RAPP 10-K & 10-Q changes, risk factors and insider trading
Rapport Therapeutics, Inc. · Nasdaq · Pharmaceutical Preparations · CIK 2012593 · All filings on SEC.gov
At a glance
What changed in the latest 10-K
Risk Factors
Largest changes
The risk of a cyber-attack, cybersecurity incident, breach, compromise, or other interruption has generally increased as the number, intensity and sophistication of attempted attacks and intrusions from around the world have increased. Such risks come from a variety of evolving threats, including but not limited to,see in full comparisonsocial-engineeringsocial engineering attacks (including through deep fakes, which may be increasinglymoredifficult to identify as fake, and phishing attacks), malicious code (such as viruses and worms), malware (including as a result of advanced persistent threat intrusions), denial-of-service attacks, credential stuffing, credential harvesting, personnel misconduct or error, ransomware attacks, supply-chain attacks, software bugs, server malfunctions, software or hardware failures, loss of data or other information technology assets, adware, attacks enhanced or facilitated by artificial intelligence (“AI”), telecommunications failures, earthquakes, fires, floods, and other similar threats. Use of generative AI models in our internal or third-party systems may create new attack surfaces or methods for adversaries, which could impact us and our vendors. Any cybersecurity incidents or data breaches related to our use of AI applications could adversely affect our reputation and results of operations.
Likewise, such legislative developments in thesee in full comparisonU.S.,UnitedseveralStatesstates,have introduced emerging compliance obligations for companies that develop or deploy AI technologies, which will impose novel risks on AI developers and users. Recently, states have advanced, and in some cases passed, dozens of laws focused on AI governance and regulation, includingColorado and California, passed laws that will take effect in 2026, to regulate various uses of artificial intelligence, including to make consequential decisions. In addition, various federal regulators have issued guidance and focused enforcement effortsonthe usedeployment of AI inregulatedhealthcaresectors.settings.TheTheseU.S.state-levelFoodinitiatives reflect a growing trend toward AI regulation in the absence of federal legislation. As a result, we may face a fragmented andDrugevolvingAdministration,complianceforlandscapeexample,thatissuedcouldguidanceincreaseonoperationalthecomplexity, regulatory scrutiny, and legal exposure associated with our use or development ofartificialAIintelligence in medical devices, requiring detailed risk management and review processes to obtain approvals.technologies. If we develop or use AI systems governed by these laws or regulations, we will need to meet higher standards of data quality, transparency, monitoring and human oversight, and we would need to adhere to specific and potentially burdensome and costly ethical, accountability, and administrative requirements, with the potential for significant enforcement or litigation in the event of any perceived non-compliance. Additionally, certain privacy laws extend rights to consumers (such as the right to delete certain personal data) and regulate automated decision making, which may be incompatible with our use of AI/ML. These obligations may make it harder for us to conduct our business using AI/ML, lead to regulatory fines or penalties, require us to change our business practices, retrain our AI/ML, or prevent or limit our use of AI/ML. For example, the Federal Trade Commission has required other companies to turn over (or disgorge) valuable insights or trainings generated through the use of AI/ML where they allege the company has violated privacy and consumer protection laws. If we cannot use AI/ML or that use is restricted, our business may be less efficient, or we may be at a competitive disadvantage.
The financial markets and the global economy may also be adversely affected by the current or anticipated impact of political uncertainty, including military conflicts, such as the ongoing conflicts between Russia and Ukraine, and Israel and Hamas, terrorism, or other geopolitical events. Sanctions imposed by the U.S. and other countries in response to such conflicts, including the one in Ukraine, may also continue to adversely impact the financial markets and the global economy, and any economic countermeasures by the affected countries or others could exacerbate market and economic instability. Additionally, recent changessee in full comparisonto policy implemented by thein U.S.Congress, the Trump administration or any new administrationpolicy have impacted and may in the future impact, among other things, the U.S. and global economy, international trade relations, unemployment, immigration, healthcare, taxation, the U.S. regulatory environment, inflation and other areas. For example,duringthepriorimplementationTrump administration, increasedof tariffswere implemented on goods imported into the U.S., particularly from China, Canada, and Mexico. On February 1, 2025,by the U.S.imposedgovernmenta 25% tariff on imports from Canada and Mexico, which were subsequently suspended for a period of one month, and a 10% additional tariff on imports from China. Historically, tariffs havehas led to increased trade disruptions and political tensions, between not only the U.S. and China, but also between the U.S. and other countries in the international community. In response to tariffs, other countries have implemented and may in the future implement additional retaliatory tariffs on U.S. goods. Political tensions as a result of trade policies could reduce trade volume, investment, technological exchange and other economic activities between major international economies, resulting in a material adverse effect on global economic conditions and the stability of global financial markets. There has also been proposed U.S. legislation that may restrict the ability of U.S. biotechnology companies to purchase services or products from, or otherwise collaborate with, certain Chinese biotechnology companies of concern without losing the ability to contract with, or otherwise receive funding from, the U.S. government. We continue to assess the legislation as it develops to determine whether it could have an effect on our contractual relationships.AnyAlthough we have taken steps to mitigate the impact of tariffs and trade restrictions on our operations, any changes in political, trade,regulatory,regulatory and economic conditions, including U.S. trade policies, could have a material adverse effect on our business, financial conditionorand results of operations. Until we know what policy changes are made, whether those policy changes are challenged and subsequently upheld by the court system and how those changes impact our business and the business of our competitors over the long term, we will not know if, overall, we will benefit from them or be negatively affected by them.
“For example, we will depend on Tenacia for the development and commercialization of RAP-219 in China. Under the terms of the Tenacia Agreement, Tenacia is responsible for the development and commercialization of RAP-219 in China. Tenacia will participate in the conduct of certain clinical trials for RAP-219 in Greater China and, as a result, we will depend on Tenacia for the enrollment of patients and the conduct of such clinical trials for RAP-219 in Greater China. …”see in full comparison
Regulators and legislators in the U.S. are increasingly scrutinizing and restricting certain personal data transfers and transactions involving foreign countries. For example,see in full comparisonthecertainBidenregulationsAdministration’sprohibitexecutivecertainordertransactionsPreventinginvolvingAccessbulktohumanAmericans’‘omicBulkdataSensitiveorPersonalhumanDatabiospecimensandfromUnitedwhichStateshumanGovernment-Related‘omicDatadatabycanCountriesbeof Concernderived, asimplementedwellby Department of Justice regulations issued in December 2024, prohibitsas data brokerage transactions involving certain sensitive personal data categories, including health data, genetic data, and biospecimens, to countries of concern, including China.TheThese regulations also restrict certain investment agreements, employment agreements and vendor agreements involving such data and countries of concern, absent specified cybersecuritycontrols.controls and the rule does not exempt key-coded or otherwise anonymized, pseudonymized, de-identified, or encrypted data. Actual or alleged violations ofthesesuchregulationsrules may be punishable by criminal and/or civil sanctions, and may result in exclusion from participation in federal and state programs.
Until such time, if ever, as we can generate substantial product revenue, we expect to finance our operations with our existing cash, cash equivalents and short-term investments, any future equity or debtsee in full comparisonfinancings andfinancings, upfront and milestone and royalty payments, if any, received under any futurelicenseslicenses, collaborations, orcollaborations.royalty agreements. If we raise additional capital through the sale of equity or convertible debt securities, or issue any equity or convertible debt securities in connection with a collaboration agreement or other contractual arrangement, the ownership interests of our stockholders will be diluted, and the terms of these securities may include liquidation or other preferences that adversely affect the rights of our stockholders. In addition, the possibility of such issuance may cause the market price of our common stock to decline.Debt financing, if available, may result in increased fixed payment obligations and involve agreements that include covenants limiting or restricting our ability to take specific actions, such as incurring additional debt, making capital expenditures, declaring dividends or acquiring, selling or licensing intellectual property rights or assets, which could adversely impact our ability to conduct our business.
Full comparison: every changed paragraph (78)
We are a clinical-stage biotechnology company with a limited operating history. We were formed in February 2022 and our operations to date have been limited to organizing and staffing our company, business planning, raising capital, developing our receptor associated protein (“RAP”) technology platform and technology, identifying potential product candidates, securing intellectual property rights, and planning and undertaking preclinical studies and clinical trials. Substantially all of our product candidates were initially developed by Janssen Pharmaceutical NV (“Janssen”),Janssen, which we in-licensed pursuant to the option and license agreement with Janssen (the “Janssen License”),License, entered into shortly after our formation. We have not yet demonstrated an ability to generate revenues, obtain regulatory approvals, manufacture any product on a commercial scale or arrange for a third party to do so on our behalf or conduct sales and marketing activities necessary for successful product commercialization. Our limited operating history as a company makes any assessment of our future success and viability subject to significant uncertainty. We will encounter risks and difficulties frequently experienced by early-stage biotechnology companies in rapidly evolving fields, and we have not yet demonstrated an ability to successfully overcome such risks and difficulties. If we do not address these risks and difficulties successfully, our business will suffer.
seek to identify, acquire and develop additional product candidates using our RAP technology platform,candidates, including through business development efforts to invest in or in-license other technologies or product candidates;
Additionally, our expenses could increase beyond our expectations if we are required by the U.S.FDA, FoodEMA, and Drug Administration (the “FDA”), European Medicines Agency (“EMA”), Medicines and Healthcare products Regulatory Agency (“MHRA”) or other comparable regulatory authorities to perform clinical trials in addition to those that we currently expect, or if there are any delays in establishing appropriate manufacturing arrangements for or in completing our clinical trials or the development of any of our product candidates.
Developing biotechnology products, including conducting preclinical studies and clinical trials, is a very time-consuming, expensive and uncertain process that takes years to complete. We expect our expenses to continue to increase in connection with our ongoing activities, particularly as we conduct clinical trials of, and seek regulatory and marketing approval for, our product candidates. Even if our current or future product candidates are approved for commercial sale, we anticipate incurring significant costs associated with commercializing any approved product candidate. To date, we have funded our operations principally through private financings andfinancings, our initial public offering (“IPO”) and concurrent private placement, which closed in June 2024.2024, and our underwritten public offering in September 2025. We expect our expenses to increase in connection with our ongoing activities, particularly as we continue the clinical and preclinical development of our product candidates, continue to identify additional targets using our RAP technology platform,targets, commence additional preclinical studies and clinical trials, and continue to identify and develop additional product candidates either through internal development or through acquisitions or in-licensing product candidates.
As of December 31, 2024,2025, we had $305.3$490.5 million of cash, cash equivalents and short-term investments, excluding restricted cash. Based upon our current operating plan, we believe that our existing cash, cash equivalents and short-term investments will enable us to fund our operating expenses and capital expenditure requirements throughinto the endsecond half of 2026.2029. We have based this estimate on assumptions that may prove to be wrong, and we could utilize our available capital resources sooner than we expect. We may also raise additional financing on an opportunistic basis in the future. For example, we may seek additional capital due to favorable market conditions or strategic considerations even if we believe we have sufficient funds for our current or future operating plans. Attempting to secure additional financing may divert our management from our day-to-day activities, which may adversely affect our ability to develop our product candidates. Our future capital requirements will depend on many factors, including but not limited to:
the number of preclinical studies and clinical trials required for regulatory approval of our current or future product candidates;
the costs and timing of future commercialization activities, including manufacturing, marketing, sales and distribution, for any of our product candidates for which we receive marketing approval;
Until such time, if ever, as we can generate substantial product revenue, we expect to finance our operations with our existing cash, cash equivalents and short-term investments, any future equity or debt financings andfinancings, upfront and milestone and royalty payments, if any, received under any future licenseslicenses, collaborations, or collaborations.royalty agreements. If we raise additional capital through the sale of equity or convertible debt securities, or issue any equity or convertible debt securities in connection with a collaboration agreement or other contractual arrangement, the ownership interests of our stockholders will be diluted, and the terms of these securities may include liquidation or other preferences that adversely affect the rights of our stockholders. In addition, the possibility of such issuance may cause the market price of our common stock to decline. Debt financing, if available, may result in increased fixed payment obligations and involve agreements that include covenants limiting or restricting our ability to take specific actions, such as incurring additional debt, making capital expenditures, declaring dividends or acquiring, selling or licensing intellectual property rights or assets, which could adversely impact our ability to conduct our business.
In July 2025, we filed a registration statement on Form S-3 (the “Registration Statement”) pursuant to which we may issue up to $400.0 million in shares of our common stock, preferred stock, debt securities, warrants and/or units. Concurrent with the filing of the Registration Statement, we entered into a common stock sales agreement, dated July 1, 2025, by and between the Company and Leerink Partners LLC and Cantor Fitzgerald & Co., acting as sales agents, which established an at-the-market offering program pursuant to which we may offer and sell shares of our common stock from time to time (the “ATM Program”). In connection with the ATM Program, we also filed a prospectus supplement with the SEC on January 7, 2026, for the offer and sale of up to $110.0 million of shares of common stock from time to time through the sales agents. As of the date of this Annual Report on Form 10-K, we have sold no shares pursuant to our ATM program.
Debt financing, if available, may result in increased fixed payment obligations and involve agreements that include covenants limiting or restricting our ability to take specific actions, such as incurring additional debt, making capital expenditures, declaring dividends or acquiring, selling or licensing intellectual property rights or assets, which could adversely impact our ability to conduct our business.
The obligations from our license agreement with Janssen may be a drain on our cash resources, or may cause us to incur debt obligations or equity dilution to satisfy the payment obligations.
Our business is highly dependent on the success of our product candidates, particularly RAP-219 for focal epilepsy.FOS. If we are unable to successfully complete clinical development, obtain regulatory approval for or commercialize one or more of our product candidates, or if we experience delays in doing so, our business will be materially harmed.
product-related adverse events (“AEs”) experienced by subjects in our clinical trials, including unexpected toxicity results, or by individuals using drugs or therapeutic biologics similar to our product candidates;
delays in submitting an Investigational New Drug (“IND”) application or other regulatory submission to the FDA, EMA, MHRA or other comparable regulatory authorities, or delays or failure in obtaining the necessary approvals from regulators to commence a clinical trial or a suspension or termination, or hold, of a clinical trial once commenced;
failure to receive the necessary regulatory approvals or a delay in receiving such approvals, which, among other things, may be caused by patients who fail the trial screening process, slow enrollment in clinical trials, patients dropping out of trials, patients lost to follow-up, length of time to achieve trial endpoints, additional time requirements for data analysis or New Drug Application (“NDA”) or similar foreign application preparation, discussions with the FDA, EMA, MHRA or other comparable regulatory authority an FDA, EMA, MHRA or other comparable regulatory request for additional preclinical or clinical data (such as long-term toxicology studies) or unexpected safety or manufacturing issues;
For example, in the fourth quarter of 2024, we were notified by the FDA that the IND submitted by us for the initiation of a Phase 2a2 proof-of-concept trial of RAP-219 in diabetic peripheral neuropathic pain (“DPNP”) was placed on clinical hold. The FDA requested additional information and amendments specific to the protocol design. WeFollowing arefurther workinginteractions with the FDA toin provideDecember 2025, the requestedFDA information.removed Should our response to theits clinical hold not be satisfactory toon the FDA,DPNP the clinical hold may not be lifted on a timely basis, or at all. Furthermore, in December 2023, we withdrew the development of another TARPg8 targeted molecule (RAP-482) in-licensed from Janssen that received a full clinical hold from the FDA prior to initiation of a Phase 1 trial, in order to prioritize development of our lead product candidate, RAP-219, and our other development candidates and programs.IND. Furthermore, the length of time necessary to complete clinical trials and submit an application for marketing approval for a final decision by a regulatory authority varies significantly from one product candidate to the next and from one country or jurisdiction to the next and may be difficult to predict.
In addition, if any of our product candidates receive marketing approval, we will be subject to significant regulatory obligations regarding the submission of safety and other post-marketing information and reports and registration, and will need to continue to comply (or ensure that our third-party providers comply) with current GoodcGMPs Manufacturingin Practicesmanufacturing (“cGMPs”)our products and Goodwith Clinical Practices (“GCPs”) for any clinical trials that we conduct post-approval. In addition, there is always the risk that we, a regulatory authority or a third party might identify previously unknown problems with a product post-approval, such as AEs of unanticipated severity or frequency. Compliance with these requirements is costly, and any failure to comply or other issues with our product candidates post-approval could adversely affect our business, financial condition and results of operations.
Our lead product candidate, RAP-219, is in clinical development for FOS, PGTCS and bipolar mania, and we continue to develop an LAI formulation. Additionally, we have two advanced discovery-stage nAChR programs stemming from our RAP technology platform. The first is our α6ß4 nAChR agonist development candidate, RAP-641, a genetically validated precision target that we are pursuing as a potential novel non-opiate, non-CNS approach for chronic pain and migraine, and the second comprises modulators of the α9α10 nAChR which third-party preclinical genetic data suggest could be an attractive target in treating hearing and vestibular disorders.
We have completed phase 1 clinical development of our lead product candidate, RAP-219, in multiple indications and are currently conducting a Phase 2a trial of RAP-219 in refractory focal epilepsy. We also intend to initiate additional Phase 2a trials of RAP-219 in bipolar mania and diabetic peripheral neuropathic pain. Our other product candidates and programs are at various stages of preclinical development. We seek to rapidly advance discovery and development of transformational small molecule medicines for patients suffering from central nervous system disorders.
At the state level, numerous U.S. states have enacted comprehensive privacy laws that impose certain obligations on covered businesses, many of which differ from each other in significant ways, thus complicating compliance efforts, including providing specific disclosures in privacy notices and affording individuals certain rights concerning their personal data. For example, in California, the California Consumer ProtectionPrivacy Act, or CCPA, established a comprehensive privacy framework for covered businesses by creating an expanded definition of personal information, establishing data privacy rights for consumers in the State of California, imposing special rules on the collection of consumer data from minors, and creating a new and potentially severe statutory damages framework for violations of the CCPA and for businesses that fail to implement reasonable security procedures and practices to prevent data breaches. While clinical trial data and information governed by HIPAA are currently exempt from the current version of the CCPA, other personal information may be applicable and possibleperiodic changes to the CCPA maycontinue to broaden its scope.
Similar laws have been passed and proposed in numerous states, as well as at the federal and local levels, and we expect more states to pass similar laws in the future. Such proposed legislation, if enacted, may add additional complexity, variation in requirements, restrictions and potential legal risk, require additional investment of resources in compliance programs, impact strategies and the availability of previously useful data and could result in increased compliance costs and/or changes in business practices and policies. The existence of comprehensive privacy laws in different states in the country would makemakes our compliance obligations more complex and costly and may increaseincreases the likelihood that we may be subject to enforcement actions or otherwise incur liability for noncompliance. While these states exempt some data processed in the context of clinical trials, these developments may further complicate compliance efforts, and increase legal risk and compliance costs for us and the third parties upon whom we rely.
Regulators and legislators in the U.S. are increasingly scrutinizing and restricting certain personal data transfers and transactions involving foreign countries. For example, thecertain Bidenregulations Administration’sprohibit executivecertain ordertransactions Preventinginvolving Accessbulk tohuman Americans’‘omic Bulkdata Sensitiveor Personalhuman Databiospecimens andfrom Unitedwhich Stateshuman Government-Related‘omic Datadata bycan Countriesbe of Concernderived, as implementedwell by Department of Justice regulations issued in December 2024, prohibitsas data brokerage transactions involving certain sensitive personal data categories, including health data, genetic data, and biospecimens, to countries of concern, including China. TheThese regulations also restrict certain investment agreements, employment agreements and vendor agreements involving such data and countries of concern, absent specified cybersecurity controls.controls and the rule does not exempt key-coded or otherwise anonymized, pseudonymized, de-identified, or encrypted data. Actual or alleged violations of thesesuch regulationsrules may be punishable by criminal and/or civil sanctions, and may result in exclusion from participation in federal and state programs.
Outside the United States, an increasing number of laws, regulations, and industry standards may govern privacy and data security. For example, the European Union’s General Data Protection Regulation (“EU GDPR”) and the United Kingdom’s GDPR (“UK GDPR”) impose strict requirements for processing personal data.
The EU GDPR and the UK GDPR (together, “GDPR”) establish stringent requirements regarding the processing of personal data, including strict requirements relating to processing sensitive data (such as health data), ensuring there is a legal basis or condition to justify the processing of personal data, where required obtaining consent from individuals, transparent disclosures about how personal data is to be used, limitations on retention of information, implementing safeguards to protect the security and confidentiality of personal data, where required providing notification of data breaches, maintaining records of processing activities and documenting data protection impact assessments where there is high risk processing and taking certain measures when engaging third-party processors.
Under GDPR, companies may face temporary or definitive bans on data processing and other corrective activities, fines of up to €20 million (approximately £17.5 million GBP) or 4% of annual global revenues, whichever is greater, and private litigation related to processing of personal data brought by data subjects or consumer protection organizations authorized at law to represent their interests. Non-compliance could also result in a material adverse effect on our business, financial position and results of operations.
In addition, we may be unable to transfer personal data from Europe and other jurisdictions to the United States or other countries due to data localization requirements or limitations on cross-border data flows. Europe and other jurisdictions have enacted laws requiring data to be localized or limiting the transfer of personal data to other countries. In particular, the European Economic Area (“EEA”) and the UK have restricted the transfer of personal data to the United States and other countries whosebased on whether those jurisdictions’ privacy laws are considered to provide adequate protection. Other jurisdictions may adopt similarly stringent interpretations of their data localization and cross-border data transfer laws. Although there are currently various mechanisms that may be used to transfer personal data from the EEA and UK to the United States in compliance with law, such as the EEA’s standard contractual clauses, the UK’s International Data Transfer Agreement / Addendum, and the EU-U.S. Data Privacy Framework and the UK extension thereto (which allows for transfers to relevant U.S.-based organizations who self-certify compliance and participate in the Framework), these mechanisms are subject to legal challenges, and there is no assurance that we can satisfy or rely on these measures to lawfully transfer personal data to the United States. If there is no lawful manner for us to transfer personal data from the EEA, the UK, or other jurisdictions to the United States, or if the requirements for a legally-compliant transfer are too onerous, we could face significant adverse consequences, including the interruption or degradation of our operations, the need to relocate part of or all of our business or data processing activities to other jurisdictions (such as Europe) at significant expense, increased exposure to regulatory actions, substantial fines and penalties, the inability to transfer data and work with partners, vendors and other third parties, and injunctions against our processing or transferring of personal data necessary to operate our business. Additionally, companies that transfer personal data out of the EEA and UK to other jurisdictions, particularly to the United States, are subject to increased scrutiny from regulators, individual litigants, and activist groups. Some European regulators have ordered certain companies to suspend or permanently cease certain transfers of personal data out of Europe for allegedly violating the GDPR’s cross-border data transfer limitations.
Although the UK is regarded as a third country under the EU GDPR, the European Commission has issued an adequacy decision recognizing the UK as providing adequate data protection and, therefore, transfers of personal data originating in the EEA to the UK remain unrestricted. Likewise, theThe UK government has confirmed that personal data transfers from the UK to the EEA remain free flowing. However,The therespective UK government is planning to reform its data protection law with the Data (Useprovisions and Access) Bill, which it has introduced to Parliament on 24 October 2024. These potential future changes to UK data protection laws may alter the similarities between the UK and EEA data protection regime and threaten the UK adequacy decision from the European Commission. The potentialenforcement of the EU GDPR and UK GDPR may further divergingdiverge in the future createsand create additional regulatory challenges and uncertaintiesuncertainties. for us. TheThis lack of clarity on future UK laws and regulations and their interaction with EU laws and regulations could add legal risk, uncertainty, complexity and cost to our handling of European personal data and our privacy and data security compliance programs and could require us to implement different compliance measures for the UK and the EEA.
Obligations related to privacy and data security (and consumers’ data privacy expectations) are quickly changing, becoming increasingly stringent, and creating uncertainty. Additionally, these obligations may be subject to differing applications and interpretations, which may be inconsistent or conflict among jurisdictions. Preparing for and complying with these obligations requires us to devote significant resources and may necessitate changes to our services, information technologies, systems, and practices and to those of any third parties that process personal data on our behalf. All of these evolving compliance and operational requirements impose significant costs, such as costs related to organizational changes, implementing additional protection technologies, training employees and engaging consultants and legal advisors, which are likely to increase over time. In addition, such requirements may require us to modify our data processing practices and policies, utilize management’s time and/or divert resources from other initiatives and projects.
In addition, such requirements may require us to modify our data processing practices and policies, utilize management’s time and/or divert resources from other initiatives and projects.
We may at times fail (or be perceived to have failed) in our efforts to comply with our privacy and data security obligations. Moreover, despite our efforts, our personnel or third parties on whom we rely may fail to comply with such obligations, which could negatively impact our business operations. If we or the third parties on which we rely fail, or are perceived to have failed, to address or comply with applicable privacy and data security obligations, we could face significant consequences, including but not limited to: government enforcement actions (e.g., investigations, fines, penalties, audits, inspections, and similar); litigation (including class-action claims) and mass arbitration demands; additional reporting requirements and/or oversight; bans on processing personal data; and orders to destroy or not use personal data. In particular, plaintiffs have become increasingly more active in bringing privacy-related claims against companies, including class claims and mass arbitration demands. Some of these claims allow for the recovery of statutory damages on a per violation basis, and, if viable, carry the potential for monumental statutory damages, depending on the volume of data and the number of violations. Any of these events could have a material adverse effect on our reputation, business, or financial condition, including but not limited to: loss of customers; interruptions or stoppages in our business operations (including, as relevant, clinical trials); inability to process personal data or to operate in certain jurisdictions; limited ability to develop or commercialize our products; expenditure of time and resources to defend any claim or inquiry; adverse publicity; or substantial changes to our business model or operations.
In the ordinary course of our business, we and the third parties upon which we rely,rely process sensitive data, and, as a result, we and the third parties upon which we rely face a variety of evolving threats that could cause cyber-attacks, cybersecurity incidents, breaches, compromises, or other interruptions. Although we take steps to develop and maintain systems and controls designed to protect our sensitive data, systems and infrastructure, there can be no assurance that our internal technology systems and infrastructure, or those of third parties upon which we rely, will be sufficient to protect against a cyber-attack, cybersecurity breach, compromise or other incident such as an industrial espionage attack, ransomware, or insider threat attack, which may compromise our system infrastructure or lead to the loss, destruction, alteration or dissemination of, or damage to, our sensitive data. Such threats are prevalent and continue to rise, are increasingly difficult to detect, and come from a variety of sources, including traditional computer “hackers,” threat actors, “hacktivists,” organized criminal threat actors, personnel (such as through theft or misuse), sophisticated nation states, and nation-state-supported actors.
The risk of a cyber-attack, cybersecurity incident, breach, compromise, or other interruption has generally increased as the number, intensity and sophistication of attempted attacks and intrusions from around the world have increased. Such risks come from a variety of evolving threats, including but not limited to, social-engineeringsocial engineering attacks (including through deep fakes, which may be increasingly more difficult to identify as fake, and phishing attacks), malicious code (such as viruses and worms), malware (including as a result of advanced persistent threat intrusions), denial-of-service attacks, credential stuffing, credential harvesting, personnel misconduct or error, ransomware attacks, supply-chain attacks, software bugs, server malfunctions, software or hardware failures, loss of data or other information technology assets, adware, attacks enhanced or facilitated by artificial intelligence (“AI”), telecommunications failures, earthquakes, fires, floods, and other similar threats. Use of generative AI models in our internal or third-party systems may create new attack surfaces or methods for adversaries, which could impact us and our vendors. Any cybersecurity incidents or data breaches related to our use of AI applications could adversely affect our reputation and results of operations.
Individuals engage in and are expected to continue to engage in cyber-attacks, including without limitation nation-state actors for geopolitical reasons and in conjunction with military conflicts and defense activities. During times of war and other major conflicts, we and the third parties upon which we rely,rely may be vulnerable to a heightened risk of cyber-attacks, including retaliatory cyber-attacks, that could materially disrupt our systems and operations, supply chain, and ability to produce, sell and distribute our services.
We have in the past experienced threats and security incidents related to our data and systems, and we may in the future experience additional threats, compromises, breaches or incidents. If we, or a third party upon whom we rely, experience a cyber-attack, cybersecurity incident, breach, compromise, or other interruption, or are perceived to have experienced a cyber-attack, cybersecurity incident, breach, compromise, or other interruption, we may experience adverse consequences, such as government enforcement actions (for example, investigations, fines, penalties, audits, and inspections); additional reporting requirements and/or oversight; restrictions on processing sensitive information (including personal data); litigation (including individual and group claims); indemnification obligations; negative publicity; reputational harm; monetary fund diversions; interruptions in our operations (including availability of data); financial loss; and other potentially significant harms. Our contracts may not contain limitations of liability, and even where they do, there can be no assurance that limitations of liability in our contracts are sufficient to protect us from liabilities, damages, or claims related to our privacy and data security obligations. Cyber liability insurance may not provide adequate coverage against potential liabilities related to any experienced cybersecurity incident or breach.
The use of new and evolving technologies, such as AI and machine learning (“ML”), in our operations, and the operations of third parties upon which we rely, may result in spending additional resources and present new risks and challenges that can impact our business including by posing security and other risks to our sensitive data,data. and asAs a resultresult, we may be exposed to reputational harm, other adverse consequences, and liability.
The use of new and evolving technologies, such as AI/ML, in our operations, and the operations of third parties upon which we rely presents new risks and challenges that could negatively impact our business.business, including cybersecurity, data privacy, IT, intellectual property, regulatory, legal, operational, competitive, reputational, and other risks and challenges. Specifically, risks related to bias, AI hallucinations, discrimination, harmful content, misinformation, fraud, scams, targeted attacks such as model poisoning or data poisoning, surveillance, data leakage, loss of consensus reality, inequality, environmental harms, and other harms may flow from our development, use, or deployment of AI technologies. The use of certain AI/ML technologies can also give rise to intellectual property risks, including compromises to proprietary intellectual property and intellectual property infringement. Additionally, several jurisdictions around the globe, including Europe and certain U.S. states, have proposed, enacted, or are considering, laws governing the development and use of AI/ML,ML. suchSuch aslaws in the EuropeanEU Union’s AI Act — the world’s first comprehensive AI law — which has entered into force on August 1, 2024 and most provisions of which will become effective on August 2, 2026. This legislation imposesimpose significant obligations on providers and deployers of high risk artificial intelligence systems, and encouragesencourage providers and deployers of artificial intelligence systems to account for EU ethical principles in their development and use of these systems.
Likewise, such legislative developments in the U.S.,United severalStates states,have introduced emerging compliance obligations for companies that develop or deploy AI technologies, which will impose novel risks on AI developers and users. Recently, states have advanced, and in some cases passed, dozens of laws focused on AI governance and regulation, including Colorado and California, passed laws that will take effect in 2026, to regulate various uses of artificial intelligence, including to make consequential decisions. In addition, various federal regulators have issued guidance and focused enforcement efforts on the usedeployment of AI in regulatedhealthcare sectors.settings. TheThese U.S.state-level Foodinitiatives reflect a growing trend toward AI regulation in the absence of federal legislation. As a result, we may face a fragmented and Drugevolving Administration,compliance forlandscape example,that issuedcould guidanceincrease onoperational thecomplexity, regulatory scrutiny, and legal exposure associated with our use or development of artificialAI intelligence in medical devices, requiring detailed risk management and review processes to obtain approvals.technologies. If we develop or use AI systems governed by these laws or regulations, we will need to meet higher standards of data quality, transparency, monitoring and human oversight, and we would need to adhere to specific and potentially burdensome and costly ethical, accountability, and administrative requirements, with the potential for significant enforcement or litigation in the event of any perceived non-compliance. Additionally, certain privacy laws extend rights to consumers (such as the right to delete certain personal data) and regulate automated decision making, which may be incompatible with our use of AI/ML. These obligations may make it harder for us to conduct our business using AI/ML, lead to regulatory fines or penalties, require us to change our business practices, retrain our AI/ML, or prevent or limit our use of AI/ML. For example, the Federal Trade Commission has required other companies to turn over (or disgorge) valuable insights or trainings generated through the use of AI/ML where they allege the company has violated privacy and consumer protection laws. If we cannot use AI/ML or that use is restricted, our business may be less efficient, or we may be at a competitive disadvantage.
The rapid evolution of AI/ML will require the application of significant resources to design, develop, test and maintain our products and services to help ensure that AI/ML is implemented in accordance with applicable law and regulation and in a socially responsible manner and to minimize any real or perceived unintended harmful impacts. Our vendors may in turn incorporate AI/ML tools into their own offerings, and the providers of these AI/ML tools may not meet existing or rapidly evolving regulatory or industry standards, including with respect to privacy and data security. Further, bad actors around the world use increasingly sophisticated methods, including the use of AI/ML, to engage in cyber-attacks, cyber espionage campaigns, exploitation of expanded attack surfaces, and other illegal activities involving the theft and misuse of sensitive data. Any of these effects could damage our reputation, result in the loss of valuable property and information, cause us to breach applicable laws and regulations, and adversely impact our business.
The results observed from preclinical studies or early-stage clinical trials of our product candidates may not necessarily be predictive of the results of later-stage clinical trials that we conduct. Similarly, positive results from such preclinical studies or early-stage clinical trials may not be replicated in our subsequent preclinical studies or clinical trials. For instance, results seen in preclinical animal models of epilepsy or pain may not translate to similar results in patients, and results from our Phase 2a proof-of-concept trial in refractory focal epilepsy may not translate to clinical seizures. Furthermore, our product candidates may not be able to demonstrate similar activity or adverse event profiles as other product candidates that we believe may have similar profiles.
In addition, in our planned future clinical trials, we may utilize clinical trial designs or dosing regimens that have not been tested in prior clinical trials. For instance, our Phase 2a proof-of-concept trial in refractory focal epilepsy utilizes a novel study design due to the biomarker-based primary endpoint, intracranial electroencephalography (“iEEG”) data. Specifically, iEEG data is recorded by an implanted responsive neurostimulation (“RNS”) system, which includes an electrode that monitors intracranial brain waves and detects the magnitude, duration and frequency of electrographic activity associated with clinical seizures. We are not aware of any other trials that have used iEEG data as a primary endpoint and have not engaged and do not plan to engage with the FDA on the use of this endpoint in our Phase 2a proof-of-concept trial, as this trial will not be used as a registrational trial. Accordingly, the FDA, EMA, MHRA or other comparable regulatory authorities may have questions around the interpretability of this data, and iEEG data may not be translatable to a clinical seizure endpoint in future registrational trials.
In addition, in our planned future clinical trials, we may utilize clinical trial designs or dosing regimens that have not been tested in prior clinical trials. There can also be no assurance that any of our clinical trials will ultimately be successful or support further clinical development of any of our product candidates. There is a high failure rate for drugs proceeding through clinical trials. Many companies in the pharmaceutical and biotechnology industries have suffered significant setbacks in late-stage clinical trials after achieving positive results in early-stage development, and we cannot be certain that we will not face similar setbacks. These setbacks have been caused by, among other things, preclinical findings made while clinical trials were underway or safety or efficacy observations made in preclinical studies and clinical trials, including previously unreported adverse effects or AEs.
Additionally, we mayhave and will continue to utilize an “open-label” clinical trial design.design in some of our clinical trials. An “open-label” clinical trial is one where both the patient and investigator know whether the patient is receiving the investigational product candidate or either an existing approved drug or placebo. Our Phase 2a proof-of-concept trial in refractorydrug-resistant focalFOS epilepsy iswas an open label study. Most open-label clinical trials test only the investigational product candidate and sometimes may do so at different dose levels. Open-label clinical trials are subject to various limitations that may exaggerate any therapeutic effect as patients in open-label clinical trials are aware when they are receiving treatment. Open-label clinical trials may be subject to a “patient bias” where patients perceive their symptoms to have improved merely due to their awareness of receiving an experimental treatment. In addition, open-label clinical trials may be subject to an “investigator bias” where those assessing and reviewing the physiological outcomes of the clinical trials are aware of which patients have received treatment and may interpret the information of the treated group more favorably given this knowledge. The results from an open-label trial may not be predictive of future clinical trial results of a product candidate when studied in a controlled environment with a placebo or active control.
Our failure to successfully initiate and complete clinical trials and to demonstrate the efficacy and safety necessary to obtain regulatory approval to market our product candidates would significantly harm our business. Our product candidate development costs will also increase if we experience delays in testing or regulatory approvals and we may be required to obtain additional funds to complete clinical trials. There can be no assurance that our clinical trials will begin as planned or be completed on schedule, if at all, or that we will not need to restructure or otherwise modify our trials after they have begun. For example, in the fourth quarter of 2024, we were notified by the FDA that the IND submitted by us for the initiation of a Phase 2a2 proof-of-concept trial of RAP-219 in DPNP was placed on clinical hold. The FDA requested additional information and amendments specific to the protocol design. WeFollowing arefurther workingdiscussions with the FDA toin provideDecember 2025, the requestedFDA information.removed Should our response to theits clinical hold not be satisfactory toon the FDA,DPNP the clinical hold may not be lifted on a timely basis, or at all.IND. Significant clinical trial delays also could shorten any periods during which we may have the exclusive right to commercialize our product candidates or allow our competitors to bring products to market before we do and impair our ability to successfully commercialize our product candidates, which may harm our business and results of operations. In addition, many of the factors that cause, or lead to, delays of clinical trials may ultimately lead to the denial of regulatory approval of our product candidates.
We may observe safety or tolerability issues beyond those we anticipate with our product candidates in ongoing or future RAP-219 clinical trials or in clinical programs for other product candidates. Additionally, it is possible that patients in our trials may experience greater side effects than observed in healthy volunteers. Additionally, adverse safety effects observed with AMPAR-inhibitors could adversely impact future labeling FDA may require for RAP-219. For example, perampanel’s FDA-approved label is accompanied by a black box warning for serious psychiatric and behavioral reactions, including aggression, hostility and homicidal ideation and threats. Furthermore, significant drug-drug interactions were reported for perampanel. The concomitant use with the other ASMs carbamazepine, phenytoin and oxcarbazepine decreased plasma levels of perampanel by approximately 50 to 67 percent. In addition, perampanel at a dose of 12 mg per day reduced exposure of levonorgestrel, an oral contraceptive, by approximately 40 percent. While we believe there are critical differences between perampanel and RAP-219, the FDA could impose similar warnings and labeling requirements on RAP-219, if approved, in the future.
We continue to learn more about our product candidates, and unfavorable pharmacology profiles, including extended half-lives, could lead to adverse outcomes or concerns by the FDA, EMA, MHRA or other comparable regulatory authorities.
We may observe safety or tolerability issues beyond those we anticipate with our product candidates in ongoing or future clinical trials. For example, while no Grade 3 or higher AEs have been observed to date, it is possible that such events may occur in our ongoing RAP-219 clinical program or in our clinical programs for other product candidates. Additionally, it is possible that human subjects with focal epilepsy, bipolar disorder and peripheral neuropathic pain may experience greater side effects in our clinical program for RAP-219 than observed in healthy volunteers. We continue to learn more about our product candidates, and unfavorable pharmacology profiles, including extended half-lives, could lead to adverse outcomes or concerns by the FDA, EMA, MHRA or other comparable regulatory authorities.
We have focused our research and development efforts on addressing disorders of the brain and nervous system. Efforts by pharmaceutical companies in this field have faced certain challenges in drug development. In particular, many neurological disorders,and psychiatric disorders such as focalFOS epilepsy,and bipolar disorder and peripheral neuropathic pain, rely on subjective patient-reported outcomes as key endpoints. This makes them more difficult to evaluate than indications with more objective endpoints. For example, in our Phase 2a proof-of-concept trial in refractory focal epilepsy, we are using change in clinical seizure frequency (measured by patient-recorded paper diaries) as a secondary endpoint. Furthermore, theseThese indications are often subject to a placebo effect, which may make it more challenging to isolate the effects of our product candidates. There can be no guarantee that we will successfully overcome these challenges with RAP-219, even with the use of the RNS system from NeuroPace Inc. (“NeuroPace”) for primary and secondary endpointsRAP-219 in our Phaseongoing 2aand proof-of-conceptfuture trialtrials infor refractory focal epilepsy,FOS or our bipolar mania and diabetic peripheral neuropathic pain candidates or that we will not encounter other challenges in the development of our product candidates. Moreover, given the history of clinical failures in this field, future clinical or regulatory failures by us or others may have result in further negative perception of the likelihood of success in this field, which may significantly and adversely affect the market price of our common stock.
the size of the patient population required for analysis of the trial’s primary and secondary endpoints;
Our pipeline includes product candidates for a variety of neuroscience diseases. There is no precise method of establishing the actual number of patients with any of these disorders in any geography over any time period. With respect to many of the indications in which we have developed, are developing, or plan to develop our product candidates, we have estimates of the prevalence of the disease or disorder. Our estimates as to prevalence may not be accurate, and the actual prevalence or addressable patient population for some or all of those indications, or any other indication that we elect to pursue, may be significantly smaller than our estimates. In estimating the potential prevalence of indications we are pursuing, or may in the future pursue, including our estimates as to the prevalence of focalFOS epilepsy,and bipolar disorder and peripheral neuropathic pain,disorder, we apply assumptions to available information that may not prove to be accurate. In each case, there is a range of estimates in the published literature and in marketing studies, which include estimates within the range that are lower than our estimates. The actual number of patients with these disease indications may, however, be significantly lower than we believe. Even if our prevalence estimates are correct, our product candidates may be developed for only a subset of patients with the relevant disease or disorder or our product candidates, if approved, may be indicated for or used by only a subset. In the event the number of patients with the diseases and disorders we are studying is significantly lower than we expect, we may have difficulties in enrolling patients in our clinical trials, which may delay or prevent development of our product candidates. If any of our product candidates are approved and our prevalence estimates with respect to any indication or our other market assumptions are not accurate, the markets for our product candidates for these indications may be smaller than we anticipate, which could limit our revenues and our ability to achieve profitability or to meet our expectations with respect to revenues or profits.
Our success depends upon the continued contributions of our key management and scientific personnel, many of whom have been instrumental for us and have substantial experience with developing therapies, identifying potential product candidates and building the technologies related to the clinical development of our product candidates. Given the specialized nature of brainneurological diseasesand psychiatric disorders and our approach, there is an inherent scarcity of experienced personnel in these fields. As we continue developing our product candidates in our pipeline, we will require personnel with medical, scientific, or technical qualifications specific to each program. The loss of key personnel, in particular our Chief Scientific Officer, neuropharmacologists and neuroscientists,personnel would delay our research and development activities. We currently do not have “key person” insurance on any of our employees. Despite our efforts to retain valuable employees, members of our team may terminate their employment with us on short notice. The competition for qualified personnel in the biotechnology and biopharmaceutical industries is intense, and our future success depends upon our ability to attract, retain, and motivate highly skilled scientific, technical and managerial employees. We face competition for personnel from other companies, universities, public and private research institutions, and other organizations. If our recruitment and retention efforts are unsuccessful in the future, it may be difficult for us to implement our business strategy, which would have a material adverse effect on our business.
In addition, we may rely on other third parties to collect, report and analyze data for our clinical trials. For example, our Phase 2a proof-of-concept clinical trial for RAP-219 in adult patients with refractorydrug-resistant focalFOS epilepsy reliesrelied on implantation of the RNS system from NeuroPace. NeuroPace ishas assistingand continues to assist us with clinical trial readiness, including identifying patients for enrollment in our trial, as well as services for the collection, reporting and analysis of patient data collected from the implanted RNS systems throughoutin the Phase 2a clinical trial.trial and in the open-label long term safety study. If NeuroPace does not successfully carry out its contractual obligations for any reason, meet expected deadlines, conduct our Phase 2a clinical trial or the open-label long term safety trial in accordance with applicable law, including regulatory and data privacy requirements, or encounters issues with its RNS system, including issues that raise questions of safety, effectiveness or data integrity, or we are otherwise unable to maintain our relationship with NeuroPace, we would have to redesign and conduct a new clinical trial to evaluate RAP-219 in patients with refractorydrug-resistant focal epilepsyFOS and our business, financial condition and prospects would be harmed.
Our use of third parties to manufacture our product candidates, including those located outside of the United States in jurisdictions such as China, may increase the risk that we will not have sufficient quantities of our product candidates, raw materials, active pharmaceutical ingredients (“APIs”) or drug products when needed or at an acceptable cost.
We currently rely on and engage third-party manufacturers to provide all of the API and the final drug product formulation of all of our product candidates that are being used in our clinical trials and preclinical studies. If we were to need an alternate manufacturer, we would incur added costs and delays in identifying and qualifying any such replacement. In addition, we typically order raw materials, API and drug product and services on a purchase order basis and do not enter into long-term dedicated capacity or minimum supply arrangements with any commercial manufacturer. We may not be able to timely secure needed supply arrangements on satisfactory terms, or at all. Our failure to secure these arrangements as needed could have a material adverse effect on our ability to complete the development of our product candidates or, to commercialize them, if approved. We may be unable to conclude agreements for commercial supply with third-party manufacturers or may be unable to do so on acceptable terms. There may be difficulties in scaling up to commercial quantities and formulation of our product candidates, and the costs of manufacturing could be prohibitive. Commitments that we make to supply drug product to collaboration partners, such as under our license agreement with Tenacia (the “Tenacia Agreement”), will also be sourced from our third-party manufacturers and our failure to do so within agreed specifications and timelines could result in contractual liabilities and harm to the collaborations.
We face significant competition in seeking appropriate collaborators. Whether we reach a definitive agreement for a partnership or collaboration will depend, among other things, upon our assessment of the collaborator’s resources and expertise, the terms and conditions of the proposed partnerships or collaboration and the proposed collaborator’s evaluation of a number of factors. Those factors may include the potential differentiation of our product candidate from competing product candidates, design or results of clinical trials, the likelihood of approval by the FDA, EMA, MHRA, or other comparable regulatory authorities and the regulatory pathway for any such approval, the potential market for the product candidate, the costs and complexities of manufacturing and delivering the product to patients and the potential of competing products. The collaborator may also consider alternative product candidates or technologies for similar indications that may be available for partnership or collaboration and whether such a partnership or collaboration could be more attractive than the one with us for our product candidate. If we elect to increase our expenditures to fund development or commercialization activities on our own, we may need to obtain additional capital, which may not be available to us on acceptable terms or at all. If we do not have sufficient funds, we may not be able to further develop our product candidates or bring them to market and generate product revenue.
IfWe wehave entered into and may enter into additional collaborations with third parties for the development and commercialization of our product candidates, and our prospects with respect to those product candidates will depend in significant part on the success of those collaborations.
We have entered into and may enter into additional collaborations for the development and commercialization of certain of our product candidates. If we enter intoUnder such collaborations, we will have limited control over the amount and timing of resources that our collaborators will dedicate to the development or commercialization of our product candidates. Our ability to generate revenues from these arrangements will depend on any future collaborators’ abilities to successfully perform the functions assigned to them in these arrangements. In addition, any future collaborators may have the right to abandon research or development projects and terminate applicable agreements, including funding obligations, prior to or upon the expiration of the agreed upon terms.
For example, we will depend on Tenacia for the development and commercialization of RAP-219 in China. Under the terms of the Tenacia Agreement, Tenacia is responsible for the development and commercialization of RAP-219 in China. Tenacia will participate in the conduct of certain clinical trials for RAP-219 in Greater China and, as a result, we will depend on Tenacia for the enrollment of patients and the conduct of such clinical trials for RAP-219 in Greater China. We do not control the individual efforts of Tenacia, and any failure by Tenacia to devote sufficient time and effort to the development of RAP-219, including enrolling patients and conducting clinical trials for RAP-219 in Greater China, or to meet their respective obligations to us, could adversely impact our ability to complete our planned clinical studies on our anticipated timelines. We depend on Tenacia to comply with all applicable local laws relative to the development and commercialization of RAP-219. If Tenacia violates, or is alleged to have violated, any laws or regulations during the performance of its obligations for us, it is possible that we could suffer financial and reputational harm or other negative outcomes, including possible legal consequences. In the case of any termination of the Tenacia Agreement, we may be required to devote additional efforts and to incur additional costs associated with pursuing the development and commercialization of RAP-219 in China, which could result in delays to our RAP-219 clinical program. Alternatively, we may attempt to identify and transact with a new sublicensee, but there can be no assurance that we would be able to identify a suitable sublicensee or transact on terms that are favorable to us.
Any regulatory approvals that we receive for our product candidates may be subject to limitations on the approved indicated uses for which the product may be marketed or to the conditions of approval, or contain requirements for potentially costly post-marketing testing, including Phase 4 clinical trials and surveillance to monitor the safety and efficacy of the product candidate. Certain endpoint data we hope to include in any approved product labeling also may not make it into such labeling, including exploratory or secondary endpoint data such as patient-reported outcome measures. The FDA may also require a risk evaluation and mitigation strategies (“REMS”) program as a condition of approval of our product candidates, which could entail requirements for long-term patient follow-up, a medication guide, physician communication plans or additional elements to ensure safe use, such as restricted distribution methods, patient registries and other risk minimization tools. In addition, if the FDA, EMAEMA, MHRA or other comparable regulatory authority approves our product candidates, we will have to comply with requirements including submissions of safety and other post-marketing information and reports and registration.
Additionally, under the Food and Drug Omnibus Reform Act (“FDORA”) sponsors of approved drugs and biologics must provide 6 months’ notice to the FDA of any changes in marketing status, such as the withdrawal of a drug, and failure to do so could result in the FDA placing the product on a list of discontinued products, which would revoke the product’s ability to be marketed. The FDA strictly regulates marketing, labeling, advertising and promotion of products that are placed on the market. Products may be promoted only for the approved indications and in accordance with the provisions of the approved label. The policies of the FDA, EMA and other comparable regulatory authorities may change and additional government regulations may be enacted that could prevent, limit or delay regulatory approval of our product candidates. We cannot predict the likelihood, nature or extent of government regulation that may arise from future legislation or administrative action, either in the United States or abroad. In addition, the U.S. Supreme Court’s July 2024 decision to overturn established case law giving deference to regulatory agencies’ interpretations of ambiguous statutory language has introduced uncertainty regarding the extent to which the FDA’s regulations, policies and decisions may become subject to increasing legal challenges, delays, and/or changes. If we are slow or unable to adapt to changes in existing requirements or the adoption of new requirements or policies, or if we are not able to maintain regulatory compliance, we may lose any marketing approval that we may have obtained and we may not achieve or sustain profitability.
Where possible, we plan to pursue accelerated development strategies in areas of high unmet need. We may seek an accelerated approval pathway for our one or more of our therapeutic candidates from the FDA, EMA, or other comparable regulatory authorities. Under the accelerated approval provisions in the Federal Food, Drug, and Cosmetic Act, and the FDA’s implementing regulations, the FDA may grant accelerated approval to a therapeutic candidate designed to treat a serious or life-threatening condition that provides meaningful therapeutic benefit over available therapies upon a determination that the therapeutic candidate has an effect on a surrogate endpoint or intermediate clinical endpoint that is reasonably likely to predict clinical benefit. The FDA considers a clinical benefit to be a positive therapeutic effect that is clinically meaningful in the context of a given disease, such as irreversible morbidity or mortality. For the purposes of accelerated approval, a surrogate endpoint is a marker, such as a laboratory measurement, radiographic image, physical sign, or other measure that is thought to predict clinical benefit, but is not itself a measure of clinical benefit. An intermediate clinical endpoint is a clinical endpoint that can be measured earlier than an effect on irreversible morbidity or mortality that is reasonably likely to predict an effect on irreversible morbidity or mortality or other clinical benefit. The accelerated approval pathway may be used in cases in which the advantage of a new drug over available therapy may not be a direct therapeutic advantage but is a clinically important improvement from a patient and public health perspective. If granted, accelerated approval is usually contingent on the sponsor’s agreement to conduct, in a diligent manner, additional post-approval confirmatory studies to verify and describe the drug’s clinical benefit. Under FDORA, the FDA is permitted to require, as appropriate, that a post-approval confirmatory study or studies be underway prior to approval or within a specified time period after the date of approval for a product granted accelerated approval. FDORA also gives the FDA increased authority to withdraw approval of a drug or biologic granted accelerated approval on an expedited basis if the sponsor fails to conduct such studies in a timely manner, send status updates on such studies to the FDA every 180 days to be publicly posted by the agency, or if such post-approval studies fail to verify the drug’s predicted clinical benefit. The FDA is empowered to take action, such as issuing fines, against companies that fail to conduct with due diligence any post-approval confirmatory study or submit timely reports to the agency on their progress.
Management's Discussion & Analysis (MD&A)
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“We are currently conducting a Phase 2a proof-of-concept trial in adult patients with refractory focal epilepsy, for which we expect to report topline results in the third quarter of 2025. We believe RAP-219 also has therapeutic potential in bipolar disorder and peripheral neuropathic pain, and we intend to initiate a Phase 2a proof-of-concept trial in bipolar mania in the third quarter of 2025 with topline results expected in the first half of 2027. We were notified in the fourth quarter of 2024 by the U.S. …”see in full comparison
“In July 2025, we entered into a common stock sales agreement, by and between the Company and Leerink Partners LLC and Cantor Fitzgerald & Co., acting as sales agents, which established an at-the-market offering program pursuant to which we may offer and sell shares of our common stock from time to time (the “ATM Program”). In connection with the ATM Program, we filed a prospectus supplement with the SEC on January 7, 2026, for the offer and sale of up to $110.0 million of shares of common stock from time to time through the sales agents. …”see in full comparison
“The NeuroPace Agreement expires on the later of three years from the effective date or the completion of all services under all statement of work agreements entered into prior to the third anniversary of the effective date. …”see in full comparison
“As of December 31, 2023, the fair value of each Series B convertible preferred stock was $1.68 per share. The risk-free interest rate is determined by reference to the U.S. Treasury yield curve for time periods approximately equal to the remaining estimated time period of achievement of the specified milestones underlying the preferred stock tranche right. …”see in full comparison
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We are a clinical-stage biotechnology company dedicated to discoveringthe discovery and developingdevelopment of small molecule precision medicines for patients with neurological or psychiatric disorders. Our foundational science has elucidated complexities of neuronal receptor biology and enables us to map and target certain neuronal receptor complexes. Neuronal receptors are complex assemblies of proteins, comprising receptor principal subunits and their receptor associated proteins (“RAPs”), the latter of which play crucial roles in regulating receptor expression and function. We believe that our deep expertise in RAP biology provides an opportunity for us to interrogate previously inaccessible targets and develop neurological and psychiatric drugs that are specific for receptor variants and neuroanatomical regions associated with certain diseases. Most neuroactive drugs lack this specificity, often resulting in undesired and intolerable side effects. Leveraging our expertise, we are developing a portfolio of precision product candidates that we believe has the potential to transform the standard of care of many neurological and psychiatric disorders.
Our founders have made pioneering discoveries related to the function of RAPs in the brain. Their findings form the basis of our RAP technology platform, which enables a differentiated approach to generate precision small molecule product candidates with the potential to overcome many limitations of conventional neurology drug discovery. RAP-219, our most advanced product candidate, is an AMPA receptor (“AMPAR”) negative allosteric modulator (“NAM”). RAP-219 is designed to achieve neuroanatomical specificity through its selective targeting of a RAP known as TARP8, which is associated with the neuronal AMPARs. Whereas AMPARs are distributed widely in the central nervous system (“CNS”), TARP8 is expressed only in discrete regions, including the hippocampusneocortex and neocortex,mesial temporal lobe, where focal onset seizures (“FOS”) often originate. By contrast, TARP8 has minimal expression in the hindbrain, where drug effects are often associated with adverse events. As such, we believe RAP-219 has the potential for a differentiated profile as compared to traditional neuroscience medications. Due to the role of AMPA biology in various neurological disorders and our precision approach of selectively targeting TARP8, we believe RAP-219 has pipeline-in-a-product potential and we are evaluating it as a potentially transformational treatment for patients with focalFOS, epilepsy,primary generalized tonic-clonic seizures (“PGTCS”), and bipolar disorder, and peripheral neuropathic pain.mania.
A total of fourSeveral Phase 1 trials in RAP-219 have been conducted to date in healthy adult volunteers:volunteers, including a single ascending dose (“SAD”) trial; a multiple ascending dose (“MAD”) trial; a second MAD trial (“MAD-2”), to assess alternative dosing regimens that may accelerate time to reach therapeutic exposure; and a human positron emission tomography (“PET”) trial, which utilized a companion PET radiotracer to confirm brain target receptor occupancy (“RO”) and brain region specificity across a range of dosing and exposure levels. In January 2025, we announced results from our PET and MAD-2 trials of RAP-219. Data demonstrated that neuroanatomical specificity can be achieved through RAP-219’s selective targeting of TARPγ8. In Cohort 1 of the human PET trial, which used the dosing regimen utilized in our ongoing Phase 2a trial in patients with refractory focal epilepsy, RAP-219 achieved target receptor occupancy associated with maximal seizure protection in preclinical models within five days and was generally well tolerated, which we believe further supports the use of such dosing regimen in the Phase 2a trial in patients with refractory focal epilepsy.
In September 2025, we announced positive topline results from our Phase 2a proof-of-concept trial of RAP-219 in adult patients with drug-resistant FOS. The trial met its primary and secondary endpoints. The trial demonstrated a statistically significant reduction in long episodes—an objective electrographic biomarker for clinical seizure reduction—compared with baseline over the 8-week treatment period. In the trial, RAP-219 also demonstrated a statistically significant and clinically meaningful reduction in clinical seizures compared with baseline. RAP-219 was generally well tolerated. In December 2025, we presented post-hoc analysis from the Phase 2a proof-of-concept trial showing treatment with RAP-219 had consistent effects in the first and second four-week segments of the treatment period. This demonstrates that there was a rapid onset of efficacy, and a consistent reduction in long episodes, and a consistent, clinically meaningful reduction in clinical seizures throughout the 8-week treatment period. We expect to present 8-week follow-up results in the second quarter of 2026. In late 2025, we initiated an open-label long term safety trial to allow patients enrolled in our Phase 2a proof-of-concept FOS trial to resume taking RAP-219. Data from the open-label trial are expected in the second half of 2026. In December 2025, we received U.S. Food and Drug Administration (“FDA”) feedback from an end-of-phase 2 meeting supporting advancement into two Phase 3 trials of RAP-219 in patients with drug-resistant FOS and expect to initiate the Phase 3 program in FOS in the second quarter of 2026.
We are also expanding our epilepsy portfolio into PGTCS, the most common type of generalized seizure and an important next step in addressing unmet need in patients with seizure disorders. With proof-of-concept established in FOS, we plan to initiate a Phase 3 trial in PGTCS in the first half of 2027.
We believe RAP-219 also has therapeutic potential in bipolar disorder. Our Phase 2 proof-of-concept trial in bipolar mania is currently enrolling patients, with topline results expected in the first half of 2027.
Additionally, we continue to develop a long-acting injectable (“LAI”) formulation of RAP-219. We believe an LAI formulation has the potential to improve patient adherence and expand the potential clinical utility across all of the RAP-219 indications. We have initiated IND-enabling activities to support a Phase 1 clinical study in healthy volunteers, with initial pharmacokinetics (“PK”) results expected in 2027.
We are currently conducting a Phase 2a proof-of-concept trial in adult patients with refractory focal epilepsy, for which we expect to report topline results in the third quarter of 2025. We believe RAP-219 also has therapeutic potential in bipolar disorder and peripheral neuropathic pain, and we intend to initiate a Phase 2a proof-of-concept trial in bipolar mania in the third quarter of 2025 with topline results expected in the first half of 2027. We were notified in the fourth quarter of 2024 by the U.S. Food and Drug Administration (“FDA”) that the Investigational New Drug (“IND”) submitted for the initiation of a Phase 2a proof-of-concept trial of RAP-219 for the treatment of diabetic peripheral neuropathic pain (“DPNP”) was placed on clinical hold. The FDA requested additional information and amendments specific to the protocol design. The clinical hold is specific to the IND for DPNP and has not impacted our ongoing Phase 2a trial in refractory focal epilepsy or planned proof-of-concept trial in bipolar mania. We believe in our ability to advance the clinical development of RAP-219 for DPNP and will provide an update on the anticipated timing of the Phase 2a trial initiation once available.
BeyondWe TARP8, wealso have two advanced discovery-stage nicotinic acetylcholine receptor (“nAChR”) programs stemming from our RAP technology platform. nAChRs have been clinically validated in patient-reported neuropathic pain and our first advanced discovery-stage nAChR program comprises agonists of the α6ß4 nAChR. α6ß4 nAChRs are selectively expressed in sensory neurons, while the α6 subunit has human genetic validation in chronic pain. We have initiated IND-enabling activities for our α6ß4 nAChR agonist development candidate, RAP-641, a genetically validated precision target that we are pursuing as a potential novel non-opiate, non-CNS approach for chronic pain and migraine. The firstsecond advanced discovery-stage nAChR program comprises modulators of 6 nAChRs that we are developing in chronic pain; and the second comprises modulators of α9α10 nAChRs that we are developing in hearing disorders.nAChR. Third-party preclinical genetic data suggest that thesethis nAChR subtypes could be an attractive drugtarget targetsin fortreating thesehearing diseases.and vestibular disorders. We continue to leverage our RAP technology platform to discover additional product candidates that we believe have the potential to provide a transformative benefitbenefits for large patient populations with neurological or psychiatric diseases with unmet needs.diseases.
Since our inception in February 2022, we have not generated any revenue from product sales or other sources and have incurred significant operating losses and negative cash flows from our operations. We have devoted substantially all of our efforts to organizing and staffing our company, business planning, research and development activities, building our intellectual property portfolio, and providing general and administrative support for these operations. In June 2024, we completed our initial public offering (“IPO”), pursuant to which we issued and sold 9,200,000 shares of common stock (inclusive of 1,200,000 shares of common stock sold pursuant to the underwriters’ exercise of their option to purchase additional shares). In addition, we issued and sold 1,058,824 shares of common stock to certain institutional accredited investors in a concurrent private placement. The aggregate net proceeds received by us from the IPO and concurrent private placement were $157.6 million, after deducting underwriting discounts and commissions, placement agent fees as well as other offering and private placement costs of $16.8 million. To date, we have funded our operations primarily with proceeds from the issuance and sale of our common stock, convertible notes and convertible preferred stock. As of December 31, 2024, we had raised aggregate gross proceeds of $424.4 million from these financings, and had cash, cash equivalents and short-term investments of $305.3 million, excluding our restricted cash.
In addition, we have several preclinical and clinical development, regulatory, and commercial milestone payment obligations under our licensing arrangements. Our net losses may fluctuate significantly from quarter-to-quarter and year-to-year, depending on the timing of our preclinical studies and plannedstudies, clinical trials and our expenditures on other research and development activities.
We believe that our existing cash and cash equivalents, and short-term investments will enable us to fund our operating expenses and capital expenditure requirements throughinto the endsecond half of 2026.2029. See the sections titled “—Liquidity and Capital Resources” and “Risk Factors—Risks Related to Our Limited Operating History, Financial Condition and Need for Additional Capital” included elsewhere in this Annual Report.
In November 2023, we entered into a master services agreement (the “NeuroPace Agreement”) with NeuroPace Inc. (“NeuroPace”), the manufacturer and distributor of the responsive neurostimulation (“RNS”) system. Pursuant to the NeuroPace Agreement and in accordance with statement of work agreements entered into from time to time, NeuroPace provides us with certain services with respect to data from the RNS systems used in our clinical trials. The NeuroPace Agreement also grants us a royalty-free, worldwide, exclusive, non-transferable license to all data collected by the RNS systems in our Phase 2aRAP-219 clinical trialtrials in drug-resistant FOS and the outcomes of algorithms that are applied to such data, as well as the ability to publish the outcomes of algorithms, subject to certain conditions. The consideration we will pay to NeuroPace for such services is set out in each statement of work agreement.
The NeuroPace Agreement contains an exclusivity provision providing that, at any time while providing services under the NeuroPace Agreement and for a period after the final clinical study report, NeuroPace may not perform any services that are the same as the services covered by the NeuroPace Agreement to any business that directly competes with us, subject to the specific terms of the NeuroPace Agreement. The NeuroPace Agreement also contains standard representations and warranties, confidentiality and intellectual property protective provisions and indemnification terms.
The NeuroPace Agreement expires on the later of three years from the effective date or the completion of all services under all statement of work agreements entered into prior to the third anniversary of the effective date. Either party may terminate the NeuroPace Agreement or any statement of work agreement (i) without cause by giving written notice to the other party within a specified period of time, (ii) by giving written notice upon a curable material breach that is not remediated within a specified period of time, or (iii) immediately upon written notice in the event of a material breach that cannot be cured.
Concurrently with the execution of the NeuroPace Agreement, the parties also entered into an initial statement of work, as amended in March 20242024, and a second SOW in July 2025 (collectively, the “NeuroPace SOWSOWs”), under the NeuroPace Agreement, pursuant to which NeuroPace agreed to provide services related to our RAP-219 Phase 2a clinical trial ofand RAP-219,planned open-label long term safety trial in RAP-219 for FOS, including, among other things, clinical trial readiness support, identification of potential patients satisfying the enrollment criteria and RNS system data reporting and data analysis. Pursuant to the payment schedule set out in the NeuroPace SOW,SOWs, wethe Company will pay NeuroPace an aggregate of up to $3.7$5.3 million over a period of approximately twofour years in connection with NeuroPace’s provision of services and achievement of certain patient enrollment and deliverable milestones. During the year ended December 31, 2024, we paid NeuroPace $0.6 million and recognized $1.8 million in research and development expense for services performed, resulting in a prepaid expense balance of $0.3 million as of December 31, 2024. The Company has incurred cumulative expenses of $4.2 million on both NeuroPace SOWs through December 31, 2025.
During the year ended December 31, 2024, we paid NeuroPace $0.6 million and recognized $1.8 million in research and development expense for services performed, resulting in a prepaid expense balance of $0.3 million as of December 31, 2024. During the year ended December 31, 2023, we paid NeuroPace $1.5 million, which was recorded as prepaid expenses and other current assets in the consolidated balance sheet as of December 31, 2023.
Interest income consists of interest earned from our cash, cash equivalents and short-term and long-term investments.
Our Series A and Series B convertible preferred stock purchase agreements provided the investors the obligation to participate in subsequent offerings of Series A and Series B convertible preferred stock upon achievement of certain specified milestones, upon the waiver of such milestone achievement by a majority vote of the respective series convertible preferred stockholders, or with respect to the Series B convertible preferred stock, upon exercise of the stockholders right to early exercise the preferred stock tranche right. The preferred stock tranche rights are classified as liabilities and initially recorded at fair value upon the issuance date of the rights. The liabilities were subsequently remeasured to fair value at each reporting date and immediately prior to being settled, and changes in fair value of the preferred stock tranche right liabilities were recognized as a component of other income (expense), net in our consolidated statements of operations and comprehensive loss. In February 2023, we closed the Series A second and third financings, resulting in full settlement of the tranche right, upon both of which we issued additional shares of Series A convertible preferred stock. Immediately prior to the issuance of such shares, the preferred stock tranche right liability was remeasured to fair value with the change in fair value recognized as a component of other income (expense), net. As a result of the Series A preferred stock tranche right settlement in February 2023, we will no longer recognize changes in the fair value of the Series A preferred stock tranche liability in ourcondensed consolidated statements of operations and comprehensive loss. In March 2024, we closed the Series B second financing, resulting in full settlement of the tranche right, upon which we issued additional shares of Series B convertible preferred stock. Immediately prior to the issuance of such shares, the preferred stock tranche right liability was remeasured to fair value with the change in fair value recognized as a component of other income (expense), net. As a result of the Series B preferred stock tranche right settlement in March 2024, we will no longer recognize changes in the fair value of the Series B preferred stock tranche liability in our condensed consolidated statements of operations and comprehensive loss.
For each of the years ended December 31, 20242025 and 2023,2024, we recorded an income tax provision of zero and $10 thousand, respectively.zero. As of December 31, 20242025 and 2023,2024, we recorded a full valuation allowance of our net deferred tax assets, as we believed it was more likely than not we would not be able to utilizerealize our deferred tax assets prior to their expiration. The income tax provision reflects the changes to tax law that was enacted during 2025.
On July 4, 2025, new U.S tax legislation was signed into law (known as the “One Big Beautiful Bill Act” or “OBBBA”) which makes permanent many of the tax provisions enacted in 2017 as part of the Tax Cuts and Jobs Act that were set to expire at the end of 2025. In addition, the OBBBA makes changes to certain U.S. corporate tax provisions, but many are generally not effective until 2026. The impacts of OBBBA are not material to the 2025 consolidated balance sheet or statements of operations and comprehensive loss.
For the year ended December 31, 2025, we adopted ASU 2023-09, Income Taxes (Topic 740): Improvements to Income Tax Disclosures (“ASU 2023-09”) on a prospective basis. The amendments require disclosure of specific categories in the rate reconciliation and provide additional information for reconciling items that meet a quantitative threshold. This standard also requires further disaggregation of income taxes paid by federal, state, and foreign taxes, and by individual jurisdictions exceeding a specific threshold. Refer to Note 11—“Income Taxes” to our annual consolidated financial statements for further information.
As part of the Protecting Americans from Tax Hikes Act of 2015 (the “PATH Act”), certain eligible companies have the ability to convert a portion of their R&D tax credits to offset payroll tax liabilities. As of December 31, 2025, the Company had converted $0.5 million of its federal R&D credits to be utilized as an offset against future payroll taxes.
ana $11.9$19.1 million increase in RAP-219 program costs, which consisted primarily of an increase of $7.4$17.0 million in clinical trial costscosts, primarily driven by costs for our Phase 2a trialtrial, inopen-label refractorylong focalterm epilepsy,safety trial, start-up costs for our Phase 2a3 trials in drug-resistant FOS, and costs for our Phase 2 proof-of-concept trial in DPNP,bipolar costs from our MAD-2 trial, costs from our PET trial, and costs from our human absorption, distribution, metabolism, and excretion study,mania, a $1.7 million increase in preclinical toxicology studies driven by initiation of long-term toxicology work, a $2.4$2.0 million increase in contract manufacturing costs related to the production of materialsclinical totrial support our additional Phase 1supply, and 2a trials,$0.8 and anmillion increase ofin $0.2RAP-219 consulting costs. These increases were offset by a $0.7 million fordecrease consultingin preclinical toxicology studies related to thelong-term DPNPtoxicology programwork;
a $10.2 million increase in preclinical program costs, which consisted primarily of a $6.0 million increase in toxicology and animal studies related to our discovery programs, a $1.9 million increase in external chemistry efforts related to our discovery programs, a $1.0 million increase in contract manufacturing costs related to the production of materials for use in our preclinical studies, a $1.2 million increase in lab supply costs due to increased preclinical activities, and a $0.2 million increase in discovery program consulting costs;
a $9.6$10.9 million increase in personnel-related costs due to an increase in headcount, which consisted primarily of salaries, bonuses, and other compensation-related costs of $7.7 million, and stock-based compensation of $2.2 million. These increases were partially offset by a decrease in general research and development consulting costs of $0.3$3.2 million; and a $1.3$3.9 million increase in other costs, consisting primarily of research and development facilitiesfacilities, expensesinformation technology, and depreciation expenseexpenses related to openingincreased headcount and continued expansion of our Boston office in September 2023, and continuing to expand our San Diego site.sites.
$9.7a $7.5 million increase in workforce expense primarily due to an increase in headcount, consisting of salaries, bonuses, and other compensation-related costs of $5.1$2.2 million and stock-based compensation of $4.6$5.4 million;
a $0.9 million increase in facility and other expenses, consisting of administrative expenses due to increased business activities and expanded general and administrative support in connection with operating as a public company; partially offset by a $0.2 million decrease in professional and consulting fees, as higher costs were incurred in 2024 due to our IPO process completed in June 2024.
$2.3 million increase in professional and consulting fees, related to expanding our administrative support to satisfy the requirements of operating as a public company along with the general growth of the Company, including outsourced legal and accounting expenses; and $1.9 million increase in other expenses, consisting primarily of administrative expenses due to increased business activities, and expanded general and administrative support in connection with operating as a public company.
Other income (expense) was $13.6 million for the year ended December 31, 2025, as compared to $4.7 million for the year ended December 31, 2024. The $7.4 million decrease in the expense from the change in fair value of preferred stock tranche right liability was due to the settlement of the Series B preferred stock tranche right liability in March 2024. The $1.5 million increase in interest income is due to the increased cash, cash equivalent and short-term investments balances from the IPO and concurrent private placement in June 2024 and the September 2025 Offering.
Interest Income
Interest income was $12.1 million for the year ended December 31, 2024, as compared to $2.5 million for the year ended December 31, 2023. The increase of $9.6 million is primarily due to opening additional interest-bearing accounts in December 2023, in addition to the increased cash, cash equivalent and short-term investments balances from the Series B convertible preferred stock financing in August 2023 and February 2024, and our initial public offering (“IPO”) in June 2024.
Change in Fair Value of Preferred Stock Tranche Right Liability
The change in fair value of the preferred stock tranche right liability expense was $7.4 million for the year ended December 31, 2024, as compared to $1.1 million for the year ended December 31, 2023 . The change in fair value of preferred stock tranche right liabilities for the year ended December 31, 2024 consisted of an increase in the fair value of the Series B preferred stock tranche right liability of $7.4 million. In conjunction with the waiver of the second tranche milestone in February 2024 and the settlement of the Series B tranche right in March 2024, the Series B tranche right liability was remeasured immediately prior to the waiver, resulting in a $7.4 million increase in fair value. The change in fair value of preferred stock tranche right liability for the year ended December 31, 2023 consisted of an increase in the fair value of the Series A preferred stock tranche right liability of $1.0 million as a result of the waiver of the second and third milestones and settlement of the Series A tranche right liability.
For the years ended December 31, 20242025 and 20232024 we recorded an income tax provision of zero and $10 thousand, respectively.zero.
Since our inception in February 2022, we have not generated any revenue from any sources and have incurred significant operating losses and negative cash flows from operations. We expect to incur significant expenses and operating losses for the foreseeable future as we advance the clinical development of our product candidates and pipeline. Further, we expect to continue to incur additional costs associated with operating as a public company. To date,Historically, we have funded our operations with proceeds from the sale of convertible notes, convertible preferred stock, and the issuance of common stock in our IPO and concurrent private placement.placement, and the September 2025 Offering. Through December 31, 2024,2025, we received aggregate gross proceeds of $424.4$711.9 million from the issuance of convertible promissory notes and the sale of our convertible preferred stock and common stock. As of December 31, 2024,2025, we had cash and cash equivalents of $56.8$52.6 million and short-term investments of $248.5$437.9 million.
In July 2025, we entered into a common stock sales agreement, by and between the Company and Leerink Partners LLC and Cantor Fitzgerald & Co., acting as sales agents, which established an at-the-market offering program pursuant to which we may offer and sell shares of our common stock from time to time (the “ATM Program”). In connection with the ATM Program, we filed a prospectus supplement with the SEC on January 7, 2026, for the offer and sale of up to $110.0 million of shares of common stock from time to time through the sales agents. To date, no shares have been sold through the ATM Program. As market conditions permit, we may offer and sell securities under the Registration Statement, including through the ATM Program, in order to fund our operations or provide additional liquidity.
During the year ended December 31, 2025 operating activities used $87.5 million of cash, resulting primarily from our net loss of $111.5 million and non-cash accretion of investments in marketable securities of $1.4 million, partially offset by $18.9 million of non-cash stock-based compensation expense, changes in operating assets and liabilities of $3.5 million, $1.0 million of non-cash depreciation expense, and $2.0 million of non-cash lease expense. The $3.5 million change in operating assets and liabilities is primarily driven by an increase in prepaid expenses and other current assets of $3.6 million, primarily due to advanced payments on new contracts related to our clinical trials, an increase in accrued expenses of $6.2 million and an increase in accounts payable of $2.2 million and due to increased expenditures and timing of payments to vendors, an increase of $0.8 million in other assets due to the payment of the security deposit for our new corporate headquarters lease and an up-front long-term clinical trial payment, and $0.5 million decrease in operating lease liabilities.
During the year ended December 31, 2023, operating activities used $27.2 million of cash, resulting primarily from our net loss of $34.8 million, partially offset by $3.5 million of non-cash stock-based compensation expense and non-cash change in fair value of preferred stock tranche right liability of $1.1 million, and net cash provided by changes in operating assets and liabilities of $2.7 million. Net cash provided by changes in operating assets and liabilities consisted primarily of increases in accrued expenses and other current liabilities of $5.4 million, partially offset by increases in prepaid expenses and other current assets of $3.2 million. The increases in accrued expenses and prepaid expenses were primarily due to increased internal and external costs associated with our research and development activities, including clinical trials and manufacturing.
During the year ended December 31, 2025, net cash used in investing activities was $187.5 million, primarily consisting of purchases of short-term investments of $354.8 million and purchases of property and equipment of $0.6 million, partially offset by maturities of short-term investments of $167.9 million.
During the year ended December 31, 2023, net cash used in investing activities was $78.9 million, primarily consisting of purchases of short-term investments of $77.2 million and purchases of property and equipment of $1.6 million.
During the year ended December 31, 2025, net cash provided by financing activities was $270.8 million, primarily consisting of net proceeds from the September 2025 Offering in the aggregate amount of $269.4 million and proceeds from exercise of stock options of $1.3 million.
During the year ended December 31, 2023, net cash provided by financing activities was $145.1 million, primarily consisting of net proceeds of $59.9 million from our additional issuance of Series A convertible preferred stock, and net proceeds of $85.3 million from our initial issuance of Series B convertible preferred stock, including tranche rights.
As of December 31, 2024,2025, we had cash, cash equivalents and short-term investments of $305.3$490.5 million, excluding our restricted cash. As of the issuance date of the consolidated financial statements for the year ended December 31, 2024,2025, we expect that our cash, cash equivalents and short-term investments will be sufficient to fund our operating expenses and capital expenditure requirements through at least 12 months from the issuance of the consolidated financial statements. We believe that our existing cash, cash equivalents and short-term investments will enable us to fund our operating expenses and capital expenditure requirements throughinto the endsecond half of 2026.2029. We have based this estimate on assumptions that may prove to be wrong, and we could exhaust our available capital resources sooner than we expect. However, our forecast for the period of time through which our financial resources will be adequate to support our operations is a forward-looking statement that involves risks and uncertainties, and actual results could vary materially. Additionally, the process of conducting preclinical studies and testing potential future product candidates in clinical trials is costly, and the timing of progress and expenses in these studies and trials is uncertain. We will need to raise substantial additional capital in the future.
Until such time, if ever, as we can generate substantial product revenue to support our cost structure, we expect to finance our cash needs through equity offerings, debt financings, or other capital sources, potentially including collaborations, licenses, and other similar arrangements. However, we may be unable to raise additional funds or enter into such other arrangements when needed on favorable terms or at all. To the extent that we raise additional capital through the sale of equity or convertible debt securities, your ownership interestinterests will be diluted, and the terms of these securities may include liquidation or other preferences that adversely affect the rights of our common stockholders. Additional debt financing and equity financing, if available, may involve agreements that include covenants limiting or restricting our ability to take specific actions, such as incurring additional debt, making capital expenditures, or declaring dividends. If we raise funds through collaborations, license arrangements, or other similar arrangements with third parties, we may have to relinquish valuable rights to our technologies, future revenue streams, research programs or potential future product candidates or grant licenses on terms that may not be favorable to us and/or may reduce the value of our common stock. Our failure to raise capital or enter into such other arrangements when needed could have a negative impact on our financial condition and on our ability to pursue our business plans and strategies. If we are unable to raise additional funds through equity or debt financings, or through other sources when needed, we may be required to delay, limit, reduce or terminate our product development or future commercialization efforts or grant rights to develop and market our product candidates and potential future product candidates even if we would otherwise prefer to develop and market such potential future product candidates ourselves.
As of the December 31, 2025, we had future minimum operating lease payments under non-cancelable leases of $13.4 million related to leases we have recognized on our consolidated balance sheet, which included existing laboratory and office leases and the new corporate headquarters lease that commenced June 1, 2025 in Boston, Massachusetts. The future minimum operating lease payments under these non-cancelable leases are due over a weighted average remaining lease term of 4.6 years.
As of the December 31, 2024, we had future minimum operating lease payments under non-cancelable leases of $1.8 million related to leases we have recognized on our consolidated balance sheet, which are due over the following 2.5 years. In addition, we have one lease in San Diego that has been entered into but has not yet commenced, for which we expect to pay approximately $9.6 million over the five-year lease term. We also entered into a sublease agreement for a facility in Boston, for which we expect to pay approximately $5.2 million over the six and a half-year sublease term.
In connection with the execution of the NeuroPace Agreement, the parties also entered into an initial statement of work (“SOW”), as amended in March 2024, and a second SOW in July 2025 (collectively, the “NeuroPace SOWs”) under the NeuroPace Agreement, pursuant to which NeuroPace agreed to provide services related to ourthe RAP-219 Phase 2a proof-of-concept clinical trial ofand RAP-219,planned open-label long term safety trial in RAP-219 for FOS, including, among other things, clinical trial readiness support, identification of potential patients satisfying the enrollment criteria and RNS system data analysisreporting and data reporting.analysis. Pursuant to the payment schedule set out in the statementNeuroPace ofSOWs, work,the weCompany will pay NeuroPace an aggregate of up to $3.7$5.3 million over a period of approximately twofour years in connection with NeuroPace’s provision of services and achievement of certain patient enrollment and deliverable milestones. During the year ended December 31, 2024, we paid NeuroPace $0.6 million and recognized $1.8 million in research and development expense for services performed, resulting in a prepaid expense balance of $0.3 million as of December 31, 2024. The Company has incurred cumulative expenses of $4.2 million on both NeuroPace SOWs through December 31, 2025.
During the year ended December 31, 2023, we paid NeuroPace $1.5 million, which was recorded as prepaid expenses and other current assets in the consolidated balance sheet as of December 31, 2023. During the year ended December 31, 2024, we paid NeuroPace $0.6 million and recognized $1.8 million in research and development expense for services performed, resulting in a prepaid expense balance of $0.3 million as of December 31, 2024.
the likelihood of achieving a liquidity event, such as an initial public offering (“IPO”) or sale of our company in light of prevailing market conditions; and the analysis of IPOs and the market performance of similar companies in the biotechnology industry.
Our Series A and Series B convertible preferred stock purchase agreements obligated the Series A and Series B investors to participate in a subsequent offering of Series A and Series B convertible preferred stock upon certain conditions being met, which we referred to as the preferred stock tranche rights. We determined that the preferred stock tranche rights were required to be recorded as liabilitiesa liability because theyit werewas a freestanding financial instrumentsinstrument that would require us to transfer assets upon exercises of the right. The preferred stock tranche rights met the definition of a freestanding financial instrument because they were legally detachable and separately exercisable from the Series A and Series B convertible preferred stock. The preferred stock tranche rights were classified as a liability and initially recorded at fair value upon the issuance date of the right. The liabilitiesliability werewas remeasured to fair value at each reporting date until settled, and changes in the fair value of the preferred stock tranche right liabilitiesliability werewas recognized as a component of other income (expense) in our consolidated statements of operations and comprehensive loss.
In February 2023, in conjunction with the amendment to the Series A convertible preferred stock purchase agreement, our existing Series A convertible preferred stockholders voted to waive the second and third tranche milestones and exercised their tranche right. As a result, an aggregate of 50,000,000 shares of Series A convertible preferred stock were issued and sold at a price of $1.00 per share, resulting in total cash proceeds of $50 million, less $61 thousand of issuance costs. As a result of this issuance, the Series A preferred stock tranche right liability, with a then fair value of $11.5 million immediately prior to the amendment and waiver, was settled in full and recognized in additional paid-in capital.
Subsequent to the original issuance, one stockholder exercised its right to early exercise the Series B preferred stock tranche right and purchased 4,769,655 shares of Series B convertible preferred stock for cash proceeds of $8.0 million. The fair value of the associated tranche right liability that was settled at the time of the sale of $0.5 million was recognized in additional paid-in capital. Additionally, the investor paid a premium of $0.8 million for these shares over their fair value which was also recorded in additional paid-in capital.
The fair value of the tranche right liabilitiesliability was determined based on significant inputs not observable in the market, which represented a Level 3 measurement within the fair value hierarchy. The fair value of the tranche right liabilitiesliability was determined using a Contingent Forward Analysis, which is a scenario-based lattice model that accounts for the different possible milestone scenarios and their associated probabilities, as estimated by us. The valuation model considered the probability of closing the tranche, the estimated future value of the convertible preferred stock to be issued at each closing and the investment required at each closing. Future values were converted to present value using a discount rate appropriate for probability-adjusted cash flows. The most significant assumptions in the Contingent Forward Analysis impacting the fair value of the preferred stock tranche rights were the fair value of the Series A and Series B convertible preferred stock as of each remeasurement date, the estimated remaining term of the tranche right as of each remeasurement date, and the probabilities of success for each tranche milestone as of each measurement date. We determined the fair value per share of the underlying convertible preferred stock by taking into consideration the most recent sales of our convertible preferred stock as well as additional factors that we deemed relevant. We assessed these assumptions and estimates on a quarterly basis as additional information impacting the assumptions was obtained. The risk-free rate was determined by reference to the U.S. Treasury yield curve for time periods approximately equal to the remaining estimated time to each tranche closing.
As of December 31, 2023, the fair value of each Series B convertible preferred stock was $1.68 per share. The risk-free interest rate is determined by reference to the U.S. Treasury yield curve for time periods approximately equal to the remaining estimated time period of achievement of the specified milestones underlying the preferred stock tranche right. As of December 31, 2023, an immediate 10 percent increase in the fair value of our Series B convertible preferred stock would have resulted in a $0.9 million increase, and in the case of a 10 percent decrease, a $0.9 million decrease to the fair value of the preferred stock tranche right liability.
What changed in the latest 10-Q
Risk Factors
Largest changes
“Further, bad actors around the world use increasingly sophisticated methods, including the use of AI/ML, to engage in cyber-attacks, cyber espionage campaigns, exploitation of expanded attack surfaces, and other illegal activities involving the theft and misuse of sensitive data. Technological developments in AI, including advances in frontier artificial intelligence models, may increase the sophistication and speed of cybersecurity incidents and other malicious attacks that we experience. …”see in full comparison
“Some of our manufacturers are located outside of the United States, including in China. There is currently significant uncertainty about the future relationship between the United States and various other countries, including China, with respect to trade policies, treaties, government regulations and tariffs. In December 2025, the National Defense Authorization Act for Fiscal Year 2026 was enacted, which included Section 851, commonly referred to as the “BIOSECURE Act.” The BIOSECURE Act restricts U.S. …”see in full comparison
“Upon losing our smaller reporting company status, we will no longer be able to rely on the scaled disclosure requirements and other accommodations available to smaller reporting companies. This may require us to provide additional disclosures in our periodic reports, including more detailed executive compensation disclosures and additional financial statement information. Due to this upcoming transition, we expect to devote significant time and effort to implement and comply with the additional standards, rules and regulations that may apply to us upon becoming a large accelerated filer. …”see in full comparison
At the state level, numerous U.S. states have enacted comprehensive privacy laws that impose certain obligations on covered businesses, many of which differ from each other in significant ways, thus complicating compliance efforts, including providing specific disclosures in privacy notices and affording individuals certain rights concerning their personal data. For example, in California, the California Consumer Privacy Act (“CCPA”) established a comprehensive privacy framework for covered businesses by creating an expanded definition of personal information, establishing data privacy rights for consumers in the State of California, imposing special rules on the collection of consumer data from minors, and creating a new and potentially severe statutory damages framework for violations of the CCPA and for businesses that fail to implement reasonable security procedures and practices to prevent data breaches. The liability framework includes civil penalties for violations, as well as a private right of action for data breaches that is expected to increase data breach litigation. While clinical trial data and information governed by HIPAA are exempt from the CCPA, other personal information may be applicable and periodic changes to the CCPA continue to broaden its scope.see in full comparison
The use of new and evolving technologies, such as AI/ML, in our operations, and the operations of third parties upon which we rely presents new risks and challenges that could negatively impact our business, including cybersecurity, data privacy, information technology, intellectual property, regulatory, legal, operational, competitive, reputational, and other risks and challenges. Specifically, risks related to bias, AI hallucinations, discrimination, harmful content, misinformation, fraud, scams, targeted attacks such as model poisoning or data poisoning, surveillance, data leakage, loss of consensus reality, inequality, environmental harms, and other harms may flow from our development, use, or deployment of AI technologies. The use of certain AI/ML technologies can also give rise to intellectual property risks, including compromises to proprietary intellectual property and intellectual property infringement. Additionally, several jurisdictions around the globe, including Europe and certain U.S. states, have proposed, enacted, or are considering, laws governing the development and use of AI/ML. Such laws in the EU impose significant obligations on providers and deployers of high risk artificial intelligence systems, come with significant penalties for non-compliance, and encourage providers and deployers of artificial intelligence systems to account for EU ethical principles in their development and use of these systems. For instance, the EU’s Artificial Intelligence Act (“AI Act”) prohibits certain uses of AI based on their risk levels and imposes various governance, risk management and transparency obligations. The AI Act may require us to implement AI governance, human oversight, documentation, monitoring, data quality, cybersecurity and transparency measures.see in full comparison
see in full comparisonSome of our manufacturers are located outside of the United States, including in China. There is currently significant uncertainty about the future relationship between the United States and various other countries, including China, with respect to trade policies, treaties, government regulations and tariffs.Increased tariffs or pending legislation that would impose federal contracting or federal funding limitations on parties directly using or connected to those using the services or equipment of certain foreign entities with known or alleged associations with foreign adversaries could potentially disrupt our existing supply chains and impose additional costs on our business. In particular, certain Chinese biotechnology companies and CMOs may become subject to trade restrictions, sanctions, and other regulatory requirements by the U.S. government, which could restrict or even prohibit our ability to work with such entities, thereby potentially disrupting our supplies and manufacturing. Additionally, it is possible further tariffs may be imposed that could affect imports of any APIs used in our product candidates in the future, or our business may be adversely impacted by retaliatory trade measures taken by China or other countries, including restricted access to such raw materials used in our product candidates. Given the unpredictable regulatory environment in China and the United States and uncertainty regarding how the U.S. or foreign governments will act with respect to tariffs, international trade agreements and policies, further governmental action related to tariffs, additional taxes, contracting matters, regulatory changes or other retaliatory trade measures in the future could occur with a corresponding detrimental impact on our business and financial condition.
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We are a clinical-stage biotechnology company with a limited operating history. We were formed in February 2022 and our operations to date have been limited to organizing and staffing our company, business planning, raising capital, developing our receptor associated protein (“RAP”) technology platform and technology, identifying potential product candidates, securing intellectual property rights, and planning and undertaking preclinical studies and clinical trials. Substantially all of our product candidates were initially developed by Janssen Pharmaceutical NV (“Janssen”), which we in-licensed pursuant to the option and license agreement with Janssen (the “Janssen License”), entered into shortly after our formation. We have not yet demonstrated an ability to generate product revenues, obtain regulatory approvals, manufacture any product on a commercial scale or arrange for a third party to do so on our behalf or conduct sales and marketing activities necessary for successful product commercialization. Our limited operating history as a company makes any assessment of our future success and viability subject to significant uncertainty. We will encounter risks and difficulties frequently experienced by early-stage biotechnology companies in rapidly evolving fields, and we have not yet demonstrated an ability to successfully overcome such risks and difficulties. If we do not address these risks and difficulties successfully, our business will suffer.
The success of our business depends primarily upon our ability to identify, develop, and commercialize product candidates based on our RAP technology platform. We do not know whether we will be able to develop any product candidates that succeed through preclinical and clinical development or products of commercial value. We have no products approved for commercial sale and have not generated any revenue from product sales to date. We will continue to incur significant research and development and other expenses related to our preclinical and clinical development and ongoing operations. As a result, we are not profitable and have incurred losses in each period since our inception. Net losses and negative cash flows have had, and will continue to have, an adverse effect on our stockholders’ equity and working capital. Our net losses totaled $19.9$76.5 million and $24.1$50.8 million for the threesix months ended MarchJune 31,30, 2026 and 2025, respectively. As of MarchJune 31,30, 2026, we have not yet generated revenues from product sales and had an accumulated deficit of $255.1$311.7 million. We expect to continue to incur significant losses for the foreseeable future, and we expect these losses to increase as we continue our research and development of, and seek regulatory approvals for, our product candidates.
As of MarchJune 31,30, 2026, we had $476.8$436.1 million of cash, cash equivalents and short-term investments, excluding restricted cash. Based upon our current operating plan, we believe that our existing cash, cash equivalents and short-term investments will enable us to fund our operating expenses and capital expenditure requirements into the second half of 2029. We have based this estimate on assumptions that may prove to be wrong, and we could utilize our available capital resources sooner than we expect. We may also raise additional financing on an opportunistic basis in the future. For example, we may seek additional capital due to favorable market conditions, including through our at-the-market program, or strategic considerations even if we believe we have sufficient funds for our current or future operating plans. Attempting to secure additional financing may divert our management from our day-to-day activities, which may adversely affect our ability to develop our product candidates. Our future capital requirements will depend on many factors, including but not limited to:
Until such time, if ever, as we can generate substantial product revenue, we expect to finance our operations with our existing cash, cash equivalents and short-term investments, any future equity or debt financings and upfront, milestone or royalty payments, if any, received under anyour current or future licenses, collaborations or royalty agreements. If we raise additional capital through the sale of equity or convertible debt securities, or issue any equity or convertible debt securities in connection with a collaboration agreement or other contractual arrangement, the ownership interests of our stockholders will be diluted, and the terms of these securities may include liquidation or other preferences that adversely affect the rights of our stockholders. In addition, the possibility of such issuance may cause the market price of our common stock to decline.
We previously entered into a common stock sales agreement (the “Sales Agreement”), dated July 1, 2025, by and between the Company and Leerink Partners LLC and Cantor Fitzgerald & Co., acting as sales agents, which established an at-the-market offering program pursuant to which we may offer and sell shares of our common stock from time to time (the “ATM Program”). In March 2026, we filed an automatic shelf registration statement on Form S-3ASR ( the “2026 Registration Statement”), containing (i) a base prospectus, which covers the offering, issuance and sale from time to time in one or more offerings of common stock, preferred stock, debt securities, warrants and/or units; and (ii) a sales agreement prospectus for the offering and issuance and sale of up to a maximum aggregate offering price of $150.0 million of common stock, to be issued pursuant to the Sales Agreement. As of MarchJune 31,30, 2026, we have not sold any shares pursuant to our ATM program.
To date, as an organization, we have not completed the development of any product candidatescandidates. andWe have initiated the Phase 3 program for RAP-219 in drug-resistant focal onset seizures (“FOS”), but nearly all of our other candidates remain in early-stage clinical or preclinical development. Our future success and ability to generate revenue from our product candidates is dependent on our ability to successfully develop, obtain regulatory approval for and commercialize one or more of our product candidates. All of our product candidates will require substantial additional investment for clinical development, regulatory review and approval in one or more jurisdictions. If any of our product candidates, particularly RAP-219, encounter safety or efficacy problems, development delays or regulatory issues or other problems, our development plans and business would be materially harmed.
Our lead product candidate, RAP-219, is in clinical development for focal onset seizures (“FOS”),FOS, primary generalized tonic-clonic seizures (“PGTCS”) and bipolar mania, and we continue to develop a long-acting injectable (“LAI”) formulation. Additionally, we have two advanced discovery-stage nAChR programs stemming from our RAP technology platform. The first is our α6ß4 nAChR agonist development candidate, RAP-641, a genetically validated precision target that we are pursuing as a potential novel non-opiate, non-CNS approach for chronic pain and migraine, and the second comprises modulators of the α9α10 nAChR which third-party preclinical genetic data suggest could be an attractive target in treating hearing and vestibular disorders.
At the state level, numerous U.S. states have enacted comprehensive privacy laws that impose certain obligations on covered businesses, many of which differ from each other in significant ways, thus complicating compliance efforts, including providing specific disclosures in privacy notices and affording individuals certain rights concerning their personal data. For example, in California, the California Consumer Privacy Act (“CCPA”) established a comprehensive privacy framework for covered businesses by creating an expanded definition of personal information, establishing data privacy rights for consumers in the State of California, imposing special rules on the collection of consumer data from minors, and creating a new and potentially severe statutory damages framework for violations of the CCPA and for businesses that fail to implement reasonable security procedures and practices to prevent data breaches. The liability framework includes civil penalties for violations, as well as a private right of action for data breaches that is expected to increase data breach litigation. While clinical trial data and information governed by HIPAA are exempt from the CCPA, other personal information may be applicable and periodic changes to the CCPA continue to broaden its scope.
Outside the United States, an increasing number of laws, regulations, and industry standards govern privacy and data security. For example, the European Union’s General Data Protection Regulation (“EU GDPR”) and the United Kingdom’s GDPR (“UK GDPR”) impose strict requirements for processing personal data. Also in Europe, the ePrivacy Directive in the EU and the Privacy and Electronic Communications Regulations in the UK impose obligations regarding our use of cookies and tracking technologies on our website and set forth requirements related to direct marketing. Non-compliance with these requirements could expose us to regulatory fines, the maximum amounts of which vary by jurisdiction.
Similarly, China’s Personal Information Protection Law (“PIPL”), which is enforced by the Cyberspace Administration of China (“CAC”), imposes comprehensive requirements on the processing of personal information, including restrictions on the export of sensitive personal information outside of China. PIPL requires data handlers to satisfy specified conditions, such as obtaining certification or entering into standard contractual arrangements prescribed by the CAC, before transferring personal information abroad. If our partners or collaborators operating in China, including Tenacia, are deemed to be in violation of PIPL's cross-border data transfer restrictions, the CAC or other competent authorities could order the suspension or cessation of such data transfers, which could prevent or materially delay the sharing of clinical trial data and other sensitive information with us and adversely affect our ability to advance the development and regulatory approval of our product candidates.
Similarly, China’s Personal Information Protection Law (“PIPL”), imposes comprehensive requirements on the processing of personal information, including restrictions on the export of sensitive personal information outside of China. PIPL requires data handlers to satisfy specified conditions, such as obtaining certification or entering into standard contractual arrangements prescribed by the CAC, before transferring personal information abroad. If our partners or collaborators operating in China, including Tenacia, are deemed to be in violation of PIPL's cross-border data transfer restrictions, the CAC or other competent authorities could order the suspension or cessation of such data transfers, which could prevent or materially delay the sharing of clinical trial data and other sensitive information with us and adversely affect our ability to advance the development and regulatory approval of our product candidates.
The risk of a cyber-attack, cybersecurity incident, breach, compromise, or other interruption has generally increased as the number, intensity and sophistication of attempted attacks and intrusions from around the world have increased. Such risks come from a variety of evolving threats, including but not limited to, social engineering attacks (including through deep fakes, which may be increasingly difficult to identify as fake, and phishing attacks), malicious code (such as viruses and worms), malware (including as a result of advanced persistent threat intrusions), denial-of-service attacks, credential stuffing, credential harvesting, personnel misconduct or error, ransomware attacks, supply-chain attacks, software bugs, server malfunctions, software or hardware failures, loss of data or other information technology assets, adware, attacks enhanced or facilitated by artificial intelligence (“AI”), telecommunications failures, earthquakes, fires, floods, and other similar threats. Use of generative AI models in our internal or third-party systems may create new attack surfaces or methods for adversaries, which could impact us and our vendors. Malicious actors may use AI tools to identify and exploit these and other software vulnerabilities, as well as use AI technologies to launch more sophisticated, automated, targeted and coordinated attacks. Any cybersecurity incidents or data breaches related to our use of AI applications could adversely affect our reputation and results of operations.
In addition, our reliance on third-party service providersprovider software could introduce new cybersecurity risks and vulnerabilities, including supply-chain attacks. We rely on third-party service providers and technologies to operate critical business systems to process sensitive data in a variety of contexts and our ability to monitor these third parties’ information security practices is limited. These third parties may not have adequate information security measures in place and if our third-party service providers experience a cyber-attack, cybersecurity incident, breach, compromise, or other interruption, we could experience adverse consequences. While we may be entitled to damages if our third-party service providers fail to satisfy their privacy or security-related obligations to us, any award may be insufficient to cover our damages, or we may be unable to recover such award.
We have in the past experienced threats and security incidents related to our data and systems, and we may in the future experience additional threats, compromises, breaches or incidents. If we, or a third party upon whom we rely, experience a cyber-attack, cybersecurity incident, breach, compromise, or other interruption, or are perceived to have experienced a cyber-attack, cybersecurity incident, breach, compromise, or other interruption, we may experience adverse consequences, such as government enforcement actions (for example, investigations, fines, penalties, audits, and inspections); additional reporting requirements and/or oversight; restrictions on processing sensitive information (including personal data); litigation (including individual and group claims); indemnification obligations; negative publicity; reputational harm; monetary fund diversions; interruptions in our operations (including availability of data); financial loss; and other potentially significant harms. Such events may require us to fundamentally change our business activities and practices in response to such litigation or investigations. Our contracts may not contain limitations of liability, and even where they do, there can be no assurance that limitations of liability in our contracts are sufficient to protect us from liabilities, damages, or claims related to our privacy and data security obligations. Cyber liability insurance may not provide adequate coverage against potential liabilities related to any experienced cybersecurity incident or breach.
The use of new and evolving technologies, such as AI/ML, in our operations, and the operations of third parties upon which we rely presents new risks and challenges that could negatively impact our business, including cybersecurity, data privacy, information technology, intellectual property, regulatory, legal, operational, competitive, reputational, and other risks and challenges. Specifically, risks related to bias, AI hallucinations, discrimination, harmful content, misinformation, fraud, scams, targeted attacks such as model poisoning or data poisoning, surveillance, data leakage, loss of consensus reality, inequality, environmental harms, and other harms may flow from our development, use, or deployment of AI technologies. The use of certain AI/ML technologies can also give rise to intellectual property risks, including compromises to proprietary intellectual property and intellectual property infringement. Additionally, several jurisdictions around the globe, including Europe and certain U.S. states, have proposed, enacted, or are considering, laws governing the development and use of AI/ML. Such laws in the EU impose significant obligations on providers and deployers of high risk artificial intelligence systems, come with significant penalties for non-compliance, and encourage providers and deployers of artificial intelligence systems to account for EU ethical principles in their development and use of these systems. For instance, the EU’s Artificial Intelligence Act (“AI Act”) prohibits certain uses of AI based on their risk levels and imposes various governance, risk management and transparency obligations. The AI Act may require us to implement AI governance, human oversight, documentation, monitoring, data quality, cybersecurity and transparency measures.
Likewise, such legislative developments in the United States have introduced emerging compliance obligations for companies that develop or deploy AI technologies, which will impose novel risks on AI developers and users. Recently, states have advanced, and in some cases passed, dozens of laws focused on AI governance and regulation, including on deployment of AI in healthcare settings. These state-level initiatives reflect a growing trend toward AI regulation in the absence of federal legislation. As a result, we may face a fragmented and evolving compliance landscape that could increase operational complexity, regulatory scrutiny, and legal exposure associated with our use or development of AI technologies. If we develop or use AI systems governed by these laws or regulations, we will need to meet higher standards of data quality, transparency, monitoring and human oversight, and we would need to adhere to specific and potentially burdensome and costly ethical, accountability, and administrative requirements, with the potential for significant enforcement or litigation in the event of any perceived non-compliance. Additionally, certain privacy laws extend rights to consumers (such as the right to delete certain personal data) and regulate automated decision making, which may bepose incompatibledifficulties within the context of our use of AI/ML. These obligations may make it harder for us to conduct our business using AI/ML, lead to regulatory fines or penalties, require us to change our business practices, retrain our AI/ML, or prevent or limit our use of AI/ML. For example, the Federal Trade Commission has required other companies to turn over (or disgorge) valuable insights or trainings generated through the use of AI/ML where they allege the company has violated privacy and consumer protection laws. If we cannot use AI/ML or that use is restricted, our business may be less efficient, or we may be at a competitive disadvantage.
The rapid evolution of AI/ML will require the application of significant resources to design, develop, test and maintain our products and services to help ensure that AI/ML is implemented in accordance with applicable law and regulation and in a socially responsible manner and to minimize any real or perceived unintended harmful impacts. Our vendors may in turn incorporate AI/ML tools into their own offerings, and the providers of these AI/ML tools may not meet existing or rapidly evolving regulatory or industry standards, including with respect to privacy and data security. Further, bad actors around the world use increasingly sophisticated methods, including the use of AI/ML, to engage in cyber-attacks, cyber espionage campaigns, exploitation of expanded attack surfaces, and other illegal activities involving the theft and misuse of sensitive data. Any of these effects could damage our reputation, result in the loss of valuable property and information, cause us to breach applicable laws and regulations, and adversely impact our business.
Further, bad actors around the world use increasingly sophisticated methods, including the use of AI/ML, to engage in cyber-attacks, cyber espionage campaigns, exploitation of expanded attack surfaces, and other illegal activities involving the theft and misuse of sensitive data. Technological developments in AI, including advances in frontier artificial intelligence models, may increase the sophistication and speed of cybersecurity incidents and other malicious attacks that we experience. These technologies may enable threat actors to better identify vulnerabilities, develop targeted exploits, conduct phishing or social engineering campaigns, or otherwise compromise or breach our information systems more efficiently. Vulnerabilities that affect us or our third-party service providers may be exploited more rapidly, and although we maintain cybersecurity risk management processes designed to address cybersecurity threats, evolving artificial intelligence capabilities may reduce these safeguards’ effectiveness and require additional investments and resources. Any of these effects could damage our reputation, result in the loss of valuable property and information, cause us to breach applicable laws and regulations, and adversely impact our business.
We are not permitted to commercialize, market, promote or sell any product candidate in the United States without obtaining regulatory approval from the FDA. Foreign regulatory authorities, such as the EMA and MHRA, impose similar requirements. The time required to obtain approval by the FDA, EMA, MHRA or other comparable regulatory authorities is inherently unpredictable, but typically takes many years following the commencement of clinical trials and depends upon numerous factors, including substantial discretion of the regulatory authorities. In addition, approval policies, regulations, or the type and amount of clinical data necessary to gain approval may change during the course of a product candidate’s clinical development and may vary among jurisdictions. ForThe instance,leadership jurisdictionschanges at the FDA or other global regulatory authorities may compound this uncertainty. Jurisdictions outside of the United States, such as the European Union or Japan, may have different requirements for regulatory approval, which may require us to conduct additional clinical, nonclinical or chemistry, manufacturing and control studies. Moreover, the U.S. Supreme Court’s July 2024 decision to overturn prior established case law giving deference to regulatory agencies’ interpretations of ambiguous statutory language has introduced uncertainty regarding the extent to which FDA’s regulations, policies, and decisions may become subject to increasing legal challenges, delays, and/or changes. To date, we have not submitted an NDA to the FDA or similar drug approval submissions to comparable foreign regulatory authorities for any product candidate. We must complete additional preclinical studies and clinical trials to demonstrate the safety and efficacy of our product candidates in humans before we will be able to obtain these approvals.
The general approach for FDA approval of a new drug is dispositive data from two or more adequate and well-controlled clinical trials of the product candidate in the relevant patient population. Adequate and well-controlled clinical trials typically involve a large number of patients, have significant costs and take years to complete. The FDA, EMA, MHRA or other comparable regulatory authorities may disagree with us about whether a clinical trial is adequate and well-controlled or may request that we conduct additional clinical trials prior to regulatory approval. In addition, there is no assurance that the doses, endpoints and trial designs that we intend to use for our planned clinical trials, including those that we have developed based on feedback from regulatory agencies or those that have been used for the approval of similar drugs, will be acceptable for future approvals. ForIn instance,June we may seek2026, FDA regulatoryissued flexibilityrevised draft guidance clarifying how sponsors can rely on one scientifically rigorous adequate and well-controlled clinical investigation with confirmatory evidence to satisfy the statutory substantial evidence of effectiveness standard. We may pursue marketing approval based on data from only one adequate and well-controlled clinical investigation. However, theFDA retains broad discretion to require additional clinical data for any product candidate, including a second adequate and well-controlled clinical trial. The FDA may be unwilling to apply regulatory flexibility and our clinical trial results may not support approval of our product candidates. In addition, our product candidates could fail to receive regulatory approval, or regulatory approval could be delayed, for many reasons, including the following:
We may observe safety or tolerability issues beyond those we anticipate with our product candidates in ongoing or future RAP-219 clinical trials or in clinical programs for other product candidates. Additionally, it is possible that patients in our trials may experience greater side effects than observed in healthy volunteers. Additionally, adverse safety effects observed with AMPAR-inhibitors could adversely impact future labeling FDA may require for RAP-219. For example, perampanel’s FDA-approved label is accompanied by a black boxboxed warning for serious psychiatric and behavioral reactions, including aggression, hostility and homicidal ideation and threats. Furthermore, significant drug-drug interactions were reported for perampanel. The concomitant use with the other ASMs carbamazepine, phenytoin and oxcarbazepine decreased plasma levels of perampanel by approximately 50 to 67 percent. In addition, perampanel at a dose of 12 mg per day reduced exposure of levonorgestrel, an oral contraceptive, by approximately 40 percent. While we believe there are critical differences between perampanel and RAP-219, the FDA could impose similar warnings and labeling requirements on RAP-219, if approved, in the future.
The process of obtaining marketing approvals, both in the United States and abroad, is expensive, may take many years if additional clinical trials are required, if approval is obtained at all, and can vary substantially based upon a variety of factors, including the type, complexity, and novelty of the product candidates involved. Changes in marketing approval policies during the development period, changes in or the enactment of additional statutes or regulations, changes in leadership, or changes in regulatory review for each submitted product application, may cause delays in the approval or rejection of an application. The FDA, EMA, MHRA and other comparable regulatory authorities have substantial discretion in the approval process and may refuse to accept any application or may decide that our data are insufficient for approval and require additional preclinical, clinical or other studies. In addition, varying interpretations of the data obtained from preclinical and clinical testing could delay, limit, or prevent marketing approval of a product candidate. Any marketing approval that we may ultimately obtain could be limited or subject to restrictions or post-approval commitments that render the approved product not commercially viable.
Factors that may inhibit our or third parties' efforts to commercialize our product candidates on our own include:
our inabilityability to recruit and retain adequate numbers of effective sales and marketing personnel;
the inabilityability of sales personnel to obtain access to or our failure to educate an adequate number of physicians on the benefits of any future products;
Our ability to develop product candidates, leverage our RAP technology platform and manage our future growth depends on attracting, hiring and retaining our key personnel and recruiting additional qualified personnel.
the possible terminationtermination, re-negotiation, or non-renewal of the manufacturing agreements by the third party, at a time or in a matter that is costly or inconvenient to us; and the possible misappropriation of our proprietary information, including our trade secrets and know-how.
Some of our manufacturers are located outside of the United States, including in China. There is currently significant uncertainty about the future relationship between the United States and various other countries, including China, with respect to trade policies, treaties, government regulations and tariffs. In December 2025, the National Defense Authorization Act for Fiscal Year 2026 was enacted, which included Section 851, commonly referred to as the “BIOSECURE Act.” The BIOSECURE Act restricts U.S. government agencies from procuring biotechnology equipment or services from, or entering into contracts with, entities that use biotechnology equipment or services from, designated “biotechnology companies of concern” (“BCCs”), and from expending federal loan or grant funds for such equipment or services. The Office of Management and Budget (“OMB”) is required to publish an initial list of BCCs by December 2026, with implementing regulations expected to follow thereafter before the procurement prohibitions formally take effect. The BCC list has not yet been published and there can be no assurance that our existing or future Chinese manufacturing partners will not be designated in the future. If any of our current or future manufacturers or vendors is designated as a BCC, or if our research institutions, collaborators, or other partners become subject to BIOSECURE-related restrictions as a result of their relationships with such manufacturers, we could be required to terminate or restructure existing arrangements, transition manufacturing or other services to alternative suppliers, or delay or suspend development activities. In addition, even absent a formal BCC designation, U.S. research institutions and other recipients of federal grants and contracts may seek to limit relationships with manufacturers flagged under related national security frameworks, which could indirectly affect our clinical development programs. Any such transition or disruption could involve significant cost, operational complexity, regulatory risk, and delays, and alternative suppliers may not be available on acceptable terms or at all.
Some of our manufacturers are located outside of the United States, including in China. There is currently significant uncertainty about the future relationship between the United States and various other countries, including China, with respect to trade policies, treaties, government regulations and tariffs. Increased tariffs or pending legislation that would impose federal contracting or federal funding limitations on parties directly using or connected to those using the services or equipment of certain foreign entities with known or alleged associations with foreign adversaries could potentially disrupt our existing supply chains and impose additional costs on our business. In particular, certain Chinese biotechnology companies and CMOs may become subject to trade restrictions, sanctions, and other regulatory requirements by the U.S. government, which could restrict or even prohibit our ability to work with such entities, thereby potentially disrupting our supplies and manufacturing. Additionally, it is possible further tariffs may be imposed that could affect imports of any APIs used in our product candidates in the future, or our business may be adversely impacted by retaliatory trade measures taken by China or other countries, including restricted access to such raw materials used in our product candidates. Given the unpredictable regulatory environment in China and the United States and uncertainty regarding how the U.S. or foreign governments will act with respect to tariffs, international trade agreements and policies, further governmental action related to tariffs, additional taxes, contracting matters, regulatory changes or other retaliatory trade measures in the future could occur with a corresponding detrimental impact on our business and financial condition.
Our failure, or the failure of our third-party manufacturers, to comply with applicable regulations could result in sanctions being imposed on us, including clinical holds, fines, injunctions, civil penalties, delays, suspension or withdrawal of approvals, seizures or voluntary recalls of product candidates, operating restrictions and criminal prosecutions, any of which could significantly affect supplies of our product candidates. The facilities used by our contract manufacturers to manufacture our product candidates must be evaluated by the FDA. We do not control the manufacturing process of, and are completelylargely dependent on, our contract manufacturing partners for compliance with cGMPs. If our contract manufacturers cannot successfully manufacture material that conforms to our specifications and the strict regulatory requirements of the FDA, EMA, MHRA or other comparable regulatory authorities, we may not be able to secure and/or maintain regulatory approval for our product candidates manufactured at these facilities. In addition, we have nolimited control over the ability of our contract manufacturers to maintain adequate quality control, quality assurance and qualified personnel. If the FDA finds deficiencies or a comparable foreign regulatory authority does not approve these facilities for the manufacture of our product candidates or if it withdraws any such approval in the future, we may need to find alternative manufacturing facilities, which wouldcould significantly impact our ability to develop, obtain regulatory approval for or market our product candidates, if approved. Contract manufacturers may face manufacturing or quality control problems causing drug substance production and shipment delays or a situation where the contractor may not be able to maintain compliance with the applicable cGMP requirements. Any failure to comply with cGMP requirements or other FDA, EMA, MHRA and comparable foreign regulatory requirements could adversely affect our clinical research activities and our ability to develop our product candidates and market our products, if approved.
Contract manufacturers may face manufacturing or quality control problems causing drug substance production and shipment delays or a situation where the contractor may not be able to maintain compliance with the applicable cGMP requirements. Any failure to comply with cGMP requirements or other FDA, EMA, MHRA and other comparable regulatory requirements could adversely affect our clinical research activities and our ability to develop our product candidates and market our products following approval, if obtained.
We plan to opportunistically pursue strategic partnerships, as the advancement of our product candidates and development programs and the potential commercialization of our current and future product candidates will require substantial additional cash to fund expenses. If we believe that additional partnerships can accelerate the development or maximize the market potential of our product candidates, we will consider entering into product, target and/or geographic specific strategic partnerships on an opportunistic basis. Likely collaborators may include large and mid-size pharmaceutical companies, regional and national pharmaceutical companies and biotechnology companies. In addition, if we are able to obtain regulatory approval for product candidates from foreign regulatory authorities, we may enter into additional partnerships or collaborations with international biotechnology or pharmaceutical companies for the commercialization of such product candidates.
As product candidates proceed through preclinical studies to late-stage clinical trials towards potential approval and commercialization, it is common that various aspects of the developmentmanufacturing program, such as the vendors used to manufacture drug product or manufacturing methods and formulation, are altered along the way in an effort to optimize processes and results. Such changes carry the risk that they will not achieve these intended objectives. Any of thesesuch changes could cause our product candidates to perform differently and affect the results of planned clinical trials or other future clinical trials conducted with the materials manufactured using altered processes.processes, and limit our ability to rely on data from clinical trials conducted with an earlier formulation of our drug candidate. Such changes may also require additional testing, FDA notification or FDA approval. This could delay or prevent completion of clinical trials, require conducting bridging clinical trials or the repetition of one or more clinical trials, increase clinical trial costs, delay or prevent approval of our product candidates and jeopardize our ability to commence sales and generate revenue.
The FDA may impose consent decrees or withdraw approval if compliance with regulatory requirements and standards is not maintained or if problems occur after the product reaches the market. Later discovery of previously unknown problems with our product candidates, including AEs of unanticipated severity or frequency, or problems with our third-party manufacturers or manufacturing processes, or failure to comply with regulatory requirements, may result in revisions to the approved labeling to add new safety information, imposition of post-market studies or clinical trials to assess new safety risks or imposition of distribution restrictions or other restrictions under a REMS program. Other potential consequences include, among other things:
fines, FDA Form 483s, warning letters or holds on clinical trials;
The ability of the FDA or comparable foreign regulatory authorities to review and approve new products can be affected by a variety of factors, including government budget and funding levels, ability to hire and retain key personnelleadership and other personnel, and ability to accept the payment of user fees, and statutory, regulatory, leadership, and policy changes. Average review times at the FDA have fluctuated in recent years as a result. In addition, government funding of other government agencies or comparable foreign regulatory authorities on which our operations may rely, including those that fund research and development activities, is subject to the political process, which is inherently fluid and unpredictable.
Without appropriation of sufficient funding for federal agencies, our business operations related to our product development activities for the U.S. market could be impacted. The ability of the FDA to review and approve new products can be affected by a variety of factors, including staffing cuts, government budget and funding levels, ability to hire and retain key personnel and ability to accept the payment of user fees and statutory, regulatory, leadership and policy changes. Average review times at the agency have fluctuated in recent years as a result. In addition, government funding of other government agencies on which our operations may rely, including those that fund research and development activities, is subject to the political process, which is inherently fluid and unpredictable.
There is substantial uncertainty as to whetherthe extent and howmanner in which the U.S. government will seek to modify or revise the requirements and policies of the FDA and other regulatory agencies with jurisdiction over our product candidates and any products for which we obtain approval. This uncertainty could present new challenges and/or opportunities as we navigate development and approval of our product candidates. Additionally, the current administration could issue or promulgate executive orders, regulations, policies or guidance that adversely affect us or create a more challenging or costly environment to pursue the development of new therapeutic candidates. Also, state governments may seek to address or react to changes at the federal level with changes to their regulatory frameworks in a manner that could impact our operations.
As of MarchJune 31,30, 2026, we had 47,807,45347,955,079 shares of common stock outstanding. Shares of unvested restricted common stock will become available for sale immediately upon the vesting of such shares, as applicable. All shares of common stock sold in our IPO or otherwise outstanding as of our IPO are able to be sold in the public market. Shares issued upon the exercise of stock options pursuant to future awards that may be granted under our equity incentive plans or pursuant to future awards granted under those plans will become available for sale in the public market to the extent permitted by the provisions of applicable vesting schedules, any applicable market stand-off and lock-up agreements, and Rule 144 and Rule 701 under the Securities Act of 1933, as amended (the “Securities Act”).
We expect to issue additional capital stock in the future that will result in dilution to all other stockholders. For example, in September 2025, we completed an underwritten public offering of 11,057,692 shares of common stock at a public offering price of $26.00 per share, which included full exercise of the underwriters’ option to purchase additional shares. Additionally, as of MarchJune 31,30, 2026, 11,179,73511,032,109 shares of our common stock were reserved for issuance upon exercise of outstanding stock options, vested restricted stock units and performance based stock units. We expect to grant equity awards to employees, directors and consultants under our stock incentive plans. We may also raise capital through equity financings in the future. As part of our business strategy, we may acquire or make investments in complementary companies, products or technologies and issue equity securities to pay for any such acquisition or investment. Any such issuances of additional capital stock may cause stockholders to experience significant dilution of their ownership interests and the per share value of our common stock to decline.
The financial markets and the global economy may also be adversely affected by the current or anticipated impact of political uncertainty, including military conflicts, such as the ongoing conflicts between Russia and Ukraine and Israel and Hamas, the recent U.S. and Israeli military action in Iran and effects thereof, terrorism, or other geopolitical events. Sanctions imposed by the U.S. and other countries in response to such conflicts, including the one in Ukraine, may also continue to adversely impact the financial markets and the global economy, and any economic countermeasures by the affected countries or others could exacerbate market and economic instability. Additionally, recent changes in U.S. policy have impacted and may in the future impact, among other things, the U.S. and global economy, international trade relations, unemployment, immigration, healthcare, taxation, the U.S. regulatory environment, inflation and other areas. For example, the implementation of tariffs by the U.S. government has led to increased trade disruptions and political tensions, between not only the U.S. and China, but also between the U.S. and other countries in the international community. In response to tariffs, other countries have implemented and may in the future implement additional retaliatory tariffs on U.S. goods. Political tensions as a result of trade policies could reduce trade volume, investment, technological exchange and other economic activities between major international economies, resulting in a material adverse effect on global economic conditions and the stability of global financial markets. TheFor Nationalexample, Defenseas Authorizationdescribed more fully above, the BIOSECURE Act for fiscal year 2026, signed into law on December 18, 2025, included a prohibition on contracting with certain biotechnology providers that may restrict the ability of U.S. biotechnology companies to purchase services or products from, or otherwise collaborate with, certain Chinese biotechnology companies of concern without losing the ability to contract with, or otherwise receive funding from, the U.S. government. We continue to assess this law to determine whether it could have an effect on our contractual relationships. Although we have taken steps to mitigate the impact of tariffs and trade restrictions on our operations, any changes in political, trade, regulatory, and economic conditions, including U.S. trade policies, could have a material adverse effect on our business, financial condition and results of operations. Until we know what policy changes are made, whether those policy changes are challenged and subsequently upheld by the court system and how those changes impact our business and the business of our competitors over the long term, we will not know if, overall, we will benefit from them or be negatively affected by them.
We are an “emerging growth company” as defined in the Jumpstart Our Business Startups Act (“JOBS Act”). For as long as we continue to be an emerging growth company, we may take advantage of exemptions from various reporting requirements that are applicable to other public companies that are not emerging growth companies, including not being required to comply with the auditor attestation requirements of Section 404, reduced disclosure obligations regarding executive compensation in our periodic reports and proxy statements and exemptions from the requirements of holding a nonbinding advisory vote on executive compensation and stockholder approval of any golden parachute payments not previously approved. In addition, as an emerging growth company, we are only required to provide two years of audited financial statements. We could be an emerging growth company until as late as December 31, 2029, although circumstances could cause us to lose that status earlier, including if we are deemed to be a “large accelerated filer,” which occurs when the market value of our common stock that is held by non-affiliates exceeds $700 million as of the prior June 30,30 unless we remain eligible to use the “smaller reporting company” requirements under the revenue test in Rule 12b-2 of the Exchange Act, or if we have total annual gross revenue of $1.235 billion or more during any fiscal year before that time, in which cases we would no longer be an emerging growth company as of the following December 31, or if we issue more than $1.0 billion in non-convertible debt during any three-year period before that time, in which case we would no longer be an emerging growth company immediately. For so long as we remain an emerging growth company, we are permitted and intend to rely on exemptions from certain disclosure requirements that are applicable to other public companies that are not emerging growth companies. These exemptions include:
not being required to comply with the auditor attestation requirements of Section 404;
providing only two years of audited financial statements in addition to any required unaudited interim financial statements and a correspondingly reduced “Management’s Discussion and Analysis of Financial Condition and Results of Operations” disclosure;
reduced disclosure obligations regarding executive compensation; and exemptions from the requirements of holding a nonbinding advisory vote on executive compensation and shareholder approval of any golden parachute payments not previously approved.
We are also a “smaller reporting company” as defined in the Exchange Act, and have elected to take advantage of certain of the scaled disclosures available to smaller reporting companies. As a smaller reporting company, we have availed ourselves of the exemption from the requirement that our independent registered public accounting firm attest to the effectiveness of our internal control over financial reporting under Section 404 of the Sarbanes-Oxley Act of 2002.
On the last business day of our second quarter in 2026, the aggregate market value of our shares of common stock held by non-affiliates exceeded $700 million. As a result, beginning with our Quarterly Report on Form 10-Q for the quarter ended March 31, 2027, we will no longer be eligible to use the scaled disclosure accommodations available to smaller reporting companies and will become subject to additional disclosure requirements.
Upon losing our smaller reporting company status, we will no longer be able to rely on the scaled disclosure requirements and other accommodations available to smaller reporting companies. This may require us to provide additional disclosures in our periodic reports, including more detailed executive compensation disclosures and additional financial statement information. Due to this upcoming transition, we expect to devote significant time and effort to implement and comply with the additional standards, rules and regulations that may apply to us upon becoming a large accelerated filer. The transition from smaller reporting company status will require additional resources, including personnel, enhanced systems and processes, and increased professional fees for accounting, legal, and compliance services. We will need to implement more comprehensive internal controls and procedures to meet the heightened requirements applicable to larger public companies. Due to the complexity and logistical difficulty of implementing the standards, rules and regulations that apply to non-smaller reporting companies, there is an increased risk that we may be found to be in non-compliance with such standards, rules and regulations or to have significant deficiencies or material weaknesses in our internal controls over financial reporting. Any failure to maintain effective disclosure controls and internal control over financial reporting could materially and adversely affect our business, results of operations and financial condition, and could cause a decline in the trading price of our common stock.
We are also a “smaller reporting company” as defined in the Exchange Act. We may continue to be a smaller reporting company even after we are no longer an emerging growth company. We may take advantage of certain of the scaled disclosures available to smaller reporting companies and will be able to take advantage of these scaled disclosures for so long as our common stock held by non-affiliates is less than $250.0 million measured on the last business day of our second fiscal quarter, or our annual revenue is less than $100.0 million during the most recently completed fiscal year and our common stock held by non-affiliates is less than $700.0 million measured on the last business day of our second fiscal quarter.
If we fail to establish and maintain an effective system of internal control over financial reporting, we may not be able to accurately report our financial results or prevent fraud. As a result, stockholders could lose confidence in our financial and other public reporting, which would harm our business and the trading price of our common stock.
Ensuring that we have adequate internal financial and accounting controls and procedures in place so that we can produce accurate financial statements on a timely basis is a costly and time-consuming effort that needs to be reevaluated frequently. Our internal control over financial reporting is a process designed to provide reasonable assurance regarding the reliability of financial reporting and the preparation of financial statements in accordance with generally accepted accounting principles. We havecurrently begun the process of documenting, reviewingmaintain and improvingreview our internal controls and procedures for compliance with Section 404 of the Sarbanes-Oxley Act, which requires an annual management assessment of the effectiveness of our internal control over financial reporting. Any failure to implement required new or improved controls, or any difficulties encountered in their implementation, could cause us to fail to meet our reporting obligations. In addition, any testing by us conducted in connection with Section 404, or any subsequent testing by our independent registered public accounting firm, may reveal deficiencies in our internal controls over financial reporting that are deemed to be material weaknesses or that may require prospective or retroactive changes to our financial statements or identify other areas for further attention or improvement. Inferior internal controls could also cause investors to lose confidence in our reported financial information, which could have a negative effect on the trading price of our stock.
We are required to disclose changes made in our internal controls and procedures on a quarterly basis and our management is required to assess the effectiveness of these controls annually. However, for as long as we are an emerging growth company or a non-accelerated filer, our independent registered public accounting firm will not be required to attest to the effectiveness of our internal controls over financial reporting pursuant to Section 404(b) of the Sarbanes-Oxley Act. We could remain an emerging growth company until as late as December 31, 2029. An independent assessment of the effectiveness of our internal controls over financial reporting could detect problems that our management’s assessment might not. Undetected material weaknesses in our internal controls over financial reporting could lead to restatements of our financial statements and require us to incur the expense of remediation.
Management's Discussion & Analysis (MD&A)
New heading “Selling, General and Administrative Expenses”
New heading “Comparison of the six months ended June 30, 2026 and 2025”
New heading “Operating Expenses”
New heading “Research and Development Expenses”
New heading “Selling, General and Administrative Expenses”
Removed heading “General and Administrative Expenses”
Largest changes
“We are also a “smaller reporting company” as defined in the Exchange Act, and have elected to take advantage of certain of the scaled disclosures available to smaller reporting companies. …”see in full comparison
Full comparison: every changed paragraph (62)
In late 2025, we initiated an open-label long term safety trial to allow patients enrolled in our Phase 2a proof-of-concept trial in drug-resistant FOS trial to resume taking RAP-219. Data from the open-label trial is expected in the secondfourth halfquarter of 2026. In December 2025, we received U.S. Food and Drug Administration (“FDA”) feedback from an end-of-Phase 2 meeting supporting advancement into two Phase 3 trials of RAP-219 in patients with drug-resistant FOSFOS. andWe we are on track to initiateinitiated the Phase 3 program in FOS in the second quarter of 2026.
We believe RAP-219 also has therapeutic potential in bipolar disorder. Our Phase 2 proof-of-concept trial in bipolar mania is progressing well and topline results are now expected in theOctober fourth quarter of 2026, ahead of the previous guidance of first half of 2027.2026. Additionally, we have modified the trial's statistical analysis plan and increased target enrollment, enabling the trial to potentially be considered as confirmatory evidence of effectiveness. Following completion of the Phase 2 trial, and subject to the results, we plan to engage with the FDA in an end-of-Phase 2 meeting to align on the design of a potential Phase 3 program to support a New Drug Application for the treatment of bipolar mania.
We also have two advanced discovery-stage nicotinic acetylcholine receptor (“nAChR”) programs stemming from our RAP technology platform. nAChRs have been clinically validated in patient-reported neuropathic pain and our first advanced discovery-stage nAChR program comprises agonists of the α6ß4 nAChR. α6ß4 nAChRs are selectively expressed in sensory neurons, and the α6 subunit has human genetic validation in chronic pain. We have initiated IND-enabling activities for our α6β46ß4 nAChR agonist development candidate, RAP-641, directed at a genetically validated precision target that we are pursuing as a potential novel non-opiate, non-CNS approach for chronic pain and migraine. The second advanced discovery-stage nAChR program comprises modulators of the α9α10 nAChR. Third-party preclinical genetic data suggest that this nAChR could be an attractive target in treating hearing and vestibular disorders. We continue to leverage our RAP technology platform to discover additional product candidates that we believe have the potential to provide transformative benefits for large patient populations with neurological or psychiatric diseases.
We have incurred significant operating losses in each year since our inception. Our ability to generate product revenue sufficient to achieve profitability will depend heavily on the successful development and eventual commercialization of any product candidates we may develop. Our net losses were $19.9$56.6 million and $24.1$26.7 million for the three months ended MarchJune 31,30, 2026 and 2025, respectively, and $76.5 million and $50.8 million for the six months ended June 30, 2026 and 2025, respectively. As of MarchJune 31,30, 2026, we had an accumulated deficit of $255.1$311.7 million. We expect our expenses and operating losses will increase substantially as we:
ultimately establish a sales, marketing and distribution infrastructure to commercialize any therapies for which we may obtain regulatory approval; and continue to incur additional costs associated with being a public company, including audit, legal, regulatory, and tax-related services associated with maintaining compliance with an exchange listing and Securities Exchange Commission (“SEC”) requirements, director and officer insurance premiums and investor relations costs.
We believe that our existing cash andcash, cash equivalents, and short-term investments will enable us to fund our operating expenses and capital expenditure requirements into the second half of 2029. See the sections titled “—Liquidity and Capital Resources” and “Risk Factors—Risks Related to Our Limited Operating History, Financial Condition and Need for Additional Capital” included elsewhere in this Quarterly Report.
Concurrently with the execution of the NeuroPace Agreement, the parties also entered into an initial statement of work, as amended in March 2024, and a second statement of work, as amended in February 2026 (collectively, the “NeuroPace SOWs”), under the NeuroPace Agreement, pursuant to which NeuroPace agreed to provide services related to our RAP-219 Phase 2a clinical trial and planned open-label long term safety trial in RAP-219 for FOS, including, among other things, clinical trial readiness support, identification of potential patients satisfying the enrollment criteria and RNS system data reporting and data analysis. Pursuant to the payment schedule set out in the NeuroPace SOWs, we will pay NeuroPace an aggregate of up to $5.8 million over a period of approximately four years in connection with NeuroPace’s provision of services and achievement of certain patient enrollment and deliverable milestones. As of MarchJune 31,30, 2026, we have incurred cumulative expenses of $4.5$4.7 million on both NeuroPace SOWs.
We have not generated any revenue from the sale of products since inception and do not expect to generate any revenue from the sale of products for several years, if ever. As discussed above and in Note 11 - License and Collaboration Agreement to our condensed consolidated financial statements, we entered into the Tenacia License Agreement in March 2026. We recognized $20.0 million of collaboration revenue from the Tenacia License Agreement during the threesix months ended MarchJune 31,30, 2026.
Our primary focus since inception has been the development of RAP-219. Our research and development costs consist primarily of personnel-related costs and external costs, such as fees paid to Contract Manufacturing Organizations (“CMOs”), Contract Research Organizations (“CROs”) and consultants in connection with our non-clinical studies, preclinical studies and clinical trials. We expense all research and development costs in the periods in which they are incurred. Because we are working on multiple research and development programs at one time, we track many of our external expenses on a program-by-program basis. We do not allocate personnel-related costs or other indirect costs,costs to specific product development programs because these costs are deployed across multiple programs and, as such, are not separately classified.
Selling, General and Administrative Expenses
GeneralSelling, general and administrative expenses consist primarily of personnel-related costs, including salaries, bonuses, benefits, and stock-based compensation charges for those individuals in executive, finance, human resources, facility operations, and other administrative functions. Other costs include legal fees relating to intellectual property and corporate matters, professional fees for auditing, accounting, tax and consulting services, office and information technology costs, insurance costs, and facilities, depreciation and other selling, general and administrative expenses, which include direct or allocated expenses for rent and maintenance of facilities and utilities.
We anticipate that our selling, general and administrative expenses will increase for the foreseeable future to support development of product candidates and our continued research activities. These increases will likely include additional costs related to the hiring of additional personnel and fees paid to outside consultants, among other expenses. We also anticipate increased expenses related to audit, accounting, legal, regulatory, and tax-related services associated with maintaining compliance with The Nasdaq Global Market (“Nasdaq”) and SEC requirements, director and officer insurance premiums, and investor relations costs associated with operating as a public company.
For the three and six months ended MarchJune 31,30, 2026 and 2025, we recorded no income tax provision. As of MarchJune 31,30, 2026 and December 31, 2025, we recorded a full valuation allowance of our net deferred tax assets, as we believed it was more likely than not we would not be able to utilize our deferred tax assets prior to their expiration.
Comparison of the three months ended MarchJune 31,30, 2026 and 2025
The following table summarizes our results of operations for the three months ended MarchJune 31,30, 2026 and 2025:
Collaboration revenue was $20.0 million for the three months ended March 31, 2026 as a result of revenue recognized related to the non-refundable upfront fee received pursuant to the Tenacia License Agreement. Through March 31, 2026, no milestones have been achieved and no royalties have been earned.
Research and development expenses were $32.7 million for the three months ended March 31, 2026, as compared to $19.6 million for the three months ended March 31, 2025. The increase of $13.1 million consisted of the following:
a $7.9 million increase in RAP-219 program costs, which consisted primarily of an increase of $6.8 million in clinical trial costs primarily driven by start-up costs for our Phase 3 trials in drug-resistant FOS, costs for our Phase 2 proof-of-concept trial in bipolar mania, costs for IND-enabling activities for our Phase 1 trial for our LAI program, and a $1.0 million increase in contract manufacturing costs related to the production of clinical supply.
a $3.9 million increase in personnel-related costs due to an increase in headcount, which consisted primarily of salaries, bonuses, and other compensation-related costs of $2.4 million, and stock-based compensation of $1.5 million;
a $1.0 million increase in preclinical program costs, which consisted primarily of a $1.2 million increase in toxicology and animal studies related to our discovery programs, offset by a $0.2 million decrease in external chemistry efforts; and a $0.4 million increase in other costs, consisting of research and development facilities, information technology, and depreciation expense related to increased headcount and continued expansion of our Boston and San Diego sites.
General and Administrative Expenses
GeneralResearch and administrativedevelopment expenses were $11.5$51.4 million for the three months ended MarchJune 31,30, 2026, as compared to $7.5$22.7 million for the three months ended MarchJune 31,30, 2025. The increase of $4.0$28.7 million consisted of the following:
a $24.4 million increase in RAP-219 program costs, which consisted primarily of an increase of $22.3 million in clinical trial costs primarily driven by start-up costs for our Phase 3 trials in drug-resistant FOS, costs for our open-label long term safety trial for RAP-219 in FOS, costs for our Phase 2 proof-of-concept trial in bipolar mania, IND-enabling costs for our LAI program, a $1.7 million increase in contract manufacturing costs related to the production of clinical supply, a $0.3 million increase in RAP-219 consulting costs, and a $0.2 million increase in preclinical toxicology studies related to long-term toxicology work;
a $2.0$4.0 million increase in workforcepersonnel-related expensecosts due to an increase in headcount, consistingwhich consisted primarily of salaries, bonuses, and other compensation-related costs of $0.7$2.7 million and stock-based compensation of $1.4$1.3 million.million;
a $0.2 million increase in other costs, consisting of research and development facility costs and information technology costs related to increased headcount and continued expansion of our Boston and San Diego sites; and a $0.1 million increase in preclinical program costs, which consisted primarily of a $0.3 million increase in toxicology and animal studies related to our discovery programs, offset by a $0.2 million decrease in laboratory supplies.
Selling, General and Administrative Expenses
Selling, general and administrative expenses were $9.4 million for the three months ended June 30, 2026, as compared to $6.8 million for the three months ended June 30, 2025. The increase of $2.6 million consisted of the following:
a $2.0 million increase in workforce expense due to an increase in headcount, consisting primarily of salaries, bonuses, and other compensation-related costs of $0.5 million and stock-based compensation of $1.5 million; and a $0.5 million increase in professional and consulting costs primarily associated with increases of $0.3 million in consulting costs and $0.2 million in legal costs.
a $2.0 million increase in professional and consulting costs primarily associated with increases of $2.2 million in consulting costs and $0.3 million in legal costs primarily due to the execution of the Tenacia License Agreement. These increases were offset by a $0.4 million decrease in accounting consulting costs.
Other income was $4.4$4.2 million for the three months ended MarchJune 31,30, 2026, as compared to $3.0$2.8 million for the three months ended MarchJune 31,30, 2025. The $1.4 million increase inwas driven by increased interest income was due to the increased cash, cash equivalent and short-term investments balances for most of the three months ended MarchJune 31,30, 2026 due to proceeds from the September 2025 Offering.
For each of the three months ended MarchJune 31,30, 2026 and 2025, we recorded an income tax provision of zero.
Comparison of the six months ended June 30, 2026 and 2025
The following table summarizes our results of operations for the six months ended June 30, 2026 and 2025:
Collaboration revenue was $20.0 million for the six months ended June 30, 2026 as a result of revenue recognized related to the non-refundable upfront fee received pursuant to the Tenacia License Agreement. Through June 30, 2026, no milestones have been achieved and no royalties have been earned.
Operating Expenses
Research and Development Expenses
Research and development expenses were $84.1 million for the six months ended June 30, 2026, as compared to $42.3 million for the six months ended June 30, 2025. The increase of $41.9 million consisted of the following:
a $32.3 million increase in RAP-219 program costs, which consisted primarily of an increase of $29.1 million in clinical trial costs primarily driven by start-up costs for our Phase 3 trials in drug-resistant FOS, costs for our open-label long term safety trial for RAP-219 in FOS, costs for our Phase 2 proof-of-concept trial in bipolar mania, costs for IND-enabling activities for our LAI program, a $2.8 million increase in contract manufacturing costs related to the production of clinical supply, and a $0.3 million increase in consulting costs;
a $7.8 million increase in personnel-related costs due to an increase in headcount, which consisted primarily of salaries, bonuses, and other compensation-related costs of $5.1 million, and stock-based compensation of $2.8 million;
a $1.2 million increase in preclinical program costs, which consisted primarily of a $1.5 million increase in toxicology and animal studies related to our discovery programs, offset by a $0.2 million decrease in external chemistry efforts; and a $0.6 million increase in other costs, consisting of research and development facility costs and information technology costs related to increased headcount and continued expansion of our Boston and San Diego sites.
Selling, General and Administrative Expenses
Selling, general and administrative expenses were $20.9 million for the six months ended June 30, 2026, as compared to $14.4 million for the six months ended June 30, 2025. The increase of $6.5 million consisted of the following:
a $4.1 million increase in workforce expense due to an increase in headcount, consisting primarily of salaries, bonuses, and other compensation-related costs of $1.2 million and stock-based compensation of $3.0 million; and a $2.6 million increase in professional and consulting costs primarily associated with increases of $2.4 million in consulting costs and $0.5 million in legal costs primarily due to the execution of the Tenacia License Agreement, offset by a $0.4 million decrease in accounting consulting costs.
Other Income
Other income was $8.5 million for the six months ended June 30, 2026, as compared to $5.8 million for the six months ended June 30, 2025. The $2.7 million increase in interest income was due to the increased cash, cash equivalent and short-term investments balances for most of the six months ended June 30, 2026 due to proceeds from the September 2025 Offering.
Income Taxes
For each of the six months ended June 30, 2026 and 2025, we recorded an income tax provision of zero.
Since our inception in February 2022, weour haverevenue nothas been generated any revenue from anypayments sourcesreceived pursuant to collaboration agreements and we have incurred significant operating losses and negative cash flows from operations. We expect to incur significant expenses and operating losses for the foreseeable future as we advance the clinical development of our product candidates and pipeline. Further, we expect to continue to incur additional costs associated with operating as a public company. Historically, we have funded our operations with proceeds from the sale of convertible notes, convertible preferred stock, and the issuance of common stock in our IPO, concurrent private placement, September 2025 Offering, and collaboration agreements. To date, we have received aggregate gross proceeds of $731.9 million from these sources. As of MarchJune 31,30, 2026, we had cash andcash, cash equivalents and short-term investments of $476.8$436.1 million, excluding our restricted cash.
In March 2026, we filed an automatic shelf registration statement on Form S-3ASR (the “2026 Registration Statement”), containing (i) a base prospectus, which covers the offering, issuance and sale from time to time in one or more offerings of an indeterminate amount of common stock, preferred stock, debt securities, warrants and/or units; and (ii) a sales agreement prospectus for the offering and issuance and sale of up to a maximum aggregate offering price of $150.0 million of common stock, to be issued pursuant to the Sales Agreement. As of MarchJune 31,30, 2026, we have not sold any shares pursuant to our ATM program. As market conditions permit, we may offer and sell securities under the 2026 Registration Statement, including through the ATM Program, in order to fund our operations or provide additional liquidity.
During the threesix months ended MarchJune 31,30, 2026, operating activities used $13.1$54.5 million of cash, resulting primarily from our net loss of $19.9$76.5 million, non-cashpartially accretionoffset by $14.2 million of investmentsnon-cash instock-based marketablecompensation securities of $0.4 million, andexpense, changes in operating assets and liabilities of $0.8$6.0 million, partially offset by $7.0 million of non-cash stock-based compensation expense, $0.2$0.4 million of non-cash depreciation expense, and $0.6$1.2 million of non-cash lease expense. The $0.8$6.0 million change in operating assets and liabilities is primarily driven by a decrease in prepaid expenses and other current assets of $0.8 million, primarily due to recognition of advanced payments on contracts related to our clinical trials, a $0.7$1.4 million decrease in operating lease liabilities, and a deceasedecrease in other assets of $0.3 million due to the movement of an upfront long-term prepaid clinical trial payment from long-term to short term, and a decrease in accruedaccounts expensespayable of $3.5$1.4 million due to timing of payments to vendors, partially offset by increases in accountsaccrued payableexpenses of $2.3$11.1 million due to financialincreased advisoryclinical feestrial costs, an increase in prepaid expenses and other current assets of $2.3 million, primarily due into connectionrecognition withof advanced payments on contracts related to our clinical trials, and an increase to accounts receivable of $0.3 million for reimbursable costs related to the Tenacia License Agreement.
During the threesix months ended MarchJune 31,30, 2025, operating activities used $20.2$45.3 million of cash, resulting primarily from our net loss of $24.1$50.8 million, non-cash accretion of investments in marketable securities of $0.4$0.5 million, and changes in operating assets and liabilities of $0.4$3.8 million, partially offset by $4.0$8.4 million of non-cash stock-based compensation expense, $0.2$0.5 million of non-cash depreciation expense, and $0.4$0.9 million of non-cash lease expense. The $0.4$3.8 million change in operating assets and liabilities is primarily driven by an increase in prepaid expenses and other current assets of $0.8$5.1 million, primarily due to advanced payments on new contracts related to our clinical trials, a $0.1 million decrease in operating lease liabilities, partially offset by an increaseincreases in accounts payable of $0.2$1.2 million and accrued expenses of $0.3$0.8 million due to timing of payments to vendors.vendors, and an increase in other assets of $0.5 million primarily due to the payment of the security deposit for our new corporate headquarters lease.
During the threesix months ended MarchJune 31,30, 2026, net cash provided by investing activities was $37.9$63.5 million, primarily consisting of maturities of short-term investments of $58.3$123.5 million and sales of short-term investments of $10.1 million, partially offset by purchases of short-term investments of $30.4 million and purchases of property and equipment of $0.1$70.0 million.
During the threesix months ended MarchJune 31,30, 2025, net cash provided by investing activities was $21.0$43.6 million, primarily consisting of maturities of short-term investments of $64.6$100.0 million, partially offset by purchases of short-term investments of $43.2$56.1 million and purchases of property and equipment of $0.3 million.
During the threesix months ended MarchJune 31,30, 2026, net cash provided by financing activities was $0.5$1.9 million, primarily consisting of proceeds from exercise of stock options of $0.8$2.4 million, partially offset by payment of deferred offering costs of $0.3$0.5 million in connection with the preparation and filing of the 2026 Registration Statement.
During the six months ended June 30, 2025, net cash used in financing activities was $0.1 million, primarily consisting of deferred offering costs in connection with the preparation and filing of the Registration Statement and the ATM Program.
During the three months ended March 31, 2025, net cash provided by financing activities was immaterial.
As of MarchJune 31,30, 2026, we had cash, cash equivalents and short-term investments of $476.8$436.1 million, excluding our restricted cash. As of the issuance date of the condensed consolidated financial statements for the threesix months ended MarchJune 31,30, 2026, we expect that our cash, cash equivalents and short-term investments will be sufficient to fund our operating expenses and capital expenditure requirements through at least 12 months from the issuance of the condensed consolidated financial statements. We believe that our existing cash, cash equivalents and short-term investments will enable us to fund our operating expenses and capital expenditure requirements into the second half of 2029. We have based this estimate on assumptions that may prove to be wrong, and we could exhaust our available capital resources sooner than we expect. However, our forecast for the period of time through which our financial resources will be adequate to support our operations is a forward-looking statement that involves risks and uncertainties, and actual results could vary materially. Additionally, the process of conducting preclinical studies and testing potential future product candidates in clinical trials is costly, and the timing of progress and expenses in these studies and trials is uncertain. We will need to raise substantial additional capital in the future.
As of the MarchJune 31,30, 2026, we had future minimum operating lease payments under non-cancelable leases of $12.5$11.6 million related to leases we have recognized on our condensed consolidated balance sheet, which included existing laboratory and office leases and the new corporate headquarters lease that commenced June 1, 2025 in Boston, Massachusetts. The future minimum operating lease payments under these non-cancelable leases are due over a weighted average remaining lease term of 4.44.2 years.
In connection with the execution of the NeuroPace Agreement, the parties also entered into an initial statement of work, as amended in March 2024, and a second statement of work, as amended in February 2026, (collectively, the “NeuroPace SOWs”) under the NeuroPace Agreement, pursuant to which NeuroPace agreed to provide services related to the RAP-219 Phase 2a proof-of-concept clinical trial and planned open-label long term safety trial in RAP-219 for FOS, including, among other things, clinical trial readiness support, identification of potential patients satisfying the enrollment criteria and RNS system data reporting and data analysis. Pursuant to the payment schedule set out in the NeuroPace SOWs, the Company will pay NeuroPace an aggregate of up to $5.8 million over a period of approximately four years in connection with NeuroPace’s provision of services and achievement of certain patient enrollment and deliverable milestones. The Company has incurred cumulative expenses of $4.5$4.7 million on the NeuroPace SOWs through MarchJune 31,30, 2026.
For information regarding our critical accounting policies and estimates, please refer to Note 2,2 — “Summary of Significant Accounting Policies” contained in our Annual Report on Form 10-K for the fiscal year ended December 31, 2025.Report. The critical accounting policy below supplements the critical accounting policies discussed in our Annual Report on Form 10-K filed with the SEC on March 10, 2026.Report.
RAPP insider buying and selling (Form 4)
Since 2026-04-11, insiders reported open-market purchases in 0 Form 4 filings and open-market sales in 17 filings (5 insiders, 20 trade dates, 1,064,842 shares, about $43.8M; 17 of these filings say the sales were made under a Rule 10b5-1 trading plan). Net open-market shares: -1,064,842 (purchases minus sales); net value about -$43.8M.Totals add up every open-market (code P and S) line in those filings, using the prices reported in the filings. Awards, option exercises, tax withholding and gifts are listed below but not counted.
| Trade date | Insider | Transaction | Shares | Price | Value |
|---|---|---|---|---|---|
| 2026-09-30 | Gault Cheryl |
Open-market sale |
7,925 | $33.04 | $261.8K |
| 2026-09-30 | Gault Cheryl |
Open-market sale |
2,075 | $33.76 | $70.0K |
| 2026-09-23 | Ceesay Abraham |
Open-market sale |
43 | $41.05 | $1.8K |
| 2026-09-23 | Ceesay Abraham |
Open-market sale |
1,952 | $39.05 | $76.2K |
| 2026-09-23 | Ceesay Abraham |
Open-market sale |
215 | $39.86 | $8.6K |
| 2026-09-08 | Ceesay Abraham |
Open-market sale |
7,141 | $45.56 | $325.3K |
| 2026-09-08 | Ceesay Abraham |
Open-market sale |
1,069 | $46.01 | $49.2K |
| 2026-08-25 | Ceesay Abraham |
Open-market sale |
18,921 | $49.57 | $937.9K |
| 2026-08-25 | Yeleswaram Krishnaswamy |
Open-market sale |
16,201 | $51.06 | $827.2K |
| 2026-08-25 | Yeleswaram Krishnaswamy |
Open-market sale |
20,799 | $50.31 | $1.0M |
| 2026-08-24 | Yeleswaram Krishnaswamy |
Open-market sale |
13,160 | $50.00 | $658.0K |
| 2026-08-19 | Yeleswaram Krishnaswamy |
Open-market sale |
404 | $49.90 | $20.2K |
| 2026-08-11 | Ceesay Abraham |
Open-market sale |
3,664 | $46.90 | $171.8K |
| 2026-08-11 | Ceesay Abraham |
Open-market sale |
19,500 | $48.04 | $936.8K |
| 2026-08-11 | Ceesay Abraham |
Open-market sale |
5,794 | $48.62 | $281.7K |
| 2026-08-10 | Ceesay Abraham |
Open-market sale |
14,171 | $45.97 | $651.4K |
| 2026-08-10 | Ceesay Abraham |
Open-market sale |
8,370 | $46.75 | $391.3K |
| 2026-07-27 | Ceesay Abraham |
Open-market sale |
2,203 | $42.11 | $92.8K |
| 2026-07-27 | Ceesay Abraham |
Open-market sale |
7 | $42.74 | $299 |
| 2026-07-14 | Ceesay Abraham |
Open-market sale |
2,729 | $40.57 | $110.7K |
| 2026-07-14 | Ceesay Abraham |
Open-market sale |
5,481 | $39.72 | $217.7K |
| 2026-06-30 | Gault Cheryl |
Open-market sale |
1,000 | $43.15 | $43.1K |
| 2026-06-30 | Gault Cheryl |
Open-market sale |
3,308 | $42.17 | $139.5K |
| 2026-06-30 | Gault Cheryl |
Open-market sale |
1,900 | $40.09 | $76.2K |
| 2026-06-30 | Gault Cheryl |
Open-market sale |
3,792 | $41.39 | $157.0K |
| 2026-06-29 | Ceesay Abraham |
Open-market sale |
876 | $40.34 | $35.3K |
| 2026-06-29 | Ceesay Abraham |
Open-market sale |
7,344 | $39.77 | $292.1K |
| 2026-05-26 | Third Rock Ventures V, L.p. |
Open-market sale |
18,572 | $40.05 | $743.8K |
| 2026-05-11 | Third Rock Ventures V, L.p. |
Open-market sale |
133,618 | $40.24 | $5.4M |
| 2026-05-08 | Third Rock Ventures Vi, L.p. |
Open-market sale |
5,018 | $40.31 | $202.3K |
| 2026-05-07 | Third Rock Ventures Vi, L.p. |
Open-market sale |
271,495 | $40.12 | $10.9M |
| 2026-04-17 | Third Rock Ventures Vi, L.p. |
Open-market sale |
426,005 | $40.32 | $17.2M |
| 2026-04-17 | Yeleswaram Krishnaswamy |
Open-market sale |
20,225 | $39.99 | $808.8K |
| 2026-04-14 | Yeleswaram Krishnaswamy |
Open-market sale |
10,200 | $35.08 | $357.8K |
| 2026-04-13 | Yeleswaram Krishnaswamy |
Open-market sale |
9,165 | $34.97 | $320.5K |
| 2026-04-10 | Yeleswaram Krishnaswamy |
Open-market sale |
500 | $34.98 | $17.5K |
Well-known investors holding RAPP (13F)
| Investor | Quarter | Shares | Reported value | % of their 13F | Change vs prior quarter |
|---|---|---|---|---|---|
| D. E. Shaw & Co. | 2026-06-30 | 214,831 | $9.0M | 0.01% | Added 22% |
| Millennium Management (Israel Englander) | 2026-06-30 | 154,908 | $6.5M | 0.0% | Reduced 52% |
| Citadel Advisors (Ken Griffin) | 2026-06-30 | 82,442 | $3.4M | 0.0% | New position |
| AQR Capital Management (Cliff Asness) | 2026-06-30 | 26,454 | $1.1M | 0.0% | Added 4% |
| Two Sigma Investments | 2026-06-30 | 24,470 | $1.0M | 0.0% | Reduced 13% |
| Renaissance Technologies | 2026-06-30 | 9,700 | $303.5K | — | Sold out |