MAZE 10-K & 10-Q changes, risk factors and insider trading

Investing in our common stock involves a high degree of risk. Before making your decision to invest in shares of our common stock, you should carefully consider the risks described below, together with the other information contained in this Annual Report on Form 10-K, including in the sections titled “Special note regarding forward-looking statements” and “Management’s discussion and analysis of financial condition and results of operations” and in our financial statements and the related notes included elsewhere in this Annual Report on Form 10-K. The risks and uncertainties described below are not the only ones we face. These disclosures reflect the Company’s beliefs and opinions as to factors that could materially and adversely affect the Company and its securities in the future. References to past events are provided by way of example only and are not intended to be a complete listing or a representation as to whether or not such factors have occurred in the past or their likelihood of occurring in the future. Additional risks and uncertainties that we are unaware of or that we deem immaterial may also become important factors that adversely affect our business. We cannot assure you that any of the events discussed below will not occur. These events could have a material and adverse impact on our business, financial condition, results of operations and prospects. If that were to happen, the trading price of our common stock could decline, and you could lose all or part of your investment.

Investment in drug development is a highly speculative undertaking and involves a substantial degree of risk. We are a clinical-stage biopharmaceutical company with a limited operating history. Our lead programs are still in early clinical and preclinical development, and we are subject to the risks of failure inherent in the development of therapeutic candidates based on novel technologies. We have not yet demonstrated an ability to successfully overcome many of the risks and uncertainties frequently encountered by companies in new and rapidly evolving fields, particularly in the biotechnology industry. Furthermore, we do not expect to generate any product revenue from commercial sales for the foreseeable future, and we expect to continue to incur significant operating losses for the foreseeable future due to the cost of clinical trials, preclinical studies, research and development, and the regulatory approval process of our therapeutic candidates. For the years ended December 31, 20242025 and 2023,2024, we incurred net income of $52.2 million and a net loss of $100.4$131.1 million and net income of $52.2 million, respectively. As of December 31, 2024,2025, we had an accumulated deficit of $358.4$489.5 million. We expect to continue to incur significant research and development and other expenses related to our ongoing operations.

Since our inception, we have focused substantially all of our efforts and financial resources on research and development activities for our programs and on establishing arrangements and collaborations with third parties for the development of our therapeutic candidates. To date, we have not generated any product revenue from product sales and have financed our operations primarily through sales of our equity and convertible promissory notes, debt financing, as well as one-time, nonrefundable, upfront licensing payments.

Our prior losses, combined with expected future losses, have had, and will continue to have, an adverse effect on our stockholders’ equity and working capital. We expect our research and development expenses to significantly increase in connection with the ongoing clinical trials for our lead programs, MZE829 and MZE782, further development of our Maze Compass platform, or our Compass platform, planned preclinical studies, IND filings and clinical trials for future therapeutic candidates. We will also incur substantial additional expenses as we seek to expand our intellectual property portfolio, including through potential in-licensing opportunities, and hire additional personnel as we scale up our operations. We expect to continue to incur additional costs associated with operating as a public company. As a result, we expect to continue to incur significant and increasing operating losses for the foreseeable future. Because of the numerous risks and uncertainties associated with developing pharmaceutical products, we are unable to predict the extent of any future losses or when we will become profitable, if at all.

Developing pharmaceutical products, including conducting clinical trials and preclinical studies, is a very time-consuming, expensive and uncertain process that takes years to complete. Our operations have consumed substantial amounts of cash since inception, and we expect our expenses to increase substantially in connection with our ongoing research and development activities, particularly as we advance our developmental programs, advance our current clinical trial and preclinical studies, commence additional clinical trials and preclinical studies and expand the breadth of our Compass platform. In addition, even if we obtain marketing approval for any of our therapeutic candidates, we expect to incur significant commercialization expenses related to product manufacturing, marketing, sales and distribution. We expect to continue to incur additional costs associated with operating as a public company. Accordingly, we will need to obtain substantial additional capital in connection with our continuing operations. Adequate additional financing may not be available to us on favorable terms, or at all. In addition, 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. If we are unable to raise capital when needed or on favorable terms, we could be forced to delay, reduce or eliminate our clinical and preclinical development, research and development programs, our commercialization plans or other operations.

As of December 31, 2024,2025, our cash, cash equivalents, and cashmarketable equivalentssecurities were $196.8$360.0 million. Based on our current operating plan, we believe our existing cash andcash, cash equivalents and marketable securities as of December 31, 2024, together with the net proceeds of approximately $127.8 million from our initial public offering completed in February 2025,2025 will enable us to fund our operating expenses and capital expenditure requirements for at least one year from the date of this Annual Report on Form 10-K. We have based this estimate on assumptions that may prove to be wrong, and we could use our capital resources sooner than we currently expect. Changes beyond our control may occur that would cause us to use our available capital before that time, including changes in and progress of our development activities and changes in regulation. Our future capital requirements will depend on many factors, including, but not limited to:

Raising additional capital may cause dilution to our stockholders, cause the price of our common stock to decline, restrict our operations or require us to relinquish rights to our technologies or therapeutic candidates.

Until such time, if ever, as we can generate substantial product revenue, we expect to finance our cash needs through a combination of equity offerings, debt financings, collaborations, strategic alliances and marketing, distribution or licensing arrangements. In addition, weWe 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. To the extent that we raise additional capital through the sale of equity or convertible debt securities, your ownership interest will be diluted, and the terms of these securities may include liquidation or other preferences that adversely affect your rights as a common stockholder. Debt financing and preferred 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 acquisitions or capital expenditures or declaring dividends.

If we raise additional funds through collaborations, strategic alliances or marketing, distribution or licensing arrangements with third parties, we may have to relinquish valuable rights to our technologies, future revenue streams, research programs or therapeutic candidates, including granting licenses to our technologies, on terms that may not be favorable to us. If we are unable to raise additional funds through equity or debt financings or other arrangements when needed, we may be required to delay, limit, reduce or terminate our research, product development or future commercialization efforts or grant rights to third parties to develop and market therapeutic candidates that we would otherwise prefer to develop and market ourselves.

Our therapeutic candidates are in early stages of development. We do not have any products that are approved for sale in any jurisdiction. We initiated a Phase 2 trial of MZE829, our most advanced lead program, in November 2024, dosed our first patient in February 2025 and expectreported topositive reporttopline clinical proof of concept data in the first quarter ofMarch 2026. We initiated a Phase 1 clinical trial of our second lead program, MZE782, in September 2024.2024 and announced initial clinical data in phenylketonuria, or PKU, and chronic kidney disease, or CKD, in September 2025. We plan to initiate two Phase 2 trials of MZE782 in PKU and CKD in 2026. Our other programs are at an early research stage, with no therapeutic candidates identified. Our research programs may not result in viable therapeutic candidates for some time, if ever, and our therapeutic candidates in development may not achieve success in our current or any future clinical trials, or obtain regulatory approval.

addressing any delays in our clinical trials, or in the manufacture or distribution of our therapeutic candidates, if approved, resulting from any major natural disasters, health pandemicspandemics, economic conditions or significant political events; and maintaining a continued acceptable safety profile of the products following approval.

We have limited experience designing and implementing clinical trials. We initiated a Phase 2 trial of MZE829, our most advanced lead program, in November 2024, dosed our first patient in February 2025 and expectreported topositive reporttopline clinical proof of concept data in the first quarter ofMarch 2026. We initiated a Phase 1 clinical trial of MZE782 in September 2024.2024 and announced initial clinical data in PKU and CKD in September 2025. We plan to initiate two Phase 2 trials of MZE782 in PKU and CKD in 2026. Failure to design any one of our clinical trials adequately, or incorrect assumptions about the design of the trial, could adversely affect the ability to initiate the trial, enroll patients, complete the trial, or use the clinical trial’s results to support an application for regulatory approval, as well as lead to increased or unexpected costs.

We may experience difficulties in patient enrollment in our clinical trials for a variety of external reasons beyond our control, including supply chain disruptions, staffing shortages and other business and economic disruptions resulting from geopolitical actions, including war and terrorism, natural disasters, including earthquakes, typhoons, floods and fires, as well as other disruptions resulting from the impact of public health factors, including pandemics or other health crises, business disruptions of our strategic partners, third-party manufacturers, suppliers and other third parties upon which we rely. Our therapeutic candidates are developed using a precision medicine approach intended to identify specific genetic targets amenable to therapeutic intervention in CVRMkidney and metabolic diseases. As a result, the potential population of patients eligible for enrollment in our clinical trials will be smaller than the population eligible for enrollment in trials targeting the entire population with the disease, and it may be difficult to identify and enroll adequate numbers of patients in our trials. For example, our therapeutic candidate MZE829 is targeted to treat kidney disease associated with genetic variations in the APOL1 gene, which are most prevalent in people of West African ancestry, including many who identify as Black, African American, Afro-Caribbean and Latina/Latino, which narrows the patient population eligible for enrollment in these trials.

In addition, the eligibility criteria of our clinical trials will further limit the pool of available trial participants. For our therapeutic candidate MZE829, we depend on and expect to perform genetic screening in order to identify candidates with the applicable genetic variations for our clinical trials. As we expect future therapeutic candidates to also target specific genetic subgroups of common conditions, as identified through our Compass platform, it is likely that future therapeutic candidates will also require identification of patients with the applicable genotype,genotype or other specific disease biomarkers, which will require genetic and/or specific clinical tests for screening and further costs and timing delays in identifying patients eligible for our clinical trials. GeneticSuch screening could involve more time consuming, costly and invasive procedures than patients may be willing to accept. If patients are not willing to undergo genetic screening, or any other eligibility criteria we have for our clinical trials, it would lead to greater costs and/or timing delays for us in order to identify eligible and the necessary patients for our clinical trials. The timely completion of clinical trials in accordance with their protocols depends, among other things, on our ability to enroll a sufficient number of eligible patients who remain in the trial until completion of treatment and adequate follow-up. In general, the enrollment and retention of patients depends on many factors, including, but not limited to:

reporting of the preliminary results of any of our clinical trials; and potential approval and commercial availability of a competitor’s product for the same indication.

In addition, our clinical trials will compete with other clinical trials for therapeutic candidates that are in the same therapeutic areas as our therapeutic candidates, and this competition will reduce the number and types of patients available to or retained by us because patients who might have opted to enroll in our trials may instead opt to enroll in a trial being conducted by one or more of our competitors. Other biopharmaceutical companies are currently conducting clinical trials, and may in the future conduct clinical trials, that compete for the same patient population as us. Since the number of qualified clinical investigation sites is limited, and often associated with a small number of academic centers, we expect to conduct some of our clinical trials at the same clinical trial sites that some of our competitors use, which will reduce the number of patients who are available for our clinical trials at such clinical trial sites. Unanticipated geopolitical events in countries where we may seek to have clinical trial sites could also negatively impact our ability to enroll or retain patients in our trials.

Our two lead programs are intended to treat chronic kidney disease, or CKD, which is a progressive condition associated with severe and often life-threatening co-morbidities. Clinical trials that involve patients with significant co-morbidities are associated with increased risks as such participants may be particularly susceptible to safety and toxicity risks. In addition, these side effects may not be appropriately recognized or managed by the treating medical staff, as safety and toxicity monitoring may be complicated and difficult to manage, which could result in patient death or other significant issues. Additionally, it can be difficult to determine if the serious adverse or unexpected side effects were caused by our therapeutic candidate or anothersome other factor, especially in subjects who may suffer from other medical conditions and take other medications.

any therapeutic candidates we develop may nevertheless be covered by third parties’ patents or other exclusive rights;

We rely on non-exclusive access to third-party data sources of genetic and paired clinical data for our Compass platform, including, among others, the University of Helsinki, Queen Mary University, the UK Biobank, Biobank Japan and the Million Veteran Program, as well as specialized databases such as the Chronic Renal Insufficiency CohortCohort, which is managed by the National Institute for Diabetic, Digestive and Kidney DiseasesDiseases, and other genetic data sets we have acquired or may acquire access to in the future. Our ability to access data from these and similar sources is vital to all phases of our Compass platform, including target identification and variant functionalization.

Our Compass platform has identified, and may identify in the future, genetic targets and therapeutic candidates outside of our core area of focus in CVRMkidney and metabolic diseases. Our business strategy includes seeking strategic collaborations to advance these targets. For example, in 2024, we granted exclusive licenses to advance two programs related to targets we were pursuing in amyotrophic lateral sclerosis, or ALS— ATXN2 and UNC13A—to other biotechnology companies, as well as an exclusive license with a pharmaceutical partner, Shionogi & Co., Ltd., or Shionogi, to advance our first clinical stage therapeutic candidate, MZE001.

Part of our business model includes creating business partnerships and new corporate entities to develop and potentially commercialize therapeutic targets identified through our Compass platform that are outside of our core area of expertise. For example, in 2020, we formed Broadwing Bio LLC, a spin-out company with Alloy Therapeutics, Inc., or Alloy, to develop antibody therapies for ANGPTL7 and another undisclosed target in ophthalmic diseases. Going forward, we may create or acquire interests in more joint venture enterprises, spin-outs, and other entities to execute our business strategy. These business collaborations involve risks that our partners may:

We rely on third parties to manufacture our clinical product supplies and therapeutic candidates and we may not be able to obtain adequate supplies at a reasonable cost or in a timely way.manner.

Further, we rely on third parties located in China for some of our contract manufacturing, and we expect to continue to use such third-party manufacturers for such purposes. For any activities conducted in China, we are exposed to the possibility of product supply disruption and increased costs in the event of changes in the policies of the United States or Chinese governments, political unrest or unstable economic conditions in China. In addition, certain Chinese biotechnology companies may become subject to trade restrictions, sanctions, other regulatory requirements, or proposed legislation by the U.S. government, which could restrict or even prohibit our ability to work with such entities, thereby potentially disrupting the supply of material to us. For example, the BIOSECURE ActAct, thata version of which was passed byin the U.S. House of Representatives, asbut wellwas as a substantially similar bill proposed in the U.S. Senate, target U.S. government contracts, grants and loans for entities that use equipment and services from certain named Chinese biotechnology companies. Given the current uncertain political and legislative environment, it is unclear what form the BIOSECURE Act will take and whether or when it will be enacted into law. If enacted asnot passed by the HouseU.S. Senate in 2024, and a revised version of Representatives,which was recently passed by the BIOSECUREU.S. ActSenate in 2025, but will be subject to the reconciliation process, would, among other things, prohibit U.S. federal agencies from entering into or renewing any contract with any entity that uses biotechnology equipment or services produced or provided by a “biotechnology company of concern” to perform that contract.contract as well as authorize the U.S. government to name additional Chinese “biotechnology companies of concern,” subject to certain “grandfathering” provisions that provide that the BIOSECURE Act’s prohibitions will not apply for five years to pre-existing contracts and agreements entered into prior to the legislation’s effective date. The BIOSECURE Act defineshas in the past and may in the future define a “biotechnology company of concern” to include WuXi Apptec and its affiliates, collectively,or WuXi,WuXi. andAlthough providesthe thatBIOSECURE additionalAct companieshas not yet become law, it or a substantially similar bill may be designated as “biotechnology companies of concern.” The version of the bill passed byor theproposed House of Representatives includes a “grandfathering” provision that provides that the BIOSECURE Act’s prohibitions will not apply to pre-existing contracts and agreements entered into prior to the legislation’s effective date until 2032.again. We are currently parties to agreements with WuXi, pursuant to which WuXi provides development and manufacturing services to us. If these bills become law, or similar laws are passed, we may be restricted in our ability to work with WuXi and other Chinese biotechnology manufacturing companies to the extent we would contract with, or otherwise receive funding from, the U.S. government. As a result, we may need to seek alternative CMO relationships, but we expect that the grandfathering provision will provide adequate time to identify and execute agreements with alternative CMOs. While we believe we will be able to identify and contract with such alternative CMOs, we cannot predict the terms of any such alternative arrangement nor what actions may ultimately be taken with respect to trade relations between the United States and China or other countries, what products and services may be subject to such actions or what actions may be taken by China or the other countries in retaliation. In addition to the BIOSECURE ACT, any unfavorable government policies on international trade, such as export controls, capital controls or tariffs, new legislation or regulations, renegotiation of existing trade agreements, or any retaliatory trade actions due to recent or future trade tension, may impede, delay, limit, or increase the cost of manufacturing our therapeutic candidates. Such events could result in our clinical or commercial supply of drug, packaging and other services being interrupted or limited, which could harm our business.

In order to market and sell our products in any jurisdiction outside the United States, we must obtain separate marketing approvals and comply with numerous and varying regulatory requirements. The approval procedure varies amongfrom countriescountry to country and canmay involve additional testing. The time required to obtain approval from foreign regulatory authorities may differ substantially from that required to obtain FDA approval. The regulatory approval process outside the United States generally includes all of the risks associated with obtaining FDA approval. In addition, in many countries outside the United States, it is required that the product be approved for reimbursement before such product can be approved for sale in that country. We may not obtain approvals from regulatory authorities outside the United States on a timely basis, if at all. Approval by the FDA does not ensure approval by regulatory authorities in other countries or jurisdictions, and approval by one foreign regulatory authority outside the United States does not ensure approval by regulatory authorities in other countries or jurisdictions or by the FDA. We may not be able to submit for marketing approvals and may not receive necessary approvals to commercialize our products in any market.

Any therapeutic candidate for which we, or any of our partners obtain marketing approval, as well as the manufacturing processes, post-approval clinical data, labeling, advertising and promotional activities for such product, will be subject to continual requirements of and review by the FDA and other regulatory authorities. These requirements include, but are not limited to, restrictions governing promotion of an approved product, submissions of safety and other post-marketing information and reports, registration and listing requirements, cGMP requirements relating to manufacturing, quality control, quality assurance and corresponding maintenance of records and documents, and requirements regarding drug distribution and the distribution of samples to physicians and recordkeeping.

Several healthcare reform initiatives culminated in the enactment of the Inflation Reduction Act in August 2022, which allows, among other things, the Department of Health and Human Services, or HHS, to negotiate the selling price of a statutorily specified number of drugs and biologics each year that the Centers for Medicare & Medicaid Services, or CMS, reimburses under Medicare Part B and Part D. The negotiated price may not exceed a statutory ceiling price. Only high-expenditure single-source drugs that have been approved for at least 7 years (11 years for single-source biologics) can qualify for negotiation, with the negotiated price taking effect two years after the selection year. For 2026, the first year in which negotiated prices become effective, CMS selected 10 high-cost Medicare Part D drugs in 2023, negotiations began in 2024, and the negotiated maximum fair price for each drug has been announced. CMS has selected 15 additional Medicare Part D drugs for negotiated maximum fair pricing in 2027. For 2028, up to an additional 15 drugs, which may be covered under either Medicare Part B or Part D, will be selected, and for 2029 and subsequent years, up to 20 additional Part B or Part D drugs will be selected. A drug or biological product that has an orphan drug designation for only one rare disease or condition will be excluded from the Inflation Reduction Act’s price negotiation requirements, but loses that exclusion if it has designations for more than one rare disease or condition, or if is approved for an indication that is not within that single designated rare disease or condition, unless such additional designation or such disqualifying approvals are withdrawn by the time CMS evaluates the drug for selection for negotiation. The Inflation Reduction Act also imposes rebates on Medicare Part B and Part D drugs whose prices have increased at a rate greater than the rate of inflation. In addition, the law eliminateseliminated the “donut hole” under Medicare Part D beginning in 2025 by significantly lowering the beneficiary maximum out-of-pocket cost and establishing a new manufacturer discount program, which requires manufacturers, in order for their drugs to be covered by Medicare Part D, to provide statutorily defined discounts on their brand (NDA) drugs dispensed to Part D enrollees. The Inflation Reduction Act also extends premium enhanced subsidies for lower income individuals purchasing health insurance coverage in ACA marketplaces through plan year 2025.2025, but those subsidies are set to expire at the end of 2025 unless they are extended by Congress. The Inflation Reduction Act permits the Secretary of HHS to implement many of these provisions through guidance, as opposed to regulation, for the initial years. Manufacturers that fail to comply with the Inflation Reduction Act may be subject to various penalties, some significant, including civil monetary penalties. These provisions arehave begun taking effect progressively starting in 2023, although they may be subject to legal challenges. For example, the provisions related to the negotiation of selling prices of high-expenditure single-source drugs and biologics have been challenged in multiple lawsuits. Thus, it is unclear how the Inflation Reduction Act will be implemented but it will likely have a significant impact on the pharmaceutical industry and the pricing of our products and therapeutic candidates. The One Big Beautiful Bill Act, or OBBBA, which was recently signed into law, reduces funding to federal healthcare programs and imposes additional requirements to be eligible for healthcare, which may result in decreased access to healthcare, particularly for Medicaid programs. The adoption of restrictive price controls in new jurisdictions, more restrictive controls in existing jurisdictions or the failure to obtain or maintain timely or adequate pricing could also adversely impact revenue. We expect pricing pressures will continue globally.

Disruptions at the FDA and other government agencies caused byby, among other factors, funding shortages or global health concerns could hinder their ability to hire, retain, or deploy key leadership and other personnel, or otherwise prevent new or modified products from being developed, approved, or commercialized in a timely manner or at all, or otherwise prevent those agencies from performing normal business functions on which the operation of our business may rely, which could negatively impact our business. In addition, there is substantial uncertainty regarding new regulatory initiatives and how these might impact the FDA, its implementation of laws, regulations, policies and guidance and its personnel. Similar initiatives may also be directed toward other government agencies. These initiatives could prevent, limit or delay development and regulatory approval of our product candidates, which would adversely affect our business.

The ability of the FDA to review and approve new products can be affected by a variety of factors, including government budget and funding levels, government shutdowns, statutory, regulatory, and policy changes, the FDA’s ability to hire and retain key personnel and accept the payment of user fees, and other events that may otherwise affect the FDA’s ability to perform routine functions. 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. Disruptions at the FDA and other agencies may also slow the time necessary for new therapeutic candidates to be reviewed and/or approved by necessary government agencies, which may adversely affect our business, financial condition, results of operations and prospects. For example, over the last several years, the U.S. government has shut down several times and certain regulatory agencies, such as the FDA, have had to furlough critical FDA employees and stop critical activities.

Failure to comply with laws governing international business practices may result in substantial civil and criminal penalties and suspension or debarment from government contracting. The Securities and Exchange Commission, or the SEC,SEC also may suspend or bar issuers from trading securities on U.S. exchanges for violations of the FCPA’sFCPA accounting provisions.

While there are currently no approved therapies for APOL1APOL1-mediated kidney disease, or AKD,AMKD, we are aware of companies advancing therapeutic candidates in clinical trials that target APOL1.

Our second most advanced lead program, MZE782, is a small molecule targeting SLC6A19, a novel target in CKD and PKU. While there are no currently approved therapies directly modulating SLC6A19, we may face competition from other companies pursuing programs targeting SLC6A19 or alternative approaches for renalkidney and metabolic indications.

Even if our Compass platform performs its intended functions, we may be unable to use the discoveries resulting from the platform to produce new therapies. Despite recent scientific advances in the life sciences and our improved understanding of biology, the roles of genes and proteins and their involvement in diseases and in other life processes is not well understood. Therefore, notwithstanding any potential promising results in earlier studies or clinical trials, we may face significant setbacks in current or future studies or clinical trials. If we are unable to use our platform discoveries platform to develop and market new drugs or therapies, our business may fail or we may never become profitable.

If we are unable to establish our own sales and marketing capabilities,capabilities and must rely on third parties to market and sell our products, our revenue from product sales and our profitability, if any, are likely to be lower than if we ourselves were to market and sell any products that we develop. In addition, we may not be successful in entering into arrangements with third parties to market and sell our therapeutic candidates or may be unable to do so on terms that are acceptable to us. Any of these third parties may fail to devote the necessary resources and attention to sell and market our products effectively. If we do not establish sales and marketing capabilities successfully, either on our own or in collaboration with third parties, we will not be successful in commercializing our therapeutic candidates.

OurFrom estimatedtime to time we may provide estimates of addressable markets and market opportunities for our therapeutic candidatescandidates, which are based on a variety of inputs, including data published by third parties, our own market insights and internal market intelligence, and internally generated data and assumptions. We have not independently verified any third-party information and cannot be assured of its accuracy or completeness. Market opportunity estimates, whether obtained or derived from third-party sources or developed internally, are subject to significant uncertainty and are based on assumptions and estimates that may prove not to be accurate, particularly given the relatively early stage of our therapeutic candidates. Although we believe our market opportunity estimates are reasonable, such information is inherently imprecise. In addition, our assumptions and estimates of market opportunities are necessarily subject to a high degree of uncertainty and risk due to a variety of factors, including but not limited to those described in this Annual Report on Form 10-K. If this third-party or internally generated data prove to be inaccurate or if we make errors in our assumptions based on that data, or if the facts underlying our assumptions change over time, our actual market may be more limited than we estimate it to be. In addition, these inaccuracies or errors may cause us to misallocate capital and other critical business resources, which could harm our business. TheOur estimates of our market opportunities included in this Annual Report on Form 10-K should not be taken as indicative of our ability to grow our business.

Moreover, we might not be able to attract or retain qualified management and other key personnel in the future due to the intense competition for qualified personnel among biotechnology, pharmaceutical and other businesses, particularly in the San Francisco Bay AreaArea, where we are headquartered, and we may be required to expend significant financial resources in our employee recruitment and retention efforts. Many pharmaceutical companies with whom we compete for qualified personnel have greater financial and other resources, different risk profiles and longer operating histories in the industry than we do. They also may provide candidates with more diverse opportunities and better chances for career advancement. If we are not able to attract and retain the necessary personnel to accomplish our business objectives, including personnel with clinical development expertise, we may experience constraints that will harm our ability to implement our business strategy and achieve our business objectives.

InWe addition,also werely haveon scientificscientific, clinical, and clinicalother technical advisors and consultants whoto assist us in formulatingwith our clinical development programs. Our future success will depend in part on our ability to engage such advisors and clinicalconsultants strategies.with Thesethe necessary expertise. In addition, these advisors and consultants are not our employees and may have commitments to, or consulting or advisory contracts with, other entities that may limit their availability to us. In addition, our advisors and consultantsThey may also have arrangements with other companies to assist those companies in developing products or technologies that may compete with ours.

Although we are still in the process of implementing our internal security controls, policies and other measures, we takehave taken steps designed to detect, mitigate, and remediate vulnerabilities in our information systems (such as our hardware and/or software, including that of third parties upon which we rely). We may not, however, detect and remediate all such vulnerabilities including on a timely basis. Further, we may experience delays in developing and deploying remedial measures and patches designed to address identified vulnerabilities. Despite the implementation of these security measures, our information technology systems and those of our third-party vendors and other contractors and consultants have been in the pastpast, and may be in the futurefuture, potentially vulnerable to breakdown or other damage or interruption from service interruptions, system malfunction, accidents by our employees or third-party service providers, natural disasters, terrorism, war, global pandemics, and telecommunication and electrical failures. We may also experience security incidents from inadvertent or intentional actions by our employees, third-party vendors, contractors, consultants, business partners and/or other third parties, including theft, fraud or unauthorized access to or use of our information technology systems, or attack or damage from hacking, cyberattacks or supply chain attacks by malicious third parties and sophisticated nation-state and nation-state-supported actors (including the deployment of harmful computer viruses and malware, ransomware, denial or degradation-of-service attacks, phishing attacks and other social engineering and other means to affect service reliability and threaten the confidentiality, integrity and availability of information), which may compromise our system infrastructure, or that of our third-party vendors and other contractors and consultants, impede our ability to conduct business, delay our financial reporting or lead to data leakage.

While we have a security consultant provide annual assessments on our systems vulnerabilities, the risk of a security incident or disruption, particularly through cyberattacks or cyber intrusion, including by computer hackers, foreign governments and cyber terrorists, hascontinues generallyto increasedincrease as the number, intensity, and sophistication of attempted attacks and intrusions from around the world have increased. We may not be able to anticipate all types of security threats, nor implement preventive measures effective against all such security threats. The techniques used by cyber criminals change frequently, may not be recognized until launched and can originate from a wide variety of sources, including outside groups such as external service providers, organized crime affiliates, terrorist organizations, or hostile foreign governments or agencies. Cyberattacks or security incidents could remain undetected for an extended period, which could potentially result in significant harm to our information technology systems, as well as unauthorized access to the information stored on and transmitted by our information technology systems. Even if identified, the full extent of a cyberattack or security incident may not be determined immediately and we may be unable to adequately investigate or remediate incidents or breaches due to attackers increasingly using tools and techniques that are designed to circumvent controls, to avoid detection, and to remove or obfuscate forensic evidence. Moreover, recovery of our information technology systems may be additionally hampered where we have outsourced the operation of information technology systems and information storage to third parties. Any breach, loss or compromise of confidential information may also subject us to liability, government enforcement actions (for example, investigations, fines, penalties, audits, and inspections); breach notification and additional reporting requirements and/or oversight; restrictions on processing sensitive information (including personal data); litigation (including class action litigation); indemnification obligations; negative publicity; reputational harm; monetary fund diversions; diversion of management attention; interruptions in our operations (including availability of data); financial loss; and other similar harms. Security incidents and attendant consequences may prevent or cause customers to stop using our products, deter new customers from using our products, and negatively impact our ability to grow and operate our business. If the information technology systems of our third-party vendors and other contractors and consultants become subject to disruptions or security incidents, we may have insufficient recourse against such third parties and we may have to expend significant resources to mitigate the impact of such an event, and to develop and implement protections to prevent future events of this nature from occurring.

We have incurred substantial losses during our history and do not expect to become profitable in the near future, and we may never achieve profitability. Unused federal net operating losses generated in tax years beginning after December 31, 2017 will not expire and may be carried forward indefinitely but the deductibility of such federal net operating losses in taxable years beginning after December 31, 2020 is limited to 80% of current year taxable income. In addition, both our current and our future unused losses and other tax attributes (such as research credits) may be subject to limitation under Sections 382 and 383 of the U.S. Internal Revenue Code of 1986, as amended, or the Code, if we undergo, or have undergone, an “ownership change,” generally defined as a greater than 50 percentage point change (by value) in our equity ownership by certain stockholders over a three-year period. As of December 31, 2024, weWe had a Section 382 study prepared which covered the period from inception through December 2024.2025. The study concluded that several ownership changes occurred since theour inception of the Company.inception. However, it was determined that the annual available NOLs under Section 382 will be sufficient to utilize in their entirety prior to their respective expiration years. It is possible that we may undergo additional ownership changes in the future, including as a result of the issuance of common stock pursuant to our initial public offering completed in February 2025 or future transactions or events that may be outside of our control. As a result, even if we attain profitability, our ability to use our pre-change net operating loss carryforwards and other pre-change tax attributes (such as research tax credits) to offset our post-change income or taxes may be limited. Similar provisions of state tax law may also apply to limit our use of accumulated state tax attributes. In addition, at the state level, there may be periods during which the use of net operating losses is suspended or otherwise limited, which could accelerate or permanently increase state taxes owed. As a result of the foregoing, even if we attain profitability, we may be unable to use all or a material portion of our net operating losses and other tax attributes, which could adversely affect our future cash flows.

Depending upon the timing, duration and specifics of any FDA marketing approval of any therapeutic candidates we may develop, one or more of our U.S. patents, if issued, may be eligible for limited patent term extension under the Drug Price Competition and Patent Term Restoration Action of 1984, or the Hatch-Waxman Amendments. The Hatch-Waxman Amendments permit a patent extension term of up to five years as compensation for patent term lost during the FDA regulatory review process, although the amount of available extension to any specific patent eligible for patent term extension depends on a variety of factors, including the date on which the patent issues and certain dates related to the regulatory review period. A patent term extension cannot extend the remaining term of a patent beyond a total of 14 years from the date of product approval, only one patent may be extended and only those claims covering the approved drug, a method for using it, or a method for manufacturing it may be extended. However, we may not be granted an extension because of, for example, failing to exercise due diligence during the testing phase or regulatory review process, failing to apply within applicable deadlines, failing to apply prior to expiration of relevant patents, or otherwise failing to satisfy applicable requirements. Moreover, the applicable time period or the scope of patent protection afforded could be less than we request. To the extent we have in-licensed any U.S. patents, we may not have control over the patent term extension process and may rely on our partners to obtain a patent term extension. Moreover, under the agreements pursuant to which we out-license or assign our MZE001, ATXN2 and UNC13A patent portfolios, the applicable licensees have the sole right to apply for patent term extensions with respect to the licensed patents and patent applications. If we or our partners are unable to obtain patent term extension or if the term of any such extension is less than we request, our competitors may obtain approval of competing products following our patent expiration, and our business, financial condition, results of operations and prospects could be materially harmed.

a severe or prolonged economic downturn; and general economic and geopolitical conditions, including fluctuating interest and tariff rates, the impact of significant political, trade and regulatory developments, disruptions in the capital and credit markets, market volatility and inflation.

We are a clinical-stage biopharmaceutical company harnessing the power of human genetics to develop novel, small molecule precision medicines for patients living with renal, cardiovascularkidney and relatedmetabolic metabolic, or CVRM diseases, including obesity.diseases. We are advancing a pipeline using our Compass platform, which allows us to identify and characterize genetic variants inassociated with health and disease and then linkdetermine thosehow variants to the biological pathways thatthese drive risk for and protection against disease in specific patient groups through a process we refer to as variant functionalization. Our Compass platform has been purpose-built to inform all phases of our drug discovery and development process through clinical trial design. We are currently advancing two whollywholly-owned owned leadclinical programs, MZE829 and MZE782, each of which represents a novel precision medicine-based approach. Our goal is to bring novel precision medicines to patients with CVRM diseases, which is where we believe we can maximize our impact on human health.MZE782.

Since our inception, we have focused substantially all of our efforts and financial resources on research and development activities for our programs and on establishing arrangements and collaborations with third parties for the development of our therapeutic candidates. To date, we have not generated any revenue from product sales and wehave dofinanced notour expectoperations toprimarily generate any revenue from commercialthrough sales forof theour foreseeableequity future.and Exceptconvertible forpromissory thenotes, debt financing, as well as one-time, nonrefundable upfront payments we received pursuant to the license agreements we entered into with several biotechnology companies in 2024, including the exclusive license agreement with Shionogi & Company, Ltd., or Shionogi,Shionogi. weWe do not expect to generate any revenue from commercial sales for the foreseeable future. We expect to continue incurring significant operating losses for the foreseeable future due to the cost of research and development, clinical trials, preclinical studies and the regulatory approval process for our therapeutic candidates.

Other than the net income of $52.2 million we recorded for the year ended December 31, 2024 as a result of license revenue recognized under our license agreements, weWe have incurred significant net losses and negative cash flows from operations since our inception.inception, except for the net income and cash provided by operations due to license revenue recognized during the fiscal year ended December 31, 2024, and we expect to continue to incur significant and increasing losses as a result of our continued research and development activities. During the years ended December 31, 20242025 and 2023,2024, we incurred net income of $52.2 million and a net loss of $100.4$131.1 million and net income of $52.2 million, respectively. As of December 31, 2024,2025, we had an accumulated deficit of $358.4$489.5 million and do not expect positive cash flows from operations infor the foreseeable future. We also expect to continue incurring significant expenses and increasing losses for the foreseeable future. Our net losses may fluctuate significantly from period to period, depending on the timing of and expenditures on our planned research and development activities, as well as upon revenue from our license agreements. We expect our research and development expenses to significantly increase in connection with the conduct of planned clinical trials for our lead programs, MZE829 and MZE782, further development of our Compass platform, planned preclinical studies, and potential Investigational New Drug Applications, or INDsINDs, and clinical trials for future therapeutic candidates. We will also incur substantial additional expenses as we seek to expand our intellectual property portfolio, including through potential in-licensing opportunities, and hire additional personnel as we scale up our operations. In addition, we expect to incur additional costs associated with operating as a public company.

We will require substantial additional capital to develop our therapeutic candidates and fund operations for the foreseeable future. Until such time as we can generate sufficient revenue from product sales, if ever, we expect to finance our operations through a combination of public or private equity offerings, debt financings, collaborations and licensing arrangements. We are party to a loan and security agreement that provides us with a line of credit of up to $50.0 million. To date, we have not drawn down any funds from this debt facility and do not currently intend to do so. Adequate additional financing may not be available to us on favorable terms, or at all. In addition, 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. If we are unable to raise capital when needed or on favorable terms, we could be forced to delay, reduce or eliminate our clinical and preclinical development, research and development programs, our commercialization plans or other operations. The amount and timing of our future funding requirements will depend on many factors including the pace and results of our development efforts. We cannot assure you that we will ever be profitable or generate positive cash flows from operating activities.

In March 2024, we entered into an exclusive license agreement, or the License Agreement, with Shionogi, pursuant to which we granted Shionogi an exclusive, worldwide, sublicensable license to research, develop, manufacture and commercialize MZE001 and certain other small molecule compounds modulating glycogen synthase 1, or the Licensed Products. As consideration for the licensed rights and the transfer of know-how and materials, we received an upfront payment of $150$150.0 million, which is the only payment we have received under the License Agreement to date. The License Agreement also requires that Shionogi pay us up to $275$275.0 million in the aggregate in milestone payments upon the completion of certain clinical and regulatory milestones and up to $330$330.0 million in the aggregate in milestone payments if certain sales milestones are achieved. In March 2026, we received notification that the first clinical development milestone under the License Agreement has been met, and we expect to receive a $20.0 million milestone payment from Shionogi in connection with the achievement of this milestone. The License Agreement also requires that Shionogi pay us tiered royalties ranging from percentages in the low double-digits to twenty on net sales of Licensed Products, subject to certain deductions.

fees paid to contract manufacturing organizations, or CMOsCMOs, in connection with the production of research products and clinical trial materials;

fees paid to clinical research organizations, or CROsCROs, in connection with clinical trials as well as preclinical and toxicology studies;

General and administrative expenses consist primarily of personnel-related costs, including salaries, bonuses, benefits, and stock-based compensation expense, for personnel in executive, finance, accounting, corporate development, and other administrative functions. General and administrative expenses also include legal fees, professional fees paid for accounting, auditing, consulting, tax, and investor relations services, insurance costs, and facility costs not otherwise included in research and development expenses, and, subsequent to December 31, 2024, will includeand public company expenses such as costs associated with compliance with the rules and regulations of the Securities and Exchange Commission, or SEC, and Nasdaq.

We expect that our general and administrative expenses will continue to increase significantly in the foreseeable future as additional administrative personnel and services are required to manage these functions ofassociated with being a public company and as our pipeline of therapeutic candidates expands.

Interest and other income, net consists of interest income earned on our cash, cash equivalents and cashmarketable equivalents,securities, interest expense, foreign currency re-measurement and transaction gains and losses. We expect interest income to increase as a result of the one-time, nonrefundable upfront payments of $167.5 million in the aggregate that we received pursuant to the license agreements we entered into with several biotechnology companies in 2024, including the exclusive license agreement with Shionogi, the receipt of $70.8 million in net proceeds from the Series D Financing that we completed in November 2024, and the receipt of net proceeds of approximately $127.8 million received from our initial public offering completed in February 2025 and net proceeds of approximately $141.3 million received from the Private Placement completed in September 2025. However, interest income may vary eachfrom reporting period to reporting period, depending on our average cash deposits, money market fund balances, and other investment balances during the period and prevailing market interest rates. We expect foreign currency gains and losses to vary each reporting period depending on the fluctuations in foreign currency exchange rates.

We issued $24.5 million and $16.2 million of convertible promissory notes during the years endedFrom December 31,2023 2024through andApril 2023,2024, respectively,we forissued a total of $40.7 million aggregate principal amount of convertible promissory notes.notes, The convertible promissory notes issued from December 2023 through early 2024which contained automatic conversion features triggered upon a qualified preferred stock financing or a qualified public offering. We elected to apply the fair value option, as per Accounting Standards Codification, or ASC, Section 825-10, to the outstanding convertible promissory notes issued. As such, the convertible promissory notes were recognized at fair value with changes in fair value recognized in the statements of operations and comprehensive income (loss). The fair value of the convertible promissory notes was $20.1 million as of December 31, 2023.income. We recorded a net loss of $3.8$8.8 million,million in the statements of operations and comprehensive (loss) income for the year ended December 31, 2024, reflecting the change in the fair value of the convertible promissory notes in the statements of operations and comprehensive income (loss) for the year ended December 31, 2023.notes. The impact of interest expense on the convertible promissory notes is included within the changes in fair value. In connection with the private placement of our Series D convertible preferred stock, or Series D Preferred Stock offering that we completedStock, in November 2024, which was a qualified preferred stock financing pursuant to the terms of the convertible promissory notes, the other outstanding convertible promissory notes issued between December 2023 and April 2024 automatically converted into 39,395,572 shares of Series D-1 Preferredconvertible Stock,preferred stock, or Series D-1 Preferred Stock, at a conversion price of $1.10336 per share. WeNo remeasuredconvertible thepromissory notes were outstanding as of December 31, 2024. Accordingly, no change in fair value of the convertible promissory notes immediately before they converted to Series D-1 Preferred Stock, resulting in a fair value of $53.4 million, andwas recorded an additional loss of $8.8 million, reflecting the change in the fair value of the convertible promissory notes in the statements of operations and comprehensive income (loss) income for the year ended December 31, 2024.2025.

We recognized a total of $167.5 million ofno license revenue for the year ended December 31, 2024.2025. License revenue for the year ended December 31, 2024 was $167.5 million. License revenue recognized in 2024 primarily related to the License Agreement with Shionogi, whereinpursuant to which we received an upfront payment of $150.0 million in exchange for the transfer of the license, know-how, and materials related to the research, development, manufacture and commercialization of MZE001 and certain other small molecule compounds modulating glycogen synthase 1. License revenue recognized in 2024 also included amounts related to the exclusive license agreement with Trace Neuroscience, Inc., or Trace, whereinpursuant to which we received an upfront payment of $15.0 million in exchange for the transfer of the license, know-how, and materials related to the research, development, manufacture and commercialization of a discovery research program targeting UNC13A for the treatment of amyotrophic lateral sclerosis, or ALS, and $2.5 million related to the exclusive license agreement with Neurocrine Biosciences, Inc., or Neurocrine, for the transfer of the license, know-how and materials related to a discovery research program targeting ATXN2. We recognized no license revenue during the year ended December 31, 2023.

General and administrative expenses were $34.5 million for the year ended December 31, 2025, compared to $26.4 million for the year ended December 31, 2024, compared to $24.6 million for the year ended December 31, 2023.2024. The increase of $1.8$8.1 million was primarily due to higheran increase of $6.0 million in personnel-related costs due to an increase in headcount and higher non-cash stock-based compensation expense, as well as higher facilities and other costs of $3.0$1.4 million,million partiallyand offsethigher bycosts lower outsidefor professional servicesservice fees of $1.5$0.3 million.

Interest and other income, net was $11.8 million for the year ended December 31, 2025, compared to $4.7 million for the year ended December 31, 2024 compared to $2.0 million for the year ended December 31, 2023.2024. This increase of $2.7$7.1 million primarily reflects an increase in interest income as a result of the higher cash, cash equivalent, and cashmarketable equivalentsecurities balances held during the year ended December 31, 20242025, from the nonrefundable upfront payments that weproceeds received pursuant to the license agreements we entered with Shionogi, Trace and Neurocrine in 2024 and proceeds from the private placement of our Series D Preferred Stock we issuedcompleted in November 2024.2024, the initial public offering completed in February 2025 and the Private Placement completed in September 2025.

The convertible promissory notes issued inbetween December 2023 and earlyApril 2024 containcontained automatic conversion features that are triggered upon a qualified preferred stock financing or a qualified public offering. We elected to apply the fair value option to the convertible promissory notes issued. As such, the convertible promissory notes arewere recorded at fair value at each reporting period with changes in fair value recognized in the statements of operations and comprehensive income (loss). income. The change in fair value of the convertible promissory notes resulted in a loss of $8.8 million and $3.8 million for the yearsyear ended December 31,31 2024 and 2023, respectively.2024. The loss recorded is primarily due to the conversion of the convertible promissory notes toissued between December 2023 and April 2024 converted into Series D-1 Preferred Stock in November 2024 atin aconnection discountwith factorthe private placement of 0.2 relative to the price paid by theour Series D Preferred StockStock, investors.which was a qualified preferred stock financing pursuant to the terms of the convertible promissory notes. No convertible promissory notes were outstanding as of December 31, 2024. Accordingly, no change in fair value of convertible promissory notes was recorded in the statements of operations and comprehensive (loss) income for the year ended December 31, 2025.

IncomeWe recognized no income tax expense wasfor the year ended December 31, 2025. We recognized income tax expense of $1.2 million for the year ended December 31, 2024 compared to zero for the year ended December 31, 2023.2024. The increaseamount recognized in 2024 was primarily due to the net income generated during the year ended December 31, 2024, as a result of the recognition of license revenue under ourthe licenseLicense agreementsAgreement with Shionogi, Traceresulting andin Neurocrine,taxable income to the Company, as compared to the net loss incurred during the year ended December 31, 2023.2025.

Since our inception, we have not generated any revenue from product sales and we do not expect to generate any revenue from commercial sales for the foreseeable future, if at all. We have incurred significant operating losses and negative cash flows from operations. We anticipate that we will continue to incur net losses for the foreseeable future. To date, we have financed our operations primarily through private placementssales of redeemable convertible preferred stock, one-time, nonrefundable upfront payments made to us in connection with our entryequity into license agreements, private placements ofand convertible promissory notes, anddebt mostfinancing, recently,as throughwell anas initialone publictime, offeringnonrefundable ofupfront ourlicensing common stock.payments. As of December 31, 2024,2025, we had cash andcash, cash equivalents and marketable securities of $196.8$360.0 million and an accumulated deficit of $358.4$489.5 million.

In February 2025, we completed our initial public offering, pursuant to which we issued and sold an aggregate of 8,750,000 shares of our common stock at thean initial public offering price of $16.00 per share, resulting in net proceeds of approximately $127.8 million, after deducting underwriting discounts, commissions and other offering expenses payablepaid by the Company of approximately $12.2 million.us.

Based on our current operating plan, we believe that our existing cash, cash equivalents and cashmarketable equivalents, together with the net proceeds from our initial public offering,securities will be sufficient to fund our operations for at least one year from the date of this Annual Report on Form 10-K. We have based this estimate on our current assumptions, which may prove to be wrong, and we may exhaust our available capital resources sooner than we expect.

If we raise additional funds by issuing equity securities, our stockholders will experience dilution. If we raise additional capital through debt financing, we may be subject to covenants that restrict our operations including limitations on our ability to incur liens or additional debt, pay dividends, repurchase our common stock, make certain investments, and engage in certain merger, consolidation or asset sale transactions. Any debt financing that we raise or additional equity that we raiseissue may contain terms that are not favorable to us or our stockholders.

If we raise additional funds through collaborations, strategic alliances or licensing arrangements with third parties, we may have to relinquish valuable rights to our proprietary technology, future revenue streams, research programs or therapeutic candidates, including granting licenses to our proprietary technologies, on terms that may not be favorable to us. If we are unable to raise additional funds through equity or debt financings or collaborations, strategic alliances or licensing arrangements with third parties when needed, we may be required to delay, limit, reduce and/or terminate our product development programs or any future commercialization efforts or grant rights to develop and market therapeutic candidates that we would otherwise prefer to develop and market ourselves.

We have entered into consortium agreements with the University of Helsinki and Queen Mary University to access genomic information from patient samples and genetic and paired clinical data. As of December 31, 2024,2025, we are obligated to pay $4.9$4.2 million under thesethe agreementsUniversity of Helsinki consortium agreement through the year ended December 31, 2027. As of December 31, 2025, we have no additional payment obligations under the Queen Mary University consortium agreement.

Net cash provided by (used in) provided by operating activities

Net cash used in operating activities was $111.9 million for the year ended December 31, 2025, and net cash provided by operating activities was $76.0 million for the year ended December 31, 2024. The net cash used in operating activities for the year ended December 31, 20232025 was primarily due to our net loss of $100.4$131.1 million, combined with a net change in our operating assets and liabilities of $6.3$2.3 million, partially offset by $19.9$21.5 million in non-cash charges such as depreciation, stock-based compensation, lease expense and thenet change in fair valueaccretion of thediscounts convertibleon promissorymarketable notes.securities.

For the year ended December 31, 2024 netNet cash provided by financing activities was $92.8$275.3 million,million for the year ended December 31, 2025, which consisted of net proceeds of $127.8 million from the issuance of convertiblecommon promissorystock notespursuant to our initial public offering, $141.3 million of $24.5 million, net proceeds from the issuancePrivate ofPlacement, Series D Preferred Stock of $70.8 million and $0.7$5.6 million from the exercise of stock option awards, net of repurchases, and $1.3 million from the issuance of common stock under the 2025 Employee Stock Purchase Plan, or 2025 ESPP, partially offset by the payment of $3.2$0.5 million for deferredthe offeringsuccess costs.fee under our previous the loan and security agreement.

For the year ended December 31, 2023, netNet cash provided by financing activities was $16.4$92.8 million,million for the year ended December 31, 2024, which consisted of net proceeds from the issuance of Series D Preferred Stock of $70.8 million, net proceeds from the issuance of convertible promissory notes of $16.2$24.5 millionmillion, and $0.2$0.7 million from the exercise of stock option awards, net of repurchases.repurchases, partially offset by the payment of $3.2 million for deferred offering costs related to our initial public offering.

Analyzing an arrangement to identify performance obligations requires the use of judgment. In arrangements that include the license of intellectual property and other promised goods or services, we first identify if the licenses are distinct from the other promises in the arrangement. If the license is not distinct, the license is combined with other promised goods or services into a single performance obligation. Factors that are considered in evaluating whether a license is distinct from other promises include, for example, whether the counterparty can benefit from the license without the promised goods and service on its own or with other readily available resources and whether the promised good or service is expected to significantly modify or customize the intellectual property. We then estimate the transaction price, which also requires the use of judgment when evaluating the fixed consideration and any variable amounts, including milestone payments. ForMilestone arrangementspayments withare evaluated for any necessary constraints based on the assessment of the probability of achievement of certain milestonedevelopment, payments,regulatory weand commercial events. We re-evaluate the probability of achievement of the related milestonemilestones each reporting period,period and any related constraints, if any, and if necessary, adjust the estimate of the overall transaction price. In addition, we are eligible to receive certain royalties on net sales of licensed products, if successfully commercialized by our licensees. The royalties are dependent on future sales which are at the full discretion of the licensee. We will recognize sales-based milestone payments in the period in which we achieve the milestone under the sales-based royalty exception allowed under accounting rules. Accordingly, we will apply a constraint to these amounts until the future sales have occurred.

DuringTo the year ended December 31, 2024,date, we have identified only single combined performance obligations for each license arrangement that we have entered for which revenue is recognized at a point in time. We may enter into arrangements in the future that include the license of intellectual property and other promised goods or services that may result in multiple performance obligations. In those circumstances, we may need to allocate the transaction price based on the relative standalone selling prices of each of the performance obligations, which will require significant judgment and the determination of significant assumptions related to such estimates.

We use the estimated fair value of our common stock to determine the fair value of restricted stock awards, or RSAs, and restricted stock units, or RSUs, on the date of grant. We estimate the fair value of service-based options to employees and non-employees using the Black-Scholes option-pricing valuation model, or Black-Scholes model. The Black-Scholes model requires the input of subjective assumptions, including the fair value of common stock, expected term, expected volatility, risk-free interest rate, and expected dividend yield, which are described in greater detail below.

Fair value of common stock—Historically,Prior asto our initial public offering, there had been no public market for our common stock prior to our initial public,and the fair value of our common stock was determined by our board of directors based in part on valuations of our common stock prepared by a third-party valuation specialist. SeeAs a public trading market for our common stock has been established in connection with the subsectionclosing entitledof “Commonour initial public offering, it will no longer be necessary for our board of directors to estimate the fair value of our common stock valuations”in below.connection with our accounting for stock option grants and other such awards we may grant, as the fair value of our common stock will be determined based on the quoted market price of our common stock.

Stock-based compensation expense for employees and non-employees is reflected in the statements of operations and comprehensive income (loss) income as follows:

As of December 31, 20242025 and 20232024 there was $30.0$28.4 million and $16.4$30.0 million, respectively, of total unrecognized compensation cost related to unvested options and RSAs for which the cost was expected to be recognized over a weighted-average period of 3.113.05 years and 2.523.11 years, respectively. As of December 31, 2025, there was $1.9 million unrecognized share-based compensation cost related to the 2025 ESPP, which is expected to be recognized over a weighted-average period of 1.16 years. As of December 31, 2025, there was $12.1 million of total unrecognized compensation cost related to RSUs, which is expected to be recognized over a weighted average period of 1.67 years.

Investment in drug development is a highly speculative undertaking and involves a substantial degree of risk. We are a clinical-stage biopharmaceutical company with a limited operating history. Our lead programs are still in early clinical development, and we are subject to the risks of failure inherent in the development of therapeutic candidates based on novel technologies. We have not yet demonstrated an ability to successfully overcome many of the risks and uncertainties frequently encountered by companies in new and rapidly evolving fields, particularly in the biotechnology industry. Furthermore, we do not expect to generate any product revenue from commercial sales for the foreseeable future, and we expect to continue to incur significant operating losses for the foreseeable future due to the cost of clinical trials, preclinical studies, research and development, and the regulatory approval process of our therapeutic candidates. For the three and six months ended MarchJune 31,30, 20262026, we incurred a net loss of $44.7 million and $68.9 million, respectively. For the three and six months ended June 30, 2025, we incurred a net loss of $24.2$33.7 million and $32.8$66.5 million, respectively. As of MarchJune 31,30, 2026, we had an accumulated deficit of $513.8$558.5 million. We expect to continue to incur significant research and development and other expenses related to our ongoing operations.

As of MarchJune 31,30, 2026, our cash, cash equivalents and marketable securities were $362.9$494.9 million. Based on our current operating plan, we believe our existing cash, cash equivalents and marketable securities as of MarchJune 31,30, 2026 will enable us to fund our operating expenses and capital expenditure requirements for at least one year from the date of this Quarterly Report on Form 10-Q. We have based this estimate on assumptions that may prove to be wrong, and we could use our capital resources sooner than we currently expect. Changes beyond our control may occur that would cause us to use our available capital before that time, including changes in and progress of our development activities and changes in regulation. Our future capital requirements will depend on many factors, including, but not limited to:

In April 2026, we completed an underwritten registered offering pursuant to which we issued and sold an aggregate of 5,540,000 shares of our common stock at a purchase price of $23.50 per share and pre-funded warrants to purchase up to an aggregate of 850,000 shares of our common stock at a purchase price of $23.499 per pre-funded warrant, which represents the per share offering price for the shares of common stock less a $0.001 per share exercise price for each pre-funded warrant, resulting in estimated net proceeds of approximately $144.7$144.6 million, after deducting underwriting discounts and commissions and estimated offering expenses.

Our therapeutic candidates are in early stages of development. We do not have any products that are approved for sale in any jurisdiction. We initiated a Phase 2 trial of MZE829, our most advanced lead program, in November 2024, dosed our first patient in February 2025 and reported positive topline clinical proof of concept data in March 2026. We plan to initiate a pivotal trial of MZE829 in the first half of 2027, subject to regulatory feedback. We initiated a Phase 1 clinical trial of our second lead program, MZE782, in September 2024 and announced initial clinical data in healthy adult volunteers in September 2025. In August 2026, we announced that the Phase 2 proof-of-concept trial of MZE782 in patients with phenylketonuria, or PKU, has initiated and chronicwe kidneyanticipate disease,reporting ortopline CKD,data from this trial in September 2025.2027. We plan to initiate twoa Phase 2 trialsproof-of-concept trial of MZE782 in PKUpatients andwith CKD in 2026.the first half of 2027. Our other programs are at an early research stage, with no therapeutic candidates identified. Our research programs may not result in viable therapeutic candidates for some time, if ever, and our therapeutic candidates in development may not achieve success in our current or any future clinical trials, or obtain regulatory approval.

We have limited experience designing and implementing clinical trials. We initiated a Phase 2 trial of MZE829, our most advanced lead program, in November 2024, dosed our first patient in February 2025 and reported positive topline clinical proof of concept data in March 2026. We plan to initiate a pivotal trial of MZE829 in the first half of 2027, subject to regulatory feedback. We initiated a Phase 1 clinical trial of MZE782 in September 2024 and announced initial clinical data in PKUhealthy andadult CKDvolunteers in September 2025. WeIn planAugust to2026, initiatewe twoannounced that the Phase 2 trialsproof-of-concept trial of MZE782 in patients with PKU has initiated and we anticipate reporting topline data from this trial in 2027. We plan to initiate a Phase 2 proof-of-concept trial of MZE782 in patients with CKD in 2026.the first half of 2027. Failure to design any one of our clinical trials adequately, or incorrect assumptions about the design of the trial, could adversely affect the ability to initiate the trial, enroll patients, complete the trial, or use the clinical trial’s results to support an application for regulatory approval, as well as lead to increased or unexpected costs.

We have filed patent applications, both in the United States and internationally, protecting our inventions related to our MZE829 and MZE782 candidates as well as our out-licensed programs. Our MZE001 patent portfolio has been exclusively out-licensed to Shionogi, who also has the exclusive right to make decisions regarding the prosecution and enforcement of patents or patent applications within that portfolio. Our ATXN2 and UNC13A patent portfoliosportfolio havehas been exclusively licensed or assigned, respectively,assigned to otherTrace biotechnologyNeuroscience, companies,Inc., who havehas the exclusive right to make decisions regarding the prosecution and enforcement of those respective portfolios.

The patenting process is expensive and time-consuming, and we and our current or future partners may not be able to file and prosecute all necessary or desirable patent applications at a reasonable cost or in a timely manner. In addition, we or our partners may not pursue or obtain patent protection in all relevant jurisdictions, particularly in markets where translation costs are high. It is also possible that we or our partners will fail to identify patentable aspects of our or their research and development output before it is too late to obtain patent protection. Although we seek to enter into non-disclosure and confidentiality agreements with parties who have access to confidential or patentable aspects of our research and development output, such as our employees, consultants, independent contractors, advisors, CMOs, CROs, hospitals, independent treatment centers, suppliers, partners and other third parties, any of these parties may breach the agreements and disclose such output before a patent application is filed, thereby jeopardizing our ability to seek patent protection. In addition, publications of discoveries in the scientific literature often lag behind the actual discoveries, and patent applications in the United States and other jurisdictions are typically not published until 18 months after filing, or in some cases not at all. Therefore, we cannot be certain that we were the first to make the inventions claimed in our current or future patent applications, or that we were the first to file for patent protection of such inventions. Moreover, in circumstances, including with respect to our exclusive out-licensing or assignment of our MZE001, ATXN2MZE001 and UNC13A patent portfolios, where we do not have the right to control the preparation, filing, prosecution, maintenance, defense or enforcement of patents or patent applications covering technologies that we currently, or may in the future, license from or license to third parties, we will be reliant on our licensors or licensees to do so. If our partners are not fully cooperative or disagree with us as to the preparation, filing, prosecution, maintenance, defense or enforcement of any licensed patent rights, such patent rights could be compromised.

Although we have issued patents in the United States and other countries and pending patent applications in the United States and other countries, filing, prosecuting, maintaining, enforcing and defending patents in all countries throughout the world would be prohibitively expensive, and our intellectual property rights in some countries outside the United States can be less extensive than those in the United States. In addition, the laws of some foreign countries do not protect intellectual property rights to the same extent as federal and state laws in the United States. Consequently, we may not be able to prevent third parties from practicing our inventions in all countries outside the United States or from selling or importing products made using our inventions in and into the United States or other jurisdictions. Competitors may use our technologies in jurisdictions where we have not obtained patent protection to develop their own products and, further, may export otherwise infringing products to territories where we have patent protection, but enforcement is not as strong as that in the United States. These products may compete with our therapeutic candidates, and our patents, the patents of our current and future partners, or other intellectual property rights may not be effective or sufficient to prevent them from competing.

Depending upon the timing, duration and specifics of any FDA marketing approval of any therapeutic candidates we may develop, one or more of our U.S. patents, if issued, may be eligible for limited patent term extension under the Hatch-Waxman Amendments. The Hatch-Waxman Amendments permit a patent extension term of up to five years as compensation for patent term lost during the FDA regulatory review process, although the amount of available extension to any specific patent eligible for patent term extension depends on a variety of factors, including the date on which the patent issues and certain dates related to the regulatory review period. A patent term extension cannot extend the remaining term of a patent beyond a total of 14 years from the date of product approval, only one patent may be extended and only those claims covering the approved drug, a method for using it, or a method for manufacturing it may be extended. However, we may not be granted an extension because of, for example, failing to exercise due diligence during the testing phase or regulatory review process, failing to apply within applicable deadlines, failing to apply prior to expiration of relevant patents, or otherwise failing to satisfy applicable requirements. Moreover, the applicable time period or the scope of patent protection afforded could be less than we request. To the extent we have in-licensed any U.S. patents, we may not have control over the patent term extension process and may rely on our partners to obtain a patent term extension. Moreover, under the agreements pursuant to which we out-license or assign our MZE001, ATXN2MZE001 and UNC13A patent portfolios, the applicable licensees have the sole right to apply for patent term extensions with respect to the licensed patents and patent applications. If we or our partners are unable to obtain patent term extension or if the term of any such extension is less than we request, our competitors may obtain approval of competing products following our patent expiration, and our business, financial condition, results of operations and prospects could be materially harmed.

We may, from time to time, elect to sell securities in a public offering or private placement. For example, in September 2025, we entered into a securities purchase agreement with certain investors, pursuant to which we issued and sold in a private placement an aggregate of 4,000,002 shares of our common stock and pre-funded warrants to purchase up to an aggregate of 5,231,090 shares of our common stock. We subsequently registered the common stock (including the shares of common stock issuable upon exercise of the pre-funded warrants) under the Securities Act for resale on Form S-1, such that these shares may now be sold by the purchasers in the public market at any time. Additionally, as of March 31, 2026, there were 4,331,090 shares of our common stock subject to outstanding pre-funded warrants, with an exercise price of $0.001 per share. In April, 2026, we completed an underwritten registered offering pursuant to which we issued and sold an aggregate of 5,540,000 shares of our common stock and pre-funded warrants to purchase up to an aggregate of 850,000 shares of our common stock. As of June 30, 2026, there were 5,181,090 shares of our common stock subject to outstanding pre-funded warrants, with an exercise price of $0.001 per share. To the extent any of these warrants are exercised, the shares underlying these warrants may be immediately sold in the public market. These sales, or the perception in the market that we or holders of a large number of shares intend to sell shares, could reduce the market price of our common stock.

Our most advanced program, MZE829, is an oral, small moleculemolecule, dual-mechanism inhibitor of apolipoprotein L1, or APOL1, for the treatment of patients with APOL1-mediated kidney disease, or AMKD, a genetically defined sub-set of chronic kidney disease, or CKD, for which there is no approved treatment today. We initiated a Phase 2 trial of MZE829 in November 2024 and dosed our first patient in February 2025. In March 2026, we announced positive topline clinical proof of concept data from our Phase 2 trial of MZE829 in patients with AMKD, in which we enrolled 15 patients. The results demonstrated that treatment with MZE829 led to a clinically meaningful mean reduction in proteinuria, as measured by urinary albumin-to-creatinine ratio. MZE829 was generally well tolerated, with no serious adverse events or severe treatment-related adverse events reported. We plan to continue enrollment in the Phase 2 trial and to advance MZE829 into a pivotal development program. We anticipate reporting additional data from the Phase 2this trial in late 2026 or early 2027. In addition, we plan to initiate a pivotal trial of MZE829 in the first half of 2027, subject to regulatory feedback.

Our second program, MZE782, is an oral, small molecule inhibitor for the treatment of patients with phenylketonuria, or PKU, an inherited metabolic disorder, and for the treatment of patients with CKD. In September 2025, we reported results from our Phase 1 clinical trial of MZE782, in which we enrolled 112 healthy adult volunteers. MZE782 was well tolerated across all doses in all cohorts and demonstrated a favorable pharmacokinetics profile after single and multiple oral doses. MZE782 produced dose-dependent increases in 24-hour urinary excretion of the neutral amino acids phenylalanine and glutamine across both single ascending dose and multiple ascending dose cohorts, confirming target engagement and SLC6A19 inhibition. We also observed dose-dependent changes in estimated glomerular filtration rate in healthy individuals with MZE782, similar to those seen with SGLT2 inhibitors, suggesting a potential beneficial effect on kidney physiology in CKD patients. WeIn planAugust to2026, initiatewe announced that the Phase 2 proof-of-concept trials of MZE782 in patients with PKU by mid-2026 and in patients with CKD in the second half of 2026. We anticipate reporting topline data from the Phase 2 trial of MZE782 in patients with PKU has initiated and we anticipate reporting topline data from this trial in 2027. We plan to initiate a Phase 2 proof-of-concept trial of MZE782 in patients with CKD in the first half of 2027.

We have incurred significant losses and negative cash flows from operations and we expect to incur significant and increasing losses for the foreseeable future as a result of our continued research and development activities. During the three and six months ended MarchJune 31,30, 20262026, we incurred a net loss of $44.7 million and $68.9 million, respectively. During the three and six months ended June 30, 2025, we incurred a net loss of $24.2$33.7 million and $32.8$66.5 million, respectively. As of MarchJune 31,30, 2026, we had an accumulated deficit of $513.8$558.5 million and do not expect positive cash flows from operations for the foreseeable future. Our net losses may fluctuate significantly from period to period, depending on the timing of and expenditures on our planned research and development activities, as well as upon revenue from our license agreements. We expect our research and development expenses to significantly increase in connection with the conduct of planned clinical trials for our lead programs, MZE829 and MZE782, further development of our Compass platform, planned preclinical studies, and potential Investigational New Drug Applications, or INDs, and clinical trials for future therapeutic candidates. We will also incur substantial additional expenses as we seek to expand our intellectual property portfolio, including through potential in-licensing opportunities, and hire additional personnel as we scale up our operations. In addition, we expect to incur additional costs associated with operating as a public company.

As of MarchJune 31,30, 2026, we had cash, cash equivalents and marketable securities of $362.9$494.9 million. Since our inception, we have financed our operations primarily through issuances of our equity and convertible promissory notes, debt financing, and license agreements with biotechnology companies.

We will require substantial additional capital to develop our therapeutic candidates and fund operations for the foreseeable future. Until such time as we can generate sufficient revenue from product sales, if ever, we expect to finance our operations through a combination of public or private equity offerings, debt financings, collaborations and licensing arrangements.agreements.

We selectively pursue strategic collaborations related to the development of certain targets, therapeutic programs, or disease areas where we believe third-party expertise or resources could be beneficial. In 2020, we formed a spin-out company, Broadwing Bio LLC, or Broadwing, with Alloy Therapeutics, Inc., or Alloy, to develop therapeutic antibody therapies for ANGPTL7 and another undisclosed target in ophthalmic diseases, informed by our Compass platform. As of MarchJune 31,30, 2026, we owned approximately 48% of the outstanding equity of Broadwing, and we expect our ownership to be significantly diluted upon the conversion of outstanding convertible notes issued by Broadwing. We retain certain opt-in rights to jointly develop and commercialize certain therapeutic candidates developed through Broadwing with Alloy. We are not involved in the development of Broadwing’s therapeutic candidate.

In March 2024, we entered into an exclusive license agreement, or the License Agreement, with Shionogi, pursuant to which we granted Shionogi an exclusive, worldwide, sublicensable license to research, develop, manufacture and commercialize MZE001 and certain other small molecule compounds modulating glycogen synthase 1, or the Licensed Products. As consideration for the licensed rights and the transfer of know-how and materials, we received an upfront payment of $150.0 million in May 2024 upon the effectiveness of the License Agreement. The License Agreement also requires that Shionogi pay us up to $275.0 million in the aggregate in milestone payments upon the completion of certain clinical development and regulatory milestones and up to $330.0 million in the aggregate in milestone payments if certain sales milestones are achieved. In April 2026, we received $20.0 million following the achievement of a clinical development milestone upon dosing of the first patient in Shionogi's Phase 2 study of MZE001 in patients with Pompe disease. The License Agreement also requires that Shionogi pay us tiered royalties ranging from percentages in the low double-digits to twenty on net sales of Licensed Products, subject to certain deductions.

The License Agreement will expire on a Licensed-Product by Licensed-Product and country-by-country basis upon the expiration of the royalty term for such Licensed Product, which will be the latest of the date when there are no remaining valid claims covering the applicable Licensed Product, the expiration of regulatory exclusivity for the applicable Licensed Product, or 11 years after the first commercial sale of the applicable Licensed Product, subject to earlier termination by the parties. As of MarchJune 31,30, 2026, we estimate that the last patent right for the only currently issued patent licensed under the License Agreement will expire in 2042, without giving effect to any potential patent term extensions, patent term adjustments, or future patents that may or may not issue with respect to the Licensed Products. Upon expiration of the License Agreement, the licenses granted to Shionogi will become fully paid-up, perpetual, irrevocable and royalty-free. Shionogi may terminate the License Agreement for convenience following a notice period and either party may terminate the License Agreement for bankruptcy or an uncured material breach. Upon termination of the License Agreement by Shionogi for convenience or by us for Shionogi’s bankruptcy or uncured material breach, Shionogi will grant us a non-exclusive, worldwide license under certain patent rights and know-how controlled by Shionogi as of the effective date of termination, solely as necessary to research, develop, manufacture and commercialize the Licensed Products in any field, and Shionogi will assign to us all regulatory materials and regulatory approvals relating to the Licensed Products. In consideration for these reversion rights, we would be required to pay to Shionogi reversion royalties on any sales of the Licensed Products determined based on the stage of clinical development of the applicable Licensed Product at the time of termination, ranging from percentages in the low-to-mid single digits if termination occurs on or after the achievement of certain Phase 2 clinical trial milestones and high single digit to mid-teens if termination occurs on or after the achievement of certain Phase 3 clinical milestones. Our obligation to pay these reversion royalties would expire on a Licensed-Product by Licensed-Product and country-by-country basis upon the latest of the date when there are no remaining valid claims covering the applicable Licensed Product, the expiration of regulatory exclusivity for the applicable Licensed Product, or ten years after the first commercial sale of the applicable Licensed Product.

professional service fees for consulting and related services; and facility costs, depreciation, and other expenses, which include direct and allocated expenses for rent and maintenance of facilities and other supplies.facilities.

We recognize research and development expenses as they are incurred. Nonrefundable advance payments for goods or services that willto be used or rendered forin future research and development activities are capitalized and expensed as the related goods are deliveredreceived or the related services are performed. WeDirect haveexternal notresearch historicallyand disclosed our directdevelopment costs betweenare ourrecorded clinicalto the specific programs they support. Internal research and development costs, including personnel, facility and laboratory supplies, are utilized across multiple research and development programs and ourare preclinicaltherefore programs.not directly attributable to any single program.

Total other income, net, primarily consists of interest income earned on our cash, cash equivalents and marketable securities and interest expense recognized on the loan and security agreement, or the Hercules Loan Agreement, with certain lenders and Hercules Capital, Inc. We expect total other income, net to increase as a result of additional interest income on the proceeds received from the initial term loan of $40.0 million under the Hercules Loan Agreement and the estimated net proceeds of approximately $144.7$144.6 million from the underwritten registered offering completed in April 2026, which is expected to be partially offset by interest expense recognized on the Hercules Loan Agreement. However, other income, net may vary each reporting period depending on our average cash deposits, money market fund and other investment balances during the period, prevailing market interest rates and the amount of interest expense incurred under the Hercules Loan Agreement.

Comparisons of the three and six months ended MarchJune 31,30, 2026 and 2025

We recognized $20.0 million in license revenue for the three months ended March 31, 2026. We recognized no license revenue for the three months ended MarchJune 31,30, 2026 and $20.0 million in license revenue for the six months ended June 30, 2026. We recognized no license revenue for the three and six months ended June 30, 2025. License revenue recognized in 2026 was related to achievement of a clinical development milestone upon dosing of the first patient in Shionogi's Phase 2 study of MZE001 in patients with Pompe disease under the License Agreement with Shionogi.

Research and development expenses were $34.1$34.9 million and $27.6$28.1 million for the three months ended MarchJune 31,30, 2026 and 2025, respectively. The increase of $6.6$6.8 million between the comparative three month periods was primarily due to an increase of $3.6$4.3 million in personnel-related costs due to an increase in headcount and higher non-cash stock-based compensation expense, an increase of $1.9$3.2 million inrelated clinical trial expenses based on theto progression of our Phase 2 clinical trialdevelopment forof the MZE829 in AMKDprogram and start-upan activities for our planned Phase 2 clinical trial for MZE782 in PKU, and higher costs for outside research and development servicesincrease of $0.9 million related to support our discovery research programs.and other programs, partially offset by a $1.4 million decrease related to the MZE782 program primarily due to higher costs incurred in 2025 for the Phase 1 clinical trial in healthy adult volunteers that completed in 2025.

General and administrative expenses were $12.4$13.1 million and $7.8$8.4 million for the three months ended MarchJune 31,30, 2026 and 2025, respectively. The increase of $4.6$4.7 million between the comparative three month periods was primarily due to an increase of $3.3 million in personnel-related costs due to an increase in headcount and higher non-cash stock-based compensation expense, higher costs for professional services of $0.8 million and higher facilities and other costs of $0.3 million.expense.

Total other income, net was $2.3$3.3 million and $2.6$2.8 million for the three months ended MarchJune 31,30, 2026 and 2025, respectively. The decreaseincrease of $0.3$0.5 million between the comparative three months periods primarily reflects interest expense recognized on the Hercules Loan Agreement, partially offset by an increase in interest income as a result of higher cash, cash equivalent, and marketable securities balances held during the three months ended MarchJune 31,30, 2026 resulting from the net proceeds received from the securitiesunderwritten purchaseregistered agreementoffering we entered intocompleted in SeptemberApril 20252026 and proceeds from the initial term loan funded under the Hercules Loan Agreement in February 2026.2026, partially offset by interest expense recognized on the Hercules Loan Agreement.

Since our inception, we have not generated any revenue from product sales and we do not expect to generate any revenue from commercial sales for the foreseeable future, if at all. We have incurred significant operating losses and negative cash flows from operations. We anticipate that we will continue to incur net losses for the foreseeable future. To date, we have financed our operations primarily through sales of our equity and convertible promissory notes, debt financing, as well as one-time, nonrefundable upfront licensingpayments payments.we received pursuant to the license agreements we entered into with several biotechnology companies in 2024, including the exclusive license agreement with Shionogi. As of MarchJune 31,30, 2026, we had cash, cash equivalents and marketable securities of $362.9$494.9 million and an accumulated deficit of $513.8$558.5 million.

Additionally, in February 2026, we entered into the Hercules Loan Agreement which provides for a senior secured term loan facility in an aggregate principal amount of up to $200.0 million. An initial term loan of $40.0 million was funded under the Hercules Loan Agreement and an aggregate of $160.0 million in additional term loans commitments will be available to us through February 2031, so long as we satisfy certain conditions precedent. The final tranche of $50.0 million is subject to approval by the lenders’ investment committee. The facility has a maturity date of February 1, 2031, and may be prepaid at any time, subject to prepayment premiums. This senior secured term facility will accrue interest at an annual rate determined by reference to the Prime Rate as reported in the Wall Street Journal, with interest rate floors that range from 7.95% to 9.25% depending on the tranche. AccruedThe Hercules Term Loan Facility provides for payment of interest is payable on the first business day of each monthonly until (a) February48 2030months after the Closing Date or (b) if certain performance and financing milestones are satisfied, 60 months after the maturityClosing date.Date. Accrued interest on the Term Loans is payable on the first business day of each month. In connection with the Hercules Loan Agreement, we terminated an existing loan and security agreement with another bank that provided us with a line of credit of up to $50.0 million. We did not draw down any funds from this terminated debt facility. See Note 10, Loan and security agreements, of this Quarterly Report on Form 10-Q for further discussion of the Hercules Loan Agreement.

In April 2026, we completed an underwritten registered offering pursuant to which we issued and sold an aggregate of 5,540,000 shares of our common stock at a purchase price of $23.50 per share and pre-funded warrants to purchase up to an aggregate of 850,000 shares of our common stockstock, or the 2026 Pre-Funded Warrants, at a purchase price of $23.499 per pre-funded warrant, which represents the per share offering price for the shares of common stock less a $0.001 per share exercise price for each pre-funded warrant, resulting in estimated net proceeds of approximately $144.7$144.6 million, after deducting underwriting discounts and commissions and estimated offering expenses, or the Underwritten Registered Offering. See Note 13,6, SubsequentCapital events,structure, of this Quarterly Report on Form 10-Q for further discussion of the Underwritten Registered Offering.

We have entered into consortium agreements with the University of Helsinki to access genomic information from patient samples and genetic and paired clinical data. As of MarchJune 31,30, 2026, we are obligated to pay $3.8$3.5 million under these agreements through the year ended December 31, 2027.

Comparisons of the threesix months ended MarchJune 31,30, 2026 and 2025

Net cash used in operating activities was $39.1$54.2 million and $29.5$59.6 million for the threesix months ended MarchJune 31,30, 2026 and 2025, respectively. The net cash used in operating activities for the threesix months ended MarchJune 31,30, 2026 was primarily due to our net loss of $24.2$68.9 million combined with a net change in our operating assets and liabilities of $23.7$4.6 million, offset by $8.8$19.3 million in non-cash charges such as depreciation, stock-based compensation, lease expense and amortization of debt discount and debt issuance costs. The net cash used in operating activities for the threesix months ended MarchJune 31,30, 2025 was primarily due to our net loss of $32.8$66.5 million,million combined with a net change in our operating assets and liabilities of $1.4$2.5 million, offset by $4.7$9.4 million in non-cash charges such as depreciation, stock-based compensation and lease expense.

Net cash provided by (used in) investing activities

Net cash providedused byin investing activities was $6.3$122.2 million and $0.7 million for the threesix months ended MarchJune 31,30, 2026,2026 whichand 2025. The net cash used in investing activities for the six months ended June 30, 2026 consisted of maturities of marketable securities of $30.0 million, offset by purchases of marketable securities of $23.6$186.0 million and purchases of property and equipment of $0.1$0.2 million, offset by maturities of marketable securities of $64.0 million. Net cash used in investing activities for the threesix months ended MarchJune 31,30, 2025 was $0.3 million, which consisted of purchases of property and equipment.equipment of $0.7 million.

Net cash provided by financing activities for the threesix months ended MarchJune 31,30, 2026 was $42.4$189.8 million, which consisted of net proceeds of approximately $144.6 million from the issuance of common stock and 2026 Pre-Funded Warrants pursuant to the Underwritten Registered Offering, net proceeds of $38.5 million from the Hercules Loan AgreementAgreement, and $4.1$5.8 million from the exercise of stock option awards,awards partiallyand offset$1.0 bymillion from the paymentissuance of $0.2common millionstock for deferred offering costs related tounder the Underwritten2025 RegisteredEmployee OfferingStock completedPurchase in April 2026.Plan. For the threesix months ended MarchJune 31,30, 2025, net cash provided by financing activities was $127.4$128.0 million, which consisted of net proceeds of approximately $127.8 million from the issuance of common stock pursuant to our initial public offeringoffering, $0.6 million from the issuance of common stock under the 2025 Employee Stock Purchase Plan and $0.1 million from the exercise of stock option awards, offset by the payment of $0.5 million for the success fee under the loan and security agreement with Banc of California.

Our significant accounting policies are described in detail in the notes to our condensed financial statements included elsewhere in this Quarterly Report on Form 10-Q. We described the accounting policies that we believe involve a significant level of estimation and uncertainty, which could have a material impact on our financial condition or results of operations and are therefore deemed critical accounting policies, in our “Management’s Discussion and Analysis of Financial Condition and Results of Operations” included in our Annual Report on Form 10-K for the year ended December 31, 2025, as filed with the SEC on March, 25, 2026. There have been no significant changes in our critical accounting policies and estimates during the threesix months ended MarchJune 31,30, 2026.