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

We are a clinical-stage biopharmaceutical company in the early stages of development with a limited operating history. Since our inception, we have focused primarily on developing our proprietary RNA Actuating Platform, or RAP Platform, identifying, developing and progressing our product candidates through preclinical and clinical development, organizing and staffing our company, research and development activities, establishing and protecting our intellectual property portfolio, and raising capital. Investment in biopharmaceutical product development is highly speculative because it entails substantial upfront capital expenditures and significant risk that any potential product candidate will fail to demonstrate adequate effect or an acceptable safety profile, gain regulatory approval and become commercially viable. We are still in the early stages of development of our product candidates and our lead product candidatecandidate, isCMP-002, onlyhas innot ayet Phase 1entered clinical trial.trials. We have no products licensed for commercial sale and have not generated any revenue to date, and we continue to incur significant research and development and other expenses related to our ongoing operations. As a result, we are not profitable and have incurred losses in each period since our inception. For the years ended December 31, 20242025 and 2023,2024, we reported net losses of $51.8$80.4 million and $49.3$51.8 million, respectively. As of December 31, 2024,2025, we had an accumulated deficit of $211.8$292.2 million. We expect to continue to incur significant losses for the foreseeable future, and we expect these losses to increase as we continue the research and development of, and seek regulatory approvals for, our lead product candidate CMP-CPS-001CMP-002 for the treatment of ureaSYNGAP1-related cycle disordersdisorder (“UCDsSYNGAP1”), along with any other current or future product candidates we may develop.

•finalize preclinical development for ourCMP-002 programand foradvance SYNGAP1-relatedinto disordersclinical trials;

•conductadvance current and future product candidates through preclinical studies and clinical trials of any future product candidates;

•contract with manufacturingthird-party sourcesmanufacturers for preclinical and clinical developmentsupply ofto support any future product candidates we may develop and for commercial supply with respect to any such product candidates that receive regulatory approval;

•add operational, legal, compliance, financial and management information systems and personnel to support our research, product development and future commercialization efforts, as well as to support our operations as a public company.efforts.

Our product candidates are in varying stages of preclinical and clinical development. To date, we have not generated any revenue. We have not completed a clinical trial of any product candidate, and we expect that it will be several years, if ever, before we have a product candidate ready for commercialization. To become and remain profitable, we must succeed in developing, obtaining the necessary regulatory approvals for, and eventually commercializing a product or products that generate significant revenue. The ability to achieve this success will require us to be effective in a range of challenging activities, including:

As of December 31, 2024,2025, we had cash and cash equivalents of $64.0$109.5 million. Based on our current operating plan, we estimate that our cash and cash equivalents as of December 31, 20242025 will be sufficient to fund our operating expenses and capital expenditure requirements into the second quarter of 2026.2028. However, we have based this estimate on assumptions that may prove to be wrong, and we could deplete our capital resources sooner than we currently expect. Our operating plans and other demands on our cash resources may change as a result of many factors currently unknown to us, and we do not expect that our existing cash and cash equivalents will be sufficient to complete development of any of our product candidates, or any future product candidates we may identify, and we will require substantial capital in order to advance our product candidates through clinical trials, regulatory approval and commercialization. Accordingly, we will need to obtain substantial additional funding in connection with our continuing operations. Our ability to raise additional funds may be adversely impacted by global economic conditions, disruptions to, and volatility in, the credit and financial markets in the United States and worldwide, and diminished liquidity and credit availability. If the equity and credit markets deteriorate, it may make any necessary debt or equity financing more difficult, more costly, and more dilutive. We cannot be certain that additional funding will be available on acceptable terms, or at all. If we are unable to raise capital when needed or on attractive terms, we could be forced to delay, reduce, or eliminate our research and development programs or any future commercialization efforts, or even cease operations. We expect to finance our cash needs through public or private equity or debt financings or other capital sources, including potential collaborations, licenses, and other similar arrangements. 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. Attempting to secure additional financing may divert our management from our day-to-day activities, which may adversely affect our ability to develop our product candidates.

•the initiation, type, number, scope, progress, expansions, results, costs and timing of preclinical studies and clinical trials of our product candidatesCMP-002 and any future product candidates we may choose to pursue, including the costs of modification to clinical development plans based on feedback that we may receive from regulatory authorities and any third-party products used as combination agents in our clinical trials;

Until such time, if ever, as we can generate substantial product revenues, we expect to finance our cash needs through a combination of equity offerings, debt financings, collaborations, strategic alliances and/or licensing arrangements. We do not have any committed external source of funds. To the extent that we raise additional capital through the sale of equity or convertible debt securities, our stockholders’ ownership interests will be diluted, and the terms of these securities may include liquidation or other preferences that adversely affect the rights of common stockholders. Any debt financing or 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, selling or licensing our assets, making capital expenditures, declaring dividends or encumbering our assets to secure future indebtedness.

We are early in our development efforts. Our product candidates are in varying stages of preclinical and clinical development and we have not completed a clinical trial of any product candidate.development. As a result, it will be many years before we commercialize a product candidate, if ever. If we are unable to identify and advance product candidates through preclinical studies and clinical trials, obtain marketing approval and ultimately commercialize them, or experience significant delays in doing so, our business will be materially harmed.

We are early in our development efforts and our lead product candidate ishas onlynot inyet a Phase 1entered clinical trial.trials. We have focused our efforts to date on developing our RAP Platform, identifying our programs and commencing the preclinical and clinical development of our product candidates. Our ability to generate product revenue, which we do not expect will occur for many years, if ever, will depend heavily on the successful development and eventual commercialization of our product candidates, which may never occur. We currently generate no revenue from sales of any product, and we may never be able to develop or commercialize a marketable product.

WePending aresuccessful currentlycompletion conductingof Good Laboratory Practices (“GLP”) toxicology studies and regulatory clearance, we intend to initiate a global Phase 1/2 clinical trial of CMP-CPS-001CMP-002 in Australia.individuals Clinicalwith SYNGAP1 as early as the second half of 2026. In each jurisdiction, clinical trials conductedare insubject Australiato usingapplicable “unapprovedregulatory therapeuticrequirements goods,”and ormust thosebe thatreviewed haveand not yet been evaluatedauthorized by the Therapeuticapplicable Goodsregulatory Association (the “TGA”) for quality, safetyauthorities and efficacy,reviewed mustand occurapproved pursuantby the applicable ethics committees or institutional review boards prior to eitherinitiation and as amended during the Clinical Trial Notification Scheme or the Clinical Trial Approval Scheme. In each case, the trial is supervised by a Human Research Ethics Committee (“HREC”), an independent review committee set up under the guidelinescourse of the Australian National Health and Medical Research Council that reviews, approves and provides continuing oversight of trial protocols and amendments, and of the methods and material to be used in obtaining and documenting informed consent of the trial subjects.trial. Commencing clinical trials in the United States is subject to acceptance by the U.S. Food and Drug Administration (the “FDA”) of an investigational new drug (“IND”) application and finalizing the trial design based on discussions with the FDA and other regulatory authorities. Commencing clinical trials outside the United States similarly requires the submission and authorization of comparable clinical trial applications or notifications, and compliance with local requirements relating to, among other things, trial conduct, informed consent, safety reporting, importation and handling of investigational product, and data integrity. In the event that the FDA or TGAany comparable foreign regulatory authority or ethics committee requires us to complete additional preclinical studies or we are required to satisfy other requests prior to commencing clinical trials, the start of our planned Phase 1/2 CMP-002 clinical trial and any future clinical trials may be delayed. Even after we receive and incorporate guidance from the FDA, an applicable HRECregulatory or the TGA,authorities, such authorities could disagree that we have satisfied their requirements to commence any clinical trial or continue or change their position on the acceptability of our trial design or the clinical endpoints selected, which may require us to complete additional preclinical studies or clinical trials or impose stricter approval conditions than we currently expect, which could delay the start or completion of such clinical trials or require more capital resources than we currently anticipate to start or complete such clinical trials.

Commercialization of any of our current or future product candidates will require preclinical and clinical development; regulatory and marketing approval issued by regulators in any jurisdiction where we seek to commercialize such product candidates, such as the FDA, TGAFDA and the European Commission (the “EC”) following a favorable assessment performed by the European Medicines Agency (the “EMA”); manufacturing supply, capacity and expertise; a commercial organization; and significant marketing efforts. The success of any of our current or future product candidates will depend on many factors, including the following:

•successful enrollment and completion of clinical trials, including under the FDA’s current Good Clinical Practices (“GCPs”), current Good Laboratory Practices (“GLPs”)GLPs, and any additional regulatory requirements from foreign regulatory authorities;

Our business is highly dependent on our lead product candidate, CMP-CPS-001, as our sole clinical-stage program,CMP-002, and we must complete clinical testing before we can seek regulatory approval and begin commercialization of any of our other product candidates. If we are unable to obtain regulatory approval for, and successfully commercialize, our current product candidates, our business may be materially harmed, and such failure may affect the viability of any future product candidates.

There is no guarantee that we will be able to advance CMP-002, our lead development candidate, into clinical trials, or that the results obtained in our ongoing Phase 1 clinical trial of CMP-CPS-001 or our plannedany future clinical trials we may conduct will be sufficient to obtain regulatory approval.approval for any current or future product candidates. In addition, because CMP-CPS-001 is our most advanced product candidate, and because our future product candidates will be based on our RAP Platform and antisense oligonucleotide (“ASO”) technology, if any of our lead product candidatecandidates encountersencounter safety or efficacy problems, developmental delays, regulatory issues, or other problems, our development plans and business related to our other future product candidates could be significantly harmed. A failure of one of our lead product candidatecandidates may affect the ability to obtain regulatory approval to continue or conduct clinical programs for our other or future product candidates.

The success of our business depends upon our ability to identify, develop and commercialize products based on our proprietary RAP Platform. All of our product candidates are still in varying stages of preclinical and clinical development. Our research programs may fail to identify additional product candidates for clinical development for a number of reasons. Our RAP Platform may be unsuccessful in identifying additional potential product candidates and our potential product candidates may be shown to have harmful side effects. In addition, our product candidates may be successful in upregulating the expression of their target genes and may nonetheless fail to show promising signals of therapeutic effect in such experiments or studies or they may have other characteristics that may make the product candidates impractical to manufacture, unmarketable or unlikely to receive marketing approval. Further, because all of our product candidates and programs are based on our RAP Platform, adverse developments with respect to one of our product candidates and programs may have a significant adverse impact on the actual or perceived likelihood of success and value of our other product candidates and programs.

In addition, we have not completed a clinical trial of any product candidate or successfully developed any product candidates, and our ability to identify and develop additional product candidates may never materialize. The process by which we identify and develop product candidates may fail to yield additional product candidates for clinical development for a number of reasons, including those discussed in these risk factors. In addition:

Before we can commence clinical trials for a product candidate, we must complete extensive preclinical testing and studies that support clearance of our regulatory filings, including IND applications to the FDA in the United States and other similar regulatory filings in other jurisdictions, including with respect to the TGA in Australia and the national competent authorities (“NCAs”) in the European Union.jurisdictions. We cannot be certain if the outcome of our preclinical studies and clinical trials will ultimately support further development of our product candidates or future programs or whether the FDA, TGA, NCAsFDA or comparable foreign regulatory authorities will accept our proposed clinical programs or whether the outcome of our preclinical testing and studies will ultimately support the further development of our product candidates. Conducting preclinical testing is a lengthy, time-consuming and expensive process. The length of time may vary substantially according to the type, complexity and novelty of the program, and often can be several years or more per program. As a result, we cannot be sure that we will be able to submit INDs, CTAsINDs and other similar regulatory filings for our preclinical programs on the timelines we expect, if at all, and we cannot be sure that submission of such regulatory filings will result in the FDA, TGA, NCAsFDA or comparable foreign regulatory authorities allowing clinical trials to begin.

•delays in opening clinical trial sites or obtaining required approval from institutional review boards (“IRBs”), HRECs or independent ethics committees, or the equivalent review groups for sites outside the United States or Australia,States, at each clinical trial site;

•failure to perform in accordance with the FDA’s GCPs, Good Manufacturing Practices (“GMP”) regulations or those of other regulatory authorities, including, but not limited to, Australia’s GMP requirementsauthorities;

Clinical trials must be conducted in accordance with the legal requirements, regulations or guidelines of the FDA, TGA, EC, NCAsFDA and other applicable regulatory authorities, and are subject to oversight by these governmental agencies and IRBs, HRECsIRBs or ethics committees at the medical institutions where the clinical trials are conducted. We could encounter delays if a clinical trial is suspended or terminated by us, by the data safety monitoring board for such trial or by the FDA, TGA, EMAFDA or any other regulatory authority, or if the IRBs or HRECs of the institutions in which such trials are being conducted suspend or terminate the participation of their clinical investigators and sites subject to their review. Such authorities may suspend or terminate a clinical trial due to a number of factors, including failure to conduct the clinical trial in accordance with regulatory requirements or our clinical protocols, inspection of the clinical trial operations or trial site by the FDA, TGA, EMAFDA or other regulatory authorities resulting in the imposition of a clinical hold, unforeseen safety issues or adverse side effects, failure to demonstrate a benefit from using a product candidate, changes in governmental regulations or administrative actions or lack of adequate funding to continue the clinical trial.

Moreover, principal investigators for our clinical trials may also serve as our scientific advisors or consultants to us from time to time and receive compensation in connection with such services. Under certain circumstances, we may be required to report some of these relationships to the FDA, TGA, EC, NCAsFDA or comparable foreign regulatory authorities. The FDA, TGA, NCAsFDA or comparable foreign regulatory authority may conclude that a financial relationship between us and a principal investigator has created a conflict of interest or otherwise affected interpretation of the trial. The FDA, TGA, EMAFDA or comparable foreign regulatory authority may therefore question the integrity of the data generated at the applicable clinical trial site and the utility of the clinical trial itself may be jeopardized. This could result in a delay in approval, or rejection, of our marketing applications by the FDA, TGA, EMAFDA or comparable foreign regulatory authority, as the case may be, and may ultimately lead to the denial of marketing approval of our product candidates.

Further, conducting clinical trials in foreign countries, such as our ongoingplanned Phase 1/2 clinical trial of CMP-CPS-001CMP-002 for the treatment of UCDs, which is being conducted in Australia,SYNGAP1, presents additional risks that may delay completion of our clinical trials. These risks include the failure of enrolled patients in foreign countries to adhere to clinical protocol as a result of differences in healthcare services or cultural customs, managing additional administrative burdens associated with foreign regulatory schemes, as well as political and economic risks relevant to such foreign countries.

We are in the early stages of development of our programs and haveplan initiatedto initiate a global Phase 1/2 clinical trial of our lead product candidate, CMP-CPS-001,CMP-002, in healthyindividuals adultwith volunteers in Australia,SYNGAP1, but we have not yet completedreceived or receivedregulatory clearance forto IND-commence or CTA-enablingclinical activities for our other product candidates or advanced any other product candidates into clinical development.CMP-002. As a result, our belief in the capabilities of our platform and potential success of our product candidatesCMP-002 is based on early research and preclinical studies. However, the results of preclinical studies may not be predictive of the results of later preclinical studies or clinical trials, and the results of any early-stage clinical trials may not be predictive of the results of later clinical trials. In addition, initial success in clinical trials may not be indicative of results obtained when such trials are completed. Moreover, preclinical and clinical data are often susceptible to varying interpretations and analyses, and many companies that have believed their product candidates performed satisfactorily in preclinical studies and clinical trials have nonetheless failed to obtain marketing approval of their product candidates. Our clinical trials may not ultimately be successful or support further clinical development of our product candidates.

Certain of the disorders we seek to treat, including UCDs and SYNGAP1-related disorders,SYNGAP1, have low prevalence and it may be difficult to identify and enroll patients with these disorders. If we experience delays or difficulties in the enrollment and/or maintenance of patients in clinical trials, our receipt of necessary regulatory approvals could be delayed or prevented.

Identifying and qualifying patients to participate in clinical trials of any of our current or future product candidates is critical to our success. Patient enrollment, a significant factor in the timing of clinical trials, is affected by many factors, including the size and nature of the patient population and competition for patients with other trials. Genetic diseases generally, and especially the rare diseases for which some of our current and any future product candidates are targeted, have low incidence and prevalence. For example, the incidence of UCDs in the United States is estimated to be approximately 1 in 35,000 births, with similar incidence estimated for Europe, and accordingly it may be difficult for us to identify and timely recruit a sufficient number of eligible patients to conduct our clinical trials. Further, the pediatric population is an important patient population for CMP-CPS-001CMP-002 and our addressable patient population estimates include pediatric populations. However, it may be more challenging to conduct studies in this population, and to locate and enroll pediatric patients. Additionally, pediatric and adolescent patients with SYNGAP1 frequently present with developmental delays, intellectual disabilities, and behavioral problems, and it may be challenging to ensure that pediatric or adolescent patientsthey adhere to clinical trial protocols. We may not be able to identify, recruit and enroll a sufficient number of patients, or those with required or desired characteristics, to complete our clinical trials in a timely manner. Patient enrollment and trial completion is affected by factors including:

We have not completed a clinical trial of any product candidate. It is impossible to predict when or if any of our current or future product candidates will prove safe in humans. There can be no assurance that our product candidates will not cause undesirable side effects.

If in the future we are unable to demonstrate that such side effects were caused by factors other than our product candidates, the FDA, the TGA, EC, NCAsFDA or other regulatory authorities could order us to cease further development of, or deny approval of, any product candidates for any or all targeted indications. Even if we are able to demonstrate that any future serious adverse events are not product-related and regulatory authorities do not order us to cease further development of our product candidates, such occurrences could affect patient recruitment or the ability of enrolled patients to complete the trial. Moreover, if we elect, or are required, to delay, suspend or terminate any clinical trial of any product candidate, the commercial prospects of such product candidates may be harmed and our ability to generate product revenues from any of these product candidates may be delayed or eliminated. Any of these occurrences may harm our ability to develop other product candidates, and may harm our business, financial condition and prospects significantly.

Because we have limited financial and managerial resources, we focus on research programs and product candidates that we identify for specific indications that we believe can be addressed by our technology among many potential options. As a result, we may forego or delay pursuit of opportunities with other product candidates or for other indications that later prove to have greater commercial potential, or we may choose to focus our efforts and resources on a potential product candidate that ultimately proves to be unsuccessful. For example, we have announced our intention, pending successful completion of GLP toxicology studies and regulatory clearance, to initiate a global Phase 1/2 clinical trial in individuals with SYNGAP1 as early as the second half of 2026 and to pursue partnership opportunities to support the further development of CMP-001 for individuals with urea cycle disorders. Our resource allocation decisions may cause us to fail to capitalize on viable commercial products or profitable market opportunities. Our spending on current and future research and development programs and product candidates for specific indications may not yield any commercially viable products. If we do not accurately evaluate the commercial potential or target market for a particular product candidate, we may relinquish valuable rights to that product candidate through collaboration, licensing or other royalty arrangements in cases in which it would have been more advantageous for us to retain sole development and commercialization rights to such product candidate. Any such event could have a material adverse effect on our business, financial condition, results of operations and prospects.

We are conducting and intend to conduct certain of our clinical trials globally. However, the FDA and other foreign equivalents may not accept data from such trials, in which case our development plans will be delayed, which could materially harm our business.

We intend to continue conductingconduct certain of our clinical trials globally. The acceptance by the FDA or other regulatory authorities of study data from clinical trials conducted outside their jurisdiction may be subject to certain conditions or may not be accepted at all. In cases where data from foreign clinical trials are intended to serve as the sole basis for marketing approval in the United States, the FDA will generally not approve the application on the basis of foreign data alone unless (i) the data are applicable to the U.S. population and U.S. medical practice; (ii) the trials were performed by clinical investigators of recognized competence and pursuant to GCP regulations; and (iii) the data may be considered valid without the need for an on-site inspection by the FDA, or if the FDA considers such inspection to be necessary, the FDA is able to validate the data through an on-site inspection or other appropriate means.

Certain of our research and product development initiatives are focused on treatments for rare diseases. Given the small number of patients who have certain of the diseases that we are currently targeting, including UCDs and SYNGAP1-related disorders,SYNGAP1, it is critical to our ability to grow and become profitable that we continue to successfully identify patients with these rare diseases. Our projections of both the number of people who have these diseases, as well as the subset of people with these diseases who have the potential to benefit from treatment with any product candidates we may develop, are based on our beliefs and estimates. These estimates have been derived from a variety of sources, including the scientific literature, surveys of clinics, patient foundations or market research that we conducted, and may prove to be incorrect or contain errors. New studies may change the estimated incidence or prevalence of these diseases. The number of patients may turn out to be lower than expected. The effort to identify patients with diseases we seek to treat is in early stages, and we cannot accurately predict the number of patients for whom treatment might be possible. Additionally, the potentially addressable patient population for each of our product candidates may be limited or may not be amenable to treatment with our product candidates, and new patients may become increasingly difficult to identify or gain access to, which would adversely affect our results of operations and our business. Further, even if we obtain significant market share for our product candidates, because the potential target populations are very small, we may never achieve profitability despite obtaining such significant market share.

Any product candidates we may develop and the activities associated with their development and commercialization, including their design, testing, manufacture, safety, efficacy, potential confirmatory studies, recordkeeping, labeling, storage, approval, advertising, promotion, sale and distribution, are subject to comprehensive regulation by the FDA and other regulatory authorities in the United States and by comparable authorities in other countries, including the TGA in Australia, the EC and the NCAs in the European Union, and by the Medicines and Healthcare products Regulatory Agency (the “MHRA”) in the United Kingdom. Failure to obtain marketing approval for a product candidate we may develop will prevent us from commercializing the product candidate in a given jurisdiction. We have not received approval to market any product candidates from regulatory authorities in any jurisdiction. We have no experience as a company in filing and supporting the applications necessary to gain marketing approvals and expect to utilize or rely on third-party experts, CROs, and other competent groups and/or individuals to assist us in this process. Securing regulatory approval requires the submission of extensive preclinical and clinical data and supporting information to the various regulatory authorities for each therapeutic indication to establish the product candidate’s safety and efficacy. Securing regulatory approval also requires the submission of information about the product manufacturing process to, and inspection of manufacturing facilities by, the relevant regulatory authority. Any product candidates we may develop may not be effective, may be only moderately effective or may prove to have undesirable or unintended side effects, toxicities, or other characteristics that may preclude our obtaining marketing approval or prevent or limit commercial use.

We have received orphan drug designation for CMP-CPS-001CMP-001 for the treatment of UCDs, and we may pursue orphan drug designation for certain of our other product candidates. We may not be able to obtain or maintain the benefits of orphan drug designation, including potential orphan drug exclusivity, and even if we do, that exclusivity may not prevent regulatory authorities from approving other competing products.

The FDA granted orphan drug designation to CMP-CPS-001CMP-001 for the treatment of UCDs in September 2024; however, we may not be able to obtain or maintain the benefits of such designation, including potential orphan drug exclusivity. Additionally, we may seek orphan drug designation for certain of our other product candidates in the future; however, we may never receive such designations. Under the Orphan Drug Act, the FDA may designate a product candidate as an orphan drug if it is a drug intended to treat a rare disease or condition, defined as a patient population of fewer than 200,000 in the United States, or a patient population greater than 200,000 in the United States where there is no reasonable expectation that the cost of developing the drug will be recovered from sales in the United States Orphan drug designation must be requested before submitting an NDA. A similar regulatory scheme governs orphan products in the EU and the United Kingdom based on, among others, prevalence of the disease or condition of less than 5 in 10,000.

The decision of the U.S. Court of Appeals for the 11th Circuit in Catalyst Pharms., Inc. v. Becerra, 14 F.4th 1299 (11th Cir. 2021) has created uncertainty regarding the scope of orphan drug exclusivity. Although the FDA subsequently announced that it intends to continue to apply its longstanding interpretation of the regulations to matters outside of the scope of the Catalyst order and continue tying the scope of orphan-drug exclusivity to the uses or indications for which a drug is approved,Finally, it is unclear how future litigation, legislation, agency decisions, andor administrative actions willmay impact the scope of the orphan drug exclusivity. The FDA may further reevaluate the Orphan Drug Act and its regulations and policies. We do not know if, when, or how the FDA may change the orphan drug regulations and policies in the future, and it is uncertain how any changes might affect our business. Depending on what changes the FDA may make to its orphan drug regulations and policies, our business could be adversely impacted.

We have received rare pediatric disease designation from the FDA for CMP-CPS-001CMP-001 for the treatment of UCDsUCDs, and we may pursue rare pediatric disease designation for certain of our other product candidates; however, there is no guarantee that a marketing application for CMP-CPS-001,such product candidates, if approved, will qualify for a rare pediatric disease priority review voucher.

Under the Rare Pediatric Disease Priority Review Voucher (“PRV”) program, a sponsor of an NDA that receives approval for a drug for a “rare pediatric disease” may qualify for a rare pediatric disease PRV that can be redeemed to obtain priority review for a subsequent application. UnderAlthough the currentPRV statutoryprogram sunsetwas provisions,reauthorized ain February 2026, the FDA’s authority to award rare pediatric disease productPRVs applicationis may be eligible for a rare pediatric disease PRV if the drug receives rare pediatric disease designation before December 20, 2024time-limited and receivesis marketingcurrently approvalscheduled beforeto terminate after September 30, 2026.2029. While we have obtained rare pediatric disease designation for CMP-CPS-001CMP-001 for the treatment of UCDs,UCDs itand ismay unlikely thatpursue this designation for other product candidates, if approval of such a product candidate will be approved before September 30, 2026. If approval is not obtained by then,September 30, 2029, we will not be eligible for a rare pediatric disease PRV,PRV unless Congress further reauthorizes the program beyond the current sunset date. Additionally, designation of a drug for a rare pediatric disease does not guarantee that an NDA for such product candidate will meet the criteria for a “rare pediatric disease priority product application” or be eligible for a rare pediatric disease PRV at the time the application is approved. The FDA may determine that a marketing application does not meet the eligibility criteria for a rare pediatric disease PRV for a number of reasons, including:

In addition, foreign regulatory authorities may change their approval policies and new regulations may be enacted. For instance, the EU pharmaceutical legislation is currently undergoing a complete revision, in the context of the Pharmaceutical Strategy for Europe initiative, launched by the European Commission in November 2020. The European Commission’s proposals for revision of several fundamental legislative instruments related to medicinal products, which may reduce the duration of regulatory data and marketing protection or exclusivity and revise the eligibility for expedited pathways in addition to other changes, was published on April 26, 2023. TheOn proposedDecember revisions11, are yet be finalized by2025, the EuropeanEU Parliament and Europeanthe CouncilCouncil, representing all EU Member States, reached agreement on a new directive and regulation that will significantly update EU pharmaceutical laws. The agreed text maintains eight years of regulatory data exclusivity for new active substances, with up to 11 years of market protection available through theadditional co-decisionincentives legislativesuch processas andaddressing theunmet proposalsmedical may therefore be substantially revised before adoption, which is not anticipated before early 2026. On April 10, 2024, the European Parliament adopted its position on the Commission proposal to reform. The revisions will however have aneeds, significant impactnew onindications, thecomparative pharmaceuticalclinical industry and our businesstrials in the longEU term.or early marketing authorization applications. For orphan medicinal products, the standard market exclusivity is nine years, while breakthrough orphan products entering therapeutic areas with no existing treatment will receive 11 years of exclusivity. To enhance patient access, Member States may require marketing authorization holders to supply medicines within three years of a request, with failure to do so resulting in a two-year reduction in exclusivity in that Member State.

For example, the Patient Protection and Affordable Care Act and Health Care and Education Reconciliation Act (the “Affordable Care Act”), which became law in the United States in 2010, contains provisions will become more salient to our business if any of our product candidates are approved. The Affordable Care Act includes, among other things, changes to the coverage and payments for products under government healthcare programs, profitability of drug products through increased rebates for drugs reimbursed by Medicaid programs, extension of Medicaid rebates to Medicaid managed care plans, mandatory discounts for certain Medicare Part D beneficiaries. and annual fees based on pharmaceutical companies’ share of sales to federal health carehealthcare programs. We may face uncertainties because of efforts to repeal, substantially modify or invalidate some or all of the provisions of the Affordable Care Act. There is no assurance that the Affordable Care Act, as currently enacted or as amended in the future, will not adversely affect our business and financial results, and we cannot predict how future federal or state legislative or administrative changes relating to healthcare reform will affect our business.

In addition, other legislative changes have been proposed and adopted since the Affordable Care Act was enacted. For example, beginning April 1, 2013, Medicare payments to providers were reduced under the sequestration required by the Budget Control Act of 2011, which will remain in effect through 2032, unless additional Congressional action is taken. Additionally, on January 2, 2013, the American Taxpayer Relief Act of 2012 was signed into law, which, among other things, further reduced Medicare payments to several providers, including hospitals, imaging centers, and cancer treatment centers, and increased the statute of limitations period for the government to recover overpayments to providers from three to five years. On March 11, 2021, the American Rescue Plan Act of 2021 was signed into law, which eliminateseliminated the statutory cap on Medicaid drug rebatesrebates, beginningeffective January 1, 2024.2024, and has allowed Medicaid to collect more from manufacturers for drugs with higher prices. The rebate was previously capped at 100% of a drug’s average manufacturer price. Additionally, the Inflation Reduction Act of 2022 includes several provisions such as drug pricing controls and Medicare redesign that are likely to impactcontinue impacting our business to varying degrees, but we cannot predict the exact nature of its ultimatefuture effecteffects on our business and the healthcare industry in generalgeneral. isAdditionally, notthe yetU.S. known.2025 reconciliation bill, or the “One Big Beautiful Bill Act,” which was signed into law on July 4, 2025, contains language that heavily impacts the Affordable Care Act’s Medicaid expansion and includes over $800 billion in Medicaid cuts. See “Healthcare lawsLaws and regulationRegulation in the United States-Healthcare reformReform” section for a more detailed discussion.

WeOur product development efforts are aenabled clinical-stageby biopharmaceuticalour companyproprietary pioneeringRAP thePlatform. discoveryWe leverage our RAP Platform to identify and developmentcharacterize of a new class of RNA-based therapeutics with the goal of upregulating gene expression and restoring healthy protein levels to treat a broad range of genetic diseases. Regulatoryregulatory RNAs (“regRNAs”), which play a central role in the regulation of every protein-coding gene by contributing to gene activation and suppression. Our approach is designed to amplify messenger RNA (“mRNA”) expression by harnessing the power of regRNAs that form localized complexes with transcription factors and regulate gene expression. Our proprietary RNA Actuating Platform, or RAP Platform,Platform allows us to rapidly and systematically identify and characterize the active regulatory elements controlling every expressed gene and tens of thousands of druggable enhancer and promoter regRNA sequences that control protein-coding genes. Once a disease-associated target gene is identified, we apply our RAP Platform to identify the controlling regRNA and rapidly generate novel antisenseASO oligonucleotide (“ASO”) candidates, which we also refer to as RNA Actuators.candidates. These ASOs are designed to bind to the identified regRNA and amplify the expression of the target gene in a specific and controllable way. We are currently focused on metabolic diseases and diseases of the central nervous system with validated disease biology, and we believe our RAP Platform allows us to address a broad range of rare and prevalent genetic diseases in which a modest increase in protein expression has the potential to be clinically meaningful.

Since our inception in 2015, we have focused substantially all of our resources primarily on developing our RAP Platform, identifying, developing and progressing our product candidates through preclinical and clinical development, organizing and staffing our company, conducting research and development (“R&D”) activities, establishing and protecting our intellectual property portfolio, and raising capital. To date, we have primarily funded our operations with proceeds from the sale of convertible preferred stock and common stock, including pursuant to an underwritten offering completed in December 2025, a private placement of our common stock and pre-funded warrants, the initial closing of which occurred in September 2025 (the “September 2025 private placement”), and our initial public offering (“IPO”), which closed on October 15, 2024, as well as through revenues from our license and collaboration agreements. Through December 31, 2024,2025, we have received grossnet proceeds of $28.1 million from our December 2025 underwritten offering, $46.7 million from the September 2025 private placement, $72.4 million from our IPO and $188.3 million from the sale of our convertible preferred stock.stock prior to our IPO. In addition, through December 31, 2024,2025, we have recognized $18.0$21.5 million in research collaboration and license revenue through our development and license agreements. Our ability to generate any product revenue and, in particular, our ability to generate product revenue sufficient to achieve profitability, will depend on the successful development and eventual commercialization of product candidates.

We have incurred significant operating losses and negative cash flows from operations since our inception. Our net losses were $51.8$80.4 million and $49.3$51.8 million for the years ended December 31, 20242025 and 2023,2024, respectively. As of December 31, 2024,2025, we had an accumulated deficit of $211.8$292.2 million. Substantially all of our net losses have resulted from costs incurred in connection with our R&D programs and, to a lesser extent, from general and administrative (“G&A”) costs associated with our operations. Our net losses may fluctuate significantly from quarter-to-quarter and year-to-year, depending on the timing of our clinical trials and preclinical studies, our other R&D activities and capital expenditures, and the timing and amount of any milestone or royalty payments due under our existing or future license or collaboration agreements. In addition, following the closing of our IPO, we expect to incur additional costs associated with operating as a public company, including significant legal, audit, accounting, regulatory and tax-related services associated with maintaining compliance with exchange listing and requirements of the Securities and Exchange Commission (“SEC”), director and officer liability insurance costs, investor and public relations costs, and other expenses that we did not incur as a private company. If we obtain regulatory approval for our product candidates, we expect to incur significant expenses related to developing our commercialization capability to support product sales, marketing and distribution. We anticipate that our expenses will increase substantially if and as we:

•finalize preclinical development for ourCMP-002 programand inadvance SYNGAP1-relatedinto disordersclinical trials;

•contract with third-party manufacturers for preclinical and clinical drug supply supportingto support any future product candidates we may develop,develop and for commercial supply with respect to any such product candidates that receive regulatory approval;

•add operational, legal, compliance, financial and management information systems and personnel to support our research, product development and future commercialization efforts, as well as to support our operations as a public company.efforts.

As of December 31, 2024,2025, we had cash and cash equivalents of $64.0$109.5 million. Based on our current operating plan, we estimate that our cash and cash equivalents as of December 31, 20242025 will be sufficient to fund our operating expenses and capital expenditure requirements into the second quarter of 2026.2028. However, we have based this estimate on assumptions that may prove to be wrong, and we could deplete our capital resources sooner than we currently expect. Our capital resources may not be sufficient to fund operations through at least the next twelve months from the date the accompanying audited consolidated financial statements as of December 31, 2024 are issued based on our expected cash needs, which raises substantial doubt about our ability to continue as a going concern. See the sections titled “—Liquidity and Capital Resources” and “Risk Factors—Risks Related to our Financial Position and Need for Additional Capital” included elsewhere in this Annual Report.

We do not own or operate,operate and currently have no plans to establish,establish any manufacturing facilities. We rely, and expect to continue to rely, on third parties for clinical supply as well as commercial supply if we obtain marketing approval. In addition, we rely on third parties to package, label, store, and distribute our clinical supply and we intend to rely on third parties to conduct the same activities for our commercial products if we obtain regulatory approval. We believe that this strategy allows us to maintain a more efficient infrastructure by eliminating the need for us to invest in our own manufacturing facilities, equipment, and personnel while also enabling us to focus our expertise and resources on the development of product candidates and continued enhancement of our RAP Platform.

In April 2018, we entered into a development and license agreement (the “CMCC Agreement”) with Children’s Medical Center Corporation (“CMCC”). The CMCC Agreement allows us to use CMCC’s proprietary intellectual property to conduct research, development and commercialization of products utilizing CMCC’s proprietary intellectual property in return for specified payments. The proprietary intellectual property licensed pursuant to this agreement is related to certain legacy programs we are not pursuing andpursuing, which were subsequently sublicensed to Fulcrum Therapeutics, Inc. (“Fulcrum”), as described below. As part of the CMCC Agreement, we issued a total of 15,123 shares of common stock to CMCC and its affiliates based on the fair value of the common stock on the date of issuance.

We are obligated to pay potential development milestone payments under the terms of the CMCC Agreement of up to $7.7 million for the first licensed target, $3.9 million for the second licensed target and $1.9 million for the third licensed target upon the achievement of certain specified contingent events. If commercial sales of a licensed product commence, we will pay CMCC royalties at percentage rates ranging in the low- to mid-single digits on net sales of licensed products in countries where such product is protected by patent rights. We incurred de minimis royalties owed to CMCC for each of the years ended December 31, 20242025 and 20232024 under the CMCC Agreement and recorded the amounts in R&D expense in the consolidated statement of operations and comprehensive loss. Further, under the terms of the CMCC Agreement, we are required to pay 10% of any upfront payment received under a sublicensing agreement entered into prior to the initiation of the first investigational new drug study. As such, we recorded de minimis amounts for each of the years ended December 31, 20242025 and 2023,2024, which is presented in R&D expenses onin the consolidated statements of operations and comprehensive loss. We reevaluate the likelihood of achieving future milestones at the end of each reporting period. As of December 31, 2024,2025, we determined that the likelihood of achieving future milestones was not probable.

We are also obligated to pay potential development milestone payments to Whitehead of up to $1.9 million upon the achievement of certain specified contingent events. In addition, if we successfully commercialize a product under the Whitehead Agreement, we are obligated to pay tiered royalties at percentage rates ranging from less than one percent to the mid-single digits of net sales or of running royalties of net sales, subject to specified reductions, until either the last-to-expire valid claim of a Whitehead patent covering the product or seven years after the first commercial sale, in each case on a product-by-product and country-by-country basis. We incurred fees of $0.2 million and $0.3 million under the Whitehead Agreement during the year ended December 31, 2024.2025 Duringand the2024, year ended December 31, 2023, the fees were de minimis.respectfully. The fees are recorded in our R&D expense in our consolidated statements of operations and comprehensive loss.

During the year ended December 31, 2023,2025, we recorded $0.4$0.6 million in license revenue in our consolidated statements of operations and comprehensive loss as a result of the receipt of a milestone payment pursuant to the Fulcrum Agreement and none in the year ended December 31, 2024.

In July 2023, we executed a Material Transfer Agreement (as amended, the “MTA”), with Eli Lilly and Company (“Eli Lilly”). As part of the MTA, we and Eli Lilly agreed to perform R&D activities to generate up to three ASOs in accordance with a prescribed workplan.work plan. As of December 31, 2025, we had completed all of the R&D activities allocated to us under the work plan and the MTA had expired in accordance with its terms. We and Eli Lilly are jointly overseeingoversaw the R&D activities under the MTA. In addition, both parties arewere exposed to the significant risks and potential rewards under the MTA. During each of the years ended December 31, 20242025 and 2023,2024, we recorded $0.1 million and $0.5 millionmillion, respectively, as a reduction in R&D expense in the consolidated statement of operations and comprehensive loss as a result of the earned R&D reimbursement from Eli Lilly. Additionally, we had ana de minimis unbilled receivable of $0.1 million recorded within prepaid expenses and other current assets on our consolidated balance sheet as of December 31, 2023 and a de minimis deferred liability as of December 31, 2024.

Research and Collaboration AgreementAgreements

In September 2024, we entered into a Collaboration and License Agreement (the “BioMarin Agreement”) with BioMarin Pharmaceutical Inc. (“BioMarin”), pursuant to which we and BioMarin agreed to collaborate with respect to the research and discovery of regulatory RNA-targeting ASOs using our proprietary RAP Platform to modulate the expression of two undisclosed genetic targets under two distinct programs. Under the terms of the BioMarin Agreement, BioMarin paid us an upfront, nonrefundable payment of $1.0 million, and will reimbursereimbursed us for certain research activities. OnIn aNovember per-program2025, basis,BioMarin weprovided willnotice beof eligibleits election to receiveterminate upthe toBioMarin $5.0Agreement, millionwhich termination became effective in futureFebruary contingent preclinical milestones, up to $75.0 million in future contingent development and regulatory milestones and up to $105.0 million in commercial sales milestones. We will be further eligible to receive tiered royalties at percentage rates ranging from low to high single digits of net sales, subject to specified reductions, until either the last-to-expire valid claim of a patent covering the product, ten years after the first commercial sale, or the expiration of any applicable regulatory exclusivity obtained for the product, in each case on a product-by-product and country-by-country basis. The agreement may be terminated in its entirety or on a program-by-program basis for convenience by BioMarin. The agreement may also be terminated by either us or BioMarin under certain other circumstances, including material breach, as set forth in the agreement. The notice periods for termination provisions range from 30 days to 270 days depending on the reason for termination.2026. During the yearyears ended December 31, 2025 and 2024, we recognized $2.9 million and $0.7 millionmillion, respectively, in collaboration revenue under the BioMarin Agreement.

For the years ended December 31, 20242025 and 2023,2024, we recognized $0.7$3.5 million and $0.4$0.7 million, respectively, in research and collaboration revenue through our collaboration and license agreements. We have not generated any revenue from the sale of products, however, and do not expect to generate any revenue from the sale of products in the foreseeable future, if at all. If our or our collaborators’ development efforts for product candidates andor any future product candidates are successful and result in regulatory approval, we may generate revenue in the future from product sales, payments from existing or potential future collaboration or license agreements with third parties, or any combination thereof.

Our operating expenses consist of R&D expenses andexpenses, G&A expenses.expenses, and impairment of right-of-use asset.

We expense R&D costs as incurred. Certain third-party costs for R&D activities are recognized based on an evaluation of the progress to completion of specific tasks using information and data provided to us by our management and scientific personnel, vendors and third-party service providers. Non-refundable advance payments for goods and services that will be used over time for R&D are deferred and capitalized as R&D prepaid expenses on our consolidated balance sheets. The capitalized amounts are recognized as an expense as the goods are delivered or as the related services are performed. Since our inception, substantially all of our external costs have been related to the development of product candidates. We use internal resources for platform development, early pipeline discovery, preclinical development, management of clinical development activities, technical operations and oversight of manufacturing partners. We do not track our R&D expenses on a program-by-program basis. Our third-party R&D expenses consist primarily of fees paid to outside consultants, CROs, CMOs and academic research laboratories in connection with our preclinical development, process development, manufacturing and clinical development activities. Our other R&D costs are internal costs primarily associated with our discovery efforts, laboratory supplies, and facilities, including depreciation, that are allocated across multiple programs.

•per patientsubject trial costs;

•the length of time required to enroll eligible patientssubjects;

•the number of patientssubjects that participate in the trials;

•the number of doses that patientssubjects receive;

•the drop-out or discontinuation rates of patientssubjects;

•the duration of patientsubjects participation in the trials and follow-up;

Other (expense) Income, Net

Other (expense) income, net primarily consists of the change in fair value of our derivative tranche liability associated with the conditional second tranche of our September 2025 private placement, interest income earned on our invested cash and cash equivalent balances and interest expense incurred on our finance lease and financing liabilities,balances, as well as other insignificant amounts.

Research and collaboration revenue was $3.5 million for the year ended December 31, 2025, compared to $0.7 million for the year ended December 31, 2024, compared to $0.4 million for the year ended December 31, 2023.2024. The increase of $0.3$2.8 million was primarily due to $0.7a $2.2 million ofincrease in revenue recognized under the BioMarin Agreement indriven theby yearincreased endedperformance Decemberactivities 31,and 2024,related comparedcosts toincurred $0.4during 2025 and $0.6 million of revenue recognized under the Fulcrum Agreement as a result of the receipt of a milestone payment in the year ended December 31, 2023.2025.

Other (Expense) Income, Net

As of December 31, 2024,2025, we had cash and cash equivalents of $64.0$109.5 million. Based on our current operating plan, we estimate that our cash and cash equivalents as of December 31, 20242025 will be sufficient to fund our operating expenses and capital expenditure requirements into the second quarter of 2026.2028. However, we have based this estimate on assumptions that may prove to be wrong, and we could deplete our capital resources sooner than we currently expect. Our capital resources may not be sufficient to fund operations through at least the next twelve months from the date the accompanying audited consolidated financial statements as of December 31, 2024 are issued based on our expected cash needs, which raises substantial doubt about our ability to continue as a going concern. In their report accompanying our audited financial statements for the years ended December 31, 2024 and 2023, our independent registered public accounting firm included an explanatory paragraph stating that our recurring losses from operations raise substantial doubt about our ability to continue as a going concern. Our future viability is dependent on our ability to generate cash from our operating activities or to raise additional capital to finance our operations. There is no assurance that we will succeed in obtaining sufficient funding on terms acceptable to us to fund continuing operations, if at all.

The timing and amount of our future funding requirements will depend on many factors, including:

•the costs, timing and outcome of regulatory meetings and reviews of our product candidates or any future product candidates, including requirements of regulatory authorities in any additional jurisdictions in which we may seek approval and any future product candidates;

WeIn haveJanuary 2023, we entered into twoa non-cancellable operating leaseslease for our office and lab space in Cambridge, Massachusetts and Boulder, Colorado,Colorado (the “Boulder Lease”), which expireexpires on June 30, 2027 and September 30, 2028, respectively. See Note 7 to our consolidated financial statements for additional details related to our noncancellable operating leases.2028.

We are continuing to invest in the clinical development of CMP-CPS-001CMP-002 and have entered into contractual obligations with CROs relating to the performance of services for our planned Phase 1/2 clinical trial services.trial. Each contract shall continue until the completion of the trial.clinical trial or until terminated in accordance with its terms. Our clinical trial costs are dependent on, among other things, the sizescope, timing, and lengthduration of our preclinical and clinical trial.development activities. We also incur R&D costs related to the enhancement of our existing and future product candidates.

The timing of when we will paypayment or receivereceipt of royalty payments is uncertain as the paymentsroyalties are contingent upon future activities, including the successful discovery, development, regulatory approval and commercialization of product candidates.

Risks Related to RegulatoryOur ApprovalFinancial Position and CommercializationNeed for Additional Capital

The following discussion and analysis of our financial condition and results of operations and the unaudited interim condensed consolidated financial statements and related notes included in this Quarterly Report on Form 10-Q (the “Quarterly Report”) should be read in conjunction with the audited financial statements and related notes thereto in our Annual Report on Form 10-K for the fiscal year ended December 31, 2025, which was filed with the Securities and Exchange Commission (the “SEC”) on March 5, 2026 (the “2025 Form 10-K”). This discussion and analysis and other parts of this Quarterly Report contain forward-looking statements. Our actual results and the timing of selected events could differ materially from those described in or implied by these forward-looking statements. Factors that could cause or contribute to such differences include, but are not limited to, those discussed in the sections of this Quarterly Report titled “Special Note Regarding Forward-Looking Statements,Statements and Industry Data,” and those risk factors described in “Part I, Item 1A, Risk Factors” of our 2025 Form 10-K and in “Part II, Item 1A, Risk Factors” in this Quarterly Report.

CMP-002 is a novel, intrathecally delivered antisense oligonucleotide (“ASO”) designed to target the SYNGAP1 gene at the transcriptional level to increase gene expression, which may increase SYNGAP protein levels in amounts sufficient to yield therapeutic benefit. In preclinical studies, intracerebroventricular injection of CMP-002 restored SYNGAP protein levels to near normal range in haploinsufficient mice carrying a single copy of the human SYNGAP1 gene after a single dose and rescued motor defects and spatial learning defects following two doses. A single dose of CMP-002 also produced a statistically significant improvement in both seizure threshold and severity of chemically induced tonic-clonic seizures in SYNGAP1 haploinsufficient mice, suggesting the potential for therapeutic benefit across both the neurodevelopmental and seizure phenotypes that characterize SYNGAP1-related disorder. In addition, biweekly intrathecal injections of CMP-002 in cynomolgus monkeys were well tolerated and significantly increased SYNGAP protein levels across multiple brain regions clinically relevant to the disease, with dose-linear increases of CMP-002 in disease-relevant brain regions. We have submittedreceived clearance from Australia’s Therapeutic Goods Administration and Argentina’s Administración Nacional de Medicamentos, Alimentos y Tecnología Médica to initiate our firstPhase regulatory filing in Australia to enable initiation of a1/2 clinical trial of CMP-002 in individuals with SYNGAP1, withand we have submitted additional global regulatory filings planned in 2026.the PendingEuropean regulatoryUnion clearance,and weUnited Kingdom to support broader enrollment across multiple sites. We intend to initiate the global Phase 1/2 clinical trial in the secondfourth halfquarter of 2026.

Our primary therapeutic focus is on diseases of the central nervous system (“CNS”), where there are numerous rare and prevalent haploinsufficient diseases with no approved treatments for which a modest increase in protein expression has the potential to be clinically meaningful. In addition to our SYNGAP1 program, we are advancing discovery programs in other DEECNS indications. We also intend to leverage strategic discovery partnerships, including our research, collaboration, and license agreement with GlaxoSmithKline, to extend the application of our RAP Platform beyond the CNS and validate our approach to gene upregulation in additional tissues and disease areas.

Since our inception in 2015, we have focused substantially all of our resources primarily on developing our RAP Platform, identifying, developing and progressing our product candidates through preclinical and clinical development, organizing and staffing our company, conducting research and development (“R&D”) activities, establishing and protecting our intellectual property portfolio, and raising capital. To date, we have primarily funded our operations with proceeds from the sale of convertible preferred stock and common stock, including pursuant to an underwritten offering completed in December 2025, a private placement of our common stock and pre-funded warrants,warrants (the “Private Placement”), the initial closing of which occurred in September 2025 (the “SeptemberInitial 2025Closing”), privateand placementthe second closing of which occurred in August 2026 (the “Second Closing”), and our initial public offering (“IPO”), which closed on October 15, 2024, as well as through revenues from our license and collaboration agreements. Through MarchJune 31,30, 2026, we have received net proceeds of $28.1 million from our December 2025 underwritten offering, $46.7 million from the SeptemberInitial 2025Closing privateof placement,the Private Placement, $72.4 million from our IPO and $188.3 million from the sale of our convertible preferred stock prior to our IPO. In addition, through MarchJune 31,30, 2026, we have recognized $22.8$24.6 million in research collaboration and license revenue through our development and license agreements. Subsequent to June 30, 2026, we received net proceeds of $46.9 from the Second Closing of the Private Placement. Our ability to generate any product revenue and, in particular, our ability to generate product revenue sufficient to achieve profitability, will depend on the successful development and eventual commercialization of product candidates.

We have incurred significant operating losses and negative cash flows from operations since our inception. Our net losses were $18.3$51.9 million and $12.4$25.0 million for the threesix months ended MarchJune 31,30, 2026 and 2025, respectively. As of MarchJune 31,30, 2026, we had an accumulated deficit of $310.5$344.0 million. Substantially all our net losses have resulted from costs incurred in connection with our R&D programs and, to a lesser extent, from general and administrative (“G&A”) costs associated with our operations. Our net losses may fluctuate significantly from quarter-to-quarter and year-to-year, depending on the timing of our clinical trials and preclinical studies, our other R&D activities and capital expenditures, and the timing and amount of any milestone or royalty payments due under our existing or future license or collaboration agreements. In addition, we incur additional costs associated with operating as a public company, including significant legal, audit, accounting, regulatory and tax-related services associated with maintaining compliance with exchange listing and requirements of the Securities and Exchange Commission (“SEC”), director and officer liability insurance costs, investor and public relations costs, and other expenses that we did not incur as a private company. If we obtain regulatory approval for our product candidates, we expect to incur significant expenses related to developing our commercialization capability to support product sales, marketing and distribution.

As of MarchJune 31,30, 2026, we had cash and cash equivalents of $99.2$86.4 million. Based on our current operating plan, we estimate that our cash and cash equivalents as of MarchJune 31,30, 20262026, together with the net proceeds of $46.9 million received from the Second Closing under the Purchase Agreement in August 2026, will be sufficient to fund our operating expenses and capital expenditure requirements intothrough the end of 2028. However, we have based this estimate on assumptions that may prove to be wrong, and we could deplete our capital resources sooner than we currently expect. See the sections titled “—Liquidity and Capital Resources” and “Risk Factors—Risks Related to our Financial Position and Need for Additional Capital” included in our 2025 Form 10-K.

We do not own or operate and currently have no plans to establish any manufacturing facilities. We rely, and expect to continue to rely, on third parties for preclinical and clinical supply as well as commercial supply if we obtain marketing approval. In addition, we rely on third parties to package, label, store, and distribute our clinical supply and we intend to rely on third parties to conduct the same activities for our commercial products if we obtain regulatory approval. We believe that this strategy allows us to maintain a more efficient infrastructure by eliminating the need for us to invest in our own manufacturing facilities, equipment, and personnel while also enabling us to focus our expertise and resources on the development of product candidates and continued enhancement of our RAP Platform.

Comparison of the Three Months Ended MarchJune 31,30, 2026 and 2025

The following table summarizes our results of operations for the three months ended MarchJune 31,30, 2026 and 2025 (in thousands):

We recognized $1.3$1.8 million in research and collaboration revenue during the three months ended MarchJune 31,30, 2026, compared to $0.9$1.5 million during the three months ended MarchJune 31,30, 2025. Research and collaboration revenue recognized in the three months ended MarchJune 31,30, 2026 primarily related to our Research, Collaboration and License Agreement with GlaxoSmithKline Intellectual Property (No. 3) Limited (“GSK”). and researchResearch and collaboration revenue recognized in the three months ended MarchJune 31,30, 2025 included $0.9 million related to our Collaboration and License Agreement with BioMarin.BioMarin and a $0.6 million milestone payment earned under the Fulcrum Agreement.

The following table summarizes our R&D expenses for the three months ended MarchJune 31,30, 2026 and 2025 (in thousands):

R&D expenses were $10.2$10.8 million for the three months ended MarchJune 31,30, 2026, compared to $10.1$10.3 million for the three months ended MarchJune 31,30, 2025. The nominalincrease increaseof $0.5 million was primarily driven by higher personnel-related costs of $0.3$0.5 million due to increased stock-based compensation expense and higheran increase of $0.4 million in clinical and preclinical costs of $0.2 million as we continue to advance CMP-002, including entering into contractual obligations with a contract research organization for services related to our planned Phase 1/2 clinical trial. These increases were partially offset by a decreasedecreases of $0.5$0.2 million in professional and consulting fees.fees and $0.1 million in facilities-related and overhead expense.

The following table summarizes our G&A expenses for the three months ended MarchJune 31,30, 2026 and 2025 (in thousands):

G&A expenses were $4.3 million for the three months ended June 30, 2026, compared to $4.2 million for the three months ended MarchJune 31, 2026, compared to $3.8 million for the three months ended March 31,30, 2025. The increase of $0.4$0.2 million was primarily driven by an increase in other expenses of $0.2 million, primarily related to travel expenses, and an increase of $0.1 million in personnel-related costs of $0.4 million due to increased stock-based compensation expense and an increase of $0.3 million in professional and consulting fees related to audit and tax costs.expense. These increases were partially offset by a decrease of $0.2 million in facilities and overhead expenses dueresulting tofrom the rent abatement period starting in October 2025 associated with the December 2025 lease modification and a decrease of $0.1 million due to a reduction in state franchise taxes.modification.

Other (expense) income, net for the three months ended MarchJune 31,30, 2026 was a $5.3$20.2 million expense, compared to $0.7$0.4 million of income for the three months ended MarchJune 31,30, 2025. The change was primarily due to a $6.2$20.9 million non-cash expense for the change in fair value of our derivative tranche liability associated with the Second Closing of ourthe Private Placement. The derivative tranche liability was subsequently settled in connection with the Second Closing, which occurred on August 3, 2026. This expense was partially offset by a $0.3$0.4 million increase in interest income due to higher average invested cash balances during the three months ended MarchJune 31,30, 2026.

Since our inception, we have not generated any revenue from product sales and have incurred significant operating losses and negative cash flows from operations. We expect to incur significant expenses and operating losses in the foreseeable future as we advance the development of product candidates. Through MarchJune 31,30, 2026, we have primarily funded our operations with proceeds from the sale of our equity securities and revenues from our license and collaboration agreements.

In connection with the filing of the Shelf Registration Statement, we also entered into a sales agreement (the “Sales Agreement”) with Leerink Partners LLC, as sales agent, pursuant to which we may issue and sell shares of our common stock for a maximum aggregate offering price of up to $100.0 million, which is included in the $300.0 million of securities that may be offered pursuant to the Shelf Registration Statement. Pursuant to the Sales Agreement, we will pay the sales agent a commission rate of up to 3.0% of the gross proceeds from the sale of any shares of our common stock. We are not obligated to make any sales of shares of our common stock under the Sales Agreement. During the three and six months ended MarchJune 31,30, 2026 and the year ended December 31, 2025, we did not issue any shares of our common stock under the Sales Agreement.

As of MarchJune 31,30, 2026, we had cash and cash equivalents of $99.2$86.4 million. Based on our current operating plan, we estimate that our cash and cash equivalents as of MarchJune 31,30, 20262026, together with the net proceeds of $46.9 million received from the Second Closing under the Purchase Agreement in August 2026, will be sufficient to fund our operating expenses and capital expenditure requirements intothrough the end of 2028. However, we have based this estimate on assumptions that may prove to be wrong, and we could deplete our capital resources sooner than we currently expect. Our future viability is dependent on our ability to generate cash from our operating activities or to raise additional capital to finance our operations. There is no assurance that we will succeed in obtaining sufficient funding on terms acceptable to us to fund continuing operations, if at all.

As of MarchJune 31,30, 2026, other than those disclosed within Notes 4, 5, and 6 to our condensed consolidated financial statements, there have been no material changes to our contractual obligations and commitments from those described under “Management’s Discussion and Analysis of Financial Condition and Results of Operations” included in our 2025 Form 10-K.

For the ThreeSix Months Ended MarchJune 31,30, 2026 and 2025

The following table provides information regarding our cash flows for the threesix months ended MarchJune 31,30, 2026 and 2025 (in thousands):

During the threesix months ended MarchJune 31,30, 2026, operating activities used $11.0$23.7 million of cash, primarily resulting from our net loss of $18.3$51.9 million,million and netthe cash used in changes in our operating assets and liabilitiestiming of $0.9working capital fluctuations of $3.5 million, partially offset by non-cash charges of $8.2$31.7 million, including stock-based compensation expense, loss from the change in fair value of our derivative tranche liability, non-cash operating lease expense, and depreciation and amortization.

During the threesix months ended MarchJune 31,30, 2025, operating activities used $14.3$24.6 million of cash, primarily resulting from our net loss of $12.4$25.0 million and netthe cash used in changes in our operating assets and liabilitiestiming of $3.7working capital fluctuations of $3.3 million, partially offset by non-cash charges of $1.9$3.7 million, including stock-based compensation expense, non-cash operating lease expense, and depreciation and amortization.

In each of the threesix months ended MarchJune 31,30, 2026 and 2025, cash used in operations was primarily related to clinical and preclinical efforts, compensation and benefits for our employees, consulting and other professional fees, and rent and overhead for our Cambridge and Boulder leases.

During the threesix months ended MarchJune 31,30, 2026 and 2025, net cash used in investing activities was $0.1 million and $0.3 million, respectively, due to purchases of property and equipment.

During the threesix months ended MarchJune 31,30, 2026, net cash provided by financing activities was $0.1 million, consisting primarily of proceeds from the exercise of stock options and issuance of common stock under the 2024 Employee Stock Purchase Plan.

During the threesix months ended MarchJune 31,30, 2025, net cash used byin financing activities was $0.2$0.1 million, consisting primarily of principal payments on finance leases.

There have been no significant changes to our critical accounting estimates in the preparation of our condensed consolidated financial statements during the threesix months ended MarchJune 31,30, 2026 compared to those disclosed in our 2025 Form 10-K.