Camp4 Therapeutics Corp
For the period ended Dec 31, 2025. Filed Mar 5, 2026.
View on SEC.govAbout this filing
A company's yearly report to the SEC, with audited financial statements. It is the fullest account a company gives of its business, its finances and the risks it sees.
FY2025 · Filed Mar 5, 2026
What changed in Management’s Discussion and Analysis?
Read the current filingPrevious report: 10-K
Period ended 2024-12-31; filed 2025-03-27.
0001736730-25-000022
Item 7. Managements Discussion and Analysis of Financial Condition and Results of OperationsCurrent report: 10-K
Period ended 2025-12-31; filed 2026-03-05.
0001736730-26-000016
Item 7. Management’s Discussion and Analysis of Financial Condition and Results of OperationsThe baseline is this issuer’s latest earlier reporting period of the same form that was filed before the current report. A previous 10-Q is a different quarter, not a year-over-year comparison. Amendments are excluded.
Compared 163 previous paragraphs and 161 current paragraphs.
96 unchanged; 38 reworded; 26 added; 29 removed; 0 previous and 1 current paragraphs rearranged.
Tables are not compared: 4 previous and 4 current tables excluded. Dates, whitespace, punctuation and numbering changes are ignored; changes to financial figures remain significant.
A changed paragraph is not evidence that risk increased or decreased. An unchanged paragraph does not establish that risk stayed the same.
Added paragraphs (26)
Current report
We are a clinical-stage biopharmaceutical company pioneering the discovery and development of a new class of RNA-targeting therapeutics with the goal of upregulating gene expression and restoring healthy protein levels to treat a broad range of genetic diseases. Our lead product candidate, CMP-002, has the potential to be the first disease-modifying therapy for the treatment of synaptic Ras GTPase activating protein 1 (“SYNGAP1”)-related disorder, or SYNGAP1, a severe developmental and epileptic encephalopathy (“DEE”) characterized by seizures, developmental delays, and cognitive impairments. SYNGAP1 is caused by haploinsufficiency of the SYNGAP1 gene, where mutation of one functional gene copy results in a reduction in SYNGAP protein levels of up to 50%. While we believe that it remains underdiagnosed, we estimate that there are approximately 21,000 individuals living with SYNGAP1 in the United States and the five largest European markets. There are no approved disease-modifying therapies for SYNGAP1.
Current 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. 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 initiated Good Laboratory Practice (“GLP”) toxicology studies for CMP-002 to support clinical trial applications and, pending successful completion and regulatory clearance, we intend to initiate a global Phase 1/2 clinical trial in individuals with SYNGAP1 as early as the second half of 2026.
Current report
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 DEE 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.
Current report
•finalize preclinical development for CMP-002 and advance into clinical trials;
Current report
•advance current and future product candidates through preclinical studies and clinical trials;
Current report
Research and Collaboration Agreements
Current report
GlaxoSmithKline Intellectual Property (No. 3) Limited (“GSK”)
Current report
In December 2025, we entered into a Research, Collaboration and License Agreement (the “GSK Agreement”) with GlaxoSmithKline Intellectual Property (No. 3) Limited (“GSK”), pursuant to which we and GSK have agreed to collaborate on the research and development of ASO therapeutics targeting regRNAs associated with multiple gene targets relevant to neurodegenerative and kidney disease indications (the “Collaboration Targets”). Under the terms of the GSK Agreement, GSK paid us a one-time, nonrefundable upfront payment of $17.5 million. In addition, we are eligible to receive up to $440.0 million in development and commercial milestone payments, subject to achievement of specified criteria, as well as tiered royalties on annual net sales of licensed products ranging from the low- to mid-single digits during a defined royalty term for each licensed product. The GSK Agreement may be terminated in its entirety or on a Collaboration Target-by-Collaboration Target basis (as defined in the GSK Agreement) for convenience by GSK and may also be terminated by either us or GSK under certain other circumstances, including material breach, as set forth in the GSK Agreement. The term of the GSK Agreement continues on a country-by-country and product-by-product basis until the expiration of the applicable royalty term, unless terminated earlier in accordance with its terms. There was no collaboration revenue recognized under the GSK Agreement during the year ended December 31, 2025.
Current report
•per subject trial costs;
Current report
Impairment of right-of-use asset
Current report
Impairment of right-of-use asset expenses is due to the impairment of our Boulder, Colorado lease's right-of-use asset. Upon vacating our Boulder location on August 29, 2025 and pursuing sublease opportunities, it was determined that the market rate for similar space is less than the base rent we are paying under the current lease. The Boulder, Colorado lease is set to expire on September 30, 2028.
Current report
Other (expense) Income, Net
Current report
R&D expenses were $38.2 million for the year ended December 31, 2025, compared to $38.8 million for the year ended December 31, 2024. The decrease of $0.6 million was primarily due to a $0.7 million decrease in clinical and preclinical expenses as we made the strategic decision to pause further investment in CMP-001. Additionally, a decrease of$0.4 million in facility-related and overhead expense due to a gain recognized in connection with the modification of our non-cancellable operating lease for office and lab space in Cambridge, Massachusetts (the “Cambridge Lease”), of which$0.2 million was included in R&D expense. These decreases were partially offset by increases in other expenses related to non-royalty sublicense fees of $0.1 million. We also incurred an increase of $0.2 million in personnel-related expenses due to lower offsetting personnel reimbursements under one of our collaboration agreements as well as severance and related costs resulting from separation agreements with certain former employees and an increase of $0.1 million in professional and consulting fees due to increased utilization of external support for clinical operations.
Current report
G&A expenses were $17.4 million for the year ended December 31, 2025, compared to $14.9 million for the year ended December 31, 2024. The increase of $2.4 million was primarily due to a $1.6 million increase in professional-related expenses, including $1.0 million of issuance costs and legal fees from our September 2025 private placement that were recognized in G&A expenses as a result of their allocation to the derivative tranche liability. The increase in facilities-related and overhead expense was driven by increased insurance premiums. In addition, there was an increase of $0.4 million in other expenses primarily due to an increase in state franchise taxes. Personnel-related expenses remained generally consistent year-over-year.
Current report
Impairment of Right-of-Use Asset
Current report
During the year ended December 31, 2025, upon vacating our Boulder, Colorado location and pursuing sublease opportunities, it was determined that the market rate for similar space is less than the base rent we are paying under our current lease. As a result, we impaired the related ROU asset and recognized an impairment charge of $0.5 million during the year ended December 31, 2025.
Current report
Other (Expense) Income, Net
Current report
Other (expense) income, net was an expense of $27.8 million for the year ended December 31, 2025, compared to income of $1.3 million for the year ended December 31, 2024. The change of $29.1 million was primarily due to a $29.8 million expense for the non-cash change in fair value of our derivative tranche liability associated with the conditional second tranche of our September 2025 private placement. This was partially offset by a $0.8 million increase in interest income due to higher average invested cash equivalent balances during the year ended December 31, 2025 and an increase in other expenses of $0.2 million primarily due to losses incurred related to the disposal of assets.
Current report
In November 2025, we filed a shelf registration statement on Form S-3 (the “Shelf Registration Statement”). Pursuant to the Shelf Registration Statement, we may offer and sell securities having an aggregate public offering price of up to $300.0 million.
Current report
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 year ended December 31, 2025, we did not issue any shares of our common stock under the Sales Agreement.
Current report
In October 2019, we entered into the Cambridge Lease, which was originally scheduled to expire on June 30, 2027.
Current report
In January 2023, we entered into a non-cancellable operating lease for office and lab space in Boulder, Colorado(the“Boulder Lease”), which expires on September 30, 2028.
Current report
In December 2025, we (i) entered into a non-cancellable operating lease for office and lab space in Watertown, Massachusetts (the“Watertown Lease”), which is targeted to commence 180 days after execution of the Watertown Lease and expires on June 30, 2030, and (ii) amended the Cambridge Lease to accelerate the termination date to the date that is thirty days after the commencement date of the Watertown Lease.
Current report
Valuation of Derivative Tranche Liability
Current report
In connection with the September 2025 private placement, we undertook a commitment to issue, and investors incurred an obligation to purchase, securities in a second, future closing at a fixed price, if specified conditions are met. The obligation to issue additional securities at a future date was determined to be a freestanding derivative instrument and is accounted for as a liability. The derivative tranche liability was accounted for at fair value at the issuance date and fair value is remeasured at the end of each reporting period until the shares are issued or the obligation expires. Changes in the fair value of the derivative tranche liability are recognized in the consolidated statement of operations.
Current report
The fair value of the derivative tranche liability was determined using a probability weighted model, which considered as inputs the probability of achieving tranche closing conditions, the estimated fair value of our common stock and a discount rate. We recognized a $29.8 million loss for the year ended December 31, 2025, related to the non-cash change in fair value of the derivative tranche liability.
Removed paragraphs (29)
Previous report
•advance our lead product candidate, CMP-CPS-001, through clinical trials;
Previous report
•finalize preclinical development for our program in SYNGAP1-related disorders;
Previous report
•conduct preclinical studies and clinical trials for our GBA1 discovery program for the treatment of Parkinson’s Disease (“PD”) and any future product candidates we may develop;
Previous report
Research and Collaboration Agreement
Previous report
•per patient trial costs;
Previous report
Other Income, Net
Previous report
R&D expenses were $38.8 million for the year ended December 31, 2024, compared to $40.6 million for the year ended December 31, 2023. The decrease of $1.8 million was primarily due to a decrease of $2.0 million in clinical and preclinical expenses primarily due to fewer external preclinical research activities, partially offset by an increase in clinical trial expenses. In addition, there was a $1.1 million decrease in personnel-related expenses primarily due to decreased average headcount. These decreases were partially offset by an increase of $0.8 million in professional and consulting fees associated with preclinical, regulatory and clinical affairs and continued development of our lead product candidate and $0.6 million in facilities and overhead expenses primarily due to increased repairs and maintenance as well as increased utilities and information technology expenses.
Previous report
G&A expenses were $14.9 million for the year ended December 31, 2024, compared to $11.6 million for the year ended December 31, 2023. The increase of $3.3 million was primarily due to an increase in personnel-related expenses of $1.9 million attributable to an increase of $0.8 million in stock-based compensation and $0.8 million in cash grants to offset the tax impact to certain executive officers associated with the forgiveness of promissory notes. In addition, professional and consulting fees increased by $1.2 million, primarily due to an increase in accounting and consulting fees as well as fees paid to maintain patents.
Previous report
Other Income, Net
Previous report
Other income, net was $1.3 million for the year ended December 31, 2024, compared to $2.6 million for the year ended December 31, 2023. The decrease of $1.3 million was primarily due to a decrease in interest income due to lower average invested cash equivalent balances in 2024.
Previous report
•the scope, timing and progress of our ongoing CMP-CPS-001 clinical trial;
Previous report
Determination of Fair Value of our Common Stock
Previous report
Prior to our IPO, given the absence of a public trading market, the fair value of our common stock was determined by our board of directors at the time of each option grant, with input from management, considering contemporaneous independent third-party valuations of common stock, and our board of directors’ assessment of additional objective and subjective factors that it believed were relevant and which may have changed from the date of the most recent valuation through the date of the grant, including: the prices at which we sold shares of our convertible preferred stock to outside investors in arms-length transactions, and the superior rights, preferences, and privileges of the convertible preferred stock relative to the common stock at the time of each grant; the progress of our company’s R&D programs, including their stages of development, and our company’s business strategy; operating and financial performance; the lack of liquidity of the common stock and trends in the broader economy and biotechnology industry also impact the determination of the fair value of the common stock; the likelihood of achieving a liquidity event for our company’s security holders, such as an IPO or a sale of the company, taking into consideration prevailing market conditions; and the hiring of key personnel and the experience of management.
Previous report
These independent third-party valuations were performed in accordance with the guidance outlined in the American Institute of Certified Public Accountants’ Auditing and Valuation Guide, Valuation of Privately-Held-Company Equity Securities Issued as Compensation (the “Guide”). The methodology to determine the fair value of our common stock included estimating the fair value of the enterprise using a market approach, which estimates the fair value of a company by including an estimation of the value of the business based on guideline public companies under several different scenarios. The Guide identifies various available methods for allocating enterprise value across classes and series of capital stock to determine the estimated fair value of common stock at each valuation date.
Previous report
In accordance with the Guide, we considered the following methods:
Previous report
•Option Pricing Method (“OPM”). Under the OPM, shares are valued by creating a series of call options with exercise prices based on the liquidation preferences and conversion terms of each equity class. The estimated fair values of the convertible preferred stock and common stock are inferred by analyzing these options. This method is appropriate to use when the range of possible future outcomes is so difficult to predict that estimates would be highly speculative, and dissolution or liquidation is not imminent.
Previous report
•Probability-Weighted Expected Return Method (“PWERM”). The PWERM is a scenario-based analysis that estimates value per share based on the probability-weighted present value of expected future investment returns, considering each of the possible outcomes available to us, as well as the economic and control rights of each share class.
Previous report
•Hybrid Method. The Hybrid Method is a hybrid between PWERM and OPM, where the equity value is estimated based on probability-weighted value across multiple scenarios where the OPM is used to estimate the allocation of value within one or more of those scenarios.
Previous report
Prior to our IPO, based on our early stage of development, the difficulty in predicting the range of specific outcomes (and their likelihood), and other relevant factors, we determined the OPM scenario was most appropriate for valuations through August 15, 2024, the date of the last grant prior to our IPO. Our board of directors considered various objective and subjective factors to determine the fair value of our common stock as of each grant date, including:
Previous report
•the prices at which we sold shares of convertible preferred stock and the superior rights and preferences of the convertible preferred stock relative to our common stock at the time of each grant;
Previous report
•the progress of our R&D programs, including the status and results of clinical and preclinical studies for our product candidates;
Previous report
•our stage of development and our business strategy;
Previous report
•external market conditions affecting the biopharmaceutical industry and trends within the biopharmaceutical industry;
Previous report
•our financial position, including cash on hand, and our historical and forecasted performance and operating results;
Previous report
•the lack of an active public market for our common stock and our convertible preferred stock;
Previous report
•the likelihood of achieving a liquidity event, such as an IPO or sale of our company considering prevailing market conditions; and
Previous report
•the analysis of IPOs and the market performance of similar companies in the biopharmaceutical industry.
Previous report
The assumptions underlying these valuations were highly complex and subjective and represented management’s best estimates, which involved inherent uncertainties and the application of management’s judgment. As a result, if we had used significantly different assumptions or estimates, the fair value of our common stock and our stock-based compensation expense could be materially different.
Previous report
After our IPO, the fair value of our common stock is determined on the date of grant based on the closing price of our common stock as reported on the Nasdaq, the primary stock exchange on which our common stock is traded.
Reworded paragraphs (38)
Previous wording
We are a clinical-stage biopharmaceutical company pioneering the discovery and development 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. Regulatory RNAs (“regRNAs”) 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, 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 antisense oligonucleotide (“ASO”) candidates, which we also refer to as RNA Actuators. 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.
Current wording
Our product development efforts are enabled by our proprietary RAP Platform. We leverage our RAP Platform to identify and characterize regulatory 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 RAP 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 ASO 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.
Previous wording
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, 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 in 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, we have received gross proceeds of $72.4 million from our IPO and $188.3 million from the sale of our convertible preferred stock. In addition, through December 31, 2024, we have recognized $18.0 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.
Current wording
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, 2025, we have received net 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 prior to our IPO. In addition, through December 31, 2025, we have recognized $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.
Previous wording
We have incurred significant operating losses and negative cash flows from operations since our inception. Our net losses were $51.8 million and $49.3 million for the years ended December 31, 2024 and 2023, respectively. As of December 31, 2024, we had an accumulated deficit of $211.8 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:
Current wording
We have incurred significant operating losses and negative cash flows from operations since our inception. Our net losses were $80.4 million and $51.8 million for the years ended December 31, 2025 and 2024, respectively. As of December 31, 2025, we had an accumulated deficit of $292.2 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.
Previous wording
•contract with third-party manufacturers for preclinical and clinical drug supply supporting any future product candidates we may develop, and commercial supply with respect to any such product candidates that receive regulatory approval;
Current wording
•contract with third-party manufacturers for preclinical and clinical supply to support any future product candidates we may develop and for commercial supply with respect to any such product candidates that receive regulatory approval;
Previous wording
•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.
Current wording
•add operational, legal, compliance, financial and management information systems and personnel to support our research, product development and future commercialization efforts.
Previous wording
As of December 31, 2024, we had cash and cash equivalents of $64.0 million. Based on our current operating plan, we estimate that our cash and cash equivalents as of December 31, 2024 will be sufficient to fund our operating expenses and capital expenditure requirements into the second quarter of 2026. 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.
Current wording
As of December 31, 2025, we had cash and cash equivalents of $109.5 million. Based on our current operating plan, we estimate that our cash and cash equivalents as of December 31, 2025 will be sufficient to fund our operating expenses and capital expenditure requirements into 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 elsewhere in this Annual Report.
Previous wording
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 and 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.
Current wording
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, 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.
Previous wording
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, 2024 and 2023 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, 2024 and 2023, which is presented in R&D expenses on 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, we determined that the likelihood of achieving future milestones was not probable.
Current wording
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 incurredde minimis royalties owed to CMCC for each of the years ended December 31, 2025 and 2024 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 recordedde minimis amounts for each of the years ended December 31, 2025 and 2024, which is presented in R&D expenses in 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, 2025, we determined that the likelihood of achieving future milestones was not probable.
Previous wording
In October 2019, we entered into a patent license agreement (as subsequently amended, the “Whitehead Agreement”) with the Whitehead Institute for Biomedical Research (“Whitehead”). Under the Whitehead Agreement, we were granted a worldwide, royalty-bearing, sublicensable license under certain patent rights owned or controlled by Whitehead. Upon entering into the Whitehead Agreement, we paid an initial $0.1 million license issuance fee, and paid de minimis additional fees in connection with subsequent amendments to the Whitehead Agreement. We are obligated to pay annual license maintenance fees for the term of the Whitehead Agreement. In addition, we are obligated to pay certain filing, prosecution and maintenance fees with respect to certain patent rights licensed.
Current wording
In October 2019, we entered into a patent license agreement (as subsequently amended, the “Whitehead Agreement”) with the Whitehead Institute for Biomedical Research (“Whitehead”). Under the Whitehead Agreement, we were granted a worldwide, royalty-bearing, sublicensable license under certain patent rights owned or controlled by Whitehead. Upon entering into the Whitehead Agreement, we paid an initial $0.1 million license issuance fee, and paidde minimis additional fees in connection with subsequent amendments to the Whitehead Agreement. We are obligated to pay annual license maintenance fees for the term of the Whitehead Agreement. In addition, we are obligated to pay certain filing, prosecution and maintenance fees with respect to certain patent rights licensed.
Previous wording
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 $0.3 million under the Whitehead Agreement during the year ended December 31, 2024. During the year ended December 31, 2023, the fees were de minimis. The fees are recorded in our R&D expense in our consolidated statements of operations and comprehensive loss.
Current wording
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, 2025 and 2024, respectfully. The fees are recorded in our R&D expense in our consolidated statements of operations and comprehensive loss.
Previous wording
In July 2023, we entered into a license agreement (the “Fulcrum Agreement”) with Fulcrum. Under the Fulcrum Agreement, we granted an exclusive license related to our intellectual property (“IP”) and granted a non-exclusive sublicense for IP obtained through the CMCC Agreement. In exchange for the license rights, Fulcrum paid us a $0.4 million upfront payment. In the event that Fulcrum achieves certain development and commercial milestones, Fulcrum will be obligated to pay us one-time milestone payments ranging from $0.6 million to $20.0 million, depending on the product developed and milestone achieved. In addition, under the Fulcrum Agreement there are potential de minimis minimum annual royalty payments as well as sales-based royalties of up to the low-double digits upon commercialization.
Current wording
In July 2023, we entered into a license agreement (the “Fulcrum Agreement”) with Fulcrum. Under the Fulcrum Agreement, we granted an exclusive license related to our intellectual property (“IP”) and granted a non-exclusive sublicense for IP obtained through the CMCC Agreement. In exchange for the license rights, Fulcrum paid us a $0.4 million upfront payment. In the event that Fulcrum achieves certain development and commercial milestones, Fulcrum will be obligated to pay us one-time milestone payments ranging from $0.6 million to $20.0 million, depending on the product developed and milestone achieved. In addition, under the Fulcrum Agreement there are potentialde minimis minimum annual royalty payments as well as sales-based royalties of up to the low-double digits upon commercialization.
Previous wording
During the year ended December 31, 2023, we recorded $0.4 million in license revenue pursuant to the Fulcrum Agreement and none in the year ended December 31, 2024.
Current wording
During the year ended December 31, 2025, we recorded $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.
Previous wording
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. We and Eli Lilly are jointly overseeing the R&D activities under the MTA. In addition, both parties are exposed to the significant risks and potential rewards under the MTA. During each of the years ended December 31, 2024 and 2023, we recorded $0.5 million 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 an 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.
Current wording
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 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 jointly oversaw the R&D activities under the MTA. In addition, both parties were exposed to significant risks and potential rewards under the MTA. During the years ended December 31, 2025 and 2024, we recorded $0.1 million and $0.5 million, 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 ade minimis unbilled receivable recorded within prepaid expenses and other current assets on our consolidated balance sheet as of December 31, 2024.
Previous wording
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 reimburse us for certain research activities. On a per-program basis, we will be eligible to receive up to $5.0 million in future 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. During the year ended December 31, 2024, we recognized $0.7 million in collaboration revenue under the BioMarin Agreement.
Current wording
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 reimbursed us for certain research activities. In November 2025, BioMarin provided notice of its election to terminate the BioMarin Agreement, which termination became effective in February 2026. During the years ended December 31, 2025 and 2024, we recognized $2.9 million and $0.7 million, respectively, in collaboration revenue under the BioMarin Agreement.
Previous wording
For the years ended December 31, 2024 and 2023, we recognized $0.7 million and $0.4 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 and 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.
Current wording
For the years ended December 31, 2025 and 2024, we recognized $3.5 million and $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, 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 or 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.
Previous wording
Our operating expenses consist of R&D expenses and G&A expenses.
Current wording
Our operating expenses consist of R&D expenses, G&A expenses, and impairment of right-of-use asset.
Previous wording
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.
Current wording
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 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.
Previous wording
•the length of time required to enroll eligible patients;
Current wording
•the length of time required to enroll eligible subjects;
Previous wording
•the number of patients that participate in the trials;
Current wording
•the number of subjects that participate in the trials;
Previous wording
•the number of doses that patients receive;
Current wording
•the number of doses that subjects receive;
Previous wording
•the drop-out or discontinuation rates of patients;
Current wording
•the drop-out or discontinuation rates of subjects;
Previous wording
•the duration of patient participation in the trials and follow-up;
Current wording
•the duration of subjects participation in the trials and follow-up;
Previous wording
Other income, net primarily consists of interest income earned on our invested cash and cash equivalent balances and interest expense incurred on our finance lease and financing liabilities, as well as other insignificant amounts.
Current wording
Other (expense) income, net 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, as well as other insignificant amounts.
Previous wording
Research and collaboration revenue was $0.7 million for the year ended December 31, 2024, compared to $0.4 million for the year ended December 31, 2023. The increase of $0.3 million was primarily due to $0.7 million of revenue recognized under the BioMarin Agreement in the year ended December 31, 2024, compared to $0.4 million of revenue recognized under the Fulcrum Agreement in the year ended December 31, 2023.
Current wording
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. The increase of $2.8 million was due to a $2.2 million increase in revenue recognized under the BioMarin Agreement driven by increased performance activities and related costs incurred during 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, 2025.
Previous wording
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 December 31, 2024, we have primarily funded our operations with proceeds from the sale of our equity securities and revenues from our license and collaboration agreements. During the year ended December 31, 2024, we received aggregate gross proceeds of $72.4 million from the sale of shares of our equity securities. In addition, for the year ended December 31, 2024, we recognized $0.7 million in research and collaboration revenue through our collaboration and license agreements.
Current wording
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 December 31, 2025, we have primarily funded our operations with proceeds from the sale of our equity securities and revenues from our license and collaboration agreements. During the year ended December 31, 2025, we received aggregate gross proceeds of $80.1 million from the sale of shares of our equity securities. In addition, for the year ended December 31, 2025, we recognized $3.5 million in research and collaboration revenue through our collaboration and license agreements.
Previous wording
As of December 31, 2024, we had cash and cash equivalents of $64.0 million. Based on our current operating plan, we estimate that our cash and cash equivalents as of December 31, 2024 will be sufficient to fund our operating expenses and capital expenditure requirements into the second quarter of 2026. 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.
Current wording
As of December 31, 2025, we had cash and cash equivalents of $109.5 million. Based on our current operating plan, we estimate that our cash and cash equivalents as of December 31, 2025 will be sufficient to fund our operating expenses and capital expenditure requirements into 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.
Previous wording
The timing and amount of our funding requirements will depend on many factors, including:
Current wording
The timing and amount of our future funding requirements will depend on many factors, including:
Previous wording
•the costs, timing and outcome of regulatory meetings and reviews of 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;
Current wording
•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;
Previous wording
We have entered into two non-cancellable operating leases for our office and lab space in Cambridge, Massachusetts and Boulder, Colorado, which expire 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.
Current wording
See Note 7 to our consolidated financial statements for additional details related to our noncancellable operating leases.
Previous wording
We are continuing to invest in the clinical development of CMP-CPS-001 and have entered into contractual obligations with CROs relating to the performance of clinical trial services. Each contract shall continue until the completion of the trial. Our clinical trial costs are dependent on, among other things, the size and length of our clinical trial. We also incur R&D costs related to the enhancement of our existing product candidates.
Current wording
We are continuing to invest in the development of CMP-002 and have entered into contractual obligations with CROs relating to the performance of services for our planned Phase 1/2 clinical trial. Each contract shall continue until the completion of the clinical trial or until terminated in accordance with its terms. Our clinical trial costs are dependent on, among other things, the scope, timing, and duration of our preclinical and clinical development activities. We also incur R&D costs related to the enhancement of our existing and future product candidates.
Previous wording
The timing of when we will pay or receive royalty payments is uncertain as the payments are contingent upon future activities, including the successful discovery, development, regulatory approval and commercialization of product candidates.
Current wording
The timing of payment or receipt of royalty payments is uncertain as the royalties are contingent upon future activities, including the successful discovery, development, regulatory approval and commercialization of product candidates.
Previous wording
During the year ended December 31, 2023, operating activities used $44.2 million of cash, primarily resulting from our net loss of $49.3 million and net cash used in changes in our operating assets and liabilities of $1.3 million, partially offset by non-cash charges of $6.4 million, including stock-based compensation expense and non-cash operating lease expense.
Current wording
During the year ended December 31, 2025, operating activities used $29.6 million of cash, primarily resulting from our net loss of $80.4 million, partially offset by net cash used in changes in our operating assets and liabilities of $13.0 million and non-cash charges of $37.8 million, including stock-based compensation expense, loss from the change in fair value of our derivative tranche liability, gain on lease modification, non-cash operating lease expense, and depreciation and amortization.
Previous wording
During the year ended December 31, 2024, operating activities used $45.6 million of cash, primarily resulting from our net loss of $51.8 million and net cash used in changes in our operating assets and liabilities of $1.3 million, partially offset by non-cash charges of $7.6 million, including depreciation and amortization, stock-based compensation expense and non-cash operating lease expense.
Current wording
During the year ended December 31, 2024, operating activities used $45.6 million of cash, primarily resulting from our net loss of $51.8 million and net cash used in changes in our operating assets and liabilities of $1.3 million, partially offset by non-cash charges of $7.6 million, including stock-based compensation expense, non-cash operating lease expense, and depreciation and amortization.
Previous wording
During the years ended December 31, 2024 and 2023, net cash used in investing activities was $0.4 million and $0.7 million, respectively, due to purchases of property and equipment.
Current wording
During the years ended December 31, 2025 and 2024, net cash used in investing activities was $0.2 million and $0.4 million, respectively, due to purchases of property and equipment.
Previous wording
During the year ended December 31, 2023, net cash provided by financing activities was $0.3 million, consisting primarily of net proceeds of $0.7 million from a financing obligation. These proceeds were partially offset by financing liability and finance lease principal payments totaling $0.6 million.
Current wording
During the year ended December 31, 2025, net cash provided by financing activities was $75.9 million, consisting primarily of proceeds of $76.0 million from our September 2025 private placement and the December 2025 underwritten offering, net of issuance costs. These proceeds were partially offset by financing liability and finance lease principal payments totaling $0.2 million.
Previous wording
During the year ended December 31, 2024, net cash provided by financing activities was $71.7 million, consisting primarily of proceeds of $72.4 million from our IPO net of offering costs. These proceeds were partially offset primarily with financing liability and finance lease principal payments totaling $0.8 million.
Current wording
During the year ended December 31, 2024, net cash provided by financing activities was $71.7 million, consisting primarily of net proceeds of $72.4 million from our IPO, net of underwriting discounts and commissions and offering costs. These proceeds were partially offset by financing liability and finance lease principal payments totaling $0.8 million.
Previous wording
•expected volatility is derived from the historical volatilities of a select group of representative public companies, for a look-back period commensurate with the expected term of the stock options, as we have no trading history of common stock.
Current wording
•expected volatility is derived from the historical volatilities of a select group of representative public companies, for a look-back period commensurate with the expected term of the stock options, as we lack sufficient trading history of common stock.
Previous wording
The intrinsic value of all outstanding options as of December 31, 2024 was $0.8 million, of which approximately $0.7 million was related to vested options and a de minimis amount was related to unvested options.
Current wording
The intrinsic value of all outstanding options as of December 31, 2025 was $3.3 million, of which approximately $1.3 million was related to vested options and $2.0 million was related to unvested options.
Rearranged current paragraphs (1)
Current report — wording carried from elsewhere
We anticipate that our expenses will increase substantially if and as we: