CAMP4 Therapeutics (NASDAQ: CAMP) details Q1 2026 results and cash runway into 2028
Filing Impact
Filing Sentiment
Form Type
10-Q
Rhea-AI Filing Summary
CAMP4 Therapeutics Corporation reports first-quarter 2026 results, showing higher collaboration revenue but a wider net loss as it advances its RNA-targeting pipeline. Revenue from research and collaboration grew to $1.3 million, mainly from its agreement with GSK. Research and development expenses were $10.2 million and general and administrative costs $4.2 million, keeping total operating expenses broadly flat versus last year.
The company recorded a non-cash $6.2 million loss from remeasuring its derivative tranche liability linked to a potential second private-placement closing, driving net loss to $18.3 million and basic and diluted net loss per share to $0.32. Operating cash outflow was $11.0 million.
CAMP4 ended the quarter with $99.2 million in cash and cash equivalents and $30.9 million in stockholders’ equity, and believes existing cash will fund operations into 2028. The company is preparing a global Phase 1/2 trial of lead candidate CMP-002 for SYNGAP1-related disorder and continues to leverage its RAP Platform and a $17.5 million GSK collaboration to support future programs.
Positive
- None.
Negative
- None.
Insights
Quarter shows stable operating spend, strong cash, and higher accounting-driven loss from a derivative.
CAMP4 recorded $1.3 million in collaboration revenue and held R&D expenses essentially flat at $10.2 million, indicating disciplined investment while it readies CMP-002 for clinical development. G&A rose modestly to $4.2 million as public-company and support costs increased.
The headline net loss of $18.3 million was driven by a $6.2 million non-cash loss from revaluing the derivative tranche liability tied to a potential second private-placement closing. Cash and cash equivalents of $99.2 million and working capital of about $85.6 million underpin management’s view that funding extends into 2028.
Strategically, the GSK collaboration, with a $17.5 million upfront now largely deferred, validates the RAP Platform and provides a revenue stream as work progresses. Investors will likely focus on progress toward regulatory clearances and initiation of the planned global Phase 1/2 trial of CMP-002 for SYNGAP1-related disorder, alongside future disclosures on the private placement’s second tranche.
Key Figures
Research and collaboration revenue: $1.3 million
Net loss: $18.3 million
Net loss per share: $0.32 basic and diluted
+5 more
8 metrics
Research and collaboration revenue
$1.3 million
Three months ended March 31, 2026
Net loss
$18.3 million
Three months ended March 31, 2026
Net loss per share
$0.32 basic and diluted
Three months ended March 31, 2026
R&D expenses
$10.2 million
Three months ended March 31, 2026
G&A expenses
$4.2 million
Three months ended March 31, 2026
Cash and cash equivalents
$99.2 million
As of March 31, 2026
Derivative tranche liability
$50.9 million
Fair value as of March 31, 2026
Operating cash flow
-$11.0 million
Net cash used in operating activities, Q1 2026
Key Terms
derivative tranche liability, RAP Platform, pre-funded warrants, orphan drug designation, +1 more
5 terms
derivative tranche liability financial
"The derivative tranche liability was recorded at its fair value on issuance and is subsequently remeasured at the end of each reporting period"
RAP Platform technical
"Our product development efforts are enabled by our proprietary RAP Platform"
pre-funded warrants financial
"the Company issued 26,717,414 shares of the Company’s common stock and 6,003,758 pre-funded warrants for net cash proceeds"
Pre-funded warrants are financial instruments that give investors the right to purchase a company's stock at a set price, but with most or all of the purchase price paid upfront. They function like a coupon or gift card for stock, allowing investors to buy shares later at a fixed price, which can be beneficial if they want to avoid future price increases. This makes them important for investors seeking flexibility and certainty in their investment plans.
orphan drug designation regulatory
"We have received orphan drug designation for CMP-001 and CMP-002 in certain jurisdictions"
Orphan drug designation is a special status given to medicines developed to treat rare diseases affecting only a small number of people. This status often provides benefits like faster approval processes and financial incentives, making it more attractive for companies to develop these drugs. For investors, it signals potential for exclusive market rights and reduced competition, which can impact the drug’s profitability.
emerging growth company regulatory
"We are an emerging growth company, as defined in the Jumpstart Our Business Startups Act of 2012"
An emerging growth company is a recently public or smaller public firm that qualifies for temporary, lighter regulatory and disclosure rules to reduce the cost and effort of being public. For investors, it means the company may provide less historical financial detail and face fewer reporting requirements than larger firms, so it can grow more quickly but also carries higher uncertainty—like buying a promising early-stage product with fewer user reviews.
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UNITED STATES
SECURITIES AND EXCHANGE COMMISSION
Washington, D.C. 20549
__________________________________________________________________
FORM 10-Q
__________________________________________________________________
(Mark One)
For the quarterly period ended March 31, 2026
or
For the transition period from ___________ to___________
Commission File Number: 001-42365
_________________________________________________________________
(Exact name of registrant as specified in its charter)
__________________________________________________________________
| (State or other jurisdiction of incorporation or organization) | (I.R.S. Employer Identification Number) | ||||
(Address of Principal Executive Offices)
(617 ) 651-8867
(Registrant’s telephone number)
__________________________________________________________________
Indicate by check mark whether the registrant (1) has filed all reports required to be filed by Section 13 or 15(d) of the Securities Exchange Act of 1934 during the preceding 12 months (or for such shorter period that the registrant was required to file such reports), and (2) has been subject to such filing requirements for the past 90 days. Yes ☒ No ☐
Indicate by check mark whether the registrant has submitted electronically every Interactive Data File required to be submitted pursuant to Rule 405 of Regulation S-T (§232.405 of this chapter) during the preceding 12 months (or for such shorter period that the registrant was required to submit such files). Yes ☒ No ☐
Indicate by check mark whether the registrant is a large accelerated filer, an accelerated filer, a non-accelerated filer, a smaller reporting company, or an emerging growth company. See the definitions of “large accelerated filer,” “accelerated filer,” “smaller reporting company,” and “emerging growth company” in Rule 12b-2 of the Exchange Act.
| Large accelerated filer | ☐ | Accelerated filer | ☐ | ||||||||||||||
| ☒ | Smaller reporting company | Emerging growth company | |||||||||||||||
If an emerging growth company, indicate by check mark if the registrant has elected not to use the extended transition period for complying with any new or revised financial accounting standards provided pursuant to Section 13(a) of the Exchange Act. ☐
Indicate by check mark whether the registrant is a shell company (as defined in Rule 12b-2 of the Exchange Act). Yes ☐ No ☒
Securities registered pursuant to Section 12(b) of the Act:
| Title of Each Class | Trading symbol | Name of Exchange on which registered | ||||||||||||
As of May 6, 2026, there were 51,925,800 shares of the registrant’s common stock, par value $0.0001 per share, outstanding.
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TABLE OF CONTENTS
| Page | ||||||||||||||
Part I | Financial Information | |||||||||||||
Item 1. | Financial Statements (Unaudited) | 5 | ||||||||||||
Unaudited Condensed Consolidated Balance Sheets as of March 31, 2026 and December 31, 2025 | 5 | |||||||||||||
Unaudited Condensed Consolidated Statements of Operations and Comprehensive Loss for the three months ended March 31, 2026 and 2025 | 6 | |||||||||||||
Unaudited Condensed Consolidated Statements of Changes in Stockholders’ Equity for the three months ended March 31, 2026 and 2025 | 7 | |||||||||||||
Unaudited Condensed Consolidated Statements of Cash Flows for the three months ended March 31, 2026 and 2025 | 8 | |||||||||||||
Notes to Condensed Consolidated Financial Statements | 9 | |||||||||||||
Item 2. | Management’s Discussion and Analysis of Financial Condition and Results of Operations | 23 | ||||||||||||
Item 3. | Quantitative and Qualitative Disclosures about Market Risk | 30 | ||||||||||||
Item 4. | Controls and Procedures | 30 | ||||||||||||
Part II | Other Information | 31 | ||||||||||||
Item 1. | Legal Proceedings | 31 | ||||||||||||
Item 1A. | Risk Factors | 31 | ||||||||||||
Item 2. | Unregistered Sales of Equity Securities and Use of Proceeds | 33 | ||||||||||||
Item 3. | Defaults Upon Senior Securities | 33 | ||||||||||||
Item 4. | Mine Safety Disclosures | 33 | ||||||||||||
Item 5. | Other Information | 33 | ||||||||||||
Item 6. | Exhibits | 33 | ||||||||||||
Exhibit Index | 34 | |||||||||||||
Signatures | 35 | |||||||||||||
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SPECIAL NOTE REGARDING FORWARD-LOOKING STATEMENTS AND INDUSTRY DATA
This Quarterly Report on Form 10-Q (“Quarterly Report”) contains forward-looking statements that involve substantial risks and uncertainties because they relate to events and depend on circumstances that may or may not occur in the future. All statements other than statements of historical fact contained in this Quarterly Report, including statements regarding our strategy, future operations, future financial position, prospects, plans, objectives of management and expected growth, are forward-looking statements. These statements are based on our current beliefs, expectations and assumptions regarding our intentions, beliefs or current expectations concerning, among other things, the future of our business, future plans and strategies, our operational results and other future conditions. Forward-looking statements involve known and unknown risks, uncertainties and other important factors that may cause our actual results, performance or achievements to be materially different from any future results, performance or achievements expressed or implied by the forward-looking statements.
In some cases, you can identify forward-looking statements by terms such as “may,” “will,” “should,” “expect,” “plan,” “anticipate,” “could,” “intend,” “target,” “project,” “estimate,” “believe,” “predict,” “potential” or “continue” or the negative of these terms or other similar expressions intended to identify statements about the future, although not all forward-looking statements contain these identifying words. These forward-looking statements include, without limitation, statements about the following:
•the initiation, timing, progress, results and costs of our research and development (“R&D”) programs and of our current and future preclinical studies and clinical trials of our product candidates, including statements regarding the timing of initiation and completion of studies or trials and related preparatory work, as well as the period during which the results of the trials are expected to become available;
•the timing of our planned regulatory submissions, initiation of planned clinical trials and timing of expected clinical results for our current and future product candidates;
•the timing of any submissions of filings for regulatory approval of, and our ability to obtain and maintain regulatory approvals for, any of our product candidates;
•our ability to obtain and maintain the benefits of regulatory designations, including orphan drug designation, for our product candidates;
•our ability to identify patients with the diseases treated by our product candidates, and to enroll patients in trials;
•our expectations regarding the size of the patient populations, market acceptance and opportunity for and clinical utility of our product candidates, if approved for commercial use;
•our reliance on third party manufacturing partners to comply with significant regulations with respect to manufacturing our products;
•our expectations regarding the scope of any approved indication for any product candidate;
•our ability to successfully commercialize our product candidates, if approved;
•our ability to leverage our RAP Platform to identify and develop future product candidates;
•our estimates of our expenses, ongoing losses, future revenue, capital requirements, and our need for or ability to obtain additional funding before we can expect to generate any revenue from product sales;
•our ability to establish or maintain strategic collaborations or arrangements, including potential business development opportunities and potential licensing partnerships, and our ability to attract collaborators with development, regulatory and commercialization expertise;
•our ability to identify, recruit and retain key personnel;
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•our reliance upon intellectual property licensed from third parties and our ability to obtain such licenses on commercially reasonable terms or at all;
•our ability to protect and enforce our intellectual property position for our product candidates, and the scope of such protection;
•our financial performance;
•the sufficiency of our existing cash and cash equivalents to fund our future operating expenses and capital expenditure requirements;
•our competitive position and the development of and projections relating to our competitors or our industry;
•our estimates regarding future expenses and needs for additional financing;
•the impact of laws and regulations;
•the effect of changes in international trade policies and general economic, industry, geopolitical and market conditions, such as uncertainties related to military conflict or war, tariffs (including tariffs that have been or may in the future be imposed by the United States or other countries), sanctions, trade protection measures or other trade barriers, inflation and financial institution instability or pandemic or epidemic disease outbreaks, many of which are beyond our control, as well as the value of our common stock and our ability to access capital markets; and
•our expectations regarding the time during which we will be an emerging growth company and smaller reporting company under the Jumpstart Our Business Startups Act of 2012 (the “JOBS Act”).
Although we base these forward-looking statements on assumptions that we believe are reasonable when made, we caution you that forward-looking statements are not guarantees of future performance and that our actual results of operations, financial condition and liquidity, and the development of the industry in which we operate may differ materially from those made in or suggested by the forward-looking statements contained in this Quarterly Report. In addition, even if our results of operations, financial condition and liquidity, and the development of the industry in which we operate are consistent with the forward-looking statements contained in this Quarterly Report, those results or developments may not be indicative of results or developments in subsequent periods.
Given these risks and uncertainties, you are cautioned not to place undue reliance on these forward-looking statements. Any forward-looking statement that we make in this Quarterly Report speaks only as of the date of such statement. Except as required by law, we assume no obligation to update these forward-looking statements publicly, or to update the reasons actual results could differ materially from those anticipated in any forward-looking statements, whether as a result of new information, future events or otherwise. Comparisons of results for current and any prior periods are not intended to express any future trends or indications of future performance, unless specifically expressed as such, and should only be viewed as historical data. You should, therefore, not rely on these forward-looking statements as representing our views as of any date subsequent to the date of this Quarterly Report.
Unless otherwise indicated, market and industry data contained in this Quarterly Report, including potential market opportunities, is based on our management’s estimates and research, as well as industry and general publications and research and studies conducted by third parties. Although we believe that the information from these third-party publications, research and studies included in this Quarterly Report is reliable, and we are responsible for the accuracy of such information, we have not independently verified the accuracy or completeness of this information. Management’s estimates are derived from publicly available information, their knowledge of our industry and their assumptions based on such information and knowledge, which we believe to be reasonable. This data involves a number of assumptions and limitations and the industry in which we operate is subject to a high degree of uncertainty and risk due to a variety of factors, including those described in Part II, Item 1A. “Risk Factors” in this Quarterly Report. These and other factors could cause our future performance to differ materially from our assumptions and estimates.
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NOTE REGARDING TRADEMARKS
“CAMP4,” “RAP Platform,” and our other registered or common law trademarks, trade names or service marks appearing in this Quarterly Report are the property of CAMP4 Therapeutics Corporation and are registered as trademarks in the United States and other countries. This Quarterly Report also contains references to trademarks belonging to other entities. Solely for convenience, trademarks and trade names referred to in this Quarterly Report, including logos, artwork and other visual displays, may appear without the ® or ™ symbols, but such references are not intended to indicate in any way that we will not assert, to the fullest extent under applicable law, our rights or the rights of the applicable licensor to these trademarks and trade names. We do not intend our use or display of other entities’ trade names, trademarks or service marks to imply a relationship with, or endorsement or sponsorship of us by, any other entity.
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PART I —FINANCIAL INFORMATION
Item 1. Financial Statements
CAMP4 Therapeutics Corporation
Unaudited Condensed Consolidated Balance Sheets
(In thousands, except share and per share amounts)
| March 31, 2026 | December 31, 2025 | ||||||||||
| Assets | |||||||||||
| Current assets: | |||||||||||
| Cash and cash equivalents | $ | $ | |||||||||
| Restricted cash, current portion | |||||||||||
| Prepaid expenses and other current assets | |||||||||||
| Total current assets | |||||||||||
| Restricted cash, net of current portion | |||||||||||
| Property and equipment, net | |||||||||||
| Operating lease right-of-use assets | |||||||||||
| Other assets | |||||||||||
| Total assets | $ | $ | |||||||||
| Liabilities and Stockholders’ Equity | |||||||||||
| Current liabilities: | |||||||||||
| Accounts payable | $ | $ | |||||||||
| Accrued expenses and other current liabilities | |||||||||||
| Current portion of deferred revenue | |||||||||||
| Operating lease liabilities, current portion | |||||||||||
| Total current liabilities | |||||||||||
| Deferred revenue, net of current portion | |||||||||||
| Operating lease liabilities, net of current portion | |||||||||||
| Derivative tranche liability | |||||||||||
| Other long-term liabilities | |||||||||||
| Total liabilities | |||||||||||
| Commitments and contingencies (Note 4) | |||||||||||
| Stockholders’ equity: | |||||||||||
Common stock, $ | |||||||||||
| Additional paid-in capital | |||||||||||
| Accumulated deficit | ( | ( | |||||||||
| Total stockholders’ equity | |||||||||||
| Total liabilities and stockholders’ equity | $ | $ | |||||||||
The accompanying notes are an integral part of these condensed consolidated financial statements.
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CAMP4 Therapeutics Corporation
Unaudited Condensed Consolidated Statements of Operations and Comprehensive Loss
(In thousands, except for share and per share data)
| Three Months Ended March 31, | |||||||||||
| 2026 | 2025 | ||||||||||
| Revenue: | |||||||||||
| Research and collaboration revenue | $ | $ | |||||||||
| Operating Expenses: | |||||||||||
| Research and development | |||||||||||
| General and administrative | |||||||||||
| Total operating expenses | |||||||||||
| Loss from operations | ( | ( | |||||||||
| Other (expense) income, net: | |||||||||||
| Interest income | |||||||||||
| Change in fair value of derivative tranche liability | ( | ||||||||||
| Other income | |||||||||||
| Total other (expense) income, net | ( | ||||||||||
| Net loss | $ | ( | $ | ( | |||||||
| Net loss per share attributable to common stockholders, basic and diluted | $ | ( | $ | ( | |||||||
| Weighted-average shares of common stock outstanding, basic and diluted | |||||||||||
| Net loss attributable to common stockholders | $ | ( | $ | ( | |||||||
| Other comprehensive income (loss) | |||||||||||
| Total comprehensive loss | $ | ( | $ | ( | |||||||
The accompanying notes are an integral part of these condensed consolidated financial statements.
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CAMP4 Therapeutics Corporation
Unaudited Condensed Consolidated Statements of Stockholders’ Equity
(In thousands, except share amounts)
| Common stock | Additional paid-in capital | Accumulated deficit | Total stockholders' equity | ||||||||||||||||||||||||||
| Shares | Amount | ||||||||||||||||||||||||||||
| Balance as of December 31, 2025 | $ | $ | $ | ( | $ | ||||||||||||||||||||||||
| Issuance of common stock under employee stock purchase plan | — | — | |||||||||||||||||||||||||||
| Stock option exercises | — | — | |||||||||||||||||||||||||||
| Stock-based compensation expense | — | — | — | ||||||||||||||||||||||||||
| Net loss | — | — | — | ( | ( | ||||||||||||||||||||||||
| Balance as of March 31, 2026 | $ | $ | $ | ( | $ | ||||||||||||||||||||||||
| Common stock | Additional paid-in capital | Accumulated deficit | Total stockholders' equity | ||||||||||||||||||||||||||
| Shares | Amount | ||||||||||||||||||||||||||||
| Balance as of December 31, 2024 | $ | $ | $ | ( | $ | ||||||||||||||||||||||||
| Issuance of common stock | — | — | — | — | |||||||||||||||||||||||||
| Vesting of restricted common stock | — | — | — | — | |||||||||||||||||||||||||
| Stock-based compensation expense | — | — | — | ||||||||||||||||||||||||||
| Net loss | — | — | — | ( | ( | ||||||||||||||||||||||||
| Balance as of March 31, 2025 | $ | $ | $ | ( | $ | ||||||||||||||||||||||||
The accompanying notes are an integral part of these condensed consolidated financial statements.
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CAMP4 Therapeutics Corporation
Unaudited Condensed Consolidated Statements of Cash Flows
(In thousands)
| Three Months Ended March 31, | |||||||||||
| 2026 | 2025 | ||||||||||
| Operating activities | |||||||||||
| Net loss | $ | ( | $ | ( | |||||||
| Adjustments to reconcile net loss to net cash used in operating activities: | |||||||||||
| Depreciation and amortization | |||||||||||
| Stock-based compensation expense | |||||||||||
| Change in fair value of derivative tranche liability | |||||||||||
| Non-cash lease expense | |||||||||||
| Non-cash interest expense | |||||||||||
| Change in operating assets and liabilities: | |||||||||||
| Prepaid expenses and other current assets | ( | ||||||||||
| Accounts payable | |||||||||||
| Accrued expenses and other liabilities | ( | ( | |||||||||
| Deferred revenue | ( | ( | |||||||||
| Operating lease liabilities | ( | ||||||||||
| Net cash used in operating activities | ( | ( | |||||||||
| Investing activities | |||||||||||
| Purchases of property and equipment | ( | ||||||||||
| Net cash used in investing activities | ( | ||||||||||
| Financing activities | |||||||||||
| Proceeds from exercise of stock options and issuance of common stock under employee stock purchase plan | |||||||||||
| Principal payments on finance leases | ( | ||||||||||
| Principal payments on financing obligation | ( | ||||||||||
| Net cash provided by (used in) financing activities | ( | ||||||||||
| Net change in cash, cash equivalents, and restricted cash | ( | ( | |||||||||
| Cash, cash equivalents and restricted cash at beginning of period | |||||||||||
| Cash, cash equivalents and restricted cash at end of period | $ | $ | |||||||||
| Supplemental disclosures of cash flow information: | |||||||||||
| Finance lease right-of-use asset converted to fixed asset | $ | $ | |||||||||
| Purchase of property and equipment in accounts payable and accrued expenses | $ | $ | |||||||||
The accompanying notes are an integral part of these condensed consolidated financial statements.
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CAMP4 Therapeutics Corporation
Notes to Condensed Consolidated Financial Statements
(Unaudited)
1. Basis of Presentation and Accounting Policies
Basis of Presentation and Principles of Consolidation
The accompanying condensed consolidated financial statements are unaudited and have been prepared in conformity with accounting principles generally accepted in the United States of America (“U.S. GAAP”) and applicable rules and regulations of the Securities and Exchange Commission for interim financial reporting, consistent in all material respects with those applied in the Company’s Annual Report on Form 10-K for the year ended December 31, 2025 (“2025 Form 10-K”) and, in the opinion of management, reflect all adjustments, which include only normal recurring adjustments, necessary to present fairly the Company’s consolidated financial position, consolidated results of operations, and consolidated cash flows for the interim periods presented. Any reference in these notes to applicable guidance is meant to refer to the authoritative standards of U.S. GAAP as found in the Accounting Standards Codification (“ASC”) and Accounting Standards Updates (“ASU”) of the Financial Accounting Standards Board (“FASB”). This report should be read in conjunction with the audited consolidated financial statements in the Company’s 2025 Form 10-K.
These condensed consolidated financial statements include CAMP4 Therapeutics Corporation and its wholly-owned subsidiary, CAMP4 Therapeutics Pty Ltd (together, the “Company”). All intercompany balances and transactions have been eliminated in consolidation. The significant accounting policies used in the preparation of these condensed consolidated financial statements for the three months ended March 31, 2026 are consistent with those described in the Company’s 2025 Form 10-K. The results of operations for the three months ended March 31, 2026 are not necessarily indicative of the operating results to be expected for the full fiscal year or future operating periods.
Private Placement
On September 9, 2025, the Company entered into a Securities Purchase Agreement (the “Purchase Agreement”) with investors pursuant to which the Company agreed to sell certain securities, in up to two closings in a private placement transaction (the “Private Placement”). The initial closing of the Private Placement occurred on September 11, 2025 (the “Initial Closing”). At the Initial Closing, the Company issued 26,717,414 shares of the Company’s common stock and 6,003,758 pre-funded warrants for net cash proceeds of $46.7 million, after deducting $3.3 million in issuance costs and legal fees (see Notes 2 and 6).
Underwritten Offering
On December 18, 2025, the Company entered into an underwriting agreement (the “Underwriting Agreement”) with Leerink Partners LLC (“Leerink Partners”) relating to an offering of shares of the Company's common stock (the “Underwritten Offering”). The closing of the Underwritten Offering occurred on December 19, 2025. At the closing, the Company issued 5,000,000 shares of the Company’s common stock for net cash proceeds of $28.1 million, after deducting $1.9 million in issuance costs.
Liquidity
As of March 31, 2026, the Company had approximately $99.2 million in cash and cash equivalents and working capital of approximately $85.6 million. The Company has a relatively limited operating history, and the revenue and income potential of the Company’s business and market are unproven. The Company has experienced net losses and negative cash flows from operations since its inception and, as of March 31, 2026, the Company had an accumulated deficit of $310.5 million. During the three months ended March 31, 2026, the Company incurred a net loss of $18.3 million and had negative cash flows from operating activities of $11.0 million. The Company will continue to incur significant costs and expenses related to its ongoing operations until it successfully develops, obtains regulatory approval for and gains market acceptance of a product candidate and achieves revenues adequate to support the Company’s operations.
From inception to March 31, 2026, the Company has funded its operations primarily with proceeds from the sale of convertible preferred stock and common stock, including the Underwritten Offering, the Initial Closing of the Private Placement, and its initial public offering (“IPO”), as well as through revenues from its license and collaboration agreements. The Company expects that its cash and cash equivalents as of March 31, 2026 will enable it to fund its
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operating expenses and capital expenditure requirements for at least 12 months from the date these condensed consolidated financial statements are issued. This evaluation is based on relevant conditions and events that are known and reasonably knowable at the date that the consolidated financial statements are issued. To the extent these conditions or events change, the Company could deplete its available capital resources sooner than it currently expects.
Use of Estimates
The preparation of the Company’s condensed consolidated financial statements in conformity with U.S. GAAP requires management to make estimates, assumptions, and judgments that affect the reported amounts of assets, liabilities, expenses and related disclosures in the accompanying notes. The Company bases its estimates, assumptions, and judgments on historical experience when available and on various factors that it believes to be reasonable under the circumstances as of the date of the accompanying condensed consolidated financial statements, including revenue recognition, stock-based compensation expense, accrued expenses, research and development expenses and related prepaid and accrued expenses, derivative tranche liability, lease accounting, and the recoverability of the Company’s net deferred tax assets and related valuation allowance. In addition, other factors may affect estimates, including the expected business and operational changes, the sensitivity and volatility associated with the assumptions used in developing estimates, and whether historical trends are expected to be representative of future trends. The estimation process often may yield a range of potentially reasonable estimates of the ultimate future outcomes, and management must select an amount that falls within that range of reasonable estimates. Actual results could differ materially from the estimates and assumptions used in the preparation of the accompanying condensed consolidated financial statements under different assumptions or conditions.
Restricted Cash
The following table provides a reconciliation of cash, cash equivalents and restricted cash reported in the condensed consolidated balance sheets to the corresponding amounts shown in the condensed consolidated statements of cash flows (in thousands):
| March 31, 2026 | December 31, 2025 | ||||||||||
| Cash and cash equivalents | $ | $ | |||||||||
| Restricted cash, current portion | |||||||||||
| Restricted cash, net of current portion | |||||||||||
| Total cash, cash equivalents, and restricted cash | $ | $ | |||||||||
Derivative Tranche Liability
The Private Placement includes a right provided to the participating investors to purchase the Company’s securities in two tranches. The second tranche was determined to be a freestanding instrument and accounted for as derivative tranche liability within the condensed consolidated balance sheet. The derivative tranche liability was recorded at its fair value on issuance and is subsequently remeasured at the end of each reporting period, with non-cash changes in fair value recorded in the condensed consolidated statement of operations until settlement.
Recently Issued Accounting Standards
In November 2024, the FASB issued ASU 2024-03, “Income Statement-Reporting Comprehensive Income-Expense Disaggregation Disclosures (Subtopic 220-40): Disaggregation of Income Statement Expenses.” The standard requires that public business entities disclose additional information about specific expense categories in the notes to financial statements for interim and annual reporting periods. For public business entities, the guidance is effective for interim and annual periods beginning after December 15, 2026. Early adoption is permitted for annual consolidated financial statements that have not yet been issued or made available for issuance. The guidance may be applied on a
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prospective basis or retrospectively for all prior periods presented in the financial statements. The Company is currently evaluating the impact that this guidance may have on its consolidated financial statements.
2. Fair Value Measurements
Financial Assets
The following tables present the financial asset instruments carried at fair value on a recurring basis as of March 31, 2026 and December 31, 2025 (in thousands) in accordance with the ASC 820 hierarchy.
| Fair Value Measurements as of March 31, 2026 | |||||||||||||||||||||||
| Level 1 | Level 2 | Level 3 | Total | ||||||||||||||||||||
| Assets | |||||||||||||||||||||||
| Cash equivalents | $ | $ | $ | $ | |||||||||||||||||||
| Fair Value Measurements as of December 31, 2025 | |||||||||||||||||||||||
| Level 1 | Level 2 | Level 3 | Total | ||||||||||||||||||||
| Assets | |||||||||||||||||||||||
| Cash equivalents | $ | $ | $ | $ | |||||||||||||||||||
Derivative Tranche Liability
The following tables present the derivative tranche liability (see Note 6) carried at fair value on a recurring basis as of March 31, 2026 and December 31, 2025 (in thousands) and in accordance with the ASC 820 hierarchy and was based on significant inputs not observable in the market, which represents a Level 3 measurement within the fair value hierarchy.
| Fair Value Measurements as of March 31, 2026 | |||||||||||||||||||||||
| Level 1 | Level 2 | Level 3 | Total | ||||||||||||||||||||
| Liabilities | |||||||||||||||||||||||
| Derivative tranche liability | $ | $ | $ | $ | |||||||||||||||||||
| Fair Value Measurements as of December 31, 2025 | |||||||||||||||||||||||
| Level 1 | Level 2 | Level 3 | Total | ||||||||||||||||||||
| Liabilities | |||||||||||||||||||||||
| Derivative tranche liability | $ | $ | $ | $ | |||||||||||||||||||
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The derivative tranche liability is valued using a Monte Carlo simulation model which uses certain assumptions, including annual volatility. The range of volatilities of comparable public companies utilized was 43.5 % - 125.8 % as of March 31, 2026. The volatility utilized in the Monte Carlo option-pricing model was determined using the 75th percentile. The following table presents the assumptions used to determine the fair value of the derivative tranche liability as of March 31, 2026 and as of December 31, 2025:
| March 31, 2026 | December 31, 2025 | ||||||||||
| Expected volatility | % | % | |||||||||
| Risk-free interest rate | % | % | |||||||||
| Expected term (in years) | |||||||||||
| Probability of achieving specified conditions | % | % | |||||||||
| Share price | $ | $ | |||||||||
The following table provides a roll-forward of the aggregate fair value of the derivative tranche liability categorized with Level 3 inputs for the three months ended March 31, 2026 (in thousands):
| Derivative Tranche Liability | |||||
| Balance as of December 31, 2025 | $ | ||||
| Change in fair value | |||||
| Balance as of March 31, 2026 | $ | ||||
The carrying amounts reflected in the condensed consolidated balance sheet for prepaid expenses and other current assets, accounts payable and accrued expenses and other liabilities are shown at their historical values, which approximate their fair values.
3. Other Balance Sheet Components
Prepaid Expenses and Other Current Assets
Prepaid expenses and other current assets consisted of the following (in thousands):
| March 31, 2026 | December 31, 2025 | ||||||||||
Prepaid research and development | $ | $ | |||||||||
| Software and subscriptions expense | |||||||||||
| Prepaid insurance costs | |||||||||||
| Prepaid lease costs | |||||||||||
| Unbilled receivable | |||||||||||
| Other | |||||||||||
| Total prepaid expenses and other current assets | $ | $ | |||||||||
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Property and Equipment, Net
Property and equipment, net consisted of the following (in thousands):
| March 31, 2026 | December 31, 2025 | ||||||||||
| Leasehold improvements | $ | $ | |||||||||
| Laboratory equipment | |||||||||||
| Computer and software | |||||||||||
| Furniture and fixtures | |||||||||||
| Construction in progress | |||||||||||
| Total property and equipment | |||||||||||
| Less: accumulated depreciation and amortization | ( | ( | |||||||||
| Total property and equipment, net | $ | $ | |||||||||
The Company incurred depreciation and amortization expense of $0.5 million and $0.4 million for the three months ended March 31, 2026 and 2025, respectively. For each of the three months ended March 31, 2026 and 2025, depreciation and amortization expense included nominal finance lease right-of-use (“ROU”) asset amortization.
Accrued Expenses and Other Current Liabilities
Accrued expenses and other current liabilities consisted of the following (in thousands):
| March 31, 2026 | December 31, 2025 | ||||||||||
| External R&D expenses | $ | $ | |||||||||
| Employee compensation and benefits | |||||||||||
| Deferred lease incentive | |||||||||||
| Professional fees | |||||||||||
| Other | |||||||||||
| Total accrued expenses and other current liabilities | $ | $ | |||||||||
4. Commitments and Contingencies
Operating Leases
Cambridge and Watertown Leases
The Company currently leases approximately 30,000 square feet of office and laboratory space in Cambridge, Massachusetts (the “Cambridge Lease”). The Cambridge Lease was originally set to expire on June 30, 2027.
On December 22, 2025, the Company entered into an amendment to the Cambridge Lease, which provided for rent abatements and refunds and accelerated the termination of the lease to the date thirty days after the commencement of the Watertown Lease (as defined below). The amendment did not result in the Company relinquishing control of the leased premises until the revised termination date.
Concurrently, on December 22, 2025, the Company entered into a lease agreement for approximately 44,000 square feet of laboratory and office space in Watertown, Massachusetts (the “Watertown Lease”) with an affiliate of the Cambridge lessor. The Watertown Lease will commence upon delivery of the premises and availability for use (the “Watertown Lease Commencement Date”), which is contractually targeted to occur approximately 180 days following execution. The initial lease term extends through June 30, 2030.
The Cambridge Lease amendment and the Watertown Lease were negotiated contemporaneously as part of a coordinated relocation transaction with affiliated lessors. Accordingly, the Company combined the arrangements for
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accounting purposes under ASC 842 and determined that the combined contract contains two separate lease components: (i) the modified Cambridge Lease and (ii) the Watertown Lease. The total consideration of the combined contract, including contractual Watertown rent, the lease modification fee (as described below) and rent refunds, was allocated to each lease component based on relative standalone prices at the effective date of the modification.
As a result of the modification, the Company remeasured the Cambridge Lease right-of-use asset and lease liability to zero and $1.1 million, respectively, and recorded a gain of approximately $0.3 million during the year ended December 31, 2025, reflecting the net impact of the remeasurement based on the allocated consideration. The gain was included within general and administrative expense and research and development expense in the consolidated statement of operations and comprehensive loss for the year ended December 31, 2025 as $0.1 million and $0.2 million, respectively.
In connection with the relocation transaction, the Company agreed to pay a lease modification fee of approximately $2.1 million, payable in installments commencing after the Company relinquishes control of the Cambridge premises. The portion of the modification fee allocated to the Cambridge Lease was included in the remeasurement described above, and the remainder was allocated to the Watertown Lease component.
As of March 31, 2026, the Watertown Lease had not commenced and, accordingly, no right-of-use asset or lease liability had been recognized for that lease component. The Watertown Lease includes an initial rent-free period of approximately 18 months. It also provides for the use of certain lessor-owned laboratory equipment during the lease term which will be accounted for as a lease incentive related to the Watertown Lease and will reduce the right-of-use asset upon lease commencement.
The Watertown Lease includes an option to extend the lease term for an additional five-year period at market rates. The Company will determine the appropriate incremental borrowing rate and recognize the related right-of-use asset and lease liability upon commencement.
Boulder Lease
The Company currently leases approximately 5,300 square feet of office and lab space in Boulder, Colorado (the “Boulder Lease”). The Boulder Lease is a five-year lease that commenced on September 1, 2023 and expires on September 30, 2028. As the rate implicit for the Boulder Lease was not readily determinable, the Company used its incremental borrowing rate of 7.12 % as of the commencement date. At commencement of the Boulder Lease, the Company recorded $1.4 million of operating ROU assets, $0.2 million of current operating lease liabilities and $1.2 million of non-current operating lease liabilities.
During the year ended December 31, 2025, the Company vacated its Boulder, Colorado facility. The Company is currently pursuing sublease opportunities for the space. During the year ended December 31, 2025, it was determined that the market rate for similar space is less than the rate the Company is paying under the Boulder Lease. As a result, the Company recognized in the consolidated statement of operations an impairment charge of $0.5 million related to the right of use (“ROU”) asset during the year ended December 31, 2025. As of March 31, 2026, the Company had $0.4 million of ROU assets, $0.3 million of current operating lease liabilities and $0.5 million of non-current operating lease liabilities related to the Boulder Lease.
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| Three Months Ended March 31, | |||||||||||
| 2026 | 2025 | ||||||||||
| Operating lease costs: | |||||||||||
| Lease expense | $ | $ | |||||||||
| Short-term lease expense | |||||||||||
| Variable lease expense | |||||||||||
| Total operating lease costs | $ | $ | |||||||||
| Other information: | |||||||||||
| Operating cash flows used for operating leases | $ | $ | |||||||||
| Weighted-average remaining lease term in years | |||||||||||
| Weighted-average incremental borrowing rate | % | % | |||||||||
Maturities of operating lease liabilities as of March 31, 2026 were as follows (in thousands):
| 2026 (nine months remaining) | $ | |||||||
| 2027 | ||||||||
| 2028 | ||||||||
| 2029 | ||||||||
| 2030 | ||||||||
| Total lease payments | ||||||||
| Less: amount representing imputed interest | ( | |||||||
| Total future minimum lease obligations | $ | |||||||
Legal Proceedings
There are no matters currently outstanding for which any liabilities have been accrued or require disclosure.
5. Collaboration and License Agreements
In-License Agreements
Children’s Medical Center Corporation
In April 2018, the Company entered into a development and license agreement (the “CMCC Agreement”) with Children’s Medical Center Corporation (“CMCC”). The CMCC Agreement allows the Company 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 deprioritized by the Company, which were subsequently sublicensed to Fulcrum Therapeutics, Inc. (“Fulcrum”), as described below. As part of the CMCC Agreement, the Company 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.
The Company is 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, the Company 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. The Company incurred de minimis royalties owed to CMCC for each of the three months ended March 31, 2026 and 2025 under the CMCC Agreement and recorded the amounts in R&D expense in the consolidated statement of operations and comprehensive loss.
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The Company re-evaluates the likelihood of achieving future milestones under the CMCC Agreement at the end of each reporting period. As of March 31, 2026, the Company determined that the likelihood of achieving future milestones under the CMCC Agreement was not probable.
Whitehead Institute for Biomedical Research
In October 2019, the Company entered into a patent license agreement (as subsequently amended, the “Whitehead Agreement”) with the Whitehead Institute for Biomedical Research (“Whitehead”). Under the Whitehead Agreement, the Company was granted a worldwide, royalty-bearing, sublicensable license under certain patent rights owned or controlled by Whitehead. Upon entering into the Whitehead Agreement, the Company paid an initial $0.1 million license issuance fee, and has paid de minimis additional fees in connection with subsequent amendments to the Whitehead Agreement. The Company is obligated to pay annual license maintenance fees for the term of the Whitehead Agreement. In addition, the Company is obligated to pay certain filing, prosecution and maintenance fees with respect to certain patent rights licensed.
The Company is also obligated to pay potential development milestones to Whitehead of up to $1.9 million upon the achievement of certain specified contingent events. In addition, if the Company successfully commercializes a product under the Whitehead Agreement, it is 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.
The Company incurred de minimis fees under the Whitehead Agreement for each of the three months ended March 31, 2026 and 2025. These fees are recorded in R&D expense in the Company’s condensed consolidated statements of operations and comprehensive loss.
Out-License Agreement
Fulcrum Therapeutics, Inc.
In July 2023, the Company entered into a license agreement (the “Fulcrum Agreement”) with Fulcrum. Under the Fulcrum Agreement, the Company granted an exclusive license related to the Company’s intellectual property (“IP”) and granted a non-exclusive sublicense for IP obtained through the CMCC Agreement. In exchange for the license rights, Fulcrum paid the Company a $0.4 million upfront payment. Under the Fulcrum Agreement, the Company was eligible to receive milestone payments ranging from $0.6 million to $20.0 million upon the achievement of specified development and commercial milestones, depending on the product developed and milestone achieved. The Fulcrum Agreement also provided for potential de minimis minimum annual royalty payments as well as sales-based royalties of up to the low-double digits upon commercialization. In April 2026, Fulcrum delivered written notice to the Company to terminate the Fulcrum Agreement for convenience. In accordance with the terms of the Fulcrum Agreement, the termination will become effective following the applicable notice period. The Company does not expect to receive any future milestone or royalty payments under the Fulcrum Agreement.
The Company assessed this arrangement in accordance with ASC Topic 606, Revenue from Contracts with Customers (“ASC 606”) and concluded that the contract counterparty is a customer. In accordance with ASC 606, the Company determined that there is one performance obligation in the Fulcrum Agreement, consisting of the exclusive and non-exclusive license rights granted to Fulcrum. The transaction price was comprised of the fixed consideration of $0.4 million and was recognized upon transfer of control of the licenses at a point in time upon contract execution. The arrangement includes significant variable consideration, primarily in the form of milestone payments, which was fully constrained at the inception of the contract. The sales-based royalty fee qualifies for the royalty constraint exception and does not require an estimate of the future transaction price. The Company did not record any license revenue pursuant to the Fulcrum Agreement for either of the three months ended March 31, 2026 or 2025.
All variable consideration is remeasured and reassessed each reporting period. As of and for the three months ended March 31, 2026 and 2025, the Company determined all remaining variable consideration was fully constrained.
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Research and Collaboration Agreements
BioMarin Pharmaceutical Inc.
In September 2024, the Company entered into a Collaboration and License Agreement (the “BioMarin Agreement”) with BioMarin Pharmaceutical Inc. (“BioMarin”). Under the terms of the BioMarin Agreement, BioMarin paid the Company an upfront, nonrefundable payment of $1.0 million, and reimbursed the Company for certain research activities. As of March 31, 2026, the BioMarin Agreement has terminated in accordance with its terms.
The Company assessed this arrangement in accordance with ASC 606 and concluded that BioMarin is a customer and there is one combined performance obligation, which includes performance of R&D activities in accordance with a contractual work plan, participation in a joint steering committee, the grant of an exclusive license to BioMarin, the grant of a non-exclusive license to the data resulting from performance of the R&D activities, and providing quarterly progress reports. The transaction price was determined to be fixed consideration of $3.8 million. The work was primarily performed during 2024 and 2025 and revenue was recognized over time based upon a cost-to-cost method. During the three months ended March 31, 2026 and 2025, the Company recognized $0.1 million and $0.9 million, respectively, in collaboration revenue under the BioMarin Agreement.
Following the termination of the BioMarin Agreement, the Company does not expect to receive any additional variable consideration.
GlaxoSmithKline Intellectual Property (No. 3) Limited (“GSK”)
In December 2025, the Company entered into a Research, Collaboration and License Agreement (the “GSK Agreement”) with GlaxoSmithKline Intellectual Property (No. 3) Limited (“GSK”). Under the terms of the GSK Agreement, GSK paid the Company a one-time, nonrefundable upfront payment of $17.5 million. In addition, the Company is 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 the Company 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.
The Company assessed this arrangement in accordance with ASC 606 and concluded that GSK is a customer and there is one combined performance obligation, which includes performance of R&D activities in accordance with a contractual work plan, participation in a joint steering committee, the grant of an exclusive license to GSK, the grant of a non-exclusive license to the data resulting from performance of the R&D activities, and providing quarterly progress reports. The transaction price was determined to be fixed consideration of $17.5 million. Performance of the work plan began in 2026 and revenue will be recognized over time based upon a cost-to-cost method. The Company recognized $1.2 million in collaboration revenue under the GSK Agreement during the three months ended March 31, 2026. Additionally, the Company recognized a contract liability in short-term deferred revenue of $11.1 million and long-term deferred revenue of $5.2 million on the condensed consolidated balance sheet as of March 31, 2026 related to the upfront payment.
The arrangement includes significant variable consideration primarily in the form of milestone payments, which was fully constrained at the inception of the contract. All variable consideration is remeasured and reassessed each reporting period. As of and for the three months ended March 31, 2026, the Company determined the variable consideration was fully constrained.
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6. Derivative Tranche Liability
Pursuant to the Purchase Agreement, subject to the occurrence of the second closing triggers, the Company has agreed to sell and the investors have agreed to purchase at a closing (the “Second Closing”) up to 32,681,866 shares of the Company’s common stock or pre-funded warrants in lieu thereof at a purchase price of $1.53 per share and $1.5299 per pre-funded warrant, and directors, consultants and members of management have agreed to purchase an additional 39,306 shares of the Company’s common stock at a purchase price of $1.65 per share, for gross proceeds to the Company of up to approximately $50.1 million. The Second Closing triggers include a certain regulatory milestone and either of the following:
1.the achievement of a volume weighted average price per share of equal to or greater than $7.50 , as defined in the Purchase Agreement, measured during any consecutive trading days during the trading days following the date of the Company’s first announcement of the regulatory milestone (the “Price Threshold”), or
2.the Company’s receipt of a signed written notice from investors holding a majority of the Securities outstanding that waives the Price Threshold for purposes of the Second Closing in which case, only the waiving investors will be obligated to participate.
The obligation to issue and purchase securities was concluded to be a forward contract derivative liability and was measured at fair value using a probability weighted model at the issuance date. The initial fair value (Level 3) of the forward contract was $14.9 million and was recorded as a derivative tranche liability and was remeasured to $44.8 million as of December 31, 2025. For the three months ended March 31, 2026, the derivative tranche liability was remeasured to $50.9 million with a change in fair-value of $6.2 million recorded in the condensed consolidated statements of operations and comprehensive loss.
7. Stockholders’ Equity
Common and Preferred Stock
The Company’s amended and restated certificate of incorporation authorizes the issuance of up to 175,000,000 shares of $0.0001 par value common stock and up to 25,000,000 shares of $0.0001 par value undesignated preferred stock. The Board may designate the rights, preferences, privileges, and restrictions of the preferred stock, including dividend rights, conversion rights, voting rights, terms of redemption, liquidation preference, and number of shares constituting any series or the designation of any series. The issuance of preferred stock could have the effect of restricting dividends on the Company’s common stock, diluting the voting power of the Company’s common stock, impairing the liquidation rights of the Company’s common stock, or delaying or preventing a change in control. As of March 31, 2026, no shares of preferred stock were outstanding.
Recent Equity Financings - Private Placement
The Initial Closing of the Private Placement occurred on September 11, 2025. At the Initial Closing, the Company issued and sold 26,681,053 shares of the Company’s common stock priced at $1.53 per share to certain investors, some of which were affiliated with directors of the Company, and 36,361 shares of common stock priced at $1.65 to directors, management and consultants of the Company (collectively, the “Shares”). In lieu of common stock, certain investors were sold pre-funded warrants to purchase 6,003,758 shares of common stock (the “Warrant Shares,” and together with the Shares, the “Securities”) at an offering price of $1.5299 per pre-funded warrant. Gross proceeds to the Company totaled $50.1 million before deducting $3.3 million of placement agent fees and other expenses.
The pre-funded warrants in the Initial Closing, and those that may be issued in the conditional Second Closing, have an exercise price of $0.0001 per Warrant Share, subject to customary adjustments, will be exercisable at any time after original issuance, will not expire until exercised in full, and will be exercisable on a net exercise “cashless” basis. The pre-funded warrants may not be exercised if the aggregate number of shares of common stock beneficially owned by the holder thereof immediately following such exercise would exceed a specified beneficial ownership limitation, not to exceed 19.99 %.
In connection with the Private Placement, on November 3, 2025, the Company filed an initial registration statement on Form S-3 (the “Initial Registration Statement”) with the SEC, which subsequently became effective, to register the Shares for resale and, as applicable, the Warrant Shares, issued in the Initial Closing. If applicable, within 30 days of the Second Closing and subject to certain allowable delays, the Company will prepare and file a second registration
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statement (the “Second Registration Statement”) with the SEC to register the shares for resale and, as applicable, the warrant shares, in each case that are issued in connection with the Second Closing. The Company will use reasonable best efforts to cause any Second Registration Statement and any amendments thereto to promptly become effective, but in no event later than the earlier of (i) the 75 th calendar day following any Second Registration Statement filing date and (ii) the fifth business day after the date the Company is notified by the SEC that any Second Registration Statement, as applicable, will not be “reviewed” or will not be subject to further review.
The Company concluded that the pre-funded warrants issued in the Initial Closing met the conditions to be classified as equity instruments.
At-the-Market Sales Agreement
On November 10, 2025, the Company filed a shelf registration statement on Form S-3 (the “Shelf Registration Statement”), which subsequently became effective. Pursuant to the Shelf Registration Statement, the Company may offer and sell up to $300.0 million of a variety of securities, including common stock, preferred stock, warrants or debt securities.
8. Stock-Based Compensation
In October 2024, the Company adopted the 2024 Equity Incentive Plan (the “2024 Plan”), which became effective in connection with the Company’s IPO. The 2024 Plan provides for the grant of stock options, stock appreciation rights, restricted and unrestricted stock awards, restricted stock unit awards, and other stock-based awards to employees, directors, and non-employee service providers of the Company.
Awards granted under the 2024 Plan expire no later than ten years from the date of grant. The price of stock options shall not be less than 100 % of the estimated fair value on the date of grant and typically vest over a four-year period, although awards may be granted with different vesting terms. The 2024 Plan initially reserved 2,143,039 shares of common stock for the issuance of future awards and provides for an automatic annual increase in the number of shares of common stock reserved for future issuance by the lesser of (i) 5 % of the number of shares of the Company’s common stock outstanding as of the close of business on the immediately preceding December 31 and (ii) the number of shares determined by the Company’s Board of Directors on or prior to such date for such year. As of March 31, 2026, there were 3,015,283 shares of common stock reserved and available for issuance pursuant to the 2024 Plan.
During the three months ended March 31, 2026, the Company granted options for the purchase of 28,000 shares of common stock.
The weighted-average assumptions used to estimate the fair value of the stock options granted during the three months ended March 31, 2026 were as follows:
| Three months ended March 31, 2026 | |||||
| Expected volatility | % | ||||
| Risk-free interest rate | % | ||||
| Expected dividend yield | % | ||||
| Expected term (in years) | |||||
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During the three months ended March 31, 2026, the weighted average grant-date fair value of options granted was $3.71 .
During the three months ended March 31, 2026, employees of the Company purchased an aggregate of 15,562 shares under the 2024 Employee Stock Purchase Plan.
The following table presents the classification of stock-based compensation expense for the three months ended March 31, 2026 and 2025 (in thousands):
| Three Months Ended March 31, | |||||||||||
| 2026 | 2025 | ||||||||||
| Research and development | $ | $ | |||||||||
| General and administrative | |||||||||||
| Total | $ | $ | |||||||||
The Company has an aggregate of $10.4 million of gross unrecognized stock-based compensation expense as of March 31, 2026, which is expected to be recognized over a weighted average period of 2.5 years.
9. Income Taxes
10. Segment Reporting
The Company manages its business activities on a consolidated basis and operates as a single operating segment: R&D, which engages in the business of developing a new class of RNA-based therapeutics to treat a broad range of genetic diseases. The Company's operations are primarily in the United States.
The Company’s CODM is its Chief Executive Officer. The CODM uses net loss, as reported on the Company’s condensed consolidated statement of operations and comprehensive loss, in evaluating performance and determining how to allocate resources. The CODM does not review assets in evaluating the results and therefore, such information is not presented.
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The operating financial results of the Company’s R&D segment for the three months ended March 31, 2026 and 2025 are as follows (in thousands):
| Three Months Ended March 31, | |||||||||||
| 2026 | 2025 | ||||||||||
| Revenue | $ | $ | |||||||||
| Less: Significant segment expenses | |||||||||||
| Personnel-related expenses | |||||||||||
| Clinical and preclinical expenses | |||||||||||
| Facilities-related and overhead | |||||||||||
| Professional and consulting fees | |||||||||||
| Corporate expenses | |||||||||||
| Travel and entertainment | |||||||||||
| Plus: Other segment (loss) income | ( | ||||||||||
| Segment net loss | $ | ( | $ | ( | |||||||
Other segment income includes total other income, net on the condensed consolidated statements of operations.
11. Net Loss Per Share Attributable to Common Stockholders
| Three Months Ended March 31, | |||||||||||
| 2026 | 2025 | ||||||||||
| Numerator: | |||||||||||
| Net loss attributable to common stockholders | $ | ( | $ | ( | |||||||
| Denominator: | |||||||||||
| Weighted-average common shares outstanding, basic and diluted (1) | |||||||||||
| Net loss per share attributable to common stockholders, basic and diluted | $ | ( | $ | ( | |||||||
The Company’s potentially dilutive securities, which include or have included convertible preferred stock, outstanding stock options, unvested restricted common stock, and warrants to purchase stock, have been excluded from the computation of diluted net loss per share as the effect would be to reduce the net loss per share. Therefore, the weighted-average number of common shares outstanding used to calculate both basic and diluted net loss per share attributable to common stockholders is the same.
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The Company excluded the following from the computation of diluted net loss per share attributable to common stockholders as of March 31, 2026 and 2025 because their inclusion would have had an anti-dilutive effect:
| Three Months Ended March 31, | ||||||||||||||
| 2026 | 2025 | |||||||||||||
| Shares issuable upon settlement of derivative tranche liability in Private Placement | ||||||||||||||
| Stock options outstanding | ||||||||||||||
| Restricted stock vesting | ||||||||||||||
| Warrants outstanding | ||||||||||||||
| Total | ||||||||||||||
12. Related Parties
In September 2015, the Company entered into consulting agreements with its two founders, related parties who hold shares of the Company’s common stock, to provide R&D and strategic planning services. For both the three months ended March 31, 2026 and 2025, the Company recognized R&D expense of $0.1 0.1 no
In March 2019, the Company entered into a consulting agreement with an executive consultant, a related party who holds shares of the Company’s common stock. For each of the three months ended March 31, 2026 and 2025, the Company recognized G&A expense totaling less than $0.1 0.1 no
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Item 2. Management’s Discussion and Analysis of Financial Condition and Results of Operations
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,” 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.
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.
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 submitted our first regulatory filing in Australia to enable initiation of a clinical trial in individuals with SYNGAP1, with additional global regulatory filings planned in 2026. Pending regulatory clearance, we intend to initiate the global Phase 1/2 clinical trial in the second half of 2026.
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.
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.
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
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October 15, 2024, as well as through revenues from our license and collaboration agreements. Through March 31, 2026, 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 March 31, 2026, we have recognized $22.8 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 $18.3 million and $12.4 million for the three months ended March 31, 2026 and 2025, respectively. As of March 31, 2026, we had an accumulated deficit of $310.5 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.
We anticipate that our expenses will increase substantially if and as we:
•finalize preclinical development for CMP-002 and advance into clinical trials;
•advance current and future product candidates through preclinical studies and clinical trials;
•expand the capabilities of our RAP Platform and seek to identify and develop additional product candidates;
•seek marketing approvals for any product candidates that successfully complete clinical trials;
•obtain, expand, maintain, defend and enforce our intellectual property portfolio;
•hire additional clinical, regulatory and scientific personnel;
•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;
•ultimately establish a sales, marketing and distribution infrastructure to commercialize any products for which we may obtain marketing approval; and
•add operational, legal, compliance, financial and management information systems and personnel to support our research, product development and future commercialization efforts.
Because of the numerous risks and uncertainties associated with the development of therapeutics, we are unable to accurately predict the timing or amount of increased expenses and when, or if, we will be able to achieve or maintain profitability. Even if we are able to generate product sales, we may not become profitable. If we fail to become profitable or are unable to sustain profitability on a continuing basis, then we may be unable to continue our operations as planned and may be forced to reduce or terminate our operations.
We do not have any products approved for sale and have not generated any revenue from product sales. We will not generate revenue from product sales unless and until we successfully complete clinical development and obtain regulatory approval for our current or any future product candidates, which we expect will take a number of years or may never occur. As a result, we will need substantial additional funding to support our continuing operations and pursue our growth strategy. Until such time as we can generate significant revenue from product sales, if ever, we expect to finance our operations through equity offerings, debt financings, or other capital sources, including current and potential future
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collaborations, license agreements, and other similar arrangements. However, we may be unable to raise additional funds or enter into such other agreements or arrangements when needed on favorable terms, or at all. If we fail to raise capital or enter into such agreements or arrangements as, and when needed, we may delay, limit, reduce or terminate our product development or future commercialization efforts or grant rights to develop and market product candidates that we would otherwise develop and market ourselves, or even cease operations.
As of March 31, 2026, we had cash and cash equivalents of $99.2 million. Based on our current operating plan, we estimate that our cash and cash equivalents as of March 31, 2026 will be sufficient to fund our operating expenses and capital expenditure requirements into 2028. However, we have based this estimate on assumptions that may prove to be wrong, and we could deplete our capital resources sooner than we currently expect. See the sections titled “—Liquidity and Capital Resources” and “Risk Factors—Risks Related to our Financial Position and Need for Additional Capital” included in our 2025 Form 10-K.
We do not own or operate and currently have no plans to establish any manufacturing facilities. We rely, and expect to continue to rely, on third parties for clinical supply as well as commercial supply if we obtain marketing approval. In addition, we rely on third parties to package, label, store, and distribute our clinical supply and we intend to rely on third parties to conduct the same activities for our commercial products if we obtain regulatory approval. We believe that this strategy allows us to maintain a more efficient infrastructure by eliminating the need for us to invest in our own manufacturing facilities, equipment, and personnel while also enabling us to focus our expertise and resources on the development of product candidates and continued enhancement of our RAP Platform.
Results of Operations
Comparison of the Three Months Ended March 31, 2026 and 2025
The following table summarizes our results of operations for the three months ended March 31, 2026 and 2025 (in thousands):
| 2026 | 2025 | Change ($) | |||||||||||||||
| Revenue | |||||||||||||||||
| Research and collaboration revenue | $ | 1,294 | $ | 858 | $ | 436 | |||||||||||
| Operating expenses: | |||||||||||||||||
| Research and development | 10,160 | 10,146 | 14 | ||||||||||||||
| General and administrative | 4,205 | 3,812 | 393 | ||||||||||||||
| Total operating expenses | 14,365 | 13,958 | 407 | ||||||||||||||
| Loss from operations | (13,071) | (13,100) | 29 | ||||||||||||||
| Other (expense) income, net: | |||||||||||||||||
| Interest income | 910 | 588 | 322 | ||||||||||||||
| Change in fair value of derivative tranche liability | (6,188) | — | (6,188) | ||||||||||||||
| Other income | 18 | 79 | (61) | ||||||||||||||
| Total other (expense) income, net | (5,260) | 667 | (5,927) | ||||||||||||||
| Net loss | $ | (18,331) | $ | (12,433) | $ | (5,898) | |||||||||||
Research and Collaboration Revenue
We recognized $1.3 million in research and collaboration revenue during the three months ended March 31, 2026, compared to $0.9 million during the three months ended March 31, 2025. Research and collaboration revenue recognized in the three months ended March 31, 2026 primarily related to our Research, Collaboration and License Agreement with GlaxoSmithKline Intellectual Property (No. 3) Limited (“GSK”) and research and collaboration revenue recognized in the three months ended March 31, 2025 related to our Collaboration and License Agreement with BioMarin.
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Research and Development Expenses
The following table summarizes our R&D expenses for the three months ended March 31, 2026 and 2025 (in thousands):
| 2026 | 2025 | Change ($) | |||||||||||||||
| Clinical and preclinical expenses | $ | 4,631 | $ | 4,479 | $ | 152 | |||||||||||
| Personnel-related expenses | 3,467 | 3,167 | 300 | ||||||||||||||
| Facilities-related and overhead expense | 1,434 | 1,446 | (12) | ||||||||||||||
| Professional and consulting fees | 384 | 834 | (450) | ||||||||||||||
| Other expenses | 244 | 220 | 24 | ||||||||||||||
| Total R&D expenses | $ | 10,160 | $ | 10,146 | $ | 14 | |||||||||||
R&D expenses were $10.2 million for the three months ended March 31, 2026, compared to $10.1 million for the three months ended March 31, 2025. The nominal increase was primarily driven by higher personnel-related costs of $0.3 million due to increased stock-based compensation expense and higher 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 decrease of $0.5 million in professional and consulting fees.
General and Administrative Expenses
The following table summarizes our G&A expenses for the three months ended March 31, 2026 and 2025 (in thousands):
| 2026 | 2025 | Change ($) | |||||||||||||||
| Personnel-related expenses | $ | 2,408 | $ | 2,019 | $ | 389 | |||||||||||
Professional and consulting fees | 1,087 | 768 | 319 | ||||||||||||||
| Facilities-related and overhead expense | 359 | 530 | (171) | ||||||||||||||
| Other expenses | 351 | 495 | (144) | ||||||||||||||
| Total G&A expenses | $ | 4,205 | $ | 3,812 | $ | 393 | |||||||||||
G&A expenses were $4.2 million for the three months ended March 31, 2026, compared to $3.8 million for the three months ended March 31, 2025. The increase of $0.4 million was primarily driven by 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. These increases were partially offset by a decrease of $0.2 million in facilities and overhead expenses due to 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.
Other (Expense) Income, Net
Other (expense) income, net for the three months ended March 31, 2026 was a $5.3 million expense, compared to $0.7 million of income for the three months ended March 31, 2025. The change was primarily due to a $6.2 million non-cash expense for the change in fair value of our derivative tranche liability associated with the Second Closing of our Private Placement. This expense was partially offset by a $0.3 million increase in interest income due to higher average invested cash balances during the three months ended March 31, 2026.
Liquidity and Capital Resources
Sources of Liquidity
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
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the foreseeable future as we advance the development of product candidates. Through March 31, 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 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.
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 months ended March 31, 2026 and the year ended December 31, 2025, we did not issue any shares of our common stock under the Sales Agreement.
As of March 31, 2026, we had cash and cash equivalents of $99.2 million. Based on our current operating plan, we estimate that our cash and cash equivalents as of March 31, 2026 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.
Future Funding Requirements
We expect our expenses to increase substantially in connection with our ongoing activities, particularly as we continue our development of, seek regulatory approval for, and potentially commercialize our product candidates and seek to discover and develop additional product candidates, conduct our ongoing and planned clinical trials and preclinical studies, continue our R&D activities, hire additional personnel, expand and protect our intellectual property, and incur additional costs associated with being a public company.
The timing and amount of our future funding requirements will depend on many factors, including:
•the initiation, type, number, scope, progress, expansions, results, costs and timing of preclinical studies and clinical trials of our product candidates 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;
•the costs and timing of manufacturing for our product candidates, including commercial manufacturing at sufficient scale, if any product candidate is approved;
•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;
•the costs of obtaining, maintaining, enforcing and protecting our patents and other intellectual property and proprietary rights;
•our efforts to enhance operational systems and hire additional personnel to satisfy our obligations as a public company, including enhanced internal control over financial reporting;
•the costs associated with hiring additional personnel and consultants as our clinical and preclinical activities increase;
•the timing and payment of milestone, royalty or other payments we must make or may receive pursuant to our existing and potential future license or collaboration agreements with third parties;
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•the costs and timing of establishing or securing sales and marketing capabilities if our product candidates or any future product candidate is approved;
•our ability to achieve sufficient market acceptance, coverage, and adequate reimbursement from third-party payors and adequate market share and revenue for any approved products;
•patients’ ability and willingness to pay out-of-pocket for any approved products in the absence of coverage and/or adequate reimbursement from third-party payors;
•the terms and timing of establishing and maintaining collaborations, licenses and other similar arrangements; and
•costs associated with any products or technologies that we may in-license or acquire.
Our operating plans and other demands for our cash resources may change because of many factors currently unknown to us, and we may need to seek additional funds sooner than planned.
We have no other committed sources of capital. Until such time, if ever, we can generate substantial product revenues, we expect to finance our operations through the sale of equity securities, debt financings, working capital lines of credit, strategic alliances and/or license arrangements, grant funding, interest income earned on invested cash balances or a combination of two or more of these sources. However, we may be unable to raise additional funds or enter into such other arrangements when needed, on favorable terms or at all. To the extent we raise additional capital through the sale of equity or convertible debt securities, investors’ ownership interest will be diluted, and the terms of these securities may include liquidation or other preferences that adversely affect the rights of a common stockholder. Debt financing and preferred equity financing, if available, may involve agreements that include covenants limiting or restricting our ability to take specific actions, such as incurring additional debt, engaging in acquisition, merger or collaboration transactions, selling or licensing our assets, making capital expenditures, redeeming our stock, making certain investments or declaring dividends. If we raise additional funds through collaborations or license agreements with third parties, we may have to relinquish valuable rights to our technologies, future revenue streams, research programs or product candidates, or grant licenses on terms that may not be favorable to us. If we are unable to raise additional funds through equity or debt financings when needed, we may be required to delay, limit, reduce or terminate our product development or future commercialization efforts or grant rights to develop and market product candidates that we would otherwise prefer to develop and market ourselves, or even cease operations.
Contractual and Other Obligations
As of March 31, 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.
Cash Flows
For the Three Months Ended March 31, 2026 and 2025
The following table provides information regarding our cash flows for the three months ended March 31, 2026 and 2025 (in thousands):
| 2026 | 2025 | ||||||||||
| Net cash used in operating activities | $ | (10,981) | $ | (14,286) | |||||||
| Net cash used in investing activities | — | (279) | |||||||||
| Net cash provided by (used in) financing activities | 57 | (151) | |||||||||
| Net change in cash, cash equivalents, and restricted cash | $ | (10,924) | $ | (14,716) | |||||||
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Operating Activities
During the three months ended March 31, 2026, operating activities used $11.0 million of cash, primarily resulting from our net loss of $18.3 million, and net cash used in changes in our operating assets and liabilities of $0.9 million, partially offset by non-cash charges of $8.2 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 three months ended March 31, 2025, operating activities used $14.3 million of cash, primarily resulting from our net loss of $12.4 million and net cash used in changes in our operating assets and liabilities of $3.7 million, partially offset by non-cash charges of $1.9 million, including stock-based compensation expense, non-cash operating lease expense, and depreciation and amortization.
In each of the three months ended March 31, 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 for our Cambridge and Boulder leases.
Investing Activities
During the three months ended March 31, 2026, there was no net cash used in or provided by investing activities.
During the three months ended March 31, 2025, net cash used in investing activities was $0.3 million, due to purchases of property and equipment.
Financing Activities
During the three months ended March 31, 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 three months ended March 31, 2025, net cash used by financing activities was $0.2 million, consisting primarily of principal payments on finance leases.
Critical Accounting Policies and Significant Judgments and Estimates
There have been no significant changes to our critical accounting estimates in the preparation of our condensed consolidated financial statements during the three months ended March 31, 2026 compared to those disclosed in our 2025 Form 10-K.
Recently Issued Accounting Standards
See Note 1 to our condensed consolidated financial statements included elsewhere in this Quarterly Report for more information.
Emerging Growth Company and Smaller Reporting Company Status
We are an emerging growth company, as defined in the Jumpstart Our Business Startups Act of 2012 (the “JOBS Act”), and we may remain an emerging growth company until December 31, 2029 or until such earlier time that we are no longer an emerging growth company. For so long as we remain an emerging growth company, we are permitted and intend to rely on certain exemptions from various public company reporting requirements, including not being required to have our internal control over financial reporting audited by our independent registered public accounting firm pursuant to Section 404 of the Sarbanes-Oxley Act, reduced disclosure obligations regarding executive compensation in our periodic reports and proxy statements, exemptions from the requirements of holding a nonbinding advisory vote on executive compensation and any golden parachute payments not previously approved and an exemption from compliance with the requirements regarding the communication of critical audit matters in the auditor’s report on financial statements.
In addition, the JOBS Act provides that an emerging growth company can take advantage of an extended transition period for complying with new or revised accounting standards. We have elected to avail ourselves of this extended transition period, which means that when a standard is issued or revised and it has different application dates for
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public or private companies, we will adopt the new or revised standard at the time private companies adopt the new or revised standard and will do so until such time that we either (i) irrevocably elect to “opt out” of such extended transition period or (ii) no longer qualify as an emerging growth company. We may choose to early adopt any new or revised accounting standards whenever such early adoption is permitted for private companies. As a result of this election, our financial statements may not be comparable to those of companies that are not emerging growth companies.
We will remain an emerging growth company until the earliest to occur of: (i) the last day of the fiscal year in which we have at least $1.235 billion in annual revenue; (ii) the last day of the fiscal year in which we are deemed to be a “large accelerated filer,” as defined in Rule 12b-2 under the Exchange Act, which would occur if the market value of our common stock held by non-affiliates exceeded $700.0 million as of the last business day of the second fiscal quarter of such year; (iii) the date on which we have issued more than $1.0 billion in nonconvertible debt securities during the prior three-year period; and (iv) December 31, 2029.
We are also a “smaller reporting company,” meaning that the market value of our shares held by non-affiliates is less than $700.0 million and our annual revenue was less than $100.0 million during the most recently completed fiscal year. We may continue to be a smaller reporting company if either: (i) the market value of our shares held by non-affiliates is less than $250.0 million; or (ii) our annual revenue was less than $100.0 million during the most recently completed fiscal year and the market value of our shares held by non-affiliates is less than $700.0 million. If we are a smaller reporting company at the time we cease to be an emerging growth company, we may continue to rely on exemptions from certain disclosure requirements that are available to smaller reporting companies. Specifically, as a smaller reporting company, we may choose to present only the two most recent fiscal years of audited financial statements in our Annual Report on Form 10-K and, similar to emerging growth companies, smaller reporting companies have reduced disclosure obligations regarding executive compensation.
Item 3. Quantitative and Qualitative Disclosures About Market Risk
Not applicable to a smaller reporting company.
Item 4. Controls and Procedures
Evaluation of Disclosure Controls and Procedures
Our management, with the participation of our principal executive officer and our principal financial officer, evaluated the effectiveness of our disclosure controls and procedures as defined in Rules 13a-15(e) and 15d-15(e) under the Securities Exchange Act of 1934 as of March 31, 2026. The term “disclosure controls and procedures,” as defined in Rules 13a-15(e) and 15d-15(e) under the Exchange Act, means controls and other procedures of a company that are designed to ensure that information required to be disclosed by us in the reports that we file or submits under the Exchange Act is recorded, processed, summarized and reported, within the time periods specified in the SEC’s rules and forms. Disclosure controls and procedures include, without limitation, controls and procedures designed to ensure that information required to be disclosed by a company in the reports that it files or submits under the Exchange Act is accumulated and communicated to the company’s management, including its principal executive and principal financial officers, or persons performing similar functions, as appropriate to allow timely decisions regarding required disclosure. Management recognizes that any controls and procedures, no matter how well designed and operated, can provide only reasonable assurance of achieving their objectives and management necessarily applies its judgment in evaluating the cost-benefit relationship of possible controls and procedures. Based on the evaluation of our disclosure controls and procedures as of March 31, 2026, our Chief Executive Officer and Chief Financial Officer concluded that, as of such date, our disclosure controls and procedures were effective at the reasonable assurance level.
Changes in Internal Control Over Financial Reporting
There were no changes in our internal control over financial reporting (as defined in Rules 13a-15(f) and 15d-15(f) under the Exchange Act) during the quarter ended March 31, 2026 that have materially affected, or are reasonably likely to materially affect, our internal control over financial reporting.
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PART II — OTHER INFORMATION
Item 1. Legal Proceedings
From time to time, we may be subject to legal proceedings. We are not currently a party to or aware of any proceedings that we believe will have, individually or in the aggregate, a material adverse effect on our business, financial condition or results of operations. Regardless of outcome, litigation can have an adverse impact on us because of defense and settlement costs, diversion of management resources, and other factors.
Item 1A. Risk Factors
In addition to the other information set forth in this Quarterly Report on Form 10-Q, you should carefully consider the factors discussed in Part I, “Item 1A. Risk Factors” in our Annual Report on Form 10-K for the year ended December 31, 2025 (the “2025 Form 10-K”), which could materially affect our business, financial condition or future results. The risk factors disclosure in our 2025 Form 10-K is qualified by the information that is described in this Quarterly Report on Form 10-Q. The risks described in our 2025 Form 10-K are not our only risks. Additional risks and uncertainties not presently known to us or that we currently believe to be immaterial also may materially adversely affect our business, financial condition or future results. There have been no material changes to our risk factors as previously disclosed in the 2025 Form 10-K except as follows:
Risks Related to Regulatory Approval and Commercialization
We have received orphan drug designation for CMP-001 and CMP-002 in certain jurisdictions, are pursuing orphan drug designation for CMP-002 in the United States, and may seek orphan drug designation for other product candidates in the future. 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-001 for the treatment of UCDs in September 2024. In addition, the EMA granted orphan drug designation to CMP-002 for the treatment of SYNGAP1 in April 2026, and we have submitted an orphan drug designation request to the FDA for CMP-002. However, we may not be able to obtain or maintain the benefits of such designations, 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.
Orphan drug designation entitles a party to financial incentives such as opportunities for grant funding towards clinical trial costs, tax advantages and application fee waivers. After the FDA grants orphan drug designation, the generic identity of the product candidate and its potential orphan use are disclosed publicly by the FDA. In addition, if a product candidate with an orphan drug designation subsequently receives the first marketing approval for the indication for which it has such designation, the product is entitled to a period of marketing exclusivity, which precludes the FDA from approving another marketing application for the same product for the same therapeutic indication for seven years.
Even if we obtain orphan drug exclusivity for a product, that exclusivity may not effectively protect the product from competition because different products can be approved for the same condition. In addition, even after an orphan drug is approved, the FDA can subsequently approve the same product for the same condition if the FDA concludes that the later product is clinically superior in that it is shown to be safer, more effective or makes a major contribution to patient care. Orphan drug exclusivity may also be lost if the FDA determines that the request for designation was materially defective or if the manufacturer is unable to assure sufficient quantity of the product to meet the needs of the patients with the rare disease or condition. Further, even if we obtain orphan drug designation, we may not be the first to obtain marketing approval for any particular orphan indication due to the uncertainties associated with developing pharmaceutical products.
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Finally, it is unclear how future litigation, legislation, agency decisions, or administrative actions may impact the scope of the orphan drug exclusivity. 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.
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Item 2. Unregistered Sales of Equity Securities and Use of Proceeds
(a)Sales of Unregistered Securities
During the quarter ended March 31, 2026, we granted stock options to two new employees to purchase a total of 16,000 shares of our common stock at a weighted average exercise price of $4.82 per share. These options were inducement grants made outside of our 2024 Equity Incentive Plan in accordance with Nasdaq Listing Rule 5635(c)(4) and Section 4(a)(2) of the Securities Act of 1933, as amended (the “Securities Act”). The options have a ten-year term and vest over four years, with 25% of the shares underlying the option award vesting on the one-year anniversary of the applicable employee’s new hire date and the remaining 75% of the shares underlying the award vesting monthly thereafter for three years. Vesting of each option is subject to the employee’s continued service with our company through the applicable vesting date. We subsequently filed a registration statement on Form S-8 to register the shares of common stock underlying these options.
(b)Use of Proceeds from Public Offering of Common Stock
On October 10, 2024, our Registration Statement on Form S-1, as amended (File No. 333-282241), was declared effective in connection with our IPO, pursuant to which we sold an aggregate of 6,820,000 shares of our common stock at a price to the public of $11.00 per share. The underwriters of our IPO were J.P. Morgan Securities LLC, Leerink Partners LLC, Piper Sandler & Co. and William Blair & Company, L.L.C.
Our IPO closed on October 15, 2024. In connection with the IPO, we granted the underwriters a 30-day option to purchase an additional 1,023,000 shares of common stock. On November 1, 2024, pursuant to the partial exercise by the underwriters of such option, we issued an additional 643,762 shares of common stock. We received aggregate gross proceeds of $82.1 million in connection with the IPO and subsequent exercise of the underwriters’ option and aggregate net proceeds of $76.4 million after deducting underwriting discounts and commissions payable by us. In connection with our IPO, no payments were made by us to directors, officers or persons owning ten percent or more of our ordinary shares or to their associates or to our affiliates. There has been no material change in the planned use of proceeds from our IPO as described in our prospectus filed pursuant to Rule 424(b)(4) under the Securities Act with the SEC on October 11, 2024. We are holding the balance of the net proceeds in cash and cash equivalents.
(c)Issuer Purchases of Equity Securities
None.
Item 3. Defaults Upon Senior Securities
Not applicable.
Item 4. Mine Safety Disclosures
Not applicable.
Item 5. Other Information
Item 6. Exhibits
See Exhibit Index.
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Table of Contents
EXHIBIT INDEX
| Exhibit No. | Description | |||||||
| 3.1 | Fifth Amended and Restated Certificate of Incorporation of CAMP4 Therapeutics Corporation (incorporated by reference to Exhibit 3.1 to the Form 8-K filed on October 15, 2024, File No. 001-42365) | |||||||
| 3.2 | Amended and Restated Bylaws of CAMP4 Therapeutics Corporation (incorporated by reference to Exhibit 3.2 to the Form 8-K filed on October 15, 2024, File No. 001-42365) | |||||||
10.1# | CAMP4 Therapeutics Corporation Amended and Restated Non-Employee Director Compensation Policy. | |||||||
| 31.1† | Certification of Chief Executive Officer Pursuant to Section 302 of the Sarbanes-Oxley Act of 2002 | |||||||
| 31.2† | Certification of Chief Financial Officer Pursuant to Section 302 of the Sarbanes-Oxley Act of 2002 | |||||||
| 32.1†* | Certification of Chief Executive Officer Pursuant to 18 U.S.C. Section 1350, as adopted pursuant to Section 906 of the Sarbanes-Oxley Act of 2002 | |||||||
| 32.2†* | Certification of Chief Financial Officer Pursuant to 18 U.S.C. Section 1350, as adopted pursuant to Section 906 of the Sarbanes-Oxley Act of 2002 | |||||||
| 101.INS† | XBRL Instance Document – the instance document does not appear in the Interactive Data File because its XBRL tags are embedded within the Inline XBRL document | |||||||
| 101.SCH† | XBRL Taxonomy Extension Schema Document | |||||||
| 101.CAL† | XBRL Taxonomy Extension Calculation Linkbase Document | |||||||
| 101.DEF† | XBRL Taxonomy Extension Definition Linkbase Document | |||||||
| 101.LAB† | XBRL Taxonomy Extension Label Linkbase Document | |||||||
| 101.PRE† | XBRL Taxonomy Extension Presentation Linkbase Document | |||||||
| 104† | Cover Page Interactive Data File (formatted in Inline XBRL and contained in Exhibit 101) | |||||||
†Filed herewith.
# Indicates management contract or compensatory plan.
*This certification will not be deemed “filed” for purposes of Section 18 of the Securities Exchange Act of 1934, as amended (the “Exchange Act”), or otherwise subject to the liability of that section. Such certification will not be deemed to be incorporated by reference into any filing under the Securities Act of 1933, as amended, or the Exchange Act, except to the extent specifically incorporated by reference into such filing.
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SIGNATURES
Pursuant to the requirements of the Securities Exchange Act of 1934, the Registrant has duly caused this report to be signed on its behalf by the undersigned, duly authorized.
Date: May 7, 2026
| CAMP4 Therapeutics Corporation | ||||||||
| By: | /s/ Josh Mandel-Brehm | |||||||
| Name: | Josh Mandel-Brehm | |||||||
| Title: | President and Chief Executive Officer | |||||||
| (Principal Executive Officer) | ||||||||
| By: | /s/ Kelly Gold | |||||||
| Name: | Kelly Gold | |||||||
| Title: | Chief Financial Officer | |||||||
| (Principal Financial Officer and Principal Accounting Officer) | ||||||||
35
FAQ
How did CAMP4 Therapeutics (CAMP) perform financially in Q1 2026?
CAMP4 reported a net loss of $18.3 million in Q1 2026, compared with $12.4 million a year earlier. Revenue from research and collaboration increased to $1.3 million, while operating cash outflow was $11.0 million as the company continued funding R&D.
What were CAMP4 Therapeutics’ cash and liquidity levels as of March 31, 2026?
As of March 31, 2026, CAMP4 held $99.2 million in cash and cash equivalents and total assets of $105.5 million. Management believes this cash balance will fund operating expenses and capital needs into 2028, assuming the current operating plan remains in place.
How much did CAMP4 Therapeutics spend on research and development in Q1 2026?
CAMP4 recorded $10.2 million in research and development expenses in Q1 2026, essentially flat versus Q1 2025. Spending reflected clinical and preclinical work, personnel costs, and platform development as the company advances CMP-002 and other discovery programs.
What drove the increase in CAMP4 Therapeutics’ net loss in Q1 2026?
The higher net loss was mainly due to a $6.2 million non-cash loss from the change in fair value of a derivative tranche liability linked to a potential second private-placement closing. Core operating expenses changed only modestly compared with the prior-year quarter.
What is CAMP4 Therapeutics’ lead program CMP-002 targeting?
CMP-002 is an antisense oligonucleotide designed to upregulate the SYNGAP1 gene for SYNGAP1-related disorder, a severe developmental and epileptic encephalopathy. Preclinical data showed restored SYNGAP protein levels and functional improvements, and CAMP4 plans a global Phase 1/2 trial starting in the second half of 2026.
How significant is the GSK collaboration to CAMP4 Therapeutics’ Q1 2026 results?
The GSK agreement provided a $17.5 million upfront payment, recognized over time. In Q1 2026, CAMP4 booked $1.2 million of collaboration revenue under this deal and reported remaining deferred revenue of $16.3 million, supporting ongoing RAP Platform and discovery activities.