Camp4 Therapeutics Corp
For the period ended Mar 31, 2026. Filed May 7, 2026.
View on SEC.govAbout this filing
A company's report for each of its first three quarters. The financial statements are not audited. It shows how the business has moved since the last annual report, and what management says changed.
Mar 31, 2026 · Filed May 7, 2026
CAMP$4.06+9.1%Stored close · as of Nov 6, 2026
What changed in Management’s Discussion and Analysis?
Read the current filingPrevious report: 10-Q
Period ended 2025-09-30; filed 2025-11-06.
0001736730-25-000094
Item 2. Management’s Discussion and Analysis of Financial Condition and Results of OperationsCurrent report: 10-Q
Period ended 2026-03-31; filed 2026-05-07.
0001628280-26-032146
Item 2. Management’s Discussion and Analysis of Financial Condition and Results of OperationsThe baseline is this issuer’s latest earlier reporting period of the same form that was filed before the current report. A previous 10-Q is a different quarter, not a year-over-year comparison. Amendments are excluded.
Compared 99 previous paragraphs and 83 current paragraphs.
52 unchanged; 14 reworded; 15 added; 30 removed; 3 previous and 2 current paragraphs rearranged.
Tables are not compared: 7 previous and 4 current tables excluded. Dates, whitespace, punctuation and numbering changes are ignored; changes to financial figures remain significant.
A changed paragraph is not evidence that risk increased or decreased. An unchanged paragraph does not establish that risk stayed the same.
Added paragraphs (15)
Current report
We are a clinical-stage biopharmaceutical company pioneering the discovery and development of a new class of RNA-targeting therapeutics with the goal of upregulating gene expression and restoring healthy protein levels to treat a broad range of genetic diseases. Our lead product candidate, CMP-002, has the potential to be the first disease-modifying therapy for the treatment of synaptic Ras GTPase activating protein 1 (“SYNGAP1”)-related disorder, or SYNGAP1, a severe developmental and epileptic encephalopathy (“DEE”) characterized by seizures, developmental delays, and cognitive impairments. SYNGAP1 is caused by haploinsufficiency of the SYNGAP1 gene, where mutation of one functional gene copy results in a reduction in SYNGAP protein levels of up to 50%. While we believe that it remains underdiagnosed, we estimate that there are approximately 21,000 individuals living with SYNGAP1 in the United States and the five largest European markets. There are no approved disease-modifying therapies for SYNGAP1.
Current report
Our primary therapeutic focus is on diseases of the central nervous system (“CNS”), where there are numerous rare and prevalent haploinsufficient diseases with no approved treatments for which a modest increase in protein expression has the potential to be clinically meaningful. In addition to our SYNGAP1 program, we are advancing discovery programs in other DEE indications. We also intend to leverage strategic discovery partnerships, including our research, collaboration, and license agreement with GlaxoSmithKline, to extend the application of our RAP Platform beyond the CNS and validate our approach to gene upregulation in additional tissues and disease areas.
Current report
•finalize preclinical development for CMP-002 and advance into clinical trials;
Current report
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.
Current report
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.
Current report
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.
Current report
In November 2025, we filed a shelf registration statement on Form S-3 (the “Shelf Registration Statement”). Pursuant to the Shelf Registration Statement, we may offer and sell securities having an aggregate public offering price of up to $300.0 million.
Current report
In connection with the filing of the Shelf Registration Statement, we also entered into a sales agreement (the “Sales Agreement”) with Leerink Partners LLC, as sales agent, pursuant to which we may issue and sell shares of our common stock for a maximum aggregate offering price of up to $100.0 million, which is included in the $300.0 million of securities that may be offered pursuant to the Shelf Registration Statement. Pursuant to the Sales Agreement, we will pay the sales agent a commission rate of up to 3.0% of the gross proceeds from the sale of any shares of our common stock. We are not obligated to make any sales of shares of our common stock under the Sales Agreement. During the 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.
Current report
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.
Current report
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.
Current report
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.
Current report
During the three months ended March 31, 2026, there was no net cash used in or provided by investing activities.
Current report
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.
Current report
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.
Current report
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.
Removed paragraphs (30)
Previous report
Overview
Previous report
Our pipeline also includes our clinical candidate, CMP-001 (formerly known as CMP-CPS-001), for the treatment of the most prevalent urea cycle disorders (“UCDs”), for which we have completed a Phase 1 clinical trial in healthy volunteers. UCDs are a group of severe, inherited metabolic diseases caused by mutations in the genes that encode one or more of the eight enzymes and transporters necessary to convert ammonia into urea. The inability of the body to properly metabolize ammonia leads to the accumulation of toxic levels in circulation, ultimately resulting in severe health outcomes, such as neurologic disability, seizure and death. CMP-001 is designed to improve urea cycle activity by amplifying expression of carbamoyl phosphate synthetase 1 (“CPS1”), an enzyme that catalyzes the first step of the urea cycle, by binding to a CPS1-specific regRNA. Our preclinical studies have demonstrated that modulating the activity of the target regRNA increases expression of the CPS1 gene, resulting in increased CPS1 enzyme levels, which allows for more ammonia to be converted into urea, thereby lowering ammonia levels to normal, healthy ranges. These preclinical studies also demonstrated that CMP-001 can increase the level of, or upregulate, the production of multiple enzymes responsible for converting ammonia into urea, potentially allowing us to address more than 85% of patients with UCDs. We have received approval of a clinical trial application in the Netherlands to enable the initiation of a Phase 1b expansion to enroll female participants who are heterozygous for a mutation of the OTC gene. We estimate that there are approximately 2,000 diagnosed female OTC heterozygotes in the United States who have inherited one copy of a gene with OTC-related changes and experience potentially addressable UCD symptoms.
Previous report
We have completed the analysis of safety, pharmacokinetic, and pharmacodynamic biomarker data from all four cohorts of the single ascending dose (“SAD”) portion of the clinical trial and from the three completed cohorts of the multiple ascending dose (“MAD”) portion of the clinical trial. In total, 86 healthy volunteer participants were observed, including 51 receiving CMP-001. CMP-001 demonstrated a favorable safety profile in both the SAD and MAD portions of the clinical trial, with no serious adverse events or discontinuations due to adverse events. Pharmacokinetic data similarly was observed to be consistent with expectations, demonstrating a dose-dependent increase in exposure (Cmax and AUC) with clear separation between groups and low-to-moderate variability in key pharmacokinetic parameters at all dose levels. There were no conclusive determinations of pharmacodynamic activity or non-activity in this healthy volunteer population, which we believe may have resulted from intrinsic variability in the ureagenesis rates of healthy individuals measured with the investigational 13C-sodium acetate test used in the study. We have made the strategic decision to pause new investment in our UCD program and to prioritize the development of our SYNGAP program. We continue to believe that CMP-001 has the potential to be the first disease-modifying therapy for the most prevalent UCDs and intend to pursue partnership opportunities to support the further development of CMP-001.
Previous report
In addition, we are party to a strategic research collaboration with BioMarin Pharmaceutical Inc. (“BioMarin”) to advance novel therapeutics that increase protein levels by targeting regRNA sequences for two genetic targets.
Previous report
•finalize preclinical development for our program in SYNGAP1-related disorders;
Previous report
We recognized $0.8 million in research and collaboration revenue during the three months ended September 30, 2025. We did not recognize any research and collaboration revenue during the three months ended September 30, 2024. The increase of $0.8 million was due to revenue recognized under our Collaboration and License Agreement with BioMarin.
Previous report
R&D expenses were $9.4 million for the three months ended September 30, 2025 compared to $9.7 million for the three months ended September 30, 2024. The decrease of $0.3 million was primarily due to a decrease of $0.3 million in clinical and preclinical expenses related to the Company’s UCD program. Other R&D expenses remained relatively comparable with the same period in the previous year.
Previous report
G&A expenses were $4.6 million for the three months ended September 30, 2025 compared to $3.8 million for the three months ended September 30, 2024. The increase of $0.8 million was partially due to an increase of $1.0 million in professional and consulting fees primarily due to private placement fees associated with our tranche liability and $0.2 million in facility-related and overhead expense due to increases in insurance premiums. These increases were partially offset by a $0.4 million decrease in personnel-related expenses primarily due to higher stock-based compensation for the three months ended September 30, 2024 associated with the forgiveness of promissory notes originally issued to certain executive officers.
Previous report
Impairment of Right-of-Use (“ROU”) Asset
Previous report
During the three months ended September 30, 2025, upon vacating our Boulder, Colorado location and pursuing a sublessee, it was determined that the market rate for similar space is less than the base rent the Company is paying under its current lease. As a result, the Company impaired the related ROU asset and recognized an impairment charge of $0.5 million.
Previous report
Comparison of the Nine Months Ended September 30, 2025 and 2024
Previous report
Research and Collaboration Revenue
Previous report
We recognized $3.2 million in research and collaboration revenue during the nine months ended September 30, 2025. We did not recognize any research and collaboration revenue during the nine months ended September 30, 2024. The increase of $3.2 million was due to revenue recognized under our Collaboration and License Agreement with BioMarin of $2.6 million and a $0.6 million milestone payment earned under the Fulcrum Agreement.
Previous report
Research and Development Expenses
Previous report
R&D expenses were $29.8 million for the nine months ended September 30, 2025 compared to $28.8 million for the nine months ended September 30, 2024. The increase of $1.0 million was primarily due to an increase of $0.4 million in personnel-related expenses due to lower offsetting personnel reimbursements under one of our collaboration agreements as well as severance and related costs resulting from separation agreements with certain former employees. We also incurred an increase of $0.4 million in professional and consulting fees due to increased utilization of external support for clinical operations. In addition, we incurred an increase of $0.2 million in clinical and preclinical expenses related to the Company’s UCD clinical trial and SYNGAP preclinical work.
Previous report
General and Administrative Expenses
Previous report
G&A expenses were $12.6 million for the nine months ended September 30, 2025 compared to $10.2 million for the nine months ended September 30, 2024. The increase of $2.4 million was partially due to an increase of $0.5 million in facility-related and overhead expense due to increases in insurance premiums. In addition, there was an increase of $0.5 million in other expenses due to an increase in state franchise taxes, as well as costs associated with operating as a public company. There was also an increase of $0.5 million in personnel-related expenses due to annual merit-related salary increases, partially offset by decreases in stock compensation expense primarily due to higher stock-based compensation for the nine months ended September 30, 2024 associated with the forgiveness of promissory notes originally issued to certain executive officers. Finally, there was an increase of $0.9 million in professional and consulting-related expenses due to costs associated with operating as a public company.
Previous report
Impairment of Right-of-Use Asset
Previous report
During the nine months ended September 30, 2025, upon vacating our Boulder, Colorado location and pursuing a sublessee, it was determined that the market rate for similar space is less than the base rent the Company is paying under its current lease. As a result, the Company impaired the related ROU asset and recognized an impairment charge of $0.5 million.
Previous report
Other (Expense) Income, Net
Previous report
Other (expense) income, net was an expense of $0.3 million for the nine months ended September 30, 2025 compared to income of $0.5 million for the nine months ended September 30, 2024. The change of $0.9 million was primarily due to a $1.8 million expense for the change in fair value of our derivative tranche liability associated with the second tranche of our private placement. This was partially offset by a $0.7 million increase in interest income due to higher average invested cash equivalent balances during the nine months ended September 30, 2025 and a decrease in other expenses of $0.2 million primarily due to lower foreign exchange losses.
Previous report
For the Nine Months Ended September 30, 2025 and 2024
Previous report
Cash flows used in operations are primarily attributed to clinical and preclinical expenses, employee compensation, rent and variable rent expenses for our Cambridge and Boulder leases as well as professional fees. Our cash flows from operating activities are significantly affected by our use of cash for operating expenses and working capital to support the business.
Previous report
Net cash used in operating activities for the nine months ended September 30, 2025 was $35.8 million and consisted of net loss of $40.1 million, non-cash adjustments of $7.9 million and a net change in assets and liabilities of $3.7 million. Non-cash items primarily included stock-based compensation of $2.8 million, loss from the change in fair value of our tranche liability of $1.8 million, non-cash operating lease expense of $1.4 million, depreciation and amortization of $1.3 million, and impairment of the Boulder lease ROU asset of $0.5 million. The net change in assets and liabilities was primarily due to $2.6 million in lease payments, a decrease in accounts payable of $0.6 million due to the timing of payments, a decrease in accrued expenses of $0.4 million due to the payout of annual employee bonuses and cash paid to certain executive officers to offset the tax impact associated with the forgiveness of promissory notes from 2024, partially offset by the current year accrual for annual employee bonuses and higher accrued R&D expenses.
Previous report
Net cash used in operating activities for the nine months ended September 30, 2025, was $1.6 million higher than the same period in 2024. The increase in cash used for operations was primarily due to the $2.0 million higher net change in assets and liabilities, partially offset by higher non-cash adjustments of $2.1 million and a lower net loss of $1.6 million. The higher net change in assets and liabilities was primarily due to the $1.6 million change in accounts payable due to the timing of payments and the increased costs of becoming a public company. The higher non-cash adjustment was primarily due to loss on the change in fair value of our tranche liability of $1.8 million and the impairment of the Boulder lease ROU asset of $0.5 million.
Previous report
Our primary investing activities consist of capital expenditures for laboratory and computer equipment as well as software. Net cash used in investing activities for the nine months ended September 30, 2025 was $0.2 million, due to purchases of laboratory equipment, partially offset by the sale of equipment used in our Boulder location. Net cash used in investing activities increased by $0.1 million during the nine months ended September 30, 2025, compared to the same period in 2024, primarily attributable to the timing of cash payments for laboratory equipment, partially offset by lower spend for the nine months ended September 30, 2025.
Previous report
Net cash provided by financing activities for the nine months ended September 30, 2025 was $47.3 million. Our primary financing activity for the nine months ended September 30, 2025 was our private placement, which resulted in cash net of issuance costs totaling $32.6 million for securities sold in the initial close and $14.9 million allocated to the derivative tranche liability. Excluding the cash provided by the private placement, net cash used in financing activities decreased $16.1 million during the nine months ended September 30, 2025, compared to the same period in 2024. The decrease was primarily attributable to cash used in 2024 of $0.8 million for deferred offering costs and a $0.4 million decrease in repayments of our finance leases and financing liability.
Previous report
Valuation of Derivative Tranche Liability
Previous report
In connection with our Private Placement in September 2025, we had a commitment and investors had an obligation to purchase securities in a second closing at a fixed price, if specified conditions are met. The obligation to issue additional securities at a future date was determined to be a freestanding derivative instrument and is accounted for as a liability. The derivative tranche liability was accounted for at fair value at the issuance date and remeasured at the end of each reporting period until the shares are issued or the obligation expires. Changes in the fair value of the derivative tranche liability are recognized in the condensed consolidated statement of operations.
Previous report
The fair value of the derivative tranche liability was determined using a probability weighted model, which considered as inputs the probability of achieving tranche closing conditions, the estimated fair value of our common stock and a discount rate. We recognized a $1.8 million loss for the nine months ended September 30, 2025, related to the change in fair value of the derivative tranche liability.
Reworded paragraphs (14)
Previous wording
We are leveraging our RAP Platform to advance a preclinical program for the treatment of synaptic Ras GTPase activating protein 1 (“SYNGAP1”)-related disorders, a group of neurodevelopmental conditions caused by pathogenic variants in the SYNGAP1 gene that result in a haploinsufficient state, reducing SYNGAP protein levels by up to 50%. SYNGAP plays a critical role in cognitive development and synaptic function. Epilepsy is a common characteristic of these disorders, and nearly all patients experience some degree of developmental delay and cognitive impairment. Incidence estimates vary significantly, ranging from one to 40 per 100,000 individuals. While we believe that SYNGAP1-related disorders remain underdiagnosed, we estimate that there are more than 10,000 individuals living with these disorders in the United States. SYNGAP1-related disorders are reported to represent 0.5% to 1.0% of all intellectual disability cases, making them among the most common causes of intellectual disability in patients with epilepsy, and indicating that the patient population may be significantly larger than incidence estimates suggest. There are no FDA-approved, disease-modifying therapies for SYNGAP1-related disorders. Treatment is often limited to supportive physical, occupational and speech therapy. A combination of non-specific anti-seizure medications may be prescribed, though SYNGAP1-related disorders have proven difficult to control with available therapeutics. Up to 50% of patients do not adequately respond to medication, in which case implantable devices, such as those for vagus nerve stimulation, may offer incremental therapeutic benefit. We are advancing our SYNGAP program to address the significant unmet need by targeting the direct cause of SYNGAP1-related disorders, haploinsufficiency, which we believe is amenable to targeting through regRNAs. Our novel approach targets the SYNGAP1 gene at the transcriptional level to restore SYNGAP function and improve symptoms by utilizing an intrathecally delivered ASO. Upregulation of SYNGAP1 gene expression may increase SYNGAP protein levels in amounts sufficient to yield therapeutic benefit. Our preclinical studies demonstrated a dose-dependent increase in SYNGAP mRNA levels accompanied by an increase in SYNGAP protein expression. On May 16, 2025, we presented preclinical data from our SYNGAP program in an oral presentation at the 28th American Society of Gene and Cell Therapy Annual Meeting. We observed that intracerebroventricular injection of our lead development candidate, CMP-002 (formerly known as CMP-SYNGAP-01), 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. Additionally, we observed that biweekly intrathecal injections of CMP-002 were well tolerated in cynomolgus monkeys and resulted in a significant increase in SYNGAP protein lev…
Excerpt shortened here; the source contains the full paragraph.
Current wording
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.
Previous wording
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. Regulatory RNAs (“regRNAs”) play a central role in the regulation of every protein-coding gene by contributing to gene activation and suppression. Our approach is designed to amplify messenger RNA (“mRNA”) expression by harnessing the power of regRNAs that form localized complexes with transcription factors and regulate gene expression. Our proprietary RNA Actuating Platform, or RAP Platform, allows us to rapidly and systematically identify and characterize the active regulatory elements controlling every expressed gene and tens of thousands of druggable enhancer and promoter regRNA sequences that control protein-coding genes. Once a disease-associated target gene is identified, we apply our RAP Platform to identify the controlling regRNA and rapidly generate novel antisense oligonucleotide (“ASO”) candidates, which we refer to as RNA Actuators. These ASOs are designed to bind to the identified regRNA and amplify the expression of the target gene in a specific and controllable way. We are primarily focused on diseases of the central nervous system with validated disease biology, and we believe our RAP Platform allows us to address a broad range of rare and prevalent genetic diseases in which a modest increase in protein expression has the potential to be clinically meaningful.
Current wording
Our product development efforts are enabled by our proprietary RAP Platform. We leverage our RAP Platform to identify and characterize regulatory RNAs (“regRNAs”), which play a central role in the regulation of every protein-coding gene by contributing to gene activation and suppression. Our approach is designed to amplify messenger RNA (“mRNA”) expression by harnessing the power of regRNAs that form localized complexes with transcription factors and regulate gene expression. Our RAP Platform allows us to rapidly and systematically identify and characterize the active regulatory elements controlling every expressed gene and tens of thousands of druggable enhancer and promoter regRNA sequences that control protein-coding genes. Once a disease-associated target gene is identified, we apply our RAP Platform to identify the controlling regRNA and rapidly generate novel ASO candidates. These ASOs are designed to bind to the identified regRNA and amplify the expression of the target gene in a specific and controllable way.
Previous wording
Since our inception in 2015, we have focused substantially all our resources primarily on developing our RAP Platform, identifying, developing and progressing our product candidates through preclinical and clinical development, organizing and staffing our company, research and development (“R&D”) activities, establishing and protecting our intellectual property portfolio, and raising capital. To date, we have primarily funded our operations with proceeds from the sale of convertible preferred stock and common stock, including our private placement, the initial closing of which occurred on September 11, 2025, and our initial public offering (“IPO”), which closed on October 15, 2024, as well as through revenues from our license and collaboration agreements. Through September 30, 2025, we have received net proceeds of $46.7 million from the initial closing of our private placement, $72.4 million from our IPO and $188.3 million from the sale of our convertible preferred stock. In addition, through September 30, 2025, we have recognized $21.2 million in research collaboration and license revenue through our development and license agreements. Our ability to generate any product revenue and, in particular, our ability to generate product revenue sufficient to achieve profitability, will depend on the successful development and eventual commercialization of product candidates.
Current wording
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.
Previous wording
We have incurred significant operating losses and negative cash flows from operations since our inception. Our net losses were $40.1 million and $38.5 million for the nine months ended September 30, 2025 and 2024, respectively. As of September 30, 2025, we had an accumulated deficit of $251.9 million. Substantially all of our net losses have resulted from costs incurred in connection with our R&D programs and, to a lesser extent, from general and administrative (“G&A”) costs associated with our operations. Our net losses may fluctuate significantly from quarter-to-quarter and year-to-year, depending on the timing of our clinical trials and preclinical studies, our other R&D activities and capital expenditures, and the timing and amount of any milestone or royalty payments due under our existing or future license or collaboration agreements. In addition, 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 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.
Current wording
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.
Previous wording
•advance current and future product candidates through preclinical and clinical studies;
Current wording
•advance current and future product candidates through preclinical studies and clinical trials;
Previous wording
•add operational, legal, compliance, financial and management information systems and personnel to support our research, product development and future commercialization efforts, as well as to support our operations as a public company.
Current wording
•add operational, legal, compliance, financial and management information systems and personnel to support our research, product development and future commercialization efforts.
Previous wording
As of September 30, 2025, we had cash and cash equivalents of $75.3 million. Based on our current operating plan, we estimate that our existing cash and cash equivalents will be sufficient to fund our projected operating expenses and capital expenditure requirements into 2027. See the sections titled “Liquidity and Capital Resources” included elsewhere in this Quarterly Report and “Risk Factors - Risks Related to our Financial Position and Need for Additional Capital” included elsewhere in this Quarterly Report and in our 2024 Form 10-K.
Current wording
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.
Previous wording
Other (expense) income, net for the three months ended September 30, 2025 was a $1.4 million expense compared to nominal income for the three months ended September 30, 2024. The change was primarily due to a $1.8 million expense for the change in fair value of our derivative tranche liability associated with the second tranche 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 September 30, 2025.
Current wording
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.
Previous wording
As of September 30, 2025, we had cash and cash equivalents of $75.3 million. Based on our current operating plan, we estimate that our cash and cash equivalents as of September 30, 2025 will be sufficient to fund our operating expenses and capital expenditure requirements into 2027. 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.
Current wording
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.
Previous wording
The timing and amount of our funding requirements will depend on many factors, including:
Current wording
The timing and amount of our future funding requirements will depend on many factors, including:
Previous wording
•the costs, timing and outcome of regulatory meetings and reviews of product candidates or any future product candidates, including requirements of regulatory authorities in any additional jurisdictions in which we may seek approval and any future product candidates;
Current wording
•timing and outcome of regulatory meetings and reviews of our product candidates or any future product candidates, including requirements of regulatory authorities in any additional jurisdictions in which we may seek approval and any future product candidates;
Previous wording
•the costs associated with hiring additional personnel and consultants as our clinical and preclinical activities increase and as we operate as a public company;
Current wording
•the costs associated with hiring additional personnel and consultants as our clinical and preclinical activities increase;
Previous wording
The following table provides information regarding our cash flows for the nine months ended September 30, 2025 and 2024 (in thousands):
Current wording
The following table provides information regarding our cash flows for the three months ended March 31, 2026 and 2025 (in thousands):
Previous wording
There have been no significant changes to our critical accounting estimates in the preparation of our condensed consolidated financial statements during the nine months ended September 30, 2025 compared to those disclosed in our 2024 Form 10-K except for the following.
Current wording
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.
Rearranged current paragraphs (2)
Current report — wording carried from elsewhere
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
Current report — wording carried from elsewhere
For the Three Months Ended March 31, 2026 and 2025
Rearranged previous paragraphs (3)
Previous report — wording carried elsewhere
The following table summarizes our results of operations for the nine months ended September 30, 2025 and 2024 (in thousands):
Previous report — wording carried elsewhere
The following table summarizes our R&D expenses for the nine months ended September 30, 2025 and 2024 (in thousands):
Previous report — wording carried elsewhere
The following table summarizes our G&A expenses for the nine months ended September 30, 2025 and 2024 (in thousands):