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Camp4 Therapeutics Corp

For the period ended Jun 30, 2025. Filed Aug 14, 2025.

View on SEC.gov

About 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.

Jun 30, 2025 · Filed Aug 14, 2025

CAMP$4.06+9.1%Stored close · as of Nov 6, 2026

What changed in Management’s Discussion and Analysis?

Read the current filing

Previous report: 10-Q

Period ended 2025-03-31; filed 2025-05-13.

0001736730-25-000039

Item 2. Management’s Discussion and Analysis of Financial Condition and Results of Operations

Current report: 10-Q

Period ended 2025-06-30; filed 2025-08-14.

0001736730-25-000070

Item 2. Management’s Discussion and Analysis of Financial Condition and Results of Operations

The 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 66 previous paragraphs and 91 current paragraphs.

43 unchanged; 19 reworded; 21 added; 2 removed; 2 previous and 8 current paragraphs rearranged.

Tables are not compared: 4 previous and 7 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 (21)

Current report

•advance current and future product candidates through preclinical and clinical studies;

Current report

G&A expenses were $8.0 million for the six months ended June 30, 2025 compared to $6.4 million for the six months ended June 30, 2024. The increase of $1.6 million was partially due to an increase of $0.9 million in personnel-related expenses due to annual merit-related salary increases, stock compensation expense, and severance costs related to separation agreements with certain former employees. 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. Lastly, there was an increase of $0.3 million in facility-related and overhead expense due to increases in insurance premiums. These increases were offset by a decrease in professional and consulting-related expenses of $0.1 million.

Current report

•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;

Current report

•the costs and timing of manufacturing for our product candidates, including commercial manufacturing at sufficient scale, if any product candidate is approved;

Current report

•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 report

•the costs of obtaining, maintaining, enforcing and protecting our patents and other intellectual property and proprietary rights;

Current report

•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;

Current report

•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 report

•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;

Current report

•the costs and timing of establishing or securing sales and marketing capabilities if our product candidates or any future product candidate is approved;

Current report

•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;

Current report

•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;

Current report

•the terms and timing of establishing and maintaining collaborations, licenses and other similar arrangements; and

Current report

•costs associated with any products or technologies that we may in-license or acquire.

Current report

Net cash used in operating activities for the six months ended June 30, 2025, was comparable to the same period in 2024. Net loss was $25.0 million for each of the six months ended June 30, 2025 and 2024. Non-cash adjustments were
Removed paragraphs (2)

Previous report

•advance our lead product candidate, CMP-CPS-001, through clinical trials;

Previous report

•conduct preclinical studies and clinical trials for our GBA1 discovery program for the treatment of PD and any future product candidates we may develop;
Reworded paragraphs (19)

Previous wording

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

Current wording

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.

Previous wording

We are also 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 approximately 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 CMP-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 CMP-SYNGAP program is a novel approach that 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 SYNGAP1 mRNA levels accompanied by a reduction in SYNGAP1 expression. We have nominated a development candidate, CMP-SYNGAP-01, and expect to initiate GLP toxicology studies in 2025 to enable the filing of a clinical trial application. We also plan to present new preclinical data in an oral presentation at the 28th American Society of Gene and Cell Therapy Annual Meeting on May 16, 2025, demonstrating a meaningful increase in SYNGAP1 protein levels following intrathecal administration of CMP-SYNGAP-01 in non-human primates.

Current 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 approximately 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 CMP-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 CMP-SYNGAP program is a novel approach that 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-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-SYNGAP-01 were well tolerated in cynomolgus monkeys and resulted in a significant increase in SYNG…

Excerpt shortened here; the source contains the full paragraph.

Previous wording

Based on our preclinical studies, we believe our clinical product candidate, CMP-CPS-001, has the potential to be the first disease-modifying therapy for the treatment of the most prevalent urea cycle disorders (“UCDs”). 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-CPS-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-CPS-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 are evaluating CMP-CPS-001 in an ongoing Phase 1 clinical trial in healthy volunteers and have completed a planned interim analysis of safety and pharmacokinetic data from all four cohorts of the single ascending dose (“SAD”) portion of the clinical trial, from which no safety trends of concern were observed. We have also completed dosing of the first three cohorts from the multiple ascending dose (“MAD”) portion of the clinical trial. We expect to report data from the SAD and MAD portions of the clinical trial, including safety, pharmacokinetic and key pharmacodynamic biomarker data, in the fourth quarter of 2025. We have also submitted a clinical trial application in Europe for a Phase 1b expansion to enroll female participants who are heterozygous for a mutation of the OTC gene, pending regulatory clearance. 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.

Current wording

We are also evaluating our clinical candidate, CMP-CPS-001, for the treatment of the most prevalent urea cycle disorders (“UCDs”), in 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-CPS-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-CPS-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 completed a planned interim analysis of safety and pharmacokinetic data from all four cohorts of the single ascending dose (“SAD”) portion of the clinical trial, and we have completed dosing of the first three cohorts from the multiple ascending dose (“MAD”) portion of the clinical trial, with no safety trends of concern observed to date. We have also submitted a clinical trial application in Europe for 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. We expect to report data from all four cohorts of the SAD portion and the first three cohorts of the MAD portion of the clinical trial, including safety, pharmacokinetic and pharmacodynamic biomarker data, in the fourth quarter of 2025 and to evaluate potential next steps for the further development of CMP-CPS-001 based on these data, which may include exploring potential partnership opportunities.

Previous wording

In addition, we have initiated a discovery program targeting a regRNA to increase GBA1 gene expression for the treatment of Parkinson’s disease (“PD”) caused by mutations in the GBA1 gene, and plan to initiate discovery programs in other CNS and metabolic indications utilizing our RAP Platform. Finally, we have entered into 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.

Current wording

In addition, we have an ongoing discovery program targeting a regRNA to increase GBA1 gene expression for the treatment of Parkinson’s disease (“PD”) caused by mutations in the GBA1 gene, and have initiated discovery programs in other CNS indications utilizing our RAP Platform. Finally, we have entered into 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 wording

Since our inception in 2015, we have focused substantially all of our resources primarily on developing our RAP Platform, identifying, developing and progressing our product candidates through preclinical and clinical development, organizing and staffing our company, research and development (“R&D”) activities, establishing and protecting our intellectual property portfolio, and raising capital. To date, we have primarily funded our operations with proceeds from the sale of convertible preferred stock and common stock, including in our initial public offering (“IPO”), which closed on October 15, 2024, as well as through revenues from our license and collaboration agreements. Through March 31, 2025, we have received net proceeds of $72.4 million from our IPO and $188.3 million from the sale of our convertible preferred stock. In addition, through March 31, 2025, we have recognized $18.9 million in research collaboration and license revenue through our development and license agreements. Our ability to generate any product revenue and, in particular, our ability to generate product revenue sufficient to achieve profitability, will depend on the successful development and eventual commercialization of product candidates.

Current wording

Since our inception in 2015, we have focused substantially all of our resources primarily on developing our RAP Platform, identifying, developing and progressing our product candidates through preclinical and clinical development, organizing and staffing our company, research and development (“R&D”) activities, establishing and protecting our intellectual property portfolio, and raising capital. To date, we have primarily funded our operations with proceeds from the sale of convertible preferred stock and common stock, including in our initial public offering (“IPO”), which closed on October 15, 2024, as well as through revenues from our license and collaboration agreements. Through June 30, 2025, we have received net proceeds of $72.4 million from our IPO and $188.3 million from the sale of our convertible preferred stock. In addition, through June 30, 2025, we have recognized $20.4 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 $12.4 million and $12.5 million for the three months ended March 31, 2025 and 2024, respectively. As of March 31, 2025, we had an accumulated deficit of $224.2 million. Substantially all of our net losses have resulted from costs incurred in connection with our R&D programs and, to a lesser extent, from general and administrative (“G&A”) costs associated with our operations. Our net losses may fluctuate significantly from quarter-to-quarter and year-to-year, depending on the timing of our clinical trials and preclinical studies, our other R&D activities and capital expenditures, and the timing and amount of any milestone or royalty payments due under our existing or future license or collaboration agreements. In addition, we expect to incur additional costs associated with operating as a public company, including significant legal, audit, accounting, regulatory and tax-related services associated with maintaining compliance with exchange listing and requirements of the SEC, director and officer liability insurance costs, investor and public relations costs, and other expenses that we did not incur as a private company. If we obtain regulatory approval for our product candidates, we expect to incur significant expenses related to developing our commercialization capability to support product sales, marketing and distribution. We anticipate that our expenses will increase substantially if and as we:

Current wording

We have incurred significant operating losses and negative cash flows from operations since our inception. Our net losses were $25.0 million for both the six months ended June 30, 2025 and 2024. As of June 30, 2025, we had an accumulated deficit of $236.8 million. Substantially all of our net losses have resulted from costs incurred in connection with our R&D programs and, to a lesser extent, from general and administrative (“G&A”) costs associated with our operations. Our net losses may fluctuate significantly from quarter-to-quarter and year-to-year, depending on the timing of our clinical trials and preclinical studies, our other R&D activities and capital expenditures, and the timing and amount of any milestone or royalty payments due under our existing or future license or collaboration agreements. In addition, 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. We anticipate that our expenses will increase substantially if and as we:

Previous wording

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

Current wording

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

Previous wording

As of March 31, 2025, we had cash and cash equivalents of $49.3 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 the second quarter of 2026, which raises substantial doubt about our ability to continue as a going concern. 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 in our 2024 Form 10-K.

Current wording

As of June 30, 2025, we had cash and cash equivalents of $39.1 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 the second quarter of 2026, which raises substantial doubt about our ability to continue as a going concern. 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.

Previous wording

We recognized $0.9 million in research and collaboration revenue during the three months ended March 31, 2025. We did not recognize any research and collaboration revenue during the three months ended March 31, 2024. The increase of $0.9 million was due to revenue recognized under our Collaboration and License Agreement with BioMarin.

Current wording

We recognized $1.5 million in research and collaboration revenue during the three months ended June 30, 2025. We did not recognize any research and collaboration revenue during the three months ended June 30, 2024. The increase of $1.5 million was due to revenue recognized under our Collaboration and License Agreement with BioMarin of $0.9 million and a $0.6 million milestone payment earned under the Fulcrum Agreement.

Previous wording

G&A expenses were $3.8 million for the three months ended March 31, 2025 compared to $3.1 million for the three months ended March 31, 2024. The increase of $0.7 million was partially due to an increase of $0.4 million in personnel-related expenses due to annual merit-related salary increases and stock compensation expense and an increase of $0.1 million in facility-related and overhead expense due to increases in insurance premiums. In addition, there was an increase of $0.2 million in other expenses due to an increase in state franchise taxes.

Current wording

G&A expenses were $4.2 million for the three months ended June 30, 2025 compared to $3.3 million for the three months ended June 30, 2024. The increase of $0.9 million was partially due to an increase of $0.5 million in personnel-related expenses due to annual merit-related salary increases and stock compensation expense and an increase of $0.2 million in facility-related and overhead expense due to increases in insurance premiums. In addition, there was an increase of $0.2 million in other expenses due to an increase in state franchise taxes.

Previous wording

Other income, net was $0.7 million for the three months ended March 31, 2025 compared to $0.4 million for the three months ended March 31, 2024. The increase of $0.3 million was primarily due to an increase in interest income due to higher average invested cash equivalent balances during the three months ended March 31, 2025.

Current wording

Other income, net was $0.4 million for the three months ended June 30, 2025 compared to $0.1 million for the three months ended June 30, 2024. The increase of $0.3 million was primarily due to an increase in interest income due to higher average invested cash balances during the three months ended June 30, 2025.

Previous wording

R&D expenses were $10.1 million for the three months ended March 31, 2025 compared to $9.7 million for the three months ended March 31, 2024. The increase of $0.4 million was primarily due to an increase of $0.1 million in clinical and preclinical expenses due to increases associated with the Company’s UCD clinical trial. In addition, there was an increase of $0.1 million in personnel-related expenses due to lower offsetting personnel reimbursements under one of our collaboration agreements. There was also an increase of $0.1 million in professional and consulting fees due to increased utilization of external support for clinical operations.

Current wording

R&D expenses were $20.5 million for the six months ended June 30, 2025 compared to $19.1 million for the six months ended June 30, 2024. The increase of $1.4 million was primarily due to an increase of $0.5 million in clinical and preclinical expenses related to the Company’s UCD clinical trial and SYNGAP preclinical work. There was also an increase of $0.5 million in professional and consulting fees due to increased utilization of external support for clinical operations. In addition, there was an increase of $0.3 million in personnel-related expenses due to lower offsetting personnel reimbursements under one of our collaboration agreements and due to annual merit-related salary increases and stock compensation expense.

Previous wording

As of March 31, 2025, we had cash and cash equivalents of $49.3 million. Based on our current operating plan, we estimate that our cash and cash equivalents as of March 31, 2025 will be sufficient to fund our operating expenses and capital expenditure requirements into the second quarter of 2026. However, we have based this estimate on assumptions that may prove to be wrong, and we could deplete our capital resources sooner than we currently expect. Our capital resources may not be sufficient to fund operations through at least the next twelve months from the date the accompanying unaudited condensed consolidated financial statements as of March 31, 2025 are issued based on our expected cash needs, which raises substantial doubt about our ability to continue as a going concern. Our future viability is dependent on our ability to generate cash from our operating activities or to raise additional capital to finance our operations. There is no assurance that we will succeed in obtaining sufficient funding on terms acceptable to us to fund continuing operations, if at all.

Current wording

As of June 30, 2025, we had cash and cash equivalents of $39.1 million. Based on our current operating plan, we estimate that our cash and cash equivalents as of June 30, 2025 will be sufficient to fund our operating expenses and capital expenditure requirements into the second quarter of 2026. However, we have based this estimate on assumptions that may prove to be wrong, and we could deplete our capital resources sooner than we currently expect. Our capital resources will not be sufficient to fund operations through at least the next twelve months from the date the accompanying unaudited condensed consolidated financial statements as of June 30, 2025 are issued based on our expected cash needs, which raises substantial doubt about our ability to continue as a going concern. Our future viability is dependent on our ability to generate cash from our operating activities or to raise additional capital to finance our operations. There is no assurance that we will succeed in obtaining sufficient funding on terms acceptable to us to fund continuing operations, if at all.

Previous wording

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 funding requirements will depend on many factors for which there have been no material changes from those described under “Management’s Discussion and Analysis of Financial Condition and Results of Operations” included in our 2024 Form 10-K.

Current wording

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.

Previous wording

The following table provides information regarding our cash flows for the three months ended March 31, 2025 and 2024 (in thousands):

Current wording

The following table provides information regarding our cash flows for the six months ended June 30, 2025 and 2024 (in thousands):

Previous wording

Net cash used in operating activities for the three months ended March 31, 2025 was $14.3 million and consisted of net loss of $12.4 million, non-cash adjustments of $1.9 million and a net change in assets and liabilities of $3.7 million. Non-cash items primarily included stock-based compensation of $0.9 million, non-cash operating lease expense of $0.5 million, and depreciation and amortization of $0.4 million. The net change in assets and liabilities was primarily due to a decrease in accrued expenses due to the timing of payout related to annual employee bonuses and cash paid to certain executive officers to offset the tax impact associated with the forgiveness of promissory notes from 2024.

Current wording

Net cash used in operating activities for the six months ended June 30, 2025 was $24.6 million and consisted of net loss of $25.0 million, non-cash adjustments of $3.7 million and a net change in assets and liabilities of $3.3 million. Non-cash items primarily included stock-based compensation of $1.9 million, non-cash operating lease expense of $1.0 million, and depreciation and amortization of $0.9 million. The net change in assets and liabilities was primarily due to a decrease in accrued expenses due to the timing of payout related to annual employee bonuses and cash paid to certain executive officers to offset the tax impact associated with the forgiveness of promissory notes from 2024.

Previous wording

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 three months ended March 31, 2025 was $0.3 million, due to purchases of laboratory equipment. Net cash used in investing activities increased by $0.3 million during the three months ended March 31, 2025, compared to the same period in 2024, primarily attributable to the timing of cash payments for laboratory equipment.

Current wording

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 six months ended June 30, 2025 was $0.3 million, due to purchases of laboratory equipment. Net cash used in investing activities increased by $0.1 million during the six months ended June 30, 2025, compared to the same period in 2024, primarily attributable to the timing of cash payments for laboratory equipment.

Previous wording

Our primary financing activities consist of principal payments on finance leases. Net cash used in financing activities for the three months ended March 31, 2025 was $0.2 primarily due to principal payments on finance leases. Net cash used in financing activities decreased by $0.1 million during the three months ended March 31, 2025, compared to the same period in 2024, primarily attributable to our completion and conversion of certain finance leases.

Current wording

Our primary financing activities for the six months ended June 30, 2025 consist of principal payments on finance leases. Net cash used in financing activities for the six months ended June 30, 2025 was $0.1 million. Net cash used in financing activities decreased by $0.9 million during the six months ended June 30, 2025, compared to the same period in 2024, primarily attributable cash used in 2024 of $0.6 million for deferred offering costs and $0.2 million in repayments for our financing liability. In addition, there was a $0.1 million decrease in principal payments on finance leases.

Previous 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, 2025 compared to those disclosed in our 2024 Form 10-K.

Current wording

There have been no significant changes to our critical accounting estimates in the preparation of our condensed consolidated financial statements during the six months ended June 30, 2025 compared to those disclosed in our 2024 Form 10-K.
Rearranged current paragraphs (8)

Current report — wording carried from elsewhere

R&D expenses were $10.3 million for the three months ended June 30, 2025 compared to $9.4 million for the three months ended June 30, 2024. The increase of $1.0 million was primarily due to an increase of $0.4 million in clinical and preclinical expenses due to increases associated with the Company’s UCD clinical trial and SYNGAP preclinical work. There was also an increase of $0.4 million in professional and consulting fees due to increased utilization of external support for clinical operations. In addition, there was an increase of $0.2 million in personnel-related expenses due to severance costs related to separation agreements with certain former employees.

Current report — wording carried from elsewhere

The following table summarizes our results of operations for the six months ended June 30, 2025 and 2024 (in thousands):

Current report — wording carried from elsewhere

We recognized $2.4 million in research and collaboration revenue during the six months ended June 30, 2025. We did not recognize any research and collaboration revenue during the six months ended June 30, 2024. The increase of $2.4 million was due to revenue recognized under our Collaboration and License Agreement with BioMarin of $1.8 million and a $0.6 million milestone payment earned under the Fulcrum Agreement.

Current report — wording carried from elsewhere

The following table summarizes our R&D expenses for the six months ended June 30, 2025 and 2024 (in thousands):

Current report — wording carried from elsewhere

The following table summarizes our G&A expenses for the six months ended June 30, 2025 and 2024 (in thousands):

Current report — wording carried from elsewhere

Other income, net was $1.1 million for the six months ended June 30, 2025 compared to $0.5 million for the six months ended June 30, 2024. The increase of $0.5 million was primarily due to an increase in interest income due to higher average invested cash equivalent balances during the six months ended June 30, 2025.

Current report — wording carried from elsewhere

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

Current report — wording carried from elsewhere

$0.3 million higher due to higher stock-based compensation and net change in assets and liabilities was $0.4 million lower due to cash paid in 2024 to certain executive officers to offset the tax impact associated with the forgiveness of promissory notes.
Rearranged previous paragraphs (2)

Previous report — wording carried elsewhere

For the Three Months Ended March 31, 2025 and 2024

Previous report — wording carried elsewhere

Net cash used in operating activities increased by $3.1 million during the three months ended March 31, 2025, compared to the same period in 2024, primarily attributable to the timing of payout related to annual employee bonuses and cash paid to certain executive officers to offset the tax impact associated with the forgiveness of promissory notes from 2024.