CAMP4 Reports Second Quarter 2025 Financial Results and Corporate Highlights

Rhea-AI Summary

CAMP4 Therapeutics (NASDAQ:CAMP) reported Q2 2025 financial results and corporate updates, highlighting progress in its RNA-targeting therapeutics pipeline. The company presented positive translational data for its SYNGAP1-related disorders program at the ASGCT Annual Meeting, demonstrating efficacy in mouse models and increased protein levels in non-human primates.

Key developments include plans to initiate GLP toxicology studies for CMP-SYNGAP-01 in Q3 2025, with potential Phase 1/2 trial initiation in H2 2026. The company completed dosing in the third MAD cohort for CMP-CPS-001, with comprehensive data expected in Q4 2025. Financial position shows $39.1 million in cash and equivalents, with Q2 R&D expenses of $10.3 million and a net loss of $12.6 million.

Positive

• Positive translational data from SYNGAP1 program showing efficacy in mouse models and increased protein levels in non-human primates
• Completed dosing in third MAD cohort for CMP-CPS-001 with data expected Q4 2025
• On track for GLP toxicology studies in Q3 2025 and potential Phase 1/2 trial initiation in H2 2026

Negative

• Cash position decreased from $49.3M to $39.1M quarter-over-quarter
• Increased R&D expenses to $10.3M from $9.4M year-over-year
• Higher G&A expenses at $4.2M compared to $3.3M year-over-year

Insights

Biotechnology Research Analyst

CAMP4 reports encouraging SYNGAP1 data while making progress in Phase 1 UCD trial, though cash position requires monitoring at $39.1M.

CAMP4 Therapeutics is making meaningful progress across its pipeline of regulatory RNA-targeting therapeutics aimed at upregulating gene expression. The company's lead programs focus on treating SYNGAP1-related disorders and urea cycle disorders (UCDs), both areas with significant unmet medical needs.

The translational data presented for their SYNGAP1 program at ASGCT demonstrates promising efficacy. In a humanized mouse model, CMP-SYNGAP-01 restored SYNGAP1 protein to near-normal levels and improved both motor and spatial learning deficits—critical behavioral outcomes that mirror patient symptoms. More impressively, studies in non-human primates showed significant increases in SYNGAP1 protein across multiple brain regions using the intended clinical route of administration (intrathecal delivery). These preclinical results strengthen the scientific rationale for advancing this candidate toward clinical studies.

For their UCD program, the company has completed dosing in the third multiple ascending dose cohort of CMP-CPS-001 in their Phase 1 trial. Data from both single and multiple ascending dose portions is expected in Q4 2025, which will provide crucial insights into safety profiles and potentially early biomarker signals.

On the financial front, CAMP4 reported $39.1 million in cash and equivalents as of June 30, representing a $10.2 million decrease from the previous quarter. R&D expenses increased to $10.3 million from $9.4 million year-over-year, while G&A expenses rose to $4.2 million from $3.3 million. The quarterly net loss remained stable at $12.6 million. At the current burn rate of approximately $10 million per quarter, their cash runway extends roughly 3-4 quarters unless additional financing is secured.

The company's timeline projections show careful planning with GLP toxicology studies for CMP-SYNGAP-01 starting in Q3 2025 and potential Phase 1/2 trial initiation in H2 2026. Management's statement about CMP-CPS-001 being "an ideal candidate for potential partnerships" suggests they may be seeking partnerships to extend their runway and advance clinical programs.

• Presented positive translational data from SYNGAP1-related disorders program showcasing efficacy in a humanized SYNGAP mouse model and increased protein in non-human primates at the 28^th American Society of Gene and Cell Therapy (ASGCT) Annual Meeting
  
  
• Initiating GLP toxicology studies evaluating CMP-SYNGAP-01 in Q3 2025
  
  
• Dosing completed in multiple ascending dose (MAD) cohort 3 of CMP-CPS-001 and data from single ascending dose (SAD) & MAD cohorts expected in Q4 2025
  

CAMBRIDGE, Mass., Aug. 14, 2025 (GLOBE NEWSWIRE) -- CAMP4 Therapeutics Corporation (“CAMP4”) (Nasdaq: CAMP), a clinical-stage biopharmaceutical company developing a pipeline of regulatory RNA-targeting therapeutics designed to upregulate gene expression with the goal of restoring healthy protein levels to treat a broad range of genetic diseases, today announced financial results for the second quarter ended June 30, 2025, and provided a corporate update.

“This past quarter, we presented compelling translational data from our SYNGAP1 program, reinforcing our confidence in CMP-SYGNAP-01's potential to transform the lives of patients living with this devastating neurological disorder, which currently has no approved treatments addressing the root cause,” said Josh Mandel-Brehm, President and Chief Executive Officer of CAMP4. “With strong translational results and a clear unmet need, we remain committed to advancing CMP-SYNGAP-01 with urgency and are on track to initiate GLP toxicology studies in the third quarter, which could support initiation of a global Phase 1/2 clinical trial in SYNGAP patients in the second half of 2026.”

Mr. Mandel-Brehm continued, “We continue to see strong potential in CMP-CPS-001 as a first-in-class, disease-modifying therapy for the most common UCDs and plan to announce safety and biomarker data from the SAD and MAD portions of the ongoing Phase 1 trial in healthy volunteers in Q4. These data could position CMP-CPS-001 as a valuable asset with an established safety profile that is ready for evaluation in symptomatic individuals, making it an ideal candidate for potential partnerships or further in-house development.”

Corporate Highlights:

• Presented new translational data from SYNGAP1-related disorders program and highlighted interim safety results from the ongoing Phase 1 UCD trial at the 28^th  American Society of Gene and Cell Therapy Annual Meeting.
  • In a humanized disease-relevant mouse model, a single intracerebroventricular dose of CMP-SYNGAP-01 restored SYNGAP1 protein to near-normal levels, and two doses rescued motor and spatial learning deficits, reflective of the disease state in patients.
  • In non-human primate studies using the clinical route of administration, intrathecal biweekly administration of CMP-SYNGAP-01 led to a significant increase in SYNGAP1 protein at levels believed to be therapeutically relevant across multiple disease-relevant brain regions.
  • Presented positive safety and pharmacokinetics data from the ongoing Phase 1 trial of CMP-CPS-001.
• Expect to initiate GLP toxicology studies evaluating CMP-SYNGAP-01 in Q3 2025; potential to initiate a global Phase 1/2 trial as early as H2 2026.
• Completed dosing in the third MAD cohort in the ongoing Phase 1 clinical trial of CMP-CPS-001; expect to report data from all four cohorts of the SAD portion and the first three cohorts of the MAD portion of the trial, including safety, pharmacokinetic and pharmacodynamic biomarker results, in Q4 2025.

Second Quarter 2025 Financial Results
Cash, cash equivalents, and marketable securities as of June 30, 2025, were $39.1 million, compared to $49.3 million as of March 31, 2025.

R&D Expenses: Research and development expenses for the quarter ended June 30, 2025, were $10.3 million, compared to $9.4 million for the quarter ended June 30, 2024. The expenses were primarily driven by an increase in clinical and preclinical study costs.

G&A Expenses: General and administrative expenses were $4.2 million for the quarter ended June 30, 2025, compared to $3.3 million for the quarter ended June 30, 2024. The expenses were primarily driven by an increase in personnel-related and overhead costs.

Net Loss: Net loss for the quarter ended June 30, 2025, was $12.6 million compared to $12.6 million for the quarter ended June 30, 2024.

About CAMP4 Therapeutics
CAMP4 is developing disease-modifying treatments for a broad range of genetic diseases where amplifying healthy protein may offer therapeutic benefits. Our approach amplifies mRNA by harnessing a fundamental mechanism of how genes are controlled. To amplify mRNA, our therapeutic ASO drug candidates target regulatory RNAs (regRNAs), which act locally on transcription factors and are the master regulators of gene expression. CAMP4’s proprietary RAP Platform™ enables the mapping of regRNAs and generation of therapeutic candidates designed to target the regRNAs associated with genes underlying haploinsufficient and recessive partial loss-of-function disorders, of which there are more than 1,200, in which a modest increase in protein expression may have the potential to be clinically meaningful. For more information, visit camp4tx.com.

Forward-Looking Statements
This press release contains forward-looking statements which involve risks, uncertainties and contingencies, many of which are beyond the control of the Company, which may cause actual results, performance, or achievements to differ materially from anticipated results, performance, or achievements. All statements other than statements of historical facts contained in this press release are 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,” “contemplate,” “believe,” “estimate,” “predict,” “potential” or “continue” or the negative of these terms or other similar expressions, although not all forward-looking statements contain these words. Forward-looking statements include, but are not limited to, statements concerning the Company’s clinical development plans and expectations regarding CMP-CPS-001; the anticipated timing and results of the Company’s ongoing and future clinical trials, including expectations regarding the timing to advance the Company’s SYNGAP1 program into a clinical trial and to report data from the CMP-CPS-001 clinical trial; the expected timing for the Company’s initiation of GLP toxicology studies relating to its SYNGAP1 program; and the therapeutic potential of the Company’s product candidates. The forward-looking statements in this press release speak only as of the date of this press release and are subject to a number of known and unknown risks, uncertainties and assumptions that could cause the Company’s actual results to differ materially from those anticipated in the forward-looking statements, including, but not limited to: the Company’s limited operating history, incurrence of substantial losses since the Company’s inception and anticipation of incurring substantial and increasing losses for the foreseeable future; the Company’s need for substantial additional financing to achieve the Company’s goals; the uncertainty of clinical development, which is lengthy and expensive, and characterized by uncertain outcomes, and risks related to additional costs or delays in completing, or failing to complete, the development and commercialization of the Company’s current product candidates or any future product candidates; delays or difficulties in the enrollment and dosing of patients in clinical trials; the impact of any significant adverse events or undesirable side effects caused by the Company’s product candidates; potential competition, including from large and specialty pharmaceutical and biotechnology companies; the Company’s ability to realize the benefits of the Company’s current or future collaborations or licensing arrangements and ability to successfully consummate future partnerships; the Company’s ability to obtain regulatory approval to commercialize any product candidate in the United States or any other jurisdiction, and the risk that any such approval may be for a more narrow indication than the Company seeks; the Company’s dependence on the services of the Company’s senior management and other clinical and scientific personnel, and the Company’s ability to retain these individuals or recruit additional management or clinical and scientific personnel; the Company’s ability to grow the Company’s organization, and manage the Company’s growth and expansion of the Company’s operations; risks related to the manufacturing of the Company’s product candidates, which is complex, and the risk that the Company’s third-party manufacturers may encounter difficulties in production; the Company’s ability to obtain and maintain sufficient intellectual property protection for the Company’s product candidates or any future product candidates the Company may develop; the Company’s reliance on third parties to conduct the Company’s preclinical studies and clinical trials; the Company’s compliance with the Company’s obligations under the licenses granted to the Company by others, for the rights to develop and commercialize the Company’s product candidates; risks related to the operations of the Company’s suppliers; and other risks and uncertainties described in the section “Risk Factors” in the Company’s Annual Report on Form 10-K for the year ended December 31, 2024 and Quarterly Report on Form 10-Q for the quarter ended June 30, 2025, as well as other information the Company files with the Securities and Exchange Commission. The forward-looking statements in this press release are inherently uncertain and are not guarantees of future events. Because forward-looking statements are inherently subject to risks and uncertainties, some of which cannot be predicted or quantified and some of which are beyond the Company’s control, you should not unduly rely on these forward-looking statements. The events and circumstances reflected in the forward-looking statements may not be achieved or occur and actual future results, levels of activity, performance and events and circumstances could differ materially from those projected in the forward-looking statements. Moreover, the Company operates in an evolving environment. New risks and uncertainties may emerge from time to time, and management cannot predict all risks and uncertainties. Investors, potential investors, and others should give careful consideration to these risks and uncertainties. Except as required by applicable law, the Company does not undertake to publicly update or revise any forward-looking statements contained herein, whether as a result of any new information, future events, changed circumstances or otherwise.

Contacts

Investor Relations:
Dan Ferry (US)
LifeSci Advisors
daniel@lifesciadvisors.com

Media:
Jason Braco, Ph.D.
LifeSci Communications
jbraco@lifescicomms.com

CAMP4 Therapeutics Corporation
Unaudited Consolidated Statements of Operations and Comprehensive Loss
(In thousands, except for share and per share data)
​		Three months ended June 30,								Six months ended June 30,
​		2025				2024				2025				2024
Revenue		​				​				​				​
Research and collaboration revenue		$	1,497			$	-			$	2,355			$	-
Operating expenses
Research and development			10,343				9,389				20,489				19,129
General and administrative			4,182				3,273				7,994				6,408
Total operating expenses			14,525				12,662				28,483				25,537
Loss from operations			(13,028	)			(12,662	)			(26,128	)			(25,537	)
Other income, net:
Interest income			453				231				1,041				626
Other income (expense)			(12	)			(145	)			67				(117	)
Total other income, net			441				86				1,108				509
Net loss attributable to common stockholders and comprehensive loss			(12,587	)			(12,576	)			(25,020	)			(25,028	)
Net loss per share attributable to common stockholders, basic and diluted			(0.62	)			(26.00	)			(1.24	)			(52.56	)
Weighted average shares of common stock outstanding, basic and diluted			20,159,666				483,640				20,155,161				476,167

Unaudited Condensed Balance Sheet Data:		June 30,				December 31,
(in thousands)		2025				2024
Cash and cash equivalents		$	39,052			$	64,039
Working capital(1)			33,751				56,785
Total assets			51,275				78,307
Total liabilities			11,288				15,163
Accumulated deficit			(236,773	)			(211,753	)
Total stockholders' equity			39,987				63,144

^{(1) Working capital is defined as total current assets less total current liabilities. See our unaudited condensed consolidated financial statements and the related notes thereto included in our Quarterly Report on Form 10-Q for the six months ended June 30, 2025 for further details regarding our current assets and current liabilities.}

FAQ

What were CAMP4's key financial results for Q2 2025?

CAMP4 reported cash and equivalents of $39.1 million, R&D expenses of $10.3 million, G&A expenses of $4.2 million, and a net loss of $12.6 million for Q2 2025.

What progress did CAMP4 make in its SYNGAP1 program?

CAMP4 presented positive translational data showing restored SYNGAP1 protein levels in mouse models and increased protein levels in non-human primates, with plans to initiate GLP toxicology studies in Q3 2025.

When will CAMP4 report data from its CMP-CPS-001 clinical trial?

CAMP4 expects to report data from all SAD cohorts and first three MAD cohorts, including safety and biomarker results, in Q4 2025.

What is the timeline for CAMP4's SYNGAP1 program clinical trials?

CAMP4 plans to initiate GLP toxicology studies in Q3 2025 and potentially begin a global Phase 1/2 clinical trial in H2 2026.

How much did CAMP4's operating expenses increase in Q2 2025?

R&D expenses increased to $10.3M from $9.4M year-over-year, while G&A expenses rose to $4.2M from $3.3M.