CAMP4 Reports Full Year 2025 Financial Results and Corporate Highlights

GLP toxicology studies ongoing for CMP-002, with initiation of global Phase 1/2 clinical trial in SYNGAP1 patients expected as early as 2H 2026

Entered strategic collaboration with GSK to advance RNA-based therapeutic discoveries, received $17.5 million upfront and eligible for milestone-based payments in addition to tiered royalties

Completed private placement with upfront gross proceeds of $50 million, with potential for up to an additional $50 million of gross proceeds, as well as underwritten offering of common stock of $30 million in gross proceeds, extending cash runway into 2028

CAMBRIDGE, Mass., March 05, 2026 (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 full year ended December 31, 2025, and provided recent corporate highlights.

"In 2025, we brought our SYNGAP1 program to the forefront of our pipeline and made significant progress against our goal of bringing a potential first-in-class treatment for SYNGAP1-related disorder into the clinic,” said Josh Mandel-Brehm, President and Chief Executive Officer of CAMP4. “Our GLP toxicology studies for CMP-002 are ongoing, and we continue to expect to initiate a global first-in-human Phase 1/2 clinical trial as early as the second half of 2026. We also made progress in our mission of developing potentially disease modifying medicines for patients with disorders marked by suboptimal gene expression by exploring new candidates for both in-house development and potential partnerships and signed a collaboration agreement with GSK to identify and develop ASO drug candidates for multiple gene targets relevant to neurodegenerative and kidney disease indications. Finally, we strengthened our balance sheet through a combination of equity financing and non-dilutive capital from collaboration partners to ensure that CAMP4 is well-capitalized to achieve its goals.”

Corporate Highlights:

• Presented compelling preclinical data for SYNGAP1 program showing that haploinsufficient SYNGAP1 mice treated with CMP-002 demonstrated an increase in SYNGAP1 protein levels and that treatment rescued multiple SYNGAP1-dependent behavioral phenotypes. This data further demonstrated that CMP-002 administration led to a significant increase in SYNGAP1 protein levels in relevant brain regions in non-human primates (NHPs).
• Initiated GLP toxicology studies for CMP-002 in support of future clinical trial applications, which could enable initiation of a global Phase 1/2 clinical trial in patients as early as H2 2026.
• Entered a strategic research, collaboration, and license agreement with GSK to identify and develop antisense nucleotide (ASO) drug candidates for multiple gene targets relevant to neurodegenerative and kidney disease indications. CAMP4 received a $17.5 million cash upfront payment and has the potential to receive up to $440 million upon achievement of certain development and commercial milestones, in addition to tiered royalties on future product sales.
• Completed oversubscribed private placement for gross proceeds of up to $100 million, $50 million of which was received in the form of an upfront payment in September, as well as underwritten offering of common stock with gross proceeds of $30 million in December to fund further development of CMP-002.
• Conducted analysis from single ascending dose (SAD) and multiple ascending dose (MAD) portions of the CMP-001 Phase 1 clinical trial and made strategic decision to pause further investment in the development of CMP-001 and explore potential partnership opportunities.
  

Full Year 2025 Financial Results

Cash and cash equivalents as of December 31, 2025, were $109.5 million, compared to $64.0 million as of December 31, 2024. The Company believes that its current cash and cash equivalents will be sufficient to fund its planned activities into 2028.

R&D Expenses: Research and development (“R&D”) expenses for the year ended December 31, 2025, were $38.2 million, compared to $38.8 million for the year ended December 31, 2024. The decrease was primarily driven by a gain on lease modification and decreased clinical costs associated with CMP-001.

G&A Expenses: General and administrative (“G&A”) expenses were $17.4 million for the year ended December 31, 2025, compared to $14.9 million for the year ended December 31, 2024. The change was primarily driven by an increase in professional expenses.

Net Loss: Net loss for the year ended December 31, 2025, was $80.4 million compared to $51.8 million for the year ended December 31, 2024. The increase in net loss was primarily driven by a $29.8 million non-cash loss recognized due to a change in fair value of the derivative tranche liability related to our September private placement.

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.

CAMP4 Therapeutics Corporation Consolidated Statements of Operations and Comprehensive Loss (In thousands, except for share and per share data)
​	Year ended December 31,
​		2025				2024
Revenue	​				​
Research and collaboration revenue	$	3,498			$	652
Operating expenses	​				​
Research and development		38,202				38,817
General and administrative		17,357				14,923
Impairment of right-of-use asset		494				—
Total operating expenses		56,053				53,740
Loss from operations		(52,555	)			(53,088	)
Other (expense) income, net:	​				​
Interest income		2,174				1,330
Change in fair value of derivative tranche liability		(29,830	)			—
Other expense		(192	)			(33	)
Total other (expense) income, net		(27,848	)			1,297
Net loss	$	(80,403	)		$	(51,791	)
Net loss per share attributable to common stockholders, basic and diluted	$	(2.65	)		$	(11.04	)
Weighted average shares of common stock outstanding, basic and diluted		30,380,567				4,690,094

​	December 31,
​Condensed Balance Sheet Data		2025				2024
(in thousands)	​				​
Cash and cash equivalents	$	109,517			$	64,039
Working capital(1)	​98,581					56,785
Total assets		117,808				78,307
Total liabilities		70,104				15,163
Accumulated deficit		(292,156	)			(211,753	)
Total stockholders' equity		47,704				63,144

(1) Working capital is defined as total current assets less total current liabilities. See our consolidated financial statements and the related notes thereto included in our Annual Report on Form 10-K for the year ended December 31, 2025, for further details regarding our current assets and current liabilities.