CAMBRIDGE, Mass., Oct. 01, 2025 (GLOBE NEWSWIRE) -- CAMP4 Therapeutics Corporation (“CAMP4” or “the Company”) (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 the initiation of toxicology studies conducted under Good Laboratory Practice (GLP) standards for its lead product candidate, CMP-S ...

Financing Overview - CAMP4 Therapeutics has secured a private placement financing of up to $100 million, with an initial $50 million in upfront proceeds to fund the SYNGAP1 program and general corporate purposes [1][2] - The financing includes the issuance of 26,681,053 shares of common stock at $1.53 per share, along with additional shares and pre-funded warrants contingent on achieving specific milestones [2][4] Clinical Development - The funds will support the Phase 1/2 clinical trial for SYNGAP1-related disorders, expected to start in the second half of 2026 [1][2] - The company aims to develop a first-in-class treatment targeting gene expression to address genetic diseases [2][9] Management Changes - Doug Williams, Ph.D., has been appointed as Board Chair, and Dan Tardiff, Ph.D., has been elevated to Chief Scientific Officer [3][5] - The board has seen resignations and transitions, with a focus on enhancing leadership in drug discovery and development [3][5] Investor Participation - The financing is led by Coastlands Capital, with participation from both new and existing investors, including Janus Henderson Investors and Balyasny Asset Management [4] - Leerink Partners is acting as the lead placement agent, supported by other financial institutions [4] Company Background - CAMP4 Therapeutics focuses on developing RNA-targeting therapeutics to restore healthy protein levels for a range of genetic diseases [9] - The company utilizes its proprietary RAP Platform to identify and target regulatory RNAs associated with various genetic disorders [9]

Core Viewpoint - CAMP4 Therapeutics Corporation reported strong execution in Q1 2025, advancing its clinical studies and achieving significant milestones in its therapeutic pipeline [2][4]. Corporate Highlights - The Phase 1 clinical trial of CMP-CPS-001 for Urea Cycle Disorders (UCDs) is ongoing, with dosing completed in multiple ascending dose (MAD) cohort 3; safety, pharmacokinetic, and pharmacodynamic data are expected in Q4 2025 [4][5]. - A Clinical Trial Application (CTA) was successfully submitted in Europe for a Phase 1b clinical trial targeting female OTC heterozygotes [4][5]. - The company nominated CMP-SYNGAP-01 as a development candidate for SYNGAP1-related disorders, with GLP toxicology studies expected to begin in 2025 [4][6]. - CAMP4 will present at the American Society of Cell and Gene Therapy (ASGCT), showcasing data on increased SYNGAP1 protein levels from CMP-SYNGAP-01 and interim safety results from the Phase 1 study of CMP-CPS-001 [4][5]. Financial Results - As of March 31, 2025, cash, cash equivalents, and marketable securities totaled $49.3 million, down from $64.0 million as of December 31, 2024 [7]. - Research and development expenses for Q1 2025 were $10.1 million, an increase from $9.7 million in Q1 2024, primarily due to higher clinical and preclinical study costs [7]. - General and administrative expenses rose to $3.8 million in Q1 2025 from $3.1 million in Q1 2024, attributed to increased personnel-related and overhead expenses [8]. - The net loss for Q1 2025 was $12.4 million, slightly improved from a net loss of $12.5 million in Q1 2024 [8][14].

Core Insights - CAMP4 Therapeutics is focused on developing regulatory RNA-targeting therapeutics aimed at upregulating gene expression to restore healthy protein levels for treating various genetic diseases [1][3] - The company will present three oral presentations at the 28th Annual Meeting of the American Society of Gene and Cell Therapy, highlighting their research and interim safety data from their Phase 1 clinical trial of CMP-CPS-001 [1][2] Group 1: Presentations Details - The first presentation will discuss targeting regulatory RNAs with antisense oligonucleotides for the potential treatment of urea cycle disorders, presented by Dan Tardiff, Ph.D. on May 16, 2025 [2] - The second presentation will also be by Dan Tardiff, Ph.D., focusing on the potential treatment of SYNGAP1-related disorders, scheduled for the same session [2] - The third presentation will provide interim safety readouts from a first-in-human double-blind, placebo-controlled study of CMP-CPS-001, presented by Yuri Maricich, M.D. on May 16, 2025 [2] Group 2: Company Overview - CAMP4 Therapeutics is developing disease-modifying treatments for over 1,200 genetic diseases where increasing healthy protein levels may provide therapeutic benefits [3] - The company's proprietary RAP Platform™ is designed to map regulatory RNAs and generate therapeutic candidates targeting regRNAs associated with haploinsufficient and recessive partial loss-of-function disorders [3]

Core Insights - CAMP4 Therapeutics is advancing its clinical programs, particularly focusing on CMP-CPS-001 for Urea Cycle Disorders (UCDs) and has initiated a Phase 1b trial for female OTC heterozygotes, addressing an underserved patient population [2][3][11] - The company has nominated CMP-SYNGAP-01 as a development candidate for SYNGAP1-related disorders, with promising preclinical data supporting its potential [2][11] - Financial results for the year ended December 31, 2024, show a net loss of $51.8 million, with cash and cash equivalents of approximately $64.0 million, sufficient to fund operations into Q2 2026 [6][7][8] Clinical Development - The Phase 1 clinical trial of CMP-CPS-001 in UCDs is ongoing, with dosing completed in two of four multiple ascending dose cohorts, and safety data expected in Q4 2025 [1][4] - The planned expansion into a Phase 1b clinical trial in female OTC heterozygotes is anticipated to begin in Q2 2025, with a Clinical Trial Application (CTA) to be submitted in Europe [2][11] - Recent studies indicate that female carriers of OTC deficiency may experience significant health risks, prompting a shift from passive observation to proactive treatment [3][11] Financial Performance - Research and development expenses for 2024 were $38.8 million, a decrease from $40.6 million in 2023, primarily due to reduced clinical and preclinical costs [6] - General and administrative expenses increased to $14.9 million in 2024 from $11.6 million in 2023, attributed to higher personnel-related costs [7] - The company reported a net loss of $51.8 million for 2024, compared to a loss of $49.3 million in 2023, with a net loss per share of $11.04 [7][15] Cash Position - As of December 31, 2024, CAMP4 had cash and cash equivalents of approximately $64.0 million, which is expected to support its planned activities into Q2 2026 [8][15] - The company’s total assets increased to $78.3 million in 2024 from $54.9 million in 2023, while total liabilities decreased slightly [15] Strategic Initiatives - CAMP4 is focusing on expanding its strategic partnerships to enhance the value of its RAP Platform, which targets regulatory RNAs to amplify gene expression [11] - The company has initiated a discovery program for GBA1-related Parkinson's Disease, with ongoing preclinical studies to identify a development candidate [11]