CAMP4 Therapeutics Receives UK Authorization for CMP-002 Clinical Trial

CAMP4 Therapeutics has received authorization from the United Kingdom’s Medicines and Healthcare Products Regulatory Agency (MHRA) to include UK sites in a global Phase 1/2 clinical trial of CMP-002 for patients with SYNGAP1-related disorder. This authorization follows recent clearances from Australia and Argentina, reinforcing CAMP4's commitment to advancing therapeutic options for patients with this rare condition.

CMP-002 is an antisense oligonucleotide (ASO) therapeutic candidate designed to upregulate SYNGAP1 gene expression, aiming to restore SYNGAP protein levels. The authorization is part of a broader plan to initiate the clinical trial in the fourth quarter of 2026, building on successful regulatory milestones in other regions.

SYNGAP1-related disorder, also known as SYNGAP1, is a rare, haploinsufficient central nervous system (CNS) disorder caused by mutations in the SYNGAP1 gene. This condition affects approximately 10,000 individuals in the United States, with nearly all patients experiencing intellectual disability, epilepsy in about 85% of cases, severe behavioral problems in 70% of cases, and sleep issues in 60% of cases. Currently, there are no approved disease-modifying therapies for this condition.

Josh Mandel-Brehm, President and CEO of CAMP4, stated, "This authorization by the MHRA expands the reach of our Phase 1/2 clinical trial and reinforces our commitment to rapidly advancing CMP-002 for patients and their families who have been without an intervention that tackles the underlying drivers of this disease."

CAMP4's RAP Platform technology is designed to target regulatory RNAs (regRNAs) that act locally on transcription factors to increase gene expression. CMP-002 specifically targets SYNGAP1-specific regRNA to increase SYNGAP1 gene expression and restore SYNGAP protein to near wild-type levels. Preclinical studies have shown that CMP-002 can increase SYNGAP protein expression in patient-derived neurons, reverse disease-relevant behavioral phenotypes in a humanized haploinsufficient mouse model, and improve seizure phenotypes and parameters in a chemically induced seizure mouse model.

The company has also submitted regulatory filings in the European Union, which remain under review. CAMP4 is developing disease-modifying treatments for a broad range of genetic diseases where amplifying healthy protein may offer therapeutic benefits. The company's approach aims to harness a fundamental mechanism of gene expression to target regRNAs associated with genes underlying haploinsufficient and recessive partial loss-of-function disorders.

With this authorization, CAMP4 is poised to expand its clinical trial to include UK sites, further advancing its mission to provide effective treatment options for patients with SYNGAP1-related disorder. The company remains focused on its goal of bringing regulatory RNA-targeting therapeutics to market to address a wide range of genetic diseases.