Camp4 Therapeutics Corporation held an Analyst Day on September 28, 2026, to provide updates on its planned Phase 1/2 ASCEND clinical trial of CMP-002 and its broader product pipeline.
The ASCEND trial is a randomized, double-blind, placebo-controlled, multiple ascending dose study designed to evaluate CMP-002 for SYNGAP1-related disorder (SRD). Participants will be randomized 3:1 to receive intrathecal administration of CMP-002 or a sham procedure. The trial includes three MAD cohorts with a minimum of eight participants each, followed by an open-label extension study. The Company expects to dose the first patient by the end of 2026 and anticipates a topline data readout in the first half of 2028.
The trial will enroll participants aged 2 to less than 18 years with genetically confirmed SRD. Key inclusion criteria require a clinical diagnosis of SRD with a protein-truncating mutation in SYNGAP1 exons 5-19, a haploinsufficiency phenotype characterized by intellectual disability and refractory epilepsy, and a stable anti-seizure medication regimen. The study will assess safety, tolerability, pharmacokinetics, and six key domains of the disorder, including seizures, sleep, motor function, communication, behavior, and development.
In addition to the ASCEND trial, Camp4 is advancing a preclinical program targeting SHANK3 for Phelan-McDermid Syndrome, a neurodevelopmental disorder with no approved treatments. The Company estimates there are more than 45,000 patients with this condition in the United States and expects to designate a development candidate in 2027. Camp4 also disclosed it is evaluating programs for two additional early-stage central nervous system haploinsufficient diseases.
