CAMP4 Therapeutics outlines Phase 1/2 ASCEND trial for CMP-002 in SYNGAP1-related disorder at Analyst Day

Camp4 Therapeutics Corp

ASCEND is a Phase 1/2 randomized, double-blind, placebo-controlled MAD trial of intrathecal CMP-002 vs sham in patients aged 2 to <18 with genetically confirmed SYNGAP1-related disorder.
Trial includes three MAD cohorts with at least eight participants each, 3:1 randomization, three doses over ~3 months, and a 28-day screening plus 4-week baseline.
Sites in UK, Australia, and Argentina approved; EU under review; >11 sites across at least 9 countries; first patient dosing expected by end of 2026, topline H1 2028.
Pipeline: preclinical SHANK3 program for Phelan-McDermid Syndrome, >45,000 US patients; development candidate expected in 2027; two additional early-stage CNS haploinsufficiency programs evaluated.
Company believes CMP-002 dose levels in all three ASCEND cohorts are within expected therapeutic range based on preclinical PK/PD modeling.

This headline and bullets were generated automatically by deepseek-v4.1-flash:cloud@v2 from the public filing. Read the source on SEC.gov before relying on any specific claim. Not investment advice. See methodology for how this pipeline works.