CAMP4 Therapeutics’ CMP-002 Increases Seizure Threshold, Reduces Severity in SYNGAP1 Model
CAMP4 Therapeutics reported that a single dose of its ASO candidate, CMP-002, produced a statistically significant increase in seizure threshold and reduced seizure severity in a SYNGAP1 haploinsufficient mouse model. The company plans to initiate a Phase 1/2 clinical trial in SYNGAP1-related disorder patients in the second half of 2026.
1. New Preclinical Data Presentation
CAMP4 Therapeutics presented preclinical findings at TIDES showing that CMP-002 improved seizure outcomes in a SYNGAP1 haploinsufficient model.
2. Efficacy in SYNGAP1 Haploinsufficient Model
A single intrathecal dose of CMP-002 led to a statistically significant increase in seizure threshold and reduction in seizure severity in PTZ-induced tonic-clonic seizure tests in juvenile mice.
3. Mechanism of Action
CMP-002 is an antisense oligonucleotide designed to bind SYNGAP1-specific regulatory RNA, amplifying SYNGAP1 mRNA and restoring protein levels toward wild-type in the central nervous system.
4. Phase 1/2 Trial Plans
Based on these results, CAMP4 plans to initiate a Phase 1/2 clinical trial in individuals with SYNGAP1-related disorder in the second half of 2026.