CAMP · CAMP4 Therapeutics Corporation

This disclosure represents a high-materiality inflection point for CAMP4, as it presents the first peer-facing preclinical evidence that CMP-002 meaningfully modulates seizure phenotypes — a defining and burdensome feature of SYNGAP1-related disorder affecting ~85% of patients. Unlike prior behavioral and motor endpoints, the PTZ-induced seizure model directly addresses a clinically urgent unmet need with no approved disease-modifying therapies. The statistically significant resistance to seizure onset and reduction in severity following a single dose suggest robust target engagement and functional rescue of neuronal excitability, reinforcing the mechanistic hypothesis that restoring SYNGAP1 protein toward wild-type levels can correct multiple disease domains. Critically, these findings de-risk the upcoming Phase 1/2 trial by providing biological plausibility for clinical efficacy in epilepsy — a key secondary endpoint likely to inform dosing, patient selection, and regulatory strategy. The timing — with clinical initiation expected in 2H 2026 — positions CAMP4 to generate first-in-human safety and biomarker data within 12–18 months, potentially catalyzing partnership interest or valuation re-rating. However, investors must weigh this progress against inherent preclinical-to-clinical translation risks highlighted in the forward-looking statements, including uncertainties around human pharmacokinetics, intrathecal delivery tolerability, and biomarker correlation in patients.