Camp4 Therapeutics Announces ASCEND Trial Timeline at Analyst Day

What Happened
Camp4 Therapeutics (CAMP) held an Analyst Day on September 28, 2026 and filed an 8‑K presenting details on its planned Phase 1/2 ASCEND clinical trial of CMP‑002 for SYNGAP1‑related disorder (SRD) and updates on its broader pipeline. The company provided study design, eligibility, site and timeline information, and furnished the Analyst Day and corporate presentations as exhibits. The filing is signed by CEO Josh Mandel‑Brehm.

Key Details

• ASCEND is a Phase 1/2, randomized, double‑blind, placebo‑controlled, multiple ascending dose (MAD) study; participants randomized 3:1 to intrathecal CMP‑002 or a sham pinprick.
• Study structure: three MAD cohorts (minimum 8 participants per cohort) → open‑label extension; total participant involvement ≈ 10 months (28‑day screen, ≥4‑week baseline, ~3‑month dosing, ~6‑month follow‑up).
• Population and endpoints: ages 2 to <18 with genetically confirmed SRD (protein‑truncating SYNGAP1 variants in exons 5–19); primary goals are safety/tolerability, PK/PD and dose selection; exploratory endpoints span seizures, sleep, motor/gait, communication, behavior, and cognition (including 24‑hour video‑EEG, actigraphy, validated clinical scales).
• Operational/timing: planned at >11 sites across ≥9 countries (UK, Australia, Argentina sites approved); first patient dosing targeted by end of 2026; topline data targeted in H1 2028.
• Pipeline note: advancing a preclinical SHANK3 program for Phelan‑McDermid Syndrome with a development candidate expected in 2027; estimates >45,000 U.S. patients for that indication; two additional early‑stage haploinsufficient CNS programs under evaluation.

Why It Matters
This 8‑K gives investors concrete clinical and operational milestones to watch: first patient dosing by end‑2026 and a topline readout in H1‑2028 are near‑term catalysts if achieved. The detailed eligibility, endpoints and multi‑domain assessments signal the company’s approach to measuring meaningful clinical effects in a rare pediatric population. Pipeline progress (SHANK3 candidate selection in 2027) highlights CAMP4’s broader strategy in haploinsufficiency CNS disorders. As the filing notes, these plans are forward‑looking and subject to clinical, regulatory and operational risks.