Camp4 Therapeutics (CAMP) investor relations material

Strategic vision and pipeline expansion

• Focus on developing disease-modifying therapies for severe neurodevelopmental disorders, starting with SYNGAP1 and expanding to SHANK3 and other haploinsufficiency-driven epileptic encephalopathies, including Phelan-McDermid Syndrome.

• Platform targets non-coding regulatory RNAs with antisense oligonucleotides (ASOs) to upregulate gene expression, aiming for broad applicability across genetic brain diseases.

• Early pipeline includes additional CNS programs leveraging expertise from SYNGAP1, with clinical initiation for SHANK3 planned for Q4 2026 and a development candidate for another CNS program in 2027.

• All programs focus on disorders with high unmet need, significant caregiver burden, and no approved disease-modifying treatments.

Clinical development and ASCEND trial design

• ASCEND phase I/II trial for CMP-002 in SYNGAP1 is a randomized, double-blind, controlled, multiple ascending dose study with global reach across 11+ sites in nine countries.

• Study population includes children aged 2 to under 18 with genetically confirmed SYNGAP1 haploinsufficiency, daily seizures, impaired sleep, and non-verbal status, focusing on protein-truncating mutations in exons 5-19.

• Three doses administered over three months, followed by a six-month follow-up; open label extension planned for maintenance dosing.

• Trial endpoints span seizure frequency, sleep, motor function, communication, behavior, and cognition, using validated and gold-standard assessments, including vEEG and actigraphy.

• First patient dosing expected by year-end 2026, with top-line results targeted for the first half of 2028 and potential for interim safety and pharmacodynamic updates.

Preclinical and translational data

• CMP-002 restores SYNGAP1 mRNA and protein levels in patient-derived neurons and animal models, with dose-dependent increases approaching wild-type levels.

• In humanized SYNGAP1 mice, CMP-002 improved neurological phenotypes across learning, memory, motor function, hyperactivity, and seizure resistance.

• Intrathecal administration in non-human primates achieved broad brain distribution and increased SYNGAP protein, with good tolerability.

• Preclinical evidence supports intervention at various ages, with no observed therapeutic window limitation.

• Platform approach validated by reversibility of phenotypes in animal models, supporting expansion to other synaptopathies.