Camp4 Therapeutics (CAMP) 44th Annual J.P. Morgan Healthcare Conference summary

Key program updates and clinical plans

• Lead program CMP-002 targets SYNGAP1-related CNS disorders with no current disease-modifying therapies, aiming for first-in-human trials by year-end following completion of GLP-tox studies this summer.

• Preclinical data in patient-derived cells, humanized mice, and primates show CMP-002 can restore SYNGAP protein to healthy levels, reverse disease phenotypes, and is safe at therapeutic doses.

• Phase I-II trial will be global, open-label, and focus on rapid enrollment, efficacy, safety, and optimal dosing, with endpoints spanning seizures, neurodevelopment, and behavior.

• Regulatory discussions are ongoing, with plans to use natural history data for trial design and potential external controls, and to pursue multiple approval pathways.

• Company is funded through 2027, with an additional $50M tranche available upon regulatory milestone achievement.

Platform and pipeline strategy

• RAP platform leverages proprietary regulatory RNA mapping and antisense oligonucleotide chemistry to selectively upregulate gene expression, enabling a pipeline beyond SYNGAP1.

• Platform differentiation lies in exclusive focus on regulatory RNAs, proprietary discovery methods, and use of validated chemistries for CNS delivery.

• Additional DEE programs will be announced later this year, with business development and partnerships (e.g., GSK) expanding reach into CNS and kidney indications.

• Platform enables targeting over 30 CNS haploinsufficiencies, with internal focus on DEEs and partnerships for larger neurodegenerative diseases.

• Chemistry advancements and intrathecal delivery are prioritized, with future interest in blood-brain barrier penetration and durable dosing.

Disease landscape and awareness

• SYNGAP1 disorder awareness has grown rapidly due to advocacy, increased genetic testing, and industry focus, with at least 20,000 patients estimated in the US and EU5.

• Only about 25% of SYNGAP1 patients are currently diagnosed, but rates are rising as new therapies and diagnostics emerge.

• SYNGAP1 causes severe intellectual, behavioral, and motor impairments, with current treatments limited to symptom management and significant caregiver burden.

• Patient organizations and KOLs play a critical role in awareness, diagnosis, and trial readiness, facilitating rapid clinical development.

• Natural history studies provide valuable data for endpoint selection and may serve as external controls in regulatory submissions.