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

Key program updates and clinical plans

• Lead program CMP-002 targets SYNGAP1-related disorders, aiming to be the first disease-modifying therapy for this CNS condition, with clinical entry planned later this year after GLP-tox studies conclude mid-summer.

• Preclinical data in patient-derived cells, humanized mice, and primates show restoration of SYNGAP protein to healthy levels, reversal of disease phenotypes, and strong translatability to clinical settings.

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

• Regulatory discussions are ongoing, with plans to start with a multiple ascending dose (MAD) design, leveraging natural history studies for patient identification and potential external controls.

• Company is funded through 2027, with an additional $50M tranche available upon regulatory milestone achievement for first-in-human study initiation.

Platform and pipeline strategy

• RAP platform leverages regulatory RNAs to selectively upregulate gene expression using antisense oligonucleotides, enabling a pipeline of programs for haploinsufficiency disorders.

• Platform differentiation lies in proprietary identification and targeting of regulatory RNAs, with chemistry based on proven modalities like Spinraza for risk mitigation.

• Business development is key, with a recent GSK partnership on CNS and kidney targets, providing non-dilutive capital and expanding platform reach.

• Additional DEE programs will be announced later this year, with the SYNGAP program serving as a cornerstone for broader pipeline expansion.

• Focus remains on CNS haploinsufficiencies, with larger neurodegeneration indications pursued through partnerships.

Disease awareness and patient impact

• SYNGAP awareness has grown significantly in the past year due to advocacy, increased genetic testing, and industry engagement, with at least 20,000 patients estimated in the US and EU5.

• Only about 25% of SYNGAP patients are currently diagnosed, but rates are rising as awareness and drug development progress.

• SYNGAP is a severe, haploinsufficient disorder affecting cognition, behavior, motor skills, and communication, with no approved or disease-modifying therapies.

• Patient organizations and natural history studies are instrumental in improving diagnosis, supporting families, and informing clinical trial design.

• The company emphasizes patient-centricity, aiming for transformative impact on both patients and caregivers.