Camp4 Therapeutics (CAMP) Stifel 2026 Virtual CNS Forum summary

Company overview and platform

• Focuses on upregulating gene expression using oligonucleotides to address haploinsufficiency disorders, particularly in the CNS.

• Targets regulatory RNAs to fine-tune gene expression, offering a tunable rather than binary approach.

• Utilizes primary human cells and next-generation sequencing to map regulatory elements for gene targets.

• Chemistry optimization is tailored for CNS delivery, with intrathecal administration as the primary route.

• Lead program CMP-002 is advancing in SYNGAP1-related disorders, with clinical entry expected in the second half of the year.

SYNGAP1-related disorder program

• SYNGAP1 disorder is a genetic epilepsy with about 10,000 patients in the US and similar numbers in the EU5.

• Patients experience intellectual disability, seizures, behavioral issues, and significant sleep problems, with no disease-modifying therapies available.

• Diagnosis rates are improving due to better access to genetic screening, reducing time to diagnosis to about one year.

• Preclinical data show dose-dependent increases in SYNGAP protein and phenotypic rescue in mouse models.

• Intrathecal delivery achieves strong brain distribution and protein upregulation in relevant regions.

Clinical development and trial design

• Phase I/II study will start in severe pediatric patients, using a MAD design and placebo control, with open-label extension.

• Seizure reduction will be the primary endpoint, with additional measures such as cognition, motor function, and communication under consideration.

• Exploring endpoints from related disorders like Angelman and Rett, and leveraging natural history studies for context.

• Study will not be statistically powered for registration but aims to inform future pivotal trials.

• Focus initially on severe population, with plans to expand to milder cases for broader proof of concept.