Camp4 Therapeutics (CAMP) 12th Annual Cantor Fitzgerald Global Healthcare Conference summary

Disease background and unmet need

• SYNGAP1 is a genetically defined rare disease caused by haploinsufficiency, leading to significant neurological symptoms including learning disabilities, cognition problems, and seizures.

• Awareness of SYNGAP1 has grown rapidly, aided by patient advocacy and public figures sharing personal connections.

• There are currently no approved therapies for SYNGAP1, representing a critical unmet need.

Clinical trial progress and design

• Clinical trial authorizations have been obtained in Australia, Argentina, and the UK, with plans to expand to other regions and the US.

• The phase I study is set to initiate in Q4, with 10-12 sites selected based on expertise, patient access, and technical capabilities.

• The study will enroll approximately 30 patients in three dose cohorts, using a multiple ascending dose (MAD) design.

• Initial enrollment will start with patients around 8-10 years old, with plans to include younger children as safety allows.

• The trial will include a placebo arm and allow for crossover to active treatment after safety data is available.

Preclinical and translational insights

• Preclinical models, including humanized mice and primates, have shown promising pharmacodynamic and safety data, supporting dose selection.

• Recent studies demonstrated the ability to reduce both frequency and severity of seizures in mouse models, addressing a key clinical endpoint.

• The regRNA approach upregulates gene expression using antisense oligonucleotides, with PK/PD properties similar to approved drugs like SPINRAZA.