Cogent Biosciences (COGT) Piper Sandler 36th Annual Healthcare Conference summary

Company overview and strategy

• Focuses on developing best-in-class small molecule inhibitors for genetically driven rare diseases, especially oncology.

• Lead asset Bezuclastinib is in three clinical programs targeting systemic mastocytosis (SM) and second-line metastatic GIST.

• Upcoming clinical catalysts expected throughout 2025, with key data readouts from APEX, SUMMIT, and PEAK studies.

• Bezuclastinib designed for high selectivity for KIT mutations and minimal CNS penetration to reduce side effects.

• Aims to deliver a superior therapeutic index by minimizing off-target toxicities.

Clinical program updates and timelines

• APEX (advanced SM) and SUMMIT (non-advanced SM) trials will have phase 1/dose escalation updates at ASH conference.

• SUMMIT Part 2, a registration-directed study, is ahead of schedule with enrollment expected to complete in Q1 2025.

• APEX study results expected mid-2025, SUMMIT in H2 2025, and PEAK (GIST) by end of 2025.

• High demand and rapid enrollment in SUMMIT attributed to strong investigator and patient interest.

• SUMMIT Part 1 established 100 mg as the recommended phase 2 dose for non-advanced SM.

Efficacy, safety, and competitive landscape

• Bezuclastinib aims to outperform Avapritinib in symptom improvement for non-advanced SM, targeting >30% mean improvement and >25% of patients achieving 50% symptom reduction.

• Safety profile characterized by asymptomatic, reversible transaminase elevations, with no evidence of clinically relevant liver toxicity.

• Advanced SM treatment goal is to match efficacy of Avapritinib but with fewer severe side effects, such as cognitive impairment and edema.

• Inclusion of Avapritinib-experienced patients in trials may support future commercial switching.

• Commercial differentiation may rely on real-world symptom improvement rather than complex scoring systems.