Cogent Biosciences (COGT) Guggenheim Inaugural Global Healthcare Conference summary

Company overview and lead asset

• Focuses on developing selective small molecule medicines for rare genetically driven diseases.

• Lead asset, bezuclastinib, is a selective KIT inhibitor in three pivotal trials: APEX, SUMMIT, and PEAK, with top-line results expected in the second half of 2025.

• Bezuclastinib is positioned as potent and selective, aiming to address unmet needs in mastocytosis and gastrointestinal stromal tumors (GIST).

• Pipeline includes four publicly announced programs, with the first phase I study for an FGFR2 inhibitor recently initiated.

Non-advanced systemic mastocytosis (SM) and SUMMIT trial

• Non-advanced SM significantly impacts quality of life, with a U.S. market opportunity estimated at $2 billion.

• SUMMIT trial data show bezuclastinib is well tolerated and highly active, with rapid reduction in disease markers.

• At 100 mg, 70% of patients had a 50% symptom improvement within 12 weeks, with minimal adverse events.

• Upcoming ASH presentation will provide longer-term data, focusing on 24-week endpoints and quality of life.

• SUMMIT part two is a pivotal, placebo-controlled study, aiming for higher efficacy benchmarks than Avapritinib.

Advanced systemic mastocytosis (ASM) and APEX trial

• ASM is a disease of mortality, with historical survival of about 3.5 years.

• Bezuclastinib aims for high activity similar to Avapritinib but with improved safety and tolerability.

• No observed liabilities such as intracranial hemorrhage or cognitive issues at effective doses.

• Goal is to match efficacy while minimizing toxicity compared to the incumbent.