Cogent Biosciences (COGT) investor relations material

Key clinical trial results and medical insights

• PEAK phase III trial in second-line GIST showed bezuclastinib plus sunitinib doubled median progression-free survival to 16.5 months and achieved a 46% objective response rate, with a 50% reduction in risk of progression or death compared to sunitinib alone.

• Combination therapy demonstrated pan-mutant coverage, addressing the heterogeneity of KIT mutations in GIST, and produced complete responses in some patients, a rare outcome in this disease.

• Safety profile of the combination was favorable, with minimal additive toxicity, no new risks compared to sunitinib monotherapy, and balanced incidence of grade 3+ adverse events; most common adverse events were reversible and manageable, and discontinuation rates were low.

• Real-world patient cases illustrated durable and deep responses to the combination, with ongoing benefit and manageable side effects.

• Expanded access program is available in the U.S., and the full PEAK data set will be presented at a major medical meeting in the first half of 2026.

Market opportunity and commercial outlook

• Second-line GIST represents a $4 billion annual opportunity in the U.S. and Europe, with no current competition and benchmark pricing for similar drugs exceeding $40,000 per month.

• The broader opportunity across GIST and advanced systemic mastocytosis is estimated at over $7.5 billion globally, primarily in the U.S. and Western Europe.

• Intellectual property protections extend to 2038 for composition of matter and potentially to 2043 for the proprietary formulation, providing a long runway before generic competition.

• Expanded access program enables immediate availability of the combination for eligible patients.

Unmet medical need and scientific rationale

• Significant unmet need persists for GIST patients resistant or intolerant to imatinib, as current therapies offer limited efficacy and are only effective against subsets of resistance mutations.

• Bezuclastinib + sunitinib combination inhibits the full spectrum of primary and secondary KIT mutations, addressing clonal heterogeneity and resistance.