Cogent Biosciences (COGT) investor relations material

Regulatory and launch readiness

• Bezuclastinib is being developed for non-advanced and advanced systemic mastocytosis and imatinib-resistant GIST; pivotal studies read out in 2025, with NDAs filed during 2026.

• The first PDUFA date, for GIST, is November 30, 2026; the CEO said approval could be days away and launch could begin within a handful of days, with orders taken immediately.

• Home-office, commercial and medical teams were in place; field professionals were onboarded in Q3 and had begun territory outreach.

• Transaminase monitoring is expected; across APEX, SUMMIT and PEAK, reported elevations were lab abnormalities, with higher-grade cases managed by dose reduction or, rarely, discontinuation and return to baseline.

• An expanded access program spans all three indications; patients have accessed treatment, while the program has also educated physicians, patients and advocates ahead of approval.

GIST opportunity and development plans

• In PEAK Phase III, mean combination treatment duration exceeded 21 months versus ~7 months for the prior standard of care; annual imatinib resistance is modeled at ~3,000 patients each in the U.S. and Western Europe.

• For peak sales in 2032 or 2037, the commercial opportunity is expected to build as patients remain on therapy across multiple years, creating a prevalence market beyond new annual cases.

• European MAAs are targeted for submission in 2027; the stated plan is to focus on European GIST access and pursue a partner only if it returns pricing authority to the innovator.

• A 40-patient first-line GIST cohort is open to any mutational profile; work with global KOLs is continuing toward a potential registration program, with more formal guidance expected in the near future.

Systemic mastocytosis positioning

• Non-advanced SM launch is anticipated in late 2026 or early 2027; diagnosis often follows visits to seven or eight physicians, and launch focus includes patients who stopped or are reluctant to start avapritinib.

• Bezuclastinib is described as KIT-selective and non-CNS-penetrant; the proposed differentiation is a wider therapeutic index for potent dosing and potential elimination of mutant mast-cell clones.

• A switch study assesses symptoms in patients moving from avapritinib 25 mg or 50 mg to bezuclastinib; some results were expected at ASH by year-end 2026, with additional data in early 2027.

• Advanced SM represents ~10% of SM patients; the CEO cited an average ~3-year lifespan from diagnosis without TKI treatment and positioned bezuclastinib’s safety profile against high-dose avapritinib.

• The elenestinib trial was described as still enrolling with a 48-week primary endpoint; the CEO estimated approval was at least several years away.