Cogent Biosciences (COGT) 44th Annual J.P. Morgan Healthcare Conference summary

Strategic focus and financial outlook

• Three pivotal trials for Bezuclastinib in GIST and systemic mastocytosis read out positively in 2025, with all NDAs targeted for submission in late 2025 and the first half of 2026.

• Commercial launch in the U.S. is planned for the second half of 2026, with preparations including a 100-person team and ex-U.S. partnerships under consideration.

• $900 million cash on hand at the end of 2025/start of 2026 provides a runway into 2028, supporting commercialization and pipeline development.

• Expansion into Europe and other global markets is under consideration, with partnerships likely outside the U.S. and possible direct investment in Europe.

• Commercial team buildout is underway, aiming for a focused, rare disease-oriented approach with about 100 employees.

Bezuclastinib clinical and commercial potential

• Bezuclastinib demonstrated significant efficacy in pivotal trials, including a 16.5-month median PFS, nearly 50% response rate in second-line GIST, and rapid, deep symptom improvement in NonAdvSM.

• The drug is positioned as the first in 20 years to show activity against an active comparator in GIST, with minimal competition in key indications.

• Intellectual property protection extends into the mid-2040s, with pending patents and regulatory exclusivity potentially securing rights through 2046.

• Commercial opportunities are estimated at $4 billion annually for both GIST and systemic mastocytosis, with a total global market opportunity exceeding $8 billion.

• Expanded access programs are providing early experience to physicians and patients ahead of full FDA approval.

Pipeline and R&D initiatives

• Pipeline includes a pan-KRAS inhibitor and a highly selective JAK2 V617F inhibitor, both targeting IND submission in 2026.

• Pan-KRAS inhibitor CGT1815 shows best-in-class potency and selectivity across KRAS mutations, with strong preclinical efficacy and avoidance of HRAS/NRAS activity.

• JAK2 V617F inhibitor CGT1145 demonstrates over 100-fold selectivity over wild type and potential as a disease-modifying therapy in preclinical models.

• Additional assets in FGF, ErbB2, and PI3K are expected to enter the clinic by the end of 2026.

• Multiple ongoing and planned trials aim to expand Bezuclastinib’s commercial utilization, including 1L and 3L+ GIST, and combination studies in SM.