Cogent Biosciences expects to file a new drug application for bezuclastinib in gastrointestinal stromal tumors early next year after what Leerink said was “the first positive trial in this disease in over a decade.”
Cogent Biosciences’ KIT inhibitor bezuclastinib, when used with the kinase inhibitor sunitinib, achieved what analysts hailed as “unprecedented” levels of efficacy in a Phase III trial of gastrointestinal stromal tumors, setting the biotech up for a regulatory run early next year.
“We view the results obtained in the 2L [setting] as offering unprecedented efficacy to date,” Leerink analysts told investors in a note on Monday, adding that the bezuclastinib combo set “a new standard of care in this disease and [generated] the first positive trial in this disease in over a decade.”
Cogent plans to submit a new drug application for bezuclastinib for gastrointestinal stromal tumors (GIST) in the first half of 2026. Shares of the company closed Monday at $32.46, up 119% from $17.64.
Data toplined on Monday come from the Phase III PEAK study, which enrolled more than 440 patients with GIST, split into several subparts to assess various outcomes, including pharmacokinetics and progression-free survival (PFS).
Patients treated with the bezuclastinib combo saw a median PFS of 16.5 months, as compared with 9.2 months in comparators given sunitinib alone. This corresponded to a 50% benefit in favor of Cogent’s drug, an effect that was highly statistically significant.
The bezuclastinib regimen also elicited a 46% objective response rate, versus 26% with sunitinib monotherapy.
As for safety, PEAK found bezuclastinib to be well-tolerated overall, with 7.4% of treated patients dropping out due to side effects, as compared with 3.8% in the sunitinib-alone arm. Meanwhile, grade 3+ liver enzyme elevations led to dose reductions in 12.7% of patients in the bezuclastinib group, as opposed to 1.5% in comparators. There were no life-threatening liver enzyme abnormalities.
PEAK is ongoing with an estimated completion date in September 2026. Cogent expects to provide more detailed findings from PEAK next year.
Given the PEAK readout, Leerink estimated peak sales of around $5.7 billion for bezuclastinib, of which $2.8 billion will come from GIST, while the remaining will be contributed by various types of systemic mastocytosis. In July, Cogent toplined the Phase III SUMMIT trial of bezuclastinib in non-advanced systemic mastocytosis, touting a nearly 9-point improvement in total symptom scores, which the biotech’s CEO Andrew Robins said at the time was “clinically meaningful and statistically significant.”
Cogent aims to file a new drug application for bezuclastinib in this indication by the end of the year.
