FDA grants Breakthrough Therapy Designation to bezuclastinib plus sunitinib in GIST
The FDA granted Breakthrough Therapy Designation to bezuclastinib plus sunitinib for previously treated GIST. The PEAK trial showed median progression-free survival of 16.5 months versus 9.2 months for sunitinib alone.
The U.S. Food and Drug Administration has granted Breakthrough Therapy Designation for bezuclastinib in combination with sunitinib for patients with gastrointestinal stromal tumors (GIST) who have received prior treatment with imatinib. The designation is based on results from the PEAK trial, and earlier this month the FDA agreed to accept the company’s NDA under the Real-Time Oncology Review program.
The PEAK trial demonstrated a substantial and highly statistically significant clinical benefit on the primary endpoint of progression-free survival, reducing risk of disease progression or death compared to the current standard of care by 50% (hazard ratio of 0.50, 95% CI: 0.39–0.65). Median progression-free survival, as assessed by blinded independent central review, was 16.5 months for the bezuclastinib combination versus 9.2 months for sunitinib monotherapy. The combination was well tolerated, and no new safety risks were observed when compared to the known safety profile of sunitinib.
The company has submitted an NDA for bezuclastinib plus sunitinib in second-line GIST under the Real-Time Oncology Review program. Breakthrough Therapy Designation is intended to expedite the review of medicines that treat a serious or life-threatening condition and have shown clinical evidence indicating the potential for substantial improvement over available therapies.
Cogent said it plans to present full results from the PEAK trial at a major medical meeting during the first half of 2026. In mid-2026, the company expects to initiate a Phase 2 trial investigating the benefit of the bezuclastinib plus sunitinib combination for first-line GIST patients with exon 9 mutations who are naive to, or recently initiated treatment with, imatinib.
In quarterly results, the company said it ended March 31, 2026 with $866.4 million in cash, cash equivalents and marketable securities and expects this to fund operations into 2028, including potential U.S. launches of bezuclastinib for systemic mastocytosis and GIST, pending approval. Net loss for the three months ended March 31, 2026 was $97.4 million, compared with $72.0 million a year earlier, driven by research and development expense of $75.4 million and general and administrative expense of $28.2 million. The company also issued $230 million of 1.625% Convertible Senior Notes due 2031 and raised $45.7 million through its at-the-market equity program.