Cogent Biosciences has achieved a significant milestone in oncology with its drug combination, bezuclastinib and sunitinib, receiving Breakthrough Therapy Designation (BTD) from the U.S. Food and Drug Administration (FDA) for treating Gastrointestinal Stromal Tumors (GIST). This designation underscores the therapy's potential to offer a substantial improvement over existing treatments, particularly for patients previously treated with imatinib, highlighting a new era of targeted cancer interventions.
The BTD was granted following compelling data from the PEAK trial, which showcased the combination's remarkable effectiveness. Patients receiving the bezuclastinib and sunitinib regimen experienced a 50% reduction in the risk of disease progression or mortality, a statistically significant outcome compared to sunitinib monotherapy. The median progression-free survival (PFS) for the combination therapy was an impressive 16.5 months, significantly extending the 9.2 months observed with sunitinib alone. This extended PFS represents a critical advantage for patients battling this aggressive form of cancer.
Importantly, the safety profile of the combined treatment was consistent with the known safety characteristics of sunitinib, indicating no new or unexpected adverse events. This favorable safety profile, combined with superior efficacy, positions the therapy as a promising option for GIST patients. The FDA's decision to accept Cogent's New Drug Application (NDA) for bezuclastinib plus sunitinib under its Real-Time Oncology Review (RTOR) program further accelerates its path to market. The RTOR program allows for an expedited review process, enabling the FDA to assess clinical trial data as it becomes available, potentially bringing this vital treatment to patients sooner.
Cogent Biosciences is moving swiftly, with plans to finalize the NDA submission by April 2026 and present the full results of the PEAK trial at a major medical conference in the first half of the same year. Looking ahead, the company also anticipates initiating a Phase 2 trial for first-line GIST patients with exon 9 mutations, who are either new to imatinib treatment or have recently started it. This continuous development pipeline illustrates Cogent's commitment to addressing unmet needs in cancer care. Additionally, the company previously submitted an NDA for bezuclastinib in Non-Advanced Systemic Mastocytosis in December 2025, following another Breakthrough Therapy Designation for that indication.
The FDA's Breakthrough Therapy Designation for bezuclastinib in conjunction with sunitinib represents a pivotal advancement in the battle against GIST, offering renewed hope for patients through a significantly improved treatment outcome and a rapid regulatory review process.