Cogent Biosciences Submits NDA for Bezuclastinib in GIST

Bezuclastinib combination demonstrated 16.5 month mPFS and 46% ORR in imatinib-resistant GIST patients

Apr. 2, 2026 at 5:19am

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Cogent Biosciences, Inc. announced the completion of the submission of its New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for bezuclastinib in patients with Gastrointestinal Stromal Tumors (GIST) who have received prior treatment with imatinib. The bezuclastinib NDA was submitted under the FDA's Real-Time Oncology Review (RTOR) program based on positive results from the PEAK trial, where the bezuclastinib combination demonstrated a substantial and highly statistically significant clinical benefit on the primary endpoint of progression free survival (PFS), reducing risk of disease progression or death compared to the current standard of care by 50%.

Why it matters

The submission of the bezuclastinib NDA represents a significant step towards bringing a new therapy to patients with second-line GIST, an area with limited treatment options. The PEAK trial data showed the bezuclastinib combination has the potential to meaningfully change the treatment landscape for these patients by dramatically improving upon the current standard of care.

The details

In the PEAK trial, the bezuclastinib combination demonstrated a median PFS of 16.5 months compared to 9.2 months for sunitinib monotherapy. Additionally, the bezuclastinib combination achieved an unprecedented objective response rate of 46% in imatinib-resistant GIST patients, compared to 26% for sunitinib. The bezuclastinib combination was generally well tolerated, and no unique risks were observed compared to the known safety profile of sunitinib.

  • The bezuclastinib NDA was submitted in April 2026 under the FDA's RTOR program.
  • Bezuclastinib was granted Breakthrough Therapy Designation as a treatment for GIST earlier in 2026.

The players

Cogent Biosciences, Inc.

A biotechnology company focused on developing precision therapies for genetically defined diseases, including bezuclastinib for GIST and advanced systemic mastocytosis.

Andrew Robbins

President and Chief Executive Officer of Cogent Biosciences.

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What they’re saying

“We are excited to complete our PEAK NDA submission which marks a significant step toward bringing a new therapy to patients with second-line GIST.”

— Andrew Robbins, President and Chief Executive Officer

What’s next

Cogent plans to present full results from the PEAK trial at a major medical meeting during the first half of 2026. The company is also on track this quarter to initiate a Phase 2 trial investigating the benefit of the bezuclastinib plus sunitinib combination for first-line GIST patients with exon 9 mutations.

The takeaway

The submission of the bezuclastinib NDA for GIST represents an important milestone in providing a potential new treatment option for patients with limited options. The positive PEAK trial data showing significant improvements in progression-free survival and response rates compared to the current standard of care highlights the promise of this targeted therapy.