Bristol Myers Squibb shares positive Phase 3 RYZ101 trial data

Bristol Myers Squibb announced positive topline results from the planned interim analysis of the Phase 3 ACTION-1 clinical trial evaluating RYZ101. The drug is an investigational targeted radiopharmaceutical therapy developed by RayzeBio, a Bristol Myers Squibb company.

The study met its primary endpoint by showing a statistically significant and clinically meaningful improvement in progression-free survival by blinded independent central review. The safety profile remained consistent with previously known data for RYZ101. The trial compares the drug against the investigator choice of standard of care in patients with somatostatin receptor-positive gastroenteropancreatic neuroendocrine tumors whose cancer progressed after Lutetium-177 therapy.

The U.S. Food and Drug Administration granted RYZ101 Orphan Drug Designation in August 2026 for this indication. RayzeBio plans to present detailed trial data at an upcoming medical meeting and discuss regulatory submission steps with the FDA. Enrolled participants will continue to be monitored for overall survival.

Gastroenteropancreatic neuroendocrine tumors affect an estimated 18,000 newly diagnosed patients each year in the United States, with a total prevalence under 200,000. In earlier testing, a Phase 1b trial in 17 patients showed no serious treatment-related adverse events. RYZ101 is also undergoing early-stage trials for hormone receptor-positive metastatic breast cancer.