Bristol Myers Squibb RYZ101 trial meets Phase 3 goal
- Bristol Myers Squibb (NYSE:BMY) reported that its RYZ101 cancer therapy met the primary endpoint in the Phase 3 ACTION-1 trial by improving progression-free survival in patients with GEP-NETs.
- The trial showed a statistically significant and clinically meaningful improvement in progression-free survival compared with standard-of-care treatments after Lu177-based therapy.
- Bristol Myers Squibb said the results support discussions with the U.S. Food and Drug Administration regarding potential regulatory steps for RYZ101.
Bristol Myers Squibb (NYSE:BMY) announced topline results from the planned interim analysis of the Phase 3 ACTION-1 trial evaluating RYZ101 (225Ac-DOTATATE) in patients with somatostatin receptor-positive gastroenteropancreatic neuroendocrine tumors (GEP-NETs).
The randomized trial compared RYZ101 with investigator-selected standard treatments, including high-dose or high-frequency long-acting somatostatin analogues, everolimus, or sunitinib, in patients whose disease progressed after Lu177-based therapy.
"This is the kind of impact we set out to deliver with actinium-based radiopharmaceuticals, and it is an important milestone supporting the pipeline we are building across our RPT platform," said Amreen Husain, MD, chief medical officer of RayzeBio.
The company said safety results were consistent with the known RYZ101 safety profile, and the U.S. Food and Drug Administration granted RYZ101 Orphan Drug Designation for GEP-NET treatment in August 2026.
Bristol Myers Squibb said detailed ACTION-1 trial results will be presented at a future medical meeting, while enrolled participants will continue receiving care under the study protocol and will be followed for overall survival.
The company is developing RYZ101 through RayzeBio as part of its targeted radiopharmaceutical therapy platform, focusing on treatments that deliver radioactive molecules directly to cancer cells.
