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News
A new alpha-emitting radiopharmaceutical, RYZ101, has met its primary endpoint in the phase 3 ACTION-1 trial, significantly improving progression-free survival in patients with rare gastroenteropancreatic neuroendocrine tumors (GEP-NETs) whose disease progressed after standard lutetium-177 therapy. The results mark the first phase 3 evidence for an alpha-based targeted radiopharmaceutical in this setting, with developer RayzeBio planning to engage the FDA on next regulatory steps.
A new treatment option may be on the horizon for patients with a rare and hard-to-treat cancer. RYZ101 (225Ac-DOTATATE), an investigational alpha-emitting radiopharmaceutical, has met the primary endpoint of the phase 3 ACTION-1 trial — delivering a statistically significant and clinically meaningful improvement in progression-free survival (PFS) compared to standard-of-care therapies in patients with somatostatin receptor-positive gastroenteropancreatic neuroendocrine tumors (GEP-NETs) that had progressed after lutetium-177 therapy.
This is a notable milestone: ACTION-1 is the first phase 3 trial to demonstrate efficacy for an alpha-based targeted radiopharmaceutical in this setting. RayzeBio, the drug's developer (now part of Bristol Myers Squibb), plans to engage the FDA on next steps toward regulatory submission. The safety profile was consistent with what was previously known about the agent.
By the Numbers:
Why it matters: For the many GEP-NET patients who eventually progress on lutetium-177 therapy, there has been no randomized phase 3 evidence to guide next steps — until now. RYZ101 could become the first approved alpha-based radiopharmaceutical in this space, offering a meaningful new option for a patient population with limited alternatives.
