Bristol Myers Squibb Announces Positive Topline Results from the Interim Analysis of Phase 3 ACTION-1 for RYZ101, RayzeBio’s Radiopharmaceutical Therapy, in Patients with GEP-NETs
Key Highlights
- ➤ACTION-1 met its primary endpoint with statistically significant PFS improvement
- ➤RYZ101 showed clinically meaningful PFS improvement versus investigator’s choice of standard care
- ➤Safety results remained consistent with RYZ101’s known safety profile
- ➤RayzeBio plans FDA discussions toward a potential regulatory submission
- ➤FDA granted RYZ101 Orphan Drug Designation for GEP-NETs in August 2026
Expert Statements
Amreen Husain, Chief medical officer of RayzeBio
“The improvement in progression-free survival is a defining moment for RYZ101 and for the patients we built this program around — people with advanced GEP-NETs whose cancer has progressed after Lu177-based therapy and who have few remaining options”
Amreen Husain, Chief medical officer of RayzeBio
“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.”
Amreen Husain, Chief medical officer of RayzeBio
“These are the first results from a Phase 3 trial of an alpha-isotope based targeted RPT ((225)Ac) in any indication, and we are moving with urgency to bring these data to the FDA and, ultimately, provide a novel option for patients.”
Simron Singh, Medical oncologist at the Odette Cancer Centre, Sunnybrook Health Sciences Centre; Professor of Medicine at the University of Toronto; principal investigator of the ACTION-1 study
“Lu177-based therapy is widely used in GEP-NETs, but unfortunately the vast majority of patients will eventually progress. Determining the optimal treatment after Lu177 has been an important question for my patients, and until now there has been no randomized Phase 3 evidence to guide it”
Simron Singh, Medical oncologist at the Odette Cancer Centre, Sunnybrook Health Sciences Centre; Professor of Medicine at the University of Toronto; principal investigator of the ACTION-1 study
“ACTION-1 provides the first Phase 3 results for an alpha-based targeted RPT, and a clinically meaningful improvement in progression-free survival is a crucial development in improving the care of patients with GEP-NETs.”
Bristol Myers Squibb Announces Positive Topline Results from the Interim Analysis of Phase 3 ACTION-1 for RYZ101, RayzeBio’s Radiopharmaceutical Therapy, in Patients with GEP-NETs
The study met the primary endpoint with a statistically significant and clinically meaningful improvement in progression free survival (PFS)
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