The centerpiece of Bristol Myers Squibb’s 2024 RayzeBio buy has read out, hitting the primary endpoint in a late-stage tumor trial.
Bristol Myers Squibb’s experimental radiopharmaceutical therapy significantly improved progression-free survival in a late-stage trial for gastroenteropancreatic neuroendocrine tumors, a rare type of cancer that typically forms in the gastrointestinal tract.
BMS picked up the radiopharma candidate, known as RYZ101, in its $4.1 billion RayzeBio acquisition announced at the end of 2023 and closed in early 2024.
Now, the Big Pharma said that an interim analysis of the Phase 3 portion of the study has met its main goal, with a statistically significant and clinically meaningful improvement in progression-free survival (PFS), as measured by blinded independent central review. BMS did not provide data behind the win in its Monday news release.
The ACTION-1 study included patients with advanced somatostatin receptor–expressing gastroenteropancreatic neuroendocrine tumors (GEP-NETs) that progressed after treatment with Novartis’ Lutathera, a targeted radiopharmaceutical therapy that serves as the condition’s current standard of care. The primary endpoint was to be measured after 143 PFS events occurred, according to ClinicalTrials.gov.
RYZ101 is made with actinium-225, a radioisotope that is teed up to be the next big thing in radiopharma—to the point of straining supply of the isotope. The high demand even prompted a pause in the enrollment of ACTION-1 back in 2024, shortly after BMS’ acquisition of RayzeBio was completed.
RYZ101 secured orphan drug status from the FDA in August based on significant unmet need, BMS said. The designation paired with the interim results support further development efforts and “plans to engage with the FDA to discuss next steps toward a regulatory submission,” according to the pharma.
Safety results were consistent with the known RYZ101 profile, BMS said. In a previous Phase 1b test in 17 patients, RYZ101 was well tolerated, with no treatment-related serious adverse events or dose discontinuations due to adverse events reported.
More detailed findings from ACTION-1 are slated for presentation at an upcoming medical meeting, the New Jersey company said.
“These are the first results from a Phase 3 trial of an alpha-isotope based targeted RPT (225Ac) in any indication, and we are moving with urgency to bring these data to the FDA and, ultimately, provide a novel option for patients,” RayzeBio Chief Medical Officer Amreen Husain said in the release.
GEP-NETs have a global prevalence of fewer than 200,000 patients. While current lutetium-177–based radiopharmaceutical therapies such as Lutathera have shown significant clinical benefit for GEP-NETs, most patients progress following treatment, according to BMS.
RYZ101 is also being evaluated in earlier-stage trials for hormone receptor–positive metastatic breast cancer.
