Bristol Myers Squibb’s (BMS) experimental radiopharmaceutical therapy has met the primary endpoint of a phase 3 trial, marking a development milestone for its cancer treatment pipeline.
The company announced that the treatment significantly improved progression-free survival compared with standard care in patients with rare, advanced gastroenteropancreatic neuroendocrine tumours (GEP-NETs).
RYZ101 combines DOTATATE with actinium-225 to deliver radiation directly to tumour cells in the aim to destroy them.
The randomised ACTION-1 study evaluated RYZ101 in patients with somatostatin receptor-positive GEP-NETs whose disease had progressed following lutetium-177-based therapy.
Patients received either the experimental RYZ101 or an investigator-selected standard treatment, including somatostatin analogues, everolimus or sunitinib.
At a planned interim analysis, the company reported a statistically significant and clinically meaningful improvement in progression-free survival, assessed through blinded independent central review.
This endpoint measures how long patients live without their disease worsening. Follow-up for overall survival will continue as the announcement does not yet establish whether the therapy helps patients live longer.
The drug, which belongs to a class of cancer therapies dubbed ‘canonballs’ for their ability to precisely target tumourous tissue, is the lead programme of RayzeBio – which BMS acquired for approximately $4.1bn in February 2024.
That transaction brought the company a radiopharmaceutical pipeline alongside manufacturing capabilities, expanding its presence in targeted cancer treatments.
While the latest result gives BMS a clinical milestone from that investment, the commercial opportunity will depend on the complete trial findings, regulatory review and the treatment’s eventual place in care.
Bristol Myers said it plans to engage with the US Food and Drug Administration to discuss the next steps towards a regulatory submission.
RYZ101 received orphan drug designation for GEP-NETs in August 2026, although this does not constitute approval.
Detailed findings are expected at a future medical meeting. The topline announcement did not disclose the magnitude of the progression-free survival benefit or a detailed breakdown of adverse events.
Those results will be important for understanding how RYZ101 compares with existing options and assessing its potential role after lutetium-177 treatment. For now, the therapy remains investigational, with regulatory discussions and further data disclosure still ahead.
Patients enrolled in the study will continue treatment under the trial protocol and will be followed to assess overall survival.
In a previous study, RYZ101 was well tolerated, with no treatment-related serious adverse events or dose discontinuations due to adverse side effects. BMS is also testing it in earlier-stage trials for hormone receptor-positive metastatic breast cancer.
Bristol Myers shares rose nearly 4% in pre-market trading on Monday as the company announced positive results for RYZ101.
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