- Bristol Myers Squibb's immunotherapies Opdivo and Yervoy did not help people with unremovable or metastatic bladder cancer live longer than did chemotherapy in a late-stage clinical trial, the pharmaceutical company reported Monday.
- Bristol Myers said it remains "blinded" to data from the trial, which will continue to assess Opdivo and Yervoy's benefit on other study goals. Specifically, the trial is testing the drugs' effect in patients who are ineligible for cisplatin-based chemotherapy, and Opdivo together with chemotherapy in patients who are.
- The study enrolled patients with unresectable or metastatic urothelial carcinoma who had not previously been treated. For the results reported Monday, participants had to test positive for a biomarker known as PD-L1 that's associated with greater treatment response to immunotherapies like Opdivo.
Urothelial carcinoma, a common cancer that occurs in cells lining the bladder, was one of the first tumor types for which immunotherapy drugs were approved, with early Food and Drug Administration approvals in 2016 and 2017 for Opdivo, Roche's Tecentriq and Merck & Co.'s Keytruda.
But in the years following, data from follow-up testing showed patients given chemotherapy were living longer than those given immunotherapies in certain settings, leading the FDA to limit the latter drugs' use only to patients whose tumors were positive for PD-L1.
Then, early last year, both Roche and AstraZeneca withdrew from market their immunotherapies for use in previously treated bladder cancer. And by April, Tecentriq's and Keytruda's approvals in first-line bladder cancer were being reviewed by FDA advisers in a meeting meant to assess whether the drugs should remain on market after negative data in confirmatory testing.
In each case, however, the advisers voted in favor of maintaining the drugs' accelerated clearances. (The FDA later converted Keytruda's accelerated approval to a full clearance, but narrowed the indication to patients with previously untreated bladder cancer who are ineligible for platinum-based chemotherapy.)
Bristol Myers did not release specific data from its trial, called CheckMate-901, which began in 2017.
"Opdivo plus Yervoy has demonstrated durable, long-term survival improvements in several challenging-to-treat advanced cancers, and we are disappointed that the final analysis of CheckMate-901 did not show this same benefit in urothelial carcinoma patients whose tumor cells express PD-L1 [of at least] 1%," said Dana Walker, head of genitourinary cancer drug development at Bristol Myers, in the company's statement.
The negative result relates to the primary portion of CheckMate-901, which enrolled 707 patients to receive either Opdivo plus Yervoy or chemotherapy. Researchers will continue to monitor the combination's effects on survival for patients who are ineligible for cisplatin-based chemotherapy, one of the primary phase's two main goals.
A sub-study of CheckMate-901 will evaluate Opdivo plus chemotherapy again chemotherapy alone in patients who are eligible for cisplatin-based chemotherapy.
While the bladder cancer results are a setback, Bristol Myers has recently had success in expanding Opdivo's use before surgery in treating early lung cancer, an area that analysts expect will become a big part of the company's business.
Shares in Bristol Myers rose by more than 1% in Monday morning trading.
Editor's note: This story has been updated to clarify which of Keytruda's indications was narrowed in the process of converting from accelerated to full approval.