Bristol Myers Squibb has ended its R&D losing streak with a hit on one of the dual primary endpoints in a phase 3 multiple myeloma trial. But with the study continuing to evaluate the other primary endpoint, it is unclear whether the drugmaker has the evidence needed to seek approval from the FDA.
The phase 3 trial evaluated the effect of combining BMS’ investigational cereblon E3 ligase modulator iberdomide with the approved CD38 drug Darzalex and steroid dexamethasone. Relapsed or refractory multiple myeloma patients either received the investigational regimen or the standard combination of Darzalex, bortezomib and dexamethasone.
BMS reported statistically significant improvement in minimal residual disease (MRD) negativity rates in the iberdomide arm, meeting one of the dual primary endpoints of the trial. Investigators are continuing to track patients to evaluate progression-free and overall survival, which are respectively a dual primary and key secondary endpoint. BMS plans to discuss the results with health authorities.
The company added MRD as a dual primary endpoint partway through the study. Samit Hirawat, M.D., who recently stepped down as chief medical officer at BMS, said in February that the company added the MRD endpoint after talking to the FDA, telling investors that the change was part of an attempt to “figure out how to accelerate the process of drug development.”
BMS added the endpoint after every member of an FDA advisory committee answered “yes” in 2024 to the question “does the evidence support the use of MRD as an accelerated approval endpoint in multiple myeloma trials?” The vote took place before the recent changes to the leadership at the FDA.
Morgan Stanley analyst Terence Flynn asked BMS CEO Chris Boerner, Ph.D., where the FDA stands on MRD and the possibility of accelerated approval earlier this month. Talking at the Morgan Stanley event, Boerner said the study was reviewed by the agency but noted that there are “some new players at [the] FDA now.”
Nonetheless, the phase 3 victory is a positive for a R&D group that has suffered a series of setbacks. This year, BMS has reported the failures of phase 3 trials in melanoma, cardiomyopathy and schizophrenia. In a recent look at BMS’ upcoming readouts, BMO Capital Markets analysts classed the phase 3 iberdomide data as being of medium importance to the company.