Bristol Myers Squibb Reports Positive Phase 3 Results in Multiple Myeloma Trial

Bristol Myers Squibb (BMS) has announced a significant breakthrough in its phase 3 multiple myeloma trial, marking an end to a series of recent setbacks in the company's R&D efforts. The study, which evaluated the combination of BMS's investigational cereblon E3 ligase modulator iberdomide with Darzalex and dexamethasone, has met one of its dual primary endpoints, showing statistically significant improvement in minimal residual disease (MRD) negativity rates.

Trial Design and Results

The phase 3 trial compared the investigational regimen of iberdomide, Darzalex (a CD38 drug), and dexamethasone against the standard combination of Darzalex, bortezomib, and dexamethasone in patients with relapsed or refractory multiple myeloma. While the study has successfully met the MRD negativity endpoint, investigators continue to track patients to evaluate progression-free and overall survival, which constitute the other primary and a key secondary endpoint, respectively.

Regulatory Implications and FDA Considerations

The addition of MRD as a dual primary endpoint midway through the study has raised questions about the potential for accelerated approval. This modification followed discussions with the FDA and was part of BMS's strategy to expedite drug development. The company's decision was bolstered by a unanimous FDA advisory committee vote in 2024, supporting the use of MRD as an accelerated approval endpoint in multiple myeloma trials.

However, recent changes in FDA leadership have introduced uncertainty regarding the agency's current stance on MRD and accelerated approval pathways. BMS CEO Chris Boerner acknowledged this shift, noting that while the study was reviewed by the agency, there are "some new players at [the] FDA now."

Implications for BMS and Industry Outlook

This phase 3 success comes as a welcome development for BMS, following a string of clinical trial failures in melanoma, cardiomyopathy, and schizophrenia earlier this year. While analysts from BMO Capital Markets have classified the iberdomide data as being of medium importance to the company, the positive results may help restore confidence in BMS's R&D capabilities.

As the pharmaceutical industry closely watches the evolving regulatory landscape, particularly regarding accelerated approval pathways, BMS's next steps with iberdomide could set important precedents for future drug development strategies in multiple myeloma and beyond.