Bristol Myers Squibb Halts Phase II Trial of Reblozyl for Rare Blood Disorder and Withdraws sBLA

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On June 6 local time, Bristol-Myers Squibb announced the discontinuation of its research program for the investigational drug Rebrozyl in patients with non-transfusion-dependent (NTD) beta-thalassemia and withdrew its supplemental Biologics License Application (sBLA). The clinical trial was halted because, based on the currently collected data, Bristol-Myers Squibb was unable to demonstrate to the FDA the benefit-risk profile of Rebrozyl in this patient population.

This decision was made several weeks after the PDUFA date for Rebrozyl was postponed. Previously, the FDA indicated that the PDUFA date might need to be delayed until the end of June. Bristol-Myers Squibb did not disclose any details regarding its decision to suspend the project under investigation, only stating that the data currently collected from the Phase II clinical trial of BEYOND is insufficient to address the FDA's questions.

BEYOND Phase II Clinical Trial is an efficacy and safety trial of Rebrozyl in adult patients with NTD β-thalassemia while providing supportive care. Bristol-Myers Squibb stated in June of last year that, in a study of 145 patients, Rebrozyl and supportive care improved anemia in 77% of patients compared to placebo. By week 24, 89.6% of patients did not require blood transfusions, compared to only 67.3% in the placebo group.

Although patients with NTD do not require lifelong regular blood transfusions, they may encounter a series of complications that necessitate occasional or even frequent transfusions. The most common side effects triggered by Rebrozyl are bone pain (36.5% in the Rebrozyl group vs. 6.1% in the placebo group), headache (30.2% in the Rebrozyl group vs. 20.4% in the placebo group), and joint pain (29.2% in the Rebrozyl group vs. 14.3% in the placebo group).

Celgene acquired the drug Rebrozyl from Acceleron for $25 million in 2011. In 2019, Bristol-Myers Squibb acquired Celgene, and subsequently, Bristol-Myers Squibb took ownership of Rebrozyl.

Notably, Rebrozyl is one of the four drugs that Bristol-Myers Squibb believes could eventually achieve $4 billion in annual sales. As early as 2020, it received approval for a second indication to treat anemia in certain adults with low-risk myelodysplastic syndromes. In 2021, the drug's annual sales reached $551 million.

Bristol-Myers Squibb claims that stopping the Phase II trial for the NTD indication will have a relatively minor impact on the overall performance expectations of Rebrozyl, and remains confident in its sales forecast reaching $4 billion.

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UPDATED: Bristol Myers pulls plug on Reblozyl program for rare blood disorder, months after missed PDUFA date