Bristol Myers Squibb (BMS) has revealed that its randomised Phase III INDEPENDENCE trial assessing Reblozyl (luspatercept-aamt) in combination with Janus kinase inhibitor (JAKi) therapy in adults with myelofibrosis-associated anaemia did not meet its primary endpoint.
INDEPENDENCE is a double-blind study that compared the safety and efficacy of Reblozyl against placebo in those with myeloproliferative neoplasms-associated myelofibrosis undergoing JAK2 inhibitor therapy and requiring red blood cell (RBC) transfusions.
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The trial’s primary goal is to achieve RBC transfusion independence while in any consecutive 12-week duration, beginning within the first 24 weeks of treatment, against a placebo.
BMS noted that despite not meeting the primary endpoint, subjects experienced a clinically meaningful improvement in RBC transfusion independence, consistent with earlier Phase II outcomes.
The trial also revealed various secondary measures where the therapy showed a benefit, including subjects who achieved a minimum of 50% decrease in RBC transfusion burden and also those achieving an increase in haemoglobin by a minimum of 1g/dL while staying transfusion independent for at least 12 consecutive weeks.
The treatment-emergent adverse events observed during the trial were in line with Reblozyl’s established safety profile.
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By GlobalDataMyelofibrosis is an uncommon type of blood cancer.
Bristol Myers Squibb haematology, oncology and cell therapy development head and senior vice-president Anne Kerber said: “We remain confident in the ability of Reblozyl to improve outcomes for patients with myelofibrosis-associated anaemia and believe the totality of these results, including meaningful improvements in transfusion burden and haemoglobin levels, support the potential to address an unmet need in patients who have few treatment options.”
BMS remains optimistic about the study’s results and plans to discuss marketing applications with regulatory authorities, including the US Food and Drug Administration (FDA) and European Medicines Agency (EMA).
According to the company, Reblozyl is a standard of care for certain types of anaemia in adults, including myelodysplastic syndromes and beta thalassemia requiring RBC transfusions.
BMS noted that the therapy is being developed and commercialised in a worldwide partnership with MSD following MSD’s acquisition of Acceleron Pharma, which included Reblozyl in its portfolio.
In September 2021, MSD announced the acquisition of Acceleron Pharma for approximately $11.5bn, which was completed in November 2021. This acquisition has expanded MSD’s portfolio, which now includes Reblozyl.
