A trial of the first oral proteasome inhibitor to win Food and Drug Administration approval for multiple myeloma that would have allowed the drug to be used among patients with previously untreated disease who are ineligible to receive bone marrow transplantation has failed, albeit just barely.
Osaka, Japan-based Takeda Pharmaceutical said Tuesday that the Phase III TOURMALINE-MM2 trial of Ninlaro (ixazomib), combined with Bristol-Myers Squibb’s Revlimid (lenalidomide) and the steroid drug dexamethasone, had failed to meet statistical significance despite showing a benefit for the three-drug combination.
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“There is a need for treatment options for transplant-ineligible patients,” said Christopher Arendt, head of Takeda’s oncology therapeutic area unit, in a statement. “We remain committed to advancing the field of multiple myeloma and continue to drive innovation through ongoing research and development.”
Results of the study showed that patients receiving Ninlaro, Revlimid and dexamethasone experienced a median progression-free survival – the amount of time that patients live without their disease worsening – of 35.3 months, while those receiving Revlimid and dexamethasone alone had a median 21.8 months’ PFS. However, despite the ostensibly large difference in months, the results missed statistical significance, with a p-value of 0.073. A p-value of 0.05 or less is considered the threshold for statistical significance, which indicates the likelihood that the result is not due to chance alone.
The results mark the second time in less than a week that a trial of an already approved multiple myeloma drug has failed in a Phase III trial among previously untreated, transplant-ineligible patients. On Monday, BMS said that its Phase III ELOQUENT-1 trial of Empliciti (elotuzumab), Revlimid and dexamethasone did not show an improvement in PFS for the triplet combination over Revlimid and dexamethasone alone.
But unlike Empliciti, a SLAMF7-targeting monoclonal antibody whose sales have not kept up with those of drugs like Revlimid, Ninlaro is a proteasome inhibitor, part of a therapeutic class considered a “backbone” of multiple myeloma treatment that also includes Takeda’s Velcade (bortezomib) and Amgen’s Kyprolis (carfilzomib). Ninlaro received FDA approval in 2015 and is labeled for combination with Revlimid and dexamethasone in multiple myeloma patients who have received at least one prior therapy.
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However, another study of the drug in first-line disease, TOURMALINE-MM4, did meet its primary endpoint. In that study, patients who had received initial therapy but had not undergone stem cell transplantation were then switched to single-agent Ninlaro as a maintenance treatment. The company announced that the drug had improved PFS relative to placebo in November, though it did not disclose the study data.
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