Bristol Myers' leukemia hopeful snags speedy FDA review

horse race
Bristol Myers Squibb's leukemia treatment extended patients’ lives a median of two years (24.7 months) versus 14.8 months for those on placebo. (Getty)

Exactly one month after filing its multiple myeloma CAR-T for FDA approval, Bristol Myers Squibb is at it again. It’s nabbed priority review status for another blood cancer program it picked up from Celgene: a leukemia treatment aimed at keeping patients in remission. The FDA is set to make a decision by Sept. 3.

The filing is based on phase 3 data revealed in December showing the drug, CC-486, beat placebo at extending the lives of patients with acute myeloid leukemia (AML). The study pitted CC-486 against placebo in 472 patients whose cancer had disappeared after front-line treatment but who could not receive stem cell therapy—or chose not to. Some of those patients whose cancer disappeared had lower-than-normal levels of some blood cells, which is linked to worse outcomes than those who achieve complete remission.

The drug extended patients’ lives a median of two years (24.7 months) versus 14.8 months for those on placebo. It also staved off relapse for twice as long as placebo—patients taking CC-486 stayed in remission for a median 10.2 months compared to 4.8 months for patients on placebo. The results were consistent across the treatment group, including the patients with low levels of some blood cells.

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The most common side effects of the drug were nausea, vomiting and diarrhea. Patients taking CC-486 and placebo experienced low blood platelet counts and anemia at roughly the same rate, while more patients taking the drug had neutropenia, or a low white blood cell count. Patients stayed on treatment until “unacceptable toxicity” or their cancer got worse, with 13% of CC-486 patients and 4% of placebo patients quitting the study because of side effects.

Adults with newly diagnosed AML often beat back their cancer with their first-line treatment, but many patients will relapse, said Noah Berkowitz, M.D., Ph.D., who leads global clinical development at Bristol Myers’ hematology unit, in a statement.

“Patients in remission are seeking treatment options that decrease the likelihood of relapse and extend overall survival,” he said.  

Bristol Myers’ $74 billion bet on Celgene keeps on delivering. After two years without a new drug approval, BMS got two in 2019 for hematology assets picked up in that deal: myelofibrosis drug Inrebic and Reblozyl, which treats anemia in patients with beta thalassemia. And just last month, the company filed its anti-BCMA CAR-T therapy with the FDA, the third of a trio of assets that could trigger a major payout to Celgene investors.

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