Lirilumab, the leukemia drug developed by Bristol-Myers Squibb Co. (BMY) and Innate Pharma, failed a mid-stage trial. (See also: Roche, Bristol-Myers Battle for Cancer Drug Dominance.)

Lirilumab failed to beat a placebo in improving patients' leukemia-free survival. A placebo is a substance that has no therapeutic effect, and is used as a control in testing new drugs.

Around 150 acute myeloid leukemia (AML) patients in the trial received lirilumab or a placebo. The results showed there was no statistically significant difference identified between lirilumab and a placebo for any of the trial endpoints.

Second Failed Trial

This was the second failed attempt for the AML drug study. In 2015, the Data and Safety Monitoring Board had recommended that the higher dose arm of lirilumab should be discontinued after it failed to beat a placebo.

Despite the trial failures, lirilumab remains a key drug candidate for Bristol-Myers and Innate Pharma. It is being investigated in six different trials sponsored by Bristol-Myers Squibb, for treating a wide range of solid and hematological cancer indications in combination with other agents, including Bristol-Myers’ bestselling drug Opdivo (nivolumab). (For more, see Bristol-Myers' Opdivo Cleared for Bladder Cancer.)

Lirilumab Tested In Combo Studies

Innate Pharma is positive on interim results from Phase 1/2 studies indicating that the combination of Opdivo and lirilumab is effective in treating squamous cell carcinoma of the head and neck. The partner companies believe that combination has the potential to help generate and spread the necessary T-cells and natural killer cells to fight cancer.

Bristol-Myers paid $35 million (around €33 million) to Innate Pharma to securre the rights to lirilumab for various combination studies. Innate also received $15 million last month from Bristol-Myers for continued exploration of lirilumab in combination with Opdivo. (See also, Assessing Bristol-Myers Post-Earnings Valuation.)

Acute myeloid leukemia (AML) is a cancer of the blood and bone marrow that affects the growth of immature blood cells, called myeloid cells. Myeloid cells interfere with the production of normal white blood cells, red blood cells, and platelets, leading to fatigue, fever, recurrent infections and bruising of the body. The disease spreads rapidly across the human body as it is a blood cancer.

Want to learn how to invest?

Get a free 10 week email series that will teach you how to start investing.

Delivered twice a week, straight to your inbox.