FDA had halted work on the study drug after 2 deaths in an unrelated trial.
Clinical results for an experimental drug showed significant weight loss for patients with type 2 diabetes, which may have been enough to lift stock prices for its manufacturer Zafgen. But for now, beloranib remains on hold with the FDA for its target population.
Regulators put beloranib on hold in December after 2 patients with the rare Prader-Willi Syndrome died after taking the drug in the study. Zafgen is now working on a “risk mitigation” plan to watch those who might develop blood clots, which caused the deaths.
Data released today involved a separate study of 152 patients, which found that severely obese patients with T2D—and do not have the syndrome—lost significant amounts of body weight. Patients taking 1.8 mg and 1.2 mg doses respectively lost 12.7% and 13.5% of their body weight. Patients taking a placebo lost 3.1% of their weight.
The company also reported that patients taking the study drug had absolute reduction in glycated hemoglobin of 2.0% compared with 0.6% for placebo.
“These results reinforce the strong efficacy profile of beloranib and further inform the potential of our MetAP2 inhibitor platform to impact metabolic disorders,” said Thomas Hughes, PhD, CEO of Zafgen in a statement.
Studies have shown that patients with T2D who lose as little as 5% of their weight can experience significant cardiovascular benefits, and even 3% weight loss can be a worthy goal.
In response to the news, Zafgen’s shares rose 12%, amid reports that the new data might convince FDA to restart testing.
Drugs to produce significant weight loss are much sought after, but several have faced safety concerns. Starting in the mid-2000s, FDA has required all new diabetes and obesity therapies to undergo rigorous testing for long-term cardiovascular effects. In this new trial 9 adverse outcomes were found in 8 patients, including pulmonary embolism and deep vein thrombosis.