Zafgen Inc. (NASDAQ: ZFGN), a small biopharmaceutical company specialized in anti-obesity drugs, announced on Wednesday that its treatment for a rare obesity disorder showed effectiveness in reducing body weight.
The company reported that boloranib, a twice-weekly injected drug, caused patients with the genetically based disorder known as Prader-Willi Syndrome, or PWS, to control their extreme eating habits and to lose a significant amount of weight. Prader-Willi Syndrome develops a chronic feeling of hunger that can lead to excessive eating and life-threatening obesity.
The drugmaker developed a double-blind, placebo-controlled study trial evaluating the safety and efficacy of the drug, in patients with Prader-Willi syndrome during a six-month randomized treatment period.
The study randomly assigned 107 patients to receive twice-weekly subcutaneous injections of 2.4 mg or 1.8 mg of beloranib or placebo. 27 patients completed the full 26 weeks of treatment per the trial protocol, and 27 patients completed at least 75% of the randomized treatment period before the trial got suspended in October 2015.
Zafgen said the drug reduced body weight by 9.45% at the higher dose and 8.2% at the lower dose. The treatment also showed a statistically significant reduction in hunger-associated behaviors based on the questionnaire used to study hyperphagia.
“This clear efficacy outcome is a crucial first step in moving discussions forward with the Food and Drug Administration regarding continued development of beloranib. While we take the previously reported adverse events very seriously, we now have the robust data to provide greater perspective on the benefit/risk relationship of beloranib in this high-risk patient population,” Thomas Hughes, Ph.D., Chief Executive Officer of Zafgen said in a statement.
On December 2nd, 2015, the Food and Drug Administration (FDA) put the beloranib investigation trial on hold due to the death of two patients. The first of them died on October due to unterminated reasons. The second patient died 2 months later from bilateral pulmonary emboli, blood clots to the lung.
Dr. Hughes claimed they are currently working to better understand the mechanisms and incidence of underlying thromboembolic disease in PWS, as well as the potential impact of beloranib treatment on thrombosis in order to develop a strategy for risk mitigation patients.
The company hosted an investor conference call on January 2oth, 2016, at 8:30 a.m., Eastern Time, to discuss the trial results in more detail.
Since the announcement, shares of the anti-obesity drug maker, gained as much as 94%. Previously, the shares had fallen 84% since the week before announcing the death of the first patient in October.