- Zafgen got a boost on Thursday when new data for its obesity drug candidate showed substantial weight loss among patients with type 2 diabetes.
- The Boston-based company has been developing beloranib for treatment of binge-eating and obesity in patients with Prader-Willi Syndrome. However, the FDA put a full clinical hold on the drug after two patients with Prader-Willi died from blood clotting during a mid-stage trial.
- This news follows separate positive data released in January from 74 patients who had completed a phase 3 trial before the clinical hold. However, the second death occurred after the patient had gone through the trial's screening.
As Zafgen puts together a risk mitigation strategy to present to the FDA, the new data from its Phase 2b clinical trial showed significant weight loss in obese patients with type 2 diabetes who had taken beloranib. Depending on dosage, patients taking beloranib lost between 12.7% and 13.5% of their body weight. Those taking the placebo lost only 3.1%.
Zafgen plans to submit the data from this trial, the previously released phase 3 results, and a risk-mitigation proposal to the FDA in hopes of lifting the clinical hold on the drug. The patients involved in this Phase 2b trial had completed six months of treatment prior to the hold.
The prevalence of type 2 diabetes, which affects roughly 9% of the population, is much higher than the prevalence of Willi-Prader Syndrome, a rare disease that affects only 1 in 13,000 people. This data could form a foundation upon which to pursue wider indications for beloranib if safety concerns are resolved.
The Department of Health and Human Services (DHHS) has published specific guidance related to unanticipated problems involving risks and adverse events in clinical trials involving human beings. According to the guidance, there are numerous corrective actions that could be taken to effectively address these problems, and therefore, mitigate risk.
Some examples of corrective actions include changes to the research protocol initiated by the investigator prior to obtaining IRB approval to eliminate apparent immediate hazards to subjects; modification of inclusion or exclusion criteria to mitigate the newly identified risks; and implementation of additional procedures for monitoring subjects.
For more information about risk mitigation after a serious adverse event has occurred during a clincal trial, you can check out HHS' guidance here.