Clinical-stage biotech Zafgen, Inc. is focused on rare diseases, selecting ZGN-1258 for the treatment of rare or orphan metabolic diseases, which includes Prader-Willi syndrome.
Zafgen is expected to begin a study on Prader-Willi syndrome in mid-2018 to add to the limited information available on the medical and clinical history of people with the disorder. Work is in progress to support an investigational new drug application with a Phase 1 trial planned by the end of 2018.
Zafgen’s previous lead drug, beloranib, showed promising result in treatment of Prader-Willi syndrome. It is a rare genetic disease where a permanent feeling of hunger leads to potentially dangerous obesity. However, in 2015, two patient deaths caused by blood clots in a Phase 3 trial led to a hold on clinical trials from the Food and Drug Administration.
The principle behind the compound targeting the rare metabolic disease is to change the mode of fat metabolizing by body leading to reduced hunger associated with the rare disease. This drug has no activity in endothelial cells, owing to which it is expected to reduce the risk of blood clots. By mid-2016, Zafgen shifted its focus to a new lead drug, ZGN-1061, for severe and complicated obesity.
According to Rare Disease Drugs Market report published by Coherent Market Insights, Rare diseases or orphan diseases are conditions that affect a small percentage of population at any given time. According to the FDA, 2017, number of orphan drug designation requests have steadily increased over the past few years. Zafgen has also pushed forward its lead MetAP2 inhibitor, ZGN-1061, a targeted drug candidate in Phase 2 for type 2 diabetes. The study aims at metabolic measures and cardiovascular risk factors including weight.