- Clinical-stage biotech Zafgen, Inc. is looking again at rare diseases, selecting ZGN-1258 for the treatment of rare or orphan metabolic diseases, including Prader-Willi syndrome, sending its shares up around 8% on Friday.
- Work to support an investigational new drug application is under way, with a Phase 1 trial planned by the end of 2018.
- Zafgen will also begin a global Prader-Willi syndrome natural history study mid-2018, to add to the limited information available on the medical and clinical history of people with the disorder.
Since a challenging time with its previous lead drug, beloranib, Zafgen, Inc. is attempting a turnaround. The company has a new CEO and Jeffrey Hatfield, a new CSO, Thomas Hughes, the current president and previous CEO. It also has a $20 million non-dilutive venture debt financing agreement with Silicon Valley Bank, extending its expected cash runway beyond the first half of 2019.
Zafgen's previous lead drug, beloranib, showed promise in Prader-Willi syndrome, 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 complete hold on clinical trials from the Food and Drug Administration. By mid-2016, Zafgen had ceased development, cut its workforce by a third, and shifted its focus to a new lead drug, ZGN-1061, for severe and complicated obesity.
The premise behind the compound targeting the rare metabolic disease is that it changes the way the body metabolizes fat and therefore reduces fat mass, as well as reducing the unrelenting hunger (hyperphagia) associated with the rare disease. Because it has no activity in endothelial cells, it is hoped that this will reduce the risk of blood clots.
Zafgen has also pushed forward its lead MetAP2 inhibitor, ZGN-1061, a targeted drug candidate in Phase 2 for type 2 diabetes. Enrollment is complete for this study in patients who are also obese, with 137 patients.
That's 14% above its target of 120 patients. The study will look at glycemic control, safety and tolerability, as well as metabolic measures and cardiovascular risk factors including weight. Topline results are expected mid-year 2018.