Zafgen, Inc., a clinical-stage biopharmaceutical company developing novel therapies for patients affected by a range of metabolic diseases, received a letter last week from the U.S. Food and Drug Administration (FDA) placing a clinical hold on the Investigational New Drug Application (IND) for its first U.S. trial of ZGN-1061, the company’s second-generation, investigational MetAP2 inhibitor currently in development for the treatment of type 2 diabetes.
The FDA cited the possibility of cardiovascular safety risk based on the company’s prior compound and outlined multiple potential paths for moving forward, including nonclinical or clinical options, to address these concerns in the ongoing development of ZGN-1061. The company plans to assess the options and request a Type A meeting with the agency to discuss next steps with the program.
Zafgen continues to advance its ongoing ex-U.S. Phase 2 clinical trial of ZGN-1061, which includes a 1.8 mg dose cohort. Dosing in this clinical trial was recently completed and, while still blinded, no cardiovascular safety signals have been observed to date. The company remains on track to report topline data from this cohort in early 2019.
The company previously reported positive full 12-week results for its initial cohort of this Phase 2 proof-of-concept clinical trial, which included a range of doses up to 0.9 mg. In that initial cohort, ZGN-1061 met all primary endpoints, demonstrating proof-of-concept efficacy with robust A1C lowering effects, and a favorable safety and tolerability profile generally comparable to placebo, with no treatment-related serious adverse events and no cardiovascular safety signals observed.
With the delay of additional clinical development for ZGN-1061, Zafgen reported it now expects the company’s cash runway will extend through calendar year 2020, with sufficient capital through multiple milestones.
(Source: Zafgen, Inc.)