Programs & Pipeline

Our pipeline is focused on complex metabolic conditions where the MetAP2 pathway plays a central role.


Type 2 diabetes represents one of the most important health challenges today, affecting 425 million people worldwide, and almost 10% of U.S. population. In the U.S., type 2 diabetes resulted in $727 billion in direct healthcare costs in 2015, and over $1 trillion in indirect costs.

ZGN-1061 is being developed for patients with difficult-to-control type 2 diabetes, with the potential to deliver clinical superiority compared with insulin. Our Phase 2 proof-of-concept trial is fully enrolled.


Prader-Willi syndrome (PWS) is a rare, genetic form of life-threatening obesity characterized by unrelenting pathologic hunger (hyperphagia) leading to dangerous food-seeking behavior. People with PWS are also impacted by slowed metabolism, psychiatric conditions and higher risk for cardiopulmonary and metabolic co-morbidities. Our best estimate of prevalence is 1:40,000 people. People with PWS have a shortened life expectancy of approximately 32 years. There is currently no approved treatment for hyperphagia or obesity in people with PWS.

Zafgen is beginning investigational new drug (IND) application-enabling work for ZGN-1258 in preparation for filing an IND with the U.S. Food and Drug Administration (FDA) and beginning Phase 1 clinical development. ZGN-1258 is designed to change the way the body metabolizes fat, reduce fat mass, and decrease hyperphagia, making it of great interest for the treatment of PWS and other severe forms of obesity.

Liver-specific candidates

We are further leveraging the MetAP2 pathway to address multiple liver diseases, such as nonalcoholic steatohepatitis (NASH). We are working toward nominating a lead liver-specific development candidate and initiate IND-enabling activities.