Our Science & Approach

Zafgen has undertaken years of research to characterize the MetAP2 pathway and its effects in various bodily systems. We are focused on leveraging our proprietary understanding of this pathway to develop transformative therapeutics that treat the underlying biological mechanisms of a variety of metabolic diseases. Our approach has the potential to significantly improve the health and well-being of patients affected by a range of both rare and more prevalent metabolic disorders where MetAP2 plays a central role.

The Science & Promise of MetAP2 Inhibition

OUR PROGRAMS & PIPELINE

Zafgen’s approach builds off a growing body of scientific evidence showing that patients affected by complex metabolic diseases metabolize fat very differently from healthy people.

With our proprietary MetAP2 development platform, advanced chemistry insights and understanding of the unique characteristics of rare and more common complex metabolic disorders, Zafgen has developed several MetAP2 inhibitors, which modulate the cellular mechanisms that control the body’s ability to make and store fat and to use fat and glucose as an energy source.

The small molecule MetAP2 inhibitors now in our pipeline have each been optimized to preferentially target specific tissue systems relevant to the disease being pursued and to minimize exposure elsewhere in the body, which we anticipate will result in clinically differentiated efficacy and safety.

PRECLINICAL DEVELOPMENT
PHASE 1
PHASE 2
PHASE 3
ZGN-1061 for type 2 diabetes
PHASE 2

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.

ZGN-1258 for Prader-Willi syndrome
PRECLINICAL DEVELOPMENT

Zafgen is initiating 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. There are currently no approved medications to aid in controlling hyperphagia.

Liver-directed candidate
PRECLINICAL DEVELOPMENT

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.

PUBLICATIONS & POSTERS

Liver-directed Candidate

poster:
Methionine Aminopeptidase 2 Inhibition Attenuates Liver Pathology in Mouse Model of NASH American Association for The Study of Liver Diseases, October 2017, Poster 2054
VIEW All Liver-directed Candidate Publications & Posters

Patient Stories

Patients and their families inspire our work.

Learn more about Marc’s story