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Zafgen shares tumble after FDA places hold on diabetes trial

Regulator concerns based on deaths with previous drug


Shares in US specialist pharma company Zafgen plunged 41% yesterday to $5.41 after the FDA placed its lead candidate on clinical hold.

The Boston-based company specialises in MetAP2 systems biology to develop novel therapies for patients affected by a range of metabolic diseases, and has seen the US regulator raise concern about possible cardiovascular safety risks associated with ZGN-1061, currently in development for type 2 diabetes.

The FDA has halted the drug’s clinical trial development at the preliminary Investigational New Drug Application (IND) stage because of concerns it could have similar problems to Zafgen’s first lead compound, beloranib.

This was abandoned in 2016 after two patients died in a phase 3 trial of the drug after suffering a pulmonary embolism. The drug was being studied to treat obesity in patients with the rare metabolic disease known as Prader-Willi Syndrome.

Like beloranib, ZGN-1061 is an injectable small molecule drug that is supposed to help the body burn more fat by inhibiting the activity of the methionine aminopeptidase 2 (MetAP2) enzyme.

Jeffrey Hatfield

Jeffrey Hatfield

Led by CEO Jeffrey Hatfield, the company says the FDA has outlined “multiple potential paths” that resolve issues including nonclinical or clinical options, and Zafgen says it will meet with the FDA soon to discuss options.

Zafgen already has a phase 2 trial of ZGN-1061 ongoing outside the US, which includes a 1.8 mg dose cohort. It says dosing in this clinical trial was recently completed and, while still blinded, no CV safety signals have been observed to date.

The company still aims to report top line data from this cohort in early 2019, and has already reported encouraging 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 favourable safety and tolerability profile generally comparable to placebo. The company adds that no treatment-related serious adverse events and no cardiovascular safety signals were observed.

Zafgen came close to running out of cash when it was forced to abandon beloranib, and had to drastically scale back its operations and staff. This time the company says the clinical hold will eat into its cash reserves, but says it has enough to keep it going until 2020.

Article by
Andrew McConaghie

27th November 2018

From: Regulatory



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