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Flex Pharma to stop two neuromuscular disease trials over safety concerns

PBR Staff Writer Published 14 June 2018

Flex Pharma said that it is scrapping two ongoing phase 2 neuromuscular disease trials, evaluating its lead drug candidate FLX-787, due to safety concerns.

The Boston-based clinical-stage biotechnology company had been conducting the mid-stage trials for the oral disintegrating tablet formulation in amyotrophic lateral sclerosis (ALS) and Charcot-Marie-Tooth (CMT).

Flex Pharma said that the two trials are being stopped because of oral tolerability concerns observed across both of them in a subset of patients treated thrice a day with 30mg of FLX-787.

The company said that it will continue to evaluate the potential of FLX-787 in dysphagia, a condition in which a person experiences difficulty in swallowing.

Due to the clinical trial failures, Flex Pharma has decided to trim down its workforce by nearly 60%, in a restructuring move to bring down its costs. The changes are expected to be executed by the month end.

Flex Pharma president and CEO Bill McVicar said: "In the past few months we have reported positive efficacy data in two serious and distinctly different neurological diseases: multiple sclerosis (MS) and ALS. We believe that these clinical data demonstrate the clear potential of FLX-787 as a symptomatic therapy to reduce painful cramps and spasms in these patient populations.

“However, recent observations of oral intolerability at the current dose and formulation, in a subset of patients, in both studies, indicate that more formulation and dose-ranging studies are required, which is challenging for the Company based upon our current resources.”

Co-founded by Rod MacKinnon, a 2003 Nobel Laureate, Flex Pharma has been developing FLX-787 under Fast Track designation for the treatment of severe muscle cramps associated with ALS.

In late March, the company reported positive results for FLX-787 from an exploratory Phase 2 trial in 57 MS patients with frequent muscle cramps/spasms and spasticity.

At a dose of 19mg, FLX-787 taken orally daily twice could register statistically significant reduction by 27.3% in the frequency of cramps/spasms compared with control in the mid-stage trial.

Image: Safety concerns force Flex Pharma to end two mid-stage trials of FLX-787. Photo: courtesy of jk1991/