US-based biotechnology firm Flex Pharma has started a Phase II clinical trial (COMMIT) of FLX-787 under an open investigational new drug (IND) application to treat Charcot-Marie-Tooth (CMT) disease in the country.
FLX-787 is a co-activator of TRPA1 and TRPV1 obtained fast-track designation from the US Food and Drug Administration (FDA) for severe muscle cramps caused by amyotrophic lateral sclerosis (ALS).
The randomised, controlled, double-blinded, parallel design Phase II COMMIT trial will assess 30mg FLX-787 given thrice daily for 28 days in patients suffering from painful, debilitating cramps due to CMT.
Flex Pharma president and CEO Dr William McVicar said: “As one of the very few companies conducting interventional studies in CMT, we are excited to be at the forefront of clinical development for this disease, an orphan indication that is the most common form of inherited neuromuscular disease.
“These new Phase II IND studies, as well as completion of the ongoing exploratory Phase II work in Australia, are expected to result in several important data readouts in 2018.”
According to COMMIT trial lead investigator and University of Utah Neurology, Paediatrics and Pathology assistant professor Dr Nicholas Johnson, the safety profile and non-systemic exposure of FLX-787 could have potential clinical advantages.
With changes in cramp frequency measured as the primary endpoint, top-line results from the Phase II trial are expected to be available in the middle of next year.
Earlier this year, Flex Pharma launched a Phase II clinical trial of FLX-787 for the treatment of ALS.