US-based biotechnology company Flex Pharma has begun a Phase ll efficacy trial of FLX-787 to treat amyotrophic lateral sclerosis (ALS) patients in Australia.
FLX-787 is a chemically synthesised, single molecule, transient receptor potential (TRP) ion channel activator that has been formulated as an oral tablet.
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The randomised, controlled, blinded, cross-over Phase ll trial has been designed to assess the safety and efficacy of FLX-787 in patients who suffer from cramps and / or spasticity as a consequence of ALS.
Flex Pharma chief medical officer Thomas Wessel said: “We have initiated three human efficacy studies this year, in nocturnal leg cramps, multiple sclerosis and now ALS, with our single agent candidate, FLX-787.
“We are hopeful that our efforts will ultimately help the many patients with severe neuromuscular diseases who suffer from these painful conditions.”
ALS is a neurological disease that leads to muscle weakness and impacts physical function.
The disease can ultimately affect the ability to control the muscles required to move, speak, eat and breathe.
According to a report by Centres for Disease Control, more than 12,000 people in the US suffer from ALS.
Flex Pharma scientific co-founder Dr Rod MacKinnon said: “Topical Chemical Neuro Stimulation is a process whereby small molecules activate TRP ion channels on sensory nerves in the oral mucosa, which we hypothesise signal through neural circuits to inhibit the hyperexcitable motor neurons in the spinal cord that can cause muscle cramps.
“We hypothesise that this approach may be generally applicable as a treatment for cramps and spasms in a spectrum of neuromuscular conditions.”
The company is currently involved in the development of new and proprietary treatments for nocturnal leg cramps, cramps and spasms related with severe neuromuscular conditions including ALS and MS, as well as exercise-associated muscle cramps.
