Flex Pharma said it plans to prioritize developing its clinical programs in neurological diseases, including amyotrophic lateral sclerosis (ALS), multiple sclerosis (MS), and peripheral neuropathies like Charcot-Marie Tooth (CMT).
One or two proof-of-concept, Phase 2 clinical trials are planned for this year in the United States to evaluate the company’s transient receptor potential (TRP) ion channel activator, FLX-787. The drug is capable of interfering with TRPV1 and TRPA1 receptors involved in pain and neurogenic inflammation, the company reported.
Flex Pharma is currently conducting a randomized, placebo-controlled, cross-over Phase 2 clinical trial in Australia evaluating FLX-787’s safety and efficacy in ALS patients with leg cramps and spasticity.
FLX-787 is also being evaluated in an exploratory Phase 1 clinical trial in MS patients with cramps, spasms, and/or spasticity. Results are expected by the end of the year.
Spasticity refers to feelings of stiffness and involuntary muscle spasms. It can vary from mild discomfort to severe, painful, uncontrollable spasms, usually in the legs. Spasticity can be aggravated by sudden movements, temperature changes, infections, or tight clothing. Left untreated, it can lead to serious complications, including contractures (frozen or immobilized joints) and pressure sores.
“By prioritizing our clinical programs to severe neurological diseases ahead of nocturnal leg cramps, we can focus on those patients with the greatest unmet need and accelerate our research efforts for cramps and spasticity,” Thomas Wessel, MD, PhD, Flex Pharma’s chief medical officer, said in a press release. “In 2017, we expect to have multiple Phase 2 studies ongoing with our single agent candidate, FLX-787, with results from our MS study towards the end of the year.”
Flex researchers said FLX-787 has shown an ability to significantly reduce human muscle cramp intensity in a dose-response way, with higher drug concentrations demonstrating superior inhibitory effect.
“We believe that topical Chemical Neuro Stimulation, the process whereby small molecules activate TRP ion channels on sensory nerves in the oral mucosa, triggers a reflex response to inhibit the hyper excitable motor neurons in the spinal cord and reduce muscle cramps,” said Rod MacKinnon, MD, Flex Pharma co-founder and a Nobel Prize winner. “Our approach has a broad range of applications that we believe will ultimately benefit the millions of people that suffer from frequent painful muscle cramps.”