Flex Pharma starts phase 2 efficacy study in multiple sclerosis

The randomized, controlled, blinded, cross-over study is designed to evaluate the safety and efficacy of FLX-787, the Company’s single molecule, chemically synthesized, transient receptor potential (TRP) ion channel activator, in approximately 50 patients who suffer from cramps, spasms and/or spasticity as a consequence of MS.

MS is an autoimmune disease in which inflammatory processes cause worsening demyelination and cell degeneration over years, resulting in a variety of neurological deficits such as loss of muscle control, sensation and vision. Spasticity is caused by damage to motor systems in the brain and spinal cord.

These lesions cause hyperactive muscle stretch reflexes, which result in spasticity. The need to treat spasticity increases as the disease progresses. According to the National Institute of Neurological Disorders and Stroke, between 250,000 and 350,000 people in the United States suffer from MS and approximately 84% of patients with MS experience spasticity.

"Neurologists have very limited therapeutic options for patients suffering from MS spasticity and the current options are often suboptimal due to their associated side effects," stated Al Sandrock, M.D., Ph.D., Biogen Chief Medical Officer and member of the Flex Pharma Scientific Advisory Board.

"The approach by Flex Pharma offers a potential important advantage as the drug candidate may reduce cramps and spasms without the sedating side effects of many current therapies."

"FLX-787 appears to have virtually no systemic exposure, thus potentially avoiding drug-drug interactions for patients on several prescription products," said Flex Pharma Chief Medical Officer Thomas Wessel, M.D., Ph.D., who served as the medical lead for three products approved in United States: Razadyne, Lunesta and Ampyra.

"We believe that Chemical Neuro Stimulation, the process whereby small molecules activate TRP ion channels topically, leads to sensory stimulation that in turn reduces hyperexcitability in motor neurons," noted Dr. Rod MacKinnon, Nobel laureate and Flex Pharma Scientific Co-Founder, Board Member, and Scientific Advisory Board Co-Chair.

"We hypothesize that this approach may be generally applicable as a treatment for cramps and spasms in a spectrum of neuromuscular conditions."

"We are encouraged by our recent positive results with the original extract formulation in nocturnal leg cramps (NLC), and we expect to have three human efficacy studies – in MS, ALS and NLC – initiated this year with our single agent candidate, FLX-787," said Laura Rosen, M.D., Ph.D., Flex Pharma Vice President, Clinical Research & Drug Safety. "I hope our efforts will ultimately help the many patients with severe neuromuscular diseases who suffer from these painful conditions."