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Flex Pharma’s Positive Human NLC Study Selected for Presentation at AAN Annual Meeting

-- Statistically Significant Efficacy Demonstrated in Randomized, Controlled, Blinded Nocturnal Leg Cramp Study --

BOSTON--(BUSINESS WIRE)--Apr. 19, 2016-- Click to Tweet

Flex Pharma, Inc. (NASDAQ: FLKS) announced that results from its recently completed study in nocturnal leg cramps (NLC) will be presented later today at the American Academy of Neurology (AAN) 68th Annual Meeting in Vancouver, B.C., Canada. As one of only 14 abstracts selected for late-breaking presentations at the AAN annual meeting, the abstract titled, “Orally-administered TRPV1 and TRPA1 activators reduce Nocturnal Leg Cramps in a randomized, blinded, placebo-controlled, crossover human trial,” will be presented during the Emerging Science Session at 6:21p.m PDT, followed by a poster presentation from 6:30-7:15pm PDT. Flex Pharma is a biotechnology company developing innovative and proprietary treatments for NLC, cramps and spasms associated with severe neuromuscular conditions such as MS and ALS, and exercise-associated muscle cramps.

The Company estimates that NLC affects four million Americans nightly; there is no approved therapeutic in the United States to treat this condition. The randomized, blinded, controlled, crossover study evaluated 50 healthy subjects (50-77 years of age) who experienced Nocturnal Leg Cramps at least four nights per week. After an initial placebo run-in period to remove placebo responders, subjects were randomized to either control or study product (extract formulation) for two weeks. Subjects were then crossed over to the other treatment for another two-week period so that each subject acted as his or her own control.

The extract formulation resulted in a median reduction of 6 total cramps over a 2-week period vs. 4 for placebo (p<0.05) and median increase of 2 total cramp-free nights vs. 1 for placebo (p<0.01). The positive effects were seen across a broad range of enrolled subjects; in addition, a subset of patients showed pronounced benefit. The magnitude of efficacy in this study on reduction in muscle cramps appears similar to published “Class 1 level” quinine efficacy studies. “Class 1 level” efficacy studies are randomized, controlled clinical trials of the intervention with masked or objective outcome assessment, in a representative population.

Quinine, the only therapeutic intervention for leg cramps with randomized, controlled, blinded study support for efficacy, is also associated with serious adverse events and was banned for the treatment of leg cramps by the FDA. In the U.K. where quinine is still available and the population is approximately one-fifth the U.S., 4.5 million prescriptions for quinine were written in 2013. The lack of any approved therapeutics for NLC in the U.S. represents a significant unmet medical need.

Statistically significant effects were also demonstrated on these key endpoints: the physician-rated Clinical Global Impression of Change (CGI-C) (p<0.01); specific sleep disturbance measures (p<0.05); and specific pain measures (p<0.01). Additionally, the product appeared to be safe and well-tolerated and there were no serious adverse events reported.

“The statistically significant human efficacy data generated in this study are highly encouraging and this approach may provide a promising new treatment in the future for the millions of patients suffering from nocturnal leg cramps who currently have no safe and effective therapeutic options,” noted John Winkelman, M.D., Ph.D., Chief of the Sleep Disorders Clinical Research Program at Massachusetts General Hospital and Flex Pharma Scientific Advisory Board member.

“These results support using specific transient receptor potential (TRP) ion channel activation to reduce nocturnal leg cramps,” noted Dr. Rod MacKinnon, Nobel laureate and Flex Pharma Scientific Co-Founder, Board Member, and Scientific Advisory Board Co-Chair. “Chemical Neuro Stimulation is a process whereby small molecules activate TRP ion channels topically, which we hypothesize leads to sensory stimulation that in turn reduces hyperexcitability in motor neurons at multiple levels in the spinal cord.”

“We believe the human efficacy demonstrated in this study are clinically meaningful and hold promise for a new approach in NLC and cramps in neurological disorders,” 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®. “Based upon the consistency of these results, we are moving forward to initiate studies with a single molecule TRP activator in NLC, MS and ALS this year.”

About Flex Pharma

Flex Pharma, Inc. is a biotechnology company that is developing innovative and proprietary treatments for nocturnal leg cramps, cramps and spasms associated with severe neuromuscular conditions such as ALS and MS, and exercise-associated muscle cramps. Flex Pharma was founded by National Academy of Science members Rod MacKinnon, M.D. (2003 Nobel Laureate), and Bruce Bean, Ph.D., recognized leaders in the fields of ion channels and neurobiology, along with Chair and CEO Christoph Westphal, M.D., Ph.D.

Cautionary Note on Forward-Looking Statements

This press release contains forward-looking statements for purposes of the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. We may, in some cases, use terms such as “predicts,” “believes,” “potential,” “proposed,” “continue,” “estimates,” “anticipates,” “expects,” “plans,” “intends,” “may,” “could,” “might,” “will,” “should” or other words that convey uncertainty of future events or outcomes to identify these forward-looking statements. Forward-looking statements include statements regarding our intentions, beliefs, projections, outlook, analyses or current expectations concerning, among other things: our expectations regarding future studies of our current product candidates, including the success and timing of these studies; our beliefs regarding the potential benefits of our current product candidates; and expectations regarding the number of individuals that may suffer from nocturnal leg cramps. These forward-looking statements are based on management’s expectations and assumptions as of the date of this press release and are subject to numerous risks and uncertainties, which could cause actual results to differ materially from those expressed or implied by such statements. These risks and uncertainties include, without limitation: the status, timing, costs, results and interpretations of our clinical studies; the uncertainties inherent in conducting clinical studies, including receiving regulatory approval to conduct these studies; the fact that we rely on third parties to manufacture and conduct the clinical studies of our product candidates, which could delay or limit future development or regulatory approval; results from ongoing and planned preclinical development; expectations of our ability to make regulatory filings and obtain and maintain regulatory approvals; our ability to develop and commercialize our consumer product; anticipated positioning and product attributes of our consumer product; results of early clinical studies as indicative of results of future trials; the inherent uncertainties associated with intellectual property; and other factors discussed in greater detail under the heading “Risk Factors” in our Annual Report on Form 10-K for the year ended December 31, 2015 and subsequent filings with the Securities and Exchange Commission (SEC). You are encouraged to read Flex Pharma’s filings with the SEC, available at www.sec.gov, for a discussion of these and other risks and uncertainties. Any forward-looking statements that we make in this press release speak only as of the date of this press release. We assume no obligation to update our forward-looking statements whether as a result of new information, future events or otherwise, after the date of this press release.

Source: Flex Pharma, Inc.

Flex Pharma, Inc.
Elizabeth Woo, 617-874-1829
SVP, Investor Relations & Corporate Communications
irdept@flex-pharma.com