Flex Pharma, Inc. (NASDAQ:FLKS), a clinical-stage biotechnology company that is developing innovative and proprietary treatments for cramps and spasticity associated with severe neurological diseases including amyotrophic lateral sclerosis (ALS), multiple sclerosis (MS) and Charcot-Marie-Tooth (CMT), today announced that the Inherited Neuropathies Consortium (INC) voted to endorse the Company’s U.S. Phase 2 Trial of FLX-787 in CMT patients who suffer from cramps. The INC is an integrated group of academic medical centers, patient support organizations, and clinical research resources dedicated to conducting clinical research in CMT and to improving the care of patients (www.rarediseasesnetwork.org/cms/inc). There are currently no drug products approved in the U.S. for this condition. The Company expects to begin enrolling U.S. patients this summer in this randomized, controlled, double-blinded, parallel design study, referred to as the COMMIT trial. FLX-787 is a small molecule co-activator of the TRPA1 and TRPV1 ion channels, and has been shown to suppress muscle cramps in a human model of electrically induced cramps, and to reduce cramping frequency and the associated pain in spontaneously occurring cramps in a population with nocturnal leg cramps.
“The Inherited Neuropathies Consortium welcomes this clinical research effort to develop a novel treatment for muscle cramps in patients with CMT neuropathy. Cramping is a very common and often disabling symptom of this disease,” remarked Dr. Michael Shy, an internationally known neuropathy researcher at the University of Iowa, which is the INC’s operational center. “We have reviewed the protocol and our organization will reach out to patients with muscle cramps to encourage their participation in this interesting study.”
“Muscle cramping can have a significant impact on our patients with CMT neuropathy, and we have compelling data in pediatric and adult populations showing the deterioration in quality of life that many of our patients experience due to frequent cramping,” stated CMT clinical trial lead investigator, Dr. Nicholas Johnson, Assistant Professor of Neurology, Pediatrics and Pathology at the University of Utah. “FLX-787 could have meaningful clinical advantages because of its safety profile and the lack of systemic exposure.”
“We are thrilled to soon be starting our second US Phase 2 trial with FLX-787. This study in CMT neuropathy is our second orphan indication in neurology,” said Flex Pharma Chief Medical Officer Thomas Wessel, M.D., Ph.D. “With two Phase 2 clinical trials in ALS and CMT expected to initiate this summer in the US, together with our ongoing exploratory Phase 2 studies in Australia, FLX-787 will be amongst the most advanced, novel compounds in the clinic for ALS and CMT.”
“We are excited that many INC study sites in the US will participate in our Phase 2 trial with FLX-787 which should speed recruitment,” commented Bill McVicar, Flex Pharma President of Research & Development. “The R&D team is focused on the execution of these new Phase 2 IND studies, as well as completion of the ongoing exploratory Phase 2 work in Australia, which are expected to result in several important data readouts in 2018.”
COMMIT Trial Design
The COMMIT trial is a Phase 2 clinical trial designed to evaluate FLX-787 in patients with CMT who suffer from cramps. This randomized, controlled, double-blinded, parallel design trial in the U.S. will include a run-in period to establish a baseline in cramp frequency. Patients will then be randomized to 30 mg of FLX-787 administered three times a day or control, for 28 days. Patients will be evaluated for changes in cramp frequency as the primary endpoint, with a number of secondary endpoints.
About Flex Pharma
Flex Pharma, Inc. is a clinical-stage biotechnology company that is developing innovative and proprietary treatments for cramps and spasticity associated with the severe neurological diseases including ALS, MS and Charcot-Marie-Tooth (CMT). 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.
This press release contains forward-looking statements for purposes of the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. Forward-looking statements include statements regarding our intentions, beliefs, projections, outlook, analyses or current expectations concerning, among other things, the design and timing of our anticipated clinical trials and expectations regarding the pace of enrollment in our study of patients with CMT. 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 interpretation of our clinical studies; the uncertainties inherent in conducting clinical studies; our ability to enroll patients in each of clinical studies on a timely basis; expectations of our ability to make regulatory filings and obtain and maintain regulatory approvals; availability of funding sufficient for our foreseeable and unforeseeable operating expenses and capital expenditure requirements; 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, 2016 and subsequent filings with the Securities and Exchange Commission (SEC). You are encouraged to read our 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.