Flex Pharma, Inc. (NASDAQ: FLKS), a clinical-stage biotechnology company that is developing innovative and proprietary treatments for cramps and spasticity associated with serious neurological diseases including amyotrophic lateral sclerosis (ALS), multiple sclerosis (MS), and Charcot-Marie-Tooth (CMT), today announced that the US Food and Drug Administration (FDA) has granted Fast Track designation for the development of FLX-787, the Company’s co-activator of TRPA1 and TRPV1, to treat severe muscle cramps in patients with ALS. There are currently no drugs approved in the US for this condition. Fast Track designation is intended to accelerate the clinical development and review of drugs to treat serious conditions that address an unmet medical need.
“We believe the receipt of Fast Track designation from the FDA highlights the serious nature of cramps for patients suffering from ALS and the current lack of safe and effective treatments. The Fast Track designation represents an opportunity for even closer collaboration with FDA and an important catalyst for our development program for FLX-787,” stated William McVicar, Ph.D., Flex Pharma interim President and CEO. “With Phase 2 clinical trials in both ALS and CMT expected to initiate this quarter in the US, FLX-787 will be amongst the most advanced, novel compounds in the clinic for these degenerative neurological diseases. 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 spasticity study in MS in Australia. These studies are expected to yield several important data readouts in 2018.”
“Our COMMEND trial, which is a Phase 2 study of primarily ALS patients in the US, has certain advantages to our ongoing ALS study in Australia, including longer run-in and treatment periods, increased dosing, a parallel design, and of course a much larger population from which patients can be recruited. As a result, we have elected to prioritize and focus our efforts on the COMMEND study and will end the exploratory Australian ALS study early, with roughly a dozen patients,” noted Flex Pharma Chief Medical Officer Thomas Wessel, M.D., Ph.D. “The data from the Australian study will inform our larger COMMEND clinical trial, particularly regarding baseline cramp frequency and intra-subject variability, which could influence sample size calculation and other aspects of trial conduct for this important US study.”
Fast Track designation allows for a more frequent dialogue with the Neurology Division at FDA on the company's drug development plan, data requirements and clinical trial design, throughout the drug development and review process, with the goal of getting important new drugs to patients more rapidly. Through the Fast Track program, a product may be eligible for priority review at the time of a New Drug Application (NDA) filing and may also be eligible to submit completed sections of the NDA on a rolling basis, before the complete application is submitted.
About the FLX-787 Clinical Trial Program
The COMMEND trial is a Phase 2 clinical trial designed to evaluate FLX-787 in patients with motor neuron disease (MND), focused on ALS, who suffer from cramps. The COMMIT trial is a Phase 2 clinical trial designed to evaluate FLX-787 in patients with CMT. These randomized, controlled, double-blinded, parallel design trials in the US 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 of ALS, MS and peripheral neuropathies such as 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 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 ongoing and anticipated clinical trials, including the timing for results of our clinical trials, and our expectations regarding the level of interaction with the FDA and other benefits associated with FLX-787 having been granted Fast Track status. 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.