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|Flex Pharma Provides FLX-787 Nocturnal Leg Cramp Regulatory and Clinical Update|
-- Efficacy Signals Seen in Subanalyses of Exploratory Human Studies of FLX-787 --
-- Parallel Design Phase 2 Trial Planned for First Half 2017 Based upon FDA Pre-IND Responses --
-- Conference Call and Webcast today at
Update on Exploratory, Proof-of-Concept Human studies:
1. Exploratory NLC Study of FLX-787 oral disintegrating tablet (ODT)
In this randomized, blinded, controlled, crossover study, 72 subjects (40-79 years of age) who reported to suffer from nocturnal leg cramps at least four nights per week were enrolled at three clinical sites. After an initial two-week placebo run-in period, subjects were randomized to either 17mg ODT FLX-787 or ODT placebo for three weeks. Subjects were then crossed over to the other treatment for an additional three weeks.
Although preliminary analysis of the entire crossover data set did not demonstrate a statistically significant difference versus placebo on the pre-specified endpoints of muscle cramp frequency or cramp-free nights, a number of concerns have been identified that potentially impact data interpretation at one of the sites. When data from this site are excluded and analysis is restricted to patients from the two other sites (n=37), FLX-787 shows a strong trend on muscle cramp frequency (p=0.06) during the initial two-week parallel design of the study versus placebo as compared to baseline run-in period, despite the limited data set not being adequately powered to show statistical significance. We continue to analyze the data between the sites to determine which of the issues, if any, may be meaningful. We believe that FLX-787 was well-tolerated, with no serious adverse events reported.
2. Exploratory, sequential, multiple crossover NLC Study of FLX-787 formulations
In order to help inform the optimal dose and design of the Phase 2 clinical trial expected to begin next year, the Company also conducted a sequential, multiple crossover study to generate safety and efficacy data in subjects exposed to different formulations and dosages of FLX-787. The 29 subjects in this study had participated in the prior NLC crossover study with the Company’s extract formulation. In this study of FLX-787, the subjects received liquid or ODT formulations of FLX-787 and matched placebos, in four rapidly successive crossover periods.
Muscle cramp frequency was reduced (p<0.05) at two weeks in the parallel
portion of the first phase which tested 19 mg of FLX-787 in liquid
formulation versus placebo. We believe this human efficacy data further
supports the use of a parallel design in future studies, consistent with
3. Human Dose-Ranging Efficacy Study in Electrically-Induced Cramps
In recent months, the Company has continued to evaluate FLX-787 using its electrically-induced cramp model. In a study of five subjects, an ODT formulation of FLX-787 reduced the intensity and duration of electrically-induced muscle cramps in a dose-dependent manner (p<0.05). Seven doses (0.5 , 2.5, 6, 10, 18, 32, and 60 mg) of FLX-787, representing more than a 100-fold range, showed an effect consistent with a classic sigmoidal dose response curve, with virtually no effect at the lowest doses and a maximal effect at the highest doses.
“We believe that the data sets reported here, which include efficacy
signals from two exploratory NLC human efficacy studies and a clear
dose-response curve in our electrically-induced human cramp model,
establish the positive effects of FLX-787 on human muscle cramping,”
“Having studied repeated dosing of FLX-787 in over 100 human subjects,
we have an unusually extensive human safety and efficacy experience for
an agent at this stage of development. We look forward to advancing our
drug development efforts,” said Dr.
Data from the human studies outlined above will be presented at future medical meetings.
Conference Call Information
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 design, 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; 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
Flex Pharma, Inc.