-- 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 8:45
a.m. ET --
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Flex Pharma, Inc. (NASDAQ: FLKS) today provided a nocturnal leg
cramp (NLC) regulatory and clinical update for FLX-787, a
topically-acting, selective transient receptor potential (TRP) ion
channel agonist, which the Company is evaluating for the treatment
of nocturnal leg cramps (NLC), multiple sclerosis (MS), amyotrophic
lateral sclerosis (ALS), and other disorders.
- In written responses to the Company’s
pre-IND meeting request received in September 2016, FDA indicated
that cramp frequency “could be an acceptable primary efficacy
endpoint.” FDA also recommended that the Company utilize a parallel
design in a planned IND-opening study due to the potential for
confounding clinical results caused by carry-over effects,
unblinding and other concerns with crossover studies.
- While statistically significant results
were seen in some, but not all, of the crossover endpoints, FLX-787
has shown positive signals on muscle cramping in the parallel
design portion of two exploratory human proof-of-concept NLC
studies. Data from these exploratory studies and a sigmoidal
dose-response curve in a human electrically-induced cramp study
warrant further evaluation and development of FLX-787 in subjects
with NLC.
- The Company is planning a parallel
design Phase 2 study in NLC to be initiated in the first half of
2017, after the IND application has been submitted and
accepted.
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 FDA recommendations. In the
crossover data sets, efficacy (p<0.05) was generally seen for
the pre-specified endpoints of muscle cramp frequency and cramp
free nights in the early study arms. In the latter arms, FLX-787
did not show statistical significance versus placebo, which we
believe resulted from a potential carryover effect.
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,” said Flex Pharma Chief Medical Officer Thomas
Wessel, M.D., Ph.D., who served as the medical lead for three
products approved in the United States: Razadyne®, Lunesta® and
Ampyra®.“ Over the past year we have gained important insights from
these exploratory studies regarding subject characteristics,
clinical endpoints, dosing and formulation that will inform our
human efficacy studies moving forward. We believe the magnitude of
beneficial effect found in these studies, as well as in our
previously reported efficacy study, bode well for our planned Phase
2 study.”
“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.
Christoph Westphal, Flex Pharma CEO.
Data from the human studies outlined above will be presented at
future medical meetings.
Conference Call Information
Flex Pharma will host a conference call and webcast Thursday,
October 13, 2016 beginning at 8:45 a.m. ET. To participate in the
conference call, please dial (855) 780-7202 (domestic) or (631)
485-4874 (international) and refer to conference ID 99423526. The
webcast will be accessible live in the Investors and Media section
of the company's website at www.flex-pharma.com.
About Flex Pharma
Flex Pharma, Inc. is a biotechnology company that is developing
innovative and proprietary treatments for nocturnal leg cramps,
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 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
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.
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version on businesswire.com: http://www.businesswire.com/news/home/20161013005397/en/
Flex Pharma, Inc.Elizabeth Woo, 617-874-1829SVP, Investor
Relations & Corporate Communicationsirdept@flex-pharma.com
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