Zynerba Pharmaceuticals Announces Top-Line Results from Phase 2 STAR 1 Trial of ZYN002 in Adult Epilepsy Patients with Focal ...
August 07 2017 - 6:45AM
Zynerba Pharmaceuticals, Inc. (NASDAQ:ZYNE), a clinical-stage
specialty pharmaceutical company dedicated to developing and
commercializing innovative transdermal pharmaceutically-produced
cannabinoid treatments, today announced top-line results from its
double-blind placebo controlled Phase 2 STAR 1
(
Synthetic
Transdermal
C
annabidiol for the T
reatment of
Epilepsy) clinical trial evaluating ZYN002 (cannabidiol [CBD]
gel) in adult epilepsy patients with focal seizures. ZYN002 did not
demonstrate a statistically significant reduction of focal seizures
during the treatment period compared to the baseline period for
either the high or low dose cohorts compared to placebo.
“We are very disappointed that the STAR 1 trial did
not meet its primary endpoint in this patient population,” said
Armando Anido, Chairman and Chief Executive Officer of Zynerba. “We
are continuing to evaluate this study and the ongoing STAR 2 open
label study to determine next steps with ZYN002 in adult epilepsy
patients with focal seizures. I’d like to thank the patients,
coordinators and investigators, as well as the development team at
Zynerba, for their time and energies in conducting this very
important trial.”
Anido continued, “Importantly, today’s results
demonstrated ZYN002 to have a very favorable safety and
tolerability profile, which is an encouraging fact as we look to
develop ZYN002 as a treatment for a wide range of indications. We
are excited that we will present top-line data from our ZYN002 STOP
trial in osteoarthritis soon, followed by top-line data from our
FAB-C study in Fragile X syndrome by the end of September.”
In the double-blind, multi-center STAR 1 trial, 188
patients were randomized to receive (i)195 mg of ZYN002 4.2% CBD
gel every 12 hours, (ii) 97.5 mg of ZYN002 4.2% CBD gel every 12
hours or (iii) placebo gel every 12 hours for 12 weeks. Patients
aged 18 to 71 years old with confirmed refractory epilepsy with
focal seizures with or without secondary generalization were
enrolled in this study. Enrolled patients had a median monthly
seizure frequency of 10.6, and were on an average of 2.5
anti-epileptic drugs (AEDs). The primary endpoint assessed the
median percentage change in seizure frequency over the 12-week
treatment period compared to the 8-week baseline
period. Secondary endpoints included proportion of patients
with ≥50% reduction from baseline in seizure frequency, percent
change from baseline in seizure frequency, change from baseline in
seizure frequency, seizure-free days, and 100% seizure free. Safety
and tolerability were also evaluated. The study was conducted at 14
sites in Australia and New Zealand. The efficacy analysis
included 186 patients.
- Primary Endpoint Data: Patients on the
low dose of ZYN002 (n=63) achieved an 18.4% median reduction in
focal seizures during the treatment period compared to baseline;
patients on the high dose of ZYN002 (n=62) achieved a 14.0% median
reduction in focal seizures during the treatment period compared to
baseline; and patients on placebo (n=63) achieved an 8.7% median
reduction in focal seizures during the treatment period compared to
baseline.
- Secondary Endpoint Data: None of the
secondary endpoints showed statistically significant differences
between ZYN002 and placebo.
- Safety Data: ZYN002 was shown to be very
well tolerated and the safety profile was consistent with
previously released data from the Phase 1 trials. Of the 188
patients in the safety database, 50% of the patients on ZYN002
(n=63) had at least one treatment emergent adverse event, compared
to 41% (n=26) of patients on placebo. Two treatment emergent
adverse events occurred in greater than 5% of the patients on
ZYN002: fatigue (5.6%, placebo, 1.6%) and headache (5.6%, placebo,
3.2%). There were no treatment related serious adverse events
reported. The discontinuation rate in the trial for ZYN002 was
10.4% compared to 1.6% in placebo.
- Pharmacokinetic Data: ZYN002 high and
low dose median plasma concentrations were dose proportional, with
the high dose levels being approximately two times the
concentration of the low dose. Despite the dose proportionality,
there was no correlation between plasma levels and efficacy.
Additional studies of ZYN002Zynerba is
evaluating the utility of ZYN002 in other indications. Enrollment
is complete in its Phase 2 STOP (Synthetic
Transdermal Cannabidiol for
Treatment of Knee Pain due to Osteoarthritis)
study in patients with osteoarthritis, and in its exploratory Phase
2 FAB-C (Treatment of Fragile X Syndrome
Anxiety and Behavioral Challenges
with CBD) study in children with Fragile X
syndrome (FXS). Delivery of top line data from these studies is on
track for August and by the end of September 2017,
respectively.
Conference call
informationZynerba management will host a live
conference call and webcast today at 8:30 am Eastern Time to
discuss the results of this clinical trial. The call can be
accessed by dialing (866) 573-0180 (U.S. and Canada) or (430)
775-1345 (international) and referencing conference ID 64535355. To
access the live webcast or the replay, visit the investor page of
the Company’s website at http://ir.zynerba.com/. The webcast
will be recorded and available on the Company’s website for 30
days.
About Our TechnologyCannabinoids are a class of
chemical compounds found in the Cannabis plant. The two primary
cannabinoids contained in Cannabis are cannabidiol, or CBD, and
∆9-tetrahydrocannabinol, or THC. Clinical and preclinical data
support the potential for CBD in treating epilepsy, arthritis and
Fragile X Syndrome, and THC has positive effects on treating pain.
Zynerba is developing therapeutic medicines that utilize innovative
transdermal technologies that, if successful, may allow for
sustained and controlled delivery of therapeutic levels of CBD and
THC. Transdermal delivery of cannabinoids may have benefits over
oral dosing because it allows the drug to be absorbed through the
skin directly into the bloodstream. This avoids first-pass liver
metabolism, potentially enabling lower dosage levels of active
pharmaceutical ingredients with a higher bioavailability and
improved safety profile. Transdermal delivery also avoids the
gastrointestinal tract, lessening the opportunity for GI related
adverse events and the potential degradation of CBD by gastric acid
into THC, which may be associated with unwanted psychoactive
effects. Using an established chemical pharmaceutical process for
manufacturing, Zynerba replicates the CBD and THC found in the
Cannabis plant. We believe that this will allow us to meet
stringent global regulatory agencies’ standards while ensuring that
we can efficiently supply the amount of product required to meet
the demand of the large markets that we are targeting.
About ZYN002Zynerba’s ZYN002 CBD gel is the
first and only pharmaceutically-produced CBD formulated as a
patent-protected permeation-enhanced gel and is being studied in
adult epilepsy patients with focal seizures, in osteoarthritis and
in children with Fragile X Syndrome. ZYN002 is a clear,
permeation-enhanced gel that is designed to provide controlled drug
delivery transdermally with once- or twice-daily dosing.
About Zynerba Pharmaceuticals, Inc. Zynerba
Pharmaceuticals (NASDAQ:ZYNE) is dedicated to improving the lives
of people with severe health conditions where there is a high unmet
medical need by developing and commercializing
pharmaceutically-produced transdermal cannabinoid medicines
designed to meet the rigorous efficacy and safety standards
established by global regulatory agencies. Through the discovery
and development of these life-changing medicines, Zynerba seeks to
improve the lives of patients battling severe, chronic health
conditions including epilepsy, Fragile X syndrome, osteoarthritis,
fibromyalgia and peripheral neuropathic pain. Learn more at
www.zynerba.com and follow the Company on Twitter at
@ZynerbaPharma.
Cautionary Note on Forward-Looking StatementsThis press release
contains forward-looking statements within the meaning 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. Such statements are
subject to numerous important factors, risks and uncertainties that
may cause actual events or results to differ materially from the
Company’s current expectations. For example, there can be no
guarantee that the Company will obtain approval for ZYN002 or
ZYN001 from the U.S. Food and Drug Administration (FDA) or foreign
regulatory authorities; even if ZYN002 or ZYN001 are approved, the
Company may not be able to obtain the label claims that it is
seeking from the FDA Management’s expectations and, therefore, any
forward-looking statements in this press release could also be
affected by risks and uncertainties relating to a number of other
factors, including the following: the success, cost and timing of
the Company’s product development activities, studies and clinical
trials; the success of competing products that are or become
available; the Company’s ability to commercialize its product
candidates; the size and growth potential of the markets for the
Company’s product candidates, and the Company’s ability to service
those markets; the Company’s ability to develop sales and marketing
capabilities, whether alone or with potential future collaborators;
the rate and degree of market acceptance of the Company’s product
candidates; and the Company’s expectations regarding its ability to
obtain and adequately maintain sufficient intellectual property
protection for its product candidates. This list is not exhaustive
and these and other risks are described in the Company’s periodic
reports, including the annual report on Form 10-K, quarterly
reports on Form 10-Q and current reports on Form 8-K, filed with or
furnished to the Securities and Exchange Commission and available
at www.sec.gov. Any forward-looking statements that the
Company makes in this press release speak only as of the date of
this press release. The Company assumes no obligation to update
forward-looking statements whether as a result of new information,
future events or otherwise, after the date of this press
release.
Zynerba Contacts
Jim Fickenscher, CFO and VP Corporate Development
484.581.7483
fickenscherj@zynerba.com
Will Roberts, VP Investor Relations and Corporate Communications
484.581.7489
robertsw@zynerba.com
Media contact
Theresa Dolge
Tonic Life Communications
Office: 215-928-2748
Theresa.Dolge@toniclc.com
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