- Clinically Meaningful Median Reduction in
Seizures Compared to Baseline of 48% to 65% Achieved with Continued
ZYN002 Treatment in STAR 2 Extension Study –
Zynerba Pharmaceuticals, Inc. (NASDAQ:ZYNE), a clinical-stage
specialty pharmaceutical company dedicated to developing and
commercializing innovative pharmaceutically-produced transdermal
cannabinoid treatments, today is reporting new clinical data
presented at the 2017 Annual Meeting of the American Epilepsy
Society (AES) in Washington, DC.
In a poster presentation entitled, “Synthetic
Transdermal Cannabidiol for the Treatment of Focal Epilepsy in
Adults (poster #2.428),” Terence O’Brien, MD of the Royal Melbourne
Hospital at The University of Melbourne presented new data from the
completed Phase 2 STAR 1
(Synthetic Transdermal
Cannabidiol for the Treatment of
Epilepsy) study and ongoing STAR 2 18-month open label
extension study evaluating ZYN002 cannabidiol (CBD) transdermal gel
in patients with focal seizures. The presentation included data
through nine months of total exposure to ZYN002 (three months of
treatment in STAR 1 and six months in STAR 2).
The key findings include that clinically meaningful
responses to ZYN002, as measured by reductions in focal seizures
from the baseline period of STAR 1, are correlated with continued
treatment with ZYN002. Patients who received ZYN002 (195mg during
STAR 1 for three months and 390mg for six months in STAR 2) for a
total of nine months achieved a median reduction in seizures of
65%. Patients who received ZYN002 (390mg for three months in
STAR 1 and six months in STAR 2) achieved a 48% median reduction in
seizures from baseline. In addition, ZYN002 was shown to be very
well tolerated through nine months of exposure.
“These new ZYN002 data are the first of their kind,
showing that focal seizures in adults may be effectively treated by
a transdermal gel delivery of pharmaceutically-produced
cannabidiol,” said Terri Browning Sebree, Zynerba’s President. “In
this population of patients, continued treatment with ZYN002 was
shown to significantly reduce seizure rates compared to baseline.
Importantly, baseline seizure frequency appears to be an important
indicator of response. These are important findings that will help
us finalize a new trial design. We expect to outline the design,
size and timing of the trial in the first quarter of 2018, and
initiate the trial later in 2018.”
“The data presented this afternoon at the American
Epilepsy Society meeting are exciting as they demonstrate that
ZYN002 may have an effect on focal seizures in adults suffering
from refractory epilepsy,” said Terence O’Brien, MD. “The epilepsy
community has been eagerly awaiting data demonstrating the
potential of pharmaceutically-produced cannabidiol formulated for
transdermal delivery. Evidence for the efficacy of CBD-based
treatments to reduce seizures in certain epilepsy populations is
emerging, but there is no previous high level clinical trial
evidence for focal seizures in adults. The potential for a
CBD-based treatment with an optimal tolerability profile would be
significant for these patients. I look forward to participating in
the next clinical trial with ZYN002, and believe that this drug
holds great promise for patients suffering from refractory
epilepsy.”
The data presented in the poster are as
follows:
Demographics and Baseline
Characteristics
- Patients randomized into STAR 1 (N=188) had a median monthly
seizure frequency of 10.6 (3-335) at baseline. By group, the median
monthly seizure frequency at baseline was 10.5 for the placebo
group, 14.0 for the ZYN002 195 mg treatment group, and 10.14 for
the ZYN002 390 mg treatment group;
- Of the 188 randomized patients, 186 were analyzed for efficacy,
and 174 completed the 12-week STAR 1 study;
- 171 patients (98% of STAR 1 completers) continued into STAR
2;
- Patients were taking a wide range of antiepileptic drugs
(AEDs), with a median of 3.0 AEDs; use of clobazam was excluded in
both the STAR 1 and STAR 2 studies.
Efficacy
- As previously disclosed on August 7, 2017, compared with
baseline, after 12 weeks of blinded treatment, the change in
seizure frequency did not statistically differ between placebo and
both doses of ZYN002, though there was a numerical difference
favoring ZYN002;
- The lack of separation of ZYN002 from placebo in STAR 1 was
likely due in part to 15 (24%) placebo-treated patients who
achieved at least a 50% reduction in focal seizures; 13 of these 15
patients had a relatively low baseline seizure rate (<15 focal
seizures per month);
- In STAR 1, patients with more severe epilepsy (defined as a
baseline seizure frequency of ≥15 per month) taking ZYN002 had a
greater percent reduction in seizures compared to patients with
severe epilepsy receiving placebo;
- Continued exposure to ZYN002 in STAR 2 (all patients dosed with
390 mg/day) resulted in clinically meaningful reductions in
seizures: ° Patients taking ZYN002 for six months
(three months during STAR 1 and three months in STAR 2) experienced
a >30% median reduction in seizures from baseline;
° Patients taking ZYN002 for nine months (three months
during STAR 1 and six months in STAR 2) experienced a >65% (195
mg in STAR 1 and 390 mg in STAR 2) and >48% (390 mg in STAR 1
and STAR 2) median reduction in seizures from baseline.
An infographic accompanying this announcement is available at
http://www.globenewswire.com/NewsRoom/AttachmentNg/a149a6dd-69c9-4abd-a7b5-2935e6d6504f
- A small number of patients in STAR 2 had an increase in their
background AEDs; the improvements in seizure frequency observed in
STAR 2 were not due to these changes to background AEDs.
Safety
- ZYN002 was very well tolerated with an incidence of adverse
events comparable to placebo and no clinically significant
differences between the active treatment groups;
- The safety profile of ZYN002 was consistent with previously
released data from Phase 1 and Phase 2 trials;
- There were no clinically significant changes in ECGs or
laboratory results in patients receiving ZYN002.
A copy of the poster presentation is currently available on the
Zynerba corporate website
at http://zynerba.com/publications/
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
children with Fragile X Syndrome, adult epilepsy patients with
focal seizures and osteoarthritis. ZYN002 is a clear,
permeation-enhanced gel that is designed to provide controlled drug
delivery transdermally with once- or twice-daily dosing.
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 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. In addition, the Company’s cash and cash equivalents may not
be sufficient to support its operating plan for as long as
anticipated. 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 ContactWill Roberts, VP Investor
Relations and Corporate
Communications484.581.7489robertsw@zynerba.com
Media contactTheresa Dolge Tonic Life
CommunicationsOffice: 215-928-2748Theresa.Dolge@toniclc.com
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