Zynerba Pharmaceuticals, Inc. (NASDAQ:ZYNE), a clinical-stage
specialty neuropsychiatric pharmaceutical company dedicated to
developing and commercializing innovative pharmaceutically-produced
transdermal cannabinoid treatments for rare and near-rare
neurological and psychiatric disorders with high unmet medical
needs, today announced the results of a positive meeting held with
the U.S. Food and Drug Administration (FDA) regarding its planned
development strategy for ZYN002 in Fragile X syndrome (FXS). FXS is
a rare genetic developmental disability that is the leading known
cause of both inherited intellectual disability and autism spectrum
disorder.
ZYN002 is the first and only
pharmaceutically-produced cannabidiol (CBD) formulated as a
patent-protected permeation-enhanced transdermal gel. Zynerba has
received U.S. Orphan Drug designation for the use of CBD as a
treatment of FXS. Currently, there are no approved therapies to
treat FXS or its most common symptoms.
Based on the company’s dialogue with the FDA, the
Company expects to initiate a single pivotal study mid-year 2018 to
support a New Drug Application (NDA) for ZYN002 in FXS. The FDA and
the Company are in agreement that the primary and key secondary
endpoints for the study should assess observable behaviors in
patients with FXS as reported by the caregiver using the validated
Aberrant Behavior Checklist in Fragile X syndrome (ABC-FXS). If the
pivotal trial meets its endpoints, approval for an indication
encompassing the treatment of behavioral symptoms associated with
Fragile X syndrome may be granted.
“The FDA meeting was an important milestone for us as we advance
the development of ZYN002 for patients and their families suffering
with the profound behavioral symptoms of Fragile X syndrome,” said
Armando Anido, Chairman and Chief Executive Officer of Zynerba. “We
are pleased with the outcome of the discussion and the guidance on
trial design, and believe we now have a path forward to advance the
development of ZYN002 to an NDA. We look forward to initiating the
pivotal study mid-year, and potentially providing FXS patients and
their families an effective and well tolerated therapy to treat the
complex behavioral symptoms of Fragile X syndrome.”
Zynerba plans to initiate a pivotal 14-week randomized, double
blind, placebo controlled clinical trial in approximately 200
pediatric and adolescent patients in the U.S., Australia and New
Zealand. Patients will be randomized 1:1 to receive either one of
two weight based doses of ZYN002, or one of two matching
administrations of placebo. Zynerba anticipates initiation of this
pivotal clinical trial mid-year 2018. Additional protocol details
will be shared at that point. All patients will be eligible to
enroll in a 12-month open label extension after completing dosing
in the pivotal study.
“We believe that ZYN002 may address core behavioral symptoms of
FXS and improve the quality of life for patients and their
families,” said Dr. Liza Squires, Zynerba’s Chief Medical Officer.
“There are currently no drugs indicated to address behavioral
symptoms in the FXS population. We believe we have designed an
efficient pivotal program that includes endpoints that measure
clinically relevant and observable behaviors in patients with FXS,
and if successful, positions us to bring the FXS community its
first targeted treatment designed with patients’ symptoms in
mind.”
About Fragile X syndrome Fragile X syndrome is
a rare genetic developmental disability that is the leading known
cause of both inherited intellectual disability and autism spectrum
disorder, affecting 1 in 3,600 to 4,000 males and 1 in 4,000 to
6,000 females. It is the most common inherited intellectual
disability in males and a significant cause of intellectual
disability in females. It is caused by a mutation in the Fragile X
Mental Retardation gene located on the X chromosome and leads to
dysregulation of the endocannabinoid pathway including the
reduction in endogenous cannabinoids (2-AG and anandamide). The
disorder negatively affects synaptic function, plasticity and
neuronal connections, and results in a spectrum of intellectual
disabilities, social anxiety and memory problems. In the US,
there are about 71,000 patients suffering with FXS.
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 and Fragile X
syndrome, and THC has positive effects on treating symptoms of
Tourette Syndrome. 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 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 transdermal gel and is being
studied in children and adolescents with Fragile X syndrome and
developmental and epileptic encephalopathies, and in adult epilepsy
patients with focal seizures. 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 a clinical-stage specialty
pharmaceutical company dedicated to developing and commercializing
innovative pharmaceutically-produced transdermal cannabinoid
treatments for rare or near-rare neuropsychiatric diseases with
high unmet medical needs. We are dedicated to improving the lives
of people with severe health conditions by developing cannabinoid
medicines designed to meet the rigorous efficacy and safety
standards established by global regulatory agencies. Through the
discovery and development of these potentially life-changing
medicines, Zynerba seeks to improve the lives of patients battling
severe, chronic health conditions including Fragile X syndrome,
refractory epilepsies, Tourette Syndrome, and other
neuropsychiatric disorders. Learn more at www.zynerba.com and
follow the Company on Twitter at @ZynerbaPharma.
Cautionary Note on Forward-Looking
Statements
This 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 from the U.S. Food and Drug
Administration (FDA) or foreign regulatory authorities; even if
ZYN002 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.
Investor Contact
Will Roberts, VP Investor Relations and Corporate
CommunicationsZynerba
Pharmaceuticals484.581.7489robertsw@zynerba.com
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