- Designation Facilitates and Expedites
Development of Drugs for Patients with Serious Unmet Medical Needs
-
- Enrollment Progressing in Pivotal CONNECT-FX
Trial of Zygel in FXS, with Data Expected in the Second Half of
2019 -
DEVON, Pa., May 06, 2019 (GLOBE NEWSWIRE) -- Zynerba Pharmaceuticals, Inc. (ZYNE), the leader in innovative pharmaceutically-produced transdermal cannabinoid therapies for rare and near-rare neuropsychiatric disorders, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track Designation for the Company’s lead development candidate Zygel™ (ZYN002 CBD gel) for treatment of behavioral symptoms associated with Fragile X Syndrome (FXS). FDA’s Fast Track program is designed to facilitate the development of drugs intended to treat serious conditions and fill unmet medical needs, and can lead to expedited review by FDA in order to get new important drugs to the patient earlier.
“The FDA’s decision to grant Fast Track Designation for Zygel underscores the significance and severity of the unmet medical need that exists for patients living with Fragile X Syndrome and their caregivers,†said Armando Anido, Chairman and Chief Executive Officer of Zynerba. “We believe that Zygel has the potential to be the first treatment indicated to directly address the core behavioral symptoms of this syndrome, and we look forward to working closely with the FDA to obtain approval to market Zygel as soon as possible.â€
Zynerba is conducting a pivotal trial to assess the efficacy and safety of Zygel as treatment for the behavioral symptoms of FXS in pediatric and adolescent patients (three through 17 years of age). Zygel is a pharmaceutically-manufactured CBD formulated as a patent-protected permeation-enhanced clear gel, designed to provide controlled transdermal drug delivery into the bloodstream.
About Fast Track
Designation
Fast Track Designation is intended to facilitate development and
expedite review of drugs to treat serious or life-threatening
conditions so that a product can reach the market expeditiously. A
drug that is intended to treat a serious or life-threatening
condition that demonstrates the potential to address an unmet
medical need may qualify for Fast Track designation. Features of
this designation include opportunities for frequent interactions
with the review team. These include meetings with the FDA to
discuss items such as study design, extent of safety data required
to support approval, dose-response concerns, accelerated approval,
the structure and content of an NDA, and other critical issues. In
addition, such a product could be eligible for priority review if
supported by clinical data at the time of NDA.
About Fragile X Syndrome
(FXS)
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. FXS is caused by a mutation
in the Fragile X Mental Retardation gene (FMR1) 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 and behavioral symptoms, such as social
avoidance and irritability. In the US, there are about 71,000
patients suffering with FXS.
About Zygelâ„¢
Zygel (CBD gel) is the first and only pharmaceutically-manufactured
CBD formulated as a patent-protected permeation-enhanced clear gel,
designed to provide controlled drug delivery into the bloodstream
transdermally (i.e. through the skin). Recent studies suggest that
FXS and other neuropsychiatric conditions may be associated with a
disruption in the endocannabinoid (EC) system. Clinical and
anecdotal data suggest that CBD may modulate the EC system and
improve certain core social and behavioral symptoms, including
social avoidance (prefers isolation from others, prefers solitary
activities, avoids new social activities), irritability (aggressive
to others, tantrums/outbursts, and stubbornness), and social
unresponsiveness/lethargy (lack of attention/interaction,
inactive/lack of movement and can resist physical contact).
Enrollment is ongoing in the multi-national, randomized, double blind placebo controlled Clinical study of Cannabidiol (CBD) in Children and Adolescents with Fragile X (CONNECT-FX), a pivotal clinical trial of ZYN002 in FXS (https://www.connectfxtrial.com/); topline data from CONNECT-FX are expected in the second half of 2019. Additionally, Zynerba expects topline data from its Phase 2 Open Label Study to Assess the Safety and Efficacy of ZYN002 Administered as a Transdermal Gel to Children and Adolescents with Developmental and Epileptic Encephalopathy (BELIEVE 1) clinical trial in the third quarter of 2019. Zynerba has also initiated a Phase 2 study of Zygel in Autism Spectrum Disorder, with data expected in the first half of 2020.
About Zynerba Pharmaceuticals,
Inc.
Zynerba Pharmaceuticals is the leader in pharmaceutically-produced
transdermal cannabinoid therapies for rare and near-rare
neuropsychiatric disorders. We are committed to improving the lives
of patients and their families living with severe, chronic health
conditions including Fragile X Syndrome, Autism Spectrum Disorder,
22q11.2 Deletion Syndrome, and a heterogeneous group of rare and
ultra-rare epilepsies known as developmental and epileptic
encephalopathies. Learn more at www.zynerba.com and
follow us 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,â€
“potential,†“proposed,†“continue,†“estimates,†“anticipates,â€
“expects,†“plans,†“intends,†“may,†“could,†“might,†“will,â€
“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
Zygel from the U.S. Food and Drug Administration (FDA) or foreign
regulatory authorities; even if Zygel is 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 Company’s cash and cash equivalents may not be
sufficient to support its operating plan for as long as
anticipated; the Company’s ability to obtain additional funding to
support its clinical development programs; the results, cost and
timing of the Company’s clinical development programs, including
any delays to such clinical trials relating to enrollment or site
initiation; clinical results for the Company’s product candidates
may not be replicated or continue to occur in additional trials and
may not otherwise support further development in a specified
indication or at all; actions or advice of the FDA and foreign
regulatory agencies may affect the design, initiation, timing,
continuation and/or progress of clinical trials or result in the
need for additional clinical trials; the Company’s ability to
obtain and maintain regulatory approval for its product candidates,
and the labeling under any such approval; the Company’s reliance on
third parties to assist in conducting pre-clinical and clinical
trials for its product candidates; delays, interruptions or
failures in the manufacture and supply of the Company’s product
candidates 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 Contact
William Roberts, Vice President, Investor Relations and Corporate
Communications
Zynerba Pharmaceuticals
484.581.7489
[email protected]