Zynerba Pharmaceuticals Provides Regulatory Update on Zygel™ in Fragile X Syndrome
December 17 2020 - 06:30AM
Zynerba Pharmaceuticals, Inc. (NASDAQ:ZYNE), the leader in
innovative pharmaceutically-produced transdermal cannabinoid
therapies for rare and near-rare neuropsychiatric disorders, today
provided an update on its meeting with the U.S. Food and Drug
Administration (FDA) regarding its Fragile X syndrome (FXS)
program. The Company plans to conduct a double-blind,
placebo-controlled pivotal trial in patients with FXS who have a
highly methylated FMR1 gene to confirm the positive results
observed in this population of responders in the CONNECT-FX trial.
In the first half of 2021, Zynerba will review the trial design and
protocol for the new trial through a Type C meeting with the FDA
and expects to initiate the pivotal trial before the end of 2021.
Zynerba believes that positive results from this confirmatory trial
would be sufficient to support the submission of a New Drug
Application (NDA) for Zygel™ in FXS.
“We believe that Zygel has the potential to meaningfully relieve
the behavioral symptoms of the most impacted individuals with
Fragile X syndrome. We are committed to bringing this important
therapy to patients and their families within the Fragile X
community,” said Armando Anido, Chairman and Chief Executive
Officer of Zynerba. “We are thankful for our ongoing constructive
dialogue with the FDA on our path forward to NDA submission.
Completing the development of Zygel in FXS and preparing for a
successful launch will be the primary focus of the Company.”
“The results reported in the analysis of children with a highly
methylated FMR1 gene are a source of considerable hope for the
patients and their families who are impacted by Fragile X
syndrome,” said Linda Sorensen, Executive Director of the National
Fragile X Foundation (NFXF). “Zynerba has been an important partner
to the families of children with Fragile X. We thank them for their
continued dedication and commitment to bringing an FDA-approved
treatment to our families and look forward to helping with the
recruitment of patients for this study.”
The Company’s development plan for Zygel in other indications
includes the following:
-
Developmental and epileptic encephalopathies
(DEE): Evaluation of potential target indications is
ongoing, and Zynerba now expects to finalize target syndrome
selection in 2021 in one or more DEE syndromes.
- 22q11.2
deletion syndrome (22q): Zynerba expects to resume
recruitment for the 14-week open label Phase 2 INSPIRE trial in
children and adolescents with genetically confirmed 22q once
COVID-19-related restrictions in Australia are eased. After
recruitment has resumed, the Company will be able to provide a
timeframe for completion of this trial.
- Autism spectrum disorder
(ASD): In the first half of 2021, Zynerba intends to
discuss data supporting the potential efficacy of Zygel in ASD,
including the results of the Phase 2 BRIGHT trial in children and
adolescents with moderate-to-severe ASD with the FDA to determine
the regulatory path forward.
Financial Impact on Cash The Company expects
that its cash runway will extend into the beginning of 2023 as a
result of these changes to its development plans.
Conference call
informationZynerba management will host a live
conference call and webcast today at 8:30 am Eastern Time to
discuss the recent FDA meeting and the regulatory pathway for Zygel
in FXS. The call can be accessed by dialing (866) 573-0180 (U.S.
and Canada) or (430) 775-1345 (international) and referencing
conference ID 4088027. 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 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,” “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. 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 expectations, projections and
estimates regarding expenses, future revenue, capital requirements,
incentive and other tax credit eligibility, collectability and
timing, and availability of and the need for additional financing;
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 U.S. Food and Drug Administration
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 planned
clinical trial evaluating Zygel™ in patients with a highly
methylated FMR1 gene may not confirm the results of the CONNECT-FX
trial or may not be determined to be sufficient to support an NDA
submission; 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;
the Company’s expectations regarding its ability to obtain and
adequately maintain sufficient intellectual property protection for
its product candidates; the timing and outcome of current and
future legal proceedings; and the extent to which health epidemics
and other outbreaks of communicable diseases, including COVID-19,
could disrupt our operations or adversely affect our business and
financial conditions. 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 ContactWilliam Roberts, Vice
President, Investor Relations and Corporate CommunicationsZynerba
Pharmaceuticals484.581.7489 robertsw@zynerba.com
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