Zynerba Pharmaceuticals Announces Clinical Development Updates for FXS, ASD, 22q and DEE
January 04 2022 - 4:05PM
Zynerba Pharmaceuticals, Inc. (Nasdaq: ZYNE), the leader in
innovative pharmaceutically-produced transdermal cannabinoid
therapies for rare and near-rare neuropsychiatric disorders, today
provided program updates for Fragile X syndrome (FXS), autism
spectrum disorder (ASD), 22q11.2 deletion syndrome (22q) and
developmental and epileptic encephalopathies (DEE). In addition to
the continued clinical development of Zygel in FXS, the Company
plans to focus on the development of Zygel in ASD and 22q. Based on
Company research and strategic prioritization, the decision has
been made to not move forward in DEE at this time.
“Committing to a Phase 3 development program for patients with
autism spectrum disorder is a key step in the advancement of a new
treatment option for patients who have high unmet medical needs and
limited FDA approved treatment options,” said Armando Anido,
Chairman and Chief Executive Officer of Zynerba. “Data from our ASD
clinical development program to date are compelling and, with a
clear regulatory path toward potential approval, we are excited to
move forward with this program.”
Anido added, “In addition, we are making good progress towards
completing enrollment in the INSPIRE trial for patients with
22q11.2 deletion syndrome and expect to have topline results
mid-year 2022. Putting resources behind a second indication beyond
FXS and completing the INSPIRE trial is consistent with our mission
of being a leader in rare and near-rare neuropsychiatric disorders,
including FXS, ASD and 22q.”
Pipeline Updates
Zygel in Fragile X Syndrome (FXS)
- The Company
continues to expect topline results from RECONNECT, a confirmatory
pivotal Phase 3 trial of Zygel in patients with FXS, in the second
half of 2023.
- The Company received
written scientific advice from the European Medicines Agency (EMA)
providing clarity and guidance on the clinical and regulatory
requirements for the submission of a marketing authorization
application (MAA) in the European Union (EU) for Zygel for the
treatment of behavioral symptoms associated with FXS. Based on the
EMA’s scientific advice, the Company believes that the successful
completion of the current development program for Zygel in FXS will
satisfy the requirements of an MAA in the EU.
- Presented data at
the BRAIN Foundation Synchrony 2021 Symposium describing how Zygel
may provide therapeutic benefit in FXS through its effects on the
endocannabinoid system. The presentation included results from the
Phase 3 CONNECT-FX trial demonstrating that Zygel was superior to
placebo in multiple analyses in the groups of patients with either
≥90% methylation or complete methylation (100%) of their FMR1 gene.
Data also showed that in the Company’s ongoing open label extension
study, Zygel continued to be well-tolerated, without reports of
clinically significant changes in vital signs, ECGs or laboratories
(including liver function) with a median length of treatment of 21
months. (Presentation)
Zygel in Autism Spectrum Disorder (ASD)
- The Company plans to advance its
program in ASD with two Phase 3 trials, the first of which is
expected to start in the second half of 2022. The Company is
finalizing the Phase 3 study protocol and will submit an
Investigational New Drug application to the U.S. Food and Drug
Administration (FDA) prior to commencing the pivotal program.
- Previous discussions with the FDA
included agreement on utilizing the irritability subscale of the
Aberrant Behavior Checklist – Community (ABC-C) as the primary
endpoint to support an indication for the treatment of irritability
in ASD. This is the same primary endpoint utilized in the pivotal
trials for the two existing FDA approved treatments for ASD.
- In the Company’s exploratory,
open-label Phase 2 BRIGHT trial in 37 children and adolescents with
ASD, all five subscales of the ABC-C showed both statistically
significant (p<0.0002) and clinically meaningful improvements at
14 weeks of treatment from baseline, including the irritability
subscale which showed a 39.1% improvement (p<0.0001).
Furthermore, as previously reported, the 18 patients who continued
through 38 weeks of treatment in the extension portion of the
BRIGHT trial saw a 56.1% improvement in the ABC-C irritability
subscale compared to baseline (p<0.0001).
- In the United States, new estimates
published in the U.S. Centers for Disease Control and Prevention’s
Morbidity and Mortality Weekly Report confirm an increasing trend
in the prevalence of ASD, with approximately one in 44 eight year
old children (or approximately 1.4 million children in total, if
extrapolated to include children ages 3 to 17 years) identified
with ASD. This increasing prevalence underscores the significant
unmet medical need, as well as opportunity, to develop effective
and well-tolerated treatment alternatives for these patients,
especially considering that there are currently only two FDA
approved treatments available for patients with ASD, each of which
are antipsychotics.
Zygel in 22q11.2 Deletion Syndrome (22q)
- The Company continues to screen and
enroll patients for the 14-week open-label Phase 2 INSPIRE trial,
and expects topline data mid-year 2022.
- In the fourth quarter of 2021, the
Company initiated an additional clinical site in the United States
to assist completion of enrollment.
- The Company plans to move forward in
22q as an orphan indication pending results from the ongoing Phase
2 INSPIRE trial in children and adolescents with genetically
confirmed 22q, and subsequent discussion with the FDA on the
regulatory path forward.
Zygel in Developmental and Epileptic Encephalopathies
(DEE)
- Feedback received from the FDA
indicated that selecting a specific DEE syndrome will be required
for a pediatric indication in epilepsy rather than evaluating
improvements in certain seizure types across all DEEs. During 2021,
the Company evaluated individual syndromes and conducted a
feasibility study. Based on Company research and strategic
prioritization, the decision has been made to maintain focus on
other important neuropsychiatric behavioral conditions and not to
pursue further development in DEE at this time.
Financial Outlook
As a result of the pipeline update and anticipated additional
clinical studies in ASD, management now believes the Company’s
$75.6 million of cash and cash equivalents as of September 30,
2021 are sufficient to fund operations and capital requirements
into the second half of 2023.
Conference Call Information
Zynerba management will host a live conference call and
webcast tomorrow, January 5, 2022, at 9:00 a.m. Eastern
Time to discuss updates to its clinical development plans. The
call can be accessed by dialing (866) 573-0180 (U.S. and Canada) or
(430) 775-1345 (international) and referencing conference ID
4181504. 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 innovative
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, and 22q11.2 deletion syndrome. 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 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.
Zynerba Contacts
Peter VozzoICR WestwickeOffice: 443.213.0505Cell:
443.377.4767Peter.Vozzo@Westwicke.com
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