Zynerba Pharmaceuticals Presents Data Supporting FMR1 Methylation Status as a Correlate to Fragile X Syndrome Severity at the...
October 15 2020 - 3:06PM
Zynerba Pharmaceuticals, Inc. (NASDAQ:ZYNE), the leader in
innovative pharmaceutically-produced transdermal cannabinoid
therapies for rare and near-rare neuropsychiatric disorders, is
presenting a poster describing data from the CONNECT-FX
(
Clinical study
Of
Ca
NNabidiol (CBD) in Childr
En and
Adoles
Cen
Ts with
Fragile
X) trial describing the
role of FMR1 methylation status in children and adolescents with
Fragile X syndrome (FXS) as a correlate to disease severity and as
a prognostic biomarker. These data are being presented at the
virtual Joint 16th International Child Neurology Congress (ICNC)
& 49th Annual Child Neurology Society (CNS) Meeting. These data
will also be presented as an oral presentation during the “Research
Pipeline: New Findings on Diagnostic and Therapeutics” session of
the virtual American Academy of Child and Adolescent Psychiatry
(AACAP) 2020 Annual Meeting on Friday, October 23rd, 2020.
A copy of the poster entitled, “ZYN002 Cannabidiol Transdermal
Gel in Children and Adolescents With Fragile X Syndrome: Role of
Methylation Status as a Correlate to Disease Severity and as a
Prognostic Biomarker” is available on the Zynerba corporate website
at http://zynerba.com/publications/.
“We are excited to provide an update on the potential role of
methylation status of the FMR1 gene as a predictive biomarker of
preferential response to Zygel™ (ZYN002) in the treatment of the
behavioral symptoms of FXS,” said Zynerba’s Chief Medical Officer,
Joseph M. Palumbo, MD, FAPA, MACPsych. “These new data demonstrate
that in patients diagnosed with FXS with a fully methylated FMR1
gene significantly more patients who received Zygel achieved a
clinically meaningful improvement in their behavioral symptoms
compared to patients who received placebo.”
CONNECT-FX is a randomized, double-blind, multinational, 14-week
pivotal study to evaluate the efficacy and safety of Zygel in
children/adolescents aged 3 to 17 years. Although the CONNECT-FX
full analysis set did not achieve statistical significance in its
endpoints, building on current scientific evidence, a pre-planned
ad hoc analysis of patients having at least 90% methylation (“full
methylation” or FMet) of the impacted FMR1 gene, representing 80%
of the overall study population, was performed. The results,
including the achievement of statistical significance (p=0.020) in
the primary endpoint of improvement at 12 weeks of treatment in the
Social Avoidance subscale of the ABC-CFXS compared to placebo,
suggest that Zygel may have benefit in patients with full
methylation of the FMR1 gene.
Zynerba utilized psychometric analyses to determine what
constitutes a clinically meaningful change from baseline as
measured by subscales of the ABC-CFXS. New results described today
include the results of these analyses which support the definition
of a clinically meaningful treatment response over 12 weeks of
treatment as an improvement of three points or greater for the
Social Avoidance subscale, nine points or greater for the
Irritability subscale, and five points or greater for the Socially
Unresponsive / Lethargic subscale. The Company determined that
58.2% of FMet patients receiving Zygel achieved a clinically
meaningful change in their socially avoidant behavior compared to
40.6% of patients receiving placebo (statistically significant;
p=0.031), and 40.3% of patients receiving Zygel achieved a
clinically meaningful change in Irritability compared to 23.8% of
patients receiving placebo (statistically significant;
p=0.036).
Figure 1: Greater Percentages of
Participants Achieved Meaningful Change in
ABC-CFXS
Social Avoidance and Irritability
with ZYN002 vs Placebo
https://www.globenewswire.com/NewsRoom/AttachmentNg/d8cf2270-883e-4618-9515-82b29393a6dc
The authors of the poster concluded that:
- To our knowledge, CONNECT-FX is the largest controlled study
ever performed in FXS.
- These results may represent an important step forward in
biomarker-driven prediction of response in FXS and neuroscience.
- Zygel was well tolerated, and the safety profile was consistent
with previously reported clinical trials.
- In the FMet group, Zygel was superior to placebo in multiple
analyses, including:
- Statistically significant mean change in Social Avoidance vs
placebo;
- Proportion of patients attaining threshold of clinically
meaningful change in Social Avoidance and Irritability;
- Caregiver reported improvements, including statistically
significant improvements in Social Avoidance and Isolation, Social
Interaction, and Irritable and Disruptive Behaviors.
- Zynerba will be meeting with the FDA in the fourth quarter of
2020 to discuss these data.
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 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 ContactWill Roberts,
VP Investor Relations and Corporate
Communications484.581.7489robertsw@zynerba.com
Media contactMolly DevlinEvoke
KYNE215.928.2199Molly.Devlin@evokegroup.com
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