Zynerba Pharmaceuticals, Inc. (NASDAQ:ZYNE), the leader in
innovative pharmaceutically-produced transdermal cannabinoid
therapies for rare and near-rare neuropsychiatric disorders, today
announced the availability of a poster describing 116-week
(two-year) data from the Phase 2 FAB-C (Treatment of
Fragile X Syndrome
Anxiety and
Behavioral
Challenges with CBD)
trial of Zygel™ (CBD transdermal gel; ZYN002) in pediatric and
adolescent patients with Fragile X syndrome (FXS). The data in the
poster show that the statistically significant improvements from
baseline that were observed at week 12 were sustained in each
subscale score of the Aberrant Behavior Checklist for Fragile X
(ABC-CFXS) through two years for patients who participated in the
open label extension.
The poster entitled Cannabidiol Transdermal Gel for the
Treatment of Fragile X Syndrome: Post Hoc Analysis and Pattern of
Efficacy on Domains of the Aberrant Behavior Checklist-Community
for FXS (ABC-CFXS) Through 116 Weeks of Treatment is available at
the 2020 American Academy of Neurology (AAN) Science Highlights
Virtual Session. The Virtual Session is online at
http://www.aan.com/2020science. A copy of the poster is also
available on the Zynerba corporate website at
http://zynerba.com/publications/.
“It’s very exciting to see that the observed early benefits of
Zygel appear to be sustained for over two years in patients who
enrolled in the open label extension of FAB-C; these data suggest
the potential for a sustained and measurable benefit for those
patients who experience an early response,” said Zynerba’s Chief
Medical Officer, Joseph M. Palumbo, MD, FAPA, MACPsych. “It’s also
reassuring to see these responses in the context of a strong
tolerability profile. We look forward to the results of our pivotal
CONNECT-FX study in children and adolescents with FXS late this
quarter.”
Open Label Phase 2 FAB-C Trial Background
The 12-week treatment results of the Phase 2 FAB-C trial were
initially announced in September 2017. These data were published in
the August 2nd, 2019 online edition of Journal of
Neurodevelopmental Disorders. (Press release)
Twenty patients aged 6 to 17 years of age with Fragile X as
confirmed by molecular documentation of FMR1 full mutation were
enrolled in the open label FAB-C study. Zygel was added on to other
medications being administered. At the completion of the 12-week
study (Period 1), patients could enter an extension study (Period
2).
Thirteen patients who completed the Period 1 rolled into Period
2. One patient who withdrew during Period 2 for reasons unrelated
to safety or efficacy had no efficacy data post week 12 and
therefore was not included in the analyses. Ten patients exceeded
two years of therapy.
Sustained Improvement in Core FXS Behaviors over Two
Years of Treatment with Zygel
Statistically significant improvements from baseline were
observed at week 12 in all six subscale scores of the ABC-CFXS in
Period 1 and these statistically significant improvements were
sustained through two years in subjects who entered Period 2. The
persistence of effect over the two-year period is as follows.
An infographic accompanying this announcement is available
at https://www.globenewswire.com/NewsRoom/AttachmentNg/b30a11af-12db-4428-9a7d-5b857a5ed25f
In addition, statistically significant improvements from
baseline were observed at week 12 in the total score and all five
subscale scores of ADAMS and these statistically significant
improvements persisted to two years.
Responder Analysis
Zynerba performed responder analyses for patients achieving at
least a 25% and 50% improvement from baseline for each subscale of
the ABC-CFXS.
Maximal 25% responder rates for each ABC-CFXS domain at any
visit in patients who completed Period 1 ranged from 72.2% to 83.3%
and emerged by week 8 for all domains. Most patients who entered
Period 2 met criteria for response at weeks 12 (≥66.7%) and 116
(≥80%).
An infographic accompanying this announcement is available
at https://www.globenewswire.com/NewsRoom/AttachmentNg/1f21f88b-08af-47f8-b7da-af5223a2c0bf
Maximal 50% responder rates for each ABC-CFXS domain at any
visit ranged from 50.0% to 77.8% in patients who completed Period 1
and were observed at week 8 for all domains except stereotypy.
Among patients who entered Period 2, 50% responder rates ranged
from 50% to 83.3% at week 12. At week 116, the range of 50.0%
responder rates was observed to be descriptively higher, ranging
from 60% to 100% across the six ABC-CFXS domains.
An infographic accompanying this announcement is available
at https://www.globenewswire.com/NewsRoom/AttachmentNg/a90b7fe1-1a26-4a3d-a12e-145546369a66
Evidence of Global, Multi-domain, and Sustained
Reduction in Behavioral Symptom Burden
Radar charts were created to visualize the proportional effect
of Zygel across the six ABC-CFXS subscales. The boundaries of the
polygon at screening and endpoint allow visualization of change
across all domains cross-sectionally and over time. These radar
charts suggest global and sustained reductions in severity with
Zygel treatment in patients who entered Period 2.
Tolerability of Zygel over Two Years of
Treatment
Zygel was well tolerated in the FAB-C trial over two years.
Treatment-emergent adverse events (TEAEs) – any event occurring
during a trial period whether unrelated or related to study drug -
are common in children and expected over a two-year period. Of the
66 TEAEs reported in 19 patients, all were either mild (85%) or
moderate (15%), and 91% were determined to be unrelated to
treatment. No treatment-related TEAEs occurred in more than one
patient. Only one serious adverse event (constipation) was reported
over two years of treatment and was not related to treatment.
The authors of the poster concluded that:
- In this post hoc analysis, the majority of patients who
completed Period 1 met important criteria for therapeutic response
(≥25% or ≥50% improvement from baseline in ABC-CFXS domains) at
weeks 4, 8, and 12 of the Phase 2 FAB-C trial; this response was
sustained or continued to improve through two years in patients who
entered Period 2;
- Simultaneous visualization of change across all ABC-CFXS
domains at baseline and endpoint through radar charts provided
additional evidence for global, multi-domain, and sustained
reduction in behavioral symptom burden among patients who entered
Period 2;
- Zygel was well tolerated through two years; all AEs were mild
or moderate and most were considered unrelated to treatment;
and
- Together, these data may suggest evidence of the clinical
efficacy and favorable safety and tolerability of Zygel in children
and adolescents with FXS when added to stable standard of care
therapies. A double‐blind, placebo‐controlled study of Zygel in FXS
called CONNECT-FX is currently in progress and will extend the
knowledge gained from this Phase 2 study.
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, and the leading genetic cause
of autism spectrum disorder (ASD). 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 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 ContactWilliam Roberts, Vice President,
Investor Relations and Corporate CommunicationsZynerba
Pharmaceuticals484.581.7489 robertsw@zynerba.com
Media contactMolly DevlinEvoke
KYNE215.928.2199Molly.Devlin@evokegroup.com
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