Zynerba Pharmaceuticals, Inc. (NASDAQ:ZYNE), the leader in
innovative pharmaceutically-produced transdermal cannabinoid
therapies for rare and near-rare neuropsychiatric disorders, today
announced the publication of data from the Phase 2 FAB-C (Treatment
of Fragile X Syndrome Anxiety and Behavioral Challenges with CBD)
clinical trial. This trial evaluated ZYN002 cannabidiol (CBD) gel
in pediatric and adolescent patients with Fragile X syndrome (FXS).
The results of the trial have been published in a paper entitled,
‘A Phase 1/2, Open Label Assessment of the Safety, Tolerability,
and Efficacy of Transdermal Cannabidiol (ZYN002) for the Treatment
of Pediatric Fragile X Syndrome’ (Heussler, Helen; Cohen, Jonathan;
Silove, Natalie, et al.) in the August 2nd online edition of
Journal of Neurodevelopmental Disorders. These data were recently
presented at the 2019 Annual Meeting of the American Psychiatric
Association (APA).
This new publication can be accessed at the following link:
https://rdcu.be/bMTnd.
“Fragile X is a family diagnosis, impacting not only the
diagnosed child but also the entire family unit,” said Honey
Heussler, MBBS, FRACP, MRCPCH, PGCAP, DM, Associate Professor,
University of Queensland and Medical Director Child Development,
Children’s Health Queensland, and an investigator in the FAB-C
study. “Children with Fragile X syndrome exhibit a number of
developmental and behavioral symptoms including anxiety, social
avoidance, hyperactivity, and socially unresponsive behaviors that
significantly impact the family, and the child’s capacity to
interact with them, their peers, and care providers. My hope is
that if we can successfully treat these symptoms, we can enhance
the child’s ability to engage and interact. In that regard, these
open label data are promising, and are an important step in the
development of ZYN002. I look forward to the results of the ongoing
double blind, placebo-controlled CONNECT-FX study.”
In summary, the authors suggest that the results from this
open-label trial indicate that ZYN002 may be an effective treatment
for many behavioral and emotional symptoms associated with FXS
although the study’s findings are limited by the open label design
and small sample size. Given the lack of medications approved for
the treatment of FXS, these open-label study findings highlight the
urgent need for randomized, controlled, clinical trials to further
assess the safety and efficacy of ZYN002 for FXS symptoms ranging
from social avoidance, irritability, social
unresponsiveness/lethargy and stereotypy, to anxiety. To that end,
enrollment is ongoing in CONNECT-FX, a multi-national, randomized,
double blind placebo controlled pivotal clinical trial of Zygel
(ZYN002) in FXS.
The FAB-C study enrolled 20 male and female pediatric patients
with FXS, six through 17 years of age at screening, with a
diagnosis of FXS confirmed through molecular documentation of FMR1
full mutation. Eligible patients were provided 12 weeks of ZYN002
as an adjunct to their existing treatments (a six-week titration
period, followed by six-week maintenance period) at three dose
levels: once daily 50 mg dose, twice daily 50 mg dose (100 mg
total), or twice daily 125 mg dose (250 mg total). During the
six-week titration period, patients could be titrated up to 250 mg
daily. Safety and tolerability were assessed bi-weekly through
physical/neurological exam, vital sign collection, 12-lead
electrocardiograms (ECGs), a Modified Suicidality Checklist, safety
laboratory tests, pregnancy tests, urinalysis, and monitoring for
adverse events (AEs). In addition, several measures assessing mood,
behavior, and functioning were selected, including the Anxiety,
Depression, and Mood Scale (ADAMS), the Aberrant Behavior Checklist
– Community (ABC-C) for FXS (ABC-CFXS), the Pediatric Anxiety
Rating Scale (PARS-R), the Pediatric Quality of Life Inventory
(PedsQL™), three Visual Analogue Scales (VAS) (anxiety,
hyperactivity/impulsivity, and tantrum/mood lability), and the
Clinical Global Impression Scale – Severity (CGI-S) and Improvement
(CGI-I).
Results from the efficacy analyses all converge to suggest a
pattern of clinical improvement in a range of key parent and
clinician rated emotional and behavioral symptoms of FXS:
- Patients experienced significant 12-week improvement over
baseline scores for the majority of study efficacy endpoints,
including the change from screening to Week 12 in the ADAMS Total
score, 4 out of the 5 ADAMS subscale scores, the six subscales of
the ABC-CFXS, PARS-R, PedsQL, VAS (anxiety,
hyperactivity/impulsivity, and tantrum/mood lability), and
CGI-I;
- The greatest emotional improvement following treatment was
observed for Anxiety (ADAMS, PARS-R, VAS (anxiety); d = 0.98 to
1.70), while behavioral improvements were most pronounced in the
domains of Social Avoidance (ABC-CFXS; d = 1.00) and Stereotypy
(ABC-CFXS; d = 0.99);
- Importantly, observed improvements were generally greater than
those demonstrated for placebo in prior controlled clinical trials
in FXS; and
- Across assessments, there was consensus in improvement in both
internalizing (e.g., anxiety, social avoidance) and externalizing
symptoms (e.g., irritability) over the course of treatment.
ZYN002 was well tolerated in this study:
- The majority of AEs were mild in severity and resolved by the
end of the 12-week treatment period with no dose adjustment;
- No serious adverse events (SAEs) were reported;
- There were no clinically meaningful trends in laboratory values
(including testosterone levels), except for an increase in
eosinophil count at Day 83 by a patient reporting an AE of moderate
rash (patient completed the study). A repeat blood collection done
one month later on this patient showed a decreased but slightly
above normal eosinophil count; and
- There were no clinically significant changes in liver function
tests.
About Zygel™ (ZYN002)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 Fragile X Syndrome
(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), social
unresponsiveness/lethargy (lack of attention/interaction,
inactive/lack of movement and can resist physical contact), and
anxiety.
Zygel has been designated a Fast Track development program by
the U.S. Food and Drug Administration for treatment of behavioral
symptoms of FXS. Enrollment is ongoing in CONNECT-FX, a
multi-national, randomized, double blind placebo controlled pivotal
clinical trial of Zygel in FXS (https://www.connectfxtrial.com/);
topline data are expected in the first half of 2020. Additionally,
Zynerba expects topline data from its Phase 2 open label BELIEVE 1
trial of Zygel in developmental and epileptic encephalopathies
(DEE) in September 2019. Zynerba has also initiated the Phase 2
BRIGHT trial in autism spectrum disorder and the Phase 2 INSPIRE
trial in 22q11.2 deletion syndrome, with data expected from both
studies 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
StatementsThis 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. 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 ContactWilliam Roberts, Vice President,
Investor Relations and Corporate CommunicationsZynerba
Pharmaceuticals484.581.7489 robertsw@zynerba.com
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