Zynerba Pharmaceuticals Announces the Completion of Enrollment in the Pivotal CONNECT-FX Trial of Zygel™ in Fragile X Syndr...
February 26 2020 - 6:50AM
Zynerba Pharmaceuticals, Inc. (NASDAQ:ZYNE), the leader in
innovative pharmaceutically-produced transdermal cannabinoid
therapies for rare and near-rare neuropsychiatric disorders, today
announced that the Company has completed enrollment in its 14-week
pivotal CONNECT-FX (
Clinical study
of Ca
nnabidiol (CBD) in
Childr
en and
Adoles
cen
ts with
Fragile
X) trial. The trial is
evaluating the efficacy and safety of Zygel™ CBD Gel as a treatment
for behavioral symptoms of Fragile X syndrome (FXS) in 210 children
ages three through 17 with full mutation FXS. Topline results are
expected late in the second quarter of 2020.
“We are excited about the potential for Zygel, if
approved by the FDA, to be the first drug ever indicated to treat
the behavioral symptoms of Fragile X, and completion of enrollment
in this trial is an important step toward that goal,” said Armando
Anido, Chairman and Chief Executive Officer of Zynerba. “I want to
thank everyone who participated in this achievement, particularly
the patients in the study, their families, the clinicians involved
in CONNECT-FX and their staff. We look forward to announcing the
topline results of this trial late in the second quarter of this
year.” The multi-national, randomized, double-blind,
placebo-controlled, 14-week CONNECT-FX trial is assessing the
efficacy and safety of Zygel for the treatment of children and
adolescents with FXS. Two hundred and ten (210) patients with
Fragile X syndrome, confirmed with the full mutation of the FMR1
gene, have been randomized at 21 clinical sites in the United
States, Australia, and New Zealand. The trial protocol targeted
enrollment of at least 204 patients. Patients have been randomized
1:1 to either trial drug or placebo. The primary endpoint is the
change from baseline to the end of the treatment period in the
Aberrant Behavior Checklist-Community FXS Specific (ABC-CFXS)
Social Avoidance subscale. Key secondary endpoints are the change
from baseline to the end of the treatment period in the ABC-CFXS
Irritability subscale score and the ABC-CFXS Socially
Unresponsive/Lethargic subscale score, and improvement in Clinical
Global Impression (CGI-I) anchored to behavioral symptoms of FXS
evaluated at the end of the treatment period. Consistent with
recent guidance from the FDA on capturing the voice of the patient
in drug development, additional qualitative data on the clinical
relevance of various FXS behaviors to caregivers and patients is
also being collected.
Baseline Characteristics of Patients
Randomized into CONNECT-FXAs intended and prospectively
designed, the trial has successfully enrolled a more severely
affected population than the population that was enrolled in the
previously completed Phase 2 FAB-C trial, as measured by baseline
behavioral symptoms, enabling the study to potentially demonstrate
the anticipated full range of efficacy of Zygel in several
behavioral domains. The ABC-CFXS mean baseline scores for the
patients randomized in the CONNECT-FX trial in comparison to the
FAB-C trial are as follows (higher baseline scores denote more
severe behaviors):
- Social Avoidance subscale (primary endpoint): 7.2 in CONNECT-FX
vs 5.1 in FAB-C;
- Irritability subscale (key secondary endpoint): 28.1 in
CONNECT-FX vs 18.2 in FAB-C;
- Socially Unresponsive/Lethargic subscale (key secondary
endpoint): 13.2 in CONNECT-FX vs 8.7 in FAB-C;
- Hyperactivity subscale: 18.4 in CONNECT-FX vs 14.5 in
FAB-C;
- Stereotypy subscale: 9.4 in CONNECT-FX vs 7.9 in FAB-C;
and
- Inappropriate Speech subscale: 6.9 in CONNECT-FX vs 6.1 in
FAB-C.
During screening, the caregivers of patients in the
trial were informed that their participating child may have the
opportunity to receive Zygel in an open label extension trial
following the child’s compliant completion of CONNECT-FX,
regardless of their child’s perceived response or actual blinded
drug assignment in CONNECT-FX. To date, 97% of the 158 patients who
have completed CONNECT-FX have enrolled in the open label extension
trial.
One hundred and fifty seven (157), or 75%, of the
enrolled patients are male and the mean age at randomization in the
study is 9.8 years.
The Company expects to announce topline results of
this study late in the second quarter of 2020. If the results are
positive, the Company expects to meet with the U.S. Food and Drug
Administration (FDA) to determine acceptability of the data as a
basis to submit its New Drug Application (NDA) for Zygel in FXS in
the second half of 2020, with potential approval by mid-year 2021.
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 Zygel™ 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
autism-related symptoms, including social avoidance and
anxiety.
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
Media contactMolly DevlinEvoke
KYNE215.928.2199Molly.Devlin@evokegroup.com
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