Zynerba Pharmaceuticals Announces that Enrollment is Nearing Completion in Pivotal CONNECT-FX Trial in Fragile X Syndrome
January 13 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
provided an update on the pivotal CONNECT-FX
(
Clinical study
of
Ca
nnabidiol (CBD) in Childr
en and
Adoles
cen
ts with
Fragile
X) trial of Zygel™ in
children and adolescents with Fragile X syndrome (FXS) in advance
of this week’s investor meetings in San Francisco.
Enrollment is nearing completion in CONNECT-FX, a
pivotal, multi-national, randomized, double blind,
placebo-controlled trial evaluating the efficacy and safety of
Zygel in treating common behavioral symptoms of FXS in three
through 17-year old patients with FXS. As of January 10, 2020, 178
patients of the 204 targeted for enrollment in the trial have been
randomized into the trial. There are also 15 patients who have been
screened but not yet randomized. The Company anticipates that
screening for the trial will close near the end of January.
“We have made great progress throughout our
pipeline in recent months,” said Armando Anido, Chairman and Chief
Executive Officer of Zynerba. “We are nearing completion of
enrollment in our pivotal CONNECT-FX study in patients with Fragile
X syndrome, and we expect to report results from this trial late in
the second quarter of 2020. We believe that the prospective
inclusion criteria have enabled us to execute on a rigorous
clinical trial of Zygel in a more severely impacted population of
children and adolescents than included in our Phase 2 FAB-C study,
which should enhance the study’s ability to demonstrate a strong
signal of activity and minimize response variability. With key
milestones expected from each of our clinical programs in the first
half of this year, 2020 has the potential to be a compelling and
exciting year.”
Baseline Data for Patients Randomized Through January 10,
2020
The primary endpoint for this trial 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 include 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
the Clinical Global Impression – Improvement scale (CGI-I) anchored
to FXS behaviors evaluated at the end of the treatment
period.
As intended and prospectively designed, the trial has enrolled a
more severely affected population than that 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 patients
randomized through January 10, 2020 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: 18.5 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, caregivers of patients in the trial are
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, 96% of the 141 patients who have completed
CONNECT-FX have enrolled in the open label extension trial.
Of the 178 patients randomized as of January 10, 2020, 135 (76%)
are male and the mean age in the study is 9.6 years.
The Company expects to disclose 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.
Other Corporate Updates
- As announced in a separate press release earlier this morning,
the Company has achieved its enrollment target of 36 patients in
the Phase 2 BRIGHT trial of Zygel for the treatment of pediatric
and adolescent patients with autism spectrum disorder (ASD).
Topline results are expected in the second quarter of 2020;
- Zynerba remains on track to report top line results from the
Phase 2 INSPIRE study of Zygel in 22q11.2 deletion syndrome (22q)
in the second quarter of 2020;
- The Company expects to meet with the FDA in the first half of
2020 to discuss the clinical path forward for Zygel in the
treatment of developmental and epileptic encephalopathies
(DEE);
- As of September 30, 2019 Zynerba had $77.5 million in cash and
cash equivalent, which it believes is sufficient to fund its
operations and capital requirements into the second half of
2021.
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 including ASD 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
Pharmaceuticals484.581.7489robertsw@zynerba.com
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