- Patients Experienced a 77% Improvement in
Aberrant Behaviors Associated with Social Avoidance Compared to
Baseline after One Year of ZYN002 Treatment -
Zynerba Pharmaceuticals, Inc. (NASDAQ:ZYNE), the leader in
innovative pharmaceutically-produced transdermal cannabinoid
therapies for rare and near-rare neuropsychiatric disorders, is
reporting new 12-month open label clinical data describing the long
term impact of ZYN002 on emotional and behavioral symptoms of
Fragile X Syndrome (FXS) in a poster presentation at the 57th
Annual Meeting of the American College of Neuropsychopharmacology.
The presentation is taking place today from 5:30 to 7:30 PM EST in
poster session III at the Diplomat Beach Resort in Hollywood,
Florida. A copy of the presentation and poster are available on the
Zynerba corporate website at http://zynerba.com/publications/.
In a poster entitled, “Transdermal Cannabidiol (CBD) Gel for the
Treatment of Fragile X Syndrome (FXS),” Steven Siegel, M.D., Ph.D.,
Professor and Chair of the Department of Psychiatry and the
Behavioral Sciences at the Keck School of Medicine, University of
Southern California, is presenting new 12-month data from the open
label Phase 2 FAB-C (Treatment of Fragile X
Syndrome Anxiety and Behavioral
Challenges with CBD) trial of ZYN002 in children
and adolescents with FXS. The data demonstrate that treatment with
ZYN002 improved core emotional and behavioral symptoms of FXS with
statistical significance versus baseline across multiple measures
of efficacy at month three, and that these improvements were
sustained through 12 months of treatment. ZYN002 continues to be
well tolerated; no serious adverse events were reported, and no
clinically meaningful trends in vital signs, ECG, or clinical
safety laboratories, including liver function tests, were
observed.
In the Social Avoidance subscale of the Aberrant Behavior
Checklist for Fragile X (ABC-CFXS), patients completing 12 months
of treatment with ZYN002 experienced a 77.2% improvement in social
avoidance behaviors versus baseline, compared to a 57.9%
improvement at three months of treatment. Both results are
statistically significant compared to baseline. The Social
Avoidance subscale of the ABC-CFXS is the primary endpoint of the
ongoing pivotal CONNECT-FX study of ZYN002.
“I am encouraged to see that improvements in FXS-associated
emotional and behavioral symptoms after 12 months of treatment with
ZYN002 are consistent with those seen at three and nine months;
these data continue to suggest the potential for sustained
responses that may be conserved over extended use of the drug,”
said Dr. Steven Siegel of the Keck School of Medicine.
“Improvements in these behaviors may enhance the child’s capacity
for interaction and engagement with their peers, families, teachers
and caregivers. These data are promising, and I am enthusiastic
about the potential opportunity for ZYN002 in these patients. I
look forward to the results of the double blind, placebo-controlled
CONNECT-FX study next year.”
Study design
Twenty patients (3:1 males) aged six through 17 years of age
(median = 9) with Fragile X Syndrome were enrolled in the open
label FAB-C study. All patients had genetic confirmation of the
full mutation of the FMR1 gene. ZYN002 was added to other
medications being administered. The first six weeks were designed
to titrate dosing in patients. Dosing was initiated at 50 mg daily
and could be increased to 250 mg daily. Weeks seven through 12 was
a maintenance period where patients were treated at the dose
established at week six. At the completion of week 12, thirteen
patients elected to enter into the extension study for up to 24
months. To date, 11 patients remain in the study and have now
exceeded 12 months of therapy with ZYN002.
The primary endpoint of the FAB-C trial was the Anxiety,
Depression, and Mood Scale (ADAMS) Total Score. Key secondary
endpoints included the ADAMS subscale scores for Social Avoidance,
Manic/Hyperactive Behavior, Depressed Mood, General Anxiety, and
Compulsive Behavior; and the Aberrant Behavior Checklist FXS Factor
Structure (ABC-CFXS) subscale scores of Social Avoidance,
Irritability, Socially Unresponsive/Lethargic, Hyperactivity,
Stereotypy, and Inappropriate Speech.
Long Term Efficacy: 12 months
The following data show the improvement in various efficacy
measures for the patients who completed three months, enrolled in
the extension trial, and have completed 12 months of treatment.
Anxiety, Depression, and Mood Scale
(ADAMS) |
|
Scale:
ADAMS |
Group Mean Percent
Improvement from Baseline |
Month
3(n=12) |
P-value vs
Baseline |
Month
9 (n=12) |
P-value vs
Baseline |
Month
12 (n=12) |
P-value vs
Baseline |
ADAMS Total Score |
48.6 |
0.0001 |
59.2 |
<0.0001 |
54.4 |
<0.0001 |
Social Avoidance |
52.5 |
0.0013 |
61.6 |
0.0007 |
55.6 |
0.0004 |
Manic/Hyperactive Behavior |
34.1 |
0.0012 |
53.4 |
0.0002 |
45.5 |
0.0014 |
Depressed Mood |
43.8 |
0.0831 |
62.5 |
0.0372 |
59.4 |
0.0032 |
General Anxiety |
55.1 |
<0.0001 |
58.2 |
<0.0001 |
58.2 |
<0.0001 |
Compulsive Behavior |
50.0 |
0.0295 |
59.4 |
0.0247 |
59.4 |
0.0213 |
A photo accompanying this chart is available
at: http://www.globenewswire.com/NewsRoom/AttachmentNg/95ff728a-4470-41b3-9659-5b1c336c148b
|
Aberrant Behavior Checklist – Community: FXS
Specific (ABC-CFXS) |
|
Scale:
ABC-CFXS |
Group Mean Percent
Improvement from Baseline |
Month
3(n=12) |
P-value vs
Baseline |
Month
9(n=9) |
P-value vs
Baseline |
Month
12(n=9) |
P-value vs
Baseline |
Social Avoidance |
57.9 |
0.0040 |
75.4 |
0.0013 |
77.2 |
0.0013 |
Irritability |
51.1 |
0.0012 |
63.7 |
0.0003 |
59.2 |
0.0007 |
Socially Unresponsive/Lethargic |
65.7 |
0.0024 |
83.3 |
0.0016 |
72.2 |
0.0035 |
Hyperactivity |
36.7 |
0.0119 |
48.2 |
0.0012 |
40.4 |
0.0037 |
Stereotypy |
60.8 |
0.0048 |
73.2 |
0.0019 |
64.9 |
0.0012 |
Inappropriate Speech |
56.5 |
0.0002 |
66.1 |
<0.0001 |
56.5 |
<0.0001 |
A photo accompanying this chart is available
at: http://www.globenewswire.com/NewsRoom/AttachmentNg/fced1b83-4584-4d28-9a38-f6945e0bc98b
Safety Summary
ZYN002 was well tolerated, and the safety profile was consistent
with previously reported clinical data, with no serious adverse
events (SAEs) reported. Through month 12, patients reported 43
treatment-emergent adverse events (TEAEs), all of which were mild
or moderate. Two out of 20 patients, who were siblings,
discontinued during the initial three-month period; one
discontinued due to worsening eczema (not considered treatment
related) and the other discontinued for administrative reasons. In
the ongoing open label extension, there has been one
discontinuation for administrative reasons. The most common TEAEs
were gastroenteritis (14%) and upper respiratory tract infection
(12%). One patient developed moderate skin rash (alternate etiology
reaction to antibiotics) and one patient developed mild dry skin;
both resolved and the patients remained in the study. There has
been no THC detected in plasma. There have been no clinically
meaningful trends in vital signs, ECGs or clinical safety labs,
including liver function tests.
About ZYN002 Zynerba’s ZYN002 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). Enrollment is ongoing in a multi-national,
randomized, double blind placebo controlled
Clinical study of
Cannabidiol (CBD) in Children and
Adolescents with
Fragile X (CONNECT-FX), a pivotal
clinical trial of ZYN002 in FXS (ClinicalTrials.gov/CONNECTFX);
topline data from CONNECT-FX are expected in the second half of
2019. Additionally, Zynerba expects to complete enrollment in its
Phase 2 Open Label Study to Assess the Safety and
Efficacy of ZYN002 Administered as a
Transdermal Gel to Children and
Adolescents with Developmental
and Epileptic Encephalopathy (BELIEVE 1) clinical
trial before year-end 2018.
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. 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 and refractory epilepsies.
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;
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.
Investor Contact
Will Roberts, VP Investor Relations and Corporate
CommunicationsZynerba
Pharmaceuticals484.581.7489robertsw@zynerba.com
Zynerba Pharmaceuticals (NASDAQ:ZYNE)
Historical Stock Chart
From Mar 2024 to Apr 2024
Zynerba Pharmaceuticals (NASDAQ:ZYNE)
Historical Stock Chart
From Apr 2023 to Apr 2024