Zynerba Pharmaceuticals, Inc. (NASDAQ:ZYNE), the leader in
innovative pharmaceutically-produced transdermal cannabinoid
therapies for rare and near-rare neuropsychiatric disorders, today
announced positive top line results from the exploratory, open
label Phase 2 BRIGHT (An Open-La
bel
Tole
rab
ility and Efficacy Study
of ZYN002 Administered as a Transdermal
Gel to
C
hildren and Adolescen
ts with
Autism Spectrum Disorder) trial.
The trial was designed to assess the safety, tolerability and
efficacy of Zygel™ in pediatric and adolescent patients with autism
spectrum disorder (ASD). Zygel was administered to patients with
moderate-to-severe symptoms of ASD as add-on therapy to their
standard of care utilizing a variety of efficacy assessments. Key
findings from the trial disclosed today include:
- All five subscales of the Aberrant Behavior Checklist –
Community (ABC-C) as well as the Parent Rated Anxiety Scale -
Autism Spectrum Disorder (PRAS-ASD) showed both statistically
significant and clinically meaningful improvements at 14 weeks of
treatment from baseline;
- The results observed in other efficacy outcome measures,
including Clinical Global Impressions - Improvement scale (CGI-I),
support the subscale results observed in the ABC-C;
- Zygel was well tolerated in this trial with no serious or
severe adverse events reported.
“We are very encouraged by the compelling top line results of
the BRIGHT trial and we expect to meet with the FDA to discuss the
clinical pathway for developing Zygel for the treatment of
behavioral symptoms of ASD in the second half of this year,” said
Armando Anido, Zynerba’s Chairman and Chief Executive Officer. “Our
goal is to develop Zygel for patients suffering from debilitating
neuropsychiatric disorders including ASD, Fragile X syndrome, 22q
and DEE. I want to thank the patients, families, physicians,
clinical staff, and the Zynerba team for their support of this key
study in ASD."
Study Design
The 14-week exploratory, open label Phase 2 BRIGHT trial
enrolled 37 patients with ASD at a single clinical site. The trial
was designed to evaluate the safety, tolerability and efficacy of
Zygel in children and adolescents ages three to 17 with ASD as
confirmed by DSM-5 diagnostic criteria. Enrolled patients received
weight-based doses of 250 mg or 500 mg daily of Zygel.
Patient Disposition and Baseline
Demographics
Thirty-seven (37) patients were enrolled in the trial and are
included in the safety analysis. One patient was lost to follow up
with no post-treatment efficacy evaluation and, as a result,
thirty-six (36) patients are included in the efficacy analyses.
Twenty-eight (28) patients completed the 14-week trial. The
discontinuation rate is consistent with other trials in ASD.
The mean age of patients enrolled in the trial was 9.2 years and
thirty-four (92%) of the patients were male. Patients weighed
between 15 and 108 kilograms (mean=41.6; median=30.2). The mean
time to diagnosis in this population was 5.4 years. Using the
Autism Diagnostic Observation Schedule (ADOS-2), 94% of enrolled
patients had moderate-to-severe symptoms of ASD. The mean
baseline ABC-C Irritability subscale score of 30.3 further supports
the severity of the enrolled patient population. Ninety-two percent
(92%) of patients entered the trial with the use of at least one
underlying medication. Sixty-five percent (65%) of patients were on
at least one psychotropic medication, for example,
anti-depressants, anxiolytics and antipsychotics.
Top-line Efficacy Results
The trial evaluated multiple efficacy assessments, including the
ABC-C, PRAS-ASD, Autism Parenting Stress Index, Autism Impact
Measure (AIM), and Clinical Global Impression – Severity (CGI-S)
and Improvement (CGI-I). The ABC-C irritability subscale was used
as the basis for approval for the two atypical antipsychotics
indicated for ASD.
Results from each of the subscales of the ABC-C after 14 weeks
of treatment with Zygel are as follows:
ABC-C
Subscale |
|
Baseline(n=36) |
|
Week 14(n=28) |
|
|
Mean % improvement |
|
|
p Value |
|
|
|
|
|
|
|
|
|
|
|
Irritability |
|
30.3 |
|
18.2 |
|
|
39.1% |
|
|
<0.0001* |
Inappropriate Speech |
|
7.4 |
|
5.2 |
|
|
42.5% |
|
|
0.0002* |
Stereotypy |
|
12.3 |
|
7.9 |
|
|
39.1% |
|
|
<0.0001* |
Social withdrawal |
|
25.1 |
|
16.5 |
|
|
36.4% |
|
|
<0.0001* |
Hyperactivity |
|
37.0 |
|
23.9 |
|
|
35.6% |
|
|
<0.0001* |
|
|
|
|
|
*Statistically
significant |
|
|
|
|
The results of other efficacy assessments reinforce the results
demonstrated in the ABC-C. For example, patients on Zygel
experienced a mean improvement of 46% at week 14 from a baseline
score of 40.8 as measured by the PRAS-ASD (p<0.0001) and 57% of
patients were assessed as “very much improved” or “much improved”
at week 14 as measured by the CGI-I.
“I am very impressed with the improvements my patients made over
the 14-week treatment period while receiving Zygel; the reduction
in irritability, communication deficits, and repetitive movements
were especially noteworthy since some of these are core autistic
behaviors,” said Helen Heussler, FRACP, Associate Professor at
Children’s Health Queensland, Medical Director Child Development
and principal investigator in the BRIGHT trial. “The magnitude
of effect on autistic behaviors in this trial is significant,
including hyperactivity and stereotypy, which are among the most
difficult behaviors to improve with therapeutic intervention. The
results of this study strongly suggest the potential of this drug
as an important treatment for ASD and I look forward to
participating in future clinical studies with Zygel.”
Safety and Tolerability
Zygel was very well tolerated, and the safety profile was
consistent with previously released data from other Zygel clinical
trials. Less than half (49%) of the patients experienced any
adverse event (whether unrelated or related to study drug), all of
which were mild (75%) or moderate (25%). Only 14% of patients
experienced an adverse event that was deemed to be
treatment-related, all of which were application site-related; most
were mild and transient. There were no severe or serious adverse
events reported during the study. Eighteen (18) patients who
completed the BRIGHT trial have rolled into the open label
extension.
Conference call information
Zynerba management will host a live conference call and
webcast today at 8:30 am Eastern Time to discuss the results
of this clinical trial. The call can be accessed by dialing (866)
573-0180 (U.S. and Canada) or (430) 775-1345 (international) and
referencing conference ID 6196218. To access the live webcast or
the replay, visit the investor page of the Company’s website
at http://ir.zynerba.com/. The webcast will be recorded and
available on the Company’s website for 30 days.
About Autism Spectrum Disorder (ASD)
Autism Spectrum Disorder is a developmental disorder that
affects communication and behavior in approximately one million
pediatric and adolescent patients between the ages of five and 17
in the U.S. It refers to a range of conditions characterized by
anxiety, repetitive patterns of behavior, impairments in social
communication including verbal and non-verbal communication, and
deficits in developing and maintaining relationships. Although
autism can be diagnosed at any age, it is said to be a
“developmental disorder” because symptoms generally appear in the
first two years of life. Research suggests that genes can act
together with influences from the environment to affect development
in ways that lead to ASD. Newer studies suggest that ASD is linked
to disruption in the endocannabinoid system.
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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