Zynerba Pharmaceuticals, Inc. (NASDAQ:ZYNE), the leader in
innovative pharmaceutically-produced transdermal cannabinoid
therapies for rare and near-rare neuropsychiatric disorders, is
presenting data this week further describing the baseline
characteristics of the pediatric and adolescent patients in the
fully-enrolled Phase 2 BRIGHT trial of Zygel™ (CBD transdermal gel;
ZYN002) in children and adolescents with autism spectrum disorder
(ASD), indicating that the trial enrolled a broad population of
patients with moderate-to-severe ASD.
The poster entitled Phase 2 BRIGHT (An Exploratory
Open-Label
Tolerability and Efficacy Study
of ZYN002 Administered as a Transdermal Gel to
Children and Adolescents with
Autism Spectrum Disorder) Trial: Baseline Characteristics (poster
#27) is being presented at the American Society for Experimental
Neurotherapeutics (ASENT) 2020 Meeting. The poster is being
presented on Tuesday, March 3 from 5:00 to 6:00 PM EST and
Wednesday, March 4 from 5:00 to 7:00 PM EST. These data will also
be presented during the ASENT Pipeline Data Blitz session on
Wednesday March 4, 2020 from 1:00 to 3:00PM EST. The meeting is
being held in Bethesda, MD on March 2nd through March 5th, 2020. A
copy of the poster is available on the Zynerba corporate website at
http://zynerba.com/publications/.
Zynerba’s Chief Medical Officer, Joseph M. Palumbo,
MD, FAPA, MACPsych, is presenting data describing the baseline
characteristics of patients enrolled in the ongoing BRIGHT trial,
which indicate a patient population with predominantly
moderate-to-severe ASD as measured by key scales used for screening
and efficacy assessment. These include the Aberrant Behavior
Checklist – Community (ABC-C); the Autism Diagnostic Observation
Schedule® (ADOS-2); and the Parent Rated Anxiety Scale–Autism
Spectrum Disorder (PRAS-ASD).
“ASD is a complex neurodevelopmental disorder
characterized by difficulties with behaviors, communication, and
social interaction,” said Dr. Palumbo. “Pediatric and
adolescent patients with ASD may also present with profound
clinical anxiety, above the rate seen in neurotypical children,
further complicating their condition and treatment regimen.
Unfortunately, current ASD management options are restricted to
cognitive behavioral therapy and a small number of approved
pharmacologic treatments, highlighting the substantial unmet need
for novel therapies in this population. We believe that we have
enrolled an appropriate population of patients into our
well-designed exploratory BRIGHT trial to enable a robust analysis
of outcomes to help inform the design of future double-blind,
placebo-controlled studies.”
The endocannabinoid system - a key modulator of
emotion and social behavior - is dysregulated in ASD, and published
data suggest that cannabidiol (CBD) may provide therapeutic
benefit. However, the efficacy and safety of CBD in patients with
ASD have not been well established. Zynerba is undertaking the
14-week BRIGHT Phase 2 exploratory trial in children and
adolescents (ages four through 17 years) with ASD as confirmed by
Diagnostic and Statistical Manual of Mental Disorders, 5th edition
(DSM-5) diagnostic criteria to assess the safety and efficacy of
Zygel in treating ASD-related behaviors as measured by a variety of
efficacy assessments which are shown in Figure 1, below. After
completing dosing in the 14-week period, participants may enroll in
a six-month extension trial.
Figure 1. Schedule of Screening and Efficacy
Assessments is available
at https://www.globenewswire.com/NewsRoom/AttachmentNg/a15d5dd4-9ab2-497d-a8ec-7782f612ebca.
The trial protocol included certain inclusion and exclusion
criteria to enrich the trial population for disease severity at
baseline, as measured by the following assessments:
ABC-C
- A 58-item caregiver-rated scale measuring behaviors across 5
subscales: irritability/agitation (maximum score: 45),
lethargy/social withdrawal (maximum score: 48), stereotypic
behavior (maximum score: 21), hyperactivity/noncompliance (maximum
score: 48), inappropriate speech (maximum score: 12);
- Each behavior is scored from 0 (“not at all a problem”) to 3
(“the problem is severe in degree”);
- Higher scores indicate greater severity of aberrant
behavior.
ADOS-2
- A diagnostic tool consisting of 5 age - and verbal ability -
dependent modules that assess social communication and core
behaviors of ASD;
- Each item is scored by a trained test administrator from 0 (“no
abnormality of type specified”) to 3 (“moderate to severe
abnormality”);
- ADOS total scores are diagnostic; however, standardized
comparison scores can be used to measure severity;
- Comparison scores range from 0-10, with scores of <5
indicating mild ASD, scores of 5-7 indicating moderate ASD, and
scores of 8-10 indicating severe ASD.
PRAS-ASD
- A 25-item parent-rated scale assessing anxiety in ASD;
- Each item is scored from 0 (“not present”) to 3 (“very frequent
and a major problem”);
- Maximum score is 75, with scores >52 indicating possible
clinical anxiety.
Baseline Disease CharacteristicsAs seen in
Table 1 below, the majority of patients had moderate or severe ASD
at baseline as measured by the ADOS-2 comparison score (94%) and
DSM-5 severity levels (92%). In addition, the mean ABC-C
Irritability score was 30.0, and 24% of the enrolled patients had
PRAS-ASD scores indicative of possible clinical anxiety, further
highlighting the severity of symptoms in the enrolled patient
population.
Table 1. Baseline Disease Characteristics of Patients Enrolled
in BRIGHT
Disease Characteristics |
Patients in BRIGHTN=37 |
|
ABC-C Irritability Subscale score (0-45) |
|
|
n |
37 |
|
Mean (range) |
30.0 (18-43) |
|
PRAS-ASD score (0-75; >52 suggests possible clinical
anxiety) |
|
|
n |
37 |
|
Mean (range) |
40.9 (21-68) |
|
>52, n (%) |
9 (24.3) |
|
DSM-5 severity leveli |
|
|
Level 1 (mild), n (%) |
3 (8.1) |
|
Level 2 (moderate), n (%) |
15 (40.5) |
|
Level 3 (severe), n (%) |
19 (51.4) |
|
ADOS®-2 comparison score |
|
|
n |
36 |
|
Mean (range) |
7.5 (4-10) |
|
<5 (mild ASD), n (%) |
2 (5.6) |
|
5-7 (moderate ASD), n (%) |
19 (52.8) |
|
8-10 (severe ASD), n (%) |
15 (41.7) |
|
|
|
|
The authors conclude that the Phase 2 BRIGHT trial has
successfully enrolled a broad patient population and was enriched
for disease severity to avoid floor effects on outcome measures.
The baseline characteristics indicate a patient population with
predominantly moderate-to-severe ASD, with a high level of
clinically significant 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
Statements
This press release contains forward-looking statements within
the meaning of The Private Securities Litigation Reform Act of
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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
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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
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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
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including the annual report on Form 10-K, quarterly reports on Form
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Zynerba ContactWill Roberts, VP Investor
Relations and Corporate
Communications484.581.7489robertsw@zynerba.com
Media contactMolly DevlinEvoke
KYNE215.928.2199Molly.Devlin@evokegroup.com
_______________i DSM-5 severity levels are based on degree of
social communication impairment and behavioral flexibility. The
levels indicate patients “requiring support” (level 1), “requiring
substantial support” (level 2), and “requiring very substantial
support” (level 3).
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