Zynerba Pharmaceuticals, Inc. (NASDAQ:ZYNE), the leader in
innovative pharmaceutically-produced transdermal cannabinoid
therapies for rare and near-rare neuropsychiatric disorders, is
presenting two posters describing the efficacy, safety and quality
of life results of the Phase 2 BELIEVE (Open La
bel
Study to Assess the Safety and
Efficacy of Zygel™
(ZYN002) Administered as a Transderma
l Gel to
Ch
ildren and Adol
escents with
De
velopmental and
Epileptic
Encephalopathy) clinical trial at the 2020 virtual annual meeting
of the American Epilepsy Society (AES). Both posters are available
on the Zynerba corporate website at
http://zynerba.com/publications/.
“These newly reported data are exciting as they describe the
compelling anti-seizure activity of Zygel in children and
adolescents suffering from developmental and epileptic
encephalopathies, or DEEs, through twelve months of treatment,”
said Zynerba’s Chief Medical Officer, Joseph M. Palumbo, MD, FAPA,
MACPsych. “This is particularly exciting, as this trial was
conducted in a refractory seizure population in which patients
failed to show adequate response despite taking as many as four
anti-seizure drugs. Additionally, we observed an equally compelling
seizure reduction in children with a coexisting diagnosis of autism
spectrum disorder.”
The first poster entitled, “ZYN002 Cannabidiol Transdermal Gel
in Children and Adolescents With Developmental and Epileptic
Encephalopathies: An Open-Label Clinical Trial [BELIEVE
(ZYN2-CL-025)]” describes the evidence of tolerability and efficacy
of Zygel observed in children and adolescents with developmental
and epileptic encephalopathies (DEE) who participated in the Phase
2 BELIEVE trial treated for up to 12 months.
As shown in Figure 1, new results presented today include
12-month treatment data showing improvements in the median
percentage reduction from baseline in monthly frequency of
consciousness-impairing seizures (focal impaired awareness
seizures, or FIAS, and tonic-clonic seizures, or TCS, including
generalized tonic-clonic seizures [GTCS] and focal to bilateral
tonic-clonic seizures [FBTCS]), ranged from 44% at month three to
73% at month 12.
Figure 1: Median
Percentage Reduction From Baseline in 28-Day
Frequency of FIAS and TCS by Time Point, Patients With FIAS
and/or TCS at Baseline
https://www.globenewswire.com/NewsRoom/AttachmentNg/774e0c2b-497d-4d92-87a2-c1341361cc52
When analyzed by seizure type, median reductions from baseline
at month six for FIAS, GTCS, and FBTCS were 45%, 60%, and 59%,
respectively. At month 12, the median reductions for FIAS, GTCS and
FBTCS were 100%, 83% and 59% respectively.
Additionally, as shown in Figure 2, a substantial percentage of
patients achieved ≥35% and ≥50% reduction in FIAS and TCS by month
three (58% and 46%, respectively), and these reductions in seizures
continued through month 12 (89% and 83%, respectively).
Figure 2: Percentage
of Patients With 35% and 50%
Reduction in
FIAS and TCS by Time Point, Patients With FIAS
and/or TCS at Baseline
https://www.globenewswire.com/NewsRoom/AttachmentNg/51acd725-7207-4e96-9f95-76e8649a27e8
Epileptic encephalopathies represent a particularly severe form
of epilepsy, associated with cognitive and behavioral deficits,
including impaired social-communication and restricted, repetitive
behaviors that are the hallmarks of autism spectrum disorder
(ASD)1. Zynerba conducted an exploratory analysis to evaluate the
efficacy of Zygel in reducing seizures in DEE patients with
coexisting ASD enrolled in BELIEVE.
As seen in Figure 3, over the 12-month treatment period, the
median percentage reduction from baseline in monthly frequency of
consciousness-impairing seizures (FIAS or TCS) in patients with DEE
and ASD improved over time, and these reductions in seizures ranged
from 45% at month three to 74% at month 12.
Figure 3. Median
Percentage Reduction From Baseline in 28-Day
Frequency of FIAS and TCS by Time Point, Patients
With Coexisting ASD at
Baseline
https://www.globenewswire.com/NewsRoom/AttachmentNg/d2655600-bf08-4e50-b2ac-185fd1c77f16
As seen in Figure 4, a substantial percentage of patients
achieved ≥35% and ≥50% reduction in FIAS and TCS by month three
(60% and 40%, respectively), and these reductions continued through
month 12 (75%).
Figure 4: Percentage
of Patients With 35% and 50% Reduction in FIAS and
TCS by Time Point, Patients With
Coexisting ASD
at Baseline
https://www.globenewswire.com/NewsRoom/AttachmentNg/8d70258a-c987-48e9-a4bb-f645bc473a54
Zygel was well tolerated in BELIEVE. Most treatment-emergent
adverse events (any event, whether unrelated or related to study
drug) were mild or moderate. There were no clinically significant
changes in vital signs, ECGs, or laboratory findings except for one
patient with a transient, benign, isolated elevation of alkaline
phosphatase at week 26 that was not considered related to study
medication.
The authors of the poster concluded that:
- These data suggest meaningful reductions in FIAS and TCS with
Zygel treatment which is maintained through to 12 months of
treatment with Zygel;
- In the subgroup of patients with ASD, Zygel demonstrated
meaningful reductions in FIAS and TCS seizures, with most children
reaching either the 35% or 50% responder threshold by month three
and month six respectively;
- Zygel was well tolerated over 18 months of treatment in a
medically fragile patient population of children and adolescents
with DEEs; and
- The positive benefit/risk profile of Zygel in this trial
supports further study in patients with DEEs and FIAS
and TCS.
The second poster entitled, “Quality of Life and Sleep
Assessments in Children with Developmental and Epileptic
Encephalopathies Treated With ZYN002 (CBD) Transdermal Gel: BELIEVE
(ZYN2-CL-025)” describes the impact of Zygel on profound sleep
disturbances experienced by DEE patients enrolled in the BELIEVE
trial.
Dr. Palumbo continued, “These new data highlight the impact
Zygel has on the profound sleep disturbance often experienced by
DEE patients in this trial. Importantly, disrupted sleep in
children with epilepsy has previously been reported to be
associated with negative outcomes in overall family functioning.
The Sleep Disturbance Scale for Children (SDSC) utilized in the
BELIEVE trial shows that half of the enrolled children exhibited
clinically significant sleep problems at study baseline, which
improved while receiving Zygel.”
The Sleep Disturbance Scale for Children (SDSC) is a 26-item
scale completed by caregivers that assesses six different sleep
categories. The authors of the poster concluded that treatment with
Zygel may be associated with improvements in disorders of
initiating and maintaining sleep, disorders of arousal/nightmares,
sleep wake transitions, and overall sleep, as well as clinically
meaningful improvements observed in vitality,
cognition/concentration, socially avoidant behaviors, seizure
severity, behavior, and mood.
As shown in Figure 5 and Table 1 below, statistically
significant improvements from baseline in sleep scores were
observed in the Total Sleep Score (p=0.012), Disorders of
Initiating or Maintaining Sleep (DIMS; p=0.006), Disorder of
Arousal/Nightmares (DA; p=0.031), and Sleep Wake Transition
Disorder (SWTD; p=0.030).
Figure 5: The Sleep Disturbance Scale
for Children (SDSC) – Percentage of Patients Above
Threshold for Clinically Significant Sleep Problems at Baseline and
Week 26
https://www.globenewswire.com/NewsRoom/AttachmentNg/f1e79e50-2018-4a4a-a9c1-b27719635f5c
Table 1. Change From
Baseline in the SDSC
SDSC Factors |
t-Score Mean
(SD) |
Change(negative number is
improvement) |
P value |
Total Score |
|
|
|
Baseline (n=46) |
71.6 (12.68) |
|
|
Week 26 (n=37) |
63.9 (13.40) |
−5.1 |
0.012* |
Disorders of Initiating and Maintaining Sleep
(DIMS) |
Baseline (n=46) |
69.6 (14.67) |
|
|
Week 26 (n=38) |
63.2 (15.76) |
−5.1 |
0.006* |
Sleep Breathing Disorders (SBD) |
Baseline (n=46) |
60.6 (15.46) |
|
|
Week 26 (n=40) |
58.9 (15.08) |
0.40 |
0.797 |
Disorders of Arousal/Nightmares (DA) |
Baseline (n=46) |
51.5 (9.91) |
|
|
Week 26 (n=39) |
49.0 (5.07) |
−1.7 |
0.031* |
Sleep Wake Transition Disorder (SWTD) |
Baseline (n=46) |
65.0 (13.09) |
|
|
Week 26 (n=39) |
60.2 (13.78) |
−4.6 |
0.030* |
Disorders of Excessive Somnolence (DOES) |
Baseline (n=46) |
68.5 (16.76) |
|
|
Week 26 (n=40) |
63.1 (13.82) |
−3.6 |
0.100 |
Sleep Hyperhidrosis (SHY) |
Baseline (n=46) |
52.7 (12.28) |
|
|
Week 26 (n=40) |
50.6 (9.95) |
−2.8 |
0.154 |
*P<0.05 for change from baseline to week 26; statistically
significant
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
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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 ContactWill Roberts,
VP Investor Relations and Corporate
Communications484.581.7489robertsw@zynerba.com
Media contactMolly DevlinEvoke
KYNE215.928.2199Molly.Devlin@evokegroup.com
1 https://www.ncbi.nlm.nih.gov/pmc/articles/PMC5481888/
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