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 on the safety, efficacy 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. These data are being presented at
the 2020 American Academy of Neurology (AAN) Science Highlights
Virtual Session. The Virtual Session is online at
http://www.aan.com/2020science. A copy of the posters are also
available on the Zynerba corporate website at
http://zynerba.com/publications/. The top line results of the
26-week Phase 2 BELIEVE trial were initially announced in September
2019. (Press release)
“These BELIEVE data continue to build on the strong safety and
tolerability profile of Zygel that we’ve seen across our clinical
trial programs, and provide evidence of its anti-seizure activity
in children and adolescents suffering from developmental and
epileptic encephalopathies, or DEEs,” said Zynerba’s Chief Medical
Officer, Joseph M. Palumbo, MD, FAPA, MACPsych. “These data are
particularly exciting because they also suggest its potential to
improve behavioral, cognition and mood deficits in these children
and adolescents, which may improve the quality of life for these
children and their family.”
The first poster entitled Cannabidiol Transdermal Gel in
Children and Adolescents with Developmental and Epileptic
Encephalopathies: An Open‐Label Clinical Trial further describes
the safety and efficacy of Zygel in children and adolescents with
developmental and epileptic encephalopathies (DEE) who participated
in the Phase 2 BELIEVE trial.
Forty-eight (48) patients with a mean age of 10.5 years
were enrolled in BELIEVE and included in the safety analysis.
Nearly three-quarters of patients (73%) had a diagnosis other than
LGS or Dravet syndrome. Patients with DEE are medically fragile.
Clinically important comorbid conditions were present in all
patients and included gait and movement disorders (46%), sleep
disturbances (40%), chronic respiratory conditions/infections
(38%), and PEG/feeding tube (15%). The modified intent-to-treat
(mITT) population comprised 46 patients; 33 patients in the mITT
population had consciousness-impairing seizures (focal impaired
awareness seizures [FIAS]; or convulsive, or tonic-clonic, seizures
[TCS] including generalized tonic-clonic seizures [GTCS] and focal
to bilateral tonic-clonic seizures [BTCS]) at baseline and
constituted the population in which the primary efficacy endpoint
was measured.
As described in the poster, the study met its primary efficacy
objective: Over the BELIEVE 26-week treatment period, the median
percentage reduction from baseline in monthly frequency of FIAS and
TCS was 43.5% (primary efficacy endpoint). Reduction from baseline
in monthly (28-day) seizure frequency was ≥44% from month two
onwards using monthly seizure frequency normalized to 28 days
(SF28). Monthly (28-day) reductions from baseline in seizure
frequency ranged from 44% to 58% from month two of the treatment
period onward. When analyzed by seizure type, the median reductions
from baseline at month six for FIAS, GTCS, and BTCS were 45.3%,
59.5%, and 58.7%, respectively.
At six months of treatment in the BELIEVE trial, 62% of patients
achieved a ≥35% reduction in FIAS and TCS from baseline, and 55% of
patients achieved a ≥50 reduction in FIAS and TCS.
An infographic accompanying this announcement is available
at https://www.globenewswire.com/NewsRoom/AttachmentNg/00997558-ff26-481a-b11c-61875243414d
SafetyZygel was well tolerated in this 26 week
Phase 2 study. Adverse events (AEs) are common in this medically
fragile patient population, and expected in a 26-week trial. All
events in the six-month period, whether unrelated or related to
study drug, were reported as AEs (e.g.: influenza, infections,
scrapes, etc.). As a result and as anticipated, most patients
experienced an adverse event during the 26-week BELIEVE trial. No
safety signal was identified. The most common treatment-related
adverse events occurred in only four patients each: application
site dryness, application site pain, and somnolence (all four
patients exhibiting somnolence were taking concomitant clobazam).
Ten (10) patients reported a serious adverse event (SAE); most were
infection-related and unrelated to study drug. Of the 10, two
patients experienced an SAE that was determined to be possibly
related to treatment (nonconvulsive status epilepticus and lower
respiratory tract infection, both of which are common in patients
with DEE). All SAEs recorded during the study resolved and did not
require dose alteration. There were no clinically significant
changes in vital signs, ECGs, or laboratory findings. No
drug-related increases in liver function tests (LFTs) were
observed.
The authors of the poster concluded that:
- These data suggest meaningful reductions in FIAS and TCS with
Zygel treatment beginning as early as month two and sustained
through 26 weeks;
- Zygel was well tolerated over 26 weeks of treatment in a
medically fragile patient population of children and adolescents
with DEEs;
- 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 Qualitative
Caregiver Assessments in Children and Adolescents with
Developmental and Epileptic Encephalopathies Treated With
Cannabidiol Transdermal Gel: An Open-Label Clinical Trial builds on
the previously disclosed data suggesting the potential of Zygel to
improve the quality of life of patients living with DEE. The key
assessments for Quality of Life (QoL) in the open label BELIEVE
trial included the Epilepsy and Learning Disabilities Quality of
Life (ELDQOL) scale - modified version, daily “good day/bad day”
questionnaire, and qualitative caregiver
assessment.ELDQOLStatistically significant reductions from baseline
in mean ELDQOL-modified subscale scores for seizure severity,
behavior, and mood were observed at week 26 (P < 0.01 for
all measures).Good Day/Bad Day Assessment At month six, the
combined proportion of “good day” and “fantastic day” reports
increased from 52% at baseline to 70%, and the combined proportion
of “terrible day” and “bad day” reports decreased from 12% at
baseline to 4%.
An infographic accompanying this announcement is available
at https://www.globenewswire.com/NewsRoom/AttachmentNg/5afa2b80-2b14-49f0-bccd-f62c93a1583b
Qualitative Caregiver FeedbackThe qualitative caregiver
assessment was administered to parents/caregivers for 43 patients.
Improvement in summary measures of qualitative assessments was
observed in most patients for most measures:
- Any improvement: 84% (n = 36)
- Improved vitality: 58% (n = 25)
- Improvement in seizures: 51% (n = 22)
- Improved cognition/concentration: 47% (n = 20)
- Improved socially avoidant behaviors: 44% (n = 19)
- Improvement in irritability: 33% (n = 14)
- School improvement: 28% (n = 12)
- Medical improvement: 14% (n = 6)
The most frequent positive qualitative statements were in the
domains of (1) behavior, cognition, and mood, and (2) seizures.
Some caregivers reported difficulty in applying gel (26%) and mild
skin reaction issues (19%). The authors concluded that treatment
with Zygel may be associated with clinically meaningful
improvements in social behaviors and cognitive symptoms and
increased QoL in children and adolescents with DEEs and their
families.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 ContactWill Roberts, VP Investor
Relations and Corporate
Communications484.581.7489robertsw@zynerba.com
Media contactMolly DevlinEvoke
KYNE215.928.2199Molly.Devlin@evokegroup.com
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