Zynerba Pharmaceuticals Announces Publication of Results of Phase 2 BELIEVE Open-Label Study of Zygel™ in Children and Adolescents with Developmental and Epileptic Encephalopathies (DEE) in JAMA Network Open
September 07 2021 - 7:00AM
Zynerba Pharmaceuticals, Inc. (Nasdaq: ZYNE), the leader in
innovative pharmaceutically-produced transdermal cannabinoid
therapies for rare and near-rare neuropsychiatric disorders, today
announced the publication of results from the Company’s open-label
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) study in the Journal of the American Medical
Association (JAMA) Network Open.
The article, titled “Safety and tolerability of
transdermal cannabidiol gel in children with developmental and
epileptic encephalopathies: A nonrandomized controlled trial” can
be accessed at HERE.
The article describes the positive results from
BELIEVE, the first trial of a non-oral formulation of cannabidiol
in children and adolescents with DEEs, and concludes that
cannabidiol transdermal gel was safe, well tolerated, and was
associated with reductions in focal impaired awareness seizures
(FIAS) and tonic-clonic seizure (TCS) frequency and disease
burden.
“DEEs are the most severe group of epilepsies
and are usually drug-resistant. Antiseizure medications are usually
administered orally, which can be extremely challenging in children
with behavioral and cognitive problems. Non-oral therapies are
needed to provide an alternative route to deliver medications to
control seizures and improve developmental outcomes,” said Ingrid
E. Scheffer, MBBS, PhD, FRS, Laureate Professor, and chair,
Pediatric Neurology Research, University of Melbourne, and the lead
investigator in the BELIEVE study. “The data from the BELIEVE study
are promising and suggest that Zygel may be a safe and
well-tolerated option to improve seizure control, challenging
behaviors and other symptoms associated with DEEs.”
Forty-eight (48) patients with a mean age of
10.5 years were enrolled in BELIEVE and included in the safety
analysis. Sixty-percent (60%) had at least one treatment-related
adverse event (AE) over the 6.5 month trial period and 96% of these
AE’s were mild or moderate. During the treatment period, 10
patients (21%) reported serious adverse events (SAEs). Two SAEs
were considered to be possibly treatment related: nonconvulsive
status epilepticus and lower respiratory tract infection, in
separate patients. All SAEs resolved, and none resulted in
alteration of study medication.
The authors indicated that the incidence of AEs
in this study, particularly related to infections, was likely due
to the high baseline rate of complex morbidities and seizure
severity of these patients at study onset, and to the relatively
long, 6.5 month duration of the trial over which events were
collected.
From an efficacy perspective, the analysis of
the 33 patients with FIAS and TCS showed a 58% median monthly
reduction in seizures at month 5, and 43.5% reduction over the 6.5
month study period (the primary efficacy endpoint). The percent of
≥50% responders in the FIAS and TCS group ranged from 42.4% at
month 2 and peaked at 62.5% at month 5. Parents/caregivers noted
improvements in social or interpersonal engagement and irritability
(33/43 [77%]); alertness, energy and sleep (23/43 [53%]); and
cognition or concentration (20/43 [47%]).
About Zynerba Pharmaceuticals,
Inc.
Zynerba Pharmaceuticals is the leader in
innovative 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.
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Zynerba Contacts
Peter VozzoWestwicke/ICROffice: 443.213.0505Cell:
443.377.4767Peter.Vozzo@Westwicke.com
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