LONDON, May 25, 2017 /PRNewswire/ --
- Pharmaceutical
formulation of cannabidiol significantly reduced convulsive seizure
frequency in children on multiple anti-epileptic
drugs with poor seizure control -
- First
well-controlled clinical study of cannabidiol in Dravet
syndrome, a rare, severe type of epilepsy
with no FDA-approved treatments -
GW Pharmaceuticals plc (Nasdaq: GWPH, "GW," "the Company" or
"the Group"), a biopharmaceutical company focused on discovering,
developing and commercializing novel therapeutics from its
proprietary cannabinoid product platform, along with its U.S.
subsidiary Greenwich Biosciences, announced today that The New
England Journal of Medicine has published results from a Phase
3 study of Epidiolex® (cannabidiol) in children with
Dravet syndrome.[1] Epidiolex, GW's lead product
candidate and the potential first in a new category of
anti-epileptic drugs, is a liquid formulation of purified,
plant-derived cannabidiol (CBD), a non-psychoactive cannabinoid,
which is being studied for the treatment of a number of rare,
severe pediatric-onset epilepsy disorders. In the study, Epidiolex
significantly reduced monthly convulsive seizure frequency compared
to placebo in highly treatment-resistant children when added to
existing treatment. Treatment with Epidiolex was generally well
tolerated, with a safety profile consistent with prior open label
experience.
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There are currently no treatments approved by the U.S. Food and
Drug Administration (FDA) for Dravet syndrome, a rare form of
epilepsy associated with a high mortality rate and significant
developmental delays. Results from this study represent the only
well-controlled clinical evaluation of a cannabinoid medication for
this devastating and drug-resistant condition. The New Drug
Application for Epidiolex remains on track for submission to the
FDA in the middle of 2017.
"Dravet syndrome is one of the most difficult types of epilepsy
to treat and many of the children in this study were experiencing
dozens, even hundreds, of seizures per month despite taking
multiple concurrent anti-epileptic medications," said Orrin Devinsky, M.D., of NYU Langone Medical
Center's Comprehensive Epilepsy Center and lead author of the
study. "These results suggest that Epidiolex can provide
clinically meaningful benefits and I look forward to the prospect
of an appropriately standardized and tested pharmaceutical
formulation of cannabidiol available as a treatment option for
these patients."
"The publication of these highly-anticipated positive results
represents an important milestone for the Dravet syndrome community
in that it provides hope that a new treatment option is within
sight for this rare and devastating disease," said Nicole Villas, Scientific Director of the Dravet
Syndrome Foundation. "As a foundation dedicated to research and
patient assistance, we hope the burden of Dravet syndrome on
patients and families is recognized and welcome new,
well-researched treatments such as this that might help ease the
burden."
The study randomized 120 children ages two to 18 years (mean age
9.8), with Dravet syndrome whose seizures were not controlled by
their current anti-epileptic regimen, to receive either Epidiolex
(20mg/kg/day) or placebo in addition to standard treatment.
Conducted in 23 study centers in the
United States and Europe,
patients in the study had tried a median of four prior
anti-epileptic drugs (range 0-26) and were taking a median of three
(range 1-5) during the study. At baseline, patients had a median
frequency of 13 convulsive seizures per month, with a wide range of
3.7 to 1,717 seizures per month.
Over the 14-week treatment period, patients taking Epidiolex had
a significantly greater median reduction in convulsive seizures (39
percent) compared to placebo (13 percent). The estimated median
treatment difference between groups was 23 percent (p=0.01). The
proportion of patients who had a 50 percent or better reduction in
convulsive seizure frequency was 43 percent with Epidiolex versus
27 percent with placebo (p=0.08). The study also measured
improvement on the Caregiver Global Impression of Change (CGIC)
scale. Sixty-two percent of patients treated with Epidiolex were
rated as improved in overall condition on the CGIC compared to 34
percent of the placebo group (p=0.02). Additionally, more patients
became seizure-free on Epidiolex than placebo (5 percent vs 0
percent: p=0.08) and total monthly seizure count was significantly
reduced with Epidiolex (p=0.03).
Epidiolex was generally well tolerated in the trial. The
most common adverse events (AEs) (>10 percent) were somnolence,
diarrhea, decreased appetite, fatigue, vomiting, pyrexia, lethargy,
convulsion, upper respiratory tract infection. Of the 93 percent of
patients on Epidiolex that experienced an AE, 84 percent reported
it to be mild or moderate. Ten patients on Epidiolex experienced a
serious adverse event compared with three patients on placebo.
Eight patients on Epidiolex discontinued treatment due to adverse
events compared with one patient on placebo. Elevations in liver
enzymes occurred in 12 patients taking Epidiolex and one patient
taking placebo, all of whom were on concomitant valproic acid. Four
of these patients withdrew from the trial, three on Epidiolex and
one on placebo. In the remaining nine patients taking Epidiolex,
elevations returned to normal while on treatment.
"The publication of results from this landmark study by The
New England Journal of Medicine and the accompanying editorial
commentary[2] highlight the potential of Epidiolex to
address the significant unmet need in Dravet syndrome," said
Justin Gover, GW's Chief Executive
Officer. "We remain committed to these patients and their families,
and are determined to make this important new medicine available to
them as quickly as possible."
About Dravet Syndrome
Dravet syndrome is a severe infantile-onset and highly
treatment-resistant epileptic syndrome frequently associated with a
genetic mutation in sodium channels. Onset of Dravet syndrome
occurs during the first year of life in previously healthy and
developmentally normal infants. Initial seizures are often
temperature related, severe, and long-lasting. Over time, people
with Dravet syndrome can develop multiple types of seizures,
including tonic-clonic, myoclonic, and atypical absences and are
prone to bouts of prolonged seizures called status epilepticus,
which can be life threatening. Risk of premature death including
SUDEP (sudden unexpected death in epilepsy) is elevated in people
with Dravet syndrome. Additionally, the majority will develop
moderate to severe intellectual and development disabilities and
require lifelong supervision and care. There are currently
no FDA-approved treatments and nearly all patients continue to
have uncontrolled seizures and other medical needs throughout their
lifetime.
About Epidiolex
Epidiolex, GW's lead cannabinoid product candidate, is a liquid
formulation of plant-derived cannabidiol (CBD), which is in
development for the treatment of a number of rare childhood-onset
epilepsy disorders. GW has conducted extensive pre-clinical
research of CBD in epilepsy since 2007. This research has shown
that CBD has significant anti-epileptiform and anticonvulsant
activity using a variety of in-vitro and in-vivo
models and efficacy in reducing seizures in acute animal models of
epilepsy. To date, GW has received Orphan Drug Designation from the
U.S. Food and Drug Administration (FDA) for Epidiolex for the
treatment of Dravet syndrome, Lennox-Gastaut syndrome (LGS),
Tuberous Sclerosis Complex (TSC) and Infantile Spasms (IS), each of
which are severe infantile-onset, drug-resistant epilepsy
syndromes. Additionally, GW has received Fast Track Designation
from the FDA for the treatment of Dravet syndrome and Orphan
Designation from the European Medicines Agency, or EMA, for
Epidiolex for the treatment of Dravet syndrome and LGS. GW is
currently evaluating additional clinical development programs in
other orphan seizure disorders.
About GW Pharmaceuticals plc
Founded in 1998, GW is a biopharmaceutical company focused on
discovering, developing and commercializing novel therapeutics from
its proprietary cannabinoid product platform in a broad range of
disease areas. GW is advancing an orphan drug program in the field
of childhood-onset epilepsy with a focus on Epidiolex®
(cannabidiol), which is in Phase 3 clinical development for the
treatment of Dravet syndrome, Lennox-Gastaut syndrome, Tuberous
Sclerosis Complex and Infantile Spasms. GW commercialized the
world's first plant-derived cannabinoid prescription drug,
Sativex® (nabiximols), which is approved for the
treatment of spasticity due to multiple sclerosis in 31 countries
outside the United States. The
Company has a deep pipeline of additional cannabinoid product
candidates which includes compounds in Phase 1 and 2 trials for
glioma, schizophrenia and epilepsy. In the United States, GW operates through its
subsidiary Greenwich Biosciences, Inc. For further information,
please visit http://www.gwpharm.com.
Forward-looking statements
This news release contains forward-looking statements that
reflect GW's current expectations regarding future events,
including statements regarding financial performance, the timing of
clinical trials, the timing and outcomes of regulatory or
intellectual property decisions, the relevance of GW products
commercially available and in development, the clinical benefits of
Epidiolex® and the safety profile and commercial potential of
Epidiolex. Forward-looking statements involve risks and
uncertainties. Actual events could differ materially from those
projected herein and depend on a number of factors, including
(inter alia), the success of GW's research
strategies, the applicability of the discoveries made therein, the
successful and timely completion of uncertainties related to the
regulatory process, and the acceptance of Epidiolex and other
products by consumer and medical professionals. A further list and
description of risks and uncertainties associated with an
investment in GW can be found in GW's filings with
the U.S. Securities and Exchange Commission, including the most
recent Form 20-F filed on 5 December
2016. Existing and prospective investors are cautioned
not to place undue reliance on these forward-looking statements,
which speak only as of the date hereof. GW undertakes no obligation
to update or revise the information contained in this press
release, whether as a result of new information, future events or
circumstances or otherwise.
1. Devinsky O, Cross JH, Laux L, et al. Trial of cannabidiol
for drug-resistant seizures in the Dravet syndrome. N Engl J Med
2017; 376;2011-20.
2. Berkovic, SF. Editorial:
Cannabinoids for epilepsy - real data, at last. N Engl J Med 2017;
376;2075-76.
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