– First well-controlled clinical study of
cannabidiol in Lennox-Gastaut syndrome, a rare, severe, form of
childhood-onset epilepsy that is difficult to treat –
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 Lancet
has published results from a Phase 3 study of Epidiolex®
(cannabidiol) in patients with Lennox-Gastaut syndrome (LGS).1
Epidiolex, GW’s lead product candidate and the potential first in a
new category of anti-epileptic drugs (AEDs), is a pharmaceutical
formulation of purified cannabidiol (CBD), a cannabinoid lacking
euphoric side effects, which is being studied for the treatment of
a number of rare, severe pediatric-onset epilepsy disorders. In
this study, Epidiolex significantly reduced monthly drop seizure
frequency compared to placebo in highly treatment-resistant
patients when added to existing treatment. Treatment with Epidiolex
was generally well tolerated, with a safety profile consistent with
prior reported experience.
A New Drug Application (NDA) submission to the
U.S. Food and Drug Administration (FDA) for Epidiolex in the
treatment of LGS and Dravet syndrome (another rare childhood-onset
epilepsy) was accepted in December with an assigned PDUFA goal date
of June 27th 2018 and, if approved, the medicine is expected to be
available in the U.S. by prescription in the second half of 2018. A
Marketing Authorisation Application (MAA) was submitted to the
European Medicines Agency (EMA) in December 2017, with an expected
decision in early 2019.
“Publication of this landmark study by The
Lancet is an exciting achievement and marks the second time that
Epidiolex data have been published in a highly prestigious journal,
following last year’s publication in The New England Journal of
Medicine,” said Justin Gover, GW's Chief Executive Officer. “These
publications highlight the potential of Epidiolex to address the
significant unmet need in LGS and Dravet syndrome, two very
challenging epilepsy conditions, and we look forward to working
with the FDA and EMA as they review our marketing applications for
Epidiolex. We are absolutely focused on the goal of making this
important new medicine available to appropriate patients and their
caregivers as quickly as possible.”
LGS is a rare, lifelong form of epilepsy that
begins in childhood and is associated with a high mortality rate2
and significant developmental delays.3,4 LGS patients suffer
from multiple types of seizures, including drop seizures which can
result in falls and other injuries. Results from this study
represent the only well-controlled clinical evaluation of a
cannabinoid medication for this severe, drug-resistant
condition.
"The publication of these positive results is an
exciting milestone for the LGS community and we are encouraged that
a new treatment option could soon be available," said Christina
SanInocencio, executive director of the Lennox-Gastaut Syndrome
Foundation. "Additional treatment options are desperately needed
for patients who continue to struggle with uncontrolled seizures
and these results offer much needed hope to those living with this
debilitating condition."
"LGS is one of the most difficult types of
epilepsy to treat and the majority of patients do not have an
adequate response to existing therapies," said Elizabeth Thiele,
MD, PhD, director of pediatric epilepsy at Massachusetts General
Hospital, professor of Neurology at Harvard Medical School and lead
author of the study publication. “These results show that
Epidiolex may provide clinically meaningful benefits for patients
with LGS.”
The study randomized 171 patients (86 to CBD; 85
to placebo) ages two to 55 years (average age 15), with LGS whose
seizures were not controlled by their current AED regimen, to
receive either Epidiolex (20mg/kg/day) or placebo in addition to
existing treatment. Conducted in 24 study centers in the United
States and Europe, on average, patients were taking approximately
three AEDs, having previously tried and discontinued an average of
six other AEDs. At baseline, patients had a median frequency of 74
drop seizures per month (drop seizures were defined as atonic,
tonic or tonic-clonic seizures involving the entire body, trunk or
head that led or could have led to a fall, injury, slumping in a
chair or hitting the patient’s head on a surface).
Over the 14-week treatment period (two-week dose
escalation period followed by 12 weeks of maintenance), patients
taking Epidiolex had a significantly greater median reduction in
drop seizures compared to placebo (44 percent vs. 22 percent;
p=0.0135), the study’s primary endpoint. Sensitivity analyses
confirmed that the treatment effect of CBD was established during
the first month of treatment and was sustained over the entire
treatment period.
Results from key secondary endpoints showed that
significantly more patients on Epidiolex experienced a 50 percent
or greater reduction in drop seizures compared to placebo (44
percent vs. 24 percent; p=0.0043) and total seizure frequency was
significantly reduced with Epidiolex compared to placebo (median
percent reduction of 41 percent vs. 14 percent; p=0.0005). CBD
patients/caregivers were significantly more likely to report an
improvement in overall condition with Epidiolex than placebo (58
percent vs. 34 percent; OR 2.54, 95% CI 1.5-4; p=0.0012) based on
the Subject/Caregiver Global Impression of Change (S/CGIC)
scale.
Epidiolex was generally well tolerated in the trial. The
most common adverse events (AEs) (>10 percent) were diarrhea,
somnolence, pyrexia, decreased appetite and vomiting. Overall, 86
percent of patients taking Epidiolex and 69 percent of patients
taking placebo experienced an AE, and most were mild or moderate.
Of those patients who experienced AEs, the events resolved by the
end of the trial for 61 percent of Epidiolex patients and 64
percent of placebo patients.
Twenty patients on Epidiolex experienced serious
AEs, including one fatal case of acute respiratory distress
syndrome (considered unrelated by the Investigator), compared with
four patients with serious AEs on placebo. Twelve patients taking
Epidiolex discontinued treatment due to AEs compared with one
patient taking placebo.
Across the Epidiolex development program, the
most common reported adverse reactions are somnolence, decreased
appetite, diarrhea, pyrexia, fatigue, lethargy, rash,
nasopharyngitis, and pneumonia; dose-related reversible elevation
of liver transaminases without elevation of bilirubin were also
observed.
“Uncontrolled seizures significantly impact the
lives of patients and their families and there is a tremendous need
for new options in difficult-to-treat epilepsies such as LGS,” said
Philip Gattone, president and CEO, Epilepsy Foundation. “This
randomized, controlled clinical study provides positive evidence of
the potential role of cannabidiol in reducing seizures and we
are excited about the possibility of a new treatment option for
LGS.”
About Lennox-Gastaut
Syndrome
The onset of LGS typically occurs between ages
of 3 to 5 years and can be caused by a number of conditions,
including brain malformations, severe head injuries, central
nervous system infections, and genetic neuro-degenerative or
metabolic conditions. In up to 30 percent of patients, no cause can
be found. Patients with LGS commonly have multiple seizure types
including drop and convulsive seizures, which frequently lead to
falls and injuries, and non-convulsive seizures. Resistance to
anti-epileptic drugs (AEDs) is common in patients with LGS. Most
children with LGS experience some degree of intellectual
impairment, as well as developmental delays and aberrant
behaviors.
About Dravet Syndrome
Dravet syndrome is a severe infantile-onset and
highly treatment-resistant epileptic encephalopathy frequently
associated with genetic mutations in the SCN1A sodium channels.
Onset of Dravet syndrome occurs typically during the first year of
life in previously healthy and developmentally normal infants.
Initial seizures are often body temperature related, severe, and
long-lasting. Over time, patients with Dravet syndrome often
develop multiple types of seizures, including tonic-clonic,
myoclonic, and atypical absences and are prone to bouts of
prolonged seizures including status epilepticus, which can be life
threatening. Risk of premature death including SUDEP (sudden
unexpected death in epilepsy) is elevated in patients 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
experience seizures and other medical needs throughout their
lifetime.
About Epidiolex®
(cannabidiol)
Epidiolex, GW's lead cannabinoid product
candidate is a pharmaceutical formulation of purified cannabidiol
(CBD), which is in development for the treatment of several rare
childhood-onset epilepsy disorders. GW has submitted a New Drug
Application with the FDA for Epidiolex as adjunctive treatment for
seizures associated with LGS and Dravet syndrome, which has been
assigned a goal date of 27 June 2018 and, if approved, the medicine
is expected to be available by prescription in the second half of
2018. GW has also submitted a Marketing Authorisation Application
(MAA) to the European Medicines Agency (EMA) in December 2017 with
an expected decision date in early 2019. To date, GW has received
Orphan Drug Designation from the FDA for Epidiolex for the
treatment of Dravet syndrome, LGS, TSC and IS. Additionally, GW has
received Fast Track Designation from the FDA for the treatment of
Dravet syndrome and conditional grant of rare pediatric disease
designation by FDA. The Company has also received Orphan
Designation from the European Medicines Agency, or EMA, for
Epidiolex for the treatment of LGS, Dravet syndrome, West syndrome
and TSC. GW is currently evaluating additional clinical development
programs in other orphan seizure disorders including Phase 3 trials
in Tuberous Sclerosis Complex and Infantile Spasms.
About GW Pharmaceuticals plc and
Greenwich Biosciences
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, along with its U.S.
subsidiary Greenwich Biosciences, is advancing an orphan drug
program in the field of childhood epilepsy with a focus on
Epidiolex (cannabidiol), for which GW has submitted an NDA to the
FDA for the adjunctive treatment of LGS and Dravet syndrome. The
Company continues to evaluate Epidiolex in additional epilepsy
conditions and currently has ongoing clinical trials in 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 numerous 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 gliobastoma, schizophrenia and epilepsy. For further
information, please visit 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® (cannabidiol) 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 and uncertainties related to the regulatory
process, and the acceptance of Sativex, 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 4 December 2017. 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.
Enquiries:
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____________________________1 Thiele EA, Marsh ED, French JA, et
al. Cannabidiol in patients with seizures associated with
Lennox-Gastaut syndrome (GWPCARE4): a randomized, double-blind
placebo-controlled phase 3 trial. Published online January 24, 2018
http://dx.doi.org/10.1016/S0140-6736(18)30136-3.2 Autry AR,
Trevathan E, Van Naarden Braun K, Yeargin-Allsopp M. Increased risk
of death among children with Lennox-Gastaut syndrome and infantile
spasms. J Child Neurol. 2010;25(4):441-447.3 LGS Foundation. About
Lennox-Gastaut Syndrome. Available at
http://www.lgsfoundation.org/aboutlgs. Accessed October 23, 2017.4
National Institute of Health. Lennox-Gastaut syndrome. Available at
https://ghr.nlm.nih.gov/condition/lennox-gastaut-syndrome#definition.
Accessed October 23, 2017.
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