GW Pharmaceuticals plc (Nasdaq: GWPH), the world leader in the
science, development, and commercialization of cannabinoid
prescription medicines, along with U.S. subsidiary Greenwich
Biosciences, today presented new data from a Phase 3 clinical trial
of EPIDIOLEX® (cannabidiol or CBD) oral solution, CV in tuberous
sclerosis complex (TSC) at the American Epilepsy Society (AES)
Annual Meeting. The study found that patients treated with
EPIDIOLEX 25 mg/kg/day or 50 mg/kg/day experienced a significantly
greater reduction in TSC-associated seizures (49% for 25 mg/kg/day
and 48% for 50 mg/kg/day) compared to placebo (27%; p=0.0009 and
p=0.0018, respectively).
“Epilepsy is the most common neurological
feature of TSC,1,2,3 a rare and severe childhood-onset disease, and
as many as two-thirds of patients experience treatment-resistant
seizures,”4 said Elizabeth Thiele, M.D, Ph.D., director of
pediatric epilepsy and director of The Carol and James Herscot
Center for Tuberous Sclerosis Complex at Massachusetts General
Hospital, professor of neurology at Harvard Medical School, Boston
and lead investigator of the trial. “There is a significant need
for new treatments to address TSC-related seizures and these
positive results suggest patients may benefit from this
pharmaceutical formulation of highly-purified CBD.”
New secondary endpoint data, presented for the
first time at AES, showed that more patients on EPIDIOLEX
experienced a 50% or greater reduction in seizures (36% for 25
mg/kg/day and 40% for 50 mg/kg/day) compared to placebo (22%;
p=0.0692 and p=0.0245, respectively). Additionally, 48% of patients
taking either dose of EPIDIOLEX in the study experienced a greater
reduction in total seizure frequency compared to placebo (27%;
p=0.0013 and p=0.0018, respectively). Caregivers and patients also
reported overall improvement with EPIDIOLEX in 69% of patients on
25 mg/kg/day and 62% of patients on 50 mg/kg/day compared to 39% on
placebo (p=0.0074 and p=0.0580, respectively). Additional analysis
showed that patients taking EPIDIOLEX in the study experienced a
greater reduction in composite focal seizures (52% for 25 mg/kg/day
and 50% for 50 mg/kg/day) compared to placebo (32%; p=0.0076 and
p=0.0116, respectively).
“We are pleased to present these results which
demonstrate the potential of EPIDIOLEX to reduce both focal and
generalized seizures associated with TSC,” said Justin Gover, CEO,
GW Pharmaceuticals. “This is promising news for patients and
clinicians, and we remain committed to helping those suffering from
seizures associated with this difficult-to-treat disease. We look
forward to submitting an sNDA to the FDA soon, with the goal of
expanding the EPIDIOLEX product label in 2020.”
The safety profile observed in the study was
generally consistent with findings from previous studies. Adverse
events (AEs) occurred in 93% of the 25 mg/kg/day group, 100% of the
50 mg/kg/day group and 95% of the placebo group. Both doses had an
acceptable safety profile, with fewer AEs reported with 25
mg/kg/day than 50 mg/kg/day. The most common AEs were diarrhea,
decreased appetite and somnolence. Eight patients on EPIDIOLEX 25
mg/kg/day, 10 on 50 mg/kg/day and 2 on placebo discontinued
treatment due to an AE. Additionally, 13% of 25 mg/kg/day patients
and 25% of 50 mg/kg/day patients experienced elevated liver
enzymes; 79% of these patients were also taking the antiepileptic
drug (AED) valproate. Elevations in ALT/AST resolved in all
patients. There were no cases of Hy’s law observed and there were
no deaths in the trial.
Study DesignThe randomized,
double-blind, placebo-controlled trial was conducted at 46 sites in
six countries. A total of 224 people (aged 1 to 65) with a
confirmed diagnosis of treatment-resistant TSC were randomized to
receive either EPIDIOLEX 25 mg/kg/day (n=75), EPIDIOLEX 50
mg/kg/day (n=73), or placebo (n=76) for 16 weeks (4-week titration
and 12-week maintenance phase), added to current AED treatment. The
primary endpoint was percent change from baseline in TSC-associated
focal and generalized seizure frequency for EPIDIOLEX vs placebo
over the treatment period. Key secondary endpoints were ≥50%
responder rate, percent reduction in total seizure frequency
(including focal sensory and epileptic spasms), and
subject/caregiver global impression of change (S/CGIC) in overall
condition. On average, patients were taking three AEDs, having
previously tried and discontinued four other AEDs. The most common
concomitant AEDs in this trial were valproate (45%), vigabatrin
(33%), levetiracetam (29%), and clobazam (27%).
About Tuberous Sclerosis Complex
(TSC)Tuberous sclerosis complex (TSC) is a rare genetic
condition that affects approximately 50,000 individuals in the U.S.
and nearly one million people worldwide.5 At least two children
born each day will develop TSC, with an estimated prevalence of one
in 6,000 newborns.5 The condition causes mostly benign tumors to
grow in vital organs of the body including the brain, skin, heart,
eyes, kidneys and lungs 6 and is a leading cause of genetic
epilepsy.7 TSC often occurs in the first year of life as either
focal seizures or infantile spasms1 and is associated with an
increased risk of autism and intellectual disability.8 The severity
of the condition can vary widely. In some children the disease is
very mild, while others may experience
life-threatening complications.6
Epilepsy is present in about 85% of patients
with TSC and may progress to become intractable to medication.1,2,3
More than 60% of individuals with TSC do not achieve seizure
control4 with standard treatments such as antiepileptic drugs,
epilepsy surgery, ketogenic diet, or vagus nerve stimulation2
compared to 30-40% of individuals with epilepsy who do not have TSC
who are drug resistant.9,10
About GW Pharmaceuticals plc and Greenwich Biosciences,
Inc.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’s lead product, EPIDIOLEX®
(cannabidiol) oral solution is commercialized in the US by its U.S.
subsidiary Greenwich Biosciences for the treatment of seizures
associated with Lennox-Gastaut syndrome (LGS) or Dravet syndrome in
patients two years of age or older. This product has received
approval in Europe under the tradename EPIDYOLEX®. The Company
continues to evaluate EPIDIOLEX in additional rare conditions
including tuberous sclerosis complex (TSC) and Rett syndrome. 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 and for which the Company is
now advancing a late stage program in order to seek FDA approval.
The Company has a deep pipeline of additional cannabinoid product
candidates which includes compounds in Phase 1 and 2 trials for
epilepsy, autism, glioblastoma, and schizophrenia. For further
information, please visit www.gwpharm.com.
Important Safety InformationImportant safety
information for EPIDIOLEX is available at Epidiolex.com.
Forward-looking statementsThis
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®/EPIDYOLEX® (cannabidiol) oral
solution and Sativex® (nabiximols), and the safety
profile and commercial potential of both medicines. 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
EPIDIOLEX®/EPIDYOLEX®, Sativex® 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.
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:
GW Pharmaceuticals plc |
Stephen Schultz, VP Investor Relations (U.S.) |
917 280 2424 / 401 500 6570 |
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U.S. Media Enquiries: |
Sam Brown Inc. Healthcare Communications |
Christy Curran |
615 414 8668 |
Mike Beyer |
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______________________1 Kingswood JC, d'Augeres GB, Belousova E,
et al. TuberOus SClerosis registry to increase disease Awareness
(TOSCA) - baseline data on 2093 patients. Orphanet J Rare Dis.
2017;12(1):2.2 Tuberous Sclerosis Alliance. Diagnosis,
Surveillance, and Management for Healthcare Professionals
https://www.tsalliance.org/healthcare-professionals/diagnosis/.
Accessed February 19, 2019.3 Jeong A, Wong M. Systemic disease
manifestations associated with epilepsy in tuberous sclerosis
complex. Epilepsia. 2016;57(9):1443-1449.4 Chu-Shore CJ, Major P,
Camposano S, Muzykewicz D, Thiele EA. The natural history of
epilepsy in tuberous sclerosis complex. Epilepsia.
2010;51(7):1236-1241.5 TS Alliance, What is TSC?
https://www.tsalliance.org/about-tsc/what-is-tsc/. Accessed April
15, 2019.6 NIH Tuberous Sclerosis Fact Sheet.
https://www.ninds.nih.gov/Disorders/Patient-Caregiver-Education/Fact-Sheets/Tuberous-Sclerosis-Fact-Sheet.
Accessed November 19, 2019.7 TS Alliance Website.
https://www.tsalliance.org/. Accessed November 19, 2019.8 de Vries
PJ, Belousova E, Benedik MP, et al. TSC-associated neuropsychiatric
disorders (TAND): findings from the TOSCA natural history study.
Orphanet J Rare Dis. 2018;13(1):157.9 Kwan P., Brodie M.J. Early
identification of refractory epilepsy. N. Engl. J.
Med. 2000;342(5):314–319.10 French JA. Refractory epilepsy:
clinical overview. Epilepsia. 2007;48 Suppl 1:3-7.
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