GW Pharmaceuticals plc (NASDAQ: GWPH, GW, the Company or the
Group), the world leader in the science, development, and
commercialization of cannabinoid prescription medicines, along with
U.S. subsidiary Greenwich Biosciences, announced today that JAMA
Neurology has published results of the company’s positive Phase 3
clinical trial of EPIDIOLEX® (cannabidiol) oral solution in
seizures associated with tuberous sclerosis complex (TSC).
EPIDIOLEX, a pharmaceutical formulation of cannabidiol (CBD), is
the first prescription, plant-derived cannabis-based medicine
approved by the U.S. Food and Drug Administration (FDA) for the
treatment of seizures associated with Lennox-Gastaut syndrome
(LGS), Dravet syndrome, or TSC in patients one year of age and
older.
The study found that individuals treated with
EPIDIOLEX 25 mg/kg/day or 50 mg/kg/day experienced a significantly
greater reduction in TSC-associated seizures (48.6% for 25
mg/kg/day and 47.5% for 50 mg/kg/day) compared to placebo (26.5%;
p<0.001 and p=0.002, respectively). This trial provided the
basis for the July 2020 FDA approval of EPIDIOLEX for seizures
associated with TSC.
“People living with TSC may experience focal
seizures and spasms as infants and continue to suffer from seizures
throughout their lifetime,” 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. “This study demonstrated
that for patients with TSC and a high baseline burden of
treatment-resistant, primarily focal, seizures, EPIDIOLEX
significantly reduced the frequency of seizures compared with
placebo.”
The safety profile observed in the study was
generally consistent with findings from previous studies of
EPIDIOLEX. Both doses had an acceptable safety profile, with fewer
adverse events (AEs) reported with 25 mg/kg/day than 50 mg/kg/day.
The most common AEs were diarrhea, decreased appetite, and
somnolence. 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. Eight
patients on EPIDIOLEX 25 mg/kg/day, 10 on 50 mg/kg/day, and two on
placebo discontinued treatment due to an AE. Additionally, 12% of
25 mg/kg/day patients and 26% 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 meeting Hy’s law criteria
observed and there were no deaths in the trial.
Secondary endpoint data showed 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=0.07 and p=0.02, respectively). Additionally, patients
taking either dose of EPIDIOLEX in the study experienced a greater
reduction in total seizure frequency (48%) compared to placebo
(27%; p<0.001 and p=0.002, respectively). Caregivers and
patients also reported overall improvement on the subject/caregiver
global impression of change (S/CGIC) more frequently with
EPIDIOLEX: 69% of patients on 25 mg/kg/day and 62% of patients on
50 mg/kg/day compared to 39% on placebo (p=0.007 and p=0.06,
respectively).
“The publication of these results in JAMA
Neurology reinforces the importance of EPIDIOLEX as a new treatment
option for people experiencing treatment-resistant seizures
associated with TSC,” said Justin Gover, Chief Executive Officer,
GW Pharmaceuticals. “We hope that the data will help clinicians to
better understand the potential of EPIDIOLEX to reduce focal and
generalized seizure frequency in their patients with this
condition.”
The results of this study were previously
presented at the 2019 American Epilepsy Society (AES) Annual
Meeting.
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 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.1 At least two children
born each day will develop TSC, with an estimated prevalence of one
in 6,000 newborns.1 The condition causes mostly benign tumors to
grow in vital organs of the body including the brain, skin, heart,
eyes, kidneys and lungs2 and is a leading cause of genetic
epilepsy.3 People with TSC may experience a variety of seizure
types. One of the most common is infantile spasms that
typically present in the first year of life; focal (or
partial) seizures are also very common.4 TSC is associated
with an increased risk of autism and intellectual disability5 and
the severity of the condition can vary widely. In some children the
disease is very mild, while others may experience
life-threatening complications.2 Epilepsy is present in about
85 percent of patients with TSC and may progress to become
intractable to medication.4,6,7 More than 60 percent of individuals
with TSC do not achieve seizure control8 with standard treatments
such as antiepileptic drugs, epilepsy surgery, ketogenic diet, or
vagus nerve stimulation6 compared to 30-40 percent 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. The Company’s
lead product, EPIDIOLEX® (cannabidiol) oral solution, is
commercialized in the U.S. by its U.S. subsidiary Greenwich
Biosciences for the treatment of seizures associated with
Lennox-Gastaut syndrome (LGS), Dravet syndrome, or tuberous
sclerosis complex (TSC) in patients one year of age and older. This
product has received approval in the European Union under the
tradename EPIDYOLEX® for the adjunctive treatment of seizures
associated with LGS or Dravet syndrome in conjunction with clobazam
in patients two years and older and is under EMA review for the
treatment of TSC. The Company has a deep pipeline of additional
cannabinoid product candidates, in particular nabiximols, for which
the Company is advancing multiple late-stage clinical programs in
order to seek FDA approval in the treatment of spasticity
associated with multiple sclerosis and spinal cord injury. The
Company has additional cannabinoid product candidates in clinical
trials for autism 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.
U.S. Media Enquiries:Sam Brown Inc.
Healthcare Communications |
Christy CurranMike Beyer |
615 414 8668312 961 2502 |
_____________________
1 TS Alliance, What is TSC?
https://www.tsalliance.org/about-tsc/what-is-tsc/. Accessed April
15, 2019.2 NIH Tuberous Sclerosis Fact Sheet. Accessed
November 19, 2019.3 TS Alliance Website.
https://www.tsalliance.org/. Accessed November 19, 2019. 4
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):2.5 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):157.6 Tuberous Sclerosis Alliance.
Diagnosis, Surveillance, and Management for Healthcare
Professionals
https://www.tsalliance.org/healthcareprofessionals/diagnosis/.
Accessed February 19, 2019.7 Jeong A, Wong M. Systemic disease
manifestations associated with epilepsy in tuberous sclerosis
complex. Epilepsia. 2016;57(9):1443-1449.8 Chu-Shore CJ, Major P,
Camposano S, Muzykewicz D, Thiele EA. The natural history of
epilepsy in tuberous sclerosis complex. Epilepsia.
2010;51(7):1236-1241.9 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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