Conference Call Today at 6:30 p.m.
ET
- New data issued in a poster presentation at the American
Academy of Neurology annual meeting on 137 patients, representing
increased patient numbers over previous disclosures
- Promising signals of efficacy with median 54% reduction in
seizures after 12 weeks treatment and maintenance of clinical
effect at 24 weeks
- Epidiolex was well tolerated and 90% of patients remained on
treatment
- Data on Lennox-Gastaut syndrome (LGS) patients presented for
first time
- Additional body of data in patients with diagnoses other than
Dravet syndrome
- GW advancing plans to commence clinical development of
Epidiolex in additional pediatric epilepsy target indication of
Tuberous Sclerosis Complex
GW Pharmaceuticals plc (Nasdaq:GWPH) (AIM:GWP) ("GW," "the Company"
or "the Group"), a biopharmaceutical company focused on
discovering, developing and commercializing novel therapeutics from
its proprietary cannabinoid product platform, today announced new
physician reports of clinical effect and safety associated with the
Epidiolex® expanded access program. These data were presented today
by study authors at the American Academy of Neurology annual
meeting in Washington DC1.
"We are pleased to report that this promising data on
significant numbers of additional children treated with Epidiolex
further reinforces and supports the signals of efficacy seen in
previous data disclosures. Epidiolex treatment was associated with
meaningful reductions in seizures across a range of childhood
epilepsies as well as a sustained response over a 6 month period
and a 90% retention rate," stated Justin Gover, GW's Chief
Executive Officer. "We believe that these findings fully support
our decision to advance Epidiolex into Phase 3 placebo-controlled,
double-blind trials in Dravet syndrome and Lennox-Gastaut syndrome
and we look forward to advancing these trials rapidly during
2015."
Physician reports of clinical effect and safety data have been
presented on 137 children and young adults with treatment-resistant
epilepsy who have been treated with GW's investigational
cannabidiol (CBD) product candidate, Epidiolex, for a period of at
least 12 weeks. These data are from eleven hospital sites in the
United States and were generated under expanded access
Investigational New Drug applications (INDs) authorized by the U.S.
Food and Drug Administration (FDA). In addition, physician reports
of safety data were presented on 213 patients (137 patients with 12
weeks treatment effect data plus additional patients still in their
first 12 weeks of treatment or who withdrew from treatment). The
expanded access program is comprised of clinical studies performed
by individual physicians.
FDA authorized expanded access programs facilitate access
to investigational drugs for treatment use for patients with a
serious or immediately life-threatening diseases or conditions who
lack therapeutic alternatives. The patients included in the
Epidiolex program had Dravet syndrome (18% of the total) and
Lennox-Gastaut syndrome (LGS) (16% of the total), epilepsy types
that can lead to intellectual disability and lifelong seizures, as
well as 10 other types of severe epilepsy. Many of the 10 other
types of epilepsy are extreme and rare, and several patients with
these epilepsies have major congenital structural brain
abnormalities.
The 137 patients were predominately children with an average age
of 11 years. In all cases, Epidiolex was added to current
anti-epileptic drug (AED) treatment regimes. On average, patients
were taking approximately 3 other AEDs.
At the request of the study authors, the data disclosed in this
press release is limited to the data presented at the AAN
meeting.
Clinical Effect Data – All Patients
Data were presented on all 137 patients who had at least 12
weeks treatment. Treatment was open label. Of these 137 patients,
48 patients had also reached 24 weeks treatment at the time of data
analysis. Information collected on all seizures (convulsive and
non-convulsive) is reported for each patient. Data are presented
showing the median reduction in seizure frequency and the
proportion of patients achieving a response of 50% or greater
compared to a 4-week baseline period.
|
|
Week 4 |
Week 8 |
Week 12 |
Week 16 |
Week 24 |
Median reduction in seizure
frequency |
-40% |
-48% |
-54% |
-51% |
-45% |
≥50% responders |
41% |
49% |
51% |
52% |
46% |
At the end of 12 weeks, 9% of all patients were
seizure-free.
Clinical Effect Data – Patients without Dravet
syndrome
Data were made available on all 112 patients with diagnoses
other than Dravet syndrome who had at least 12 weeks treatment. Of
these 112 patients, 39 patients had also reached 24 weeks treatment
at the time of data analysis. Information collected on all seizures
(convulsive and non-convulsive) is reported for each patient. Data
are presented showing the median reduction in seizure frequency and
the proportion of patients achieving a response of 50% or greater
compared to a 4-week baseline period.
|
|
Week 4 |
Week 8 |
Week 12 |
Week 16 |
Week 24 |
Median reduction in seizure
frequency |
-37% |
-45% |
-50% |
-50% |
-38% |
≥50% responders |
39% |
48% |
50% |
49% |
41% |
Clinical Effect Data – Dravet syndrome patients
only
Data were presented on 25 patients with Dravet syndrome who had
at least 12 weeks treatment data. Of these 25 patients, 9 patients
had also reached 24 weeks treatment at the time of data analysis.
Information collected on all seizures (convulsive and
non-convulsive) is reported for each patient. Data are presented
showing the median reduction in seizure frequency and the
proportion of patients achieving a response of 50% or greater
compared to a 4-week baseline period.
|
|
Week 4 |
Week 8 |
Week 12 |
Week 16 |
Week 24 |
Median reduction in seizure
frequency |
-50% |
-60% |
-65% |
-56% |
-59% |
≥50% responders |
52% |
54% |
56% |
61% |
67% |
At the end of 12 weeks, 16% of Dravet syndrome patients were
seizure-free.
Of the 25 patients with Dravet syndrome, 23 patients had
convulsive seizures at baseline. At the end of 12 weeks, the median
reduction in convulsive seizures in these patients was 53%2.
Clinical Effect Data – Drop ("atonic")
Seizures
Data were made available on 27 patients with drop seizures at
baseline who had at least 12 weeks treatment, of which 11 patients
had LGS2. Drop seizures are the types of seizures generally
considered by FDA in assessing primary efficacy for LGS trials.
- For all 27 patients with drop seizures, there was a 67% median
reduction in the number of atonic seizures at the end of 12
weeks
- For the 11 LGS patients with drop seizures, there was a 55%
median reduction in the number of atonic seizures at the end of 12
weeks2
Safety Data
Safety data were made available on 213 patients (137 patients
with 12 weeks treatment effect data plus 76 additional patients for
whom 12 week treatment effect data are not yet available or who
withdrew from treatment) and represents approximately 59
patient-years of exposure to Epidiolex.
- The most common adverse events (occurring in 10% or more
patients and resulting from all causes) were:
- Somnolence - 21% of patients
- Diarrhea - 17% of patients
- Fatigue - 17% of patients
- Decreased appetite - 16% of patients
- Most adverse events were mild or moderate and transient
- 10 patients (5%) reported an adverse event leading to
discontinuation, 3 of whom subsequently restarted treatment with
Epidiolex
- There were 14 withdrawals from treatment due to lack of
clinical effect
- Serious adverse events were reported in 52 patients. Of these,
SAEs in 22 patients were deemed possibly related to treatment,
including status epilepticus (10), diarrhea (3), weight loss
(2), pneumonia (2), lethargy (1), and hepatotoxicity (1)
- Serious adverse events include 2 deaths, neither of which were
deemed related to Epidiolex by the independent investigators. One
death was from SUDEP (sudden unexpected death in epilepsy) and one
from respiratory failure due to aspiration.
- There were no clinically significant changes in hematologic
markers or renal function
- Occasional increases in liver transaminases were noted, thought
unlikely to be related to CBD by the independent investigators
GW Epidiolex Development Program – Dravet and
LGS
GW's initial focus is on conducting formal development programs
for Epidiolex in the treatment of both Dravet syndrome and LGS. The
Company has received from the FDA Orphan Drug Designations for
Epidiolex for both Dravet syndrome and LGS, as well as Fast Track
Designation for Dravet syndrome. GW has recently commenced two
Phase 3 trials of Epidiolex in the treatment of Dravet syndrome. GW
also expects to commence two Phase 3 trials in LGS in the second
quarter of 2015.
New Target Indication – Tuberous Sclerosis Complex
(TSC)
Based on the findings in the expanded access program, GW has
continued to explore options to commence clinical development of
Epidiolex in an additional pediatric epilepsy indication. GW has
now concluded that this additional target indication should be
Tuberous Sclerosis Complex (TSC) and expects to commence clinical
development in TSC later this year.
TSC is a genetic disorder that causes non-malignant tumors to
form in many different organs, primarily in the brain, eyes, heart,
kidney, skin and lungs. TSC results from a mutation in tumor
suppression genes TSC1 or TSC2. TSC is estimated to affect
approximately 50,000 patients in the U.S. The most common symptom
of TSC is epilepsy, which occurs in 75-90% of patients, about 70%
of whom experience seizure onset in their first year of life.
Approximately 60% of TSC patients have treatment-resistant
seizures. There are significant co-morbidities associated with TSC
including cognitive impairment in 50%, autism spectrum disorders in
up to 40% and neurobehavioral disorders in over 60% of individuals
with TSC.
A number of patients with TSC have been treated with Epidiolex
in the expanded access program. According to Geffrey AL et al in a
poster presentation at the American Epilepsy Society annual meeting
in December 2014, of 5 TSC patients treated with Epidiolex, 4
patients experienced a reduction in seizures of 97% (n=2), 77%
(n=1) and 40% (n=1) at week 16 compared to a 4-week baseline
period. All three patients with cognitive impairment experienced
cognitive gains and one of the two subjects with behavioral
problems showed improvements. Since this poster presentation,
additional TSC patients have commenced treatment with Epidiolex in
the expanded access program.
Conference Call and Presentation
Information
GW Pharmaceuticals will host a conference call and broadcast a
slide show to discuss these data today at 6:30 p.m. EDT. To
participate in the conference call, please dial 877-407-8133 (toll
free from the U.S. and Canada) or 201-689-8040 (international). To
view the slide show while using the audio dial-in, please go to the
investor relations (presentation and events) section of GW's
website at http://www.gwpharm.com. Investors may also access a live
audio webcast of the call via the Company's website. A replay of
the call will also be available through the GW website shortly
after the call. Replay Numbers: (toll free): 1-877-660-6853,
(international): 1-201-612-7415. For both dial-in numbers, please
use conference ID # 13607527.
References
1. Devinsky et al, Epidiolex
(Cannabidiol) in Treatment Resistant Epilepsy, American Academy of
Neurology Annual Meeting, Poster, April 22 2015
2. Devinsky et al, American Academy of
Neurology Annual Meeting, Abstract, April 22 2015
Note Regarding Expanded Access Studies
Expanded access studies are uncontrolled, carried out by
individual investigators, and not typically conducted in strict
compliance with Good Clinical Practices, all of which can lead to a
treatment effect which may differ from that in placebo-controlled
trials. These studies provide only anecdotal evidence of efficacy
for regulatory review. These studies contain no control or
comparator group for reference and these patient data are not
designed to be aggregated or reported as study results. Moreover,
data from such small numbers of patients may be highly variable.
Information obtained from these studies, including the statistical
principles that the independent investigators have chosen to apply
to the data, may not reliably predict data collected via systematic
evaluation of the efficacy in company-sponsored clinical trials or
evaluated via other statistical principles that may be applied in
those trials. Reliance on such information to design our clinical
trials may lead to Phase 2 and 3 trials that are not adequately
designed to demonstrate efficacy and could delay or prevent our
ability to seek approval of Epidiolex. Expanded access programs
provide supportive safety information for regulatory review.
Physicians conducting these studies may use Epidiolex in a manner
inconsistent with the protocol, including in children with
conditions beyond those being studied in GW-sponsored trials. Any
adverse events or reactions experienced by subjects in the expanded
access program may be attributed to Epidiolex and may limit our
ability to obtain regulatory approval with labeling that we
consider desirable, or at all.
Forward-looking statements
This news release may contain forward-looking statements that
reflect GW's current expectations regarding future events,
including statements regarding the therapeutic benefit, safety
profile and commercial value of the company's investigational drug
Epidiolex®, the development and commercialization of Epidiolex,
plans and objectives for product development, plans and objectives
for present and future clinical trials and results of such trials,
plans and objectives for regulatory approval. 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 the 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 Sativex®,
Epidiolex®, and other products by consumer and medical
professionals. A further list and description of risks,
uncertainties and other risks 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.
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 commercialized the world's first plant-derived
cannabinoid prescription drug, Sativex®, which is approved for the
treatment of spasticity due to multiple sclerosis in 27 countries
outside the United States. GW is advancing an orphan drug program
in the field of childhood epilepsy with a focus on Epidiolex®,
which is in Phase 3 clinical development for the treatment of
Dravet syndrome and which is also expected to enter Phase 3
clinical trials in the treatment of Lennox-Gastaut syndrome. GW has
a deep pipeline of additional cannabinoid product candidates which
includes Sativex in Phase 3 clinical development as a potential
treatment of pain associated with advanced cancer, as well as
compounds in Phase 1 and 2 trials for glioma, ulcerative colitis,
type 2 diabetes, and schizophrenia. For further information, please
visit www.gwpharm.com.
CONTACT: Enquiries:
GW Pharmaceuticals plc
(Today) +44 20 3727 1000
Stephen Schultz, VP Investor Relations
(U.S.) 917 280 2424 / 401 500 6570
FTI Consulting (Media Enquiries)
Ben Atwell / Simon Conway (UK)
+ 44 20 3727 1000
Robert Stanislaro (U.S.)
212 850 5657
Trout Group, LLC (U.S. investor relations)
Todd James / Chad Rubin
646 378 2900
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