The following excerpt is from the company's
SEC filing.
- Three Phase 3 Epidiolex clinical trials fully recruited above
target sample size – on track for initial data in Q1 2016 -
- Significant clinical activity in multiple cannabinoid pipeline
programs -
London, UK, 7 Dec 2015
: 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, announces financial
results for the fourth quarter and twelve months end ed 30
September 2015.
“This last year has seen GW maintain a rapid pace in the
progress of our pediatric epilepsy research program, including the
start and completion of recruitment of multiple Phase 3 trials for
Epidiolex, and the expansion of our expanded access program which
has continued to yield promising safety and efficacy data,” stated
Justin Gover, GW’s Chief Executive Officer. “We expect to carry
this momentum through 2016 with top-line data from four Epidiolex
pivotal trials, our first NDA filing, build-out of our U.S.
commercial organization, and ongoing data read-outs from a number
of clinical pipeline programs.”
2015 HIGHLIGHTS
(CBD) childhood epilepsy program:
Company sponsored Phase 3 development programs in Dravet
syndrome and Lennox-Gastaut syndrome
First Phase 3 Dravet syndrome trial fully enrolled above
original target sample size (120 randomized) data expected Q1
2016
First LGS Phase 3 trial fully enrolled above original target
sample size (171 randomized). Data expected Q2 2016
Second LGS Phase 3 trial fully enrolled above original target
sample size. (>210 expected to randomize). Data expected Q2
2016
Phase 3 Dravet syndrome trial ongoing. Data expected mid
2016
NDA submission with FDA expected Q4 2016
Phase 3 Tuberous Sclerosis Complex trial expected to commence Q1
2016
Additional clinical development for Epidiolex beyond initial
three indications expected to commence in H2 2016
Expanded access program:
Separate data update announcement issued today at the American
Epilepsy Society December 2015 Annual Meeting
Approximately 350 children on treatment at 32 U.S. clinical
sites
Over 850 children authorized for treatment by FDA under Expanded
Access Treatment INDs and 6 U.S. State programs
Strategic agreement with Government of New South Wales in
Australia to conduct Epidiolex and CBDV clinical trials
CBD and CBDV patent portfolio strengthened
Advanced clinical programs in multiple cannabinoid pipeline
product candidates:
Phase 2a CBD schizophrenia study data shows positive
proof-of-concept with a reassuring safety profile
Phase 2 CBDV epilepsy study in adults underway with data
expected H2 2016
Neonatal Hypoxic-Ischemic Encephalopathy (NHIE) IV CBD Phase 1
clinical program expected to commence in H2 2016
Orphan Drug and Fast Track Designations granted from FDA and
EMA
Clinical trials within the field of autism spectrum disorders
expected to commence in H2 2016
Phase 1b/2a study for the treatment of Recurrent Glioblastoma
Multiforme (GBM) fully enrolled with data expected in mid-2016
Orphan Drug Designation granted from FDA
Phase 2 study in type-2 diabetes fully enrolled with data
expected mid 2016
Phase 2 study of Sativex in spasticity due to cerebral palsy
ongoing with data expected mid 2016
Pre-clinical progress addressing a number of areas of unmet need
including autism spectrum disorders, duchenne muscular dystrophy,
glioma, ovarian and pancreatic cancers, and chemotherapy-induced
cachexia
U.S. operations established in Carlsbad, California
GW’s CEO, Justin Gover, relocates to the U.S.
Seasoned industry executive Julian Gangolli appointed as
President, North America
Epilepsy specialist team build-out underway
FINANCIAL HIGHLIGHTS
Balance sheet further strengthened with successful NASDAQ
follow-on offering raising net proceeds after expenses of £127.5
million ($193.3 million)
Revenue for the twelve months ended 30 September 2015 of £28.5
million ($43.2 million) compared to £30.0 million for the twelve
months ended 30 September 2014.
Loss for the twelve months ended 30 September 2015 of £44.6
million ($67.4 million) compared to £14.7 million for the twelve
months ended 30 September 2014.
Cash and cash equivalents at 30 September 2015 of £234.9 million
($355.3 million) compared to £164.5 million as at 30 September
2014.
Conference Call and Webcast Information
GW Pharmaceuticals will host a conference call and webcast to
discuss the 2015 fourth quarter and year-end financial results
today at 4:30 p.m. EST / 9:30 p.m. GMT. To participate in the
conference call, please dial 877-407-8133 (toll free from the U.S.
and Canada), or 0800-756-3429 (toll free from the UK) or
201-689-8040 (international). Investors may also access a live
audio webcast of the call via the investor relations section of the
Company’s website at
http://www.gwpharm.com
. A replay of the call will also be available through the GW
website shortly after the call and will remain available for 90
days. Replay Numbers: (toll free):1-877-660-6853,
(international):1-201-612-7415. For both dial-in numbers please use
conference ID #13626013.
Enquiries:
GW Pharmaceuticals plc
Stephen Schultz, VP Investor Relations
401 500 6570
FTI Consulting (Media Enquiries)
Ben Atwell / Simon Conway
+ 44 20 3727 1000
Peel Hunt LLP (UK NOMAD)
James Steel
+44 20 7418 8900
(“GW” or “the Company” or “the Group”)
Preliminary Results for the Fourth Quarter and Year Ended 30
September 2015
OPERATIONAL OVERVIEW
GW Overview
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. In its 17 years of operations, GW has established a
world leading position in the development of plant-derived
cannabinoid therapeutics through its proven drug discovery and
development processes, intellectual property portfolio and
regulatory and manufacturing expertise. 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 28 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 Lennox-Gastaut syndrome and which is also
expected to enter Phase 3 clinical trials in the treatment of
Tuberous Sclerosis Complex in early 2016. GW has a deep pipeline of
additional cannabinoid product candidates which includes clinical
development across a variety of compounds for both orphan and
non-orphan indications.
U.S. Operations Update
In June 2015, GW announced the relocation of the Company’s CEO,
Justin Gover, to the U.S. and the appointment of Julian Gangolli to
the newly created position of President, North America. Previously,
Mr Gangolli was President of the North American Pharmaceutical
division of Allergan Inc., with responsibility for a 1,400-person
integrated commercial operation with sales exceeding $3.8 billion
in 2014. Together, Mr. Gangolli and Mr. Gover are leading GW’s
growth in the U.S., preparations for the expected NDA submission
and build-out of GW’s in-house U.S. commercial infrastructure to
support the launch of Epidiolex. Mr. Gangolli and Mr. Gover are
based at GW’s new U.S. office in Carlsbad, California.
U.S. Follow-on Offering
In May 2015, GW successfully completed a U.S. follow-on offering
on the NASDAQ Global Market issuing a total of 1,840,000 American
Depositary Shares (“ADSs”) at a price of $112.00 per ADS. This
total included the full exercise of the underwriter’s option and
resulted in total net proceeds after expenses of approximately
$193.3 million (£127.5 million). The funds raised in this offering
are primarily intended to support further expansion of GW’s
Epidiolex manufacturing capability and build-up of inventory in
preparation for U.S. launch, if Epidiolex is approved; clinical
development of other orphan indication opportunities for Epidiolex,
with an initial focus on Tuberous Sclerosis Complex; the
advancement of other pipeline opportunities, including an
intravenous CBD formulation in the treatment of Neonatal
Hypoxic-Ischemic Encephalopathy
(NHIE); pre-launch commercialization activities for Epidiolex in
the United States; and for other general corporate purposes.
Epilepsy in the United States
Epilepsy is one of the most common neurological disorders in
children. According to Russ et al in the February 2012 edition of
Pediatrics, there are 466,000 childhood epilepsy patients in the
United States and 765,000 patients in Europe, of which 30%, or
about 140,000 patients in the United States and about 230,000 in
Europe, are deemed medically intractable or pharmacoresistant.
According to Kwan and Brodie in the February 2000 edition of the
New England Journal of Medicine, 36% of patients with epilepsy were
pharmacoresistant. Of the patients in the study, 47% became
seizure-free during treatment with their first antiepileptic drug,
13% became seizure-free during treatment with a second
anti-epileptic drug as a monotherapy, and 4% became seizure-free
with a third anti-epileptic drug or treatment with multiple
anti-epileptic drugs. The remaining 36% of patients were classified
by the authors as having pharmacoresistant epilepsy. Furthermore it
is recognized that some of those that do find relief often suffer
side effects severe enough with their current medication that an
alternative or adjunct is often sought. The costs of uncontrolled
epilepsy are significant, with direct and indirect costs associated
with epilepsy totaling more than $15 billion per year. 50,000
epilepsy related deaths occur each year, more than breast cancer
deaths annually.
Epilepsy Drug Development Programs
GW’s epilepsy franchise centers around two product candidates,
Epidiolex, a liquid formulation of pure plant-derived cannabidiol
(CBD), and GWP42006 (CBDV). GW is currently pursuing the
development of CBD for a series of individual orphan
epilepsy-related indications providing GW with significant new
market opportunities. GW retains all rights to commercialize any
and all products that evolve from these programs.
GW is undertaking a formal development program for Epidiolex in
the field of severe, drug-resistant childhood epilepsy with initial
focus on conducting formal development programs in the treatment of
three indications - Dravet syndrome, Lennox-Gastaut syndrome (LGS)
and Tuberous Sclerosis Complex (TSC). The Company has to date
received Orphan Drug Designation from the U.S. Food and Drug
Administration (FDA) for Epidiolex for the treatment of both Dravet
syndrome and LGS. Additionally, GW has received Fast Track
Designation from the FDA and Orphan Designation from the European
Medicines Agency (EMA) for Epidiolex for the treatment of Dravet
syndrome.
There is a significant effort to identify the mechanisms of
action that underpin the clinical effectiveness of Epidiolex (and
other cannabinoids) in epilepsy, including investigation of the
effect of cannabinoids on epilepsy associated gene expression.
Based on recent findings in
The Journal of Pharmacology and Experimental Therapeutics
, CBD is likely to be acting via more than one mechanism of
action with the effect of reducing neuronal hyperexcitability.
Importantly, the anti-seizure effects of CBD are not dependent on
cannabinoid receptors, nor on sodium channels.
Dravet Syndrome
According to Forsgren L. et al. in the 2004 edition of Epilepsy
in Children, the incidence of epilepsy in the first year of life is
1.5 per 1,000 people, or, by our estimate, 6,450 new epilepsies per
year worldwide. According to Dravet et al. in the 2012 edition of
Epileptic Syndromes in Infancy, Childhood and Adolescence, up to 5%
of epilepsies diagnosed in the first year of life are Dravet
syndrome, equating to 320 new cases per year in the United States
with a mortality rate that studies have shown may be as high as 15%
in the first 20 years of life, or, by our estimate, 5,440 patients
with Dravet in the United States under the age of 20 years.
Applying the same assumptions in Europe, we believe there are an
estimated 6,710 Dravet patients in the European Union. It is likely
that these figures are a low estimate, however GW believes that
this syndrome is likely underdiagnosed.
Epidiolex development in Dravet syndrome
GW commenced a Phase 2/3 clinical trial of Epidiolex for Dravet
syndrome in October 2014. This trial is designed as a two-part
randomized double-blind, placebo-controlled parallel group dose
escalation, safety, tolerability, pharmacokinetic and efficacy
trial of single and multiple doses of Epidiolex to treat Dravet
syndrome in children who are not responding adequately to other
anti-epileptic drugs. The first part of this trial was completed in
February 2015, and included a dose-ranging pharmacokinetic and
safety evaluation in a total of 34 patients over a 3-week treatment
period.
Following a review of the Part A data by an independent panel,
Part B of the trial commenced in March 2015 and is a Phase 3
pivotal placebo-controlled safety and efficacy evaluation of
Epidiolex (at a dose of 20mg/kg per day) over a 3-month treatment
period. Originally expected to recruit 100 patients, this trial
reached a total of 120 patients randomized. In April 2015, GW
commenced an additional dose-ranging Phase 3 trial in Dravet
syndrome which is recruiting 150 patients. This placebo-controlled
trial differs from the first Phase 3 trial in that it includes two
Epidiolex dose arms, at 20mg/kg and at 10mg/kg. Both of these
studies will be the largest known controlled trials in Dravet
syndrome.
GW expects to report top-line results from the Phase 2/3 pivotal
safety and efficacy study in the first quarter of 2016 and results
from the second Phase 3 trial around mid 2016.
The primary measure of efficacy in both trials will be the
comparison between Epidiolex and placebo in the percentage change
from baseline in number of convulsive seizures.
It is important to note that the protocols for both the
Epidiolex Dravet syndrome and LGS Phase 3 trials allow for a
prospective pooling of data within each indication, which is
endorsed by the FDA in its recent guidance (Integrated Summary of
Effectiveness “ISE” – October 2015). The recommendations in this
guidance reflect the FDA’s current thinking regarding information
that industry should include in an ISE to provide an integrated
analysis that offers insights beyond those observable in individual
clinical trials, where NDA filers are “encouraged to provide an ISE
because it represents an opportunity to present a coherent analysis
and presentation of the drug’s benefits.”
Lennox-Gastaut Syndrome (LGS)
The Lennox-Gastaut syndrome (LGS) is a type of epilepsy with
multiple types of seizures, particularly tonic (stiffening) and
atonic (drop) seizures. LGS accounts for about 3% to 4% of
childhood epilepsies with the cause of the disorder unknown in 1
out of 4 children.
LGS affects between 14,500 – 18,500 children under the age of 18
in the U.S. and over 30,000 children and adults in the U.S.
80% of children with LGS continue to experience seizures,
psychiatric, and behavioral deficits in adulthood. Seizures due to
LGS are hard to control and they generally
require life-long treatment as LGS usually persists into the
adult years. Historically
, patients with LGS have had few effective treatment options.
Intellectual and behavioral problems associated with LGS are common
and add to the complexity of this syndrome and the difficulties in
managing life with LGS.
Epidiolex development in LGS
In May 2015 we commenced the Phase 3 pivotal trials program for
Epidiolex in LGS. The first Phase 3 trial is a placebo-controlled
safety and efficacy evaluation of Epidiolex (at a dose of 20mg/kg
per day) over a 3-month treatment period. Originally expected to
recruit 100 patients, this trial reached a total of 171 patients
randomized. The second placebo-controlled Phase 3 trial differs
from the first Phase 3 LGS trial in that it includes two Epidiolex
dose arms, at 20mg/kg and at 10mg/kg. Originally expected to
recruit 150 patients, it has completed enrolment and is estimated
to have a total of more than 210 patients randomized. We expect to
report top-line results from both trials in the second quarter of
2016.The primary measure of efficacy in both trials will be the
comparison between Epidiolex and placebo in the percentage change
from baseline in number of drop attacks.
TSC is a genetic disorder that causes non-malignant tumors to
form in many different organs, with the brain and skin being the
most commonly affected tissues. TSC results from a mutation in
tumor suppression genes TSC1 or TSC2 and is estimated to affect
approximately 50,000 patients in the United States. The most common
clinical feature 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 these TSC patients (or
approximately 25,000 of patients in the U.S.) 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.
Epidiolex development in TSC
Based on the findings in the physician-led expanded access
program, GW announced earlier this year that its third target
indication will be TSC and expects to commence Phase 3 clinical
development in early 2016.
Epidiolex U.S. Expanded Access Program
In parallel with the GW’s formal clinical trial program, the FDA
has granted 20 intermediate expanded access INDs to independent
physician investigators in the U.S to treat a total of over 450
children and young adults suffering from intractable epilepsy with
Epidiolex. In addition, the FDA has granted further INDs to treat
400 additional patients under expanded access programs supported by
6 U.S. states and for which GW is supplying Epidiolex. The FDA may
authorize expanded access programs to facilitate access to
investigational drugs for treatment use for patients with a serious
or immediately life-threatening disease or condition who lack
therapeutic alternatives. The FDA has also granted to physicians 11
individual emergency INDs and 3 individual patient non-emergency
INDs. As at mid November, approximately 350 children are on
treatment at 32 U.S. clinical sites
Seven abstracts related to GW programs were presented at the
69
Annual Meeting of the American Epilepsy Society including data
from the physician-led Epidiolex expanded access program. As
reported today in a separate press release from GW, physician
reports of clinical effect and safety data were presented on 261
children and young adults with treatment-resistant epilepsy who
have been treated with Epidiolex, for a period of at least 12 weeks
(Devinsky et al). These data are from 16 clinical sites in the
United States and were generated under expanded access INDs
authorized by the FDA. In addition, physician reports of safety
data were presented on 313 patients (261 patients with 12 weeks
treatment effect data plus additional 52 patients still in their
first 12 weeks of treatment or who withdrew from treatment). The
expanded access program comprises clinical studies performed by
individual physicians. Highlights from this data include:
Included within the information presented is new data issued in
a poster presentation on 261 patients enrolled in this expanded
access program, representing a two-fold increase in patient numbers
over the previous disclosure in April 2015
Promising signals of efficacy have been reported, with median
reduction in total seizures of 45% across all patients after 12
weeks treatment, and maintenance of clinical effect at 36 weeks
47% of patients experienced a ≥50% reduction in seizures after
12 weeks treatment
Epidiolex was well tolerated - only 4% of patients withdrew due
to side effects
Additional data in patients with diagnoses of Dravet syndrome,
LGS and TSC, the targets of the GW sponsored Phase 3 clinical trial
program for Epidiolex, were presented and this data further
supports GW’s choice of these indications
Additional information on the presentations at the annual
meeting can be found online at
www.aesnet.org
CBDV (cannabidivarin) Development Program
GW is developing a second epilepsy product candidate, GWP42006,
which features the non-psychoactive cannabinoid CBDV as the primary
cannabinoid. CBDV is distinct in chemical structure to CBD and has
shown anti-seizure properties across a range of
in vitro
in vivo
models. GW has completed a Phase 1 trial of GWP42006 by the oral
and intravenous route in 66 healthy subjects. In this trial,
GWP42006 was well tolerated even at the highest tested dose. There
were no serious or severe adverse events, nor any withdrawals due
to adverse events.
In 2015, GW commenced a Phase 2 study of GWP42006 in patients
with epilepsy and expects data from that study in the second half
of 2016.
GWP42006 has the potential for development in the field of
pediatric epilepsy as well as the broader epilepsy market.
In October 2015, GW announced that it had signed a Memorandum of
Understanding (MOU) with the Government of New South Wales in
Australia to progress a research program for Epidiolex and CBDV in
children with severe, drug resistant childhood epilepsy. The MOU
will facilitate:
A world first, Phase 2 clinical trial in children for GWP42006
(CBDV)
A compassionate access program for Epidiolex
Provision for NSW to host additional Phase 3 clinical trials of
Epidiolex
A Phase 4 clinical trial of Epidiolex (to follow completion of
the Phase 3 studies)
CBD/CBDV Intellectual Property Portfolio
GW’s patent portfolio related to the use of CBD and/or CBDV
includes fourteen patent families containing one or more pending
and/or issued patents with claims in the treatment of epilepsy,
compositions, extraction techniques, CBD and CBDV extracts and
highly purified plant-derived CBD.
These include Notices of Allowance received this year from the
U.S. Patent and Trademark Office for patent applications protecting
the use of CBD in the treatment of partial seizures and for CBDV in
the treatment of patients with epilepsy. The issued patents from
these applications will provide an exclusivity period until June
2030 and March 2031 respectively. We have also recently had a
patent granted in the UK which claims the use of CBD in combination
with certain standard anti-epileptic drugs in the treatment of
epilepsy. Equivalent patent applications are being prosecuted in
the US and at the European Patent Office.
Epidiolex Manufacturing & Production
GW is actively scaling its growing and manufacturing of
Epidiolex to meet anticipated commercial demand, if approved. From
its extensive experience in growing CBD botanical raw material, GW
is able to utilize a range of growing methods to generate
significant quantities of CBD botanical raw material derived from
its proprietary CBD plant chemotypes. In 2015, production increased
by a factor of approximately 20 times compared with the previous
year and is expected to double again in 2016. This will equate to
approximately 200 Tonnes in 2016 and when purified and formulated,
results in approximately 1.6 million 100mg/ml bottles of Epidiolex.
With expectations of significant demand for Epidiolex upon
approval, GW plans to continue expansion of its Epidiolex plant
growing capacity well beyond this 2016 goal. The finished product,
Epidiolex, is a liquid formulation of pure CBD and there are
several processing steps beyond growing to ensure that the product
is pure, meets the required FDA specification, and can be
manufactured at scale to current Good Manufacturing Practice
Regulations (cGMP). Each step has already been scaled and a further
increase in scale is anticipated during 2016.
GW believes it is on track to be ready for FDA pre-approval
inspection from H2 2016 onwards.
U.S. Legislative Update
Following NDA approval of a drug containing a Schedule I
controlled substance, that substance must be rescheduled as a
Schedule II, III, IV or V substance before it can be marketed. On
November 17, 2015, H.R. 639, Improving Regulatory Transparency for
New Medical Therapies Act, passed through both houses of Congress
and on November 25, the Bill was signed into law. The new law
removes uncertainty associated with timing of the DEA rescheduling
process after NDA approval. Specifically, it requires DEA to issue
an “interim final rule,” pursuant to which a manufacturer may
market its product within 90 days of FDA approval. The new law also
preserves the period of orphan marketing exclusivity for the full
seven years such that this period only begins after DEA scheduling.
This contrasts with the previous situation whereby the orphan
“clock” began to tick upon FDA approval, even though the product
could not be marketed until DEA scheduling was complete.
Other Orphan/Neurology Pipeline Programs
Schizophrenia
GW’s product candidate, an oral formulation of GWP42003 (CBD),
has shown notable anti-psychotic effects in accepted pre-clinical
models of schizophrenia and has also demonstrated the ability to
reduce the characteristic movement disorders induced by currently
available anti-psychotic agents.
In September 2015, GW announced positive top line results from
an exploratory Phase 2a placebo-controlled clinical trial of CBD in
88 patients with schizophrenia who had previously failed to respond
adequately to first line anti-psychotic medications. Over a series
of exploratory endpoints, CBD was consistently superior to placebo,
with the most notable differences being in the PANSS positive
sub-scale (p=0.018), the Clinical Global Impression of Severity
(p=0.04) and Clinical Global Impression of Improvement (p=0.02).
The proportion of responders (improvement in PANSS Total score
greater than 20%) on CBD was higher than that of participants on
placebo and the Scale for Assessment of Negative Symptoms showed a
trend in favour of CBD which reached statistical significance for
patients taking CBD together with one of the leading first line
anti-psychotic medications. The safety profile of CBD was
particularly reassuring, with no serious adverse events and an
overall frequency of adverse events very similar to placebo
GW believes that the signals of efficacy demonstrated in this
trial, together with a notably reassuring safety profile, provide
the Company with the prospect of new and distinct cannabinoid
neuropsychiatric product pipeline opportunity as the mechanism of
CBD does not appear to rely on the dopamine D2 receptor
augmentation of standard antipsychotics. GW is now analysing the
data to fully understand the appropriate next steps regarding
product development in schizophrenia with future research likely
focused on pediatric orphan neuropsychiatric indications.
Neonatal Hypoxic-Ischemic Encephalopathy (NHIE)
NHIE is acute or sub-acute brain injury resulting from
deprivation of oxygen during birth (hypoxia). GW estimates 6,500 to
12,000 cases of NHIE occur in the U.S. each year. Of these, 35% are
expected to die in early life and 30% are expected to develop
persistent neurologic disability. There are currently no
FDA-approved medicines specifically indicated for NHIE. The current
standard of care for NHIE patients is to induce whole-body
hypothermia. Even if a patient is put into induced hypothermia
there is still a significant rate of morbidity and mortality, with
a meta-analysis of the available data revealing a 27% death rate.
Among the patients who survive NHIE, 28% suffer from major
neurodevelopment issues and 26% develop Cerebral Palsy.
GW intends to commence a clinical development program with the
FDA and expects to start a Phase 1 clinical study in healthy
volunteers for an intravenous CBD formulation in the treatment of
NHIE in H1 2016. GW has received Orphan Drug and Fast Track
Designations from the FDA for CBD for the treatment of NHIE and has
recently received Orphan Designation from the European Medicines
Agency for the treatment of Perinatal Asphyxia, an alternate term
that describes the same condition.
Autism Spectrum Disorders
Many of the pediatric intractable epilepsy conditions within the
Epidiolex Expanded Access Program share considerable overlap with
Autism Spectrum Disorders (ASD). Early clinical observations from
treating physicians suggest a potential role for cannabinoids in
addressing problems associated with ASD; they may be able to treat
deficits in cognition, behavior and communication. Consequently,
there are several ongoing initiatives within GW to evaluate a range
of cannabinoids in pre-clinical models of ASD, with a focus on, but
not limited to, those caused by single genetic aberrations. These
conditions often fall...
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