In the U.S. alone each year,
roughly 150k people develop some form of the seizure spectrum disorder known as
epilepsy, which is the fourth most common neurological disorder known to man
and which is characterized by not only unpredictable seizures, but other health
problems as well, like increased risk of heart disease and stroke. With some 50
million people currently afflicted with epilepsy worldwide and the current
standards of care producing a variety of side effects in up to 90 percent of
patients, as many as 25 percent of all patients discontinue treatment due to
experiencing adverse side effects (many others simply try to cope). Side
effects which range from mood changes, abrupt stoppage-related seizure
frequency increases and liver toxicity, to decreased bone marrow function, and
even birth defects. A staggering one out of every twenty six people will
develop epilepsy at some point in their lives, but the highest incident rates
are among the elderly and the very young, with over 466k children diagnosed
here in the U.S., and over 765k in Europe, according to AAP data published in
Pediatrics back in 2012.
Severe seizures lasting longer
than five minutes (status epilepticus), SUDEP (sudden unexpected death in
epilepsy, which accounts for 34 percent of deaths in children with epilepsy)
and the often-associated social and developmental problems which tragically
lead to an inordinately high incident rate of suicides among epilepsy sufferers
mean that patients with epilepsy have a statistically increased risk of death
compared to the general population, which ranges from 1.6 times higher (low end
of SE/SUDEP), up to as much as 6 times higher (high end of suicide rate).
Thankfully, the emerging clinical understanding of the key role that the human
endogenous cannabinoid system (endocannabinoids) plays in regulating a wide
variety of physiological processes has led to paradigm-shattering advances in
recent years that may soon do away with the current generation of epilepsy
drugs like carbamazepine, phenytoin, valproate (the one most clearly linked to
birth defects), and the barbiturate known as phenobarbital.
One of the companies at the
forefront of cannabinoid science today, which specializes in pioneering the
kind of cutting-edge research that leads not only to new clinical data on the
pharmacokinetics of plant-based cannabinoids (phytocannabinoids) like CBD (the
non-psychoactive component of the cannabis sativa plant, known as cannabidiol)
and THC (the psychoactive component, delta-9-tetrahydrocannabinol), but powerful
new IP for the creation of market-defining therapies, is London-based GW
Pharmaceuticals (NASDAQ: GWPH) (LSE: GWP). The company’s growing understanding
of the importance of the human body’s currently identified (there may be more)
cannabinoid receptors CB1 and CB2, respectively found in neurological tissues,
as well as glands (CB1), and in immune system tissues (CB2), has led to an
impressive product pipeline that has managed to not only garner significant
attention from the medical community, but also seriously impress industry
regulators like the FDA.
The ability of CBD and THC to
act as neuroprotectant antioxidants, helping to secure vital brain function in
the face of degenerative diseases, as well as severe injuries and stroke, is
paramount when it comes to understanding the neuromodulatory potential of
cannabinoid therapies. And the more recent evidence that cannabinoids have
broad-spectrum potential in anti-inflammatory, pain relief and immunomodulatory
applications should give investors the kind of over-the-horizon radar needed to
understand the upper limits for a company like GWPH, whose proprietary
cannabinoid development platform is really just getting warmed up.
The company’s lead candidate
Epidiolex® has seen barriers for its application in Dravet and Lennox-Gastaut
syndromes, two cripplingly severe forms of epilepsy, melt away in recent years.
With FDA Fast Track granted to Epidiolex in Dravet syndrome and pivotal Phase 3
studies now engaged in for both Dravet and Lennox-Gastaut, broader application
in general epilepsy (currently in Phase 2) looks solid. The entry into Phase 2
clinical trials of cannabidivarin (CBDV) in adult patients with epilepsy last
month shows how far-reaching the company’s next-gen epilepsy therapy platform
really is. If the pre-clinical safety and efficacy data is any indication, with
CBDV having been well tolerated at even maximum dosages, with no side effects
or withdrawals, CBDV could soon be stealing even Epidiolex’s thunder.
Hence the recent move of the
company’s CEO, Justin Gover, to the U.S., as well as the appointment of Julian
Gangolli, whose distinguished career at specialty pharma giant Allergan speaks
for itself, to the role of President of the company’s North America operations.
The company is now eagerly setting up shop in SoCal and looks forward to the
possible commercial launch of Epidiolex, for which the most recent physician
reports out in April, referring to its use in children and young adults with
treatment-resistant forms of epilepsy, look extremely promising. GWPH is
banking on big things in North America for the future of their pipeline, and
has struck hard and fast ahead of the curve in order to establish a firm
commercial infrastructure footprint here well before they clear Phase 3, in
order to maximize profitability when the time comes.
With median seizure reduction
after a 12-week run of around 54 percent in reported from the company’s
expanded access program and a clear ability to maintain clinical effect all the
way in at the 24 week mark, as well as a 90 percent patient retention, in whom
Epidiolex was found to be well tolerated, GW Pharmaceuticals is now confidently
gearing up to aggress further pediatric epilepsy targets like TSC (Tuberous
Sclerosis Complex). Having kicked off Phase 3 clinical trials in Lennox-Gastaut
syndrome (LGS) last month, subsequent to the reception of Orphan Drug
Designation from the FDA, GW Pharmaceuticals is roaring towards an NDA as early
as 2016 for Epidiolex in LGS, which continues to be one of the most drug-resistant
and difficult to treat forms of epilepsy, and usually starts before the age of
four in afflicted children.
The importance of a safe,
well-tolerated therapy derived from plants, like the purified and formulated
CBD indication Epidiolex, is absolutely essential for treating children who are
diagnosed as being at risk for epilepsy, as no parent wants to start their
young child out on a lifelong commitment to taking potentially dangerous
synthetic pharmaceuticals like those mentioned earlier. The ability to make a
comfortable decision with something like Epidiolex, secure in the knowledge
that the seizures can be treated without causing the kinds of long-term side
effects like those seen with other epilepsy drugs, is a factor that could make
Epidiolex far more than a massive revenue generation engine for GWPH – it could
make Epidiolex into a meaningfully capable new standard of care.
Moreover, unlike the company’s
other top cannabis-based candidate, the oral spray Sativex® – which is approved
in Europe for multiple sclerosis-related spasticity and which is being worked
up as a cancer pain indication with applicability in other types of pain –
Epidiolex is not licensed to majors like Bayer, Novartis, and Otsuka
Pharmaceutical. This key fact puts GWPH in the pole position when it comes to
capitalizing on the commercialization of Epidiolex, and it should be of
particular note to savvy investors.
Take a closer look by visiting
www.gwpharm.com
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