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GW Pharmaceuticals (GWPH) Safe, Effective Cannabinoid Pipeline Advancing Through & Beyond Epilepsy into Pain Management

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.

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