How cannabis-based drugmaker GW finally cracked America

GW's painstaking efforts to develop medicines from cannabis, and win the approval of regulators, are paying off

When a pair of British doctors set out to make medicines from the cannabis plant 16 years ago, they knew it would take some time to crack the world's biggest pharmaceuticals market.

But Geoffrey Guy and Britain Whittle could hardly have predicted that when their company, GW Pharmaceuticals, finally did break into the US, it would come amid such fanfare.

The fortunes of GW have taken a dramatic turn in the last year after US authorities turned to them for a neat solution to a political conundrum. It has now landed a starring role in one of America's most high profile issues: using cannabis derivatives to treat children with life-threatening epilepsy.

In November, GW was granted permission to supply its experimental epilepsy drug Epidiolex, never before tested on humans, to a handful of specialist paediatricians in the US to use on their patients.

It is supplying the medicine free of charge, but in return has seen the first evidence that the drug could work in real patients. Twenty-seven children with debilitating forms of epilepsy - the type that can cause tens or even hundreds of seizures a day - were enrolled in the programme, Thirteen saw their seizures drop by a half after three months of treatment and four became completely seizure-free.

These results could be game-changing for a drug which had been on GW's backburner for more than a decade, during which it quietly amassed a body of scientific evidence on its medical potential.

A major clinical will start later this year, spanning patients across the US and Europe, including Great Ormond Street Hospital in London. GW hopes this will confirm early signs that the oil works, and that it could be approved as early as 2016.

"In the pharmaceuticals industry you live and dream for a moment when you feel like you've got an opportunity to make a dramatic difference to a set of patients with an unmet need," says Justin Gover, who has led the company since its founding in 1998.

"A huge number of collective forces have come together in the last year to put us in that position."

A perfect political storm provided the catalyst for GW's rise to prominence in the US last year. Parents of severely epileptic children were flocking to illegal cannabis growers, desperate to get hold of an extract known as cannabidiol, or CBD, which had been shown to dramatically reduce seizure frequency.

Their willingness to go to such lengths is unsurprising in light of reports claiming the oil could dramatically decrease or even eliminate seizures.

Washington, under increasing pressure to decriminalise the actions of despairing parents, could either change the law - still unthinkable on a federal level despite several states legalising medical marijuana - or find a legitimate source of CBD.

GW's new found acceptance in the US has had an intoxicating effect on its stock: from a low of 39.50p in March last year the shares have soared more than tenfold and currently trade for around £4.

At first glance it looks like an overnight success, but Gover says GW owes its shot at prime time to long hard graft.

"We started talking to the US authorities about our programme in 1999, a year after the company was founded, and a full seven years before we ever met with the FDA," he says.

"We realised the importance of the US market for our products but also the time and effort it would take to ensure that we had the standing in front of the right agencies, the track record and the quality of science to show we were serious about this."

GW's history in the US has coincided with a loosening of attitudes towards cannabis. Since 1998, around half of all states have passed laws permitting the medical use of the drug. This year, a handful of states went further to completely legalise the drug. Nonetheless, cannabis is still categorised as a "schedule one" substance on the federal level, sharing a category with heroin and putting it one notch above cocaine.

Gover does not see a link between the wider acceptance of medical cannabis and his company's improving prospects in the country, since the FDA operates on a federal level. But he is pleased that the swell of state-wide legalisation has increased awareness of the medical potential of the cannabis plant.

"The prospect of cannabinoids as therapeutics is [now] much more commonly understood and appreciated. In 1998 people would say [people feel better] because they're high. Now it seems more obvious there are medical properties here which need to be explored further," he says.

Still, GW is careful to extract itself from the controversial world of "medical marijuana".

Gover insists that his efforts to brandish GW's unique position as a regular pharmaceutical company which happens to use compounds from the cannabis plant do not amount to lobbying, though GW has recruited some political heavyweights to aid its cause in the past.

In 2005 it hired former deputy drugs czar and high profile medical marijuana critic Andrea Barthwell to help GW distinguish its products from medical cannabis.

"It was a part of an evolution," says Gover, adding that Barthwell and other recruits in that period understood the tension between concerns about cannabis being used as an herbal material, even for medical purposes, and the potential for the plant to be exploited in a pharmaceutical way.

GW has marched by this drumbeat ever since its founding. In the early days, Dr Guy described his scientific goal as "bottling the essence of cannabis", but the company's language has moved on since then to push an even bigger wedge between its products and the plant they come from.

"It was always about harnessing the potential of the molecules in the cannabis plant, which seem to have multiple therapeutic targets," he says.

Epidiolex may have secured the spotlight in the last year, but it is GW's much longer journey to US approval for its lead product, Sativex, that best reflects its tenacity.

The company waited until Sativex, which relieves muscle spasms in multiple sclerosis, had been given the green light in Canada before approaching the FDA in 2005.

"We felt the first approach we made to the FDA needed to be at the right time with the right data, because it would set the tone for all the future interaction," says Gover.

"[Canadian approval] was a serious and credible development."

The waiting game paid off. The FDA opened the door for US patients to be involved in a major clinical trial on Sativex in cancer pain in early 2006. GW has not yet completed clinical testing, but the FDA has endorsed the medicine with a "fast track" status, meaning it will be accelerated through regulatory approval.

As far as winning US approval for Sativex in multiple sclerosis is concerned, GW has proceeded with caution. Last year it agreed to run a trial in American patients to determine the safety of the drug, despite having already received the green light from regulators in 22 other countries.

But as GW's prospects brighten in America, they look to be dimming at home, in the country which enabled the research to happen in the first place.

The British Government was instrumental in GW's founding by giving the company special permission to grow industrial quantities of the illegal drug in a top secret, high security location thought to be near the south coast of England.

But recent draft guidelines on multiple sclerosis treatment from the UK's National Institute for Health and Care Excellence advised against the use of Sativex for spasm relief, sparking outrage from patient groups who say they were not consulted.

If the final document, expected in October, leaves the guidance unchanged, patients will be forced to pay out of pocket to get Sativex. In an unusual twist, the drug will be made widely available in Wales, where the medicines adviser has conducted its own cost-benefit analysis on the drug and recommended its use.

Gover is frustrated by this development, though he is hoping for an about-turn in the final guidelines.

"The message it sends is go back to 1998 and smoke cannabis, essentially.

"Which is not the message we or any authority would want to send."

http://www.telegraph.co.uk/finance/newsbysector/pharmaceuticalsandchemicals/11037982/How-cannabis-based-drugmaker-GW-finally-cracked-America.html 16/08/2014