GW Phar­ma de­tails an im­pres­sive PhI­II case for a cannabis-based drug for se­vere epilep­sy as it preps an NDA

Or­rin Devin­sky, NYU Lan­gone Med­ical Cen­ter

GW Phar­ma­ceu­ti­cals lined up a straight shot at a key ap­proval for Epid­i­olex to­day, pub­lish­ing its im­pres­sive Phase III da­ta for a se­vere form of child­hood epilep­sy which in­spired a block­buster fore­cast for the cannabis-based ther­a­py.

GW an­nounced last spring that Epid­i­olex — a liq­uid ther­a­py us­ing pu­ri­fied cannaba­di­ol — trig­gered a mean re­duc­tion in con­vul­sive seizures of 39% among treat­ment-re­sis­tant pa­tients with Dravet syn­drome, com­pared to on­ly 13% among the place­bo group. A re­port pub­lished in The New Eng­land Jour­nal of Med­i­cine adds to­day that 43% of the drug group had a mean drop of 50% or more — com­pared to 27% in the place­bo group. And 5% stopped hav­ing seizures, with none in the place­bo arm do­ing as well.

GW Phar­ma $GW­PH now plans to file for an FDA ap­proval some­time in the com­ing weeks, with an­a­lysts pro­ject­ing peak sales over a bil­lion dol­lars. GW has had suc­cess­ful tri­als for both Dravet syn­drome as well as an­oth­er rare form of epilep­sy called Lennox-Gas­taut syn­drome, with plans to file for ap­provals on both.

GW’s stock jumped 4.4% on the pub­li­ca­tion of the da­ta, which helps ver­i­fy their re­sults.

We al­so got a much bet­ter look at the safe­ty da­ta in the NE­JM ar­ti­cle. A to­tal of 93% of the pa­tients tak­ing the drug ex­pe­ri­enced a side ef­fect, though most were mild or mod­er­ate. AEs were reg­is­tered for som­no­lence, di­ar­rhea, de­creased ap­petite, fa­tigue, vom­it­ing, pyrex­ia, lethar­gy, con­vul­sion and up­per res­pi­ra­to­ry tract in­fec­tions. Ten pa­tients in the drug arm dropped out of the study, com­pared to 3 pa­tients on place­bo.

Most se­ri­ous­ly, 12 pa­tients ex­pe­ri­enced a spike in liv­er en­zymes — a clas­sic red flag on tox­i­c­i­ty — and 4 dropped out of the study. Of the oth­er 9 who stayed, all saw liv­er en­zyme lev­els re­turn to nor­mal.

Leerink’s Paul Mat­teis, though, sound­ed a note of con­cern about two is­sues that could trip up GW Phar­ma. In a note to in­vestors, he wrote:

From our con­ver­sa­tions with in­vestors we’ve heard two bear points on Epid­i­olex ahead of an NDA fil­ing: (1) that clobazam may have been a ma­jor con­trib­u­tor to Epid­i­olex’s ef­fi­ca­cy [and that this could ul­ti­mate­ly prove to be im­por­tant to or prob­lem­at­ic for FDA]; and (2) that liv­er en­zyme el­e­va­tions on Epid­i­olex could in part hin­der broad use, pos­si­bly via un­fa­vor­able la­bel­ing lan­guage. We don’t re­al­ly un­der­stand the lat­ter con­cern since val­proate – an an­ti-epilep­tic with a black box warn­ing for he­pa­to­tox­i­c­i­ty and more se­ri­ous liv­er en­zyme is­sues than ob­served in GW’s ph3 pro­gram – is broad­ly used in the re­frac­to­ry epilep­sy set­ting. How­ev­er, re­gard­ing the clobazam drug-drug in­ter­ac­tion, seizure re­duc­tion sub-group analy­ses re­main of in­ter­est. GW has sug­gest­ed that these analy­ses could be pre­sent­ed be­fore a po­ten­tial FDA ad­vi­so­ry com­mit­tee but spe­cif­ic guid­ance has not been com­mu­ni­cat­ed.

“Dravet syn­drome is one of the most dif­fi­cult types of epilep­sy to treat and many of the chil­dren in this study were ex­pe­ri­enc­ing dozens, even hun­dreds, of seizures per month de­spite tak­ing mul­ti­ple con­cur­rent an­ti-epilep­tic med­ica­tions,” said Or­rin Devin­sky, the lead au­thor from the NYU Lan­gone Med­ical Cen­ter’s Com­pre­hen­sive Epilep­sy Cen­ter. “These re­sults sug­gest that Epid­i­olex can pro­vide clin­i­cal­ly mean­ing­ful ben­e­fits and I look for­ward to the prospect of an ap­pro­pri­ate­ly stan­dard­ized and test­ed phar­ma­ceu­ti­cal for­mu­la­tion of cannabid­i­ol avail­able as a treat­ment op­tion for these pa­tients.”

Norbert Bischofberger. Kronos

Backed by some of the biggest names in biotech, Nor­bert Bischof­berg­er gets his megaround for plat­form tech out of MIT

A little over a year ago when I reported on Norbert Bischofberger’s jump from the CSO job at giant Gilead to a tiny upstart called Kronos, I noted that with his connections in biotech finance, that $18 million launch round he was starting off with could just as easily have been $100 million or more.

With his first anniversary now behind him, Bischofberger has that mega-round in the bank.

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Francesco De Rubertis

Medicxi is rolling out its biggest fund ever to back Eu­rope's top 'sci­en­tists with strange ideas'

Francesco De Rubertis built Medicxi to be the kind of biotech venture player he would have liked to have known back when he was a full time scientist.

“When I was a scientist 20 years ago I would have loved Medicxi,’ the co-founder tells me. It’s the kind of place run by and for investigators, what the Medicxi partner calls “scientists with strange ideas — a platform for the drug hunter and scientific entrepreneur. That’s what I wanted when I was a scientist.”

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Af­ter a decade, Vi­iV CSO John Pot­tage says it's time to step down — and he's hand­ing the job to long­time col­league Kim Smith

ViiV Healthcare has always been something unique in the global drug industry.

Owned by GlaxoSmithKline and Pfizer — with GSK in the lead as majority owner — it was created 10 years ago in a time of deep turmoil for the field as something independent of the pharma giants, but with access to lots of infrastructural support on demand. While R&D at the mother ship inside GSK was souring, a razor-focused ViiV provided a rare bright spot, challenging Gilead on a lucrative front in delivering new combinations that require fewer therapies with a more easily tolerated regimen.

They kept a massive number of people alive who would otherwise have been facing a death sentence. And they made money.

And throughout, John Pottage has been the chief scientific and chief medical officer.

Until now.

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Chas­ing Roche's ag­ing block­buster fran­chise, Am­gen/Al­ler­gan roll out Avastin, Her­ceptin knock­offs at dis­count

Let the long battle for biosimilars in the cancer space begin.

Amgen has launched its Avastin and Herceptin copycats — licensed from the predecessors of Allergan — almost two years after the FDA had stamped its approval on Mvasi (bevacizumab-awwb) and three months after the Kanjinti OK (trastuzumab-anns). While the biotech had been fielding biosimilars in Europe, this marks their first foray in the US — and the first oncology biosimilars in the country.

Seer adds ex-FDA chief Mark Mc­Clel­lan to the board; Her­cules Cap­i­tal makes it of­fi­cial for new CEO Scott Bluestein

→ On the same day it announced a $17.5 million Series C, life sciences and health data company Seer unveiled that it had lured former FDA commissioner and ex-CMS administrator Mark McClellan on to its board. “Mark’s deep understanding of the health care ecosystem and visionary insights on policy reform will be crucial in informing our thinking as we work to bring our liquid biopsy and life sciences products to market,” said Seer chief and founder Omid Farokhzad in a statement.

Daniel O'Day

No­var­tis hands off 3 pre­clin­i­cal pro­grams to the an­tivi­ral R&D mas­ters at Gilead

Gilead CEO Daniel O’Day’s new task hunting up a CSO for the company isn’t stopping the industry’s dominant antiviral player from doing pipeline deals.

The big biotech today snapped up 3 preclinical antiviral programs from pharma giant Novartis, with drugs promising to treat human rhinovirus, influenza and herpes viruses. We don’t know what the upfront is, but the back end has $291 million in milestones baked in.

Vas Narasimhan, AP Images

On a hot streak, No­var­tis ex­ecs run the odds on their two most im­por­tant PhI­II read­outs. Which is 0.01% more like­ly to suc­ceed?

Novartis CEO Vas Narasimhan is living in the sweet spot right now.

The numbers are running a bit better than expected, the pipeline — which he assembled as development chief — is performing and the stock popped more than 4% on Thursday as the executive team ran through their assessment of Q2 performance.

Year-to-date the stock is up 28%, so the investors will be beaming. Anyone looking for chinks in their armor — and there are plenty giving it a shot — right now focus on payer acceptance of their $2.1 million gene therapy Zolgensma, where it’s early days. And CAR-T continues to underperform, but Novartis doesn’t appear to be suffering from it.

So what could go wrong?

Actually, not much. But Tim Anderson at Wolfe pressed Narasimhan and his development chief John Tsai to pick which of two looming Phase III readouts with blockbuster implication had the better odds of success.

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On a glob­al romp, Boehringer BD team picks up its third R&D al­liance for Ju­ly — this time fo­cused on IPF with $50M up­front

Boehringer Ingelheim’s BD team is on a global deal spree. The German pharma company just wrapped its third deal in 3 weeks, going back to Korea for its latest pipeline pact — this time focused on idiopathic pulmonary fibrosis.

They’re handing over $50 million to get their hands on BBT-877, an ATX inhibitor from Korea’s Bridge Biotherapeutics that was on display at a science conference in Dallas recently. There’s not a whole lot of data to evaluate the prospects here.

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Servi­er scoots out of an­oth­er col­lab­o­ra­tion with Macro­Gen­ics, writ­ing off their $40M

Servier is walking out on a partnership with MacroGenics $MGNX — for the second time.

After the market closed on Wednesday MacroGenics put out word that Servier is severing a deal — inked close to 7 years ago — to collaborate on the development of flotetuzumab and other Dual-Affinity Re-Targeting (DART) drugs in its pipeline.

MacroGenics CEO Scott Koenig shrugged off the departure of Servier, which paid $20 million to kick off the alliance and $20 million to option flotetuzumab — putting a heavily back-ended $1 billion-plus in additional biobuck money on the table for the anti-CD123/CD3 bispecific and its companion therapies.