GW on fire: PhI­II epilep­sy da­ta sig­nals suc­cess ahead for first CBD drug

Im­age: Justin Gov­er, GW Phar­ma

The British com­pa­ny shoot­ing to be the first to launch a drug de­rived from the cannabis plant just got a big nod from the sci­en­tif­ic com­mu­ni­ty, paving the way for what could be one of the biggest block­buster launch­es of the year.

Peer-re­viewed jour­nal The Lancet pub­lished Phase III da­ta on GW Phar­ma­ceu­ti­cals’ cannabid­i­ol med Epid­i­olex, which was stud­ied in a kind of pe­di­atric epilep­sy called Lennox-Gas­taut syn­drome (LGS).

The drug is a phar­ma­ceu­ti­cal for­mu­la­tion of CBD, a com­pound de­rived from cannabis that lacks the psy­chotrop­ic ef­fects of THC. Al­though a syn­thet­ic ver­sion of THC was ap­proved by the FDA back in the 1980s, this would be the first reg­u­la­to­ry ap­proval of a weed-de­rived phar­ma­ceu­ti­cal prod­uct, if ap­proved.

GW trum­pet­ed its promis­ing PhI­II da­ta in LGS af­ter the tri­al wrapped up back in 2016, but The Lancet’s val­i­da­tion is mean­ing­ful to both pa­tients and in­vestors, GW’s CEO Justin Gov­er tells me. That’s be­cause Epid­i­olex is what biotech com­pa­nies like to call a “pipeline in a prod­uct,” as it’s be­ing test­ed in sev­er­al epilep­tic in­di­ca­tions. Suc­cess in a dif­fi­cult-to-treat epilep­sy such as LGS boosts in­vestors’ con­fi­dence that the drug will per­form well in oth­er epilep­tic con­di­tions. Wall Street has es­ti­mat­ed peak sales could reach $800 mil­lion-plus, with bull­ish an­a­lysts pre­dict­ing clos­er to $2 bil­lion.

Of these in­di­ca­tions GW is tak­ing on, LGS is no­to­ri­ous­ly dif­fi­cult to treat. LGS pa­tients of­ten de­vel­op a re­sis­tance to the treat­ments they’re on, which leads to pa­tients tak­ing sev­er­al drugs at once.

“For this pa­tient pop­u­la­tion, un­for­tu­nate­ly pa­tients have tried and failed mul­ti­ple drugs — three drugs on av­er­age. The prob­a­bil­i­ty of see­ing im­prove­ment from adding an­oth­er an­ti-epilep­tic drug of a sim­i­lar type is very low,” Gov­er said. “The im­por­tance of CBD in this con­text is that it rep­re­sents a very dif­fer­ent type of med­ica­tion, with a dif­fer­ent mech­a­nism of ac­tion and a dif­fer­ent side ef­fect pro­file.”

In LGS, Epid­i­olex trig­gered a me­di­an re­duc­tion of 44% in drop seizures (the kind of seizures that send pa­tients to the ground, of­ten caus­ing in­jury). The place­bo group saw a re­duc­tion of 22%, by com­par­i­son. The p-val­ue came in at 0.0135.

Mild to mod­er­ate ad­verse events showed up in 74 of the 86 pa­tients on the drug, the most com­mon be­ing di­ar­rhea, som­no­lence, pyrex­ia, de­creased ap­petite, and vom­it­ing. Twelve pa­tients dropped out of the study, and one died. The Lancet pa­per notes the pa­tient death “was con­sid­ered un­re­lat­ed to treat­ment.”

GW is test­ing Epid­i­olex in sev­er­al epilep­tic in­di­ca­tions, in­clud­ing Dravet syn­drome and tuber­ous scle­ro­sis com­plex.

Epid­i­olex has al­so shown equal­ly no­table ef­fect against Dravet syn­drome, in which it trig­gered a 39% mean re­duc­tion in con­vul­sive seizures com­pared to 13% in the place­bo group in late-stage tri­als.

In­vestors ap­pear to be pleased with the da­ta be­hind the po­ten­tial drug fran­chise so far, as GW man­aged to raise $300 mil­lion in a pub­lic of­fer­ing last month. Gov­er said the com­pa­ny is well cap­i­tal­ized, with a cash bal­ance of just over $600 mil­lion. That mon­ey will go straight in­to the com­mer­cial launch of Epid­i­olex, FDA ap­proval will­ing.

Ap­pli­ca­tions have been sub­mit­ted to both the FDA and the EMA for Epid­i­olex in LGS and Dravet syn­drome. The PDU­FA date in the US is June 27, while Gov­er ex­pects to hear back on the Eu­ro­pean ap­proval in 2019.

Hal Barron, GSK

Break­ing the death spi­ral: Hal Bar­ron talks about trans­form­ing the mori­bund R&D cul­ture at GSK in a crit­i­cal year for the late-stage pipeline

Just ahead of GlaxoSmithKline’s Q2 update on Wednesday, science chief Hal Barron is making the rounds to talk up the pharma giant’s late-stage strategy as the top execs continue to woo back a deeply skeptical investor group while pushing through a whole new R&D culture.

And that’s not easy, Barron is quick to note. He told the Financial Times:

I think that culture, to some extent, is as hard, in fact even harder, than doing the science.

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Aca­dia is mak­ing the best of it, but their lat­est PhI­II Nu­plazid study is a bust

Acadia’s late-stage program to widen the commercial prospects for Nuplazid has hit a wall. The biotech reported that their Phase III ENHANCE trial flat failed. And while they $ACAD did their best to cherry pick positive data wherever they can be found, this is a clear setback for the biotech.

With close to 400 patients enrolled, researchers said the drug flunked the primary endpoint as an adjunctive therapy for patients with an inadequate response to antipsychotic therapy. The p-value was an ugly 0.0940 on the Positive and Negative Syndrome Scale, which the company called out as a positive trend.

Their shares slid 12% on the news, good for a $426 million hit on a $3.7 billion market cap at close.

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Some Big Phar­mas stepped up their game on da­ta trans­paren­cy — but which flunked the test?

The nonprofit Bioethics International has come out with their latest scorecard on data transparency among the big biopharmas in the industry — flagging a few standouts while spotlighting some laggards who are continuing to underperform.

Now in its third year, the nonprofit created a new set of standards with Yale School of Medicine and Stanford Law School to evaluate the track record on trial registration, results reporting, publication and data-sharing practice.

Busy Gilead crew throws strug­gling biotech a life­line, with some cash up­front and hun­dreds of mil­lions in biobucks for HIV deal

Durect $DRRX got a badly needed shot in the arm Monday morning as Gilead’s busy BD team lined up access to its extended-release platform tech for HIV and hepatitis B.

Gilead, a leader in the HIV sector, is paying a modest $25 million in cash for the right to jump on the platform at Durect, which has been using its technology to come up with an extended-release version of bupivacaine. The FDA rejected that in 2014, but Durect has been working on a comeback.

In­tec blitzed by PhI­II flop as lead pro­gram fails to beat Mer­ck­'s stan­dard com­bo for Parkin­son’s

Intec Pharma’s $NTEC lead drug slammed into a brick wall Monday morning. The small-cap Israeli biotech reported that its lead program — coming off a platform designed to produce a safer, more effective oral drug for Parkinson’s — failed the Phase III at the primary endpoint.

Researchers at Intec, which has already seen its share price collapse over the past few months, says that its Accordion Pill-Carbidopa/Levodopa failed to prove superior to Sinemet in reducing daily ‘off’ time. 

Cel­gene racks up third Ote­zla ap­proval, heat­ing up talks about who Bris­tol-My­ers will sell to

Whoever is taking Otezla off Bristol-Myers Squibb’s hands will have one more revenue stream to boast.

The drug — a rising star in Celgene’s pipeline that generated global sales of $1.6 billion last year — is now OK’d to treat oral ulcers associated with Behçet’s disease, a common symptom for a rare inflammatory disorder. This marks the third FDA approval for the PDE4 inhibitor since 2014, when it was greenlighted for plaque psoriasis and psoriatic arthritis.

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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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Vlad Coric (Biohaven)

In an­oth­er dis­ap­point­ment for in­vestors, FDA slaps down Bio­haven’s re­vised ver­sion of an old ALS drug

Biohaven is at risk of making a habit of disappointing its investors.

Late Friday the biotech $BHVN reported that the FDA had rejected its application for riluzole, an old drug that they had made over into a sublingual formulation that dissolves under the tongue. According to Biohaven, the FDA had a problem with the active ingredient used in a bioequivalence study back in 2017, which they got from the Canadian drugmaker Apotex.

Apotex, though, has been a disaster ground. The manufacturer voluntarily yanked the ANDAs on 31 drugs — in late 2017 — after the FDA came across serious manufacturing deficiencies at their plants in India. A few days ago, the FDA made it official.

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