3 for 3 in PhI­II: GW Phar­ma rac­ing to the FDA af­ter bag­ging an­oth­er late-stage epilep­sy suc­cess

Ex­tend­ing its win­ning streak, GW Phar­ma­ceu­ti­cals says the sec­ond Phase III for Epid­i­olex in treat­ing Lennox-Gas­taut syn­drome — a rare form of epilep­sy — has al­so come through with solid­ly pos­i­tive ef­fi­ca­cy da­ta. And the UK biotech is now plan­ning to hus­tle ahead with a new drug ap­pli­ca­tion at the FDA in the com­ing months.

Or­rin Devin­sky, New York Uni­ver­si­ty Lan­gone Med­ical Cen­ter

The da­ta from this sec­ond study on the com­pa­ny’s lead cannabi­noid ther­a­py were even bet­ter than the first, with a me­di­an 42% drop in month­ly seizures, com­pared to 17% in the place­bo group for a 20 mg dose. The 10 mg dose was al­so a suc­cess, with a 37% drop in the seizure rate.

Shares of GW Phar­ma $GW­PH shot up 15% on the lat­est suc­cess, which may well leave the com­pa­ny poised for a mar­ket launch.

Back in March the com­pa­ny ex­cit­ed in­vestors with re­sults for the first of four late-stage stud­ies on Epid­i­olex, with the drug re­duc­ing con­vul­sions in chil­dren with treat­ment-re­sis­tant Dravet syn­drome by a me­di­an av­er­age of 39% com­pared to on­ly 13% in the place­bo arm. That da­ta helped in­spire block­buster-sized peak sales es­ti­mates for this drug.

GW Phar­ma had a pre-NDA meet­ing with the FDA last sum­mer, af­ter it racked up the pos­i­tive Phase III study for Dravet syn­drome. Now it plans to hus­tle along an NDA for both Dravet and LGS at the same time, rather than wait for an­oth­er round of late-stage da­ta on Dravet syn­drome

Stat­ed Or­rin Devin­sky, M.D., of New York Uni­ver­si­ty Lan­gone Med­ical Cen­ter’s Com­pre­hen­sive Epilep­sy Cen­ter and prin­ci­pal in­ves­ti­ga­tor in the tri­al:

“The pos­i­tive out­come in this sec­ond tri­al of Epid­i­olex in pa­tients with Lennox-Gas­taut syn­drome demon­strates the ef­fec­tive­ness of this prod­uct in this par­tic­u­lar­ly dif­fi­cult to treat, child­hood-on­set epilep­sy. The da­ta from the Epid­i­olex Dravet and LGS stud­ies of­fers the prospect of an FDA-ap­proved CBD med­i­cine that shows both clin­i­cal­ly mean­ing­ful seizure re­duc­tion and a con­sis­tent safe­ty and tol­er­a­bil­i­ty pro­file. I be­lieve Epid­i­olex has the po­ten­tial to be­come an im­por­tant new op­tion with­in the field of treat­ment-re­sis­tant epilep­sy.”

One pa­tient on 10mg/kg Epid­i­olex dis­con­tin­ued treat­ment due to an ad­verse event, in­ves­ti­ga­tors said, com­pared with six pa­tients on 20mg/kg and one pa­tient on place­bo.

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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