Ver­tex ex­ecs whip up a cheer from the an­a­lysts af­ter hedg­ing their bet by pick­ing their two top triples for PhI­II

Ver­tex mapped out its late-stage strat­e­gy for a new triple ther­a­py for cys­tic fi­bro­sis on Wednes­day evening, earn­ing con­sid­er­able ap­plause from an ap­pre­cia­tive group of an­a­lysts cov­er­ing the biotech.

Jeff Lei­den

The biotech’s $VRTX ex­ec­u­tive team has stoked block­buster hopes around these com­bi­na­tion stud­ies, key to their plan to broad­en the scope of pa­tients they can cov­er, and they did their best to keep ex­pec­ta­tions high as ri­vals look to carve in­to the mar­ket.

In the end, the team clear­ly likes VX-659 in com­bi­na­tion with teza­caftor and Ka­ly­de­co, not­ing that their Phase II da­ta demon­strat­ed that the high dose im­proved a mea­sure of lung func­tion by 13.3%.

That helped earn a 6% boost in their stock price in af­ter-mar­ket trad­ing.

But with the com­pa­ny’s fu­ture be­ing built on the out­comes of the Phase III stud­ies to come, they al­so want to pur­sue a Phase III tri­al that match­es an­oth­er ex­per­i­men­tal ther­a­py, VX-445, for the triple — though that tri­al will get start­ed af­ter the 659 study gets un­der­way.

Why two Phase II­Is?

“(W)e’re tak­ing two for­ward be­cause that’s one way of mod­i­fy­ing one risk and that’s the risk of some rare off tar­get tox­i­c­i­ty due to the next-gen cor­rec­tor in one of these reg­i­mens and ob­vi­ous­ly by tak­ing two for­ward, we mit­i­gate that risk,” CEO Jeff Lei­den told an­a­lysts dur­ing the quar­ter­ly call.

The 445 triple ac­tu­al­ly just bare­ly edged out 659 for the high-dose re­sponse, with a 13.8% im­prove­ment in FEV1. But the sec­ond high­est dose on­ly came in at a sin­gle-dig­it im­prove­ment rate, with a low­er rate of re­duc­tion for sweat chlo­ride as well as two trou­bling dis­con­tin­u­a­tions in one of the drug arms.

“(A)t the mar­gin, VX-659 looks to be the clean­er and more ef­fec­tive of the two mol­e­cules, al­though the pa­tient num­bers are still small (40-50 pa­tients per tri­al),” not­ed Leerink’s Ge­of­frey Porges Thurs­day morn­ing. “The com­pa­ny ap­pears to be fo­cus­ing on VX-659 as the pre­ferred sec­ond gen­er­a­tion cor­rec­tor com­bi­na­tion part­ner for fur­ther de­vel­op­ment, al­though VX-445 is al­so be­ing ad­vanced in­to piv­otal tri­als. The first triple com­bi­na­tion piv­otal tri­al (VX-659+ Tez/Iva) will be­gin in Q2, pend­ing fi­nal­iz­ing study de­sign with the FDA, and will ini­tial­ly fo­cus on het min pa­tients, al­though piv­otal tri­als in ho­mozy­gous delF508 pa­tients are like­ly to be on­ly a few months be­hind. Ver­tex ex­pects to start a sec­ond piv­otal tri­al pro­gram with VX-445 in mid year, most like­ly in­cor­po­rat­ing VX-561, (deuter­at­ed iva­caftor li­censed from Con­cert) and con­sist­ing of all med­i­cines dosed on a once dai­ly ba­sis.”

“The da­ta an­nounced to­day are re­mark­able and demon­strate the po­ten­tial for sig­nif­i­cant and con­sis­tent clin­i­cal ben­e­fits in pa­tients with one F508del mu­ta­tion and a min­i­mal func­tion mu­ta­tion when treat­ed with a triple com­bi­na­tion reg­i­men con­tain­ing ei­ther VX-659 or VX-445,” not­ed Lei­den.

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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