Bio­gen shares tum­ble af­ter key ex­ec notes a change­up to piv­otal ad­u­canum­ab study due to 'vari­abil­i­ty'

With news of Mer­ck’s flag­ship BACE drug go­ing down to com­plete fail­ure in a late-stage Alzheimer’s study still re­ver­ber­at­ing through a dispir­it­ed R&D field, Bio­gen is now pitch­ing in to make things even worse.

Al San­drock, Bio­gen

Shares of Bio­gen $BI­IB tum­bled about 7% Wednes­day af­ter­noon af­ter their CMO Al San­drock shocked some at­ten­dees at the Leerink Health­care Con­fer­ence by say­ing that the com­pa­ny had de­cid­ed to add 510 pa­tients to its piv­otal study for the close­ly-watched drug ad­u­canum­ab.

San­drock told at­ten­dees that they need­ed to add hun­dreds of pa­tients to the study due to some “vari­abil­i­ty” they were see­ing in the da­ta from the first round of pa­tients to reach the pri­ma­ry end­point.

Bio­gen couldn’t have picked a worse time for the dis­clo­sure.

The drum­beat of neg­a­tive re­sults for every late-stage ef­fort aimed at amy­loid be­ta has be­gun to shake a once-sol­id be­lief that the clus­ters of tox­ic pro­tein found in many — though not all — pa­tients had to be the cul­prit for a dis­ease that af­flicts mil­lions of peo­ple the world over.

Ge­of­frey Porges, who fol­lows the drug close­ly, not­ed:

The ex­pan­sion in en­roll­ment de­ci­sion was made by the com­pa­ny af­ter they an­a­lyzed blind­ed re­sults for the per­for­mance of the pri­ma­ry end­point (18 month CDR-SB) in the pa­tients who have reached the pri­ma­ry end­point so far. These ad­di­tion­al pa­tients are re­quired to main­tain 90% pow­er for the tri­als due to “vari­abil­i­ty”. Bio­gen has not seen the da­ta by arm, but to main­tain the pow­er­ing need­ed to add the ad­di­tion­al en­rollees giv­en the vari­ance seen so far. Bio­gen in­di­cat­ed that be­cause of the ac­cel­er­at­ed ear­ly en­roll­ment in the tri­al they could still main­tain ap­prox­i­mate­ly the same tim­ing for full en­roll­ment, and sug­gest­ed that they ex­pect full en­roll­ment “in the sum­mer” and ex­pect fi­nal re­sults now in the ear­ly part of 2020. The com­pa­ny al­so com­ment­ed that the drop out rate had been low­er than ex­pect­ed, and that the rate of se­ri­ous ad­verse events, and ARIA-E in par­tic­u­lar, had been with­in their ex­pec­ta­tions. In the con­text of Mer­ck’s (MP) re­cent dis­ap­point­ment with the Mer­ck BACE in­hibitor verube­ce­s­tat, Bio­gen’s ad­u­canum­ab is one of what is now on­ly a hand­ful of Alzheimer’s med­i­cines still ac­tive in piv­otal tri­als.

The pos­i­tive da­ta that Bio­gen had dis­closed from small­er stud­ies had gone a long way to rais­ing hopes that Bio­gen could ac­tu­al­ly make a break­through here, even as se­ri­ous safe­ty is­sues arose. Any­thing that rais­es doubts about ef­fi­ca­cy can’t help Bio­gen, even as it in­sists that the com­pa­ny re­mains con­fi­dent in the amy­loid be­ta the­o­ry.

Michael Yee at Jef­feries not­ed this af­ter­noon that the study da­ta re­mains blind­ed and is even less like­ly to read out sig­nif­i­cant da­ta points be­fore it’s com­plet­ed way out in 2020. He sees to­day’s event as a wake up call for Bio­gen to get busy and buy some­thing the mar­ket won’t fret about.

Mar­ket will swing back to un­cer­tain­ty on the Alzheimer’s pen­du­lum based on tri­al changes, and giv­en da­ta is not like­ly un­til 2020 (in our view) and an in­ter­im seems un­like­ly giv­en dis­clo­sures about vari­abil­i­ty, while we main­tain a pos­i­tive stance on da­ta in 2020, we think co needs to go out and buy de-risked neu­ro/or­phan com­pa­nies to “change the nar­ra­tive” to be­ing a bi­na­ry Alzheimer’s com­pa­ny to one with prod­ucts even if Alzheimer’s doesn’t work.

Good luck with that.

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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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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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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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.