J&J ax­es its big BACE pro­gram in asymp­to­matic Alzheimer's pa­tients as the drum­beat of PhI­II fail­ures rolls on

Re­searchers at J&J $JNJ have pulled the plug on yet an­oth­er BACE pro­gram for Alzheimer’s.

Un­like Mer­ck, which scrapped their lead­ing BACE pro­gram for verube­ce­s­tat af­ter it clear­ly failed to help pa­tients on cog­ni­tion and func­tion in a ma­jor Phase III study, J&J was forced to halt the Phase IIb/III tri­al of atabece­s­tat (JNJ-54861911) af­ter some of the pa­tients in the drug arm be­gan to demon­strate se­ri­ous spikes in liv­er en­zymes.

Mer­ck’s study was a clas­sic piv­otal at­tempt to bend the curve of the ero­sion of cog­ni­tion and func­tion among pa­tients suf­fer­ing from what’s been de­fined as an ear­ly stage of the dis­ease, with symp­toms of an ail­ment that is wip­ing the mem­o­ries of mil­lions of pa­tients around the world. And it was a clas­sic fail­ure, join­ing a long and grow­ing list of late-stage prat­falls de­spite do­ing what the re­searchers ex­pect­ed it to — halt­ing the flow of amy­loid be­ta in­to the brain.

J&J’s study, though, was one of sev­er­al that want to see if the BACE ap­proach can work in pre­vent­ing the on­set of the dis­ease in asymp­to­matic pa­tients. They launched their main study — along­side a longterm safe­ty tri­al — back in the fall of 2015. In­ves­ti­ga­tors had treat­ed more than 600 peo­ple with an eye to com­plet­ing a marathon tri­al that would stretch out to 2024, even­tu­al­ly in­clud­ing 1650 sub­jects.

J&J had good rea­son to be leery of tox. Eli Lil­ly had to scrap their ini­tial BACE ef­fort in 2013 for the same rea­son, and Vi­tae Phar­ma and Boehringer In­gel­heim’s BI 1181181 was sus­pend­ed two years lat­er due to skin rash­es.

Eli Lil­ly lat­er went on to in-li­cense As­traZeneca’s BACE drug, pay­ing to jock­ey it in­to a Phase III tri­al for symp­to­matic pa­tients. But a host of an­a­lysts are shak­ing their heads over its prospects now that Mer­ck has of­fered some com­pelling ev­i­dence to show that strat­e­gy is a non-starter.

Alzheimer’s has test­ed the pa­tience and bud­gets of a large group of phar­ma gi­ants and biotechs. Pfiz­er ex­e­cut­ed one of the more re­cent ex­its, ax­ing a group of 300 in the process. And Ax­o­vant shares were shred­ded af­ter its un­sur­pris­ing col­lapse. Still, new play­ers like Cel­gene and De­nali have been ex­pand­ing their pipelines in a hunt for a drug that can help pa­tients. Any­one who’s suc­cess­ful, even mar­gin­al­ly, will have a big league block­buster on their hands. But the odds against it are daunt­ing.

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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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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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.