Thumbs Up/Thumbs Down: The new buzz in R&D, block­buster work on a bud­get and why what’s dead may nev­er die

End­points as­sess­es the big bio­phar­ma R&D sto­ries of the week, with a lit­tle added com­men­tary on what they mean for the in­dus­try.

Bio­elec­tron­ics gets a star turn

Let’s get it out front. I have a weak­ness for big think­ing about new R&D fields. So when I got wind of the tie-up be­tween Glax­o­SmithK­line and Ver­i­ly (Lord Google’s life sci­ence arm), I got up at 3 am and wrote the sto­ry. The idea that nan­otech based de­vices can be used to or­ches­trate a se­ries of elec­tric puls­es to treat a dis­ease can on­ly thrill a re­al R&D en­thu­si­ast. And at this stage, every­thing has great po­ten­tial. We’re a cou­ple of years out from proof of con­cept, so this is far from a com­mer­cial sto­ry. (Which, let’s face it, is what GSK re­al­ly needs now.) Sci­ence lovers will fol­low it as close­ly as they can. Count me in.

Bio­gen is do­ing a deal? Re­al­ly?

There’s been no doubt at all, for more than a year now, that Bio­gen needs to get se­ri­ous and do some big deals. But in­stead of pacts that can grab Wall Street’s at­ten­tion with some late-stage piz­zazz, Bio­gen has been deal­ing with a man­age­ment change­up. Doug Williams and Steve Holtz­man left last year, both find­ing new biotechs to run, and Bio­gen couldn’t get its act to­geth­er in time to save George Scan­gos’ perch at the top. The ear­ly-stage work it com­mit­ted to in gene ther­a­py is fas­ci­nat­ing, but it lacks the kind of near term cat­a­lysts that Bio­gen hungers for. Alzheimer’s is a huge, ul­tra-risky op­por­tu­ni­ty. Man­age­ment told us again this week that great deals are com­ing. We’re still wait­ing. And that’s not a good thing.

Re­gen­eron’s stel­lar Q2 up­date

This is how you set up a late-stage pipeline, on a bud­get. At $1.45 bil­lion, Re­gen­eron’s 2016 re­search bud­get won’t make the top 20. But its late-stage pipeline has sev­er­al po­ten­tial block­busters in it. I just saw a new an­a­lyst take on the com­pa­ny, not­ing that the ap­proval of drugs like dupilum­ab is baked in now. That’s an in­di­ca­tion that Re­gen­eron not on­ly knows a sol­id med­ical ad­vance, it plans and ex­e­cutes on clin­i­cal tri­als like few com­pa­nies can. Sanofi al­so gets cred­it for par­tic­i­pat­ing in its work, where it has seen far more suc­cess than its in-house ef­fort has pro­duced. Fail­ures are so fre­quent in bio­phar­ma R&D, it’s great to ap­plaud Re­gen­eron. I’m look­ing for­ward to this com­pa­ny’s third act in the clin­ic.

  Shire fol­lows Take­da in re­or­ga­niz­ing R&D.

Last week, Take­da made it clear that it is cut­ting staffers in places like Cam­bridge, UK so it can con­cen­trate its re­search forces in the Cam­bridge/Boston and Japan. This week Shire out­lined the first cut in its three-year plan: to re­duce R&D costs $210 mil­lion af­ter ac­quir­ing Bax­al­ta. Bax­al­ta, of course, was just get­ting start­ed with its so­lo act when Shire stepped in. These are all re­al-world re­sults when you are A) grow­ing through ac­qui­si­tions and B) chang­ing the cul­ture. They are painful and of­ten nec­es­sary al­ter­ations in the land­scape. But you can’t help but feel for the peo­ple whose jobs are ly­ing in the way of per­pet­u­al change. R&D has been a vol­canic field for the past decade. Job se­cu­ri­ty is a thing of the past. This has like­ly helped pro­duc­tiv­i­ty, at the cost of play­ing hell with peo­ple’s lives.

Resver­a­trol: Show me the da­ta.

Some re­search sto­ries won’t die. But truth­ful­ly, why bring back resver­a­trol from the dead at this point? The Wall Street Jour­nal made the un­for­tu­nate de­ci­sion to do that, based on some mighty thin pick­ings from a few on­go­ing re­search projects in odd pock­ets of the world. You’ll re­call GSK’s en­thu­si­asms men­tioned at the top? They got a look at resver­a­trol’s an­i­mal da­ta at Sir­tris, which turned out to be more than a lit­tle con­tro­ver­sial, and paid $720 mil­lion for it. Sir­tris nev­er got very far and GSK wound it up and shipped the re­main­ing projects south to Philly. One day, some­body may ac­tu­al­ly land re­al ev­i­dence that they found a way to turn this mir­a­cle drug in­to a re­al­i­ty. Un­til then, let’s leave it as a side show.

Deci­bel hears the call of a new biotech neigh­bor­hood.

Biotech, of course, can be pur­sued just about any­where on the plan­et, but grav­i­tates to the most ex­pen­sive places in the world. One of those places is Kendall Square in Cam­bridge, where Big Phar­ma has been mov­ing in and gen­tri­fy­ing the neigh­bor­hood with big bucks for new re­search cen­ters. They have an un­der­stand­able de­ter­mi­na­tion to learn new things from the movers and shak­ers of the sci­ence world. But as Deci­bel’s Steve Holtz­man made clear, they’re pric­ing star­tups like his out of the mar­ket. So he’s jump­ing the Charles and mak­ing his new home in Fen­way. Boston/Cam­bridge will long re­main one of the most vi­tal re­search hubs in the world. But on­ly if you think out­side the bound­aries, and that in­cludes town lines.

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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