Aca­dia shares slide — again — af­ter FDA con­firms it has launched a safe­ty re­view of Nu­plazid

Aca­dia’s suf­fer­ing shares $ACAD took an­oth­er hit on Wednes­day, plung­ing 22% af­ter the FDA con­firmed to­day that reg­u­la­tors have be­gun a re­view of its an­ti-psy­chosis drug Nu­plazid — fol­low­ing CNN’s re­port ques­tion­ing the safe­ty of the drug.

A ’break­through’ drug ush­ered in­to the mar­ket to treat Parkin­son’s dis­ease psy­chosis, CNN re­cent­ly high­light­ed a non­prof­it’s warn­ing on 244 deaths in­volv­ing pa­tients on the drug in less than a year af­ter its launch. Aca­dia re­spond­ed by say­ing there’s no rea­son to be­lieve the drug was di­rect­ly re­spon­si­ble for any of the deaths CNN spot­light­ed in its sto­ry. And the FDA not­ed at the time that there is no rea­son to be­lieve there is any safe­ty is­sue that isn’t al­ready cov­ered by the la­bel. 

In a re­cent ex­change with law­mak­ers, though, FDA com­mis­sion­er Scott Got­tlieb — who took the helm well af­ter the drug was ap­proved — said he would “take an­oth­er look” at Nu­plazid, CNN re­port­ed.

A spokesper­son for the FDA con­firmed to me that the drug is un­der re­view, say­ing:

The FDA is con­duct­ing an eval­u­a­tion of avail­able in­for­ma­tion about Nu­plazid. This re­view has been go­ing on for sev­er­al weeks. We have noth­ing more to share at this time.

The at­ten­tion on safe­ty is­sues has pushed a steep drop in Aca­dia shares, which have lost more than half their val­ue over the last 6 months.

Aca­dia won over an ex­pert pan­el two years ago, de­spite a crit­i­cal re­view by FDA in­sid­ers that clear­ly red-flagged their safe­ty con­cerns, not­ing a dis­tinct in­crease in the num­ber of deaths as well as ad­verse events among the pa­tients tak­ing the drug com­pared to the con­trol arm of the study — even if there was no ob­vi­ous clue what was trig­ger­ing those events. The agency’s ap­proval came with a black box warn­ing on the risks, not un­com­mon at the FDA.

Steve Davis, Aca­dia CEO

Aca­dia had set out to prove that the drug could al­so be used for Alzheimer’s dis­ease psy­chosis, claim­ing a win in a clin­i­cal tri­al on the da­ta. The re­sults, though, quick­ly drew scruti­ny from an­a­lysts and one Alzheimer’s ex­pert, Lon Schnei­der, who not­ed in a sub­se­quent re­view of the da­ta that in­ves­ti­ga­tors had on­ly tracked a tem­po­rary wors­en­ing in the place­bo arm be­fore claim­ing suc­cess. The biotech lat­er switched strate­gies, though, chang­ing to a mix of dis­eases as­so­ci­at­ed with psy­chosis.

In­ves­ti­ga­tors last fall de­scribed their new ap­proach for the piv­otal study, re­cruit­ing a mix of pa­tients with Alzheimer’s dis­ease, de­men­tia with Lewy bod­ies, Parkin­son’s dis­ease de­men­tia, vas­cu­lar de­men­tia and fron­totem­po­ral de­men­tia.

The added scruti­ny of Nu­plazid comes as the FDA has been work­ing on ac­cel­er­at­ing the speed of drug de­vel­op­ment and agency re­views. Any is­sues with one of its ‘break­through’ drugs could con­ceiv­ably slow that process down.

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.