Neu­ro­crine bags an FDA OK for tar­dive dysk­i­ne­sia, trump­ing Te­va as it preps first com­mer­cial launch

Kevin Gor­man, CEO of Neu­ro­crine Bio­sciences, presents at a Jef­feries in­vestor con­fer­ence in 2013. Bloomberg/via Get­ty Im­ages


The FDA has fol­lowed through with an ap­proval for Neu­ro­crine Bio­science’s In­grez­za (val­be­nazine) for tar­dive dysk­i­ne­sia, set­ting up a po­ten­tial show­down just a week af­ter Te­va scored with their ri­val ther­a­py, now ap­proved for Hunt­ing­ton’s chorea.

Un­like Te­va, Neu­ro­crine’s la­bel has come through with­out a black box warn­ing on de­pres­sion and sui­ci­dal ideation, giv­ing it a dis­tinct edge in any mar­ket­ing con­fronta­tion to come as Te­va waits for the FDA to de­cide on its own ap­pli­ca­tion to hit the TD mar­ket. Neu­ro­crine shares jumped 28% on the news, though some an­a­lysts had fret­ted about the la­bel.

The ap­proval marks a sea change for San Diego-based Neu­ro­crine $NBIX, which has been build­ing up a sales force for its first com­mer­cial launch. An­a­lysts next want to see late-stage Tourette syn­drome da­ta on In­grez­za and NDAs for elagolix — part­nered with Ab­b­Vie — in en­dometrio­sis and uter­ine fi­broids as the biotech jour­neys through a trans­for­ma­tive year.

Bar­clays, for one, has of­fered Neu­ro­crine’s drug its stamp of block­buster-wor­thi­ness, es­ti­mat­ing peak sales at more than $1 bil­lion for tar­dive dysk­i­ne­sia alone. Eval­u­atePhar­ma has al­so tracked a block­buster fu­ture for this drug as well, de­signed to treat in­vol­un­tary move­ments spurred by the pro­longed use of an­tipsy­chotics. Hunt­ing­ton’s chorea is a re­lat­ed con­di­tion in­volv­ing in­vol­un­tary twist­ing and writhing.

“This is a mo­men­tous oc­ca­sion for us,” said CEO Kevin Gor­man in a Tues­day evening call with an­a­lysts. Few com­pa­nies can say they dis­cov­ered a drug in their lab, de­vel­oped it and grabbed an FDA ap­proval, he added. This is the first drug ap­proved for TD.

What Gor­man and his team didn’t say, though, was how much the drug will cost. Like a grow­ing num­ber of com­pa­nies, Neu­ro­crine wants a clean shot at cel­e­brat­ing the ap­proval with­out hav­ing to ex­plain the cost — a sen­si­tive top­ic with man­u­fac­tur­ers and pay­ers. That will come with the launch, slat­ed for May 1.

The lat­est ap­proval came through af­ter Neu­ro­crine pub­lished the full set of da­ta in its fa­vor, in­clud­ing a note that its drug scored for 40% of the pa­tients in the drug arm who reg­is­tered at least a 50% im­prove­ment in the AIMS dysk­i­ne­sia score, com­pared to 8.7% of those on place­bo.

“Un­til now, one of the few op­tions for physi­cians, when man­ag­ing TD, was to stop, change or low­er the dose of an­tipsy­chot­ic med­ica­tion, po­ten­tial­ly jeop­ar­diz­ing pa­tients’ psy­chi­atric sta­bil­i­ty,” said Christoph U. Cor­rell, MD, Pro­fes­sor, Psy­chi­a­try and Mol­e­c­u­lar Med­i­cine, Hof­s­tra North­well School of Med­i­cine. “In clin­i­cal tri­als, IN­GREZ­ZA sig­nif­i­cant­ly and rapid­ly im­proved TD symp­toms com­pared to place­bo, re­duc­ing in­vol­un­tary move­ments acute­ly and through 48 weeks of treat­ment with­out com­pro­mis­ing un­der­ly­ing psy­chi­atric care. These re­sults, com­bined with con­ve­nient once-dai­ly dos­ing, rep­re­sent a tremen­dous break­through for pa­tients suf­fer­ing from TD.”

Last fall Te­va in­cit­ed a con­sid­er­able amount of spec­u­la­tion about how it could one day ri­val Neu­ro­crine in tar­dive dysk­i­ne­sia with Auste­do (SD-809, deutetra­benazine). But their mixed re­sults al­so sparked more than a lit­tle kick­back from an­a­lysts who pre­fer In­grez­za. The FDA hand­ed Te­va’s drug — ac­quired 2 years ago in a $3.5 bil­lion Aus­pex buy­out — a pri­or­i­ty re­view for TD in Feb­ru­ary and as­sign­ing them an Au­gust 30 PDU­FA date.

In­ves­ti­ga­tors for Te­va say that a high and mid-range dose of their drug hit the pri­ma­ry end­point on the move­ment scale rat­ing for a “mod­i­fied in­tent to treat” group in their sec­ond late-stage study. The low dose failed to sep­a­rate sig­nif­i­cant­ly from the place­bo. But the high dose, mean­while, missed beat­ing the place­bo based on in­ves­ti­ga­tors’ as­sess­ment of how their pa­tients were do­ing.

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.

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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Norbert Bischofberger. Kronos

Backed by some of the biggest names in biotech, Nor­bert Bischof­berg­er gets his megaround for plat­form tech out of MIT

A little over a year ago when I reported on Norbert Bischofberger’s jump from the CSO job at giant Gilead to a tiny upstart called Kronos, I noted that with his connections in biotech finance, that $18 million launch round he was starting off with could just as easily have been $100 million or more.

With his first anniversary now behind him, Bischofberger has that mega-round in the bank.

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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.

Seer adds ex-FDA chief Mark Mc­Clel­lan to the board; Her­cules Cap­i­tal makes it of­fi­cial for new CEO Scott Bluestein

→ On the same day it announced a $17.5 million Series C, life sciences and health data company Seer unveiled that it had lured former FDA commissioner and ex-CMS administrator Mark McClellan on to its board. “Mark’s deep understanding of the health care ecosystem and visionary insights on policy reform will be crucial in informing our thinking as we work to bring our liquid biopsy and life sciences products to market,” said Seer chief and founder Omid Farokhzad in a statement.

Daniel O'Day

No­var­tis hands off 3 pre­clin­i­cal pro­grams to the an­tivi­ral R&D mas­ters at Gilead

Gilead CEO Daniel O’Day’s new task hunting up a CSO for the company isn’t stopping the industry’s dominant antiviral player from doing pipeline deals.

The big biotech today snapped up 3 preclinical antiviral programs from pharma giant Novartis, with drugs promising to treat human rhinovirus, influenza and herpes viruses. We don’t know what the upfront is, but the back end has $291 million in milestones baked in.

Vas Narasimhan, AP Images

On a hot streak, No­var­tis ex­ecs run the odds on their two most im­por­tant PhI­II read­outs. Which is 0.01% more like­ly to suc­ceed?

Novartis CEO Vas Narasimhan is living in the sweet spot right now.

The numbers are running a bit better than expected, the pipeline — which he assembled as development chief — is performing and the stock popped more than 4% on Thursday as the executive team ran through their assessment of Q2 performance.

Year-to-date the stock is up 28%, so the investors will be beaming. Anyone looking for chinks in their armor — and there are plenty giving it a shot — right now focus on payer acceptance of their $2.1 million gene therapy Zolgensma, where it’s early days. And CAR-T continues to underperform, but Novartis doesn’t appear to be suffering from it.

So what could go wrong?

Actually, not much. But Tim Anderson at Wolfe pressed Narasimhan and his development chief John Tsai to pick which of two looming Phase III readouts with blockbuster implication had the better odds of success.

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H1 analy­sis: The high-stakes ta­ble in the biotech deals casi­no is pay­ing out some record-set­ting win­nings

For years the big trend among dealmakers at the major players has been centered on ratcheting down upfront payments in favor of bigger milestones. Better known as biobucks for some. But with the top 15 companies competing for the kind of “transformative” pacts that can whip up some excitement on Wall Street, with some big biotechs like Regeneron now weighing in as well, cash is king at the high stakes table.

We asked Chris Dokomajilar, the head of DealForma, to crunch the numbers for us, looking over the top 20 deals for the past decade and breaking it all down into the top alliances already created in 2019. Gilead has clearly tipped the scales in terms of the coin of the bio-realm, with its record-setting $5 billion upfront to tie up to Galapagos’ entire pipeline.

Dokomajilar notes:

We’re going to need a ‘three comma club’ for the deals with over $1 billion in total upfront cash and equity. The $100 million-plus club is getting crowded at 164 deals in the last decade with new deals being added towards the top of the chart. 2019 already has 14 deals with at least $100 million in upfront cash and equity for a total year-to-date of over $9 billion. That beats last year’s $8 billion and sets a record.

Add upfronts and equity payments and you get $11.5 billion for the year, just shy of last year’s record-setting $11.8 billion.

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