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.

Pi­o­neer­ing Click Chem­istry in Hu­mans

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Despite this significant progress in therapeutic targeting, why are we still seeing such a high mortality rate? The reason is that promising therapies are often limited by their therapeutic index, which is a measure of the effective dose of a drug, relative to its safety. If we could broaden the therapeutic indices of currently available medicines, it would revolutionize cancer treatments. We are still on the quest to find the ultimate cancer medicine – highly effective in several cancer types, safe, and precisely targeted to the tumor site.

Justin Klee (L) and Joshua Cohen, Amylyx co-CEOs (Cody O'Loughlin/The New York Times; courtesy Amylyx)

Ad­vo­cates, ex­perts cry foul over Amy­lyx's new ALS drug, cit­ing is­sues with price, PhI­II com­mit­ment

Not 24 hours after earning the first ALS drug approval in five years, Amylyx Pharmaceuticals’ Relyvrio is already drawing scrutiny. And it’s coming from multiple fronts.

In an investor call Friday morning, Amylyx revealed that it would charge about $158,000 per year, a price point that immediately drew backlash from ALS advocates and some outside observers. The cost reveal had been highly anticipated in the immediate hours after Thursday evening’s approval, though Amylyx only teased Relyvrio would cost less than previously approved drugs.

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Land­mark Amy­lyx OK spurs de­bate; Some... pos­i­tive? Alzheimer's da­ta; Can­cer tri­al bot­tle­neck; Sanofi's CRISPR bet; and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

After brief stops in Paris and Boston, John Carroll and the Endpoints crew are staying on the road in October with their return for a live/streaming EUBIO22 in London. The hybrid event fireside chats and panels on mRNA, oncology and the crazy public market. We hope you can join him there.

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Joshua Cohen (L) and Justin Klee, Amylyx co-CEOs

Up­dat­ed: Af­ter long and wind­ing road, FDA ap­proves Amy­lyx's ALS drug in vic­to­ry for pa­tients and ad­vo­ca­cy groups

For just the third time in its 116-year history, the FDA has approved a new treatment for Lou Gehrig’s disease, or ALS.

US regulators gave the thumbs-up to the drug, known as Relyvrio, in a massive win for patients and their families. The approval, given to Boston-area biotech Amylyx Pharmaceuticals, comes after two years of long and contentious debates over the drug’s effectiveness between advocacy groups and FDA scientists, following the readout of a mid-stage clinical trial in September 2020.

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#AAO22: J&J’s first look at com­mon eye dis­ease port­fo­lio pads the case for PhII of gene ther­a­py

CHICAGO — While the later-stage drug developers in the geographic atrophy field are near the finish line, Johnson & Johnson’s Janssen is taking a more deliberate route, with a treatment that it hopes to be a one-time fix.

The Big Pharma will take its Hemera Biosciences-acquired gene therapy into a Phase II study later this year in patients with GA, a common form of age-related macular degeneration that impacts about five million people worldwide. To get there, Janssen touted early-stage safety data at the American Academy of Ophthalmology annual conference Saturday morning, half a day after competitors Apellis and Iveric Bio revealed their own more-detailed Phase III analyses.

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Nooman Haque, head of life sciences and healthcare at Silicon Valley Bank, and John Carroll

I’m head­ed to Lon­don soon for #EU­BIO22. Care to join me?

It was great getting back to a live ESMO conference/webinar in Paris followed by a live pop-up event for the Endpoints 11 in Boston. We’re staying on the road in October with our return for a live/streaming EUBIO22 in London.

Silicon Valley Bank’s Nooman Haque and I are once again jumping back into the thick of it with a slate of virtual and live events on October 12. I’ll get the ball rolling with a virtual fireside chat with Novo Nordisk R&D chief Marcus Schindler, covering their pipeline plans and BD work.

Some­one old, some­one new: Mod­er­na pro­motes CTO, raids No­var­tis for re­place­ment amid pipeline push

Moderna CEO Stéphane Bancel made clear on the last quarterly call that “now is not the time to slow down.” On Thursday, he made a bit more room in the cockpit.

The company unveiled a new executive role on Thursday, promoting former chief technical operations and quality officer Juan Andres to president of strategic partnerships and enterprise expansion, and poaching a former Novartis exec to take his place.

Gilead names 'k­ing­pin­s' in coun­ter­feit HIV med law­suit

Gilead is mounting its counterfeit drug lawsuit, naming two “kingpins” and a complex network of conspirators who allegedly sold imitation bottles of its HIV meds, some of which ended up in US pharmacies.

The pharma giant on Wednesday provided an update on what it called a “large-scale, sophisticated counterfeiting conspiracy,” accusing two new defendants of “leading and orchestrating” a scheme to sell hundreds of millions of dollars in illegitimate drugs posing as meds such as Biktarvy and Descovy.

Tar­sus looks to raise aware­ness of eye­lid mite dis­ease in cam­paign aimed at eye­care spe­cial­ists

Eyelid mite disease may be “gross” but it’s also fairly common, affecting about 25 million people in the US.

Called demodex blepharitis, it’s a well-known condition among eyecare professionals, but they often don’t always realize how common it is. Tarsus Pharmaceuticals wants to change that with a new awareness campaign called “Look at the Lids.”

The campaign and website debut Thursday — just three weeks after Tarsus filed for FDA approval for a drug that treats the disease.

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