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.

A fa­vorite in Alex­ion’s C-suite is leav­ing, and some mighty sur­prised an­a­lysts aren’t the least bit hap­py about it

Analysts hate to lose a biotech CFO they’ve come to trust and admire — especially if they’re being blindsided by a surprise exit.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 59,900+ biopharma pros reading Endpoints daily — and it's free.

David Grainger [file photo]

'Dis­con­nect the bas­tard­s' — one biotech's plan to break can­cer cell­s' uni­fied de­fens­es

Chemotherapy and radiotherapy are the current gladiators of cancer treatment, but they come with well-known limitations and side-effects. The emergence of immunotherapy — a ferocious new titan in oncologist’s toolbox — takes the brakes off the immune system to kill cancer cells with remarkable success in some cases, but the approach is not always effective. What makes certain forms of cancer so resilient? Scientists may have finally pieced together a tantalizing piece of the puzzle, and a new biotech is banking on a new approach to fill the gap.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 59,900+ biopharma pros reading Endpoints daily — and it's free.

While No­var­tis ban­ish­es Zol­gens­ma scan­dal scars — Bio­gen goes on a Spin­raza 'of­fen­sive'

While Novartis painstakingly works to mop up the stench of the data manipulation scandal associated with its expensive gene therapy for spinal muscular atrophy (SMA) Zolgensma— rival Biogen is attempting to expand the use of its SMA therapy, Spinraza. 

The US drugmaker $BIIB secured US approval for Spinraza for use in the often fatal genetic disease in 2016. The approval covered a broad range of patients with infantile-onset (most likely to develop Type 1) SMA. 

Jason Kelly. Mike Blake/Reuters via Adobe

Eye­ing big ther­a­peu­tic push, Gink­go bags $290M to build a cell pro­gram­ming em­pire

Ginkgo Bioworks is on a roll. Days after publicizing a plan to nurture new startups via partnerships with accelerators Y Combinator and Petri, the Boston biotech says it has raised another $290 million for its cell programming platform to reach further and wider.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 59,900+ biopharma pros reading Endpoints daily — and it's free.

UP­DAT­ED: Speak­er Nan­cy Pelosi to un­veil bill for fed­er­al­ly ne­go­ti­at­ed drug prices

After months of buzz from both sides of the aisle, Speaker Nancy Pelosi will today introduce her plan to allow the federal government to negotiate prices for 250 prescription drugs, setting up a showdown with a pharmaceutical industry working overtime to prevent it.

The need to limit drug prices is a rare point of agreement between President Trump and Democrats, although the president has yet to comment on the proposal and will likely face pressure to back a more conservative option or no bill at all. Republican Senator Chuck Grassley is reportedly lobbying his fellow party members on a more modest proposal he negotiated with Democratic Senator Ron Wyden in July.

Jeff Kindler's Cen­trex­ion re­news bid to make pub­lic de­but

Jeffrey Kindler’s plan to take his biotech — which is developing a slate of non-opioid painkillers — public, is back on.

The Boston based company, led by former Pfizer $PFE chief Kindler, originally contemplated a $70 million to $80 million IPO last year— but eventually postponed that strategy. On Wednesday, the company revived its bid to make a public debut in a filing with the SEC — although no pricing details were disclosed.

It’s fi­nal­ly over: Bio­gen, Ei­sai scrap big Alzheimer’s PhI­I­Is af­ter a pre­dictable BACE cat­a­stro­phe rais­es safe­ty fears

Months after analysts and investors called on Biogen and Eisai to scrap their BACE drug for Alzheimer’s and move on in the wake of a string of late-stage failures and rising safety fears, the partners have called it quits. And they said they were dropping the drug — elenbecestat — after the independent monitoring board raised concerns about…safety.

We don’t know exactly what researchers found in this latest catastrophe, but the companies noted in their release that investigators had determined that the drug was flunking the risk/benefit analysis.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 59,900+ biopharma pros reading Endpoints daily — and it's free.

Zachary Hornby. Boundless

'A fourth rev­o­lu­tion in can­cer ther­a­pies': ARCH-backed Bound­less Bio flash­es big check, makes big­ger promis­es in de­but

It was the cellular equivalent of opening your car door and finding an active, roaring engine in the driver seat.

Scientists learned strands of DNA could occasionally appear outside of its traditional home in the nucleus in the 1970s, when they appeared as little, innocuous circles on microscopes; inexplicable but apparently innate. But not until UC San Diego’s Paul Mischel published his first study in Science in 2014 did researchers realize these circles were not only active but potentially overactive and driving some cancer tumors’ superhuman growth.

Scott Gottlieb, AP Images

Scott Got­tlieb is once again join­ing a team that en­joyed good times at the FDA un­der his high-en­er­gy stint at the helm

Right after jumping on Michael Milken’s FasterCures board on Monday, the newly departed FDA commissioner is back today with news about another life sciences board post that gives him a ringside chair to cheer on a lead player in the real-world evidence movement — one with very close ties to the FDA.

Aetion is reporting this morning that Gottlieb is joining their board, a group that includes Mohamad Makhzoumi, a general partner at New Enterprise Associates, where Gottlieb returned after stepping out of his role at the FDA 2 years after he started.

Gottlieb — one of the best connected execs in biopharma — knows this company well. As head of FDA he championed the use of real-world evidence to help guide drug developers and the agency in gaining greater efficiencies, which helped set up Aetion as a high-profile player in the game.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 59,900+ biopharma pros reading Endpoints daily — and it's free.