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.

BiTE® Plat­form and the Evo­lu­tion To­ward Off-The-Shelf Im­muno-On­col­o­gy Ap­proach­es

Despite rapid advances in the field of immuno-oncology that have transformed the cancer treatment landscape, many cancer patients are still left behind.1,2 Not every person has access to innovative therapies designed specifically to treat his or her disease. Many currently available immuno-oncology-based approaches and chemotherapies have brought long-term benefits to some patients — but many patients still need other therapeutic options.3

President Donald Trump (left) and Moncef Slaoui, head of Operation Warp Speed (Alex Brandon, AP Images)

UP­DAT­ED: White House names fi­nal­ists for Op­er­a­tion Warp Speed — with 5 ex­pect­ed names and one no­table omis­sion

A month after word first broke of the Trump Administration’s plan to rapidly accelerate the development and production of a Covid-19 vaccine, the White House has selected the five vaccine candidates they consider most likely to succeed, The New York Times reported.

Most of the names in the plan, known as Operation Warp Speed, will come as little surprise to those who have watched the last four months of vaccine developments: Moderna, which was the first vaccine to reach humans and is now the furthest along of any US effort; J&J, which has not gone into trials but received around $500 million in funding from BARDA earlier this year; the joint AstraZeneca-Oxford venture which was granted $1.2 billion from BARDA two weeks ago; Pfizer, which has been working with the mRNA biotech BioNTech; and Merck, which just entered the race and expects to put their two vaccine candidates into humans later this year.

Is a pow­er­house Mer­ck team prepar­ing to leap past Roche — and leave Gilead and Bris­tol My­ers be­hind — in the race to TIG­IT dom­i­na­tion?

Roche caused quite a stir at ASCO with its first look at some positive — but not so impressive — data for their combination of Tecentriq with their anti-TIGIT drug tiragolumab. But some analysts believe that Merck is positioned to make a bid — soon — for the lead in the race to a second-wave combo immuno-oncology approach with its own ambitious early-stage program tied to a dominant Keytruda.

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GSK presents case to ex­pand use of its lu­pus drug in pa­tients with kid­ney dis­ease, but the field is evolv­ing. How long will the mo­nop­oly last?

In 2011, GlaxoSmithKline’s Benlysta became the first biologic to win approval for lupus patients. Nine years on, the British drugmaker has unveiled detailed positive results from a study testing the drug in lupus patients with associated kidney disease — a post-marketing requirement from the initial FDA approval.

Lupus is a drug developer’s nightmare. In the last six decades, there has been just one FDA approval (Benlysta), with the field resembling a graveyard in recent years with a string of failures including UCB and Biogen’s late-stage flop, as well as defeats in Xencor and Sanofi’s programs. One of the main reasons the success has eluded researchers is because lupus, akin to cancer, is not just one disease — it really is a disease of many diseases, noted Al Roy, executive director of Lupus Clinical Investigators Network, an initiative of New York-based Lupus Research Alliance that claims it is the world’s leading private funder of lupus research, in an interview.

UP­DAT­ED: Es­ti­mat­ing a US price tag of $5K per course, remde­sivir is set to make bil­lions for Gilead, says key an­a­lyst

Data on remdesivir — the first drug shown to benefit Covid-19 patients in a randomized, controlled trial setting — may be murky, but its maker Gilead could reap billions from the sales of the failed Ebola therapy, according to an estimate by a prominent Wall Street analyst. However, the forecast, which is based on a $5,000-per-course US price tag, triggered the ire of one top drug price expert.

Leen Kawas, Athira CEO (Athira)

Can a small biotech suc­cess­ful­ly tack­le an Ever­est climb like Alzheimer’s? Athi­ra has $85M and some in­flu­en­tial back­ers ready to give it a shot

There haven’t been a lot of big venture rounds for biotech companies looking to run a Phase II study in Alzheimer’s.

The field has been a disaster over the past decade. Amyloid didn’t pan out as a target — going down in a litany of Phase III failures — and is now making its last stand at Biogen. Tau is a comer, but when you look around and all you see is destruction, the idea of backing a startup trying to find complex cocktails to swing the course of this devilishly complicated memory-wasting disease would daunt the pluckiest investors.

Covid-19 roundup: Mod­er­na read­ies to en­ter PhI­II in Ju­ly, As­traZeneca not far be­hind; EU ready to ne­go­ti­ate vac­cine ac­cess with $2.7B fund

Moderna may soon add another first to the Covid-19 vaccine race.

In March, the mRNA biotech was the first company to put a Covid-19 vaccine into humans. Next month, they may become the first company to put their vaccine into the large, late-stage trials that are needed to prove whether the vaccine is effective.

In an interview with JAMA editor Howard Bauchner, NIAID chief Anthony Fauci said that a 30,000-person, Phase III trial for Moderna’s vaccine could start in July. The news comes a week after Moderna began a Phase II study that will enroll several hundred people.

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José Basel­ga finds promise in new class of RNA-mod­i­fy­ing can­cer tar­gets, lock­ing in 3 pre­clin­i­cal pro­grams with $55M

Having dived early into some of the RNA breakthroughs of the last decades — betting on Moderna’s mRNA tech and teaming up with Silence on the siRNA front — AstraZeneca is jumping into a new arena: going after proteins that modify RNA.

Their partner of choice is Accent Therapeutics, which is receiving $55 million in upfront payment to steer a selected preclinical program through to the end of Phase I. After AstraZeneca takes over, the Lexington, MA-based startup has the option to co-develop and co-commercialize in the US — and collect up to $1.1 billion in milestones in the long run. The deal also covers two other potential drug candidates.

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Pfiz­er’s Doug Gior­dano has $500M — and some ad­vice — to of­fer a cer­tain breed of 'break­through' biotech

So let’s say you’re running a cutting-edge, clinical-stage biotech, probably public, but not necessarily so, which could see some big advantages teaming up with some marquee researchers, picking up say $50 million to $75 million dollars in a non-threatening minority equity investment that could take you to the next level.

Doug Giordano might have some thoughts on how that could work out.

The SVP of business development at the pharma giant has helped forge a new fund called the Pfizer Breakthrough Growth Initiative. And he has $500 million of Pfizer’s money to put behind 7 to 10 — or so — biotech stocks that fit that general description.

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