A spot­light schiz­o­phre­nia drug in Neu­ro­crine's $2B Take­da deal flunks its first ma­jor test. But it's not giv­ing up yet

When Take­da spun out a pipeline of ex­per­i­men­tal psy­chi­a­try drugs to Neu­ro­crine in a $2 bil­lion deal amid a post-merg­er shake­out, R&D chief Andy Plump de­scribed the ther­a­pies as “very in­ter­est­ing but still dif­fi­cult.”

On Tues­day, we got some idea of how dif­fi­cult.

Eiry Roberts

San Diego-based Neu­ro­crine re­vealed that one of the three spot­light clin­i­cal pro­grams they’d ac­quired failed the pri­ma­ry end­point in a Phase II tri­al for schiz­o­phre­nia, reg­is­ter­ing a neg­a­tive out­come on the change from base­line in the pos­i­tive and neg­a­tive syn­drome scale/neg­a­tive symp­tom fac­tor score (PANSS NS­FS).

CMO Eiry Roberts not­ed the IN­TER­ACT study, which en­rolled 256 pa­tients and test­ed three dos­ing lev­els of lu­vadax­i­s­tat in a place­bo-con­trolled set­ting, was “well-de­signed and ex­e­cut­ed” — leav­ing lit­tle room to ma­neu­ver in that end­point. The PANSS score is de­signed to as­sess sever­i­ty of schiz­o­phre­nia symp­toms.

From RBC an­a­lyst Bri­an Abra­hams:

Our sense is that the miss on the pri­ma­ry end­point, the PANSS neg­a­tive score, was like­ly due to a true lack of ef­fi­ca­cy, rather than any con­duct is­sues such as base­line im­bal­ances, glob­al site vari­abil­i­ty or high place­bo ef­fect, though the com­pa­ny has not yet com­plet­ed full analy­sis of PK char­ac­ter­i­za­tion (re­call there is a mod­est food ef­fect). The com­pa­ny is not re­port­ing whether there were any no­table trends or dose de­pen­dent sig­nals, sug­gest­ing there like­ly were none.

There is, though, still a sil­ver lin­ing. The drug — al­so known as TAK-831 — met the sec­ond end­points as­sess­ing cog­ni­tive per­for­mance, and the side ef­fects ap­peared con­sis­tent with pre­vi­ous stud­ies.

All of that backs fur­ther clin­i­cal work with the help of Take­da, Roberts added.

Neu­ro­crine had put down $120 mil­lion up­front to get its hands on a pack­age of three clin­i­cal-stage drugs, plus four more pre­clin­i­cal ther­a­pies. Take­da is then in line for $495 mil­lion in de­vel­op­ment mile­stones plus $1.4 bil­lion in com­mer­cial goal cash as well as roy­al­ties. Or it can trade in some of those mile­stones in fa­vor of a 50:50 prof­it share — should it choose to opt-in.

The deal marked one of the last steps of a ma­jor R&D re­org at Take­da, which was look­ing to shed some weight and earn some cash af­ter pay­ing $62 bil­lion to merge with Shire.

And the turn to fo­cus on sec­ondary end­points was an op­tion baked in­to the lu­vadax­i­s­tat pro­gram, ex­ecs told Stifel an­a­lyst Paul Mat­teis.

“(A)t the out­set of this pro­gram, Take­da had been de­bat­ing on whether to de­vel­op in schiz­o­phre­nia neg­a­tive symp­toms or cog­ni­tion, so a sig­nal on the lat­ter does align with some of the orig­i­nal sci­en­tif­ic hy­pothe­ses,” he wrote, adding that they al­so em­pha­sized “cog­ni­tive im­pair­ment in schiz­o­phre­nia is a big un­met need and worth pur­su­ing. That said, there’s al­so a lot of opac­i­ty here and ze­ro da­ta to an­a­lyze, so while the sec­ondary end­point sig­nal seems in­ter­est­ing and may cush­ion the hit to the stock, it will take a full pre­sen­ta­tion/pub­li­ca­tion to re­al­ly un­der­stand the prospects of ‘831.”

Shares in Neu­ro­crine $NBIX end­ed up falling 7.05% to $101.71.

We will have to wait for the da­ta. The com­pa­ny says it’s eval­u­at­ing the re­sults to de­ter­mine the next steps.

Kevin Gor­man, Neu­ro­crine CEO

Big Phar­ma's Twit­ter ex­o­dus; Mer­ck wa­gers $1.35B on buy­out; $3.5M gene ther­a­py; 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.

As you start planning for #JPM23, we hope you will consider joining Endpoints News for our live and virtual events. For those who are celebrating Thanksgiving, we hope you are enjoying the long weekend with loved ones. And if you’re not — we’ll see you next week!

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Paul Perreault, CSL Behring CEO

CSL lands FDA ap­proval for he­mo­phil­ia B gene ther­a­py, sets $3.5M list price

The FDA has approved the world’s first gene therapy for hemophilia B, ushering into the market a treatment that’s historic in both what it promises to do and how much it will cost.

CSL will be marketing the drug, Hemgenix, at a list price of $3.5 million — which sets a new record for the most expensive single-use gene therapy in the US.

In a statement provided to Endpoints News, the Australian company noted that the current costs of treating people with moderate to severe hemophilia B can be significant over a lifetime. By some estimates, healthcare systems could spend more than $20 million per person.

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Image: Shutterstock

MIT re­searchers re­veal DNA "Paste" tech be­hind lat­est gene edit­ing start­up

MIT scientists have developed a tool that they say can insert large gene sequences where they want in the genome.

In a paper published Thursday in Nature Biotechnology, MIT fellows Omar Abudayyeh, Jonathan Gootenberg and colleagues detail a technology they call PASTE, which they say can potentially be used to insert long strands of DNA and treat genetic diseases caused by many different mutations, such as cystic fibrosis and Leber congenital amaurosis, a rare eye disorder that causes blindness.

Elon Musk (GDA via AP Images)

Biggest drug com­pa­nies halt­ed Twit­ter ad buys af­ter Lil­ly in­sulin spoof

Almost all of the drug industry’s biggest advertisers cut their spending on Twitter to zero or near-zero over the last two weeks amid worries about impersonation of their brands by pranksters and the future of the social media company.

Among 18 of the biggest pharmaceutical advertisers in the US market, 12 cut their Twitter ad spending to nothing for the week beginning Nov. 14, according to Pathmatics, which tracks data on prescription drug ad spending as well as general corporate advertising. The list of drugmakers cutting spending to zero includes Merck, AstraZeneca, Eli Lilly, Novartis, Pfizer and others.

J&J's Spra­va­to pulls a PhI­II win against Sero­quel XR in treat­ment-re­sis­tant de­pres­sion

A day before Thanksgiving, J&J’s Janssen has a new cut of Phase III Spravato data to be grateful for.

The pharma giant announced on Wednesday that its nasal spray, also known as esketamine, beat extended-release quetiapine, previously sold by AstraZeneca as Seroquel XR, in treatment-resistant depression (TRD). Of 676 adults, a significantly higher number of patients on Spravato were able to achieve remission and avoid relapse after 32 weeks, according to J&J.

Rob Davis, Merck CEO

Up­dat­ed: No Seagen here: 'Do more' means a small $1.35B pur­chase of Ima­go for Mer­ck

Merck is making an acquisition, the Big Pharma announced before Monday’s opening bell. No, Seagen is not entering the fold, as had been speculated for quarters.

Folding under Merck’s wings will be Pfizer-backed Imago BioSciences. For nearly a year, Merck CEO Rob Davis has been saying the pharma giant needs to “do more” on the business development front after its 2021 $11.5 billion acquisition of Acceleron.

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Isao Teshirogi, Shionogi president and CEO (Kyodo via AP Images)

Sh­ionogi's Covid an­tivi­ral lands first ap­proval in Japan's new emer­gency ap­proval path­way

Japanese regulators on Tuesday signed off on Shionogi’s homegrown antiviral for Covid-19, known as Xocova (ensitrelvir), making it the first approval under Japan’s emergency regulatory approval system.

The emergency approval, following a back-and-forth with regulators since last February, is based on a safety profile with more than 2,000 patients who have accessed the pill, and clinical symptomatic efficacy for five typical Omicron-related symptoms (primary endpoint) and antiviral efficacy (key secondary endpoint) in patients with mild to moderate SARS-CoV-2 infection, regardless of risk factors or vaccination status, and during the Omicron-dominant phase of the pandemic.

Dermavant Sciences' first consumer TV ad for its Vtama psoriasis med shows people ready for a new topical treatment.

Roivant’s Der­ma­vant de­buts first-ever TV com­mer­cial for pso­ri­a­sis cream Vta­ma

Dermavant Sciences has been marketing its first product, psoriasis med Vtama, to dermatologists for months, but on Tuesday it rolled out its first consumer campaign. The debut DTC effort including a streaming TV commercial encourages patients to a “Topical Uprising” in a nod to Vtama being a topical cream.

In the new commercial, a swell of people discards scarves and jacket coverings, gathering in the street to converge on a pharmacy to demand a steroid-free prescription. A moment of levity follows when a pharmacist says, “You know you can just talk to your doctor, right?” The gathered crowds collectively says, “Oh.”

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FDA preps for DMD drug gener­ics as Sarep­ta has yet to fin­ish its con­fir­ma­to­ry tri­al

The FDA typically releases guidance to help generic drug manufacturers develop new copycats of small molecule drugs, oftentimes in preparation for a brand name product’s patents or exclusivity to expire.

This week, FDA released such bioequivalence guidance for any generic drugmakers looking to take on Sarepta’s Duchenne muscular dystrophy (DMD) drug Exondys 51 (eteplirsen), even though the drug’s sponsor has yet to convert the accelerated approval to a full approval, showing clinical benefit.