Neu­ro­crine steers its ‘break­through’ tar­dive dysk­i­ne­sia drug to an FDA de­ci­sion

San Diego-based Neu­ro­crine Bio­sciences an­nounced that it has filed their NDA on a new drug for tar­dive dysk­i­ne­sia to­day, but the com­pa­ny isn’t wait­ing for the FDA to make up its mind be­fore lay­ing the ground­work on a mar­ket­ing cam­paign.

Bri­an Sko­r­ney, Baird an­a­lyst

Com­pa­ny ex­ecs not­ed in their Q2 call with an­a­lysts that they are al­ready build­ing a mar­ket­ing group for the drug, val­be­nazine, which has achieved a break­through drug des­ig­na­tion that could put it on track to an ap­proval as ear­ly as next spring. The drug is a VMAT2 in­hibitor, de­signed to mod­u­late dopamine re­lease dur­ing nerve com­mu­ni­ca­tion. And it’s al­so in Phase II for Tourette syn­drome.

Neu­ro­crine $NBIX has a $4.3 bil­lion mar­ket cap.

Tar­dive dysk­i­ne­sia, though, is no easy af­flic­tion to mar­ket to. The dis­ease is char­ac­ter­ized by repet­i­tive and un­con­trol­lable body move­ments, like gri­mac­ing, tongue move­ments and lip smack­ing. And for Neu­ro­crine to live up to an­a­lysts’ projects of a block­buster bil­lion dol­lars or more in rev­enue, the com­pa­ny has to over­come some built-in mar­ket hur­dles while pur­su­ing ad­di­tion­al ap­provals. Chief among those hur­dles will be the one-on-one ed­u­ca­tion of doc­tors who cur­rent­ly don’t re­al­ly know how to rec­og­nize or treat the con­di­tion.

Here’s Neu­ro­crine’s Chief Med­ical Of­fi­cer Chris O’Brien from the call:

(I)f you go out and talk to 100 docs or psy­chi­a­trists, you’ll find ex­am­ples where you’ll find some docs say TD doesn’t ex­ist any­more, we don’t see it. You’ll find oth­er docs say, this prob­lem is more per­va­sive than I ever re­al­ized it was. I’m see­ing TD as com­mon­ly now as I did 20 years ago, but it’s just not as se­vere. You’ll then see docs that say, yes, my pa­tients have TD, but I don’t want to do any­thing that is go­ing to desta­bi­lize them and ob­vi­ous­ly that was a key fo­cus of our tri­al de­signs is that we had a sim­ple once-a-day add-on med­ica­tion that re­quired no changes in un­der­ly­ing ther­a­py and we showed no wors­en­ing of psy­chi­atric state.

So, once you talk to docs about what val­be­nazine has shown in tri­als to date, that is when as Kevin (Gor­man) said, they start to get ex­cit­ed about it. They say, you mean, I can use some­thing that’s once-a-day, easy to use and you’ve shown me videos about the phe­nom­e­nol­o­gy of what Tar­dive Dysk­i­ne­sia is. I can do this safe­ly. Then, their at­ti­tude changes. But this is ob­vi­ous­ly part of what has to hap­pen for suc­cess­ful launch of the drug.

Baird’s Bri­an Sko­r­ney had this to say to­day:

Though val­be­nazine has yet to launch in tar­dive dysk­i­ne­sia, we are ea­ger to see what else the drug can do. Com­ing out of open-la­bel re­sults last year in Tourette, the up­com­ing Phase 2 da­ta from chil­dren, ado­les­cents, and adults (T-Force GREEN, T-For­ward) will be able to give us a read on how the drug stacks up. Re­call, the two stud­ies are mea­sur­ing tic sever­i­ty through the Yale Glob­al Tic Sever­i­ty Scale. Be­cause the place­bo ef­fect in neu­ro­log­i­cal and psy­chi­atric dis­eases, es­pe­cial­ly Tourette, can be high, this will be the first op­por­tu­ni­ty for us to see if the drug pro­vides a ben­e­fit over place­bo. We are not cur­rent­ly mod­el­ing the Tourette op­por­tu­ni­ty, but see po­ten­tial for it to add an in­cre­men­tal $300-500M in peak sales on top of the over $1B peak po­ten­tial we es­ti­mate in TD.

Martin Shkreli [via Getty]

Pris­on­er #87850-053 does not get to add drug de­vel­op­er to his list of cred­its

Just days after Retrophin shed its last ties to founder Martin Shkreli, the biotech is reporting that the lead drug he co-invented flopped in a pivotal trial. Fosmetpantotenate flunked both the primary and key secondary endpoints in a placebo-controlled trial for a rare disease called pantothenate kinase-associated neurodegeneration, or PKAN.

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We­bi­nar: Re­al World End­points — the brave new world com­ing in build­ing fran­chise ther­a­pies

Several biopharma companies have been working on expanding drug labels through the use of real world endpoints, combing through the data to find evidence of a drug’s efficacy for particular indications. But we’ve just begun. Real World Evidence is becoming an important part of every clinical development plan, in the soup-through-nuts approach used in building franchises.

I’ve recruited a panel of 3 top experts in the field — the first in a series of premium webinars — to look at the practical realities governing what can be done today, and where this is headed over the next few years, at the prodding of the FDA.

ZHEN SU — Merck Serono’s Senior Vice President and Global Head of Oncology
ELLIOTT LEVY — Amgen’s Senior Vice President of Global Development
CHRIS BOSHOFF — Pfizer Oncology’s Chief Development Officer

A premium subscription to Endpoints News is required to attend this webinar. Please upgrade to either an Insider or Enterprise plan for access. Already have Endpoints Premium? Please sign-in below. You can contact our Subscriptions team at help@endpointsnews.com with any issues.

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Hal Barron. GSK

GSK's Hal Bar­ron her­alds their sec­ond pos­i­tive piv­otal for cru­cial an­ti-BC­MA ther­a­py, point­ing to a push for quick OKs in a crowd­ed field

Hal Barron has his second positive round of Phase III data in hand for his anti-BCMA antibody drug conjugate belantamab mafodotin (GSK2857916). And GSK’s research chief says the data paves the way for their drive in search of an FDA approval for treating multiple myeloma.

It’s hard to overestimate the importance of this drug for GSK, a cornerstone of Barron’s campaign to make a dramatic impact on the oncology market and provide some long-lost excitement for the pharma giant’s pipeline. They’re putting this BCMA program at the front of that charge — looking to lead a host of rivals all aimed at the same target.

We don’t know what the data are yet, but DREAMM-2 falls on the heels of a promising set of data delivered 5 months ago for DREAMM-1. There investigators noted that complete responses among treatment-resistant patients rose to 15% in the extra year’s worth of data to look over, with a median progression-free survival rate of 12 months, up from 7.9 months reported earlier. The median duration of response was 14.3 months.

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Brian Kaspar. AveXis via Twitter

AveX­is sci­en­tif­ic founder fires back at No­var­tis CEO Vas Narasimhan, 'cat­e­gor­i­cal­ly de­nies any wrong­do­ing'

Brian Kaspar’s head was among the first to roll at Novartis after company execs became aware of the fact that manipulated data had been included in its application for Zolgensma, now the world’s most expensive therapy.

But in his first public response, the scientific founder at AveXis — acquired by Novartis for $8.7 billion — is firing back. And he says that not only was he not involved in any wrongdoing, he’s ready to defend his name as needed.

I reached out to Brian Kaspar after Novartis put out word that he and his brother Allen had been axed in mid-May, two months after the company became aware of the allegations related to manipulated data. His response came back through his attorneys.

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Why would Am­gen want to buy Alex­ion? An­a­lysts call hot­ly ru­mored takeover un­like­ly, but seize the mo­ment

A rumor that Amgen is closing in on buyout deal for Alexion has sparked a guessing game on just what kind of M&A strategy Amgen is pursuing and how much Alexion is worth.

Mizuho analyst Salim Syed first lent credence to the report out of the Spanish news outlet Intereconomía, which said Amgen is bidding as much as $200 per share. While the source may be questionable, “the concept of this happening doesn’t sound too crazy to me,” he wrote.

FDA asks why No­var­tis took two months to launch for­mal in­ter­nal probe, af­ter AveX­is flagged da­ta ma­nip­u­la­tion

And the plot thickens. Novartis $NVS officials are reportedly now scrambling to explain to the FDA why it took them two months to open an internal investigation into data discrepancies for their $2.1 million gene-therapy for spinal muscular dystrophy — the world’s most expensive drug.

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Build­ing on suc­cess­ful PD-1 pact, Eli Lil­ly li­cens­es di­a­betes drug to Chi­nese part­ners at In­novent

Eli Lilly is expanding its partnership with China’s Innovent in a deal involving a diabetes drug sitting in its Phase I reserves.

The two companies had jointly developed one of China’s first homegrown PD-1 agents, scoring an approval for Tyvyt (sintilimab) late last year for relapsed/refractory classical Hodgkin’s lymphoma. This time around, Lilly is out-licensing a piece of its diabetes pipeline, a leading franchise that has historically produced the top-selling Trulicity and Humalog.

Am­gen, Al­ler­gan biosim­i­lar of Roche's block­buster Rit­ux­an clears an­oth­er US piv­otal study 

Novartis $NVS may have given up, but Amgen $AMGN and Allergan $AGN are plowing ahead with their knockoff of Roche’s blockbuster biologic Rituxan in the United States.

Their copycat, ABP 798, was found to have a clinically equivalent impact as Rituxan — meeting the main goal of the study involving CD20-positive B-cell non-Hodgkin’s lymphoma patients. This is the second trial supporting the profile of the biosimilar. In January, it came through with positive PK results in patients with rheumatoid arthritis.

Trump ad­min­is­tra­tion re­vives bid to get drug list prices on TV ads

The Trump administration is not giving up just yet. On Wednesday, the HHS filed an appeal against a judge’s decision in July to overturn a ruling obligating drug manufacturers to disclose the list price of their therapies in television adverts — hours before it was stipulated to go into effect.

In May, the HHS published a final ruling requiring drugmakers to divulge the wholesale acquisition cost— of a 30-day supply of the drug — in tv ads in a bid to enhance price transparency in the United States. The pharmaceutical industry has vehemently opposed the rule, asserting that list prices are not what a typical patient in the United States pays for treatment — that number is typically determined by the type of (or lack thereof) insurance coverage, deductibles and out-of-pocket costs. Although there is truth to that claim, the move was considered symbolic in the Trump administration’s healthcare agenda to hold drugmakers accountable in a climate where skyrocketing drug prices have incensed Americans on both sides of the aisle.