Block­buster per­for­mance: Re­gen­eron, Sanofi add stel­lar PhI­II dupilum­ab da­ta on dis­play at EADV

Af­ter a steady drum­beat of praise from Re­gen­eron and its part­ners at Sanofi, in­ves­ti­ga­tors to­day backed up their un­blush­ing op­ti­mism over their late-stage drug prospect dupilum­ab with a batch of stel­lar late-stage tri­al da­ta for atopic der­mati­tis, adding to the pos­i­tive pro­file al­ready sketched out.

The sci­en­tists turned up at the Eu­ro­pean Acad­e­my of Der­ma­tol­ogy and Venere­ol­o­gy in Vi­en­na to re­view the da­ta from SO­LO1 and SO­LO2. And it was worth the wait for a drug they plan to mar­ket as Dupix­ent as they look to start rack­ing up rev­enue against the multi­bil­lion-dol­lar peak sales es­ti­mates that have hov­ered around this drug.

On every ma­jor score, dupilum­ab hand­i­ly out­per­formed the place­bo, un­der­scor­ing the part­ners’ high ex­pec­ta­tions for a ther­a­py that’s al­so in late-stage test­ing for asth­ma. And new da­ta on itch­ing and de­pres­sion should help close the sale.

Gi­an­lu­ca Pirozzi

“I tru­ly be­lieve this is go­ing to be rev­o­lu­tion­ary for many pa­tients,” Gi­an­lu­ca Pirozzi, the glob­al project head for Sanofi, tells me. This is the first mon­o­clon­al an­ti­body that blocks both Il-4 and IL-13 cy­tokines, and there’s noth­ing that looks like a ri­val threat on the near hori­zon, leav­ing these two big play­ers a lot of room to move.

The pa­tients in these sto­ries rep­re­sent a se­vere­ly af­flict­ed group, he adds, with no chance of re­lief. And while a long way from a uni­ver­sal ef­fect, the da­ta in­di­cate that this drug has a tremen­dous amount of com­mer­cial po­ten­tial, with lit­tle in the way of safe­ty is­sues to fret about.

The full re­port card now in­cludes:

On the Pru­ri­tus NRS ranges from 0 (no itch) to 10 (worst itch imag­in­able). At 16 weeks, for SO­LO 1 and SO­LO 2, re­spec­tive­ly, 41 and 36 per­cent of pa­tients who re­ceived Dupix­ent 300 mg every two weeks, and 40 and 39 per­cent of pa­tients who re­ceived Dupix­ent 300 mg week­ly, achieved a four-point or greater re­duc­tion in their NRS score com­pared to 12 and 10 per­cent with place­bo.

Lest any­one for­get, the two big part­ners have al­so post­ed an im­pres­sive amount of pos­i­tive da­ta.

On the pri­ma­ry end­point: 37 and 36 per­cent of adult pa­tients who re­ceived Dupix­ent 300 mg week­ly, and 38 and 36 per­cent of pa­tients who re­ceived Dupix­ent 300 mg every two weeks, achieved clear­ing or near-clear­ing of skin le­sions as mea­sured by the 5-point In­ves­ti­ga­tor’s Glob­al As­sess­ment (IGA) scale. In the place­bo arm, those rates were tracked at 10 and 8 per­cent with place­bo.

On an­oth­er EU pri­ma­ry and US sec­ondary end­point: 52 and 48 per­cent of adult pa­tients who re­ceived Dupix­ent 300 mg week­ly, and 51 and 44 per­cent of pa­tients who re­ceived Dupix­ent 300 mg every two weeks, achieved a 75 per­cent or greater re­duc­tion in their Eczema Area and Sever­i­ty In­dex score (EASI-75). In the place­bo arm that hit 15 and 12 per­cent.

On the per­cent im­prove­ment in EASI from base­line:  The score was 72 and 69 per­cent in pa­tients who re­ceived the 300 mg week­ly dose, and 72 and 67 per­cent for pa­tients who re­ceived Dupix­ent 300 mg every two weeks, com­pared to 38 and 31 per­cent for place­bo.

Based on the da­ta they’ve seen so far, it looks like they will go with a drug dosed once every two weeks. Dupilum­ab was re­cent­ly filed with the FDA and is get­ting a pri­or­i­ty re­view, with a PDU­FA date on March 29. So far, this drug has been sail­ing through a big Phase III with­out a hitch.

An­a­lysts haven’t over­looked the po­ten­tial here, but there has been some fret­ting that pay­ers are al­ready lay­ing in wait for a big new ther­a­py like this. And that could make it hard for these part­ners to re­al­ize the kind of big gains that have been fore­cast. Ge­of­frey Porges notes:

We now look to the part­nered com­mer­cial or­ga­ni­za­tions to ex­plain how they plan to price and pro­mote dupi, at a man­age­able ex­pense in­vest­ment, to avoid the for­mi­da­ble road­blocks that have stalled the re­cent launch­es of oth­er chron­ic use spe­cial­ty ther­a­peu­tics with large com­mer­cial op­por­tu­ni­ties. Oth­er­wise we have con­cerns that the stock is fac­ing launch es­ti­mates that are too high, and launch rev­enue that falls short of po­ten­tial.

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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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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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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UP­DAT­ED: An em­bold­ened As­traZeneca splurges $95M on a pri­or­i­ty re­view vouch­er. Where do they need the FDA to hus­tle up?

AstraZeneca is in a hurry.

We learned this morning that the pharma giant — not known as a big spender, until recently — forked over $95 million to get its hands on a priority review voucher from Sobi, otherwise known as Swedish Orphan Biovitrum.

That marks another step down on price for a PRV, which allows the holder to slash 4 months off of any FDA review time.

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Bob Smith, Pfizer

Pfiz­er is mak­ing a $500M state­ment to­day: Here’s how you be­come a lead play­er in the boom­ing gene ther­a­py sec­tor

Three years ago, Pfizer anted up $150 million in cash to buy Bamboo Therapeutics in Chapel Hill, NC as it cautiously stuck a toe in the small gene therapy pool of research and development.

Company execs followed up a year later with a $100 million expansion of the manufacturing operations they picked up in that deal for the UNC spinout, which came with $495 million in milestones.

And now they’re really going for it.

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Video: Putting the AI in R&D — with Badhri Srini­vasan, Tony Wood, Rosana Kapeller, Hugo Ceule­mans, Saurabh Sa­ha and Shoibal Dat­ta

During BIO this year, I had a chance to moderate a panel among some of the top tech experts in biopharma on their real-world use of artificial intelligence in R&D. There’s been a lot said about the potential of AI, but I wanted to explore more about what some of the larger players are actually doing with this technology today, and how they see it advancing in the future. It was a fascinating exchange, which you can see here. The transcript has been edited for brevity and clarity. — John Carroll

As­traZeneca’s Imfinzi/treme com­bo strikes out — again — in lung can­cer. Is it time for last rites?

AstraZeneca bet big on the future of their PD-L1 Imfinzi combined with the experimental CTLA-4 drug tremelimumab. But once again it’s gone down to defeat in a major Phase III study — while adding damage to the theory involving targeting cancer with a high tumor mutational burden.

Early Wednesday the pharma giant announced that their NEPTUNE study had failed, with the combination unable to beat standard chemo at overall survival in high TMB cases of advanced non-small cell lung cancer. We won’t get hard data until later in the year, but the drumbeat of failures will call into question what — if any — future this combination can have left.

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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.