Re­gen­eron and Sanofi land the big one, win­ning FDA ap­proval for their megablock­buster hope­ful Dupix­ent

George Yan­copou­los, Pres­i­dent and CSO, Re­gen­eron

The FDA has ap­proved the new eczema drug dupilum­ab from Re­gen­eron and Sanofi, of­fi­cial­ly launch­ing a new drug that is like­ly on course to cre­at­ing a megablock­buster fran­chise for these two part­ners. Wide­ly tapped as the biggest pro­gram in the in­dus­try’s pipeline this year, dupilum­ab now will be brand­ed as Dupix­ent. And the mar­ket here could be worth up­wards of $5 bil­lion a year.

“Peo­ple with mod­er­ate-to se­vere atopic der­mati­tis cope with in­tense, some­times un­bear­able symp­toms that can im­pact them for most of their lives,” said Julie Block, the CEO of the Na­tion­al Eczema As­so­ci­a­tion. “To date, there have been few op­tions avail­able to treat peo­ple with mod­er­ate-to-se­vere atopic der­mati­tis who have un­con­trolled dis­ease. That’s why to­day’s ap­proval of Dupix­ent is so im­por­tant for our com­mu­ni­ty. Now we have a treat­ment that is ex­pect­ed to help ad­dress pa­tients suf­fer­ing from this dev­as­tat­ing dis­ease.”

The drug will be priced at $37,000 a year, a shade high­er than many an­a­lysts had ex­pect­ed, but not sig­nif­i­cant­ly out of line.

“We have set a fair and re­spon­si­ble price for Dupix­ent,” says a Sanofi spokesper­son. “We are en­cour­aged by the on­go­ing con­ver­sa­tions we’ve had with health plans and phar­ma­cy ben­e­fit man­agers about cov­er­age for Dupix­ent for pa­tients, and we have a shared com­mit­ment with them to en­sure pa­tients who should re­ceive it can re­ceive it.”

Ge­of­frey Porges, Leerink

Some top an­a­lysts wouldn’t dis­agree with that re­mark. Leerink’s Ge­of­frey Porges not­ed that the dis­count price should fall right around $30,000, which should be ac­cept­able to pay­ers. He not­ed:

While pay­ers may still be­lieve Dupix­ent is an ex­pen­sive ther­a­py, the drug is cost-ef­fec­tive for the in­di­cat­ed pa­tients, ac­cord­ing to the in­flu­en­tial ICER re­port. In­deed, in to­day’s press Ex­press Scripts’ (ES­RX, MP) CMO Steven Miller com­ment­ed that the man­u­fac­tur­ers had set a “re­spon­si­ble price,” and pay­ers ap­pear to be tak­ing cred­it for in­flu­enc­ing the pric­ing de­ci­sion. Re­cent drug launch­es have been harmed by un­clear ef­fi­ca­cy, broad­ly-la­beled pa­tient pop­u­la­tions that do not match avail­able clin­i­cal tri­al da­ta, and high list prices. By con­trast, Dupix­ent’s well-de­fined la­belled in­di­ca­tion and clear out­comes ben­e­fits, as well as the com­plete lack of FDA-ap­proved med­i­cines for post-top­i­cals mod­er­ate-to-se­vere AD, should lim­it the im­pact of step-ed­its and pri­or au­tho­riza­tion re­quire­ments.

Baird’s Bri­an Sko­r­ney ran a physi­cian sur­vey and found a large num­bers of doc­tors are ea­ger to start pre­scrib­ing this drug.

Bri­an Sko­r­ney, Baird an­a­lyst

“Im­por­tant­ly, physi­cian sen­ti­ment points to a mar­ket for Dupix­ent of about 300,000-350,000 pa­tients, in line with man­age­ment’s ex­pec­ta­tions,” he not­ed Tues­day morn­ing. “We wouldn’t be sur­prised to see an ex­cep­tion­al­ly strong launch here, but we con­tin­ue to point out that con­sen­sus ex­pec­ta­tions are al­ready build­ing that in.

The two bio­phar­ma part­ners — with Re­gen­eron whip­ping up the IL-4/IL-13 an­ti­body and Sanofi weigh­ing in with its late-stage mus­cle — have divvied up the mar­ket 50/50. And they’ve al­ready fired off a pre­emp­tive le­gal blast against a liti­gious Am­gen, their arch neme­sis which is sit­ting on IP for both tar­gets.

Am­gen has ag­gres­sive­ly pur­sued a law­suit against the Re­gen­eron/Sanofi’s team for their PC­SK9 drug Pralu­ent, which com­petes against Am­gen’s Repatha. At one point, Am­gen scored a stun­ning up­set, win­ning a court rul­ing in fa­vor of shelv­ing the ri­val drug, which has since been set aside.

In the part­ners’ suit against Am­gen, they say that they’ve learned that Am­gen has hired coun­sel to pur­sue a claim in­volv­ing patent ‘487, which in­volves IL-4 and IL-13 drugs. Am­gen tried, and failed, to de­vel­op their own IL4/IL-13 drug — AMG317 — and failed in Phase II. And Re­gen­eron and Sanofi are seek­ing a pre­emp­tive rul­ing against Am­gen, which loves to field its ag­gres­sive le­gal team when­ev­er it suits their strat­e­gy or the lawyers be­lieve there’s some rev­enue to grab off the ta­ble.

Re­gen­eron and Sanofi have much to pro­tect here, if the an­a­lysts are right. Pfiz­er scored an ap­proval for its atopic der­mati­tis drug Eu­crisa (crisabo­role), a PDE-4 in­hibitor, which has some block­buster am­bi­tions of its own. But Dupix­ent is ex­pect­ed to be game chang­er for pa­tients.

Just a few weeks ago in­ves­ti­ga­tors spelled out a new batch of stel­lar da­ta, com­plet­ing a ma­jor Phase III ef­fort that care­ful­ly re­viewed safe­ty and ef­fi­ca­cy da­ta for a wide range of pa­tients.

In the new CHRONOS da­ta re­viewed at the an­nu­al meet­ing of the Amer­i­can Acad­e­my of Der­ma­tol­ogy in Or­lan­do ear­li­er this month, in­ves­ti­ga­tors not­ed that the se­vere itch­ing pa­tients are af­flict­ed by was re­duced by 55% and 58% in the two drug arms af­ter 16 weeks, com­pared to 29% of pa­tients on TCS alone. And the dis­ease score rat­ing for pa­tients dropped by 4 or more points among 77% of the pa­tients in the drug arms com­pared to 37% of the place­bo/top­i­cal cor­ti­cos­teroid group.

Re­gen­eron and Sanofi need to make a quick suc­cess of this drug, and they’ll like­ly roll out an ag­gres­sive mar­ket­ing ef­fort now that they have re­ceived a long-await­ed green light from the FDA. They still be­lieve that they can make a big suc­cess of Repatha, but pay­ers have kept it care­ful­ly bot­tled up, wait­ing for car­dio out­comes. Rev­enue is mar­gin­al.

Then there’s sar­ilum­ab, held up by the FDA months ago so that Sanofi could clear up some em­bar­rass­ing man­u­fac­tur­ing is­sues.

Dupix­ent is a go. And the Re­gen­eron/Sanofi team is now tak­ing the mar­ket­ing field with a care­ful­ly planned blitz. They can’t af­ford an­oth­er set­back.

Sanofi in par­tic­u­lar, which has been sad­dled with a large­ly in­ef­fec­tive French R&D group, needs to show it can de­vel­op and field a ma­jor new drug. Now they have an­oth­er shot at glo­ry.

“Dupix­ent is the re­sult of years of tire­less re­search by our sci­en­tists in­to the un­der­ly­ing caus­es of al­ler­gic and atopic dis­eases. In atopic der­mati­tis, Dupix­ent was shown to help clear the skin and man­age the in­tense itch caused by the dis­ease,” said George D. Yan­copou­los, the found­ing sci­en­tist and CSO at Re­gen­eron. “To­day’s ap­proval would not be pos­si­ble with­out the ded­i­ca­tion of the clin­i­cal in­ves­ti­ga­tors and the par­tic­i­pa­tion of the pa­tients who took part in the glob­al LIB­ER­TY AD clin­i­cal pro­gram.”

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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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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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