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.

A new era of treat­ment: How bio­mark­ers are chang­ing the way we think about can­cer

AJ Patel was recovering from a complicated brain surgery when his oncologist burst into the hospital room yelling, “I’ve got some really great news for you!”

For two years, Patel had been going from doctor to doctor trying to diagnose his wheezing, only to be dealt the devastating news that he had stage IV lung cancer and only six months to live. And then they found the brain tumors.

“What are you talking about?” Patel asked. He had never seen an oncologist so happy.

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Michael Corbo, Pfizer CDO of inflammation & immunology

UP­DAT­ED: Plan­ning ahead for crowd­ed ul­cer­a­tive col­i­tis mar­ket, Pfiz­er spells out PhI­II da­ta on $6.7B Are­na drug

Pfizer has laid out the detailed results behind its boast that etrasimod — the S1P receptor modulator at the center of its $6.7 billion buyout of Arena Pharma — is the winner of the class, potentially leapfrogging an earlier entrant from Bristol Myers Squibb.

Pivotal data from the ELEVATE program in ulcerative colitis — which consists of two Phase III trials, one lasting 52 weeks and the other just 12 weeks — illustrate an “encouraging balance of efficacy and safety,” according to Michael Corbo, chief development officer of inflammation & immunology at Pfizer. The company is presenting the results as a late breaker at Digestive Disease Week.

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Mihael Polymeropoulos, Vanda Pharmaceuticals CEO

Phar­ma com­pa­ny con­tin­ues its FDA law­suit spree, this time af­ter agency de­nies fast-track des­ig­na­tion

Vanda Pharmaceuticals is making a name for itself, at least in terms of suing the FDA.

The DC-headquartered firm on Monday filed its latest suit against the agency, with the company raising concerns over the FDA’s failure to grant a fast track designation for Vanda’s potential chronic digestive disorder drug tradipitant, which is a neurokinin 1 receptor antagonist.

Specifically, Vanda said FDA’s “essential point” in its one-page denial letter on the designation pointed to “the lack of necessary safety data,” which was “inconsistent with the criteria for … Fast Track designation.”

An NYU surgeon transplants an engineered pig kidney into the outside of a brain-dead patient (Joe Carrotta/NYU Langone Health)

'Xeno­trans­plan­ta­tion is com­ing': New NE­JM pa­per gives de­tailed look in­to 2 pig-to-hu­man kid­ney trans­plant cas­es

The thymokidney is a curious organ, if you could call it that. It’s a sort of Frankensteinian creation — a system of pig thymus embedded underneath the outer layer of a pig’s kidney, made for human transplantation.

In the first case of pig-to-human xenotransplantation of a kidney into a brain-dead patient, the thymokidney quietly featured front and center.

In that experiment, which took place in September of last year, NYU researchers led by Robert Montgomery sutured a pig thymokidney onto the leg of a brain-dead 66-year-old woman. That case was widely reported on by a horde of major media outlets, including the New York Times, the BBC, and an in-depth feature by USA Today.

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Vlad Coric, Biohaven CEO

UP­DAT­ED: Fresh off $11.6B sale to Pfiz­er, New Bio­haven hits Phase III set­back just weeks af­ter Vlad Coric chalked up promise

When Pfizer bought up Biohaven’s migraine portfolio in the largest M&A deal of the year earlier this month, Biohaven CEO Vlad Coric promised the rest of the pipeline, which will live on under the umbrella of New Biohaven, still has a lot to offer. But that vision took a dent Monday as the drugmaker revealed it’s once again flopped on troriluzole.

The glutamate regulator failed to meet the primary endpoint on a Phase III study in patients with spinocerebellar ataxia, an inherited disorder that impairs a person’s ability to walk, speak and swallow. SCA can also lead to premature death.

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Mod­er­na seeks to dis­miss Al­ny­lam suit over Covid-19 vac­cine com­po­nent, claim­ing wrong venue

RNAi therapeutics juggernaut Alnylam Pharmaceuticals made a splash in March when it sued and sought money from both Pfizer and Moderna regarding their use of Alnylam’s biodegradable lipids, which Alnylam claims have been integral to the way both companies’ mRNA-based Covid-19 vaccines work.

But now, Moderna lawyers are firing back, telling the same Delaware district court that Alnylam’s claims can only proceed against the US government in the Court of Federal Claims because of the way the company’s contract is set up with the US government. The US has spent almost $10 billion on Moderna’s Covid-19 vaccine so far.

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Cracks in the fa­cade: Is phar­ma's pan­dem­ic ‘feel good fac­tor’ wan­ing?

The discordant effects of the Covid-19 pandemic on pharma reputation continues. While the overall industry still retains a respectable halo from its Covid-19 quick response and leadership, a new patient group study reveals a different story emerging in the details.

On one hand, US patient advocacy groups rated the industry higher-than-ever overall. More than two-thirds (67%) of groups gave the industry a thumbs up for 2021, a whopping 10 percentage point increase over the year before, according to the PatientView annual study, now in its 9th year.

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Saqib Islam, SpringWorks CEO

Pfiz­er spin­out Spring­Works will ship its first drug to the FDA be­fore year’s end with PhI­II win

SpringWorks Therapeutics thinks it has cemented the backbone for its first “pipeline-in-a-product” oncology treatment and will send it to the FDA before the clock strikes 2023 with a Phase III win on Tuesday.

The oral gamma secretase inhibitor, dubbed nirogacestat, beat placebo on the primary goal of progression-free survival in adults with progressing desmoid tumors.

The soft-tissue tumors can lead to long-lasting pain, disfigurement and amputation, and there are currently no approved meds for the rare oncology indication. The tumors typically impact patients aged 20 to 44 years old and disproportionately affect women at rates 2 to 3 times higher, with up to a total of 1,650 new cases diagnosed in the US annually, according to SpringWorks.

Robert Califf (Michael Brochstein/Sipa USA via AP Images)

House Re­pub­li­cans at­tack Chi­na-on­ly da­ta in FDA sub­mis­sions, seek new in­ves­ti­ga­tion in­to re­search in­spec­tions

Three Republican representatives are calling on the FDA to take a closer look at the applications including only clinical data from China.

The letter to FDA commissioner Rob Califf late last week comes as the agency recently rejected Eli Lilly’s anti-PD-1 antibody, which attempted to bring China-only data but ran into a bruising adcomm that may crush the hopes of any other companies looking to bring cheaper follow-ons based only on Chinese data.

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