Pfiz­er of­fers 3rd round of JAK da­ta, this time in kids. Can it com­pete?

Pfiz­er has re­leased the 3rd round of large and late-stage da­ta for their JAK in­hibitor and they say — well, they say pret­ty much what the first 2 stud­ies said, just in a new set of pa­tients.

The lat­est study, called JADE TEEN, was the first to study their JAK in­hibitor abroc­i­tinib ex­clu­sive­ly on chil­dren and ado­les­cents, test­ing it against place­bo in just un­der 300 atopic der­mati­tis pa­tients age 12 to 18. The youth pop­u­la­tion for AD is sig­nif­i­cant, with be­tween 15% and 20% of chil­dren af­fect­ed an­nu­al­ly by the in­flam­ma­to­ry con­di­tion world­wide.

As in the adult stud­ies — which al­lowed for pa­tients as young as 12 but had a mean age as high as 35 — the tri­al met its two pri­ma­ry end­points: a pair of met­rics that mea­sures how much the drug re­duces area of red­ness and itch­i­ness. But al­so like the adult stud­ies, the teens on the drug arm showed high­er rates of ad­verse ef­fects. The full de­tails weren’t dis­closed, but 62.8% of pa­tients on the high dose drug arm had side ef­fects, com­pared to 52.1% for place­bo.

In atopic der­mati­tis, Pfiz­er is go­ing up against an en­trenched com­peti­tor in Re­gen­eron and Sanofi’s Dupix­ent, an IL-4/IL-13 in­hibitor. In that strug­gle, the NJ phar­ma has a few things for them: It’s giv­en oral­ly as op­posed to sub­cu­ta­neous­ly, and over­all it has looked sim­i­lar in its abil­i­ty to treat the itch and pain that af­flict pa­tients.

“The ef­fect of abroc­i­tinib was fast on­set and could be ob­served as ear­ly as week 2, which con­curred our KOL’s im­pres­sion in pre­vi­ous in­ter­views,” Jef­feries’ Biren Amin wrote in a note to in­vestors af­ter the last round of abroc­i­tinib da­ta was pub­lished in JA­MA on June 3. “Our KOL con­sid­ers JA­Ki as an at­trac­tive op­tion for se­vere pts who are un­con­trolled by dupi.”

JAK in­hibitors, though, have broad­ly faced ques­tions about their safe­ty, ques­tions that have lim­it­ed the adop­tion of drugs that were long thought to have block­buster po­ten­tial. Ab­b­Vie’s JAK in­hibitor Rin­voq, for in­stance, got a black­box warn­ing af­ter stud­ies showed 1% of pa­tients got a se­ri­ous in­fec­tion, op­por­tunis­tic in­fec­tion, or her­pes zoster. In the JA­MA study, 1.9% of pa­tients on 100mg got a se­ri­ous in­fec­tion but none on 200mg. Two 200mg pa­tients, though, were in­fect­ed with her­pes.

There were al­so con­cerns about platelet re­duc­tion, with platelet counts drop­ping 26% for the 200mg dose and 19% for the 100mg group com­pared to es­sen­tial­ly no re­duc­tion on place­bo, though they even­tu­al­ly re­turned to nor­mal.

The lat­est top line read­out can’t an­swer those ques­tions. An­a­lysts will have to wait for the full da­ta. Still, with Sanofi and Re­gen­eron re­port­ing over $2 bil­lion in sales from Dupix­ent and atopic der­mati­tis af­fect­ing up to 20% of chil­dren and 3% adults, it’s a mar­ket that could have room for mul­ti­ple com­peti­tors.

Pfiz­er, which has gained a break­through ther­a­py des­ig­na­tion for the drug, is plan­ning to file for FDA ap­proval lat­er this year.

Cell and Gene Con­tract Man­u­fac­tur­ers Must Em­brace Dig­i­ti­za­tion

The Cell and Gene Industry is growing at a staggering 30% CAGR and is estimated to reach $14B by 20251. A number of cell, gene and stem cell therapy sponsors currently have novel drug substances and products and many rely on Contract Development Manufacturing Organizations (CDMO) to produce them with adherence to stringent regulatory cGMP conditions. Cell and gene manufacturing for both autologous (one to one) and allogenic (one to many) treatments face difficult issues such as: a complex supply chain, variability on patient and cellular level, cell expansion count and a tight scheduling of lot disposition process. This complexity affects quality, compliance and accountability in the entire vein-to-vein process for critically ill patients.

Phase III read­outs spell dis­as­ter for Genen­tech’s lead IBD drug

Roche had big plans for etrolizumab. Eyeing a hyper-competitive IBD and Crohn’s market where they have not historically been a player, the company rolled out 8 different Phase III trials, testing the antibody for two different uses across a range of different patient groups.

On Monday, Roche released results for 4 of those studies, and they mark a decided setback for both the Swiss pharma and their biotech sub Genentech, potentially spelling an end to a drug they put over half-a-decade and millions of dollars behind.

Eric Shaff (Seres)

UP­DAT­ED: Af­ter a 4-year so­journ, strug­gling mi­cro­bio­me pi­o­neer Seres claims a break­out PhI­II come­back. And shares re­spond in fren­zied spike

Almost exactly 4 years ago, Seres Therapeutics $MCRB experienced one of those soul-crunching failures that can raise big questions about a biotech’s future. Out front in their pursuit of a gut punch to C. difficile infection (CDI), the Phase II test was a flat failure, and investors wiped out a billion dollars of equity value that never returned in the years that followed.

Seres, though, pressed ahead, changing out CEOs a year ago — bidding Merck vet Roger Pomerantz farewell from the C suite — and pushing through a Phase III, hoping that amping up the dosage would make the key difference. And this morning, they unveiled a claim that they had aced the Phase III and positioned themselves for a run at a landmark FDA OK.

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Michel Vounatsos, Biogen CEO (via YouTube)

UP­DAT­ED: Bio­gen scores a pri­or­i­ty re­view for its Alzheimer's drug ad­u­canum­ab, mov­ing one gi­ant leap for­ward in its con­tro­ver­sial quest

Biogen scored a big win at the FDA today as regulators accepted their application for the controversial Alzheimer’s drug aducanumab and gave it a priority review.

The PDUFA date is March 7, 2021.

Significantly, Biogen says it did not use its priority review voucher to win special treatment at the FDA. The agency handed that out gratis.

That’s the ideal scenario Biogen was looking for as disappointed analysts wondered aloud about the delayed application earlier in the year.

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In­novent and Eli Lil­ly chal­lenge Mer­ck­'s mega-block­buster Keytru­da in non-small cell lung can­cer field

China-based Innovent Biologics and its multinational ally Eli Lilly shared Phase III evidence that their PD-1 inhibitor combo can delay the progression of nonsquamous non-small cell lung cancer.

But the drugmakers will face stiff competition in China from Merck’s Keytruda, the ruling PD-1 which is already approved to treat both squamous and nonsquamous NSCLC and boasts positive overall survival rates.

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Anap­tys­Bio's etokimab pro­vides more dis­ap­point­ing re­sults, rais­ing ques­tions about com­pound's fu­ture

The lead program for AnaptysBio’s in-house pipeline has hit another setback.

Etokimab, an IL-33 inhibitor, did not achieve statistically significant improvement in a Phase II trial for patients suffering from chronic rhinosinusitis with nasal polyps. Researchers measured the individuals’ bilateral nasal polyps score and sino-nasal outcome test, finding that neither improved upon a placebo after both four- and eight-week time markers, though they did demonstrate improvement over baseline levels of the examinations.

Brian Stuglik, Verastem CEO

The du­velis­ib hot pota­to is tossed to a new own­er as Ve­rastem looks to re­or­ga­nize around the pipeline

When Infinity put up duvelisib for a no-money-down instant deal, the biotech was looking for a quick exit from a clinical disaster. AbbVie had walked away from their alliance after looking at how the data stacked up in a crowded field.

And while it was approvable, it wasn’t looking pretty to anyone who thought in commercial terms.

One Big Pharma’s trash, though, was seen as a biotech treasure as a deeply troubled Verastem stepped up to grab the PI3K-delta/gamma — promising to run it across the goal lines at the FDA. And they did just that, only with little to show for it.

DFC CEO Adam Boehler and Kodak CEO Jim Continenza (Kodak)

Covid-19 roundup: Cure­Vac beefs up its uni­corn IPO dreams as bil­lion­aire own­er takes this Covid-19 mR­NA play­er on a forced march to Nas­daq; Ko­dak's $765M deal is put on hold

When CureVac initially jotted down $100 million for its IPO raise a couple of weeks ago, it seemed small. The German mRNA player, after all, had jumped into a Covid-19 race that swelled the sails of Moderna and BioNTech by tens of billions. And after raising $640 million in a slate of deals, $100 million in a hot market like this seemed like a pittance in the bigger scheme of things.

Today, we got a look at a figure that probably comes closer to the game-changing number the top execs probably have in mind. Selling 15.3 million shares at the high end of their $14 to $16 range would net a $243 million bounty. Majority owner Dietmar Hopp is putting in another €100 million, bringing the total to around $350 million. And what are the chances they want to do even better than that?

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Warren Huff, Reata CEO

Rea­ta sug­gests Friedre­ich's atax­ia pro­gram could be de­layed, send­ing stock plung­ing

Reata Pharmaceuticals $RETA made waves last October when its drug omaveloxolone produced positive trial results in treating a rare neurological disorder, but the candidate’s path forward became much murkier Monday.

In a report of quarterly earnings, the biotech divulged that the FDA is considering delaying omaveloxolone’s NDA pending completion of a second trial. That could push back approval by at least a year given that the target population, individuals with Friedreich’s ataxia, is limited and progression of the hard-to-treat illness is notoriously slow. The Covid-19 pandemic would also hinder Reata’s ability to complete an additional trial.