Rux­oli­tinib pass­es an­oth­er PhI­II test for graft-ver­sus-host dis­ease, a win for In­cyte and No­var­tis

Rux­oli­tinib has proven it­self a stel­lar mon­ey­mak­er for In­cyte and No­var­tis, and it should come as no sur­prise that the drug’s ef­fi­ca­cy reached ex­pec­ta­tions in yet an­oth­er Phase III tri­al.

The two com­pa­nies an­nounced Thurs­day that rux­oli­tinib — sold as Jakafi by In­cyte in the US and as Jakavi by No­var­tis else­where — met its pri­ma­ry end­point in a study eval­u­at­ing treat­ment in pa­tients with chron­ic graft-ver­sus-host dis­ease who did not re­spond well to steroids. Mea­sured against the best avail­able ther­a­py, rux­oli­tinib showed a sig­nif­i­cant­ly im­proved over­all re­sponse rate af­ter 24 weeks.

Full da­ta haven’t been re­leased yet, but the drug al­so met both sec­ondary end­points im­prov­ing fail­ure-free sur­vival and pa­tient-re­port­ed symp­toms. The lat­ter was mea­sured by the mod­i­fied Lee cGVHD Symp­tom Scale.

This news comes just a few months af­ter a sep­a­rate tri­al il­lus­trat­ed the drug’s ef­fi­ca­cy in treat­ing acute GVHD, which man­i­fests much ear­li­er than its chron­ic coun­ter­part. That study mea­sured over­all re­sponse rate af­ter on­ly 28 days of rux­oli­tinib treat­ment.

Graft-ver­sus-host dis­ease oc­curs af­ter pa­tients re­ceive a trans­plant that us­es stem cells. The stem cells es­sen­tial­ly give the body a new im­mune sys­tem but treat the ex­ist­ing tis­sue as for­eign. GVHD can oc­cur at any time af­ter a trans­plant, and the risk is greater if the donor is un­re­lat­ed.

Acute GVHD usu­al­ly ap­pears with­in 100 days of a trans­plant, where­as chron­ic GVHD takes longer than that bench­mark. Pa­tients with chron­ic GVHD can al­so ex­hib­it some symp­toms sim­i­lar to au­toim­mune dis­eases.

The topline re­sults “un­der­score the po­ten­tial for Jakafi to pro­vide a mean­ing­ful treat­ment op­tion, not on­ly for pa­tients with acute GVHD, but al­so for the chron­ic form of the con­di­tion,” said Pe­ter Lang­muir, In­cyte’s group VP for on­col­o­gy tar­get­ed ther­a­pies.

Rux­oli­tinib it­self is a JAK in­hibitor tar­get­ing JAK1 and JAK2 and had pre­vi­ous­ly been ap­proved in the US and EU as a sec­ond-line treat­ment for poly­cythemia ve­ra, as well as a first-line treat­ment for adults with myelofi­bro­sis, or the buildup of scar tis­sue in bone mar­row. Pa­tients who ex­hib­it MF af­ter be­ing treat­ed for PV can al­so be pre­scribed rux­oli­tinib.

De­spite rux­oli­tinib’s con­tin­ued suc­cess, net­ting In­cyte $1.7 bil­lion in sales last year, the Delaware-based biotech has seen its share of set­backs in re­cent years. Back in 2018, there was the epaca­do­stat de­ba­cle when In­cyte had to halt a Phase III tri­al re­search­ing ef­fi­ca­cy in metasta­t­ic melanoma. And ear­ly in 2020, a sep­a­rate acute GVHD treat­ment dubbed itac­i­tinib flopped in a first-line, Phase III treat­ment study.

But rux­oli­tinib has kept the com­pa­ny afloat for now, and the drug’s con­tin­ued suc­cess in Phase III tri­als demon­strates its longevi­ty. In the US, a top­i­cal Jakafi cream passed two Phase III read­outs in treat­ing atopic der­mati­tis ear­li­er 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.

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.

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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