In­cyte scores much need­ed PhI­II suc­cess — and of course it’s de­liv­ered by rux­oli­tinib

In­cyte’s ef­forts to breathe a sec­ond life in­to rux­oli­tinib — its JAK in­hibitor sold in pill form as Jakafi — has been greet­ed with clear, if pre­lim­i­nary and un­sur­pris­ing, Phase III suc­cess.

Topline da­ta from the TRuE-AD2 ce­ments rux­oli­tinib’s foun­da­tion­al im­por­tance for In­cyte, and gives an­a­lysts hope that there might yet be room for growth in a pipeline that’s suf­fered mul­ti­ple R&D set­backs.

Com­par­ing two dos­es of top­i­cal rux­oli­tinib to a place­bo cream, re­searchers found that “sig­nif­i­cant­ly more pa­tients” with atopic der­mati­tis on the drug arms (0.75% and 1.5%, re­spec­tive­ly) achieved treat­ment suc­cess as de­fined by the in­ves­ti­ga­tor’s glob­al as­sess­ment score, or IGA score, of 0 or 1. To qual­i­fy, pa­tients would al­so have to see at least a two-point im­prove­ment from base­line at Week 8.

That’s as much de­tail as we’re go­ing to get un­til the biotech presents at a con­fer­ence. The pre­cise num­bers on that pri­ma­ry end­point, as well as the slate of sec­ondary mea­sures in­clud­ing mul­ti­ple vari­a­tions of the Eczema Area and Sever­i­ty In­dex (EASI), will have to wait.

Al­so com­ing lat­er this quar­ter: re­sults from a sec­ond piv­otal study dubbed TRuE-AD1. To­geth­er the pro­gram en­rolled 1,200 pa­tients, who will be fol­lowed for up to a year for long-term safe­ty and ef­fec­tive­ness.

In­cyte po­si­tioned the drug as an al­ter­na­tive to cor­ti­cos­teroids — the stan­dard of care — and a safe op­tion in mild-to-mod­er­ate AD.

“With­in mild-mod­er­ate dis­ease, a pro­por­tion of pa­tients are not well con­trolled on TCS/TCIs, and / or are not el­i­gi­ble for steroids be­cause of dis­ease on the face,” the com­pa­ny wrote in an email to End­points News. “In ad­di­tion, the ma­jor­i­ty of pa­tients seek­ing treat­ment for AD in a der­ma­tol­o­gists of­fice have tried TCS pre­vi­ous­ly (many have tried mul­ti­ple dif­fer­ent ones).”

How­ev­er, Evan Seiger­man of Cred­it Su­isse not­ed that the tri­al “was not de­signed to show rel­a­tive­ly ef­fi­ca­cy to SoC of top­i­cal cor­ti­cos­teroids, which could lim­it its use among der­ma­tol­o­gists.”

That said, In­cyte does have some com­para­tor da­ta go­ing for it, Cowen an­a­lysts wrote when it un­veiled Phase II da­ta last June. Drop­ping in on pre­sen­ta­tions at the World Derm con­fer­ence, they saw that lack of ref­er­ence to es­tab­lished treat­ments in­deed turned out to be a prob­lem for Pfiz­er’s Eu­crisa.

“This has been cit­ed as a hin­drance to adop­tion in ad­di­tion to its mod­est ef­fi­ca­cy (31-33% IGA re­sponse at week 8) and as­so­ci­a­tion with ap­pli­ca­tion site pain,” they wrote.

In con­trast, 1.5% rux­oli­tinib cream, ap­plied twice dai­ly, was di­rec­tion­al­ly su­pe­ri­or to the steroid tri­am­ci­nolone at week 4 on EASI (72% vs. 60%) and IGA (38% vs. 26%).

And for those who see AD and think Dupix­ent, Seiger­man point­ed out that rux­oli­tinib tar­gets a dif­fer­ent pa­tient pop­u­la­tion with less se­vere dis­ease.

“We agree with the sci­en­tif­ic ra­tio­nale for us­ing top­i­cal rux­oli­tinib in AD, and would ex­pect that the top­i­cal ad­min­is­tra­tion would more like­ly to lim­it se­ri­ous side ef­fects more com­mon with sys­temic treat­ments,” he wrote.

He es­ti­mates that the top­i­cal for­mu­la­tion, which is slat­ed for a 2020 NDA sub­mis­sion and 2021 launch, could add $300 mil­lion in US sales for In­cyte by 2025. An­drew Berens at SVB Leerink pegged peaks for top­i­cal rux­oli­tinib at $1.1 bil­lion.

In­cyte’s top team, led by CEO Hervé Hop­penot, is hold­ing on­to every dol­lar and op­ti­mistic sen­ti­ment around the cash cow fran­chise to buy time for some R&D rein­vig­o­ra­tion. The IDO dis­as­ter with epaca­do­stat and the re­cent Phase III flop with itac­i­tinib blew a huge hole in the late-stage pipeline that the biotech has now filled part­ly with Mor­phoSys’ CD19-tar­get­ed taf­a­sita­m­ab. It cost $900 mil­lion in cash.

There’s more work go­ing on with Jakafi in chron­ic graft ver­sus host dis­ease, among oth­er projects (the oth­er mid-stage prospects tar­get FGFR1/2/3 and PI3Kδ, re­spec­tive­ly).

“We are most in­ter­est­ed in the da­ta from the TRuE-V tri­als of top­i­cal rux. in vi­tili­go (giv­en the high­er un­met med­ical need and lack of treat­ment op­tions), not ex­pect­ed to read out un­til 2021,” Seiger­man wrote.

Un­til then the up­side is rel­a­tive­ly mod­est, he pre­dict­ed. In­cyte shares $IN­CY are up 2.14% to $77.7.

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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Ryan Watts, Denali CEO

Bio­gen hands De­nali $1B-plus in cash, $1B-plus in mile­stones to part­ner on late-stage Parkin­son’s drug

Biogen is handing over more than a billion dollars cash to partner with the up-and-coming neurosciences crew at Denali on a new therapy for Parkinson’s. And the big biotech is ready to pile on more than a billion dollars more in milestones — if the alliance is a success.

For Biogen $BIIB, the move on Denali’s small molecule inhibitors of LRRK2 puts them in line to collaborate on a late-stage program for DNL151, which is scheduled to start next year.

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Moncef Slaoui, Getty Images

When will it end? Big Phar­ma's top vac­cine ex­pert at OWS of­fers a speedy time­line for a Covid-19 vac­cine — ei­ther be­fore or right af­ter the elec­tion

Moncef Slaoui hasn’t started making plans for his summer vacation next year. But he offers high odds that all Americans will be able to do that in the not too distant future.

In an interview with a pair of sympathetic podcasters at the conservative American Enterprise Institute, Slaoui provides an education to listeners on how any drug or vaccine can be sped through trials. And he leaves the door wide open to the notion that the leading vaccine developers can demonstrate efficacy and safety in a compelling fashion as early as October — or as late as the end of this year.

Levi Garraway, Roche CMO (Source: Genentech)

UP­DAT­ED: FDA hands out a quick OK for po­ten­tial SMA block­buster ris­diplam, giv­ing Genen­tech and Roche a chance to chal­lenge ri­vals on the price

US regulators handed Roche and Genentech a big win Friday afternoon, one that has market-shaping potential for its high-priced rivals from Novartis and Biogen.

The FDA has green-lit the companies’ spinal muscular atrophy drug risdiplam, which will be marketed as Evrysdi in the US, for use in patients two months and older. It’s the first SMA drug that can be taken orally, as Biogen’s Spinraza is injected into the spine while Novartis’ Zolgensma is a gene therapy.

Per­cep­tive fields SPAC #3 as an­oth­er group of biotechs scoops up $364M in lat­est Nas­daq romp

There’s no sign that the windfall of cash dropping biotech’s way on Wall Street is abating. Three more biopharmas priced IPOs on Thursday and Friday morning, riding a historic boom with a $364 million payoff.

London-based biotech Freeline Therapeutics took home the lion’s share of the cash with $159 million after pricing 8,823,529 shares at $18 a pop. Checkmate Pharmaceuticals, of Cambridge, MA, raised $75 million with an offer of 5 million shares at $15 — right at the midpoint of its range. And Arya Sciences Acquisition Corp III, the third in a series from Perceptive, priced 13,000,000 shares at $10 per share.

Covid-19 roundup: Gates Foun­da­tion pours $150M in­to In­dia’s Serum In­sti­tute; Pfiz­er teams with Gilead on remde­sivir

By CEO and scion Adar Poonawalla’s estimation, the Serum Institute in India has already poured hundreds of millions of dollars into scaling up the unproven Covid-19 vaccine being developed by AstraZeneca and Oxford for use in low and middle income countries. It’s meant taking on a risk that other companies, including AstraZeneca, have mitigated with huge amounts of government funding.

Now, for the first time, Poonawalla is getting some outside help. The Gates Foundation has agreed to pay the institute $150 million to supply 100 million vaccines to India and other emerging economies next year, Reuters reported. That includes both the vaccine being developed by AstraZeneca and the one being developed by Novavax. Those vaccines will be available in 92 countries and be priced at $3 per dose.

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In sur­pris­ing set­back, com­bo of Roche’s Tecen­triq and chemo fails to help pa­tients with triple-neg­a­tive breast can­cer

Roche broke ground last year when they secured the first FDA approval for a checkpoint therapy in triple-negative breast cancer, a notoriously difficult-to-treat indication that has been passed over by the wave of targeted therapies.

Now, though, doctors are puzzling over why a combination of drugs meant to make that therapy more potent instead appeared to make it less effective.

Roche said Thursday that in a Phase III trial, combining their PD-1/L1 checkpoint therapy Tecentriq with the chemotherapy paclitaxel, did not significantly improve progression-free survival for patients with locally advanced or metastatic triple-negative breast cancer over giving those patients chemotherapy alone. In fact, patients on the Tecentriq-chemo arm had lower overall survival than patients on chemo, although the drugmaker cautioned that the trial was not powered for that endpoint and the data were immature.

Douglas Fambrough, Dicerna CEO (Boehringer Ingelheim via YouTube)

Roche-backed Dicer­na push­es in­to the pack rac­ing to­ward the block­buster hep B goal line, armed with PhI da­ta

Dicerna has lined up a set of proof-of-concept data from a small cohort of hepatitis B patients in a match-up against some heavyweight rivals which got out in front of this race. And right in the front row you’ll find a team from Roche, which paid $200 million in cash and offered another $1.5 billion in milestones to partner with Dicerna $DRNA on their RNAi program for hep B.

Right now it’s looking competitive, with lots of big challenges ahead.

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Jan Hatzius (Photographer: Christopher Goodney/Bloomberg via Getty Images)

When will it end? Gold­man econ­o­mist gives late-stage vac­cines a good shot at tar­get­ing 'large shares' of the US by mid-2021 — but the down­side is daunt­ing

It took decades for hepatitis B research to deliver a slate of late-stage candidates capable of reining the disease in.

With Covid-19, the same timeline has devoured all of 5 months. And the outcome will influence the lives of billions of people and a multitrillion-dollar world economy.

Count the economists at Goldman Sachs as optimistic that at least one of these leading vaccines will stay on this furiously accelerated pace and get over the regulatory goal line before the end of this year, with a shot at several more near-term OKs. That in turn should lead to the production of billions of doses of vaccines that can create herd immunity in the US by the middle of next year, with Europe following a few months later.

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