In­cyte keeps rolling on top­i­cal cream for JAK in­hibitor, pass­ing two PhI­II tests in vi­tili­go

As In­cyte pre­pares to po­ten­tial­ly hit the mar­ket with a top­i­cal for­mu­la­tion of its cash cow rux­oli­tinib in atopic der­mati­tis, the Wilm­ing­ton, DE-based com­pa­ny is beef­ing up its da­ta pack­age for an­oth­er in­di­ca­tion: vi­tili­go.

In­cyte re­leased Phase III re­sults from two of its clin­i­cal vi­tili­go pro­grams Mon­day morn­ing, say­ing both stud­ies met their pri­ma­ry end­points of pa­tients achiev­ing at least 75% im­prove­ment from base­line in repig­men­ta­tion of the face. The da­ta will like­ly lead In­cyte to ask for ap­proval in both the US and Eu­rope for those old­er than 12 be­fore the end of the year.

The FDA is cur­rent­ly re­view­ing the rux­oli­tinib cream in an NDA for AD, which re­ceived pri­or­i­ty re­view thanks to a vouch­er used by In­cyte. In­cyte sub­mit­ted the NDA in De­cem­ber, and the PDU­FA date for AD is June 21.

Mon­day’s re­sults come from the two tri­als that looked at 300 pa­tients each, re­quir­ing in­di­vid­u­als to meet cer­tain in­clu­sion cri­te­ria for how much of their skin was af­fect­ed by the con­di­tion. They were ran­dom­ized in­to two arms, one us­ing 1.5% rux­oli­tinib cream twice a day and the oth­er uti­liz­ing a con­trol for 24 weeks. Both stud­ies hit sparkling p-val­ues of p<0.0001.

Jim Lee

The 75% num­ber is mea­sured by a scor­ing in­dex sim­i­lar to those used in oth­er skin con­di­tions like eczema and pso­ri­a­sis, in­flam­ma­tion and au­toim­mu­ni­ty chief Jim Lee told End­points News. That fig­ure rep­re­sents the point at which both pa­tients feel a mean­ing­ful ben­e­fit and re­searchers see less “back­ground noise” in re­gards to a place­bo ef­fect, he said.

Sec­ondary end­points in the stud­ies in­clud­ed pa­tients who saw at least 50% and 90% im­prove­ment from base­line, two oth­er bench­marks that had been de­bat­ed in the past, and In­cyte hit sta­tis­ti­cal sig­nif­i­cance in these too.

Rux­oli­tinib, sold as Jakafi in the US and Jakavi by No­var­tis else­where, has long been a mon­ey mak­er for In­cyte. Orig­i­nal­ly ap­proved in 2011 to treat myelofi­bro­sis, the drug is a JAK in­hibitor tar­get­ing JAK1 and JAK2. Since then, it gained the FDA OK in poly­cythemia ve­ra in 2014 and acute graft-ver­sus-host dis­ease in 2019.

But the top­i­cal ver­sion has rep­re­sent­ed a new push for rux­oli­tinib in­to ar­eas that present much dif­fer­ent­ly than its oth­er in­di­ca­tions. Where­as the first three ar­eas all have to do with blood can­cers and trans­plant re­jec­tions, AD and vi­tili­go are skin con­di­tions, the lat­ter of which can re­sult in pa­tients’ loss of skin pig­men­ta­tion in many parts of the body.

For In­cyte, it didn’t make sense to de­vel­op oral rux­oli­tinib in these in­di­ca­tions, Lee said. The top­i­cal for­mu­la­tion can be ap­plied di­rect­ly to the af­fect­ed ar­eas while lim­it­ing the ex­po­sure that oth­er ad­min­is­tra­tion meth­ods might have had.

Ad­di­tion­al­ly, this cream is on­ly for use in mild to mod­er­ate cas­es of vi­tili­go, where af­fect­ed ar­eas make up about 10% to 20% of a pa­tient’s body. In AD, that al­so might trans­late in­to some­thing where pa­tients’ on­ly af­fect­ed ar­eas are on their el­bows or be­hind their knees, Lee added. And there’s the com­mon JAK de­nom­i­na­tor in­volved in all of the dis­eases, too.

Re­gard­ing the se­vere cas­es, “It’s not very prac­ti­cal to lath­er a cream all over your body twice a day for an ex­tend­ed pe­ri­od of time,” Lee said. “But we know that 90% of pa­tients or more have lim­it­ed dis­ease, and cream you can ap­ply to those lim­it­ed ar­eas is re­al­ly a very sen­si­ble, prac­ti­cal ap­proach.”

In­cyte had long been strug­gling to de­vel­op new drugs, with Jakafi be­ing its on­ly ap­proved prod­uct through the end of 2019. But last year came two new ap­provals — Pe­mazyre for bile duct can­cer, and the Mor­phoSys-part­nered Mon­ju­vi for re­frac­to­ry dif­fuse large B-cell lym­phoma.

Un­pack­ing the Aduhelm de­ci­sion, Ver­tex's half full glass, a $525M J&J breakup, and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

By now you have surely read about the FDA’s controversial approval of Biogen’s Alzheimer’s drug and all its reverberations. But I’d still recommend checking out the meaty recap below to make sure you didn’t miss all the angles that the Endpoints team has covered. If you’d rather look ahead, look no further than our three-day virtual panels next week at BIO, where we will discuss what the new normal means for every part of the industry.

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What does a clear ma­jor­i­ty of the bio­phar­ma in­dus­try think of the FDA ap­proval of ad­u­canum­ab? 'Hor­ri­fy­ing' 'Dan­ger­ous' 'Con­fus­ing' 'Dis­as­ter'

Over the years, we’ve become used to seeing a consensus emerge early in our industry polls at Endpoints News. And when we took the pulse of drug hunters on the heels of a controversial FDA approval for aducanumab this week, it became immediately apparent that the vast majority of our readers — heavily concentrated among biopharma staffers and execs — were incensed by what they had just witnessed.

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Aaron Kesselheim (Scott Eisen/AP Images for AIDS Healthcare Foundation)

Har­vard’s Aaron Kessel­heim re­signs from ex­pert pan­el in wake of ad­u­canum­ab OK, blast­ing FDA for ‘worst drug ap­proval de­ci­sion in re­cent U.S. his­to­ry'

A third member of the FDA’s Peripheral and Central Nervous System Drugs Advisory Committee has resigned in the wake of Biogen’s controversial Aduhelm approval, slamming the agency as he left and further deepening the controversy surrounding the decision.

Harvard University professor Aaron Kesselheim quit in protest Thursday afternoon, calling the Aduhelm OK “probably the worst drug approval decision in recent U.S. history.” Kesselheim follows both Joel Perlmutter, a neurologist from Washington University in St. Louis, and David Knopman, a neurologist from the Mayo Clinic, out the door.

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David Knopman (Mayo Clinic via YouTube)

A sec­ond ad­comm mem­ber aban­dons his post in af­ter­math of con­tro­ver­sial ad­u­canum­ab de­ci­sion

As the fallout from the FDA’s approval of Alzheimer’s med aducanumab grows, a second member of the adcomm overseeing that drug’s review has walked away. But even with two experts now having resigned from that committee in protest, is there enough broad-level outrage to prevent another aducanumab from getting approved?

The FDA on Wednesday lost another member of its Peripheral and Central Nervous System Drugs Advisory Committee as Mayo Clinic neurologist David Knopman hit the exit over the agency’s decision to approve Biogen’s Alzheimer’s drug Aduhelm despite the committee’s near-unanimous vote against it.

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Reshma Kewalramani, Vertex CEO (BIO via YouTube)

UP­DAT­ED: Ver­tex strikes out on its lat­est big shot at a rare ge­net­ic dis­ease. But they're go­ing to keep on swing­ing

It’s been several months since Vertex culled one of its small molecules for alpha-1 antitrypsin deficiency (AATD), taking a big hit after evidence of liver damage surfaced in a key Phase II trial. Now we learned that the company has whiffed on its second shot, and there’s nothing left in the clinic to treat the rare genetic disease — but that won’t stop it from trying.

Despite avoiding the safety issues that plagued the last candidate, Vertex $VRTX is taking the axe to VX-864 after Phase II results revealed the magnitude of the drug’s response is “unlikely to translate into substantial clinical benefit.” As a result of the news, the company’s stock fell 12.5% after hours.

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FDA au­tho­rizes about 10M J&J vac­cine dos­es, trash­es 60M more from trou­bled Emer­gent plant

The FDA on Friday released about 10 million doses of J&J’s vaccine for use, and disposed of another 60 million doses that were manufactured at the now-shuttered Emergent BioSolutions facility in Baltimore where cross-contamination occurred.

The agency said it’s not yet ready to allow the Emergent plant to be included in the J&J EUA, but that may occur soon. FDA came to the decision to authorize some of the doses after reviewing facility records and quality testing results.

Paul Hudson, Sanofi CEO (Eric Piermont/AFP via Getty Images)

Months af­ter FDA re­jec­tion, Sanofi touts piv­otal win for rare dis­ease drug su­tim­limab as it preps to re­file

One of the pillar drugs of Sanofi’s $11.6 billion pickup of Bioverativ hit a big setback late last year when the FDA sent its application for approval back. Now, as Sanofi gears up to resubmit the drug for review, the drugmaker is touting pivotal data it hopes will help take it over the finish line.

Sanofi’s sutimlimab nailed all three of its primary endpoints in its Phase III CADENZA study for patients with cold agglutinin disease, a rare disorder that can cause severe anemia, without a recent history of blood transfusion, the French drugmaker said Friday. The topline results will be presented at this weekend’s virtual EHA meeting.

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Ver­tex and CRISPR Ther­a­peu­tics un­veil more pos­i­tive gene ther­a­py da­ta, but busul­fan again casts a shad­ow over the field

Less than 12 hours after revealing a flop on its second shot for alpha-1 antitrypsin deficiency, Vertex plowed ahead with another data drop from its partnership with CRISPR Therapeutics. And though the topline proved positive, concerns over conditioning agents continue to linger over the collaboration, as well as the entire gene therapy space.

Presenting data from two trials at the European Hematology Association annual meeting, the pair announced that follow-up data of at least three months for 22 patients with genetic blood disorders indicated a “consistent and sustained” response to the experimental drug CTX001. All 15 patients with transfusion-dependent beta thalassemia did not need further blood transfusions and all seven with severe sickle cell disease were pain free, the biotechs announced.

Janet Woodcock, acting FDA commissioner, at Thursday's Senate Appropriations hearing (Bill Clark/CQ Roll Call via AP Images)

Sen­a­tors lam­bast new Alzheimer’s drug’s price but give Janet Wood­cock a free pass on the ap­proval de­ci­sion

Senate Finance Democrats took aim at Biogen’s pricey new Alzheimer’s drug on Thursday, but members on both sides of the aisle at a separate appropriations hearing didn’t question acting FDA commissioner Janet Woodcock on the approval.

“I was appalled that Biogen priced their Alzheimer’s drug approved by the FDA at $56,000 per year — I’m not going to debate whether this is effective or not, but it’s double the household median income for Michiganders over the age of 65,” Sen. Debbie Stabenow (D-MI) said at the finance hearing.