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.

At the In­flec­tion Point for the Next Gen­er­a­tion of Can­cer Im­munother­a­py

While oncology researchers have long pursued the potential of cellular immunotherapies for the treatment of cancer, it was unclear whether these therapies would ever reach patients due to the complexity of manufacturing and costs of development. Fortunately, the recent successful development and regulatory approval of chimeric antigen receptor-engineered T (CAR-T) cells have demonstrated the significant benefit of these therapies to patients.

All about Omi­cron; We need more Covid an­tivi­rals; GSK snags Pfiz­er’s vac­cine ex­ec; Janet Wood­cock’s fu­ture at FDA; 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.

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Merck's new antiviral molnupiravir (Quality Stock Arts / Shutterstock)

As Omi­cron spread looms, oral an­tivi­rals ap­pear to be one of the best de­fens­es — now we just need more

After South African scientists reported a new Covid-19 variant — dubbed Omicron by the WHO — scientists became concerned about how effective vaccines and monoclonal antibodies might be against it, which has more than 30 mutations in the spike protein.

“I think it is super worrisome,” Dartmouth professor and Adagio co-founder and CEO Tillman Gerngross told Endpoints News this weekend. Moderna CEO Stéphane Bancel echoed similar concerns, telling the Financial Times that experts warned him, “This is not going to be good.”

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Pfiz­er, Am­gen and Janssen seek fur­ther clar­i­ty on FDA's new ben­e­fit-risk guid­ance

Three top biopharma companies are seeking more details from the FDA on how the agency conducts its benefit-risk assessments for new drugs and biologics.

While Pfizer, Amgen and Janssen praised the agency for further spelling out its thinking on the subject in a new draft guidance, including a discussion of patient experience data as part of the assessment, the companies said the FDA could’ve included more specifics in the 20-page draft document.

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Lisa Deschamps, AviadoBio CEO

Ex-No­var­tis busi­ness head hops over to a gene ther­a­py start­up — and she's reeled in $80M for a dash to the clin­ic

Neurologist and King’s College London professor Christopher Shaw has been researching neurodegenerative diseases like ALS and collaborating with drugmakers for the last 25 years in the hopes of pushing new therapies forward. But unfortunately, none of those efforts have come anywhere close to fruition.

“So, you know, after 20 years in the game, I said, ‘Let’s try and do it ourselves,’” he told Endpoints News. 

Vas Narasimhan, Novartis CEO (Thibault Camus/Pool via AP Images)

With gener­ic com­pe­ti­tion heat­ing up, Vas Narasimhan out­lines No­var­tis' growth plans at R&D day

Thursday marks Novartis’ annual R&D day, and with it comes CEO Vas Narasimhan’s attempt to spotlight the company’s pipeline strategy and emerging stars.

The biggest question entering Thursday’s presentation dealt with how the big biopharma will make up revenues from upcoming generic competition — Novartis says within the next five years, generics will eat away roughly $9 billion in sales. To offset this, Narasimhan outlined a strategy for 4% growth or higher until 2026, focusing on six key medicines he believes will see multibillion dollar profits during this time.

In­cor­po­rat­ing Ex­ter­nal Da­ta in­to Clin­i­cal Tri­als: Com­par­ing Dig­i­tal Twins to Ex­ter­nal Con­trol Arms

Most drug development professionals are familiar with the nerve-racking wait for the read-out of a large trial. If it’s negative, is the investigational therapy ineffective? Or could the failure result from an unforeseen flaw in the design or execution of the protocol, rather than a lack of efficacy? The team could spend weeks analyzing data, but a definitive answer may be elusive due to insufficient power for such analyses in the already completed trial. These problems are only made worse if the trial had lower enrollment, or higher dropout than expected due to an unanticipated event like COVID-19. And if a trial is negative, the next one is likely to be larger and more costly — if it happens at all.

Reshma Kewalramani, Vertex CEO (Vertex via YouTube)

Bat­tling a line­up of skep­tics, Ver­tex claims an­oth­er ear­ly clin­i­cal win — this time in kid­ney dis­ease

Vertex claimed its second early-stage win of the fall Wednesday, announcing positive results in a small study on a genetically defined form of kidney disease.

The 16-patient, Phase II trial focused on patients with focal segmental glomerulosclerosis, a rare disease where kidneys are unable to filter blood properly. Over 13 weeks on an experimental pill, the level of protein in the patients’ urine fell by an average of 47.6%.

Ab­b­Vie tacks on a new warn­ing to Rin­voq la­bel as safe­ty frets crimp JAK class

The safety problems that continue to plague the JAK class as new data highlight some severe side effects are casting a large shadow over AbbVie’s Rinvoq.

As a result of a recent readout highlighting major adverse cardiac events (MACE), malignancy, mortality and thrombosis with Xeljanz a couple of months ago, AbbVie put out a notice late Friday afternoon that it is adding the new class risks to its label for their rival drug.

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