In­cyte's rux­oli­tinib scores its sec­ond ap­proval in two days, this time in chron­ic graft-ver­sus-host dis­ease

A day af­ter ap­prov­ing a top­i­cal for­mu­la­tion of In­cyte’s cash cow rux­oli­tinib for atopic der­mati­tis, reg­u­la­tors are back with more good news for the com­pa­ny’s fa­vorite JAK in­hibitor.

The FDA has giv­en Jakafi, the oral ver­sion of the drug, the green light to treat chron­ic graft-ver­sus-host dis­ease af­ter one or two lines of sys­temic ther­a­py, In­cyte said Wednes­day, mark­ing the drug’s sec­ond ap­proval in just as many days.

GVHD is a con­di­tion that oc­curs af­ter some al­lo­gene­ic stem cell trans­plants, in which the donor stem cells view the host as for­eign and at­tack. Jakafi won ap­proval to treat the acute form of the dis­ease back in 2019, which typ­i­cal­ly oc­curs with­in 100 days of the trans­plant and can cause rash­es, dis­col­oration and vom­it­ing. But the chron­ic form usu­al­ly comes lat­er than 100 days and can be worse, trig­ger­ing symp­toms like short­ness of breath, pain, mus­cle weak­ness and more. Both forms can be dead­ly, and can af­fect mul­ti­ple or­gan sys­tems.

It’s es­ti­mat­ed that more than 14,000 peo­ple in the US are liv­ing with GVHD, and around half don’t re­spond to steroids, leav­ing few treat­ment op­tions.

“This ap­proval rep­re­sents a sig­nif­i­cant ad­vance­ment in the treat­ment of ap­pro­pri­ate pa­tients with chron­ic GVHD — for both the pa­tients who face a poor prog­no­sis and the health­care providers who strug­gle to ef­fec­tive­ly treat them,” prin­ci­pal in­ves­ti­ga­tor Robert Zeis­er said in a state­ment.

In the Phase III REACH3 study, Jakafi achieved an over­all re­sponse rate of 49.7% at Week 24, com­pared to 25.6% in a group of pa­tients giv­en the best avail­able ther­a­py (p<0.0001). The ORR af­ter six full treat­ment cy­cles was 70% for Jakafi com­pared to 57% for BAT, ac­cord­ing to In­cyte.

The most com­mon side ef­fects rat­ed Grade 3 or high­er were throm­bo­cy­tope­nia (a low blood platelet count), ane­mia, neu­trope­nia (low­er-than-nor­mal lev­els of neu­trophils, a type of white blood cell), and pneu­mo­nia, ac­cord­ing to full re­sults pub­lished back in Ju­ly. How­ev­er, deaths re­port­ed as pri­mar­i­ly due to chron­ic GVHD com­pli­ca­tions and/or its treat­ment were high­er in the Jakafi arm (13.3%, ver­sus 7.9% in the con­trol arm), In­cyte said.

Rux­oli­tinib is In­cyte’s great­est rev­enue stream — bring­ing in near­ly $2 bil­lion in 2020 — and has kept In­cyte sail­ing over the years de­spite set­backs across their pipeline. It’s al­so ap­proved for poly­cythemia ve­ra (PV) in adults who have had an in­ad­e­quate re­sponse to or are in­tol­er­ant of hy­drox­yurea, in­ter­me­di­ate or high-risk myelofi­bro­sis (MF), and — as of yes­ter­day — mild to mod­er­ate atopic der­mati­tis.

The drug will now go up against Kad­mon’s Rezurock, which scored an OK in chron­ic GVHD back in Ju­ly — six weeks be­fore its PDU­FA date. Up­on re­leas­ing the first glimpse at piv­otal da­ta back in 2019, Kad­mon framed their drug as one of a grow­ing num­ber of op­tions pa­tients might use, not­ing that many of the pa­tients in the tri­al didn’t re­spond to In­cyte’s Jakafi or Ab­b­Vie and J&J’s Im­bru­vi­ca.

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.

Stéphane Bancel, Moderna CEO

'This is not go­ing to be good': Mod­er­na CEO Ban­cel warns of a 'ma­te­r­i­al drop' in vac­cine ef­fi­ca­cy as Omi­cron spreads

Even as public health officials remain guarded about their comments on the likelihood Omicron will escape the reach of the currently approved Covid-19 vaccines, there’s growing scientific consensus that we’re facing a variant that threatens to overwhelm the vaccine barricades that have been erected.

Stéphane Bancel, the CEO of Moderna, one of the leading mRNA players whose quick vault into the markets with a highly effective vaccine created an instant multibillion-dollar market, added his voice to the rising chorus early Tuesday. According to Bancel, there will be a significant drop in efficacy when the average immune system is confronted by Omicron. The only question now is: How much?

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Philip Dormitzer, new GSK global head of vaccines R&D

Glax­o­SmithK­line poach­es Pfiz­er's vi­ral vac­cines lead in rush to cap­i­tal­ize on fu­ture of mR­NA

GlaxoSmithKline has appointed Philip Dormitzer, formerly chief scientific officer of Pfizer’s viral vaccines unit, as its newest global head of vaccines R&D, looking to leverage one of the leading minds behind Pfizer and BioNTech’s RNA collaboration that led to Covid-19 jab Comirnaty, the British drug giant said Tuesday.

Dormitzer had been with Pfizer for a little more than six years, joining up after a seven-year stint with Novartis, where he reached the role of US head of research and head of global virology for the company’s vaccines and diagnostics unit.

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In­tro­duc­ing End­points Stu­dio, a new way to ad­ver­tise with End­points-craft­ed brand­ing cam­paigns

Since our start in 2016, Endpoints has grown fast while executing our mission to cover biopharma’s most critical developments for industry pros worldwide. As readership has grown, our advertising business has too. Endpoints advertising partners support the mission and engage their desired audiences through announcements on our email and web platforms, brand recognition in our event coverage and sponsorships of Endpoints daily and weekly reports.

Lan Huang, BeyondSpring CEO

Months af­ter shock­ing in­vestors with lung can­cer win, Be­yond­Spring's lead drug hits road­block at the FDA

BeyondSpring shocked investors in early August after its once-marginal lead drug suddenly showed a lot of promise in a common form of lung cancer. With hopes high, the FDA has now slammed the door on that drug in another indication — does that spell bad news for BeyondSpring’s Cinderella story?

The FDA issued BeyondSpring a complete response letter for its plinabulin in combination with granulocyte colony-stimulating factor (G-CSF) for the prevention of chemotherapy-induced neutropenia, effectively shutting down the drug’s immediate chances at a marketing approval, the biotech said Wednesday.

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With on­ly burns to show in gene ther­a­py, Astel­las inks deal with AAV spe­cial­ist Dyno in push for a bet­ter cap­sid

On the hunt for a better AAV capsid for gene therapy, Eric Kelsic’s Dyno Therapeutics has set itself apart with its focus on machine learning to help speed discovery. Now, Japanese drugmaker Astellas — fresh off a slate of gene therapy burns — is taking a bet on Dyno as it looks to the future.

Astellas and Dyno will work together as part of an R&D pact to develop next-gen AAV vectors for gene therapy using Dyno’s CapsidMap platform directed at skeletal and cardiac muscle, the companies said Wednesday. Under the terms of the deal, Dyno will design AAV capsids for gene therapy, while Astellas will be responsible for conducting preclinical, clinical and commercialization activities for gene therapy product candidates using the capsids.

J&J and Sanofi's mul­ti­ple myelo­ma tit-for-tat con­tin­ues, as sub­cu­ta­neous Darza­lex wins com­bo ap­proval

J&J and Sanofi have gone back and forth in their multiple myeloma tug-of-war. Earlier this year, Sanofi notched an approval of Sarclisa in combination with Amgen’s Kyprolis to try to outflank the big conglomerate, but J&J is clapping back.

Wednesday afternoon, Amgen announced that the subcutaneous version of J&J’s blockbuster Darzalex is also now approved as a combo with Kyprolis and dexamethasone. The green light came through for adults with relapsed or refractory multiple myeloma who had progressed on one to three earlier lines of therapy.

As first Omi­cron case in US crops up, re­searchers won­der: which an­ti­bod­ies, vac­cines will hold up?

As Covid-19 drug and vaccine developers race to figure out which of their products might be hampered by the new variant, the CDC on Wednesday afternoon announced the first confirmed case of the Omicron variant (B.1.1.529) in the US, found in San Francisco.

The unidentified individual was a traveler who returned from South Africa on Nov. 22, 2021, was fully vaccinated, and had mild symptoms that the CDC described as improving. All close contacts have been contacted and have tested negative, the centers said.

FDA ad­comm nar­row­ly votes in fa­vor of Mer­ck­'s an­tivi­ral for out­pa­tient Covid-19

With little explanation for why Merck’s potential Covid-19 antiviral was less effective in reducing Covid hospitalizations and deaths in a full analysis of a Phase III trial versus an interim look, the FDA’s antimicrobial drugs advisory committee on Tuesday voted 13-10 in favor of the pill’s benefits outweighing the risks for adults within 5 days of developing Covid symptoms.

Molnupiravir will likely be authorized by FDA in the coming days for adults with mild or moderate Covid-19. While Pfizer’s antiviral may prove to be more effective, Merck’s pill will be another weapon in the armamentarium of Covid-19 treatments for countries around the world, adding to the mAb treatments already in use in the outpatient space from Regeneron, Eli Lilly and Vir/GlaxoSmithKline.