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.

Grow­ing ac­cep­tance of ac­cel­er­at­ed path­ways for nov­el treat­ments: but does reg­u­la­to­ry ap­proval lead to com­mer­cial suc­cess?

By Mwango Kashoki, MD, MPH, Vice President-Technical, and Richard Macaulay, Senior Director, of Parexel Regulatory & Access

In recent years, we’ve seen a significant uptake in the use of regulatory options by companies looking to accelerate the journey of life-saving drugs to market. In 2018, 73% of the novel drugs approved by the U.S. Federal Drug Administration (FDA) were designated under one or more expedited development program categories (Fast Track, Breakthrough Therapy, Priority Review, and Accelerated Approval).ᶦ

Sanofi out­lines big API plans as coro­n­avirus out­break re­port­ed­ly threat­ens short­age of 150 drugs

As the world becomes increasingly dependant on Asia for the ingredients of its medicines, Sanofi sees business to be done in Europe.

The French drugmaker said it’s creating the world’s second largest active pharmaceutical ingredients (API) manufacturer by spinning out its six current sites into a standalone company: Brindisi (Italy), Frankfurt Chemistry (Germany), Haverhill (UK), St Aubin les Elbeuf (France), Újpest (Hungary) and Vertolaye (France). They have mapped out €1 billion in expected sales by 2022 and 3,100 employees for the new operations headquartered in France.

UP­DAT­ED: NGM Bio takes leap for­ward in crowd­ed NASH field

South San Francisco-based NGM Bio may have underwhelmed with its interim analysis of a key cohort from a mid-stage NASH study last fall — but stellar topline data unveiled on Monday showed the compound induced significant signs of antifibrotic activity, NASH resolution and liver fat reduction, sending the company’s stock soaring.

There are an estimated 50+ companies focused on developing drugs for non-alcoholic steatohepatitis, or NASH, a common liver disease that has long flummoxed researchers. The first wave of NASH drug developers struggled with efficacy as well as safety — and companies big and small have crashed and burned.

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Mickey Kertesz, KidsandArtOrg via YouTube

Soft­Bank's newest, $165M biotech in­vest­ment looks for in­fec­tious traces in the blood

SoftBank has found its newest biotech investment.

The Japanese bank has invested $165 million into Karius, a company that uses blood tests to diagnose infectious diseases, as part of its new Vision Fund 2. The full scope of the new fund has yet to be announced, but the first and newly-beleaguered Vision Fund poured $100 billion into technology companies, including the biotechs Vir Biotechnology and Roivant and the sequencing company 10x Genomics.

Methicillin-resistant Staph aureus (Shutterstock)

FDA grants ‘break­through’ sta­tus to an­tibi­ot­ic al­ter­na­tive as Con­tra­Fect rush­es to join fight against su­per­bug

An experimental drug that promises to be the first anti-infective agent to prove superior to vancomycin — an antibiotic approved in 1958 — has notched the FDA’s “breakthrough” status.

ContraFect said the designation was based on Phase II data in which exebacase was tested against a superbug known as methicillin-resistant Staph aureus, or MRSA. In a subgroup analysis, the clinical responder rate at day 14 was 42.8% higher than that among those treated with standard of care, the company said (p=0.010).

Zhong Nanshan, CGTN via YouTube

Har­vard joins coro­n­avirus fight with $115 mil­lion and a high-pro­file Chi­nese part­ner

For two months, as the novel coronavirus swelled from a few early cases tied to a Wuhan market to a global epidemic, most of the world’s focus and dollars have flowed toward emergency initiatives: building vaccines at a record pace, plucking experimental antivirals out of freezers to see what sticks and immunizing mice for new antibodies.

Now a new and well-funded collaboration between Harvard and a top Chinese research institute will play the long game. In a 5-year, $115 million initiative backed by China Evergrande Group, researchers from the Harvard Medical School, Harvard T.H. Chan School of Public Health and Guangzhou Institute for Respiratory Health will study the virus in an effort to develop therapies against infections by the novel coronavirus, known as SARS–CoV-2, and to prevent new ones.

No­var­tis gets a boost in block­buster mul­ti­ple scle­ro­sis race with Roche

In the first step of what’s likely to be a long and uphill battle for the drugmaker, the FDA has accepted Novartis’s BLA submission for a new multiple sclerosis drug and given it priority review. The PDUFA date for the potential blockbuster drug is in June.

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Juergen Horn

An­i­mal health vet Juer­gen Horn makes new an­ti­body play for pets, rak­ing $15M in Se­ries A haul

Zoetis forked over $85 million in 2017 to acquire Nexvet Biopharma and its pipeline of monoclonal antibodies. Juergen Horn, Nexvet’s former chief product development officer, has now secured $15 million for his own biologic company for animals: Invetx.

Buoyed by emerging advances in gene therapies for humans, scientists have started looking at harnessing the technology for animals setting up companies such as Penn-partnered Scout Bio and George Church-founded Rejuvenate Bio. But akin to Nexvet, Invetx is working on leveraging the time-tested science of monoclonal antibodies to treat chronic diseases that afflict man’s best friend.

As coro­n­avirus out­break reach­es 'tip­ping point,' GSK lends ad­ju­vant tech to Chi­nese part­ner armed with pre­clin­i­cal vac­cine

As the coronavirus originating out of Wuhan spreads to South Korea, Italy and Iran, stoking already intense fears of a pandemic, GlaxoSmithKline has found another pair of trusted hands to place its adjuvant system. China’s Clover Biopharmaceuticals will add the adjuvant to its preclinical, protein-based vaccine candidate against SARS-CoV-2.

Clover, which is based in the inland city of Chengdu, boasts of a platform dubbed Trimer-Tag that produces covalently-trimerized fusion proteins. Its candidate, COVID-19 S-Trimer, resembles the viral spike (S)-protein found in the virus.