Flemming Ørnskov, Galderma CEO

UP­DAT­ED: Gal­der­ma drug pass­es PhI­II in rare skin dis­ease, set­ting the stage for ap­proval re­quest

Der­ma­tol­ogy be­he­moth Gal­der­ma is one step clos­er to reg­u­la­tors’ doorsteps for a mon­o­clon­al an­ti­body it li­censed from Chugai Phar­ma­ceu­ti­cal af­ter a Phase III read­out Wednes­day.

The for­mer Nestlé Skin Health unit said that the drug, named nemolizum­ab, passed muster in one of two Phase III tri­als in the rare skin dis­ease pruri­go nodu­laris. The drug, test­ed on its own, was able to clear most skin le­sions and re­duce itch­ing in pa­tients with the rare skin dis­ease.

The Cetaphil mak­er will pack­age the re­sults and sub­mit to reg­u­la­tors once gath­er­ing da­ta from the sec­ond Phase III study, CEO Flem­ming Ørn­skov told End­points News in an email. The for­mer Shire CEO de­clined to dis­close when the sec­ond part of the OLYMPIA piv­otal pro­gram would read out in the rare in­flam­ma­to­ry dis­ease, which is de­void of any ap­proved ther­a­pies.

“If ap­proved, nemolizum­ab would be a con­ve­nient ther­a­py with a month­ly in­jec­tion as op­posed to some of the ex­ist­ing bi­o­log­ic ther­a­pies which re­quire bi­week­ly in­jec­tions,” the CEO said.

Gal­der­ma is al­so test­ing nemolizum­ab in atopic der­mati­tis. The Phase III AD tri­als, un­der the clin­i­cal pro­gram um­brel­la AR­CA­DIA, are ex­pect­ed to com­plete in late 2022 or the first half of 2023, Ørn­skov said. An­oth­er long-run­ning biotech work­ing to get in­to the AD space re­ceived FDA ap­proval last De­cem­ber, as Leo Phar­ma won a nod for an IL-13-tar­get­ing drug, named Ad­bry.

Af­ter 16 weeks of treat­ment, 38% of pa­tients treat­ed with Gal­der­ma’s in­ves­ti­ga­tion­al med saw skin le­sion clear­ance or al­most-clear­ance as com­pared to 11% in the place­bo group. On the itch­ing im­prove­ment, Gal­der­ma’s med led to a four-point re­duc­tion or greater in itch­ing in 56% of nemolizum­ab-treat­ed pa­tients com­pared to 21% in the con­trol co­hort.

All key sec­ondary end­points were al­so achieved, the Swiss der­ma­tol­ogy com­pa­ny said Wednes­day. Pa­tients re­ceived two sub­cu­ta­neous in­jec­tions at base­line and then ei­ther one or two in­jec­tions, de­pend­ing on weight, every four weeks across 16 weeks.

Gal­der­ma is al­so in­ves­ti­gat­ing the drug in mul­ti­ple mid- and late-stage tri­als in atopic der­mati­tis.

Orig­i­nal­ly de­vel­oped by Chugai Phar­ma­ceu­ti­cal, the an­ti-IL-31 mAb re­ceived ap­proval in Japan in March for treat­ing pru­ri­tus (itch­ing) as­so­ci­at­ed with atopic der­mati­tis. Japan­ese biotech Maruho is mar­ket­ing the drug as Mitch­ga. Maruho has Japan and Tai­wan rights to the Chugai drug and Gal­der­ma has the ex­clu­sive glob­al li­cense else­where un­der a Ju­ly 2016 deal, for which fi­nan­cials were not dis­closed.

The Phase III re­sults prop up the strat­e­gy be­hind Gal­der­ma’s in­creased fo­cus on R&D since spin­ning out of Nestlé. With “dis­rup­tive po­ten­tial,” nemolizum­ab is cen­tral to the com­pa­ny’s bi­o­log­ics fu­ture, Ørn­skov said.

Ed­i­tor’s note: This sto­ry was up­dat­ed to clar­i­fy that Leo Phar­ma’s Ad­bry is an IL-13 and Gal­der­ma’s mon­o­clon­al an­ti­body tar­gets IL-31.

The Fac­tors Dri­ving a Rapid Evo­lu­tion of Gene & Cell Ther­a­py and CAR-T Clin­i­cal Re­search in APAC

APAC is the fastest growing region globally for cell & gene therapy trials representing more than a third of all cell & gene studies globally, with China leading in the region. 

APAC is the leading location globally for CAR-T trials with China attracting ~60% of all CAR-T trials globally between 2015-2022. The number of CAR-T trials initiated by Western companies has rapidly increased in recent years (current CAGR of about 60%), with multiple targets being explored including CD19, CD20, CD22, BCMA, CD30, CD123, CD33, CD38, and CD138.

The End­points 11; blue­bird's $3M gene ther­a­py; Bio­gen tout new neu­ro da­ta; Harsh re­views for can­cer drugs; 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.

Reading about John Carroll’s pick of biotech’s most promising startups has become a treasured tradition. If you ever get curious about previous classes of the Endpoints 11, you can find all of them (plus a number of our other regular specials) here.

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EMA warns of short­ages of two Boehringer heart drugs due to a spike in de­mand

The EMA is putting EU member states on alert over the shortage of two drugs that counter heart attacks due to an uptick in demand.

On Friday, the EMA sent out a warning that two Boehringer Ingelheim drugs are experiencing a shortage: Actilyse and Metalyse. The drugs are used as emergency treatments for adults experiencing acute myocardial infarction, or a heart attack, by dissolving blood clots that have formed in the blood vessels.

The End­points 11: The top pri­vate biotechs in pur­suit of new drugs. Push­ing the en­ve­lope with pow­er­ful new tech­nolo­gies

Right around the beginning of the year, we got a close-up look at what happens after a boom ripples through biotech. The crash of life sciences stocks in Q1 was heard around the world.

In the months since, we’ve seen the natural Darwinian down cycle take effect. Reverse mergers made a comeback, with more burned out shells to go public at a time IPOs and road shows are out of favor. And no doubt some of the more recent arrivals on the investing side of the business are finding greener pastures.

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Mene Pangalos (AstraZeneca via YouTube)

As­traZeneca shuts the PhI­II door for Ion­is' PC­SK9 drug de­spite pos­i­tive PhI­Ib

When Ionis and AstraZeneca unveiled the first round of mid-stage data for their antisense PCSK9 drug, Mene Pangalos, AstraZeneca’s EVP of biopharmaceuticals R&D, underscored the drug’s “potential best-in-class efficacy profile.”

But now that the second batch is in, it appears AZD8233 isn’t hitting the mark after all.

Ionis announced Friday morning that although the candidate, also dubbed ION449, met the primary endpoint in the Phase IIb SOLANO trial, its partners at AstraZeneca have decided not to move it into Phase III studies because the “results did not achieve pre-specified efficacy criteria.”

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Up­dat­ed: Bio­gen throws it­self back in­to mud­dled da­ta ar­gu­ments with more de­tails on its an­ti­sense ALS drug

With a highly watched FDA decision deadline coming in late January, Biogen and Ionis dropped the full data on the Phase III study of their ALS drug tofersen in the New England Journal of Medicine on Wednesday.

Biogen is looking for approval for tofersen in a very small subset of ALS patients — some 2%, according to the paper — who have a SOD1 gene mutation, which has previously been linked to ALS. Tofersen is meant to reduce levels of mutant SOD1 proteins.

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As­traZeneca, Mer­ck cull one Lyn­parza in­di­ca­tion in heav­i­ly pre­treat­ed ovar­i­an can­cer pa­tients

Just one day after blockbuster Lynparza got access to another indication in China, its Big Pharma owners have decided to withdraw it in certain patients after reviewing Phase III data.

The two companies that work together on Lynparza decided to recall one of the indications several weeks ago in a specific type of ovarian cancer, Lynparza’s first indication when it was first FDA-approved in 2014. Initial data showed that rates of overall survival in patients with at least three rounds of chemo before getting on the PARP inhibitor were lower than in patients with less previous chemo treatment.

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Solicitor General Elizabeth Prelogar

Should SCO­TUS hear Am­gen's Repatha case? So­lic­i­tor gen­er­al says no

Back in April, Amgen said it was encouraged by the solicitor general’s anticipated review of its Supreme Court petition to rehear a Repatha patent case. They’re likely much less optimistic about the outcome now.

Solicitor General Elizabeth Prelogar wrote in a recent 27-page brief that Amgen’s arguments “lack merit and further review is not warranted.”

The case traces back to a suit filed in 2014 against Sanofi and Regeneron’s Praluent, which ended up beating Amgen’s PCSK9 blockbuster Repatha to market by a month just a year later.

Phil Sharp, Nobel Prize laureate (L), and John Carroll, Endpoints News co-CEO (via Michael Last)

The End­points 11: Fire­side chat with No­bel Prize lau­re­ate Phil Sharp

On Thursday evening in Boston I had the great good fortune to talk about the creation of the biotech industry with Nobel Prize-winning scientist Phil Sharp. I learned quite a bit about the early days of Genentech, Biogen and Alnylam, which all helped birth this unusual drug development ecosystem. And that’s why we can do things like the Endpoints 11. Here’s my talk with Phil Sharp, which you can either watch or read below.

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