Joe Wiley, Amryt CEO

Am­ryt earns a pri­or­i­ty re­view in a rare dis­ease that makes pa­tients' skin frag­ile as but­ter­fly wings

A year af­ter flash­ing mixed Phase III da­ta, an Irish drug­mak­er no­to­ri­ous for own­ing some of the coun­try’s most ex­pen­sive drugs is get­ting an ex­pe­dit­ed look at its treat­ment for epi­der­mol­y­sis bul­losa (EB) — a rare dis­ease where a pa­tient’s skin be­comes as frag­ile as but­ter­fly wings.

The FDA grant­ed pri­or­i­ty re­view to Am­ryt’s Oleogel-S10, ac­cel­er­at­ing the drug’s as­sess­ment time from 10 months to six months, the com­pa­ny said on Thurs­day. The agency set its PDU­FA date for Nov. 30, and CEO Joe Wi­ley said launch plans are “well ad­vanced.”

Am­ryt orig­i­nal­ly picked up the drug, al­so known as episal­van, from the small Ger­man drug­mak­er Birken. The drug was al­ready EMA-ap­proved for par­tial thick­ness wounds, but Am­ryt saw po­ten­tial in EB and rushed it in­to a Phase III tri­al al­most im­me­di­ate­ly.

The FDA’s de­ci­sion will be based on da­ta from Am­ryt’s EASE tri­al, in which 41.3% of pa­tients who re­ceived Oleogel-S10 saw first com­plete clo­sure of EB tar­get wounds with­in 45 days. That com­pares to 28.9% of pa­tients in the con­trol group, trans­lat­ing to a 44% in­crease in the prob­a­bil­i­ty of tar­get wound clo­sure (and a p-val­ue of 0.013), ac­cord­ing to Am­ryt.

The drug missed on key sec­ondary end­points, though, in­clud­ing the pro­por­tion of com­plete­ly closed tar­get wounds with­in the 90-day treat­ment pe­ri­od. In­ves­ti­ga­tors say they saw a greater re­duc­tion of to­tal body wound bur­den in those on Oleogel-S10 — mea­sured by a com­mon­ly used scale and to­tal sur­face area of EB par­tial thick­ness wounds — but the dif­fer­ence wasn’t sta­tis­ti­cal­ly sig­nif­i­cant.

At the time, Wi­ley cel­e­brat­ed the re­sults as a “sig­nif­i­cant mile­stone” for the com­pa­ny, and he’s al­ready brand­ed the EB ver­sion of the drug as Fil­su­vez.

There are cur­rent­ly no ap­proved treat­ments for EB, which af­fects rough­ly 1 in 20,000 peo­ple. Chron­ic and re­laps­ing wounds leave pa­tients vul­ner­a­ble to re­peat­ed in­fec­tions, ac­cord­ing to the FDA, and some end up with fi­bro­sis, de­for­mi­ties and cu­ta­neous car­cino­gen­e­sis.

“Skin le­sions are as­so­ci­at­ed with itch­ing and pain, the lat­ter ag­gra­vat­ed dur­ing dress­ing changes,” the agency said in a 2019 state­ment, up­on re­leas­ing new guid­ance to help com­pa­nies de­vel­op treat­ments.

Sev­er­al oth­ers are work­ing on treat­ments for the rare con­di­tion, in­clud­ing Abeona, which last sum­mer reini­ti­at­ed a Phase III tri­al for its cell ther­a­py af­ter a brief pause due to Covid-19. Krys­tal Biotech said it com­plet­ed en­roll­ment in a Phase III study for its own dy­s­troph­ic EB can­di­date back in March, and ex­pects top-line re­sults in Q4. Cas­tle Creek Phar­ma­ceu­ti­cals bought out Fi­bro­cell Sci­ence back in De­cem­ber 2019, and is now in Phase III with its re­ces­sive dy­s­troph­ic EB can­di­date FCX-007.

Fil­su­vez would make Am­ryt’s fourth ap­proved prod­uct, af­ter Episal­van and two drugs the com­pa­ny picked up from Aege­ri­on: Myalept and Jux­tapid. The lat­ter two have con­sis­tent­ly ranked among the most ex­pen­sive drugs in the US. Myalept now is fourth on GoodRx’s list of the most cost­ly drugs in the coun­try.

In­side Track: Be­hind the Scenes of a Ma­jor Biotech SPAC

Dr. David Hung and Michelle Doig are no strangers to the SPAC phenomenon. As Founder and CEO of Nuvation Bio, a biotech company tackling some of the greatest unmet needs in oncology, Dr. Hung recently took the company public in one of this year’s biggest SPAC related deals. And as Partner at Omega Funds, Doig not only led and syndicated Nuvation Bio’s Series A, but is now also President of the newly formed, Omega-sponsored, Omega Alpha SPAC (Nasdaq: OMEG; oversubscribed $138m IPO priced January 6, 2021).

Barry Greene, Sage CEO

UP­DAT­ED: Sage's sec­ond chance at de­pres­sion hits the PhI­II pri­ma­ry, but ques­tions re­main over dura­bil­i­ty, side ef­fects

Looking to make a comeback after a big Phase III flop, Sage Therapeutics revealed data they believe could change the entire depression treatment landscape, given the vast array of failures in the field. But some results are spooking investors, sending Sage $SAGE shares down early Tuesday.

First, the primary: Sage and Biogen reported Phase III data for once-daily zuranolone Tuesday morning, saying the experimental drug hit its primary endpoint by spurring a statistically significant change from baseline in the 17-item Hamilton Rating Scale for Depression total score. After 15 days, patients in the drug arm saw an average change of -14.1 points, compared to -12.3 on placebo.

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Harith Rajagopalan (Fractyl)

Af­ter a decade in the Type 2 di­a­betes game, Fractyl Lab­o­ra­to­ries recharges with a fresh $100M and a new name

Harith Rajagopalan compared the way Type 2 diabetes is managed to sticking your fingers in a dam that’s leaking from a number of places.

You can take drugs to lower your blood sugar, cholesterol, or blood pressure, but you’re not addressing what he says is the core issue — the metabolic abnormality that causes the disease.

“We’re so busy plugging the holes in the dam, we don’t have time to see that the whole infrastructure is at risk,” he said. “That infrastructure is a full-body systemic metabolic abnormality called metabolic syndrome, that we’re ignoring while we’re so busy trying to treat all of the individual symptoms of the condition.”

Bio­gen sig­nals a big PhI­II fail­ure as the lead gene ther­a­py in their $800M Night­star buy­out goes down in flames

That $800 million buyout of Nightstar has turned into a bust for Biogen as the lead therapy in the deal failed a pivotal study, signaling a severe setback for the biotech’s ambitions in gene therapies.

The big biotech put out the word after the market closed on Monday that the gene therapy they picked up in the deal for a degenerative blindness called choroideremia failed the Phase III study, just a month after their #2 drug in the deal also flopped in a mid-stage study.

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Hal Barron, GSK R&D chief (Endpoints News)

Hal Bar­ron gam­bles $625M cash on high-wire TIG­IT act, throw­ing Glax­o­SmithK­line in­to heat­ed race and com­plet­ing next-gen I/O trin­i­ty

Count Hal Barron and GlaxoSmithKline in for the TIGIT fight.

The stakes are as high as the risks: While a growing pack of Big Pharma rivals is lending credence to the hypothesis that TIGIT will be the next big immune checkpoint and cancer drug target, the first clinical trials have shown response rates that can be described as modest at best. But Barron’s bet is on the whole “axis” that the receptor sits on, with an eye on testing its new anti-TIGIT antibody not just in combo with PD-1 but also in triplets.

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Michel Sade­lain puts his name and new cell en­gi­neer­ing tech be­hind 'ag­nos­tic' CAR-T start­up chas­ing epi­ge­net­ic anti­gens

It felt natural for Alain Maiore and Sebastian Amigorena to bring in Michel Sadelain as a co-founder of Mnemo Therapeutics. A CAR-T pioneer, Sadelain had been involved as an advisor since the early days — enthusiastic about Amigorena’s work in a genetic knockout that could enhance T cell memory and a new class of potential targets he’s discovered — and could introduce some well-known technologies to the toolbox. So they got the initial cash from Sofinnova Partners to plant roots in Paris and New York in early 2019; within a few months, they began to see more clearly just what the antigen discovery platform might unlock.

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Patrizia Cavazzoni, CDER

FDA’s Cavaz­zoni calls for ad­comms to ‘get back to the sub­stance’

While her comments were recorded prior to the FDA’s recent approval of Biogen’s controversial Alzheimer’s drug, CDER Director Patrizia Cavazzoni presciently called for substantial reforms to the advisory committee process at the agency.

Short on examples of the adcomms she was referring to, Cavazzoni said at a BIO event aired on Monday that some recent committees show “how they can be swayed by emotion in the face of hard facts,” but they need to “get back to the fundamentals, which is listening to thoughtful input from experts in response to thoughtful questions that we ask them.”

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75M vac­cine dos­es wast­ed: FDA spells out Emer­gent fa­cil­i­ty de­fi­cien­cies at length in new memo

The FDA is offering a fuller account of what went wrong at Emergent BioSolutions’ Covid-19 manufacturing facility in Baltimore, where the cross contamination of J&J and AstraZeneca vaccines led to the discarding of about 75 million vaccine doses.

CBER director Peter Marks released a memo on Saturday with new specifics, making clear up front that no vaccine manufactured at this plant has been distributed for use in the US yet.

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Lynn Fitch, Mississippi Attorney General (Rogelio V. Solis/AP Images)

Mis­sis­sip­pi sues Eli Lil­ly, Sanofi and No­vo over in­sulin prices as in­ter­change­able biosim­i­lars may ar­rive soon

Mississippi Attorney General Lynn Fitch last week sued the top three insulin manufacturers, which collectively cover almost the entire US insulin market, alleging that they’ve colluded to raise their prices in lockstep, and in some cases by more than 1,000% for drugs that are decades old.

“Because of Manufacturer Defendants’ collusive price increases, nearly a century after the discovery of insulin, diabetes medications have become unaffordable for many diabetics,” the lawsuit says.

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