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.

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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Janet Woodcock (AP Images)

Janet Wood­cock plots her fu­ture at FDA, with se­nior ad­vi­sor role to fall back on if Califf wins con­fir­ma­tion

Acting FDA commissioner Janet Woodcock has been the face of just about every drug approval decision at the agency since the turn of the century. Since the pandemic began, she’s moved between the top of the drugs center to the head of therapeutics at Operation Warp Speed, leading the drive for work on Covid-targeted mAbs and antivirals.

Looking forward — and pending a quick Senate confirmation to cement Rob Califf’s return to the top of FDA early next year — Woodcock’s role at the agency will again be in flux.

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Richard Pazdur (via AACR)

Ac­cel­er­at­ed ap­proval re­forms need mean­ing­ful con­fir­ma­to­ry tri­al im­prove­ments, pro­fes­sors write in Sci­ence

Outside of Covid-19, 2021 has been the year of the accelerated approval.

Beginning last spring, FDA openly challenged six “dangling” accelerated approvals (hadn’t confirmed their clinical benefit yet), three of which were later pulled by the companies.

Then in June, FDA pulled out the accelerated approval pathway, seemingly out of nowhere, to sign off on Biogen’s controversial Alzheimer’s drug Aduhelm. It hadn’t even been mentioned at the drug’s adcomm.

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. 

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.

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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