Pro­tal­ix, Chiesi fire up ac­cel­er­at­ed ap­proval pitch for Fab­ry dis­ease treat­ment

When Pro­tal­ix Bio­Ther­a­peu­tics re­port­ed its 2018 re­sults this March, it spent much of its time talk­ing up the po­ten­tial of nab­bing an ac­cel­er­at­ed ap­proval for its Fab­ry dis­ease treat­ment pe­gu­ni­gal­si­dase al­fa.

Weeks lat­er, the Is­raeli biotech — which has an un­ap­pe­tiz­ing pen­ny stock val­u­a­tion $PLX — and part­ner Chiesi are go­ing for it, op­ti­mistic af­ter months of dis­cus­sion with the FDA. The BLA fil­ing is sched­uled for Q1 2020.

Like Shire’s Re­pla­gal, which it’s be­ing com­pared against in a pair of late-stage tri­als, pe­gu­ni­gal­si­dase al­fa (or PRX-102) is an en­zyme re­place­ment ther­a­py de­signed to break down the ex­cess fat mol­e­cules build­ing up in Fab­ry pa­tients. Syn­the­sized from plants, this par­tic­u­lar re­com­bi­nant al­pha-galac­tosi­dase A en­zyme can be in­fused every four weeks in­stead of Re­pla­gal’s two.

An ini­tial glimpse of the Phase III study showed that high lev­els of the drug cir­cu­lat­ed in the blood­stream af­ter 28 days, with a mean con­cen­tra­tion of 138 ng/mL among 15 evalu­able pa­tients. In con­trast, Fab­razyme — an­oth­er ERT de­vel­oped by Sanofi Gen­zyme — had his­tor­i­cal­ly shown a mean con­cen­tra­tion of 20 ng/mL at 10 hours post in­fu­sion, HC Wain­wright an­a­lysts added in a Feb­ru­ary note.

But those are not the on­ly com­peti­tors Pro­tal­ix will face. Am­i­cus has trav­eled down its own wind­ing path to an ap­proval for Galafold, an oral drug that works by ad­dress­ing some mis­fold­ed α-GalA en­zymes. New ther­a­pies to in­tro­duce healthy copies of the GLA gene, which en­codes for the en­zyme, are com­ing up in the pipeline, with a no­table ef­fort from Avro­bio.

“This reg­u­la­to­ry ap­proval path is a sig­nif­i­cant achieve­ment as it means that we can start the ap­pli­ca­tion process and po­ten­tial­ly at­tain mar­ket ap­proval sig­nif­i­cant­ly ear­li­er than the ini­tial plan of da­ta from our on­go­ing phase III BAL­ANCE clin­i­cal tri­al,” CEO Moshe Manor said in a state­ment. “We plan to con­tin­ue the BAL­ANCE study to fur­ther strength­en the pro­file of pe­gu­ni­gal­si­dase al­fa.”

Chiesi holds the US rights to the drug from a deal that looped in $760 mil­lion in reg­u­la­to­ry and com­mer­cial mile­stones. Pro­tal­ix cur­rent­ly has one ap­proved prod­uct for Gauch­er dis­ease, mar­ket­ed by Pfiz­er.

That 2012 ap­proval, though, has done lit­tle to lift Pro­tal­ix’s suf­fer­ing stock af­ter a con­found­ing de­ci­sion in 2007 to price a sec­ondary of­fer­ing at an 85% dis­count to its trad­ing price the day pri­or. Shares plum­met­ed from $35 to $5 overnight and has bare­ly gone over $10 since, trad­ing way be­low $1 these days.

So­cial im­age: Shut­ter­stock

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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Ap­peals court puts the fi­nal nail in the cof­fin for Tec­fidera patent, adding to Bio­gen's bur­geon­ing set­backs

In another setback for Biogen, the big biotech lost its appeal to revive a patent for the once-blockbuster drug Tecfidera, marking a likely conclusion to the case.

The US Court of Appeals for the Federal Circuit issued the ruling Tuesday morning, saying Biogen failed to satisfy the “written description” requirement for patent law. As a result, Mylan-turned-Viatris will be able to sell its multiple sclerosis generic without fear of infringement and Biogen will have to find a new revenue driver elsewhere.

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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.

As lead drug runs in­to a wall, De­ci­phera slims down its pipeline, puts 140 jobs on the chop­ping block

Barely a month after disappointing data shattered hopes for a major label expansion for the GI tumor drug Qinlock, Deciphera is making a major pivot — scrapping development plans for that drug and discarding another while it hunkers down and focuses on two remaining drugs in the pipeline.

As a result, 140 of its staffers will be laid off.

The restructuring, which claims the equivalent of 35% of its total workforce, will take place across all departments including commercial, R&D as well as general and administrative support functions, Deciphera said, as it looks to streamline Qinlock-related commercial operations in the US while concentrating only on a “select number of key European markets.”

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How to use reg­istry da­ta to sup­port FDA de­ci­sion mak­ing: Agency ex­plains in new guid­ance

Drugmakers looking to design a new registry or use an existing one to support a regulatory decision on a drug’s effectiveness or safety will need to consult with a new draft guidance released Monday by the FDA.

The agency’s reliance on registry data for regulatory decisions dates back more than two decades, at least, as in 1998 Bayer won approval for its anticoagulant Refludan (withdrawn from the market in 2013 for commercial reasons) based in part on a historical control group pulled from a registry.

Tillman Gerngross (Adagio)

Till­man Gern­gross on Omi­cron: 'It is a grim sit­u­a­tion...we’re go­ing to see a sig­nif­i­cant drop in vac­cine ef­fi­ca­cy'

Tillman Gerngross, the rarely shy Dartmouth professor, biotech entrepreneur and antibody expert, has been warning for over a year that the virus behind Covid-19 would likely continue to mutate, potentially in ways that avoid immunity from infection and the best defenses scientists developed. He spun out a company, Adagio, to build a universal antibody, one that could snuff out any potential mutation.

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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.