Eiger nabs the first FDA ap­proval for Prog­e­ria, an ul­tra-rare pre­ma­ture ag­ing dis­ease, with an old Mer­ck drug

Eiger Bio­Phar­ma­ceu­ti­cals $EIGR has re­ceived an FDA OK for a drug Mer­ck li­censed to them at no cost — and now re­port­ed­ly plans to charge a lev­el con­sis­tent with oth­er ul­tra-rare dis­ease med­i­cines.

The biotech an­nounced Fri­day evening that reg­u­la­tors had ap­proved lon­a­farnib for the treat­ment of Hutchin­son-Gil­ford prog­e­ria syn­drome, al­so known as Prog­e­ria, as well as some progeroid laminopathies in chil­dren old­er than one year. It’s the first ap­proval grant­ed for the con­di­tion, and the drug will be mar­ket­ed and sold as Zok­in­vy.

In­vestors met the news warm­ly, send­ing the stock up rough­ly 12% in post-mar­ket trad­ing Fri­day. By ear­ly Mon­day morn­ing, the price had lev­eled off to up about 5%.

One of the rarest dis­eases in the world with an es­ti­mat­ed 350 to 400 cas­es at any one time, Prog­e­ria is a ge­net­ic dis­or­der that caus­es pre­ma­ture ag­ing in chil­dren. About 180 in­di­vid­u­als with the dis­ease have been iden­ti­fied, Eiger says, in­clud­ing 20 in the US and 23 in Eu­rope. Those di­ag­nosed with the dis­ease have an av­er­age life ex­pectan­cy of about 14 years and gen­er­al­ly suf­fer from com­pli­ca­tions such as heart fail­ure, stroke or hard­en­ing of the ar­ter­ies.

Lon­a­farnib, an oral far­ne­syl­trans­ferase in­hibitor, helps pre­vent the buildup of de­fec­tive prog­erin or prog­erin-like pro­teins. Two clin­i­cal tri­als en­rolling 62 pa­tients have shown a re­duc­tion in mor­tal­i­ty by 60 per­cent (p=0.0064), and chil­dren tak­ing the drug have lived about 2.5 years longer on av­er­age af­ter the 11-year fol­low-up pe­ri­od.

With the ap­proval, Eiger al­so won a Pri­or­i­ty Re­view Vouch­er for rare pe­di­atric dis­eases, which it plans to sell. The biotech will split the pro­ceeds 50-50 with the Prog­e­ria Re­search Foun­da­tion, with whom it de­vel­oped the drug.

Eiger li­censed the drug in 2010 for an­ti-vi­ral in­di­ca­tions, in­clud­ing He­pati­tis D, af­ter Mer­ck shut down an in­ves­tiga­tive pro­gram for can­cer. The com­pa­nies ex­pand­ed their col­lab­o­ra­tion in 2018 to in­clude Prog­e­ria, giv­ing Eiger full com­mer­cial and dis­tri­b­u­tion rights for any li­censed and ap­proved in­di­ca­tions. Eiger was re­spon­si­ble for all reg­u­la­to­ry, com­mer­cial­iza­tion and dis­tri­b­u­tion as­pects of lon­a­farnib, and Mer­ck will not re­ceive any mile­stone pay­ments on Prog­e­ria sales.

The biotech now plans to price the drug at lev­els com­pa­ra­ble to oth­er ul­tra-rare dis­ease med­i­cines, ac­cord­ing to the Boston Globe, and does not ex­pect “cost to be a bar­ri­er to pa­tient ac­cess.” Eiger did not re­spond to an End­points News re­quest for com­ment on pric­ing ear­ly Mon­day morn­ing.

Lon­a­farnib’s ap­proval proves a big win for Eiger fol­low­ing a rough cou­ple of years in which two tri­als for uben­imex flopped in pul­monary ar­te­r­i­al hy­per­ten­sion and low­er leg lym­phede­ma. Eiger has scrapped work for that med­i­cine un­less an­oth­er com­pa­ny ex­press­es in­ter­est in de­vel­op­ing it. In 2018, Eiger saw some good news when its avex­i­tide pro­gram re­turned pos­i­tive Phase II re­sults in post-bariatric hy­po­glycemia, or low blood glu­cose lev­els ex­pe­ri­enced af­ter weight-loss surgery.

The com­pa­ny is al­so con­duct­ing a Phase III tri­al for lon­a­farnib in He­pati­tis D, as well as a Phase III for pegin­ter­fer­on lamb­da in the same in­di­ca­tion.

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.

FDA can­cels ODAC meet­ing this week to re­view two more dan­gling ac­cel­er­at­ed ap­provals — but won't ex­plain why

The FDA’s Oncologic Drugs Advisory Committee has decided to cancel a planned meeting on Thursday to discuss two cancer drugs that previously won accelerated approvals but failed to confirm clinical benefit in required follow-up trials or have taken a long time to finish those trials.

The FDA said in a statement that the meeting “is no longer needed” but did not offer further detail on why exactly it was canceled, telling Endpoints News to contact the companies. Attempts to contact both Secura Bio and Acrotech went unreturned. The companies may have decided to pull these treatments from the market, or they’ve come to new agreements with the agency on their confirmatory trials.

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.