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.

Da­ta Lit­er­a­cy: The Foun­da­tion for Mod­ern Tri­al Ex­e­cu­tion

In 2016, the International Council for Harmonisation (ICH) updated their “Guidelines for Good Clinical Practice.” One key shift was a mandate to implement a risk-based quality management system throughout all stages of a clinical trial, and to take a systematic, prioritized, risk-based approach to clinical trial monitoring—on-site monitoring, remote monitoring, or any combination thereof.

Mer­ck scraps Covid-19 vac­cine pro­grams af­ter they fail to mea­sure up on ef­fi­ca­cy in an­oth­er ma­jor set­back in the glob­al fight

After turning up late to the vaccine development game in the global fight against Covid-19, Merck is now making a quick exit.

The pharma giant is reporting this morning that it’s decided to drop development of 2 vaccines — V590 and V591 — after taking a look at Phase I data that simply don’t measure up to either the natural immune response seen in people exposed to the virus or the vaccines already on or near the market.

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Ron Cooper, Albireo CEO

Al­bireo just ad­vanced down to the 10-yard line at the FDA. And Ron Coop­er’s team is get­ting prepped for the next big play

When Albireo Pharma’s board $ALBO moved to bring in Ron Cooper as the CEO more than 5 years ago, the development-stage company went with an experienced commercial player who had a big-time position on his resume after running Bristol Myers’ commercial ops in Europe.

Now, after successfully navigating a pivotal study, putting them in a foot race with a rival toward an FDA OK, Cooper is getting a boost from regulators on the last drive back to an arena he understands completely.

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More than five years after Corey Fishman and Michael Dunne dusted sulopenem off Pfizer’s shelves — the second castoff antibiotic they’ve brought out of the pharma giant — and founded Iterum Therapeutics around that single drug, they have lined up a quick shot at approval with priority review from the FDA.

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Matt Gline (L) and Vivek Ramaswamy

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Over the past 7 years since founding Roivant, Vivek Ramaswamy has been a constant blur of biotech building motion.

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Janet Woodcock (AP Images)

End­points poll: Janet Wood­cock takes the (in­ter­im) helm at the FDA. And a large ma­jor­i­ty of our read­ers want her to stay there

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Hal Barron, GSK via YouTube

What does $29B buy you in Big Phar­ma? In Glax­o­SmithK­line’s case, a whole lot of un­com­fort­able ques­tions about the pipeline

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A little over a week ago, GSK R&D chief Hal Barron marked his third anniversary at the research helm by taking a turn at the virtual podium during JP Morgan to make the case that he and his team had built a valuable late-stage pipeline capable of churning out more than 10 blockbusters in the next 5 years.

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Bahija Jallal, Immunocore

Buried in Im­muno­core's IPO fil­ings? A kick­back scheme from a now for­mer em­ploy­ee

Immunocore spent much of 2019 dealing with the fallout of the Neil Woodford scandal, as the former star investor’s fall crashed the biotech’s valuation out of unicorn range. Now it turns out that the company spent 2020 dealing with another internal scandal.

The longtime UK biotech darling disclosed in their IPO filing last week that they had fallen victim to an alleged kickback scheme involving one of their employees. After a whistleblower came forward, they said in their F-1, they spent the summer and spring investigating, finding fraud on the part of an employee and two outside vendors.

Stéphane Bancel, Moderna CEO (Steven Ferdman/Getty Images)

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As Merck bows out of the global race to develop vaccines for Covid-19, Moderna is doubling down to make sure they can quell new variants that have recently emerged and quickly spread.

The Cambridge, MA-based biotech put out word on Monday that in vivo studies indicate their mRNA vaccine works well enough against two strains first detected in the UK and South Africa. But with a six-fold reduction in neutralizing titers observed against the latter strain, the company is launching a new study of a booster version to make sure it can do the job.

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