Hu­mana spells out its con­di­tion­al Ex­ondys 51 cov­er­age pol­i­cy — strings at­tached

Hu­mana’s de­ci­sion to cov­er Sarep­ta’s $SRPT con­tro­ver­sial Duchenne mus­cu­lar dy­s­tro­phy drug Ex­ondys 51 (eteplirsen) comes with some thick strings at­tached to it.

In a new cov­er­age pol­i­cy post­ed at the big in­sur­er, Hu­mana says that it will on­ly pro­vide cov­er­age for the $300,000-plus ther­a­py pro­vid­ed pa­tients are still am­bu­la­to­ry, con­tin­ue to prove that they re­main able to walk and meet the cri­te­ria for pa­tients who may ben­e­fit from an Ex­on 51-skip­ping drug.

Un­der con­di­tions for cov­er­age, Hu­mana notes: “The mem­ber re­mains am­bu­la­to­ry (e.g. able to walk with as­sis­tance, not wheel­chair de­pen­dent).”

Trans­la­tion: Any pa­tient who con­tin­ues to de­te­ri­o­rate to the point that they re­quire a wheel­chair will no longer be cov­ered.

That pol­i­cy cov­er­age de­ci­sion fol­lows An­them’s ver­dict that it won’t cov­er the drug be­cause in their view it re­mains ex­per­i­men­tal, even though the FDA has ap­proved it for mar­ket­ing. Oth­er big in­sur­ers have said that they will cov­er it, but have not yet post­ed their con­di­tions.

Janet Wood­cock

Janet Wood­cock’s de­ci­sion to over­rule the point­ed ob­jec­tions of the re­view team as well as se­nior-lev­el col­leagues — wide­ly cel­e­brat­ed in the DMD com­mu­ni­ty that fought for this drug — con­tin­ues to draw heavy crit­i­cism in some cir­cles. Ear­li­er this week two FDA pol­i­cy ex­perts fret­ted over the “wor­ri­some mod­el” that the agency had en­dorsed in back­ing eteplirsen for an ap­proval, urg­ing a fresh look at how sim­i­lar drugs backed by pa­tient ad­vo­cates ahead of con­vinc­ing da­ta should be han­dled in the fu­ture, in­clud­ing a sug­ges­tion to make them avail­able with­out al­low­ing a prof­it. In­side the FDA, mean­while, John Jenk­ins has been ea­ger to sug­gest that that Sarep­ta was a once-off, with all the same tra­di­tion­al de­mands on ef­fi­ca­cy and safe­ty da­ta still in place.

That con­tro­ver­sy is now spilling over in­to the pay­er com­mu­ni­ty, and some clear­ly don’t like the idea of cov­er­ing a drug that some be­lieve re­mains ex­per­i­men­tal, re­gard­less of the FDA’s de­ci­sion.

Jef­feries’ Gena Wang, who bet against an ap­proval, says she wasn’t sur­prised by the de­ci­sion. And more such con­di­tion­al poli­cies may ap­pear. Her note in­clud­ed:

Re­call we had pre­vi­ous­ly not­ed that pay­ers ranked ev­i­dence of clin­i­cal ben­e­fit high­er than FDA ap­proval sta­tus and in­de­pen­dent analy­sis would be like­ly re­quired. Hu­mana’s de­ci­sion to lim­it cov­er­age with­in am­bu­la­to­ry pts is al­so in-line with our ex­pec­ta­tions of ad­di­tion­al re­stric­tions based on clin­i­cal tri­al de­mo­graph­ics (am­bu­la­to­ry pts with base­line age 7-10). Fur­ther­more, the 6 month ini­tial ap­proval pe­ri­od and the re­quire­ment for pts to re­main am­bu­la­to­ry for con­tin­u­al treat­ment, al­so echo the feed­back we re­ceived from pay­ers on pos­si­ble con­tin­u­ous mon­i­tor­ing of drug ef­fi­ca­cy. Among oth­er large pay­ers, Aet­na plans to con­duct a full clin­i­cal re­view (ac­cord­ing to Reuters), Unit­ed (via our com­mu­ni­ca­tion with a spokesper­son) have not­ed their in­ter­est in cov­er­ing the drug with a pri­or au­tho­riza­tion (de­tails undis­closed) and Cigna has con­firmed in­ter­est in pro­vid­ing cov­er­age (via email to Bloomberg). Ex­press Scripts al­so has plans to con­duct a full clin­i­cal re­view but de­tails are un­known.

From left to right: Lilian Kim, Associate Director Business Development; John Moller, CEO; Yooni Kim, Executive Director, Asia Operations; Michelle Park, Director South Korea Operations.

Novotech CRO sees 26% growth in Asia tri­al ac­tiv­i­ty from biotechs, but still plen­ty of ca­pac­i­ty

As the Asia-Pacific clinical trials sector continues to grow rapidly, Novotech the Asia-Pacific-based CRO is seeing biotech clinical activity up by 26%. But says there is still plenty of capacity in the region that features advanced medical facilities, supportive regulatory environments, and more than 2.3 billion people, largely treatment naïve, living in urban areas.

China, South Korea and Australia have the most studies registered as recruiting or about to recruit according to ClinicalTrials.Gov.

Pfizer, South San Francisco — Jeff Rumans for Endpoints News

UP­DAT­ED: Pfiz­er takes aim at a flag­ship fran­chise at Sanofi and Re­gen­eron — and scores a few di­rect hits

Count Pfizer in as a top player in the blockbuster game of JAK1 inhibitors.

Over the weekend the pharma giant posted some stellar Phase III efficacy data for their heavyweight contender abrocitinib in atopic dermatitis (eczema) that lines up ahead of a booming Dupixent (dupilumab), a blockbuster in the portfolios of Regeneron and Sanofi. And they put some real distance ahead of Eli Lilly’s trailing Olumiant, which made a delayed initial arrival on the market for rheumatoid arthritis after the FDA hobbled it with some additional hurdles on safety concerns.

JADE-MONO-1 scores well for Pfizer, teeing up what will be an intensely followed breakdown of the JADE MONO-2 data, which the pharma giant recently top-lined as “similar” to the first Phase III when tested against a placebo — a control group that has been easily outclassed by all the drugs in this market niche.

As of now, Pfizer looks to be equipped to run into the review stage — advantaged by a breakthrough therapy designation that is intended to speed up the regulatory process.

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A preda­tor's world? Top an­a­lyst sees the 'haves' and the 'haven't­s' di­verge as biotech bub­bles form — and col­lapse

Josh Schimmer

We’ve all seen the deluge of cash that’s been pouring into biotech from every angle: VCs, IPOs and follow-ons have generated billions in capital for new and emerging drug developers with ready access to some powerful new tech. But Evercore ISI’s Josh Schimmer is asking where we’re headed from here.

His answer is neither apocalyptic nor universally blissful, but if he’s right — and this is a discussion we’re hearing much, much more about at a time of growing economic and industry uncertainty — we may well be at a crossroads that could affect valuations, M&A and the entire global industry that has formed over the past 5 years.

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US mulls tar­iffs on Swiss drug ex­ports, weigh­ing on No­var­tis and Roche –  re­port

The leading Swiss newspaper has reported that the US is considering placing tariffs on pharmaceuticals from Switzerland. Roche and Novartis stock each fell 1% after the news broke.

Neue Zürcher Zeitung reported that US Trade Representative Robert Lighthizer told pharmaceutical representatives the Trump administration was considering the move. Tariffs do not appear to be in the immediate offing, but they would potentially affect Swiss giants Novartis and Roche along with other companies that manufacture in Switzerland, including Merck KGaA and US biotech Biogen, which is currently constructing a new facility in the country.

Neil Kumar, Endpoints

Bridge­Bio drops bid to re­claim Ei­dos af­ter di­rec­tors spurn 3 of­fers

A couple of months ago a newly public BridgeBio turned some heads by disclosing that it had made a bid for subsidiary Eidos Therapeutics in hopes of gobbling up the 34% stake that it doesn’t already own. Two offers later, the parties are calling it off.

A special committee of independent directors at the smaller biotech led by RA Capital’s Rajeev Shah and ex-Portola CEO William Lis first rejected the parent company’s initial offer — which would swap 1.3 BridgeBio shares for each Eidos share — on September 12. In the latest announcement, BridgeBio revealed that it eventually raised the offer to 1.5 shares and made $110 million available for all-cash or mixed consideration options, but Eidos still wasn’t interested.

Mark Foley, Revance

HR vi­o­la­tion push­es Re­vance co-founder out, vault­ing for­mer Zel­tiq chief to the helm

Months after Revance amended the terms of its Botox biosimilar collaboration with Mylan, the Newark, California-based drug developer disclosed its co-founder Dan Browne is stepping down, in what appears to be mysterious circumstances.

The company — which is also developing a rival to Allergan’s formidable Botox franchise — on Monday said Browne is departing “due to misjudgment in handling an employee matter,” that has also culminated in his resignation from Revance’s board of directors.

In-house FDA re­view flags a sus­pi­cious im­bal­ance in deaths as Sh­iono­gi hunts an OK for an­tibi­ot­ic

Shionogi has some big questions to answer if they plan to win an FDA panel’s backing for their new antibiotic.

While investigators have provided positive efficacy data for their new product to treat cases of complex urinary tract infections, an FDA review has flagged an imbalance of deaths between the antibiotic and a control arm. And they want the agency’s outside advisers to take a good hard look at that when they meet on Wednesday.

Cell ther­a­py start­up rais­es $16 mil­lion to fund its quest for the Holy Grail in re­gen­er­a­tive med­i­cine

In 2006, Shinya Yamanaka shook stem cell research with his discovery that mature cells can be converted into stem cells, relieving a longstanding political-ethical blockage and throwing open medical research on everything from curbing eye degeneration to organ printing.

But that process still has pitfalls, including in risk and scalability, and some researchers are exploring another way first hinted at years ago: new technology to convert mature cells directly into other mature cells without the complex and time-consuming process of first making them into stem cells.

Eye­ing $86M, Galera leads a pack of three mod­est biotech IPOs push­ing past high pro­file stum­bles

Exactly one year after kicking off a pivotal Phase III study for its lead drug — a companion for cancer patients receiving radiotherapy — Galera is looking to the Nasdaq for some new cash to complete the clinical work and fuel its commercial drive.

CEO Mel Sorensen has penciled in an $86 million ask, which was filed on the same day as liver disease company 89bio and rare disease diagnostics shop Centogene. The trio marks the first batch of IPO filings in the wake of two highly anticipated but ultimately disappointing public debuts by BioNTech and Vir, signaling dwindling biotech fervor on Wall Street. 89bio and Centogene are seeking $70 million and $69 million, respectively.