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.

Brent Saunders [Getty Photos]

UP­DAT­ED: Ab­b­Vie seals $63B deal to buy a trou­bled Al­ler­gan — spelling out $1B in R&D cuts

Brent Saunders has found his way out of the current fix he’s in at Allergan $AGN. He’s selling the company to AbbVie for $63 billion in the latest example of the hot M&A market in biopharma.

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Sanofi/Re­gen­eron mus­cle ahead of a ri­val No­var­tis/Roche team, win first ap­proval in key rhi­nos­i­nusi­tis field

Re­gen­eron and their part­ners at Sanofi have beat the No­var­tis/Roche team to the punch on an­oth­er key in­di­ca­tion for their block­buster an­ti-in­flam­ma­to­ry drug Dupix­ent. The drug team scored an ac­cel­er­at­ed FDA ap­proval for chron­ic rhi­nos­i­nusi­tis with nasal polyps, mak­ing this the first such NDA for the field.

An­a­lysts have been watch­ing this race for awhile now, as Sanofi/Re­gen­eron won a snap pri­or­i­ty re­view for what is now their third dis­ease in­di­ca­tion for this treat­ment. And they’re not near­ly done, build­ing up hopes for a ma­jor fran­chise.

Af­ter rais­ing $158M, this up­start's founders have star back­ers and plans to break new ground in gene ther­a­py

Back in 2014, Stephanie Tagliatela opted to take an early exit out of her PhD program after working in Mark Bear’s lab at MIT, where she specialized in the synaptic connections between neuronal cells in the brain. She never finished that PhD, but she and fellow MIT student Kartik Ramamoorthi — who was on the founding team at Voyager — came away with some ideas for a gene therapy startup.

Today, fully 5 years later, she and Ramamoorthi are taking the wraps off of a $104 million mega-round designed to take the cumulative work of their preclinical formative stage for Encoded Therapeutics into human studies. They’ve now raised $158 million since starting out in Illumina’s incubator in the Bay Area, and they believe they are firmly on track to do something unique in gene therapy.

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Novotech CEO Dr. John Moller

Novotech CRO Award­ed Frost & Sul­li­van Best Biotech CRO Asia-Pa­cif­ic 2019

Known in the in­dus­try as the Asia-Pa­cif­ic CRO, Novotech is now lead CRO ser­vices provider for the grow­ing num­ber of in­ter­na­tion­al biotechs se­lect­ing the re­gion for their stud­ies.

Re­flect­ing this Asia-Pa­cif­ic growth, Novotech staff num­bers are up 20% since De­cem­ber 2018 to 600 in-house clin­i­cal re­search peo­ple across a full range of ser­vices, across the re­gion.

Novotech’s ca­pa­bil­i­ties have been rec­og­nized by an­a­lysts like Frost & Sul­li­van, most re­cent­ly with the pres­ti­gious Asia-Pa­cif­ic CRO Biotech of the year award for best prac­tices in clin­i­cal re­search for biotechs for the fifth year. See oth­er awards here.

Richard Gonzalez testifying in front of Senate Finance Committee, February 2019 [AP Images]

Ab­b­Vie's $63B buy­out spot­lights the re­turn of ma­jor M&A deals — de­spite the back­lash

Big time M&A is back. But for how long?

Over the past 18 months we’ve now seen three major buyouts announced: Takeda/Shire; Bristol-Myers/Celgene and now AbbVie/Allergan. And with this latest deal it’s increasingly clear that the sharp fall from grace suffered by high-profile players which have seen their share prices blasted has created an opening for the growth players in big pharma to up their game — in sharp contrast to the popular bolt-on deals that have been driving the growth strategy at Novartis, Merck, Roche and others.

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Two biotech uni­corns swell pro­posed IPOs, eye­ing a $600M-plus wind­fall

We’ve been wait­ing for the ar­rival of Bridge­Bio’s IPO to top off the wave of new biotech of­fer­ings sweep­ing through Nas­daq at the end of H1. And now we learn that it’s been sub­stan­tial­ly up­sized.

Ini­tial­ly pen­ciled in at a uni­corn-sized $225 mil­lion, the KKR-backed biotech has spiked that to the neigh­bor­hood of $300 mil­lion, look­ing to sell 20 mil­lion shares at $14 to $16 each. That’s an added 5 mil­lion shares, re­ports Re­nais­sance Cap­i­tal, which fig­ures the pro­posed mar­ket val­u­a­tion for Neil Ku­mar’s com­pa­ny at $1.8 bil­lion.

No­var­tis holds back the copy­cat brigade's at­tack on its top drug fran­chise — for now

A fed­er­al judge has put a gener­ic chal­lenge to No­var­tis’ block­buster mul­ti­ple scle­ro­sis drug Gilenya on hold while a patent fight plays out in court.

Judge Leonard P. Stark is­sued a tem­po­rary in­junc­tion ear­li­er this week, forc­ing My­lan, Dr. Red­dy’s Lab­o­ra­to­ries and Au­robindo Phar­ma to shelve their launch plans to al­low the patent fight to pro­ceed. He ruled that al­low­ing the gener­ics in­to the mar­ket now would per­ma­nent­ly slash the price for No­var­tis, even if it pre­vails. 

Top an­a­lyst finds a sil­ver lin­ing in Ab­b­Vie’s $63B Al­ler­gan buy­out — but there’s a catch

Af­ter get­ting beat up on all sides from mar­ket ob­servers who don’t much care for the lat­est mega-deal to ar­rive in bio­phar­ma, at least one promi­nent an­a­lyst now is start­ing to like what he sees in the num­bers for Ab­b­Vie/Al­ler­gan.

But it’s go­ing to take some en­cour­age­ment if Ab­b­Vie ex­ecs want it to last.

Ab­b­Vie’s mar­ket cap de­clined $20 bil­lion on Tues­day as the stock took a 17% hit dur­ing the day. And SVB Leerink’s Ge­of­frey Porges can see a dis­tinct out­line of an up­side af­ter re­view­ing the fun­da­men­tals of the deal.

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While Ako­rn works to re­vive its for­tunes, the FDA hits it with an­oth­er warn­ing let­ter

Ako­rn just can’t dig it­self out of its hole.

The spe­cial­ty gener­ic drug­mak­er has re­ceived yet an­oth­er warn­ing let­ter from the FDA this year. With­out dis­clos­ing any specifics, the Lake For­est, Illi­nois-based drug­mak­er on Wednes­day said the US reg­u­la­tor had is­sued the let­ter, cit­ing an in­spec­tion of its Som­er­set, New Jer­sey man­u­fac­tur­ing fa­cil­i­ty in Ju­ly and Au­gust of 2018. The com­pa­ny’s shares $AKRX dipped about 1.7% to $4.65 be­fore the bell.