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.

Michel Vounatsos, Biogen CEO (via YouTube)

UP­DAT­ED: Bio­gen spot­lights a pair of painful pipeline set­backs as ad­u­canum­ab show­down looms at the FDA

Biogen has flagged a pair of setbacks in the pipeline, spotlighting the final failure for a one-time top MS prospect while scrapping a gene therapy for SMA after the IND was put on hold due to toxicity.

Both failures will raise the stakes even higher on aducanumab, the Alzheimer’s drug that Biogen is betting the ranch on, determined to pursue an FDA OK despite significant skepticism they can make it with mixed results and a reliance on post hoc data mining. And the failures are being reported as Biogen was forced to cut its profit forecast for 2020 as a generic rival started to erode their big franchise drug.

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A new chap­ter in the de­cen­tral­ized clin­i­cal tri­al ap­proach

Despite the promised decentralized trial revolution, we haven’t yet moved the needle in a significant way, although we are seeing far bolder commitments to this as we continue to experience the pandemic restrictions for some time to come. The vision of grandeur is one thing, but operationalizing and execution are another and recognising that change, particularly mid-flight on studies, is worthy of thorough evaluation and consideration in order to achieve success. Here we will discuss one of the critical building blocks of a Decentralized and Remote Trial strategy: TeleConsent; more than paper under glass, it is a paradigm change and key digital enabler.

Pfiz­er scoops up an an­tibi­ot­ic in rare M&A deal, bag­ging a vir­tu­al start­up op­er­at­ing on a shoe­string bud­get

Pfizer is stepping up with a rare antibiotics buyout deal today, grabbing Palo Alto, CA-based Arixa Pharmaceuticals in a bid to add a new oral version of avibactam, a beta lactamase inhibitor — or BLI — approved back in 2015 as part of the IV treatment Avycaz.

The Arixa acquisition follows some encouraging Phase I responses demonstrating that 60% to 80% of the oral drug is absorbed into the bloodstream. Only 7% of the IV version is absorbed orally, far below the 30% threshold Arixa has pointed to as a therapeutic threshold. The buyout gives Pfizer’s hospital group a line on a new oral combo with antibiotics like ceftibuten to go after drug-resistant cases of urinary tract infections and other ailments.

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Can B cells break the bound­aries of cell ther­a­py? Long­wood start­up has $52M to prove a new en­gi­neer­ing tech

Back in December 2017, as the cell therapy world was still basking in the virtually back-to-back approvals of two pioneering CAR-Ts, researchers at Seattle Children’s Research Institute reported a scientific first in a different corner of the field: engineer B cells to treat disease.

The team, led by David Rawlings and Richard James, eventually worked with Longwood Fund to start a biotech around those findings. And now Atlas Venture and RA Capital Management are coming on board to lead a $52 million launch round, joined by Alta Partners, for Be Biopharma.

Jason Kelly, Gingko Bioworks CEO (Mike Blake/Reuters via Adobe)

Ex­clu­sive: Eye­ing big Covid-19 test­ing ex­pan­sion, Gink­go rolls out 50M rapid anti­gen di­ag­nos­tics

In what they hope will be a key part of an extensive effort to boost Covid-19 testing in the US, Ginkgo Bioworks is acquiring and distributing 50 million rapid antigen tests that can potentially be used for virus surveillance in schools and communities and for quick, on the ground diagnoses.

The tests, developed by SD Biosensor, are in line with proposals from the Rockefeller Foundation and Harvard epidemiologists, among others, to blanket the country with fast, low-cost tests. Although not yet authorized in the US, they are a key part of testing efforts from the Bill and Melinda Gates Foundation, who announced plans last month to distribute 120 million of them in low and middle income countries. Roche has commercialized the diagnostic in Europe.

Bo Cumbo, AavantiBio CEO (file photo)

Bo Cum­bo jumps from the top com­mer­cial post at Sarep­ta to the helm of a gene ther­a­py start­up with some in­flu­en­tial back­ers, big plans and $107M

After a 7-year stretch building the commercial team at Sarepta, longtime drug salesman Bo Cumbo is jumping to the entrepreneurial side of the business, taking the helm of a startup that’s got several deep-pocket investors. And he’s not just bringing his experience in selling drugs.

He tells me that when he told Sarepta CEO Doug Ingram about it, his boss got excited about the venture and opted to jump in with a $15 million investment from Sarepta to add to the launch money, alongside 3 of the busiest investors in biotech.

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HHS secretary Alex Azar (at the podium) and FDA commissioner Stephen Hahn (Pete Marovich/Getty Images)

Covid-19 roundup: Azar open­ly plan­ning Hahn ouster — re­port; Vul­ner­a­ble pop­u­la­tions like­ly to get vac­cines by Jan­u­ary

The relationship between HHS secretary Alex Azar and FDA commissioner Stephen Hahn has deteriorated to the point where Azar has suggested replacing Hahn, according to a Politico report.

Azar was angered by the FDA’s pushback of the Trump administration’s proposals for authorizing Covid-19 vaccines, so much so that he began openly floating potential replacements for Hahn. The report cited six unnamed sources that said Azar discussed bringing up Hahn’s removal to the White House.

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Jean-Pierre Sommadossi, Atea president and CEO (file photo)

Roche wades deep­er in­to Covid-19 fight, ink­ing an­tivi­ral pact with $350M cash fol­low­ing Re­gen­eron deal

Roche is making its first bet on an antiviral against Covid-19 in style, shelling out $350 million in cash to grab ex-US rights.

The drug comes from Atea Pharmaceuticals, the 7-year-old biotech created by Pharmasset co-founder Jean-Pierre Sommadossi, which essentially rebranded itself as a Covid-19 fighter in May when it closed a whopping $215 million venture round. Over a dozen investors bought in, including marquee names like Bain Capital and RA Capital.

CEO Marc Gleeson (Azura)

Azu­ra Oph­thalmics gets a $20M boost for its R&D work on eye dis­eases

Three years after closing a $16 million Series B, the same group of investors are back to give Azura Ophthalmics a $20 million boost.

That brings the Tel Aviv-Yafo, Israel-based biotech’s total fundraise to $38 million, and should pave the way for a registration study of its lead candidate in Meibomian gland dysfunction (MGD) and related eye diseases, CEO Marc Gleeson told Endpoints News.

The topical candidate, dubbed AZR-MD-001, is designed to address abnormal hyperkeratinization, or the build-up and shedding of proteins at the opening of or within the Meibomian gland. When Meibomian glands become dysfunctional, rapid evaporation of the tear film can occur, leading to dry eye disease.