An­a­lysts track a pay­er re­volt against Sarep­ta’s con­tro­ver­sial Duchenne drug

Janet Wood­cock

When the FDA’s Janet Wood­cock pushed through a con­tro­ver­sial ap­proval for Sarep­ta’s Ex­ondys 51 for Duchenne mus­cu­lar dy­s­tro­phy in Sep­tem­ber, she cre­at­ed a test case for pay­ers who were es­sen­tial­ly be­ing asked to re­im­burse for an ex­per­i­men­tal drug.

But in­stead of rolling with the sit­u­a­tion and pay­ing a price that starts at $300,000 or so a year, many of the pay­ers are kick­ing back hard, ac­cord­ing to a sur­vey of pay­ers con­duct­ed by Jef­feries.

Gena Wang, Jef­fries

“We con­duct­ed in depth due dili­gence on Ex­ondys 51 launch with 4 pri­vate pay­ers, 3 Med­ic­aid ex­perts, and 2 DMD KOLs with ex­pe­ri­ence in ad­min­is­ter­ing Ex­ondys 51,” not­ed Jef­feries Gena Wang, who had been in the large con­tin­gent of an­a­lysts who had pre­dict­ed a re­jec­tion for Sarep­ta. In ad­di­tion we com­piled a com­pre­hen­sive list of pub­lished med­ical poli­cies from 21 pri­vate pay­ers (5 na­tion­al & 15 re­gion­al MCOs; 1 PBM). Based on avail­able da­ta, 3/5 na­tion­al and 8/15 re­gion­al man­aged care or­ga­ni­za­tions (MCOs) have de­nied/re­strict­ed cov­er­age for Ex­ondys 51, in line with our ex­pec­ta­tion of push­back from pri­vate pay­ers.”

That push­back has cost Sarep­ta $SRPT plen­ty. The biotech has seen its share price fade back by about half since the ap­proval more than two months ago as ques­tions have con­tin­ued to cir­cu­late about pay­ers’ po­si­tion on the drug.

An­them got the back­lash rolling with its po­si­tion that it will not cov­er an ex­per­i­men­tal drug, even though it’s been giv­en an ac­cel­er­at­ed ap­proval. And Hu­mana fol­lowed up with re­stric­tive rules that re­quires pa­tients be­ing able to walk so long as they are re­im­burs­ing for the drug. It’s clear from the Jef­feries sur­vey that a slight ma­jor­i­ty of pay­ers don’t want to han­dle this like any oth­er ap­proved ther­a­py for a rare and dead­ly dis­ease.

The Ex­ondys 51 ap­proval trig­gered an in­ter­nal civ­il war at the FDA, pit­ting top reg­u­la­tors against Wood­cock, who ex­pressed sup­port for the drug and the com­pa­ny, even though it had on­ly been stud­ied in a tiny tri­al and the da­ta had been harsh­ly re­viewed by in­ter­nal reg­u­la­tors. That view helped set the stage for its re­jec­tion by a pan­el of out­side ex­perts, which al­so didn’t help the drug’s chances once it was hand­ed over to pay­ers to de­cide.

The kick­back from pay­ers is go­ing to make it much hard­er for some of these fam­i­lies to get this drug, just when they felt that the biggest hur­dle had al­ready been cleared.

How one start­up fore­told the neu­ro­science re­nais­sance af­ter '50 years of shit­show'

In the past couple of years, something curious has happened: Pharma and VC dollars started gushing into neuroscience research.

Biogen’s controversial new Alzheimer’s drug Aduhelm has been approved on the basis of removing amyloid plaque from the brain, but the new neuro-focused pharma and biotechs have much loftier aims. Significantly curbing or even curing the most notorious disorders would prove the Holy Grail for a complex system that has tied the world’s best drug developers in knots for decades.

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Bob Bradway, Amgen CEO (Scott Eisen/Bloomberg via Getty Images)

Am­gen bel­lies back up to the M&A ta­ble for an­oth­er biotech buy­out, this time with a $2.5B deal for an an­ti­body play­er fo­cused on PS­MA

Five months after Amgen CEO Bob Bradway stepped up to the M&A table and acquired Five Prime for $1.9 billion, following up with the smaller Rodeo acquisition, he’s gone back in for another biotech buyout.

This time around, Amgen is paying $900 million cash while committing up to $1.6 billion in milestones to bag the privately held Teneobio, an antibody drug developer that has expertise in developing new bispecifics and multispecifics. In addition, Amgen cited Teneobio’s “T-cell engager platform, which expands on Amgen’s existing leadership position in bispecific T-cell engagers by providing a differentiated, but complementary, approach to Amgen’s current BiTE platform.”

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Ryan Watts, Denali CEO

De­nali slips as a snap­shot of ear­ly da­ta rais­es some trou­bling ques­tions on its pi­o­neer­ing blood-brain bar­ri­er neu­ro work

Denali Therapeutics had drummed up considerable hype for their blood-brain barrier technology since launching over six years ago, hype that’s only intensified in the last 14 months following the publications of a pair of papers last spring and proof of concept data earlier this year. On Sunday, the South San Francisco-based biotech gave the biopharma world the next look at in-human data for its lead candidate in Hunter syndrome.

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Why is On­col­o­gy Drug De­vel­op­ment Re­search Late to the Dig­i­tal Bio­mark­ers Game?

During the recent Annual ASCO Meeting, thousands of cancer researchers and clinicians from across the globe joined together virtually to present and discuss the latest findings and breakthroughs in cancer research and care. There were more than 5000+ scientific abstracts presented during this event, yet only a handful involved the use of motion-tracking wearables to collect digital measures relating to activity, sleep, mobility, functional status, and/or quality of life. Although these results were a bit disappointing, they should come as no surprise to those of us in the wearable technology field.

Art Levinson (Calico)

Google-backed Cal­i­co dou­bles down on an­ti-ag­ing R&D pact with Ab­b­Vie as part­ners ante up $1B, start to de­tail drug tar­gets

Seven years after striking up a major R&D alliance, AbbVie and Google-backed anti-aging specialist Calico are doubling down on their work with a joint, $1 billion commitment to continuing their work together. And they’re also beginning to offer some details on where this project is taking them in the clinic.

According to their statement, each of the two players is putting up $500 million more to keep the labs humming.

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UP­DAT­ED: Pan­el of neu­ro­science ex­perts lays out the com­pli­ca­tions with us­ing Bio­gen's new Alzheimer's drug

Treatment of early Alzheimer’s patients with Biogen’s new drug Aduhelm should closely resemble how the drug was studied in its pivotal clinical trials, according to new recommendations from a panel of neuroscience experts led by UNLV’s Jeffrey Cummings.

“Those considering aducanumab therapy should understand that the expected benefit is slowing of cognitive and functional decline; improvement of the current clinical state is not anticipated,” they wrote Tuesday in The Journal of Prevention of Alzheimer’s Disease, noting that some of their recommendations are more specific or more restrictive than the information provided in the FDA’s prescribing information.

Busi­ness­es and schools can man­date the use of Covid-19 vac­cines un­der EUAs, DOJ says

As public and private companies stare down the reality of the Delta variant, many are now requiring that their employees or students be vaccinated against Covid-19 prior to attending school or to returning or starting a new job. Claims that such mandates are illegal or cannot be used for vaccines under emergency use authorizations have now been dismissed.

Setting the record straight, the Department of Justice on Monday called the mandates legal in a new memo, even when used for people with vaccines that remain subject to EUAs.

Ugur Sahin, BioNTech CEO (Bernd von Jutrczenka/dpa via AP Images)

BioN­Tech is spear­head­ing an mR­NA vac­cine de­vel­op­ment pro­gram for malar­ia, with a tech trans­fer planned for Africa

Flush with the success of its mRNA Covid-19 vaccine, BioNTech is now gearing up for one of the biggest challenges in vaccine development — which comes without potential profit.

The German mRNA pioneer says it plans to work on a jab for malaria, then transfer the tech to the African continent, where it will work with partners on developing the manufacturing ops needed to make this and other vaccines.

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J&J’s Rem­i­cade — the poster child for how to block biosim­i­lars — fi­nal­ly set­tles Pfiz­er suit

Biosimilars have proven time and again (although mostly in Europe) that competition works to bring down the cost of a once-pricey biologic, and can even expand its use.

J&J’s Remicade, however, has always proven to be an outlier.

Back in 2016, Pfizer won FDA approval for its infliximab biosimilar, known as Inflectra, but when the launch foundered, the company sued J&J, claiming that the company’s plan to block biosimilar competition worked incredibly well. Pfizer even went on to win FDA approval for a second infliximab biosimilar in 2017, known as Ixifi, but decided to never launch it.

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