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.

Ryan Watts, Denali CEO

Bio­gen hands De­nali $1B-plus in cash, $1B-plus in mile­stones to part­ner on late-stage Parkin­son’s drug

Biogen is handing over more than a billion dollars cash to partner with the up-and-coming neurosciences crew at Denali on a new therapy for Parkinson’s. And the big biotech is ready to pile on more than a billion dollars more in milestones — if the alliance is a success.

For Biogen $BIIB, the move on Denali’s small molecule inhibitors of LRRK2 puts them in line to collaborate on a late-stage program for DNL151, which is scheduled to start next year.

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Michel Vounatsos, Biogen CEO (via YouTube)

UP­DAT­ED: Bio­gen scores a pri­or­i­ty re­view for its Alzheimer's drug ad­u­canum­ab, mov­ing one gi­ant leap for­ward in its con­tro­ver­sial quest

Biogen scored a big win at the FDA today as regulators accepted their application for the controversial Alzheimer’s drug aducanumab and gave it a priority review.

The PDUFA date is March 7, 2021.

Significantly, Biogen says it did not use its priority review voucher to win special treatment at the FDA. The agency handed that out gratis.

That’s the ideal scenario Biogen was looking for as disappointed analysts wondered aloud about the delayed application earlier in the year.

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Levi Garraway, Roche CMO (Source: Genentech)

UP­DAT­ED: FDA hands out a quick OK for po­ten­tial SMA block­buster ris­diplam, giv­ing Genen­tech and Roche a chance to chal­lenge ri­vals on the price

US regulators handed Roche and Genentech a big win Friday afternoon, one that has market-shaping potential for its high-priced rivals from Novartis and Biogen.

The FDA has green-lit the companies’ spinal muscular atrophy drug risdiplam, which will be marketed as Evrysdi in the US, for use in patients two months and older. It’s the first SMA drug that can be taken orally, as Biogen’s Spinraza is injected into the spine while Novartis’ Zolgensma is a gene therapy.

Moncef Slaoui, Getty Images

When will it end? Big Phar­ma's top vac­cine ex­pert at OWS of­fers a speedy time­line for a Covid-19 vac­cine — ei­ther be­fore or right af­ter the elec­tion

Moncef Slaoui hasn’t started making plans for his summer vacation next year. But he offers high odds that all Americans will be able to do that in the not too distant future.

In an interview with a pair of sympathetic podcasters at the conservative American Enterprise Institute, Slaoui provides an education to listeners on how any drug or vaccine can be sped through trials. And he leaves the door wide open to the notion that the leading vaccine developers can demonstrate efficacy and safety in a compelling fashion as early as October — or as late as the end of this year.

President Trump (AP Images)

UP­DAT­ED: FDA takes the lead on defin­ing es­sen­tial un­der Trump's 'Buy Amer­i­can' ex­ec­u­tive or­der — as phar­ma warns of sup­ply chain dis­rup­tion

President Donald Trump has signed an executive order detailing how the federal government should help on-shore drug manufacturing — and the FDA will play a central role.

The agency now has three months to draw up the list of “essential medicines, medical countermeasures, and their critical inputs” that the US must have available at all times. Various departments and agencies are then directed to buy these drugs and their ingredients from American manufacturers.

Covid-19 roundup: Gates Foun­da­tion pours $150M in­to In­dia’s Serum In­sti­tute; Pfiz­er teams with Gilead on remde­sivir

By CEO and scion Adar Poonawalla’s estimation, the Serum Institute in India has already poured hundreds of millions of dollars into scaling up the unproven Covid-19 vaccine being developed by AstraZeneca and Oxford for use in low and middle income countries. It’s meant taking on a risk that other companies, including AstraZeneca, have mitigated with huge amounts of government funding.

Now, for the first time, Poonawalla is getting some outside help. The Gates Foundation has agreed to pay the institute $150 million to supply 100 million vaccines to India and other emerging economies next year, Reuters reported. That includes both the vaccine being developed by AstraZeneca and the one being developed by Novavax. Those vaccines will be available in 92 countries and be priced at $3 per dose.

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UP­DAT­ED: No­vavax her­alds the lat­est pos­i­tive snap­shot of ear­ly-stage Covid-19 vac­cine — so why did its stock briefly crater?

High-flying Novavax $NVAX became the latest of the Covid-19 vaccine players to stake out a positive set of biomarker data from its early-stage look at its vaccine in humans.

Their adjuvanted Covid-19 vaccine was “well-tolerated and elicited robust antibody responses numerically superior to that seen in human convalescent sera,” the company noted. According to the biotech:

All subjects developed anti-spike IgG antibodies after a single dose of vaccine, many of them also developing wild-type virus neutralizing antibody responses, and after Dose 2, 100% of participants developed wild-type virus neutralizing antibody responses. Both anti-spike IgG and viral neutralization responses compared favorably to responses from patients with clinically significant COVID‑19 disease. Importantly, the IgG antibody response was highly correlated with neutralization titers, demonstrating that a significant proportion of antibodies were functional.

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Bio­haven adds near­ly $1B in Nurtec deals with Roy­al­ty Phar­ma, Sixth Street

Biohaven just added nearly $1 billion to their balance sheet.

On Friday morning, the neuroscience biotech announced a pair of creative agreements with Royalty Pharma and the investment firm Sixth Street to bolster the commercial launch of their new migraine drug, Nurtec. Biohaven will sell a sliver of its royalties on Nurtec and 3% of the royalties on their experimental migraine drug zavegepant to Royalty Pharma as part of a larger agreeement that will pay $450 million. At the same time, the company announced they took out a $500 million loan from Sixth Street.

Ab­b­Vie set­tles in­sur­ance fraud suit, agrees to tweak nurse am­bas­sador pro­gram; CStone aims for NSCLC OK with pos­i­tive PhI­II da­ta

AbbVie has resolved a California lawsuit alleging insurance fraud in the promotion of its cash cow Humira, paying $24 million to settle things with the state’s insurance regulator.

The settlement comes almost four years after a whistleblower first reported AbbVie’s practice of deploying registered nurses to visit patients at home or call them by phone to ensure that Humira prescriptions are filled. AbbVie was also charged with providing illegal kickbacks to doctors in an attempt to encourage them to prescribe Humira for a range of anti-inflammatory diseases.