Michel Vounatsos, Biogen CEO (Credit: World Economic Forum/Valeriano Di Domenico)

Bio­gen de­fends slow roll­out of new Alzheimer's drug, crit­i­cizes neg­a­tive me­dia at­ten­tion

As Bio­gen ex­ecs be­moaned the neg­a­tive me­dia cov­er­age around Aduhelm’s ap­proval a month ago, the biotech isn’t gain­ing much trac­tion yet in us­ing its new drug, large­ly due to a lack of in­sur­ance cov­er­age, ac­cord­ing to an earn­ings call Thurs­day.

Man­age­ment in­di­cat­ed that of the near­ly 900 sites that were prepped and ready fol­low­ing Aduhelm’s ap­proval, 325 of those, or about 35%, have com­plet­ed a pos­i­tive phar­ma­cy and ther­a­peu­tics (P&T) re­view or won’t re­quire one. The re­view is a step some hos­pi­tals or health sys­tems take pri­or to us­ing a new drug. Some ma­jor sites, how­ev­er, have said they won’t par­tic­i­pate.

Bio­gen’s top re­search chief Al San­drock al­so said on the call that he wel­comed a for­mal re­view in­to the in­ter­ac­tions be­tween FDA and Bio­gen, as “a bet­ter un­der­stand­ing is good for every­one in­volved.” He al­so of­fered an open let­ter on Thurs­day to “cor­rect some of the mis­in­for­ma­tion” around the ap­proval. For in­stance, San­drock took is­sue with the idea that ad­u­canum­ab’s tri­al re­sults are “post hoc,” al­though the com­pa­ny did stop the tri­als due to fu­til­i­ty. He al­so stressed that the ac­cel­er­at­ed ap­proval path­way, while not­ing that col­lab­o­ra­tion be­tween in­dus­try and reg­u­la­to­ry agen­cies is com­mon, ap­pro­pri­ate and ben­e­fi­cial.

A for­mer se­nior FDA of­fi­cial told End­points that any dis­cus­sion at a con­fer­ence be­tween agency of­fi­cials and a com­pa­ny on an ap­pli­ca­tion’s de­tails or tim­ing, is im­prop­er and should not hap­pen. Both STAT News and NYT al­so con­firmed sep­a­rate­ly that FDA’s neu­ro­science head Bil­ly Dunn met pri­vate­ly at a con­fer­ence to dis­cuss Aduhelm’s ap­pli­ca­tion.

San­drock al­so seemed to blame the con­tro­ver­sy around the Aduhelm ap­proval on neg­a­tive me­dia at­ten­tion. Jay Ol­son, a re­search an­a­lyst at Op­pen­heimer, opened the call’s ques­tion­ing with one about all of the “neg­a­tive me­dia” cov­er­age, which CEO Michel Vounatsos thanked him for ask­ing and said he agreed with.

Vounatsos said he does ex­pect re­gion­al Medicare MAC/Ad­van­tage plans will pro­vide cov­er­age while Medicare’s NCD re­view is on­go­ing. But the NCD will over­ride any of those lo­cal Medicare plan de­ci­sions that are in­sti­tut­ed pri­or to the NCD, said Al­isha Alaimo, pres­i­dent of Bio­gen’s US or­ga­ni­za­tion. That NCD de­ci­sion from CMS isn’t ex­pect­ed for an­oth­er 9 months.

But, ac­cord­ing to one Bio­gen em­ploy­ee, that re­gion­al cov­er­age has not hap­pened yet, and com­mer­cial in­sur­ers have been hes­i­tant to cov­er the new drug. The com­pa­ny said it brought in $2 mil­lion in quar­ter­ly Aduhelm sales, but Vounatsos said to as­sume that a “big chunk” of that $2 mil­lion is in­ven­to­ry and does not re­veal how many pa­tients have been treat­ed.

Bio­gen ex­pects “mod­est” sales in 2021 but did not pro­vide any fi­nan­cial es­ti­mates.

“It’s still ear­ly days,” Vounatsos said. “Over­all, it’s a bit slow­er than what we as­sumed, but we’re mak­ing progress.”

Al San­drock

Alaimo said Bio­gen is mak­ing every ef­fort to get in front of these lo­cal Medicare de­ci­sion mak­ers and there’s a “high lev­el of in­ter­est” in Bio­gen’s pro­gram where it pays for a test that can iden­ti­fy be­ta amy­loid in po­ten­tial pa­tients.

She not­ed that al­though no in­sur­ers have come out with a cov­er­age pol­i­cy yet, so there are no man­dates on find­ing amy­loid be­ta yet, “that’s the ex­pec­ta­tion.” There are sev­er­al ar­eas of the coun­try where ac­cess to amy­loid PET is not avail­able, she said, not­ing that Bio­gen has re­quest­ed PET and CSF tests, but the com­pa­ny is work­ing on gain­ing PET re­im­burse­ment through CMS.

Vounatsos al­so promised that Bio­gen will pub­lish its Phase III tri­al re­sults for Aduhelm in a peer-re­viewed jour­nal. Those da­ta are im­por­tant as some ex­perts are now rais­ing ques­tions about what the FDA re­leased along­side the ac­cel­er­at­ed ap­proval, and as three ad­comm mem­bers have now re­signed from the FDA pan­el.

Adap­tive De­sign Meth­ods Of­fer Rapid, Seam­less Tran­si­tion Be­tween Study Phas­es in Rare Can­cer Tri­als

Rare cancers account for 22 percent of cancer diagnoses worldwide, yet there is no universally accepted definition for a “rare” cancer. Moreover, with the evolution of genomics and associated changes in categorizing tumors, some common cancers are now characterized into groups of rare cancers, each with a unique implication for patient management and therapy.

Adaptive designs, which allow for prospectively planned modifications to study design based on accumulating data from subjects in the trial, can be used to optimize rare oncology trials (see Figure 1). Adaptive design studies may include multiple cohorts and multiple tumor types. In addition, numerous adaptation methods may be used in a single trial and may facilitate a more rapid, seamless transition between study phases.

Matt Gline (L) and Pete Salzmann

UP­DAT­ED: Roivant bumps stake in Im­muno­vant with a $200M deal. But with M&A off the ta­ble, shares crater

Roivant has worked out a deal to pick up a chunk of stock in its majority-owned sub Immunovant $IMVT, but the stock buy falls far short of its much-discussed thoughts about buying out all of the 43% of shares it doesn’t already own.

Roivant, which recently inked a SPAC move to the market at a $7 billion-plus valuation, has forged a deal to boost its ownership in Immunovant by 6.3 points, ending with 63.8% of the biotech’s stock following a $200 million injection. That cash will bolster Immunovant’s cash reserves, giving it a $600 million war chest to fund a slate of late-stage studies for its big drug: the anti-FcRn antibody IMVT-1401.

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Sanofi preps a multi­bil­lion-dol­lar buy­out of an mR­NA pi­o­neer af­ter falling be­hind in the race for a Covid-19 jab — re­port

It looks like Sanofi CEO Paul Hudson is dead serious about his intention to vault directly into contention for the future of mRNA vaccines.

A year after paying Translate Bio a whopping $425 million in an upfront and equity payment to help guide the pharma giant to the promised land of mRNA vaccines for Covid-19, Sanofi is reportedly ready to close the deal with a buyout.

Translate’s stock $TBIO soared 78% after the market closed Monday. A spokesperson for Sanofi declined to comment on the report, telling Endpoints News that the company doesn’t comment on market rumors.

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Bris­tol My­ers pulls lym­phoma in­di­ca­tion for Is­to­dax af­ter con­fir­ma­to­ry tri­al falls flat

Amid an industrywide review of cancer drugs with accelerated approval, Bristol Myers Squibb had to make the tough call last month to yank an approval for leading I/O drug Opdivo after flopping a confirmatory study. Now, a second Bristol Myers drug is on the chopping block.

Bristol Myers has pulled aging HDAC inhibitor Istodax’s indication in peripheral T cell lymphoma after a Phase III confirmatory study for the drug flopped on its progression-free survival endpoint, the drugmaker said Monday.

Rick Pazdur (via AACR)

FDA's on­col­o­gy head Rick Paz­dur de­fends the ac­cel­er­at­ed ap­proval path­way, claim­ing it is 'un­der at­tack'

The FDA is sounding the alarm over its accelerated approval pathway as backlash continues over the recent nod in favor of Biogen’s Alzheimer’s drug Aduhelm, and an ODAC meeting on six such approvals that could potentially be pulled from the market — two of which already have.

“Do you think accelerated approval is under attack? I do,” Rick Pazdur, head of FDA’s Oncology Center of Excellence, said at a Friends of Cancer Research webinar on Thursday.

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UP­DAT­ED: Watch out Glax­o­SmithK­line: As­traZeneca's once-failed lu­pus drug is now ap­proved

Capping a roller coaster journey, AstraZeneca has steered its lupus drug anifrolumab across the finish line.

Saphnelo, as the antibody will be marketed, is the only treatment that’s been approved for systemic lupus erythematosus since GlaxoSmithKline’s Benlysta clinched an OK in 2011. The British drugmaker notes it’s also the first to target the type I interferon receptor.

Mirroring the population that the drug was tested on in late-stage trials, regulators sanctioned it for patients with moderate to severe cases who are already receiving standard therapy — setting up a launch planned for the end of August, according to Ruud Dobber, who’s in charge of AstraZeneca’s biopharmaceuticals business unit.

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How the bio­phar­ma in­dus­try is help­ing to pay for the bi­par­ti­san in­fra­struc­ture bill

Senators on Sunday finalized the text of a massive, bipartisan infrastructure bill that contains little that might impact the biopharma industry other than two ways the legislators are planning to pay for the $1.2 trillion deal.

On the one hand, senators are seeking to further delay a Trump-era Medicare Part D rule related to drug rebates, this time until 2026. Senators claim the rule could end up saving about $49 billion, but the PBM industry has attacked it as it would remove rebates from a safe harbor that provides protection from federal anti-kickback laws. The pharmaceutical industry, however, is in favor of the rule and opposes this latest delay as it continues to point its finger at the PBM industry for the rising cost of out-of-pocket expenses.

Not all mR­NA vac­cines are cre­at­ed equal. Does it mat­ter?; Neu­ro is back; Pri­vate M&A af­fair; and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

As part of our broader and deeper drive, Endpoints has been pairing webinars with our special reports to cover more angles on a given topic. In conjunction with Max Gelman’s neuroscience feature, Kyle Blankenship moderated an insightful panel to discuss where the field is headed. You can register to watch it on demand here.

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FTC pulls re­main­ing case against Ab­b­Vie; New EU clin­i­cal tri­als sys­tem com­ing in 2022; Abing­worth bets big on CymaBay

The Federal Trade Commission on Friday withdrew its remaining case against AbbVie after the Supreme Court declined to review a lower court’s ruling.

The punt by SCOTUS means that while the Illinois pharma company illegally blocked patients’ access to lower-cost alternatives to its testosterone drug AndroGel, the FTC will no longer be able to return about $500 million directly to AndroGel consumers.