Bio­gen's new Alzheimer's drug serves as a ral­ly­ing cry for drug pric­ing re­forms

A coali­tion of em­ploy­ers and health care pur­chasers are call­ing on the top House and Sen­ate lead­ers to take a hard look at the “as­tro­nom­i­cal price” of Bio­gen’s new Alzheimer’s drug Aduhelm and “move bold­ly” to en­act pre­scrip­tion drug pric­ing re­form.

The group Em­ploy­er­sRx, which in­cludes the Amer­i­can Ben­e­fits Coun­cil and the Na­tion­al Al­liance of Health­care Pur­chas­er Coali­tions among oth­ers, says that while it strong­ly sup­ports mar­ket so­lu­tions to es­tab­lish rea­son­able prices, when no mar­ket ex­ists — as is the case for ad­u­canum­ab — “we sup­port al­low­ing the fed­er­al gov­ern­ment to en­ter in­to mean­ing­ful ne­go­ti­a­tions on the price of drugs on be­half of all pay­ers.”

Al­low­ing the gov­ern­ment to ne­go­ti­ate prices has been a ma­jor stick­ing point for in­dus­try groups and lob­by­ists, who con­tend that such ne­go­ti­a­tions are akin to price con­trols and will se­vere­ly con­strict the bio­phar­ma in­dus­try’s abil­i­ty to win over new VC fund­ing and take risks on po­ten­tial new treat­ments.

But if 1 mil­lion Medicare ben­e­fi­cia­ries re­ceive Aduhelm, which may be on the low­er end of Bio­gen’s ex­pec­ta­tions, ac­cord­ing to the Kaiser Fam­i­ly Foun­da­tion, spend­ing on Aduhelm would sur­pass spend­ing on all oth­er Part B-cov­ered drugs com­bined. The US in­sur­er for se­niors may end up spend­ing more on Aduhelm than on the CDC, or even NASA.

Em­ploy­er­sRx al­so notes that Medicare’s Part B pay­ment pol­i­cy pro­vides a fi­nan­cial in­cen­tive for clin­i­cians to pre­scribe high­er-cost ther­a­peu­tic drugs, which may work in Bio­gen’s, but not Medicare’s, fa­vor. Wall Street biotech an­a­lysts, how­ev­er, have said Aduhelm sales like­ly won’t reach $7 bil­lion per year un­til 2025 (and Bio­gen has un­til 2030 to com­plete its con­fir­ma­to­ry tri­al).

“Ne­go­ti­at­ed prices should align with clin­i­cal ef­fi­ca­cy, con­sid­er the price of the drug in oth­er in­dus­tri­al­ized coun­tries, and pro­tect true in­no­va­tion,” Em­ploy­er­sRx said in its let­ter on Mon­day to House Speak­er Nan­cy Pelosi (D-CA), House mi­nor­i­ty leader Kevin Mc­Carthy (R-CA), Sen­ate ma­jor­i­ty leader Chuck Schumer (D-NY) and Sen­ate mi­nor­i­ty leader Mitch Mc­Connell (R-KY). “To en­sure that these poli­cies do not re­sult in cost-shift­ing to pri­vate pay­ers, and to pro­vide di­rect re­lief to fam­i­lies cov­ered by pri­vate in­sur­ance, it is es­sen­tial that any poli­cies to di­rect­ly man­age drug prices ex­tend to all pay­ers, not just pub­lic pro­grams.”

The coali­tion of em­ploy­ers and pur­chasers al­so raised con­cerns about the im­pli­ca­tions of this ap­proval at the FDA, as, “if drug man­u­fac­tur­ers know that the FDA will ap­prove drugs with lit­tle-to-no val­ue over cur­rent treat­ments, they will lose re­al in­cen­tives to de­vel­op tru­ly in­no­v­a­tive, life­sav­ing med­ica­tions.”

“The un­prece­dent­ed ap­proval and un­jus­ti­fied pric­ing of ad­u­canum­ab pro­vides a cau­tion­ary tale of a bro­ken sys­tem that is in pro­found need for re­form,” the let­ter says.

The let­ter is part of a grow­ing out­cry over the de­ci­sion from the FDA and its im­pli­ca­tions. Non­prof­it watch­dog Pub­lic Cit­i­zen last week called for the top three FDA of­fi­cials re­spon­si­ble for the ap­proval, in­clud­ing act­ing com­mis­sion­er Janet Wood­cock, to be re­moved from their po­si­tions.

Marc Good­man

But SVB Leerink an­a­lyst Marc Good­man ex­plained re­cent­ly in a note to in­vestors how many un­knowns there are still on the launch of Aduhelm.

“We don’t know how wide­spread physi­cians will pre­scribe with­in the spec­trum of AD pa­tients. We don’t know how long physi­cians will rec­om­mend that pa­tients stay on the drug or pay­ers will re­im­burse treat­ment. What is the right pric­ing giv­en the com­pli­cat­ed dos­ing titra­tion? Will physi­cians titrate dos­ing in line with la­bel­ing rec­om­men­da­tions? We don’t know yet how dif­fi­cult CMS and oth­er pay­ers (e.g., Medicare Ad­van­tage plans) will make it for re­im­burse­ment,” he wrote. “How many pa­tients will dis­con­tin­ue treat­ment over time? And how will the mar­ket fac­tor in fu­ture com­pe­ti­tion from the Ei­sai and Lil­ly drugs that have the same mech­a­nism and ap­pear to be more ro­bust in clear­ing amy­loid than Adu?”

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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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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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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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.

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.