Bio­gen push­es for broad CMS cov­er­age of Alzheimer's drug — while Eli Lil­ly looks to es­cape an NCD

The nine-month quest for CMS to fig­ure out how (or how not) to cov­er Bio­gen’s new Alzheimer’s drug Aduhelm, and po­ten­tial­ly more amy­loid-tar­get­ed mon­o­clon­al an­ti­bod­ies, kicked off in earnest on Tues­day with an open pub­lic hear­ing of com­ments from var­i­ous stake­hold­ers stress­ing the pros and cons of cov­er­ing this new, pricey drug with un­known clin­i­cal ben­e­fits.

Bio­gen and its af­fil­i­ates stressed the need for a quick and pos­i­tive Na­tion­al Cov­er­age De­ter­mi­na­tion from CMS as oth­er physi­cians and a speak­er from ICER called on CMS to not cov­er the drug or to lim­it cov­er­age in cer­tain ways as Bio­gen’s clin­i­cal tri­als didn’t show strong signs of clin­i­cal ben­e­fit, the $56,000 an­nu­al price tag doesn’t match those ben­e­fits, and as the tri­als didn’t ad­dress a di­verse group of pop­u­la­tions, like African Amer­i­cans or Lat­inx, who are fre­quent­ly af­fect­ed by Alzheimer’s.

While an all-out re­jec­tion in cov­er­age from CMS seems un­like­ly giv­en the his­to­ry of the cen­ters’ pri­or NCDs on CAR-T ther­a­pies and oth­er treat­ments and de­vices, Bio­gen opened Tues­day’s call with the first two com­ments from ex­ec­u­tives re­it­er­at­ing the ne­ces­si­ty for wide­spread and ear­ly cov­er­age.

Ma­ha Rad­hakr­ish­nan

“Ther­a­py should not be lim­it­ed to aca­d­e­m­ic cen­ters as many un­der­served and un­der-rep­re­sent­ed pa­tients don’t have ac­cess to these cen­ters,” Ma­ha Rad­hakr­ish­nan, CMO at Bio­gen, said.

An ear­li­er Bio­gen ex­ec called on the agency to en­cour­age MACs to cov­er Aduhelm at the lo­cal lev­el while the NCD process is on­go­ing, echo­ing com­ments from an anony­mous Bio­gen em­ploy­ee who told End­points News last week that he de­clined to use his re­la­tion­ship with a large aca­d­e­m­ic med­ical cen­ter to en­cour­age lo­cal Medicare pol­i­cy­mak­ers to cov­er Aduhelm as he didn’t think it was eth­i­cal.

But it was in the writ­ten com­ments (see be­low) where a strong force of aca­d­e­mics and oth­er Alzheimer’s re­searchers called on Medicare to do what FDA didn’t do, and re­ject cov­er­age for Aduhelm

Not all amy­loid drugs?

But it was an Eli Lil­ly doc­tor who called on CMS to not make any rash de­ci­sion that pre­ma­ture­ly short­changes the en­tire class of amy­loid-tar­get­ed drugs.

Lil­ly said late last month it’s fil­ing its amy­loid-tar­get­ed do­nanemab with the FDA lat­er this year based on Phase II da­ta now that the FDA con­sid­ers amy­loid clear­ance rea­son­ably like­ly to lead to cog­ni­tive ben­e­fit.

Brandy Matthews

Brandy Matthews, a neu­rol­o­gist and se­nior med­ical ad­vis­er to Lil­ly, not­ed the com­pa­ny’s “con­cern” at the out­set of its pub­lic com­ment that a class-based NCD “might be pre­ma­ture at this point” be­cause dif­fer­ent mon­o­clon­al an­ti­bod­ies are at dif­fer­ent stages of de­vel­op­ment and ev­i­dence might be­come avail­able dur­ing or af­ter the NCD.

Each Alzheimer’s drug should be eval­u­at­ed on its own amy­loid plaque re­duc­tion, safe­ty pro­file and clin­i­cal ben­e­fit, Matthews said. CMS cov­er­age poli­cies to eval­u­ate ther­a­peu­tics should oc­cur for each one, she said while urg­ing CMS for flex­i­bil­i­ty that does not lim­it ac­cess to fu­ture ther­a­pies.

“Lim­i­ta­tions on the first ap­proved ther­a­py should not au­to­mat­i­cal­ly ap­ply to fu­ture ther­a­pies,” she said.

As Bio­gen-fund­ed aca­d­e­mics sig­naled how to use Aduhelm on Tues­day, Matthews called on CMS to use the en­roll­ment cri­te­ria for each clin­i­cal tri­al as the start­ing point for cov­er­age.

Flood of neg­a­tive writ­ten com­ments

Out­side of Tues­day’s call, a pile-up of writ­ten com­ments on the na­tion­al cov­er­age analy­ses shows just how many think FDA was wrong to ap­prove Aduhelm and that CMS should not pro­vide cov­er­age.

Roger Al­bin, a pro­fes­sor of neu­rol­o­gy at the Uni­ver­si­ty of Michi­gan, wrote, “The da­ta sup­port­ing util­i­ty of ad­u­canum­ab for Alzheimer Dis­ease isn’t even weak. The FDA should nev­er have ap­proved this agent. CMS should de­cline to pro­vide cov­er­age for ad­u­canum­ab treat­ment.”

James Lah, di­rec­tor of the Cog­ni­tive Neu­rol­o­gy Pro­gram at Emory Uni­ver­si­ty School of Med­i­cine, added, “The FDA made sev­er­al mis­takes in re­view­ing ad­u­canum­ab, but per­haps fore­most was their fail­ure to re­quest the ad­vi­so­ry pan­el con­sid­er the op­tion of Ac­cel­er­at­ed Ap­proval. Had they done so, I sus­pect there would have been fruit­ful dis­cus­sion to in­form the many de­ci­sions now left to us as providers and pay­ors to an­swer.”

“The fact that this drug was FDA ap­proved is a trav­es­ty; please do not make it worse. The mon­ey that would cov­er this drug could go to much bet­ter use for those pa­tients and care­givers,” wrote An­drea Bial, pro­gram di­rec­tor at the Loy­ola-Hines Geri­atric Med­i­cine Fel­low­ship Pro­gram.

Bri­enne Min­er, as­so­ciate pro­fes­sor of geri­atrics at Yale Uni­ver­si­ty, wrote, “I am deeply trou­bled by the pres­sure to use an un­proven and po­ten­tial­ly dan­ger­ous treat­ment in our pa­tients, many of whom are des­per­ate to try any­thing that might give them hope. I don’t see how they can re­al­ly make an in­formed de­ci­sion here.”

Saman­tha Hold­en, an as­sis­tant pro­fes­sor in the De­part­ment of Neu­rol­o­gy at the Uni­ver­si­ty of Col­orado, al­so not­ed, “As a cog­ni­tive neu­rol­o­gist and de­men­tia spe­cial­ist, I have long wait­ed for an ef­fec­tive dis­ease-mod­i­fy­ing ther­a­py for my pa­tients. I have not been con­vinced that ad­u­canum­ab is that drug. I am ex­treme­ly con­cerned re­gard­ing the pre­sent­ed da­ta, con­flict­ing re­sults be­tween EN­GAGE and EMERGE, the costs, and po­ten­tial to in­crease in­equities in neu­ro­log­i­cal care in this coun­try.”

Michael Car­ome, di­rec­tor of the non­prof­it Pub­lic Cit­i­zen’s Health Re­search Group, al­so called on CMS to ex­clude cov­er­age for Aduhelm, ar­gu­ing that the drug “can­not pos­si­bly be deemed rea­son­able and nec­es­sary” as “based on the cur­rent­ly avail­able sci­en­tif­ic ev­i­dence, re­duc­tion of amy­loid-be­ta plaques in the brain it­self is not a clin­i­cal­ly mean­ing­ful out­come.”

Mov­ing for­ward, the ini­tial writ­ten pub­lic com­ment pe­ri­od ends on Au­gust 11, and then CMS will re­view those com­ments over 6 months in de­vel­op­ing a pro­posed NCD and mem­o­ran­dum. No lat­er than Jan. 12, 2022, the pro­posed NCD will be avail­able, and the pub­lic will have one more 30-day com­ment pe­ri­od. A fi­nal NCD will be com­plet­ed no lat­er than April 12, 2022, CMS said Tues­day.

Medicare pay­ment rates and codes are gen­er­at­ed out­side the NCD process.

M&A: a crit­i­cal dri­ver for sus­tain­able top-line growth in health­care

2021 saw a record $600B in healthcare M&A activity. In 2022, there is an anticipated slowdown in activity, however, M&A prospects remain strong in the medium to long-term. What are future growth drivers for the healthcare sector? Where might we see innovations that drive M&A? RBC’s Andrew Callaway, Global Head, Healthcare Investment Banking discusses with Vito Sperduto, Global Co-Head, M&A.

15 LGBTQ lead­ers in bio­phar­ma; Paul Stof­fels’ Gala­pa­gos re­vamp; As­traZeneca catch­es up in AT­TR; 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.

A return to in-person conferences also marks a return to on-the-ground reporting. My colleagues Beth Synder Bulik and Nicole DeFeudis were on-site at Cannes Lions, bringing live coverage of pharma’s presence at the ad festival — accompanied by photos from Clara Bui, our virtual producer, that bring you right to the scene. You can find a recap (and links to all the stories) below.

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David Loew (Ipsen)

Ipsen snags an ap­proved can­cer drug in $247M M&A deal as an­oth­er bat­tered biotech sells cheap

You can add Paris-based Ipsen to the list of discount buyers patrolling the penny stock pack for a cheap M&A deal.

The French biotech, which has had plenty of its own problems to grapple with, has swooped in to buy Epizyme $EPZM for $247 million in cash and a CVR with milestones attached to it. Epizyme shareholders, who had to suffer through a painfully soft launch of their EZH2a inhibitor cancer drug Tazverik, will get $1.45 per share along with a $1 CVR tied to achieving $250 million in sales from the drug over four consecutive quarters as well as an OK for second-line follicular lymphoma by 1 Jan. 2028.

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AstraZeneca's new Evusheld direct to consumer campaign aims to reach more immunocompromised patients.

As­traZeneca de­buts first con­sumer cam­paign for its Covid-19 pro­phy­lac­tic Evusheld — and a first for EUA drugs

AstraZeneca’s first consumer ad for Evusheld is also a first for drugs that have been granted emergency use authorizations during the pandemic.

The first DTC ad for a medicine under emergency approval, the Evusheld campaign launching this week aims to raise awareness among immunocompromised patients — and spur more use.

Evusheld nabbed emergency authorization in December, however, despite millions of immunocompromised people looking for a solution and now more widespread availability of the drug.

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De­spite a slow start to the year for deals, PwC pre­dicts a flur­ry of ac­tiv­i­ty com­ing up

Despite whispers of a busy year for M&A, deal activity in the pharma space is actually down 30% on a semi-annualized basis, according to PwC’s latest report on deal activity. But don’t rule out larger deals in the second half of the year, the consultants said.

PwC pharmaceutical and life sciences consulting solutions leader Glenn Hunzinger expects to see Big Pharma companies picking up earlier stage companies to try and fill pipeline gaps ahead of a slew of big patent cliffs. Though a bear market continues to maul the biotech sector, Hunzinger said recent deals indicate that pharma companies are still paying above current trading prices.

Joe Wiley, Amryt Pharma CEO

Am­ryt Phar­ma sub­mits a for­mal dis­pute res­o­lu­tion to the FDA over re­ject­ed skin dis­ease drug

The story of Amryt Pharma’s candidate for the genetic skin condition epidermolysis bullosa, or EB, will soon enter another chapter.

After the Irish drugmaker’s candidate, dubbed Oleogel-S10 and marketed as Filsuvez, was handed a CRL earlier this year, the company announced in a press release that it plans to submit a formal dispute resolution request for the company’s NDA for Oleogel-S10.

Sen. Thom Tillis (R-NC) (J. Scott Applewhite/AP Images)

Phar­ma-friend­ly sen­a­tor calls on FDA for a third time to show patent pro­tec­tions should­n't be blamed for high drug prices

North Carolina Republican Sen. Thom Tillis made a name for himself in the 2020 election cycle as the darling of the pharma industry, accepting hundreds of thousands in campaign contributions, even from the likes of Pfizer CEO Albert Bourla.

Those contributions have led Tillis to attempt to re-write patent laws in pharma’s favor, a move which failed to gain steam in 2019, and request for a third time since January that the FDA should help stop “the false narrative that patent protections are to blame for high drug prices.”

EMA signs off on 3 drugs re­cent­ly re­ject­ed by FDA, in­clud­ing Bio­Mar­in's new he­mo­phil­ia gene ther­a­py

The EMA’s human medicines committee on Friday recommended three new drugs for approval or conditional approval, even as their US counterparts have rejected these three for various reasons.

In a major move, CHMP offered a thumbs-up to a conditional marketing authorization for the first gene therapy to treat severe hemophilia A, although the agency cautioned that it’s so far unknown how long the effects of infusion will last.

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Joe Papa (Ryan Remiorz/The Canadian Press via AP, File)

Joe Pa­pa re­signs as chair of Bausch Health as bil­lion­aire John Paul­son takes over

Joe Papa, chair of Bausch Health, officially resigned on Thursday and the board appointed billionaire hedge fund manager John Paulson as the new chair, effective immediately.

The specialty pharma company sought to make clear that Papa’s abrupt departure “was not due to any dispute or disagreement with the Company, its management or the Board on any matter relating to the Company’s operations, policies or practices.”

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