Alzheimer's ad­comm mem­bers ques­tion FDA: How can you use amy­loid as the ba­sis for an ac­cel­er­at­ed ap­proval?

The FDA has not yet pro­vid­ed a per­sua­sive sci­en­tif­ic ba­sis for de­clar­ing be­ta-amy­loid as a rea­son­able sur­ro­gate for an ac­cel­er­at­ed ap­proval, sev­en mem­bers of the ad­comm that re­viewed ad­u­canum­ab and unan­i­mous­ly vot­ed against its ap­proval, wrote Wednes­day in the New Eng­land Jour­nal of Med­i­cine.

Al­though the FDA has re­leased key re­views on the in­ter­nal dis­sent around the agency’s de­ci­sion to OK Bio­gen’s new con­tro­ver­sial Alzheimer’s drug, the ex­perts (three of whom have since re­signed from the ad­comm in protest over the ap­proval) ex­plained how more than two dozen ther­a­pies based on this amy­loid hy­poth­e­sis have un­der­gone late-stage clin­i­cal tri­als, and none have shown mean­ing­ful clin­i­cal ben­e­fit.

Pa­trizia Cavaz­zoni

Top FDA of­fi­cials who signed off on the ap­proval, in­clud­ing neu­ro­science head Bil­ly Dunn and CDER di­rec­tor Pa­trizia Cavaz­zoni, si­mul­ta­ne­ous­ly pub­lished a let­ter to the ed­i­tor of NE­JM on Wednes­day, not­ing they dis­agreed with the ad­comm mem­bers’ as­ser­tion that the ben­e­fit of ad­u­canum­ab is “high­ly un­cer­tain.”

They point­ed to their own JA­MA ed­i­to­r­i­al on the is­sue (Bio­gen’s da­ta has not been pub­lished in a peer-re­viewed jour­nal yet) to ex­plain how “a con­trolled dose–re­sponse clin­i­cal tri­al of ad­u­canum­ab was pos­i­tive for both clin­i­cal end points as­sess­ing men­tal func­tion­ing, and one of the two phase 3 tri­als was strong­ly pos­i­tive, show­ing sta­tis­ti­cal­ly sig­nif­i­cant ef­fects on all four end­points as­sess­ing men­tal and dai­ly life func­tion— stan­dard end points in Alzheimer’s tri­als.”

The first au­thor of the NE­JM per­spec­tive Caleb Alexan­der, pro­fes­sor at Johns Hop­kins Bloomberg School of Pub­lic Health, dis­agreed, telling End­points News that the ev­i­dence speaks for it­self, and that he’s al­ready heard peo­ple mis­stat­ing that the ev­i­dence is clear when it is not. He said the sus­tained pub­lic out­cry push­ing back on the ap­proval is a tes­ta­ment to how ir­reg­u­lar the agency’s de­ci­sion-mak­ing was.

Caleb Alexan­der

“It is hard to rec­on­cile the lack of con­vinc­ing clin­i­cal ben­e­fit across two prospec­tive ran­dom­ized tri­als with this ex­pec­ta­tion of some clin­i­cal ben­e­fit of un­known mag­ni­tude over an un­known pe­ri­od,” Alexan­der and co-au­thors wrote. They al­so not­ed that the “sci­en­tif­i­cal­ly un­jus­ti­fied la­bel” for Aduhelm may lead to bil­lions of dol­lars in un­nec­es­sary Medicare ex­pen­di­tures, even if CMS re­stricts cov­er­age in some way.

Bio­gen yes­ter­day called for a wide NCD on a pub­lic com­ment hear­ing with CMS, as well as ear­li­er pay­er cov­er­age be­fore that de­ci­sion is in the books. When that might hap­pen is un­clear, but some ma­jor med­ical cen­ters like the Cleve­land Clin­ic are de­clin­ing to use Aduhelm and some in­sur­ers are de­clin­ing cov­er­age too.

The ad­comm mem­bers al­so com­mend­ed Janet Wood­cock, in­ter­im FDA com­mis­sion­er, for her de­ci­sion to call for an Of­fice of the In­spec­tor Gen­er­al in­ves­ti­ga­tion, which they said “should be ex­pe­di­tious­ly per­formed, so as to learn how this reg­u­la­to­ry fail­ure oc­curred and to en­sure that it doesn’t oc­cur again.”

Australia’s Avance Clinical: no IND required and a 43.5% rebate on clinical spend for CGT biotechs

No IND Re­quired for Cell and Gene Ther­a­py Stud­ies with Aus­tralia’s Ac­cred­it­ed CRO Avance Clin­i­cal

Avance Clinical is the specialist Australian CRO, with CGT accreditation, for international biotechs that leverages Australia’s supportive clinical trials environment which includes no IND requirement plus a 43.5% Government incentive rebate on clinical spend.

Learn more about Avance ClinicReady here.
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The cell and gene therapies (CGT) sector offers unprecedented opportunities for patient disease management across virtually all therapeutic areas. However, finding the right accredited clinical teams to take a therapy through to the clinic and manage the regulatory process can be a major challenge for biotechs with a CGT product.

Ursula von der Leyen, President of the European Commission

Omi­cron: Re­searchers scram­ble as new coro­n­avirus mu­ta­tion takes flight around the globe — Pfiz­er/BioN­Tech, Mod­er­na vow swift re­sponse

As Americans were waking up for their Black Friday rituals, they were greeted with the news that a new mutation of the Covid-19 virus has appeared and been sequenced — after it caught an international flight to Hong Kong. And two of the leading Covid-19 vaccine developers promised delivery of a new vaccine “within 100 days” if necessary while a third spelled out its 3-prong strategy hours later.

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Thanks­giv­ing edi­tion: Top 15 End­points sto­ries of 2021; Can you name that vac­cine?; Mer­ck­'s Covid an­tivi­ral dis­ap­points; FDA nom­i­nee's in­dus­try ties; 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.

Happy Thanksgiving to all those who are celebrating it — although, if we are being honest, this week’s abbreviated edition is really for those who are not. Wherever you’re tuning in from, we appreciate your support, hope you find this recap helpful and we wish you a wonderful weekend.

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Troy Wilson, Kura CEO

UP­DAT­ED: FDA hits the red light on an ear­ly-stage AML study af­ter a pa­tient dies

The FDA has slapped a clinical hold on the early-stage program for one of Kura Oncology’s cancer drugs following a patient’s death in a clinical trial.

The biotech $KURA reported early Wednesday that the Phase Ib study of KO-539 for acute myeloid leukemia would be halted, suspending enrollment, while researchers and the FDA probed the death. Patients already on the drug can continue taking it.

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What were End­points read­ers tun­ing in­to this year? Here’s a look at our 15 most pop­u­lar re­ports of the year (so far)

At the beginning of this year, I laid out a basic objective for Endpoints News as we headed to our 5th anniversary. We’ve long been doing a fine job covering the breaking news in R&D — if I do say so myself — but we needed to expand our horizons on industry coverage, increase the staff and go much, much deeper when the stories demanded it.

In a phrase: broader and deeper.

It’s safe to say, based on our daily web traffic, that you all seemed to like this idea. We’ve doubled the staff — thanks to a growing group of paid subscribers — ramped up the daily report and now publish a regular slate of in-depth articles. And traffic — those clicks you always read about — have gone up in volume too. Monthly sessions are up 43%, to close to 1.5 million. Unique readers are up 63%, to 874,480 in October, after setting a record of close to a million the month before. Page views are running at 3 million-plus a month. And the overall number of subscribers has surged to 124,000.

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Mar­ket­ingRx roundup: Am­gen, Lil­ly, Bio­haven mi­graine brand re­call low, study says; No­var­tis looks to re­make drug launch mod­el

Forget the migraine marketing brand wars. When it comes to patients, many can’t even name one despite substantial advertising efforts, according to a new study from Phreesia that concludes CGRP migraine drugmakers still need to work on brand recognition.

Almost half (47%) of the patients Phreesia surveyed couldn’t name one preventative migraine brand. The best performer was Topamax, a small molecule anticonvulsant that’s been around since 2004, which 26% of migraine patients could recall. Among the new CGRP brand names recognized, Amgen’s Aimovig ranked highest with 8% recall, while Eli Lilly’s Emgality and Biohaven’s Nurtec tied at 7% and Teva’s Ajovy was remembered by 3% of patients.

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Emma Walmsley, GlaxoSmithKline CEO (Fang Zhe/Xinhua/Alamy Live News)

Glax­o­SmithK­line places a risky bet on Ar­row­head­'s RNA drug in the fail­ure-strewn NASH field

As activist investors champ at the bit for change at drug giant GlaxoSmithKline, the pharma giant has turned over many rocks to find an R&D success to present to its detractors. In NASH, a field strewn with failures, GSK hopes a new license deal can churn out a much-needed winner.

GSK will pay $120 million in upfront cash and $910 million in downstream milestones to develop and sell ARO-HSD, Arrowhead Pharmaceuticals’ RNA interference drug targeting fatty liver disease nonalcoholic steatohepatitis (NASH), the companies said Monday.

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Name that vac­cine: From Comir­naty to Spike­vax to Nu­vax­ovid, Covid-19 shot­s' brand names re­main lit­tle-known

Most people know if they’re “Team Pfizer” or “Team Moderna,” but few know if they got the Comirnaty or Spikevax Covid-19 vaccine. Those are the brand names of Pfizer and Moderna vaccines, respectively, however they have yet to take hold with consumers, media or even medical professionals.

And there are others. Covid vaccine brand names also include AstraZeneca’s Vaxzevria, Novavax’s Nuvaxovid, and Sanofi and GlaxoSmithKline’s Vidprevtyn. J&J’s Janssen-developed Covid vaccine is the lone major holdout and is still yet to be named, if ever. In EMA filings approving its conditional use, the brand name is listed simply as “Covid-19 Vaccine Janssen.”

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Joan Perelló, Sanifit CEO

Joan Perel­ló set out 17 years ago to de­vel­op a drug. And to­day he's be­ing re­ward­ed with a $424M biotech buy­out

Joan Perelló beat all the odds with his little Spanish biotech startup Sanifit.

Working on the far perimeter of the big US/European drug development scene, he took a drug born out of his PhD work and got enough seed cash to get started. That’s one near miracle. In the second near miracle he gathered a previously unheard of venture raise in Spain — helping build an industry ecosystem from scratch — to pursue a successful search for solid human data for his drug, SNF472. And while gathering a virtual team of developers from Europe and the US, the CEO/co-founder steered it into the late-stage arena.

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