Ex­perts urge trans­paren­cy, ad­vi­so­ry com­mit­tee re­view for Covid-19 vac­cines

At a vir­tu­al brief­ing on Tues­day, sev­er­al ex­perts told mem­bers of the House Com­mit­tee on Over­sight and Re­form Sub­com­mit­tee on Eco­nom­ic and Con­sumer Pol­i­cy that a trans­par­ent process and ad­vi­so­ry com­mit­tee re­view will be key to the ef­fec­tive roll­out of a vac­cine to pre­vent coro­n­avirus dis­ease.

The brief­ing comes just two weeks af­ter the FDA is­sued guid­ance on the de­vel­op­ment and li­cen­sure of vac­cines against SARS-CoV-2, the virus that caus­es Covid-19, and at a time when some of the most ad­vanced vac­cine can­di­dates are mov­ing in­to Phase 3 tri­als.

Bruce Gellin

Dur­ing the brief­ing, Bruce Gellin, pres­i­dent, glob­al im­mu­niza­tion at the Sabin Vac­cine In­sti­tute, said that FDA’s re­cent guid­ance helps pro­vide trans­paren­cy about its de­ci­sion-mak­ing process for an even­tu­al Covid-19 vac­cine.

“Guid­ances like these, while di­rect­ed to in­dus­try, are re­al­ly for all to see, so all know the con­sid­er­a­tions that go in­to the process,” Gellin said, warn­ing that cut­ting cor­ners in the race to ap­prove a vac­cine would be a “mis­step” with “se­ri­ous im­pli­ca­tions,” such as fu­el­ing vac­cine skep­ti­cism and erod­ing the pub­lic’s trust in FDA’s ap­proval process.

“I strong­ly rec­om­mend that any vac­cine that’s be­ing con­sid­ered for any type of ap­proval by the FDA be re­viewed in an open pub­lic meet­ing by FDA’s Vac­cine and Re­lat­ed Bi­o­log­i­cal Prod­ucts Ad­vi­so­ry Com­mit­tee [VRB­PAC],” Gellin said, not­ing his past par­tic­i­pa­tion on the com­mit­tee.

“With­out such a pub­lic re­view, I have no doubt that sto­ries will ap­pear about vac­cine ap­provals made be­hind closed doors be­cause the gov­ern­ment didn’t want the pub­lic to see what or who may have in­flu­enced those de­ci­sions,” he said.

Ruth Kar­ron

Two oth­er ex­perts, Ruth Kar­ron, di­rec­tor of the Cen­ter for Im­mu­niza­tion Re­search at Johns Hop­kins Bloomberg School of Pub­lic Health, and Ja­son Schwartz, as­sis­tant pro­fes­sor of health pol­i­cy at the Yale School of Pub­lic Health, con­curred.

Kar­ron, who pre­vi­ous­ly chaired the com­mit­tee, said that VRB­PAC’s “ad­vice will be crit­i­cal as the FDA re­views vac­cine tri­al da­ta and makes de­ci­sions about emer­gency use au­tho­riza­tions (EUAs) and li­cen­sure.”

Schwartz added that both VRB­PAC and the Cen­ters for Dis­ease Con­trol and Pre­ven­tion’s Ad­vi­so­ry Com­mit­tee on Im­mu­niza­tion Prac­tices (ACIP) have helped to sup­port the gov­ern­ment’s reg­u­la­to­ry and pol­i­cy­mak­ing work for vac­cines for decades.

“The clos­er that our gov­ern­ment health agen­cies ad­here to their well-es­tab­lished, time test­ed process­es for eval­u­at­ing the safe­ty and ef­fec­tive­ness of Covid-19 vac­cines and de­vel­op­ing ev­i­dence-based ap­proach­es for their de­ploy­ment, the more con­fi­dence the pub­lic can have in the in­tegri­ty and qual­i­ty of those de­ci­sions and those vac­cines,” Schwartz said.

Ap­provals and EUAs

Jesse Good­man

Jesse Good­man, di­rec­tor of the Cen­ter on Med­ical Prod­uct Ac­cess, Safe­ty and Stew­ard­ship at George­town Uni­ver­si­ty and for­mer chief sci­en­tist at FDA, said he thinks the 50% ef­fec­tive­ness thresh­old set in FDA’s guid­ance is “a rea­son­able start­ing point.”

Good­man al­so said that with ad­e­quate cau­tion, an EUA or ex­pand­ed ac­cess pro­to­col could pro­vide ear­ly ac­cess to a vac­cine.

“We need to be very thought­ful about any vac­cine ac­cess pri­or to ap­proval. There may be cir­cum­stances where use of an un­ap­proved vac­cine could be ap­pro­pri­ate in a se­vere out­break. For ex­am­ple, if a vac­cine with doc­u­ment­ed safe­ty and promis­ing ef­fec­tive­ness were avail­able, but all the da­ta need­ed [was] not yet sub­mit­ted, FDA could pro­vide ac­cess through an ex­pand­ed ac­cess pro­gram or through an emer­gency use au­tho­riza­tion,” Good­man said.

Ja­son Schwartz

Both Good­man and Schwartz raised con­cerns that the pub­lic might not un­der­stand the dif­fer­ence be­tween an EUA and full ap­proval.

“I would be con­cerned … that an emer­gency use au­tho­riza­tion would not be un­der­stood among the pub­lic to be dif­fer­ent from the full ap­proval with all the de­tails from the FDA,” Schwartz said, be­fore adding that is­su­ing an EUA pri­or to hav­ing Phase 3 da­ta in hand would be a “very se­ri­ous gam­ble.”

Good­man sug­gest­ed that FDA re­quire in­formed con­sent as part of the process for ad­min­is­ter­ing a vac­cine un­der an EUA and said that such use should be “tar­get­ed to those at high­est risk of in­fec­tion and com­pli­ca­tions.”

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Reimagining cancer treatments

Cancer is a leading cause of death worldwide, accounting for nearly 10 million deaths in 2020, which is nearly one in six deaths. Recently, we have seen incredible advances in novel cancer therapies such as immune checkpoint inhibitors, cell therapies, and antibody-drug conjugates that have revamped cancer care and improved survival rates for patients.

Despite this significant progress in therapeutic targeting, why are we still seeing such a high mortality rate? The reason is that promising therapies are often limited by their therapeutic index, which is a measure of the effective dose of a drug, relative to its safety. If we could broaden the therapeutic indices of currently available medicines, it would revolutionize cancer treatments. We are still on the quest to find the ultimate cancer medicine – highly effective in several cancer types, safe, and precisely targeted to the tumor site.

Justin Klee (L) and Joshua Cohen, Amylyx co-CEOs (Cody O'Loughlin/The New York Times; courtesy Amylyx)

Ad­vo­cates, ex­perts cry foul over Amy­lyx's new ALS drug, cit­ing is­sues with price, PhI­II com­mit­ment

Not 24 hours after earning the first ALS drug approval in five years, Amylyx Pharmaceuticals’ Relyvrio is already drawing scrutiny. And it’s coming from multiple fronts.

In an investor call Friday morning, Amylyx revealed that it would charge about $158,000 per year, a price point that immediately drew backlash from ALS advocates and some outside observers. The cost reveal had been highly anticipated in the immediate hours after Thursday evening’s approval, though Amylyx only teased Relyvrio would cost less than previously approved drugs.

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Land­mark Amy­lyx OK spurs de­bate; Some... pos­i­tive? Alzheimer's da­ta; Can­cer tri­al bot­tle­neck; Sanofi's CRISPR bet; 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.

After brief stops in Paris and Boston, John Carroll and the Endpoints crew are staying on the road in October with their return for a live/streaming EUBIO22 in London. The hybrid event fireside chats and panels on mRNA, oncology and the crazy public market. We hope you can join him there.

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Joshua Cohen (L) and Justin Klee, Amylyx co-CEOs

Up­dat­ed: Af­ter long and wind­ing road, FDA ap­proves Amy­lyx's ALS drug in vic­to­ry for pa­tients and ad­vo­ca­cy groups

For just the third time in its 116-year history, the FDA has approved a new treatment for Lou Gehrig’s disease, or ALS.

US regulators gave the thumbs-up to the drug, known as Relyvrio, in a massive win for patients and their families. The approval, given to Boston-area biotech Amylyx Pharmaceuticals, comes after two years of long and contentious debates over the drug’s effectiveness between advocacy groups and FDA scientists, following the readout of a mid-stage clinical trial in September 2020.

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Nooman Haque, head of life sciences and healthcare at Silicon Valley Bank, and John Carroll

I’m head­ed to Lon­don soon for #EU­BIO22. Care to join me?

It was great getting back to a live ESMO conference/webinar in Paris followed by a live pop-up event for the Endpoints 11 in Boston. We’re staying on the road in October with our return for a live/streaming EUBIO22 in London.

Silicon Valley Bank’s Nooman Haque and I are once again jumping back into the thick of it with a slate of virtual and live events on October 12. I’ll get the ball rolling with a virtual fireside chat with Novo Nordisk R&D chief Marcus Schindler, covering their pipeline plans and BD work.

Up­dat­ed: Al­ny­lam re­in­forces APOL­LO-B patisir­an da­ta be­fore head­ing to the FDA

Weeks after uncorking some mostly positive data for patisiran in transthyretin-mediated (ATTR) amyloidosis with cardiomyopathy, Alnylam is bolstering its package with new exploratory and subgroup data before shipping it off to regulators.

The RNAi drug maintained “generally consistent” benefits in efficacy and quality of life across several prespecified subgroups at month 12, Alnylam announced on Friday afternoon, including age, baseline tafamidis use, ATTR amyloidosis type, baseline six-minute walk test score and others.

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#AAO22: J&J’s first look at com­mon eye dis­ease port­fo­lio pads the case for PhII of gene ther­a­py

CHICAGO — While the later-stage drug developers in the geographic atrophy field are near the finish line, Johnson & Johnson’s Janssen is taking a more deliberate route, with a treatment that it hopes to be a one-time fix.

The Big Pharma will take its Hemera Biosciences-acquired gene therapy into a Phase II study later this year in patients with GA, a common form of age-related macular degeneration that impacts about five million people worldwide. To get there, Janssen touted early-stage safety data at the American Academy of Ophthalmology annual conference Saturday morning, half a day after competitors Apellis and Iveric Bio revealed their own more-detailed Phase III analyses.

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In­dus­try groups call to block WTO IP waiv­er ex­pan­sion to Covid-19 ther­a­peu­tics

The WTO’s TRIPS Council in mid-October is expected to debate whether to extend the IP waiver for Covid-19 vaccines to therapeutics and diagnostics too.

While the Biden administration backed the original vaccine waiver, which critics note has not done much to expand access to vaccines as demand has dried up, US trade officials haven’t offered any perspective yet on whether to expand the waiver to Covid treatments.

Jerome Durso, Intercept Pharmaceuticals CEO

In­ter­cep­t's OCA fails a PhI­II NASH tri­al, rais­ing fresh doubts about its years­long quest for an OK

Intercept Pharmaceuticals has run into another big setback in its yearslong quest to win an approval for OCA in NASH. The biotech put out word Friday morning that its Phase III REVERSE study failed the primary endpoint for the liver disease, sending its share price into a tailspin.

There was no significant improvement in fibrosis among the patients suffering from cirrhosis who were treated with obeticholic acid, with investigators hunting for a minimum 1-stage histological improvement in the disease after 18 months of therapy.

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