Joel Perlmutter

Ad­comm mem­ber who warned about risk of ap­prov­ing Bio­gen's ad­u­canum­ab has re­signed fol­low­ing his­toric OK — re­ports

One of the 11 ex­perts the FDA had con­vened to dis­cuss Bio­gen’s ad­u­canum­ab — be­fore over­rid­ing their rec­om­men­da­tion and ap­prov­ing the Alzheimer’s drug — has re­signed from the pan­el, STAT News and Pink Sheet re­port­ed.

Joel Perl­mut­ter, a neu­rol­o­gist at Wash­ing­ton Uni­ver­si­ty in St. Louis who’s been on the Pe­riph­er­al & Cen­tral Ner­vous Sys­tem Drugs Ad­vi­so­ry Com­mit­tee ros­ter since 2015, told STAT that he quit on Mon­day “due to this rul­ing by the FDA with­out fur­ther dis­cus­sion with our ad­vi­so­ry com­mit­tee.”

The ad­comm’s opin­ion was near-unan­i­mous: When asked whether it is rea­son­able to con­sid­er the pos­i­tive Phase III Bio­gen sub­mit­ted “as pri­ma­ry ev­i­dence of ef­fec­tive­ness” for the treat­ment of Alzheimer’s dis­ease, 10 vot­ed no, none said yes and one ab­stained. Dur­ing the full-day ses­sion last No­vem­ber, ex­perts ex­co­ri­at­ed both the com­pa­ny and the agency for what they saw as in­suf­fi­cient da­ta and a bi­ased pre­sen­ta­tion of in­for­ma­tion.

At one point, Perl­mut­ter warned that an ap­proval for ad­u­canum­ab could push back a tru­ly ef­fec­tive treat­ment “for more than a cou­ple of years, for many years.”

“Alzheimer’s treat­ment is a huge, ur­gent, un­met need, but I al­so think if we ap­prove some­thing where the da­ta is not strong, we have a risk of de­lay­ing good treat­ment,” he was quot­ed as say­ing.

The FDA’s his­toric ap­proval of ad­u­canum­ab was a re­sound­ing vic­to­ry for Bio­gen, which stands to gain bil­lions in rev­enue from the drug, now dubbed Aduhelm, with a broad la­bel priced at $56,000 per year. While pa­tient ad­vo­ca­cy groups and some lead­ing neu­rol­o­gists hailed the mile­stone, oth­ers de­cried pos­si­ble false hope and ex­pressed con­cerns about the prece­dent that reg­u­la­tors have now set re­gard­ing every­thing from the va­lid­i­ty of be­ta-amy­loid as an Alzheimer’s tar­get to the thresh­old for clin­i­cal ev­i­dence.

Bio­gen has nine years to com­plete the con­fir­ma­to­ry tri­al.

But the FDA is now back-track­ing on that time­line, say­ing in an emailed state­ment to End­points News, “We con­sid­er the nine-year time­line a con­ser­v­a­tive es­ti­mate, which is an ap­pro­pri­ate start­ing point for de­vel­op­ment of the draft pro­to­col from the spon­sor for its glob­al con­fir­ma­to­ry study. The agency be­lieves it’s im­por­tant that spon­sors set achiev­able clin­i­cal tri­al tar­get time­lines. We ful­ly ap­pre­ci­ate the high lev­el of in­ter­est in get­ting these an­swers quick­ly and will care­ful­ly mon­i­tor tri­al progress and sup­port ef­forts to com­plete this tri­al in the short­est pos­si­ble time­line.”

FDA al­so says it will con­tin­ue to work ac­tive­ly with Bio­gen in the months ahead on plans for this con­fir­ma­to­ry tri­al, in­clud­ing re­view and dis­cus­sion of the as­sump­tions for the pro­posed time­line.

“We ex­pect the spon­sor to com­mit all re­sources need­ed to move this tri­al for­ward as ef­fec­tive­ly as pos­si­ble, with the aim of com­plet­ing the tri­al as soon as is fea­si­ble, while as­sur­ing the qual­i­ty of the da­ta and the ro­bust­ness of the re­sults. Alzheimer’s tri­als take time to com­plete,” FDA said.

In­side Track: Be­hind the Scenes of a Ma­jor Biotech SPAC

Dr. David Hung and Michelle Doig are no strangers to the SPAC phenomenon. As Founder and CEO of Nuvation Bio, a biotech company tackling some of the greatest unmet needs in oncology, Dr. Hung recently took the company public in one of this year’s biggest SPAC related deals. And as Partner at Omega Funds, Doig not only led and syndicated Nuvation Bio’s Series A, but is now also President of the newly formed, Omega-sponsored, Omega Alpha SPAC (Nasdaq: OMEG; oversubscribed $138m IPO priced January 6, 2021).

Barry Greene, Sage CEO

UP­DAT­ED: Sage's sec­ond chance at de­pres­sion hits the PhI­II pri­ma­ry, but ques­tions re­main over dura­bil­i­ty, side ef­fects

Looking to make a comeback after a big Phase III flop, Sage Therapeutics revealed data they believe could change the entire depression treatment landscape, given the vast array of failures in the field. But some results are spooking investors, sending Sage $SAGE shares down early Tuesday.

First, the primary: Sage and Biogen reported Phase III data for once-daily zuranolone Tuesday morning, saying the experimental drug hit its primary endpoint by spurring a statistically significant change from baseline in the 17-item Hamilton Rating Scale for Depression total score. After 15 days, patients in the drug arm saw an average change of -14.1 points, compared to -12.3 on placebo.

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CEO Harith Rajagopalan (Fractyl)

Af­ter a decade in the type 2 di­a­betes game, Fractyl Lab­o­ra­to­ries recharges with a fresh $100M and a new name

Harith Rajagopalan compared the way type 2 diabetes is managed to sticking your fingers in a dam that’s leaking from a number of places.

You can take drugs to lower your blood sugar, cholesterol, or blood pressure, but you’re not addressing what he says is the core issue — the metabolic abnormality that causes the disease.

“We’re so busy plugging the holes in the dam, we don’t have time to see that the whole infrastructure is at risk,” he said. “That infrastructure is a full-body systemic metabolic abnormality called metabolic syndrome, that we’re ignoring while we’re so busy trying to treat all of the individual symptoms of the condition.”

Bio­gen sig­nals a big PhI­II fail­ure as the lead gene ther­a­py in their $800M Night­star buy­out goes down in flames

That $800 million buyout of Nightstar has turned into a bust for Biogen as the lead therapy in the deal failed a pivotal study, signaling a severe setback for the biotech’s ambitions in gene therapies.

The big biotech put out the word after the market closed on Monday that the gene therapy they picked up in the deal for a degenerative blindness called choroideremia failed the Phase III study, just a month after their #2 drug in the deal also flopped in a mid-stage study.

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Hal Barron, GSK R&D chief (Endpoints News)

Hal Bar­ron gam­bles $625M cash on high-wire TIG­IT act, throw­ing Glax­o­SmithK­line in­to heat­ed race and com­plet­ing next-gen I/O trin­i­ty

Count Hal Barron and GlaxoSmithKline in for the TIGIT fight.

The stakes are as high as the risks: While a growing pack of Big Pharma rivals is lending credence to the hypothesis that TIGIT will be the next big immune checkpoint and cancer drug target, the first clinical trials have shown response rates that can be described as modest at best. But Barron’s bet is on the whole “axis” that the receptor sits on, with an eye on testing its new anti-TIGIT antibody not just in combo with PD-1 but also in triplets.

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Michel Sade­lain puts his name and new cell en­gi­neer­ing tech be­hind 'ag­nos­tic' CAR-T start­up chas­ing epi­ge­net­ic anti­gens

It felt natural for Alain Maiore and Sebastian Amigorena to bring in Michel Sadelain as a co-founder of Mnemo Therapeutics. A CAR-T pioneer, Sadelain had been involved as an advisor since the early days — enthusiastic about Amigorena’s work in a genetic knockout that could enhance T cell memory and a new class of potential targets he’s discovered — and could introduce some well-known technologies to the toolbox. So they got the initial cash from Sofinnova Partners to plant roots in Paris and New York in early 2019; within a few months, they began to see more clearly just what the antigen discovery platform might unlock.

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Stephen Hahn (AP Images)

UP­DAT­ED: Ex-FDA com­mish Stephen Hahn joins Flag­ship, a ven­ture group that spawned Covid-19 vac­cine mak­er Mod­er­na

That revolving door between the FDA and industry is spinning even faster than usual.

Former FDA commissioner Stephen Hahn is joining Flagship Pioneering, the venture outfit that founded Moderna — which raced its way to an FDA EUA for a Covid-19 vaccine that is making billions of dollars– as the new CMO of its Preemptive Medicine and Health Security initiative.

Flagship confirmed the hire — first reported in The Washington Post — in a statement out late Monday.

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Patrizia Cavazzoni, CDER

FDA’s Cavaz­zoni calls for ad­comms to ‘get back to the sub­stance’

While her comments were recorded prior to the FDA’s recent approval of Biogen’s controversial Alzheimer’s drug, CDER Director Patrizia Cavazzoni presciently called for substantial reforms to the advisory committee process at the agency.

Short on examples of the adcomms she was referring to, Cavazzoni said at a BIO event aired on Monday that some recent committees show “how they can be swayed by emotion in the face of hard facts,” but they need to “get back to the fundamentals, which is listening to thoughtful input from experts in response to thoughtful questions that we ask them.”

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Paul Burton, incoming Moderna CMO (J&J/file photo)

Look­ing be­yond the pan­dem­ic, Mod­er­na ap­points J&J vet­er­an Paul Bur­ton as new CMO

Moderna is turning to one of its Covid-19 vaccine competitors to fill its open CMO slot, but this time, it’s not the vaccine experience they’re after.

Paul Burton, who’s spent 16 years at J&J, most recently as chief global medical affairs officer of Janssen, will take over as Moderna CMO on July 6.

With an eye toward a future beyond the pandemic, the mRNA biotech went with Burton, who earned his MD and PhD degrees in London, as he offers experience on a range of therapeutic areas, as well as work as a cardiothoracic surgeon and leading tech projects with Apple.

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