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.

Health­care Dis­par­i­ties and Sick­le Cell Dis­ease

In the complicated U.S. healthcare system, navigating a serious illness such as cancer or heart disease can be remarkably challenging for patients and caregivers. When that illness is classified as a rare disease, those challenges can become even more acute. And when that rare disease occurs in a population that experiences health disparities, such as people with sickle cell disease (SCD) who are primarily Black and Latino, challenges can become almost insurmountable.

The End­points 11: They've got mad mon­ey and huge am­bi­tions. It's time to go big or go home

These days, selecting a group of private biotechs for the Endpoints 11 spotlight begins with a sprint to get ahead of IPOs and the M&A teams at Big Pharma. I’ve had a couple of faceplants earlier this year, watching some of the biotechs on my short list choose a quick leap onto Nasdaq or into the arms of a buyer.

Vividion, you would have been a great pick for the Endpoints 11. I’m sorry I missed you.

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Dave Lennon, former president of Novartis Gene Therapies

So what hap­pened with No­var­tis Gene Ther­a­pies? Here's your an­swer

Over the last couple of days it’s become clear that the gene therapy division at Novartis has quietly undergone a major reorganization. We learned on Monday that Dave Lennon, who had pursued a high-profile role as president of the unit with 1,500 people, had left the pharma giant to take over as CEO of a startup.

Like a lot of the majors, Novartis is an open highway for head hunters, or anyone looking to staff a startup. So that was news but not completely unexpected.

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Who are the women su­per­charg­ing bio­phar­ma R&D? Nom­i­nate them for this year's spe­cial re­port

The biotech industry has faced repeated calls to diversify its workforce — and in the last year, those calls got a lot louder. Though women account for just under half of all biotech employees around the world, they occupy very few places in C-suites, and even fewer make it to the helm.

Some companies are listening, according to a recent BIO survey which showed that this year’s companies were 2.5 times more likely to have a diversity and inclusion program compared to last year’s sample. But we still have a long way to go. Women represent just 31% of biotech executives, BIO reported. And those numbers are even more stark for women of color.

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

The fire un­der Glax­o­SmithK­line's Em­ma Walm­s­ley grows as an­oth­er well-known ac­tivist in­vestor grabs its pitch­fork — re­port

Bluebell Capital Partners, a proxy brawler fresh off a campaign to oust global food giant Danone’s CEO and most of its board of directors, has bought a stake in UK drugmaker GlaxoSmithKline with its eyes trained directly on Emma Walmsley, the Financial Times reported Wednesday.

The London-based hedge fund joins another notorious activist firm in Paul Singer’s Elliott Management, which earlier this year called for a shakeup in leadership at GSK to handle what the company described as a wealth of riches across the drug giant’s portfolio hindered by limited vision from top staff.

FDA+ roundup: Bs­U­FA III ready for show­time, court tells FDA to re-work com­pound­ing plan, new guid­ance up­dates and more

The FDA has now spelled out what exactly will be included in the third iteration of Biosimilar User Fee Act (BsUFA) from 2023 through 2027, which similarly to the prescription drug deal, sets fees that industry has to pay for submitting applications, in exchange for firm timelines that the agency must meet.

This latest deal includes several sweeteners for the biosimilar industry, which has yet to make great strides in the US market, with shorter review timelines for safety labeling updates and updates to add or remove an indication that does not contain efficacy data.

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David Meek, new Mirati CEO (Marlene Awaad/Bloomberg via Getty Images)

Fresh off Fer­Gene's melt­down, David Meek takes over at Mi­rati with lead KRAS drug rac­ing to an ap­proval

In the insular world of biotech, a spectacular failure can sometimes stay on any executive’s record for a long time. But for David Meek, the man at the helm of FerGene’s recent implosion, two questionable exits made way for what could be an excellent rebound.

Meek, most recently FerGene’s CEO and a past head at Ipsen, has become CEO at Mirati Therapeutics, taking the reins from founding CEO Charles Baum, who will step over into the role of president and head of R&D, according to a release.

FDA au­tho­rizes Pfiz­er's vac­cine boost­er for se­niors, those at high risk for se­vere Covid-19

The Biden administration’s goal of kicking off its booster shot drive for the entire US population this week is not quite going as planned.

First, Pfizer applied for approval of a supplemental application for the booster shots, but since last Friday’s adcomm reviewing them, the plan has devolved into an EUA, which the FDA issued late Thursday evening.

The population that is now eligible for the booster, six months after receiving the first pair of Pfizer-BioNTech vaccines, also narrowed from what Pfizer applied for (everyone who’s eligible for the initial Pfizer shots) to just those who are 65 or older, or at high-risk of a Covid infection, including health care workers and others with occupational hazards.

Stéphane Bancel, AP Images

Fi­nal analy­sis of US-fund­ed Mod­er­na Covid vac­cine tri­al shows 98% ef­fi­ca­cy against se­vere dis­ease

A final look at the results of the placebo-controlled Moderna trial in the New England Journal of Medicine, published Thursday afternoon, shows how the vaccine continues to prevent Covid-19 and severe cases after more than five months following the second shot.

Of the more than 30,000 enrolled in the trial that ultimately led to the vaccine’s EUA, only two people in the vaccine group got a severe form of the disease, compared to 106 in the placebo group — leading to an efficacy of 98%.