Blake Ferguson, 12, receives his first dose of the Pfizer vaccine (Photo by Anthony Behar/Sipa USA)(Sipa via AP Images)

Covid-19 vac­cines for kids: FDA spells out ques­tions on im­muno­bridg­ing, safe­ty fol­lowup ahead of ad­comm

NI­AID chief An­tho­ny Fau­ci pre­dict­ed days ago that Covid-19 vac­cines for younger chil­dren will be avail­able by the end of the year or ear­ly next year. But what re­quire­ments should they sat­is­fy be­fore get­ting the clear­ance from the FDA? The agency laid out some of its thoughts on brief­ing doc­u­ments for an ad­vi­so­ry com­mit­tee set for Thurs­day.

As with the first ex­pert pan­els con­vened to dis­cuss Covid-19 vac­cine reg­u­la­tions, the ad­comm won’t be talk­ing about spe­cif­ic prod­ucts. Rather, the goal is to gauge their opin­ion on frame­work, such as da­ta need­ed and ap­pro­pri­ate end­points.

Right now, Pfiz­er and BioN­Tech have the on­ly shot that’s au­tho­rized for ado­les­cents above the age of 12 — but noth­ing is sanc­tioned yet for those younger. (Cana­da and the EU, though, have green­light­ed Mod­er­na’s jab for the same pop­u­la­tion.)

Reg­u­la­tors set the stage by not­ing that al­though pe­di­atric pop­u­la­tions seem less like­ly to be in­fect­ed by the coro­n­avirus (and when they do, tend not to get as sick), many have still been hos­pi­tal­ized or died from Covid-19:

As of May 25, 2021, ap­prox­i­mate­ly 553,000 and 2.7 mil­lion COVID-19 cas­es, re­spec­tive­ly, were re­port­ed in chil­dren 0-4 and 5-17 years of age in the US, among which 121 (0.02%) and 278 (0.01%), re­spec­tive­ly, were fa­tal, and more than 38,000 of which re­sult­ed in hos­pi­tal­iza­tions re­port­ed since Au­gust 1, 2020, in pa­tients 0-17 years of age

The FDA em­pha­sized that any au­tho­riza­tion for pe­di­atric use should fol­low “sub­stan­tial ev­i­dence of ef­fec­tive­ness from ad­e­quate and well-con­trolled tri­als” plus am­ple safe­ty da­ta. Yet with three vac­cines from Pfiz­er/BioN­Tech, Mod­er­na and J&J al­ready show­ing ef­fi­ca­cy in adults, they won­der if the ev­i­dence could take a dif­fer­ent shape.

Giv­en the low­er in­fec­tion, hos­pi­tal­iza­tion and death rates in chil­dren, af­ter all, the dy­nam­ics are quite dif­fer­ent.

FDA ac­knowl­edges that di­rect demon­stra­tion of ef­fec­tive­ness in field ef­fi­ca­cy tri­als may not be fea­si­ble in pe­di­atric pop­u­la­tions and thus, fol­low­ing di­rect demon­stra­tion of pro­tec­tion in adults, ef­fec­tive­ness of the same vac­cine could be in­ferred in pe­di­atric pop­u­la­tions by im­muno­bridg­ing. … In this ap­proach, an im­mune re­sponse bio­mark­er(s) elicit­ed by the vac­cine in a pe­di­atric age group is com­pared to the same im­mune re­sponse bio­mark­er(s) elicit­ed by the same vac­cine in a rel­e­vant adult age group, with for­mal sta­tis­ti­cal hy­poth­e­sis test­ing to demon­strate that the mea­sured im­mune re­sponse in the pe­di­atric age group is non-in­fe­ri­or to that in adults.

There are al­so ques­tions about whether there should be a longer safe­ty fol­lowup than the two months man­dat­ed for the adult and ado­les­cent OKs.

One oth­er spe­cif­ic con­sid­er­a­tion for chil­dren, the FDA added, would be how the Covid-19 vac­cine might in­ter­fere with oth­er rou­tine­ly rec­om­mend­ed im­mu­niza­tions. Vac­cine mak­ers are en­cour­aged to in­clude in­for­ma­tion but will not be re­quired to as­sess con­comi­tant ad­min­is­tra­tion to sup­port li­cen­sure.

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).

Janet Woodcock, acting FDA commissioner (Al Drago/Bloomberg via Getty Images)

New Alzheimer's drug ap­proval fall­out: Pub­lic Cit­i­zen seeks re­moval of FDA's Wood­cock, Cavaz­zoni and Dunn

As Capitol Hill begins to wake up to the financial and scientific mess behind the FDA’s approval of Biogen’s new controversial Alzheimer’s drug Aduhelm, nonprofit watchdog Public Citizen is now calling for the top three FDA officials who are responsible to be removed from their positions.

In a letter to HHS Secretary Xavier Becerra on Wednesday, the group highlighted the “litany of flaws” in the FDA’s approval of the new drug, including the “unprecedented, inappropriately close” collaboration between the FDA and Biogen in the analysis of key trial data, basing approval on an unvalidated surrogate endpoint, not following the advice of its expert advisory committee (3 members of which have since resigned), and the wide label that the agency granted.

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In a first, Re­gen­eron's an­ti­body cock­tail re­duces deaths for a sub­group of hos­pi­tal­ized Covid-19 pa­tients

Scientists have come up with the first evidence that Regeneron’s antibody cocktail, which has so far only been authorized for the outpatient setting, may reduce deaths of hospitalized Covid-19 patients — albeit only a subset.

The combination of casirivimab and imdevimab is the subject of the latest data cut from RECOVERY, the large-scale UK-based trial testing a variety of potential treatments. In total, 9,785 patients hospitalized with Covid-19 were enrolled in this arm of the study and were randomly assigned to receive either usual care plus the intravenous combo or usual care alone.

Aus­tralian spin­out eyes the clin­ic with a pro­phy­lac­tic nasal spray for Covid-19

Whether patients are immunocompromised or just don’t like needles, one fledgling Australian biotech says it has an alternative to Covid-19 vaccines that doesn’t involve a jab in the arm — and on Tuesday, it pulled in a fresh round of funding to take it into the clinic.

ENA Respiratory, which spun out of ENA Therapeutics last year, has pulled in nearly $24.7 million (AU $30 million) to advance its nasal spray for respiratory viral infections, the company said Tuesday.

Someit Sidhu, JATT

An­oth­er life sci­ences SPAC has popped up from a small biotech CEO with the help of Take­da, No­var­tis vet­er­ans

Editor’s note: Interested in following biopharma’s fast-paced IPO market? You can bookmark our IPO Tracker here.

The SPAC train has slowed down since the extraordinarily high levels from late 2020 into early this year, but Tuesday saw the filing of a new blank-check company targeting the life sciences industry.

Jatt Acquisition submitted its SEC paperwork Tuesday, penciling in an estimated raise of $120 million as it sets its sights on a reverse merger partner. The SPAC is run by Someit Sidhu, a co-founder of Pathios Therapeutics, as well as CEO of Akaza Bioscience and Izana Bioscience. Sidhu will control about 97% of the blank-check company’s shares, per the S-1.

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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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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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.”

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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