In his­toric vote, mR­NA vac­cines go 2 and 0 as FDA ex­perts unan­i­mous­ly back Mod­er­na's Covid-19 vac­cine

Come Sat­ur­day, the US will like­ly have two Covid-19 vac­cines.

The FDA’s ad­vi­so­ry com­mit­tee on vac­cines vot­ed unan­i­mous­ly Thurs­day — with one ab­sten­tion — to rec­om­mend an emer­gency use au­tho­riza­tion for Mod­er­na’s mR­NA-based Covid-19 shot, set­ting the agency up to like­ly is­sue the EUA on Fri­day. The agency does not have to lis­ten to the com­mit­tee but usu­al­ly does.

“The ev­i­dence that has been stud­ied in great de­tail on this vac­cine high­ly out­weighs any is­sues that we’ve seen,” Stan­ford in­fec­tious dis­ease pe­di­a­tri­cian Hay­ley Gans said. “And I think re­al­ly sup­ports us be­ing able, with the pan­dem­ic, move for­ward and fi­nal­ly pro­vide a safe and ef­fec­tive way of get­ting to herd im­mu­ni­ty.”

The unan­i­mous vote con­trast­ed with last week’s ad­vi­so­ry hear­ing on Pfiz­er-BioN­Tech’s own mR­NA shot, where four com­mit­tee mem­bers vot­ed against the ques­tion put to them and one ab­stained. But the dis­crep­an­cy may come down to a mi­nor dif­fer­ence be­tween the two ap­pli­ca­tions.

Pfiz­er asked for an EUA to vac­ci­nate any­one 16 years of age or old­er, but some mem­bers of the com­mit­tee were con­cerned that Pfiz­er did not con­duct suf­fi­cient test­ing on 16 and 17 year olds and vot­ed ac­cord­ing­ly, even though they sup­port­ed vac­ci­nat­ing any­one over 18. Mod­er­na on­ly re­quest­ed an EUA for peo­ple 18 and up, as­suag­ing the com­mit­tee’s con­cerns.

Nev­er­the­less, some ad­vi­so­ry mem­bers still sparred with the FDA over the word­ing of the ques­tion put be­fore the com­mit­tee, fear­ing that the word­ing may mis­lead the pub­lic in­to be­liev­ing that the vac­cine has been ful­ly ap­proved. They of­fered adding “ex­per­i­men­tal” or “emer­gency use au­tho­riza­tion” to the ques­tion.

“This can be in­ter­pret­ed as rec­om­mend­ing full ap­proval,” said NCAT’s Michael Kuril­la. “That may… pre­clude not on­ly ad­e­quate eval­u­a­tion of this vac­cine but al­so fu­ture and on­go­ing Covid vac­cines in de­vel­op­ment.

FDA of­fi­cials and oth­er mem­bers, how­ev­er, quick­ly shot down the idea.

“The ques­tion is nev­er when do you know every­thing, it’s when do you know enough,” said Paul Of­fit, an in­fec­tious dis­ease physi­cian at Chil­dren’s Hos­pi­tal of Philadel­phia.

Kuril­la ab­stained over the word­ing, as well as con­cerns that an ex­pand­ed ac­cess pro­to­col may have been more ap­pro­pri­ate than an EUA, al­low­ing tri­als to con­tin­ue to col­lect ran­dom­ized da­ta.

An EUA for Mod­er­na would rough­ly dou­ble the amount of vac­cine im­me­di­ate­ly avail­able in the U.S., from over 20 mil­lion dos­es to over 40 mil­lion dos­es. The vac­cine is al­so eas­i­er to dis­trib­ute as it can be han­dled at the same tem­per­a­ture as most ap­proved vac­cines — un­like Pfiz­er’s shot — and the U.S. has ex­er­cised op­tions to ac­quire 200 mil­lion dos­es through June.

While prof­fer­ing dozens of ques­tions, the com­mit­tee nev­er ex­pressed se­ri­ous con­cerns about the safe­ty or ef­fec­tive­ness of the vac­cine. Mod­er­na an­nounced pre­vi­ous­ly that the vac­cine was 94.1% ef­fec­tive at pre­vent­ing symp­to­matic Covid-19. Al­though the FDA re­view re­vealed that the vac­cine ap­peared less ef­fec­tive in vol­un­teers over 65 — 86.5% pro­tec­tion — com­pa­ny ex­ec­u­tives not­ed that the dif­fer­ence was not sta­tis­ti­cal­ly sig­nif­i­cant.

In­stead, dis­cus­sion cen­tered on rare po­ten­tial side ef­fects and Mod­er­na’s plan for what to do with ad­verse events. The FDA’s re­view turned up one sur­pris­ing se­ri­ous event: Two vol­un­teers who had pre­vi­ous­ly re­ceived a der­mal fill­ing ex­pe­ri­enced fa­cial swelling af­ter re­ceiv­ing the vac­cine.

The pan­el asked re­peat­ed­ly the al­ler­gic re­ac­tions that have emerged in the two weeks since Pfiz­er’s vac­cine was in­oc­u­lat­ed. Al­though Mod­er­na said they did not see any cas­es of ana­phy­lax­is in their study — out­side of one al­ler­gic re­ac­tion two months out, from some­one with a soy al­ler­gy — they al­so not­ed it as one po­ten­tial se­ri­ous event to mon­i­tor long term.

Still, Mod­er­na CMO Tal Zaks cau­tioned that each com­po­nent of the com­pa­ny’s lipid nanopar­ti­cle, which may have trig­gered the al­ler­gic re­ac­tion in Pfiz­er re­cip­i­ents, was dif­fer­ent chem­i­cal­ly than the Pfiz­er-BioN­Tech vac­cine.

“While we all might say, oh there’s an LNP here, there’s a lipid and mR­NA, there­fore they must be all be the same,” he said. “I would not nec­es­sar­i­ly as­sume that.”

The on­ly point of con­tin­u­al fric­tion be­tween Mod­er­na and the pan­el arose around the ques­tion of what the com­pa­ny should do with their Phase III tri­al af­ter an EUA is is­sued. The group was skep­ti­cal last week when Pfiz­er pro­posed grad­u­al­ly un­blind­ing par­tic­i­pants as they be­come el­i­gi­ble for vac­cine un­der CDC pri­or­i­ty guide­lines. Mod­er­na want­ed to go fur­ther, im­me­di­ate­ly of­fer­ing the vac­cine to all place­bo par­tic­i­pants, re­gard­less of whether they would be el­i­gi­ble.

Mod­er­na ex­ec­u­tives said that they need­ed to un­blind to pre­vent vol­un­teers from drop­ping out of the study, and be­cause place­bo par­tic­i­pants are and would con­tin­ue to be in­fect­ed. They said they could vac­ci­nate them with clin­i­cal sup­ply of the vac­cine that would not draw from their com­mer­cial sta­ble.

“Ad­di­tion­al se­vere cas­es and death are a ques­tion more of when than a ques­tion of if,” Mod­er­na se­nior vice pres­i­dent Jacque­line Miller said.

Many ad­vi­sors re­ject­ed the ap­proach, propos­ing that Mod­er­na ei­ther fol­low Pfiz­er’s plan or adopt an ap­proach where the tri­al would stay blind­ed but par­tic­i­pants in the vac­cine arm would get place­bo and vol­un­teers in the place­bo arm would re­ceive vac­cine, al­low­ing com­par­isons on dura­bil­i­ty and long-term ad­verse events. A few, how­ev­er, backed the Mod­er­na ap­proach, say­ing the blind­ing pro­pos­al and even the Pfiz­er ap­proach may be in­fea­si­ble.

The de­bate got to the point that at least two dif­fer­ent ad­vi­sors pro­posed the agency al­low the pan­elists to vote on the is­sue, al­though they had not orig­i­nal­ly been asked to do so.

“It seems to me that there’s an as­sump­tion that Mod­er­na is go­ing to do what it’s go­ing to do. It doesn’t have to be that way,” Shel­don Taub­man, the con­sumer ad­vo­cate on the pan­el, said. “We weren’t asked to vote on it, but we could vote on it.”

Mar­i­on Gru­ber, di­rec­tor of the of­fices of vac­cine re­search at the FDA, though, neu­tral­ized that idea, cit­ing the “com­plex­i­ty of the sit­u­a­tion” and the fact that they did not ask the com­mit­tee to vote on the same is­sue at the Pfiz­er tri­al. The hear­ing end­ed with­out a res­o­lu­tion.

“I think we gave FDA a sense of our with that we could do a crossover blind­ed de­sign, but the re­al­iza­tion that that may be im­pos­si­ble,” com­mit­tee chair Arnold Mon­to said. “FDA will be ne­go­ti­at­ing with Mod­er­na about the way they will ad­dress this prob­lem.”

IDC: Life Sci­ences Firms Must Em­brace Dig­i­tal Trans­for­ma­tion Now

Pre-pandemic, the life sciences industry had settled into a pattern. The average drug took 12 years and $2.9 billion to bring to market, and it was an acceptable mode of operations, according to Nimita Limaye, Research Vice President for Life Sciences R&D Strategy and Technology at IDC.

COVID-19 changed that, and served as a proof-of-concept for how technology can truly help life sciences companies succeed and grow, Limaye said. She recently spoke about industry trends at Egnyte’s Life Sciences Summit 2022. You should watch the entire session, free and on-demand, but here’s a brief recap of why she’s urging life sciences companies to embrace digital transformation.

Tom Barnes, Orna Therapeutics CEO

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Merck went in deep on its collaboration with Moderna on new mRNA programs, and dropped them all over time, including their RSV partnership. But after writing off what turned out as one of the most successful infectious disease players in the business, Merck is coming in this morning with a new preclinical alliance — this time embracing a biotech that hopes to eventually outdo the famously successful mRNA in a new run at vaccines and therapeutics.

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Bayer's first DTC ad campaign for chronic kidney disease drug Kerendia spells out its benefits

Bay­er aims to sim­pli­fy the com­plex­i­ties of CKD with an ABC-themed ad cam­paign

Do you know the ABCs of CKD in T2D? Bayer’s first ad campaign for Kerendia tackles the complexity of chronic kidney disease with a play on the acronym (CKD) and its connection to type 2 diabetes (T2D).

Kerendia was approved last year as the first and only non-steroidal mineralocorticoid receptor antagonist to treat CKD in people with type 2 diabetes.

In the TV commercial launched this week, A is for awareness, B is for belief and C is for cardiovascular, explained in the ad as awareness of the connection between type 2 and kidney disease, belief that something can be done about it, and cardiovascular events that may be reduced with treatment.

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James Mock, incoming CFO at Moderna

Mod­er­na taps new CFO from PerkinElmer af­ter for­mer one-day CFO oust­ed

When Moderna hired a new CFO last year,  it didn’t expect to see him gone after only one day. Today the biotech named his — likely much more vetted — replacement.

The mRNA company put out word early Wednesday that after the untimely departure of then brand-new CFO Jorge Gomez, it has now found a replacement in James Mock, the soon-to-be former CFO at diagnostics and analytics company PerkinElmer.

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Kate Haviland, Blueprint Medicines CEO

Blue­print met all its end­points in bid for ex­pand­ed Ay­vak­it la­bel — but stock trends low­er any­way

Blueprint Medicines announced this morning that the second part of its study on Ayvakit in non-advanced systemic mastocytosis (SM) — a rare disease in which a type of white blood cells known as mast cells builds up — met all endpoints, but the biopharma left key questions unanswered.

In 212 patients, with 141 in the treatment arm and 71 in the control arm, patients who got Ayvakit saw an average 15.6-point decrease in their symptom scores compared to a 9.2-point decrease in the placebo arm at 24 weeks. In an extension study, those on Ayvakit saw their symptom scores drop by 20.2 points by week 48.

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Up­dat­ed: Hit by an­oth­er PhI­II flop, Sanofi culls breast can­cer drug — sound­ing alarm for the class

Sanofi is officially giving up on its oral SERD.

The French drugmaker put out word Wednesday morning that it will discontinue the global development program of amcenestrant, the selective estrogen receptor degrader once billed as a top late-stage prospect. Having already failed a Phase II monotherapy test earlier this year, a combo with the drug also missed the bar in a second trial for breast cancer, triggering the decision to drop the whole program.

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Etleva Kadilli, director of UNICEF’s supply division

GSK lands first-ever UNICEF con­tract for malar­ia vac­cine worth $170M

GSK has landed a new first from UNICEF the first-ever contract for malaria vaccines, worth up to $170 million for 18 million vaccine doses distributed over the next three years.

The vaccine, known as Mosquirix or RTS,S, won WHO’s backing last October after a controversial start, but UNICEF said these doses will potentially save thousands of lives every year.

“We hope this is just the beginning,” Etleva Kadilli, director of UNICEF’s supply division, said. “Continued innovation is needed to develop new and next-generation vaccines to increase available supply, and enable a healthier vaccine market. This is a giant step forward in our collective efforts to save children’s lives and reduce the burden of malaria as part of wider malaria prevention and control programmes.”

Joe Jonas (Photo by Anthony Behar/Sipa USA)(Sipa via AP Images)

So­lo Jonas broth­er car­ries Merz's new tune in Botox ri­val cam­paign

As the lyrics of his band’s 2019 pop-rock single suggest, Joe Jonas is only human — and that means even he gets frown lines. The 33-year-old singer-songwriter is Merz’s newest celebrity brand partner for its Botox rival Xeomin, as medical aesthetics brands target a younger audience.

Merz kicked off its “Beauty on Your Terms” campaign on Tuesday, featuring the Jonas brother in a video ad for its double-filtered anti-wrinkle injection Xeomin.

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Paul Perreault, CSL Behring CEO

CSL CEO Paul Per­reault de­ter­mined to grow plas­ma col­lec­tion af­ter full-year sales dip

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However, CEO Paul Perreault assured investors and analysts on the full-year call that the team has left “no stone unturned” when assessing options to grow plasma volumes. The chief executive also spelled out positive results for the company’s monoclonal antibody garadacimab in hereditary angioedema (HAE), though he isn’t revealing the exact numbers just yet.