Albert Bourla, Pfizer CEO (Gian Ehrenzeller/Keystone via AP Images)

Pfiz­er, BioN­Tech say Covid-19 vac­cine is more than 90% ef­fec­tive, ex­ceed­ing ex­pert ex­pec­ta­tions and set­ting up an EUA sub­mis­sion late this month

For 10 months, the US and the world have won­dered and watched and wait­ed for when an ef­fec­tive Covid-19 vac­cine will ar­rive. Pfiz­er said Mon­day morn­ing that it has.

The Big Phar­ma and its part­ner BioN­Tech said that, on an in­ter­im look, their vac­cine was at least 90% ef­fec­tive in pre­vent­ing symp­to­matic Covid-19 cas­es. If the da­ta hold up, it would mean a pan­dem­ic in­oc­u­la­tion vast­ly more ef­fec­tive than an­tic­i­pat­ed through­out 2020, when ex­perts warned that the first vac­cines could be as low as 60% or 70% ef­fec­tive and the FDA in­di­cat­ed it was will­ing to ap­prove jabs that sur­passed the 50% mark.

Pfiz­er said it will sub­mit for an emer­gency use au­tho­riza­tion around the third week of No­vem­ber, when it sur­pass­es a safe­ty bench­mark set by the FDA. FDA of­fi­cials have in­di­cat­ed they could move to en­act an EUA at record speed, al­though not be­fore con­ven­ing an ad­vi­so­ry com­mit­tee to re­view Pfiz­er’s da­ta.

A da­ta safe­ty and mon­i­tor­ing com­mit­tee didn’t flag any ma­jor safe­ty is­sues, al­though the com­pa­ny still has to reach the me­di­an two months of fol­lowup the FDA has re­quired Covid-19 vac­cine mak­ers reach in their Phase III tri­als.

A sea of ques­tions still looms for the mR­NA can­di­date, though. It’s not clear how well the vac­cine pre­vents in­fec­tion or se­vere cas­es of Covid-19 — an is­sue raised at the re­cent FDA ad­vi­so­ry com­mit­tee on vac­cines — and no one knows how long pro­tec­tion will last. The da­ta have al­so not been peer-re­viewed, draw­ing crit­i­cism from ac­tivists who have called for com­pa­nies not to re­lease Covid-19 re­sults by press re­lease.

Pub­lic Cit­i­zen, the left-lean­ing Wash­ing­ton-based NGO, called it “bad sci­ence,” not­ing the lin­ger­ing un­knowns on safe­ty and se­vere cas­es.

“Cru­cial in­for­ma­tion ab­sent from the com­pa­nies’ an­nounce­ment is any ev­i­dence that the vac­cine pre­vents se­ri­ous COVID-19 cas­es or re­duces hos­pi­tal­iza­tions and deaths due to the dis­ease,” Michael Car­ome, the NGO’s lead health re­searcher, said in a state­ment. “More im­por­tant­ly, crit­i­cal safe­ty da­ta from the phase 3 tri­al of the Pfiz­er and BioN­Tech vac­cine is not yet avail­able.”

Still, the re­sults pro­vide a ma­jor boon for hopes that vac­cines can even­tu­al­ly help curb the pan­dem­ic in the US and world­wide. Mod­er­na, the vac­cine de­vel­op­er like­ly to read­out next, al­so us­es mR­NA, a tech­nol­o­gy that had nev­er been test­ed in large scale tri­als be­fore 2020, and vir­tu­al­ly all ma­jor can­di­dates tar­get the coro­n­avirus spike pro­tein that Pfiz­er tar­gets.

Even if the vac­cine holds up and is au­tho­rized by the FDA, though, it will still be in short sup­ply. Pfiz­er has said that around 50 mil­lion dos­es of the vac­cine could be avail­able by the end of the year, with 1 bil­lion dos­es avail­able in 2021. The vac­cine re­quires two dos­es to be ef­fec­tive.

The 90% fig­ure comes from an analy­sis of 94 con­firmed cas­es of Covid-19 across both arms of the com­pa­ny’s Phase III tri­al, which has en­rolled 44,000 vol­un­teers to date. The com­pa­ny had orig­i­nal­ly planned to con­duct its first analy­sis at 32 cas­es, prompt­ing CEO Al­bert Bourla to in­sist they’d know if it worked by Oc­to­ber, but af­ter dis­cus­sions with the FDA, agreed to look af­ter 62 cas­es. By that time, though, 94 cas­es had al­ready ac­crued.

In a state­ment, Bourla cast the re­sults as a ma­jor mile­stone in the fight against the pan­dem­ic, not­ing that it comes at a time when cas­es are surg­ing to record lev­els. Those surg­ing cas­es like­ly helped Pfiz­er reach the ef­fi­ca­cy da­ta faster than they would have if the virus was un­der con­trol.

“To­day is a great day for sci­ence and hu­man­i­ty,” he said. “The first set of re­sults from our Phase 3 COVID-19 vac­cine tri­al pro­vides the ini­tial ev­i­dence of our vac­cine’s abil­i­ty to pre­vent COVID-19. We are reach­ing this crit­i­cal mile­stone in our vac­cine de­vel­op­ment pro­gram at a time when the world needs it most with in­fec­tion rates set­ting new records, hos­pi­tals near­ing over-ca­pac­i­ty and economies strug­gling to re­open.”

Al­though Pfiz­er has in­di­cat­ed they would want to un­blind the study once re­sults were in, the com­pa­ny said that it will con­tin­ue un­til it reach­es 164 con­firmed cas­es. At that point, how­ev­er, it’s un­clear whether the FDA would want them to con­tin­ue the blind­ed study, which would al­low them to col­lect bet­ter long term safe­ty da­ta.

The an­nounce­ment marks a ma­jor suc­cess for Bourla, who was pro­mot­ed to CEO two years ago to re-en­er­gize a slow-foot­ed Amer­i­can gi­ant and who ar­guably bet more on his com­pa­ny’s Covid-19 vac­cine ef­forts than any oth­er ex­ec­u­tive on the plan­et. Team­ing with the Ger­man mR­NA biotech BioN­Tech, he es­chewed gov­ern­ment fund­ing on the prin­ci­ple that they could move faster alone. Asked by Time in Ju­ly what would hap­pen if the FDA re­ject­ed the vac­cine they in­vest­ed over a $1 bil­lion in, he said: “We will just have to write it off and call it a day.”

Pfiz­er, a com­pa­ny that first made its mark mass-pro­duc­ing peni­cillin dur­ing World War II, could now stand to reap both the pres­tige and fi­nan­cial wind­fall of the first ef­fec­tive Covid-19 shot. The gov­ern­ment has agreed to pay the New York phar­ma $1.95 bil­lion for 100 mil­lion dos­es of its vac­cine. Pro­jec­tions for fu­ture sales vary great­ly, but SVB Leerink  pegged them for over $3 bil­lion in 2021 and JP Mor­gan has put that fig­ure as high as $5.5 bil­lion.

With the world like­ly fac­ing a short­age of coro­n­avirus vac­cines for sev­er­al years to come and vac­ci­nat­ed peo­ple like­ly to need boost­er shots, the prod­uct could be a block­buster for a con­sid­er­able time.

For a look at all End­points News coro­n­avirus sto­ries, check out our spe­cial news chan­nel.

Pascal Soriot (Getty)

As­traZeneca CEO So­ri­ot plans new study to test that con­tro­ver­sial 90% ef­fi­ca­cy fig­ure, wait­ing for US da­ta be­fore go­ing to FDA

Pascal Soriot spent the long Thanksgiving weekend digging AstraZeneca out of a hole, promising to put an end to the questions around its interim Phase III vaccine data by conducting a new study while going to regulators with a large part of what it already has.

AstraZeneca and its partners at Oxford had initially touted high-level results from two studies conducted in the UK and Brazil as positive. But the enthusiasm was soon shadowed by confusion as observers probed into how the highest, 90% efficacy was seen in a dosing regimen given to a small group of volunteers due to an error. Among a larger cohort given the intended shots, the vaccine was only 62% effective, a rate that would’ve been respectable had Pfizer/BioNTech and Moderna not posted efficacy rates of 94%, 95% for their mRNA candidates. And many weren’t sure what to make of the average 70% number that AstraZeneca ran in headlines.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 94,300+ biopharma pros reading Endpoints daily — and it's free.

Scoop: Google’s GV spear­heads the Spot­light syn­di­cate — back­ing an up­start biotech aimed at ‘de­moc­ra­tiz­ing’ gene edit­ing

CRISPR had no sooner started to shake the very foundations of drug development before its limitations began to loom large. Gene editing could change the world — if only you could get around the hurdles that threatened to trip up every program.

So it’s only natural to see CRISPR 2.0 taking shape before the pioneers can get the lead therapies through development. And who better than Google’s GV venture arm to take the lead spot in a small syndicate backing some scientists with their own unique twist on a solution?

Biogen CEO Michel Vounatsos (via Getty Images)

With ad­u­canum­ab caught on a cliff, Bio­gen’s Michel Vounatsos bets bil­lions on an­oth­er high-risk neu­ro play

With its FDA pitch on the Alzheimer’s drug aducanumab hanging perilously close to disaster, Biogen is rolling the dice on a $3.1 billion deal that brings in commercial rights to one of the other spotlight neuro drugs in late-stage development — after it already failed its first Phase III.

The big biotech has turned to Sage Therapeutics for its latest deal, close to a year after the crushing failure of Sage-217, now dubbed zuranolone, in the MOUNTAIN study.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 94,300+ biopharma pros reading Endpoints daily — and it's free.

FDA gives Rhythm the green light for set­melan­otide, a drug aimed at re­duc­ing obe­si­ty in cer­tain ge­net­ic dis­or­ders

A little over a year after completing successful pivotal trials, Rhythm Pharmaceuticals $RYTM has its first drug approval on its hands.

The Boston-based biotech announced Friday that the FDA gave setmelanotide the thumbs-up for three rare genetic disorders that result in obesity in patients six and older. It’s the agency’s first such approval, Rhythm said, with the indicated deficiencies being the POMC, PCSK1 and LEPR genes. Rhythm will market the drug as Imcivree, and plans to have it on the shelves in the first quarter of 2021.

Robert Clarke (Kinaset)

Ki­naset launch­es with $40M and a JAK in­hibitor from Vec­tura's old pipeline

Kinaset Therapeutics is joining the search for a better severe asthma treatment, picking up where Vectura left off when it decided to clear house last year.

UK-based Vectura — which took a big hit when its most advanced candidate flopped in a Phase III asthma trial back in 2018 — recently shifted to a CDMO model, offloading all of its R&D programs. Robert Clarke, who’s worked on inhalable therapeutics for 21-plus years, had close contacts at the company and took a look at what they were offering. After doing some research, he was attracted by VR475, a pan-JAK inhibitor.

Stephané Bancel (Endpoints at JPM20)

Mod­er­na cal­i­brates fi­nal Covid-19 vac­cine ef­fi­ca­cy at 94.1% — and to­day it's gun­ning for the EUA

Nearly a year ago, as the coronavirus emerged in China, the NIH and four major companies bet on an unproven genetic technology as the best tool for developing a vaccine to stem the outbreak. Today, a second such vaccine is heading to the FDA.

Moderna said Monday that they will request an emergency use authorization from the FDA after a final analysis showed their mRNA vaccine was 94.1% effective at preventing symptomatic Covid-19. The data confirm the results from an interim analysis and matches efficacy Pfizer and BioNTech showed in a Phase III study, setting the biotech up to potentially nab one of the first two Covid-19 vaccine OKs.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 94,300+ biopharma pros reading Endpoints daily — and it's free.

Jason Kelly, Ginkgo Bioworks CEO (Kyle Grillot/Bloomberg via Getty Images)

Af­ter Ko­dak de­ba­cle, US lends $1.1B to a syn­thet­ic bi­ol­o­gy com­pa­ny and their big Covid-19, mR­NA plans

In mid-August, as Kodak’s $765 million government-backed push into drug manufacturing slowly fell apart in national headlines, Ginkgo Bioworks CEO Jason Kelly got a message from his company’s government liaison: HHS wanted to know if they, too, might want a loan.

The government’s decision to lend Kodak three quarters of a billion dollars raised eyebrows because Kodak had never made drugs before. But Ginkgo, while not a manufacturing company, had spent the last decade refining new ways to produce materials inside cells and building automated facilities across Boston.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 94,300+ biopharma pros reading Endpoints daily — and it's free.

FDA hands Liq­uidia and Re­vance a CRL and de­fer­ral, re­spec­tive­ly, as Covid-19 cre­ates in­spec­tion chal­lenge

Two biotechs said they got turned away by the FDA on Wednesday, in part due to pandemic-related travel restrictions.

North Carolina-based Liquidia Technologies was handed a CRL for its lead pulmonary arterial hypertension drug, citing the need for more CMC data and on-site pre-approval inspections, which the FDA hasn’t been able to conduct due to travel restrictions. The agency also deferred its decision on Revance Therapeutics’ BLA for its frown line treatment, because it needs to inspect the company’s northern California manufacturing facility. The action, Revance emphasized, was not a CRL.

Covid-19 roundup: Eu­rope pur­chas­es 80M dos­es of Mod­er­na's vac­cine; CO­V­AXX se­cures $2.8B in emerg­ing mar­ket pre-or­ders

With the announcement of its vaccine efficacy data last week, Moderna is starting to line up customers for its Covid-19 mRNA jabs.

The Massachusetts-based biotech announced Wednesday it has agreed to sell an initial round of 80 million doses to the European Commission, with the option to double the amount to 160 million. Once the member states rubber stamp the approval, the deal will be finalized.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 94,300+ biopharma pros reading Endpoints daily — and it's free.