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.

2023 Spot­light on the Fu­ture of Drug De­vel­op­ment for Small and Mid-Sized Biotechs

In the context of today’s global economic environment, there is an increasing need to work smarter, faster and leaner across all facets of the life sciences industry.  This is particularly true for small and mid-sized biotech companies, many of which are facing declining valuations and competing for increasingly limited funding to propel their science forward.  It is important to recognize that within this framework, many of these smaller companies already find themselves resource-challenged to design and manage clinical studies themselves because they don’t have large teams or in-house experts in navigating the various aspects of the drug development journey. This can be particularly challenging for the most complex and difficult to treat diseases where no previous pathway exists and patients are urgently awaiting breakthroughs.

Dipal Doshi, Entrada Therapeutics CEO

Ver­tex just found the next big ‘trans­for­ma­tive’ thing for the pipeline — at a biotech just down the street

Back in the summer of 2019, when I was covering Vertex’s executive chairman Jeff Leiden’s plans for the pipeline, I picked up on a distinct focus on myotonic dystrophy Type I, or DM1 — one of what Leiden called “two diseases (with DMD) we’re interested in and we continue to look for those assets.”

Today, Leiden’s successor at the helm of Vertex, CEO Reshma Kewalramani, is plunking down $250 million in cash to go the extra mile on DM1. The lion’s share of that is for the upfront, with a small reserve for equity in a deal that lines Vertex up with a neighbor in Seaport that has been rather quietly going at both of Vertex’s early disease targets with preclinical assets.

Endpoints Premium

Premium subscription required

Unlock this article along with other benefits by subscribing to one of our paid plans.

WIB22: Am­ber Salz­man had few op­tions when her son was di­ag­nosed with a rare ge­net­ic dis­ease. So she cre­at­ed a bet­ter one

This profile is part of Endpoints News’ 2022 special report about Women in Biopharma R&D. You can read the full report here.

Amber Salzman’s life changed on a cold, damp day in Paris over tiny plastic cups of lukewarm tea.

She was meeting with Patrick Aubourg, a French neurologist studying adrenoleukodystrophy, or ALD, a rare genetic condition that causes rapid neurological decline in young boys. It’s a sinister disease that often leads to disability or death within just a few years. Salzman’s nephew was diagnosed at just 6 or 7 years old, and died at the age of 12.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 154,300+ biopharma pros reading Endpoints daily — and it's free.

Ahead of ad­comm, FDA rais­es un­cer­tain­ties on ben­e­fit-risk pro­file of Cy­to­ki­net­ic­s' po­ten­tial heart drug

The FDA’s Cardiovascular and Renal Drugs Advisory Committee will meet next Tuesday to discuss whether Cytokinetics’ potential heart drug can safely reduce the risk of cardiovascular death and heart failure in patients with symptomatic chronic heart failure with reduced ejection fraction.

The drug, known as omecamtiv mecarbil and in development for more than 15 years, has seen mixed results, with a first Phase III readout from November 2020 hitting the primary endpoint of reducing the odds of hospitalization or other urgent care for heart failure by 8%. But it also missed a key secondary endpoint analysts had pegged as key to breaking into the market.

Ab­b­Vie slapped with age dis­crim­i­na­tion law­suit, fol­low­ing oth­er phar­mas

Add AbbVie to the list of pharma companies currently facing age discrimination allegations.

Pennsylvania resident Thomas Hesch filed suit against AbbVie on Wednesday, accusing the company of passing him over for promotions in favor of younger candidates.

Despite 30 years of pharma experience, “Hesch has consistently seen younger, less qualified employees promoted over him,” the complaint states.

Rami Elghandour, Arcellx CEO

Up­dat­ed: Gilead, Ar­cel­lx team up on an­ti-BC­MA CAR-T as biotech touts a 100% re­sponse rate at #ASH22

Gilead and Kite are plunking down big cash to get into the anti-BCMA CAR-T game.

The pair will shell out $225 million in cash upfront and $100 million in equity to Arcellx, Kite announced Friday morning, to develop the biotech’s lead CAR-T program together. Kite will handle commercialization and co-development with Arcellx, and profits in the US will be split 50-50.

Concurrent with the deal, Arcellx revealed its latest cut of data for the program known as CART-ddBCMA, ahead of a full presentation at this weekend’s ASH conference — a 100% response rate among patients getting the therapy. Investors jumped at the dual announcements, sending Arcellx shares $ACLX up more than 25% in Friday’s morning session.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 154,300+ biopharma pros reading Endpoints daily — and it's free.

WIB22: Lead­ing NK cell re­searcher re­flects on roots in Iran, the UK and Texas

This profile is part of Endpoints News’ 2022 special report about Women in Biopharma R&D. You can read the full report here.

In a small but widely-cited 11-person study published in NEJM in 2020, seven patients saw signs of their cancer completely go away after getting a new therapy made from natural killer cells. The study was one of the earliest to provide clinical proof that the experimental treatment method had promise.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 154,300+ biopharma pros reading Endpoints daily — and it's free.

WIB22: Chas­ing af­ter ever-evolv­ing sci­ence takes a drug hunter across the pond

This profile is part of Endpoints News’ 2022 special report about Women in Biopharma R&D. You can read the full report here.

Like many scientists, Fiona Marshall would tell you that she loved the natural world growing up — going to look at crabs running around the beach near her childhood home, pondering about the tides. But one thing about biology, in particular, stood out: It was constantly changing, and changing very quickly.

Endpoints News

Keep reading Endpoints with a free subscription

Unlock this story instantly and join 154,300+ biopharma pros reading Endpoints daily — and it's free.

Christian Itin, Autolus CEO (UKBIO19)

Au­to­lus tips its hand, bags $220M as CAR-T show­down with Gilead looms

The first batch of pivotal data on Autolus Therapeutics’ CAR-T is in, and execs are ready to plot a path to market.

With an overall remission rate of 70% at the interim analysis featuring 50 patients, the results set the stage for a BLA filing by the end of 2023, said CEO Christian Itin.

Perhaps more importantly — given that Autolus’ drug, obe-cel, is going after an indication that Gilead’s Tecartus is already approved for — the biotech highlighted “encouraging safety data” in the trial, with a low percentage of patients experiencing severe immune responses.