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.

The DCT-OS: A Tech­nol­o­gy-first Op­er­at­ing Sys­tem - En­abling Clin­i­cal Tri­als

As technology-enabled clinical research becomes the new normal, an integrated decentralized clinical trial operating system can ensure quality, deliver consistency and improve the patient experience.

The increasing availability of COVID-19 vaccines has many of us looking forward to a time when everyday things return to a state of normal. Schools and teachers are returning to classrooms, offices and small businesses are reopening, and there’s a palpable sense of optimism that the often-awkward adjustments we’ve all made personally and professionally in the last year are behind us, never to return. In the world of clinical research, however, some pandemic-necessitated adjustments are proving to be more than emergency stopgap measures to ensure trial continuity — and numerous decentralized clinical trial (DCT) tools and methodologies employed within the last year are likely here to stay as part of biopharma’s new normal.

Angela Merkel (AP Photo/Michael Sohn)

Covid-19 roundup: Pfiz­er sub­mits vac­cine for full ap­proval; Merkel op­pos­es Biden pro­pos­al to sus­pend IP for vac­cines

Pfizer and BioNTech said Friday that they’ve submitted a biologics license application to the FDA for full approval of their mRNA vaccine for those over the age of 16.

How long it will take the FDA to decide on the BLA will be set once it’s been formally accepted by the agency.

Peter Marks, director of the FDA’s Center for Biologics Evaluation and Research, previously told Endpoints News that the review of the BLA should take between three and four months, but it may be even faster than that.

Stéphane Bancel, Getty

Mod­er­na CEO brush­es off US sup­port for IP waiv­er, eyes more than $19B in Covid-19 vac­cine sales in 2021

Moderna is definitively more concerned with keeping pace with Pfizer in the race to vaccinate the world against Covid-19 than it is with Wednesday’s decision from the Biden administration to back an intellectual property waiver that aims to increase vaccine supplies worldwide.

In its first quarter earnings call on Thursday, Moderna CEO Stéphane Bancel shrugged off any suggestion that the newly US-backed intellectual property waiver would impact his company’s vaccine or bottom line. Still, the company’s stock price fell by about 9% in early morning trading.

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Onno van de Stolpe, Galapagos CEO (Thierry Roge/Belga Mag/AFP via Getty Images)

Gala­pa­gos chops in­to their pipeline, drop­ping core fields and re­or­ga­niz­ing R&D as the BD team hunts for some­thing 'trans­for­ma­tive'

Just 5 months after Gilead gutted its rich partnership with Galapagos following a bitter setback at the FDA, the Belgian biotech is hunkering down and chopping the pipeline in an effort to conserve cash while their BD team pursues a mission to find a “transformative” deal for the company.

The filgotinib disaster didn’t warrant a mention as Galapagos laid out its Darwinian restructuring plans. Forced to make choices, the company is ditching its IPF molecule ’1205, while moving ahead with a Phase II IPF study for its chitinase inhibitor ’4617.

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EMA safe­ty com­mit­tee seeks more in­fo on heart in­flam­ma­tion fol­low­ing Pfiz­er Covid-19 vac­cine

The European Medicines Agency’s safety committee said Friday that it’s aware of cases of inflammation of the heart muscle and inflammation of the membrane around the heart, mainly reported following vaccination with Pfizer’s Covid-19 vaccine, known in Europe as Comirnaty.

“There is no indication that these cases are due to the vaccine,” the EMA’s Pharmacovigilance Risk Assessment Committee said.

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An­oth­er failed tri­al for Or­p­hazyme's 'pipeline-in-a-pro­duc­t' leaves shad­ow on drug's fu­ture

The tumultuous ride for Orphazyme continued on Friday as the company announced that a pivotal trial for its lead drug arimoclomol failed yet again, this time in the treatment of ALS, seeding doubt in a drug that had recently been cleared by the FDA for priority review. The latest failure casts a darker shadow on the upcoming decision despite Orphazyme’s upbeat outlook.

In a statement, the Danish biotech announced that the drug did not meet its primary or secondary endpoints evaluating function and survival. But the company has not announced any data surrounding the failure, instead saying that it will publish the complete results later this year.

In­cyte ponies up $12M to set­tle char­i­ty foun­da­tion kick­back claims; US ex­er­cis­es op­tion for more dos­es of mon­key­pox vac­cine

One in a string of lawsuits targeting copay charity foundations, the DOJ has been hunting drugmaker Incyte for what prosecutors alleged was a kickback scheme to court patients. Now, Incyte is clearing its name.

Incyte will shell out $12.6 million to settle claims it funneled funds through a charity foundation to cover federal copays for patients taking its JAK inhibitor Jakafi, the DOJ said this week.

CEO Khurem Farooq (Gyroscope)

Hours be­fore ex­pect­ed de­but, Gy­ro­scope post­pones its IPO as 2 oth­er biotechs hold the line on their march to Nas­daq

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

In a surprising turn of events, UK-based Gyroscope Therapeutics has postponed its IPO mere hours before it was set to debut on Nasdaq.

Working on a gene therapy for wet AMD, Gyroscope was all set and ready to go public earlier this week, setting terms for a $142 million raise with a price range of $20 to $22. But in the wee hours of Friday morning, the company put out a press release saying they would delay their debut “in light of market conditions,” CEO Khurem Farooq said in a statement.

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Karyopharm taps long­time Pfiz­er, Am­gen vet to steer the ship; With Mer­ck in the rearview mir­ror, Roger Perl­mut­ter stakes his claim to a CEO job — and it's a sur­pris­ing choice

Like many who work in biopharma, Richard Paulson got started in the field because of a love of science.

Paulson had just finished business school and was looking to start a career that married his two passions. While looking for jobs, he thought of his grandmother who had struggled with Alzheimer’s disease, recalling how he saw first-hand what innovative medicines can do for patients. Ultimately, he started his first job in the space as a sales rep at Glaxo Wellcome, one of GlaxoSmithKline’s predecessors before its merger with SmithKline Beecham in 2000.