Mene Pangalos (via AstraZeneca)

Un­der fire, As­traZeneca hus­tles out a pri­ma­ry cut of the num­bers for its Covid-19 vac­cine sup­port­ing pos­i­tive ef­fi­ca­cy

Af­ter spend­ing the last 48 hours in a pres­sure cook­er of wide­spread crit­i­cism, As­traZeneca has up­dat­ed its da­ta on its con­tro­ver­sial Covid-19 vac­cine.

The phar­ma gi­ant an­nounced Wednes­day evening that their vac­cine was 76% ef­fec­tive in the pri­ma­ry analy­sis, a small slip on over­all ef­fi­ca­cy from its first cut of the da­ta. There was 100% ef­fi­ca­cy in pre­vent­ing se­vere dis­ease and hos­pi­tal­iza­tion. The slight re­vi­sion still bears up well in com­par­i­son with the 72% ef­fi­ca­cy rate cit­ed by J&J for its vac­cine, leav­ing As­traZeneca in a sol­id po­si­tion to win an emer­gency au­tho­riza­tion from the FDA.

The da­ta came from 190 over­all cas­es of Covid-19, in­clud­ing 49 new cas­es since their pre­vi­ous da­ta cut. There were 8 cas­es of se­vere dis­eases, all of them in the place­bo group.

As­traZeneca R&D leader Mene Pan­ga­los says the com­pa­ny is push­ing straight ahead with its re­quest for emer­gency au­tho­riza­tion in the US.

The pri­ma­ry analy­sis is con­sis­tent with our pre­vi­ous­ly re­leased in­ter­im analy­sis, and con­firms that our COVID-19 vac­cine is high­ly ef­fec­tive in adults, in­clud­ing those aged 65 years and over. We look for­ward to fil­ing our reg­u­la­to­ry sub­mis­sion for Emer­gency Use Au­tho­riza­tion in the US and prepar­ing for the roll­out of mil­lions of dos­es across Amer­i­ca.

As­traZeneca had earned ku­dos on Mon­day with a round of in­ter­im da­ta that spot­light­ed a 79% rate in pre­vent­ing symp­to­matic Covid-19 with a 100% elim­i­na­tion of se­vere dis­ease. For the multi­na­tion­al op­er­a­tion, that score marked a dra­mat­ic turn­around from the roast­ing it was get­ting in Eu­rope and around the world as ex­perts and health of­fi­cials ques­tioned its ef­fec­tive­ness in com­bat­ing vari­ants or in peo­ple over the age of 65.

But that mo­men­tary turn of events evap­o­rat­ed late Mon­day, af­ter the DSMB charged with over­see­ing the tri­al alert­ed of­fi­cials at the NIH that the com­pa­ny had ig­nored its rec­om­men­da­tion to tell the pub­lic that the da­ta they were see­ing in Feb­ru­ary and March in­di­cat­ed a rate of 69% to 74%.

It amount­ed to sim­ple cher­ry pick­ing of the da­ta, they not­ed. In their let­ter, which The Wash­ing­ton Post ob­tained, the ob­servers not­ed:

The point that is clear to the board is that the [vac­cine ef­fi­ca­cy num­ber] . . . they chose to re­lease was the most fa­vor­able for the study as op­posed to the most re­cent and most com­plete. De­ci­sions like this are what erode pub­lic trust in the sci­en­tif­ic process.

That sort of em­bell­ish­ment was wide­ly con­demned, and Scripps’ Er­ic Topol added to the din of crit­i­cism, re­peat­ed­ly as­sert­ing on Twit­ter that As­traZeneca could in­stant­ly clear the air.

NI­AID chief An­tho­ny Fau­ci, mean­while, con­sis­tent­ly as­sert­ed that while the da­ta may have been wrong, the fi­nal num­bers would bear out its over­all val­ue.

The new da­ta help con­firm that as­ser­tion, al­though the num­bers are still not en­tire­ly set­tled: As­traZeneca said that there were 14 ad­di­tion­al pos­si­ble Covid-19 cas­es that had yet to be ful­ly ad­ju­di­cat­ed.

At this stage of the game, af­ter so much crit­i­cism in a storm of con­tro­ver­sy, the vac­cine is still like­ly to run in­to stiff re­sis­tance — par­tic­u­lar­ly in the more af­flu­ent na­tions that have a choice. Poor coun­tries, though, now may have lit­tle choice in the mat­ter.

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

BY­OD Best Prac­tices: How Mo­bile De­vice Strat­e­gy Leads to More Pa­tient-Cen­tric Clin­i­cal Tri­als

Some of the most time- and cost-consuming components of clinical research center on gathering, analyzing, and reporting data. To improve efficiency, many clinical trial sponsors have shifted to electronic clinical outcome assessments (eCOA), including electronic patient-reported outcome (ePRO) tools.

In most cases, patients enter data using apps installed on provisioned devices. At a time when 81% of Americans own a smartphone, why not use the device they rely on every day?

Chris Gibson (Photo By Vaughn Ridley/Sportsfile for Web Summit via Getty Images)

Re­cur­sion founders gin for­tunes as IPO back­ers show­er $436M on one of the biggest boasts in AI -- based on some very small deals

In the AI drug development world, boasting often comes with the territory. Yet few can rival Recursion when it comes to claiming the lead role in what company execs like to call the industrialization of drug development, with promises of continued exponential growth in the number of drugs it has in the pipeline.

On Friday, the Salt Lake City-based biotech translated its unicorn-sized boasts into a killer IPO, pricing more than 24 million shares at the high end of its range and bringing in $436 million — with a large chunk of that promised by some deep-pocket backers.

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Image: Shutterstock

Eli Lil­ly asks FDA to re­voke EUA for Covid-19 treat­ment

Eli Lilly on Friday requested that the FDA revoke the emergency authorization for its Covid-19 drug bamlanivimab, which is no longer as effective as a combo therapy because of a rise in coronavirus variants across the US.

“With the growing prevalence of variants in the U.S. that bamlanivimab alone may not fully neutralize, and with sufficient supply of etesevimab, we believe now is the right time to complete our planned transition and focus on the administration of these two neutralizing antibodies together,” Daniel Skovronsky, Lilly’s CSO, said in a statement.

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Covid-19 vac­cine halt drags on, an FDA ap­point­ment at long last, the great CRO con­sol­i­da­tion, and more

Welcome back to Endpoints Weekly, your review of the week’s top biopharma headlines. Want this in your inbox every Saturday morning? Current Endpoints readers can visit their reader profile to add Endpoints Weekly. New to Endpoints? Sign up here.

Conference season is upon us, and while we’d much prefer to be wandering down the hallways and presentation rooms in person, the team is ready to cover the most consequential data coming out of these scientific meetings. Get in touch early if you have news to share.

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Ex­clu­sive in­ter­view: Pe­ter Marks on why full Covid-19 vac­cine ap­provals could be just months away

Peter Marks, director of the FDA’s Center for Biologics Evaluation and Research, took time out of his busy schedule last Friday to discuss with Endpoints News all things related to his work regulating vaccines and the pandemic.

Marks, who quietly coined the name “Operation Warp Speed” before deciding to stick with his work regulating vaccines at the FDA rather than join the Trump-era program, has been the face of vaccine regulation for the FDA throughout the pandemic, and is usually spotted in Zoom meetings seated in front of his wife’s paintings.

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J&J faces CDC ad­vi­so­ry com­mit­tee again next week to weigh Covid-19 vac­cine risks

The CDC’s Advisory Committee on Immunization Practices punted earlier this week on deciding whether or not to recommend lifting a pause on the administration of J&J’s Covid-19 vaccine, but the committee will meet again in an emergency session next Friday to discuss the safety issues further.

The timing of the meeting likely means that the J&J vaccine will not return to the US market before the end of next week as the FDA looks to work hand-in-hand with the CDC to ensure the benefits of the vaccine still outweigh the risks for all age groups.

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Near­ly a year af­ter Au­den­tes' gene ther­a­py deaths, the tri­al con­tin­ues. What hap­pened re­mains a mys­tery

Natalie Holles was five months into her tenure as Audentes CEO and working to smooth out a $3 billion merger when the world crashed in.

Holles and her team received word on the morning of May 5 that, hours before, a patient died in a trial for their lead gene therapy. They went into triage mode, alerting the FDA, calling trial investigators to begin to understand what happened, and, the next day, writing a letter to alert the patient community so they would be the first to know. “We wanted to be as forthright and transparent as possible,” Holles told me late last month.

The brief letter noted two other patients also suffered severe reactions after receiving a high dose of the therapy and were undergoing treatment. One died a month and a half later, at which point news of the deaths became public, jolting an emergent gene therapy field and raising questions about the safety of the high doses Audentes and others were now using. The third patient died in August.

“It was deeply saddening,” Holles said. “But I was — we were — resolute and determined to understand what happened and learn from it and get back on track.”

Eleven months have now passed since the first death and the therapy, a potential cure for a rare and fatal muscle-wasting disease called X-linked myotubular myopathy, is back on track, the FDA having cleared the company to resume dosing at a lower level. Audentes itself is no more; last month, Japanese pharma giant Astellas announced it had completed working out the kinks of the $3 billion merger and had restructured and rebranded the subsidiary as Astellas Gene Therapies. Holles, having successfully steered both efforts, departed.

Still, questions about precisely what led to the deaths of the 3 boys still linger. Trial investigators released key details about the case last August and December, pointing to a biological landmine that Audentes could not have seen coming — a moment of profound medical misfortune. In an emerging field that’s promised cures for devastating diseases but also seen its share of safety setbacks, the cases provided a cautionary tale.

Audentes “contributed in a positive way by giving a painful but important example for others to look at and learn from,” Terry Flotte, dean of the UMass School of Medicine and editor of the journal Human Gene Therapy, told me. “I can’t see anything they did wrong.”

Yet some researchers say they’re still waiting on Astellas to release more data. The company has yet to publish a full paper detailing what happened, nor have they indicated that they will. In the meantime, it remains unclear what triggered the events and how to prevent them in the future.

“Since Audentes was the first one and we don’t have additional information, we’re kind of in a holding pattern, flying around, waiting to figure out how to land our vehicles,” said Jude Samulski, professor of pharmacology at UNC’s Gene Therapy Center and CSO of the gene therapy biotech AskBio, now a subsidiary of Bayer.

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Mer­ck scraps their $425M Covid-19 drug in lat­est pan­dem­ic set­back

Seven months after paying $425 million cash to acquire it, Merck is scrapping a Covid-19 drug they hoped could provide one of the only treatments for severe hospitalized patients.

Merck’s decision comes after they faced significant and unexpected regulatory delays in getting the drug, known as MK-7110 or CD24Fc, across the finish line. The Big Pharma licensed the drug under the belief that it had already shown sufficient benefit in severe patients and they could help scale it up far faster than OncoImmune, its former owner, could. But in February, the company reported that the FDA insisted Merck run a new trial before seeking authorization.

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Severin Schwan, Roche CEO (Georgios Kefalas/Keystone via AP Images)

Look­ing to ce­ment its lead in packed MS mar­ket, Roche's Ocre­vus un­corks new da­ta in ear­ly-stage pa­tients

Among a positively jam-packed multiple sclerosis market, Roche’s Ocrevus has managed to stand out for what the Swiss drugmaker is calling the most successful launch in its long history. But in order to press its advantage, Ocrevus is looking to earlier-stage patients, and new interim data should help build its case there.

After 48 weeks on Roche’s Ocrevus, 85% of newly diagnosed primary progressing or relapsing MS patients without a history of disease modifying therapy posted no disease activity, including disease progression or relapse, according to interim data set to be presented this weekend at the virtual American Academy of Neurology meeting.