Covid-19 roundup: As­traZeneca paus­es vac­cine tri­al for chil­dren as blood clot probe con­tin­ues; EMA to in­ves­ti­gate whether Sput­nik V tri­als met GCP stan­dards — re­port

As­traZeneca and part­ners at Ox­ford Uni­ver­si­ty have halt­ed dos­ing of their Covid-19 vac­cine to chil­dren in a small clin­i­cal tri­al in the lat­est hic­cup caused by safe­ty scares.

MHRA — the UK’s med­i­cines reg­u­la­tor — has been con­duct­ing what they called a “thor­ough and de­tailed re­view” to in­ves­ti­gate a pos­si­ble link be­tween the vac­cine and rare blood clots in adults.

“Whilst there are no safe­ty con­cerns in the pae­di­atric clin­i­cal tri­al, we await ad­di­tion­al in­for­ma­tion from the MHRA on its re­view of rare cas­es of throm­bo­sis/throm­bo­cy­topae­nia that have been re­port­ed in adults, be­fore giv­ing any fur­ther vac­ci­na­tions in the tri­al,” An­drew Pol­lard, chief in­ves­ti­ga­tor of the tri­al, told the BBC.

Around 300 vol­un­teers had signed up for the study, which start­ed around mid-Feb­ru­ary. It’s un­clear how many have al­ready re­ceived the vac­cine (or how many dos­es they got).

The ac­knowl­edg­ment of a pause comes af­ter a top vac­cine ex­pert at the EMA said in an in­ter­view with Ital­ian news­pa­per Il Mes­sag­gero that there is a “clear” as­so­ci­a­tion be­tween the vac­cines and throm­bot­ic events, but the caus­es re­main un­known.

It was the first time a health of­fi­cial pub­licly sug­gest­ed a con­nec­tion, fol­low­ing a tense few weeks of coun­tries across Eu­rope sus­pend­ing use of the shot due to re­ports of blood clots.

Days ago, the EMA came out with “a clear sci­en­tif­ic con­clu­sion that this is a safe and ef­fec­tive vac­cine,” and called for vac­ci­na­tions to con­tin­ue while coun­tries raise aware­ness of these pos­si­ble rare con­di­tions. The agency is set to pro­vide up­dat­ed guide­lines lat­er in the week. — Am­ber Tong

EMA to in­ves­ti­gate whether Sput­nik V tri­als met GCP stan­dards — re­port

As the EU strug­gles with vac­cine roll­out, the EMA will launch an in­ves­ti­ga­tion next week in­to whether Rus­sia’s con­tro­ver­sial Sput­nik V vac­cine meets “good clin­i­cal prac­tice” (GCP) stan­dards, the Fi­nan­cial Times re­port­ed.

Un­named sources told the FT that the reg­u­la­tor is tak­ing a deep­er look due to con­cerns that the Sput­nik V tri­als weren’t eth­i­cal­ly run. Reuters has pre­vi­ous­ly re­port­ed that state em­ploy­ees were pres­sured by their su­pe­ri­ors to en­roll in the tri­als.

One mu­nic­i­pal of­fi­cer, Sergei Martyanov, al­leged­ly told col­leagues to re­cruit friends and fam­i­ly in the tri­als, send­ing out the mes­sage: “At least two peo­ple per em­ploy­ee!” ac­cord­ing to Reuters.

Kir­ill Dmitriev, head of the Russ­ian Di­rect In­vest­ment Fund, de­nied the al­le­ga­tions to the FT. He said the vac­cine has al­ready been au­tho­rized in 59 coun­tries, and that there was “no pres­sure [on par­tic­i­pants] and Sput­nik V com­plied with all clin­i­cal prac­tices.”

Sput­nik V be­came the world’s first reg­is­tered Covid-19 vac­cine back in Au­gust, when the coun­try au­tho­rized the jab be­fore it cleared Phase III tri­als. In Feb­ru­ary, re­searchers wrote in The Lancet that the vac­cine had an ef­fi­ca­cy rate of 91.6%, based on the num­ber of Covid-19 cas­es tracked 21 days af­ter the first dose was ad­min­is­tered in a Phase III tri­al.

But last month, the head of the EMA’s man­age­ment board ad­vised EU coun­tries to hold off on au­tho­riz­ing the vac­cine while the EMA re­views its safe­ty and ef­fi­ca­cy, prompt­ing the shot’s de­vel­op­ers to de­mand an apol­o­gy.

“We de­mand a pub­lic apol­o­gy from EMA’s Christa Wirthumer-Hoche for her neg­a­tive com­ments on EU states di­rect­ly ap­prov­ing Sput­nik V,” a post from the of­fi­cial Sput­nik V Twit­ter ac­count read. “Her com­ments raise se­ri­ous ques­tions about pos­si­ble po­lit­i­cal in­ter­fer­ence in the on­go­ing EMA re­view.” — Nicole De­Feud­is

This wasn’t the first time Emer­gent BioSo­lu­tions has tossed mil­lions of vac­cine dos­es — re­port

Days af­ter Emer­gent BioSo­lu­tions lost con­trol of its Bal­ti­more plant for botch­ing mil­lions of Covid-19 vac­cine dos­es, re­ports have emerged that this wasn’t the first time the com­pa­ny had to toss dos­es.

Last week, the feds di­rect­ed J&J to take con­trol of the plant af­ter Emer­gent mixed up vac­cines from J&J and As­traZeneca, wast­ing up to 15 mil­lion dos­es. Emer­gent was tagged for mul­ti­ple op­er­at­ing vi­o­la­tions.

But ac­cord­ing to the New York Times, this has hap­pened be­fore. The news or­ga­ni­za­tion re­port­ed that Emer­gent threw out five lots of As­traZeneca’s vac­cine be­tween Oc­to­ber and Jan­u­ary — each con­tain­ing 2 to 3 mil­lion dos­es — due to con­t­a­m­i­na­tion or sus­pect­ed con­t­a­m­i­na­tion.

Au­dits viewed by the Times re­vealed that Emer­gent hadn’t fol­lowed ba­sic in­dus­try stan­dards at the Bal­ti­more plant, as well as risks of vi­ral cross-con­t­a­m­i­na­tion, mold prob­lems, and poor dis­in­fec­tion.

Emer­gent spokesper­son Matt Hartwig told the Times the com­pa­ny was work­ing with the feds to “ad­dress is­sues,” adding: “Any al­le­ga­tion that our safe­ty, qual­i­ty and com­pli­ance sys­tems are not work­ing or that we do not take these re­spon­si­bil­i­ties se­ri­ous­ly is false.”

The com­pa­ny has won more than $1.3 bil­lion in con­tracts from the gov­ern­ment as well as J&J and As­traZeneca to pro­duce dos­es. — Nicole De­Feud­is 

Cure­Vac co-founder calls out ‘US first’ sup­ply chains for Covid-19 vac­cine man­u­fac­tur­ing dis­rup­tions in EU

For vac­cine play­ers in the EU look­ing to bring their Covid-19 jabs to mar­ket, a dearth of ma­te­ri­als orig­i­nat­ing from US man­u­fac­tur­ers has slowed pro­duc­tion. Now, mR­NA play­er Cure­Vac’s co-founder is call­ing out an Amer­i­ca-first man­u­fac­tur­ing man­date for those short­ages.

“Glob­al sup­ply chains are dis­rupt­ed,” Flo­ri­an von der Mülbe, who’s al­so Cure­Vac’s chief pro­duc­tion of­fi­cer, said to the Rheinis­che Post. “Be it chem­i­cals, equip­ment, fil­ters or hoses: U.S. man­u­fac­tur­ers are oblig­ed first to meet Amer­i­can de­mand, and that means we are slip­ping down the list.”

Von der Mülbe added that vac­cine mak­ers look­ing to house pro­duc­tion in Eu­rope are at a dis­ad­van­tage be­cause sup­pli­ers in the US are re­quired to cater to the home mar­ket at first un­der the De­fense Pro­duc­tion Act, ac­cord­ing to Reuters. Cure­Vac’s cur­rent pro­duc­tion is based at its Tübin­gen, Ger­many plant.

In Ju­ly, Glax­o­SmithK­line pledged to pump $293 mil­lion in­to the mR­NA-based Cure­Vac vac­cine. The in­vest­ment meant a big bet on mR­NA as the core tech­nol­o­gy be­hind fu­ture vac­cines and mon­o­clon­al an­ti­bod­ies. Per­haps most no­tably, that deal left Cure­Vac re­spon­si­ble for man­u­fac­tur­ing, all the way to the mar­ket.

Cure­Vac has made ef­forts to stay ahead of the game as new Covid-19 vari­ants have emerged. Two weeks ago, the com­pa­ny an­nounced that it was ex­pand­ing tri­al pro­to­col for its Phase IIb/III in Eu­rope and Latin Amer­i­ca to de­ter­mine ef­fi­ca­cy for dif­fer­ent strains of the virus. The com­pa­ny hopes to file for au­tho­riza­tion in Eu­rope in the sec­ond quar­ter.

Right now, Cure­Vac has an al­liance with No­var­tis to help man­u­fac­ture the drug in Aus­tria, with plans to pro­duce up to 50 mil­lion dos­es by 2021. — Josh Sul­li­van

NIH to launch study on vac­cine al­ler­gic re­ac­tions

The NIH on Wednes­day an­nounced that a clin­i­cal tri­al is un­der­way to study al­ler­gic re­ac­tions to the Mod­er­na and Pfiz­er-BioN­Tech vac­cines.

The tri­al aims to de­ter­mine whether those who are high­ly al­ler­gic or have a mast cell dis­or­der are at in­creased risk to a re­ac­tion from the vac­cine, a re­lease from the NIH said. If an al­ler­gic re­ac­tion oc­curs in study par­tic­i­pants, in­ves­ti­ga­tors will try to de­ter­mine if a re­ac­tion is more com­mon in these sub­jects than those with no al­ler­gy his­to­ry.

“The in­for­ma­tion gath­ered dur­ing this tri­al will help doc­tors ad­vise peo­ple who are high­ly al­ler­gic or have a mast cell dis­or­der about the risks and ben­e­fits of re­ceiv­ing these two vac­cines,” NI­AID Di­rec­tor An­tho­ny Fau­ci said in the state­ment. “How­ev­er, for most peo­ple, the ben­e­fits of COVID-19 vac­ci­na­tion far out­weigh the risks.”

The Phase II tri­al is spon­sored and fund­ed by the NI­AID.

Fol­low­ing re­ports of ear­ly al­ler­gic re­ac­tions to the Pfiz­er-BioN­Tech vac­cine in Jan­u­ary, Mod­er­na in­ves­ti­gat­ed one of its own vac­cine batch­es for the same rea­son out of an “abun­dance of cau­tion.” — Josh Sul­li­van

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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Joe Biden (Carolyn Kaster, AP Images)

Covid-19 roundup: Biden in­vests $1.7B to ad­dress Covid vari­ants; EU puts faith in Pfiz­er with new vac­cine deals

The Biden administration said Friday that it’ll pump $1.7 billion into various programs to address Covid-19 variants as the original strain of Covid-19 makes up only about half of all US cases today.

Most of those new funds, $1 billion in total, will go to expand genomic sequencing so the CDC, states and other jurisdictions can improve their capacity to identify Covid mutations and monitor the circulation of variants. Back in February, US labs were only sequencing about 8,000 Covid-19 strains per week, although the rate of sequencing has increased substantially since then, the administration said.

Osman Kibar (Samumed, now Biosplice)

Os­man Kibar lays down his hand at Sa­mumed, step­ping away from CEO role as his once-her­ald­ed an­ti-ag­ing biotech re­brands

Samumed made quite the entrance back in 2016, when it launched with some anti-aging programs and a whopping $12 billion valuation. That level of fanfare was nowhere to be found on Thursday, when the company added another $120 million to its coffers and quietly changed its name to Biosplice Therapeutics.

Why the sudden rebrand?

“We did that for obvious reasons,” CFO and CBO Erich Horsley told Endpoints News. “The name Biosplice echoes our science much more than Samumed does.”

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