Covid-19 roundup: Mer­ck pre­pares new study on their $425M Covid drug; CO­V­AX be­gins roll­out but huge dis­par­i­ties still loom

Mer­ck spent $425 mil­lion to buy out On­coIm­mune in No­vem­ber, pre­sum­ably un­der the be­lief that they could scale up their drug for se­vere Covid-19 and get it through the FDA on the strength of a Phase III tri­al the small­er biotech com­plet­ed in Sep­tem­ber.

The FDA threw a wrench in those plans. The agency told Mer­ck last week that the 203-per­son study wasn’t suf­fi­cient to show the drug was ef­fec­tive. Now, Bloomberg re­ports, Mer­ck is prepar­ing an ad­di­tion­al Phase III study, one that will push re­sults — and any chance of ap­proval — back to the end of the year.

It’s an­oth­er Covid-19 set­back for Mer­ck, a Big Phar­ma long known for elite re­search, par­tic­u­lar­ly in in­fec­tious dis­ease; two years ago, they de­vel­oped the first vac­cine for Ebo­la. The com­pa­ny joined the pan­dem­ic fight late, and the var­i­ous tech­nolo­gies they’ve de­vel­oped have since stum­bled. They axed their two vac­cine pro­grams af­ter dispir­it­ing ear­ly re­sults, and they’ve said lit­tle about an Emory-de­vel­oped oral an­tivi­ral they li­censed and put in Phase III.

The FDA’s de­ci­sion is par­tic­u­lar­ly sur­pris­ing giv­en how the Mer­ck-On­coIm­mune buy­out came about. Bloomberg re­port­ed that Op­er­a­tion Warp Speed chief Mon­cef Slaoui bro­kered the deal af­ter the small biotech said their drug re­duced the risk of res­pi­ra­to­ry fail­ure or death in a Phase III tri­al. HHS then gave Mer­ck a con­tract for $356 mil­lion to sup­ply 100,000 dos­es by June.

“It’s not that the agency wasn’t im­pressed by the da­ta, be­cause they were,” Nicholas Kart­so­nis, Mer­ck’s clin­i­cal in­fec­tious dis­ease chief told Bloomberg. “They just want more of it, and more com­fort that if we’re in­deed go­ing to bring this for­ward, that the da­ta gets repli­cat­ed.”

CO­V­AX be­gins roll­out but huge dis­par­i­ties still loom

CO­V­AX, the WHO and Gates-backed vac­cine-shar­ing pro­gram, has had a rocky start, edged out in the hunt for vac­cines by the wealth­i­est coun­tries in the world. As shots rolled out in the US, the UK, and Eu­rope, CO­V­AX large­ly stood on the side­lines: At the end of Feb­ru­ary, more than 100 of the world’s poor­est coun­tries had yet to re­ceive a sin­gle dose.

The pro­gram, though, is be­gin­ning to set bench­marks. They post­ed their first round of glob­al vac­cine al­lo­ca­tions on Tues­day, sched­ul­ing 237 mil­lion dos­es of the As­traZeneca-Ox­ford vac­cine to ship to 142 na­tions by the end of May. The first ones be­gan on Mon­day, af­ter 600,000 dos­es land­ed in Ghana and 500,000 dos­es in Côte d’Ivoire.

The al­lo­ca­tions still amount to drops in the buck­et com­pared to the roll­out in the US alone, where more than 500 mil­lion dos­es have been se­cured in the same time pe­ri­od. Pres­i­dent Biden said Tues­day that the US would have enough vac­cines for its en­tire el­i­gi­ble adult pop­u­la­tion by May —  a bench­mark most of the world may not hit for years.

In ad­di­tion to shots from As­traZeneca, a com­pa­ny that pri­or­i­tized glob­al scale and ac­cess, CO­V­AX will soon be­gin to see shots from oth­er ma­jor de­vel­op­ers. Just over 1 mil­lion Pfiz­er-BioN­Tech vac­cines are in­clud­ed in the first al­lo­ca­tion and No­vavax, which has yet to be au­tho­rized any­where but re­cent­ly showed its vac­cine was high­ly ef­fec­tive in the UK and mod­er­ate­ly ef­fec­tive in South Africa, has pledged 1.1 bil­lion dos­es.

NIH pulls plug on con­va­les­cent plas­ma in pa­tients

The NIH is singing last rites on con­va­les­cent plas­ma.

More than a year af­ter doc­tors around the coun­try be­gan band­ing to­geth­er to get con­va­les­cent plas­ma off the ground as an emer­gency, stop­gap treat­ment for a new, emer­gency virus — and six months af­ter for­mer Pres­i­dent Trump pushed the FDA au­tho­rize it — the NIH has halt­ed its tri­al in con­va­les­cent plas­ma for mod­er­ate pa­tients af­ter a da­ta safe­ty and mon­i­tor­ing board found it had lit­tle ef­fect.

The study, called C3PO and launched in Au­gust, was one of the best hopes for prov­ing con­va­les­cent plas­ma could be an ef­fec­tive treat­ment for Covid-19. Al­though oth­er stud­ies, in­clud­ing the Mayo Clin­ic’s con­tro­ver­sial ex­pand­ed ac­cess pro­gram, fo­cused on se­vere pa­tients, re­searchers be­lieved it should work bet­ter the ear­li­er it was ad­min­is­tered. Small­er stud­ies from oth­er coun­tries bol­stered that be­lief.

The study had en­rolled 511 out of a planned 900 pa­tients. It showed the ther­a­py was safe but un­like­ly to help the group stud­ied.

The FDA al­ready be­gan to roll back its con­tro­ver­sial EUA for con­va­les­cent plas­ma last month, in the face of new ev­i­dence point­ing to its in­ef­fec­tive­ness in hos­pi­tal­ized pa­tients. They said it would on­ly be used in hos­pi­tal­ized pa­tients with ear­ly-stage dis­ease or an im­paired abil­i­ty to make their own an­ti­bod­ies.

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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