Neil King (University of Washington)

Is a 'su­per-sea­son­al' flu vac­cine on the hori­zon? The NIH and UW are tak­ing a shot in­to the clin­ic this April

Sev­er­al biotechs and phar­ma com­pa­nies are look­ing to dis­cov­er a uni­ver­sal flu vac­cine that could pro­tect hu­mans from any strain of the virus, re­gard­less of which is pre­dict­ed to cir­cu­late. But giv­en how many of these ef­forts are like­ly years away at best, are there any in­ter­me­di­ate steps avail­able to bridge that gap?

That’s the ques­tion Uni­ver­si­ty of Wash­ing­ton re­searcher Neil King and a group of NIH sci­en­tists are hop­ing to an­swer. In a new Na­ture pa­per pub­lished Wednes­day, the team was able to demon­strate broad ef­fec­tive­ness in an­i­mal mod­els for a “su­per-sea­son­al” flu vac­cine by dis­play­ing mul­ti­ple flu strains at the same time. And King says it’s start­ing hu­man tri­als at the end of April.

The big idea is in­stead of get­ting dif­fer­ent flu shots every year as the virus mu­tates, one could get pro­tec­tion from one vac­cine for three to five years, King said.

“Even that would be a mas­sive ad­vance,” King told End­points News. “The virus mu­tates a lit­tle bit and you’re still cov­ered. And the next year it mu­tates a lit­tle bit more and you’re still cov­ered.”

The tech­nol­o­gy King used to ac­com­plish this is sim­i­lar to virus-like par­ti­cles, though he says he would not use that term for “very tech­ni­cal rea­sons.” It’s still used in a sim­i­lar fash­ion, how­ev­er, on­ly in­stead of uti­liz­ing a cap­sid that lacks a genome, the group de­signed pro­tein nanopar­ti­cles that can self-as­sem­ble.

Es­sen­tial­ly what you get are two of these nanopar­ti­cles, which sep­a­rate­ly don’t do much. But when you mix them to­geth­er in a test tube, King said, they as­sem­ble them­selves in­to a new mol­e­cule that can func­tion like a VLP. The tech­nol­o­gy is the same that’s be­ing used by Icosavax, though the com­pa­ny was not in­volved in this spe­cif­ic pa­per.

Su­per-sea­son­al flu shots have been at­tempt­ed be­fore, but the un­der­ly­ing twist here is while the in vit­ro as­sem­bly of the nanopar­ti­cles was the same, King and the NIH team man­aged to codis­play mul­ti­ple flu strains on the same nanopar­ti­cle. They man­aged to in­duce broad re­spons­es in mice, fer­rets and non-hu­man pri­mates, hit­ting not just the “head” of the spe­cif­ic strains but al­so the “stem.”

The stem of the virus strain — whose sci­en­tif­ic jar­gon name is hemag­glu­tinin — is what many sci­en­tists have been af­ter for a uni­ver­sal flu shot, King said, as cur­rent flu shots on­ly go for the head. Some ap­proach­es in­volve cut­ting off the head to fo­cus on­ly on the stem, but the nanopar­ti­cle ap­proach man­aged to train the im­mune sys­tem to at­tack both.

“Tra­di­tion­al­ly that’s been very hard. The im­mune sys­tem just doesn’t re­act very strong­ly to the stem,” King said. “But some­thing about putting these hemag­glu­tinins on our nanopar­ti­cles taught the im­mune sys­tem, ‘Hey, give me the head … but al­so go get that stem too.’ And that is what’s pro­vid­ing the pro­tec­tive breadth from these vac­cines.”

On top of that, the group used the same strains cur­rent­ly in com­mer­cial flu shots. That proved a key part in their ef­forts, King said, be­cause it makes a po­ten­tial reg­u­la­to­ry path through the FDA much eas­i­er.

Be­cause their ex­per­i­men­tal vac­cine has al­ready been man­u­fac­tured, the NIH is bar­rel­ing ahead with a Phase I tri­al planned to start next month. By the end of the year, King says the nanopar­ti­cle tech­nol­o­gy will have four vac­cine pro­grams in the clin­ic: the su­per-sea­son­al flu shot, two po­ten­tial Covid-19 vac­cines and a pro­gram for res­pi­ra­to­ry syn­cy­tial virus, or RSV.

But in or­der to ful­ly com­mer­cial­ize the flu shots and take them all the way through piv­otal Phase III stud­ies, King said they still need a phar­ma com­pa­ny to li­cense the pro­gram. There has been ini­tial in­ter­est from at least two com­pa­nies, though King stayed mum on who’s look­ing.

At the end of the day, the su­per-sea­son­al flu shots are still a ways away from their first sales and even longer from re­plac­ing con­ven­tion­al flu vac­cines, which still use tech­nol­o­gy from the 1930s by grow­ing the pre­dict­ed strains in­side chick­en eggs, King said. A tra­di­tion­al time­line pre-Covid-19 might have tak­en 10 years, but now every­thing is up in the air.

“Af­ter what we saw in 2020 with SARS-CoV-2, I don’t know,” King said about a time­line. “It could be faster. But this thing is en­ter­ing Phase I next month, you have to do Phase II, Phase III, these things do take time. So my guess is it would be at least five years, but who re­al­ly knows?”

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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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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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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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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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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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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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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Pascal Soriot (AstraZeneca via YouTube)

Af­ter be­ing goad­ed to sell the com­pa­ny, Alex­ion's CEO set some am­bi­tious new goals for in­vestors. Then Pas­cal So­ri­ot came call­ing

Back in the spring of 2020, Alexion $ALXN CEO Ludwig Hantson was under considerable pressure to perform and had been for months. Elliott Advisers had been applying some high public heat on the biotech’s numbers. And in reaching out to some major stockholders, one thread of advice came through loud and clear: Sell the company or do something dramatic to change the narrative.

In the words of the rather dry SEC filing that offers a detailed backgrounder on the buyout deal, Alexion stated: ‘During the summer and fall of 2020, Alexion also continued to engage with its stockholders, and in these interactions, several stockholders encouraged the company to explore strategic alternatives.’

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