Joseph Payne, Arcturus CEO (Arcturus via YouTube)

Arc­turus touts $63M BAR­DA award for new mR­NA in­fluen­za vac­cine, hop­ing to prep against fu­ture pan­demics

A San Diego mR­NA biotech is look­ing to ex­pand its ef­forts in in­fluen­za, and a fed­er­al award will bring it clos­er to that goal.

Arc­turus Ther­a­peu­tics said Wednes­day that it won an award from HHS’s BAR­DA worth $63.2 mil­lion, a grant that was award­ed as part of the en­ti­ty’s pan­dem­ic in­fluen­za ef­forts. The grant will be divvied out over the next three years to boost Arc­turus’ self-am­pli­fy­ing mR­NA vac­cine plat­form tech on mul­ti­ple as­pects, such as pre­clin­i­cal re­search, man­u­fac­tur­ing, non-clin­i­cal safe­ty stud­ies and reg­u­la­to­ry sup­port.

The goal, per an Arc­turus state­ment, is to push a can­di­date for in­fluen­za from pre­clin­i­cal through Phase I clin­i­cal stud­ies.

CEO Joseph Payne tells End­points News that there’s a big dif­fer­ence be­tween sea­son­al in­fluen­za and pan­dem­ic in­fluen­za. One is the kind for which some­one gets their an­nu­al shot, and the oth­er is one that could mu­tate and be­come more of a threat.

“This pan­dem­ic flu is some­thing that is of sig­nif­i­cant con­cern for the Unit­ed States gov­ern­ment, be­cause it could be very lethal — far more lethal than Covid ever was,” Payne added.

The CEO not­ed that there’s in­creased en­er­gy in the US and around the world to be more proac­tive on pan­dem­ic pre­pared­ness, and right now there’s a fo­cus on pan­dem­ic flu.

In the mean­time, the next im­me­di­ate step for Arc­turus is to start pre­clin­i­cal re­search.

“We’re go­ing to be work­ing with the Unit­ed States gov­ern­ment to make sure that we’re go­ing af­ter the strains that are of most con­cern in the pan­dem­ic flu space,” Payne said.

Arc­turus’ vac­cine de­sign, ac­cord­ing to Payne, hinges on its abil­i­ty to be self-am­pli­fy­ing, es­sen­tial­ly the abil­i­ty to ex­press an anti­gen for a week or two once it en­ters a cell, in­stead of one or two days. The CEO not­ed that it would al­low the vac­cine dose to be no big­ger than five µg, a sub­stan­tial dif­fer­ence com­pared to re­cent mR­NA boost­ers at 30 or 50 µg.

On top of that, the fi­nal prod­uct that the com­pa­ny hopes to de­liv­er to BAR­DA would by lyophilized, or freeze-dried, and would al­low for po­ten­tial­ly mul­ti-year stor­age in the na­tion­al strate­gic stock­pile.

The biotech pre­vi­ous­ly had shared re­sults from a “Phase I/II/III study” in late April from its lead can­di­date, a Covid-19 mR­NA vac­cine. While it was 55% ef­fec­tive at stop­ping symp­to­matic Covid-19 in­fec­tion and 95% ef­fec­tive at pre­vent­ing se­vere dis­ease, the da­ta came out af­ter more than a third of Amer­i­cans had al­ready got­ten their first boost­er. In­vestors sent the stock price $ARCT down 15%.

Fast for­ward to to­day, where the stock price is up 2% as of Thurs­day morn­ing.

BAR­DA, for its part, has been busy in re­cent months. On top of mul­ti­ple or­ders of Bavar­i­an Nordic’s mon­key­pox vac­cine Jyn­neos, the of­fice gave out $11 mil­lion to Grand Riv­er Asep­tic Man­u­fac­tur­ing ear­li­er this week, which had agreed to pro­vide fill and fin­ish ser­vices for Bavar­i­an Nordic’s vac­cine.

Has the mo­ment fi­nal­ly ar­rived for val­ue-based health­care?

RBC Capital Markets’ Healthcare Technology Analyst, Sean Dodge, spotlights a new breed of tech-enabled providers who are rapidly transforming the way clinicians deliver healthcare, and explores the key question: can this accelerating revolution overturn the US healthcare system?

Key points

Tech-enabled healthcare providers are poised to help the US transition to value, not volume, as the basis for reward.
The move to value-based care has policy momentum, but is risky and complex for clinicians.
Outsourced tech specialists are emerging to provide the required expertise, while healthcare and tech are also converging through M&A.
Value-based care remains in its early stages, but the transition is accelerating and represents a huge addressable market.

Lat­est on ul­tra-rare dis­ease ap­proval; Pos­i­tive, if mixed, signs for Bio­gen's ALS drug; Clay Sie­gall finds a new job; and more

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Over the last four years, we’ve honored 80 women whose extraordinary accomplishments have changed the game in biopharma R&D. You can now nominate someone to be highlighted in this year’s special report. Details are here.

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FDA spells out how can­cer drug de­vel­op­ers can use one tri­al for both ac­cel­er­at­ed and full ap­provals

The FDA’s Oncology Center of Excellence has been a bright spot within the agency in terms of speeding new treatments to patients. That flexibility was on full display this morning as FDA released new draft guidance spelling out exactly how oncology drug developers can fulfill both the accelerated and full approval’s requirements with just a single randomized controlled trial.

While Congress recently passed legislation that will allow FDA to require confirmatory trials to be recruiting and ongoing prior to granting an accelerated approval, the agency is now making clear that the initial trial used to win the AA, if designed appropriately, can also serve as the trial for converting the accelerated approval into a full approval.

Clay Siegall, Morphimmune CEO

Up­dat­ed: Ex-Seagen chief Clay Sie­gall emerges as CEO of pri­vate biotech

Clay Siegall will be back in the CEO seat, taking the helm of a private startup working on targeted cancer therapies.

It’s been almost a year since Siegall resigned from Seagen, the biotech he co-founded and led for more than 20 years, in the wake of domestic violence allegations by his then-wife. His eventual successor, David Epstein, sold the company to Pfizer in a $43 billion deal unveiled last week.

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FDA ad­vi­sors unan­i­mous­ly rec­om­mend ac­cel­er­at­ed ap­proval for Bio­gen's ALS drug

A panel of outside advisors to the FDA unanimously recommended that the agency grant accelerated approval to Biogen’s ALS drug tofersen despite the drug failing the primary goal of its Phase III study, an endorsement that could pave a path forward for the treatment.

By a 9-0 vote, members of the Peripheral and Central Nervous System Drugs Advisory Committee said there was sufficient evidence that tofersen’s effect on a certain protein associated with ALS is reasonably likely to predict a benefit for patients. But panelists stopped short of advocating for a full approval, voting 3-5 against (with one abstention) and largely citing the failed pivotal study.

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Sijmen de Vries, Pharming CEO

FDA ap­proves Pharm­ing drug for ul­tra-rare im­mun­od­e­fi­cien­cy dis­ease

US regulators cleared an ultra-rare drug from Pharming Group, by way of Novartis, on Friday afternoon.

The Dutch biotech said the FDA greenlit leniolisib for an immunodeficiency disease known as activated phosphoinositide 3-kinase delta (PI3Kδ) syndrome, or APDS. People 12 years and older can receive the oral drug, to be marketed as Joenja, beginning early next month, Pharming said, five days ahead of the decision deadline set by the FDA as part of a priority review.

No­vo Nordisk oral semaglu­tide tri­al shows re­duc­tion in blood sug­ar, plus weight loss

Novo Nordisk is testing higher levels of its oral version of its GLP-1, semaglutide, and its type 2 diabetes trial results released today show reductions in blood sugar as well as weight loss.

In the Phase IIIb trial, Novo compared its oral semaglutide in 25 mg and 50 mg doses with the 14 mg version that’s currently the maximum approved dose. The trial looked at how the doses compared when added to a stable dose of one to three oral antidiabetic medicines in people with type 2 diabetes who were in need of an intensified treatment.

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Ly­me vac­cine test com­ple­tion is pushed back by a year as Pfiz­er, Val­ne­va say they'll ad­just tri­al

Valneva and Pfizer have adjusted the end date for the Phase III study of their investigational Lyme disease vaccine, pushing it back by a year after issues at a contract researcher led to thousands of US patients being dropped from the test.

In a March 20 update to, Valneva and Pfizer moved the primary completion date on the trial, called VALOR, from the end of 2024 to the end of 2025.

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Zhi Hong, Brii Biosciences CEO

Brii Bio­sciences stops man­u­fac­tur­ing Covid-19 an­ti­body com­bo, plans to with­draw EUA re­quest

Brii Biosciences said it will stop manufacturing its Covid-19 antibody combination, sold in China, and is working to withdraw its emergency use authorization request in the US, which it started in October 2021.

The Beijing and North Carolina biotech commercially launched the treatment in China last July but is now axing the work and reverting resources to other “high-priority programs,” per a Friday update. The focus now is namely hepatitis B viral infection, postpartum depression and major depressive disorders.

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