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.

Big Phar­ma's Twit­ter ex­o­dus; Mer­ck wa­gers $1.35B on buy­out; $3.5M gene ther­a­py; 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.

As you start planning for #JPM23, we hope you will consider joining Endpoints News for our live and virtual events. For those who are celebrating Thanksgiving, we hope you are enjoying the long weekend with loved ones. And if you’re not — we’ll see you next week!

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Paul Perreault, CSL Behring CEO

CSL lands FDA ap­proval for he­mo­phil­ia B gene ther­a­py, sets $3.5M list price

The FDA has approved the world’s first gene therapy for hemophilia B, ushering into the market a treatment that’s historic in both what it promises to do and how much it will cost.

CSL will be marketing the drug, Hemgenix, at a list price of $3.5 million — which sets a new record for the most expensive single-use gene therapy in the US.

In a statement provided to Endpoints News, the Australian company noted that the current costs of treating people with moderate to severe hemophilia B can be significant over a lifetime. By some estimates, healthcare systems could spend more than $20 million per person.

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J&J's Spra­va­to pulls a PhI­II win against Sero­quel XR in treat­ment-re­sis­tant de­pres­sion

A day before Thanksgiving, J&J’s Janssen has a new cut of Phase III Spravato data to be grateful for.

The pharma giant announced on Wednesday that its nasal spray, also known as esketamine, beat extended-release quetiapine, previously sold by AstraZeneca as Seroquel XR, in treatment-resistant depression (TRD). Of 676 adults, a significantly higher number of patients on Spravato were able to achieve remission and avoid relapse after 32 weeks, according to J&J.

Elon Musk (GDA via AP Images)

Biggest drug com­pa­nies halt­ed Twit­ter ad buys af­ter Lil­ly in­sulin spoof

Almost all of the drug industry’s biggest advertisers cut their spending on Twitter to zero or near-zero over the last two weeks amid worries about impersonation of their brands by pranksters and the future of the social media company.

Among 18 of the biggest pharmaceutical advertisers in the US market, 12 cut their Twitter ad spending to nothing for the week beginning Nov. 14, according to Pathmatics, which tracks data on prescription drug ad spending as well as general corporate advertising. The list of drugmakers cutting spending to zero includes Merck, AstraZeneca, Eli Lilly, Novartis, Pfizer and others.

Rob Davis, Merck CEO

Up­dat­ed: No Seagen here: 'Do more' means a small $1.35B pur­chase of Ima­go for Mer­ck

Merck is making an acquisition, the Big Pharma announced before Monday’s opening bell. No, Seagen is not entering the fold, as had been speculated for quarters.

Folding under Merck’s wings will be Pfizer-backed Imago BioSciences. For nearly a year, Merck CEO Rob Davis has been saying the pharma giant needs to “do more” on the business development front after its 2021 $11.5 billion acquisition of Acceleron.

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Andrew Phillips, Nexo Therapeutics CEO

Scoop: Ver­sant, NEA launch new biotech helmed by ex-CEO of pro­tein de­grad­er C4 Ther­a­peu­tics

Long-time biotech venture firms Versant and New Enterprise Associates are backing a new startup run by former C4 Therapeutics chief executive Andrew Phillips.

The fledgling biotech has raised at least $30 million so far, according to paperwork filed with the SEC this week. The round could balloon to $60 million.

Phillips, who left protein degradation startup C4 in 2020 to be a managing director at Cormorant Asset Management, is running the show of the new venture as president, the SEC filing outlines. He also served as interim CEO of Cormorant-backed and Hansoh Pharmaceutical-partnered Blossom Bioscience last year.

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Isao Teshirogi, Shionogi president and CEO (Kyodo via AP Images)

Sh­ionogi's Covid an­tivi­ral lands first ap­proval in Japan's new emer­gency ap­proval path­way

Japanese regulators on Tuesday signed off on Shionogi’s homegrown antiviral for Covid-19, known as Xocova (ensitrelvir), making it the first approval under Japan’s emergency regulatory approval system.

The emergency approval, following a back-and-forth with regulators since last February, is based on a safety profile with more than 2,000 patients who have accessed the pill, and clinical symptomatic efficacy for five typical Omicron-related symptoms (primary endpoint) and antiviral efficacy (key secondary endpoint) in patients with mild to moderate SARS-CoV-2 infection, regardless of risk factors or vaccination status, and during the Omicron-dominant phase of the pandemic.

Alzheimer’s drug bites the dust; Re­struc­ture, re­struc­ture, re­struc­ture; Land­mark di­a­betes OK; 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.

Being in the news business can give one a warped sense of time — it feels like quite a while since we published some of these stories below. But next Saturday’s Endpoints Weekly will definitely be shorter, as we take off Thursday and Friday for Thanksgiving. We will still have the abbreviated edition in your inbox at the usual time.

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MIT re­searchers re­veal DNA "Paste" tech be­hind lat­est gene edit­ing start­up

MIT scientists have developed a tool that they say can insert large gene sequences where they want in the genome.

In a paper published Thursday in Nature Biotechnology, MIT fellows Omar Abudayyeh, Jonathan Gootenberg and colleagues detail a technology they call PASTE, which they say can potentially be used to insert long strands of DNA and treat genetic diseases caused by many different mutations, such as cystic fibrosis and Leber congenital amaurosis, a rare eye disorder that causes blindness.