Michael Watson (Moderna via YouTube)

As­traZeneca teams up with Im­pe­r­i­al off­shoot Vax­E­quity on an RNA 3.0 ef­fort

Robin Shat­tock

Last year, Im­pe­r­i­al Col­lege Lon­don pro­fes­sor Robin Shat­tock was giv­en close to $50 mil­lion by the UK gov­ern­ment to make an RNA-based Covid-19 vac­cine. While that fell through, the deal his com­pa­ny Vax­E­quity just signed with Big Phar­ma could be worth al­most four times that amount — with in­creased ex­po­sure to boot.

In­tro­duce As­traZeneca, which just signed a deal worth $195 mil­lion in mile­stones with Shat­tock’s com­pa­ny on a plat­form based on Shat­tock’s work on a new type of RNA as self-am­pli­fy­ing RNA, or saR­NA. As­traZeneca and Vax­E­quity will col­lab­o­rate on up to 26 drug tar­gets, which re­main undis­closed.

Ex­ec­u­tive chair­man Mike Wat­son told End­points News that one of the goals for the part­ner­ship is to val­i­date the plat­form with As­traZeneca and es­sen­tial­ly get right back in­to the clin­ic. As parts of the plat­form, which he calls “RNA 3.0,” have al­ready been test­ed clin­i­cal­ly with Shat­tock’s Phase I/II Covid-19 study con­duct­ed last year, it may not be a tra­di­tion­al 5-10 year R&D route, as Wat­son put it.

“We would hope with­in two to three years, we’ll have good pre­clin­i­cal da­ta that will con­firm that this is work­ing as well as we think it is,” he said.

The main point of saR­NA is that you could es­sen­tial­ly get a stronger ef­fect with a small­er dose, Wat­son said. A com­pa­ny us­ing saR­NA could make RNA-based ther­a­peu­tics and vac­cines us­ing a small­er dosage and still be ef­fec­tive — us­ing a dosage any­where from 1/3 to 1/10th the amount of a dose you might see in an mR­NA treat­ment. This could al­low for both de­creased price and in­creased scal­a­bil­i­ty.

The new deal comes as the in­dus­try broad­ly dives deep­er in­to RNA tech­nol­o­gy fol­low the suc­cess of the mR­NA-based Covid-19 vac­cines by Pfiz­er and BioN­Tech and Mod­er­na over the past year. GSK signed deals worth sev­er­al hun­dred mil­lion dol­lars with mR­NA com­pa­ny Cure­Vac as Cure­Vac and Sanofi com­mit­ted to build­ing out its own mR­NA cen­ter, in part with as­sets from a $2 bil­lion buy­out of Trans­late Bio.

Speak­ing of Mod­er­na, the Cam­bridge biotech tried to fo­cus on mR­NA treat­ments — not just vac­cines — just four years ago, un­til they ran in­to safe­ty prob­lems. They were, at the time, strug­gling to find a bal­ance be­tween not us­ing enough mR­NA to be ef­fec­tive with­out trig­ger­ing side ef­fects in an­i­mals.

Wat­son, who led Mod­er­na’s in­fec­tious dis­ease team for four years, said that be­cause their RNA self-am­pli­fies af­ter it en­ters the cell, you should be able to dose less of­ten and at low­er amounts than con­ven­tion­al and ac­com­plish many of the things Mod­er­na — which has kept work on mR­NA treat­ments as a sec­ond pri­or­i­ty — orig­i­nal­ly sketched out.

“If your RNA con­tin­ues to self-am­pli­fy for days and weeks, you can start with a low­er dose. The area un­der the curve is vast­ly larg­er than if you’ve got a fleet­ing­ly trans­lat­ing mod­i­fied RNA,” Wat­son said. “So, that means you can think about a whole range of ther­a­peu­tic tar­gets, whether that be an­ti­bod­ies, whether that be ther­a­peu­tic pro­teins, whether that be gene ther­a­py.”

A small firm con­sist­ing of on­ly five em­ploy­ees, Vax­E­quity plans to add 5 more em­ploy­ees with­in the next 2-3 weeks, ef­fec­tive­ly dou­bling their cur­rent em­ploy­ee base, Wat­son said. They plan to ad­vance their own in­ter­nal pipeline, while al­so work­ing on new types of RNA al­to­geth­er.

“Covid has been fan­tas­tic in pro­vid­ing this sort of … im­pe­tus to bring RNA through very, very quick­ly. We’re see­ing RNA ver­sion 1.0. This is prob­a­bly ver­sion 3.0. And it’s not self am­pli­fy­ing RNA, it’s mod­i­fied self am­pli­fy­ing RNA. What about 4.0? What about 5.0? …We’ll be look­ing at that as well,” Wat­son said.

2023 Spot­light on the Fu­ture of Drug De­vel­op­ment for Small and Mid-Sized Biotechs

In the context of today’s global economic environment, there is an increasing need to work smarter, faster and leaner across all facets of the life sciences industry.  This is particularly true for small and mid-sized biotech companies, many of which are facing declining valuations and competing for increasingly limited funding to propel their science forward.  It is important to recognize that within this framework, many of these smaller companies already find themselves resource-challenged to design and manage clinical studies themselves because they don’t have large teams or in-house experts in navigating the various aspects of the drug development journey. This can be particularly challenging for the most complex and difficult to treat diseases where no previous pathway exists and patients are urgently awaiting breakthroughs.

Val­neva's chikun­gun­ya vac­cine on track for FDA nod af­ter long-term an­ti­body da­ta

The chikungunya virus met its match in Valneva’s vaccine candidate, VLA1553, according to data from an antibody persistence trial released today.

The trial found that 99% of 363 participants retained neutralizing antibodies 12 months after a single dose of vaccine in the company’s earlier, larger Phase III trial, called VLA1553-301. The participants from the long-term study will continue to be monitored for at least five years.

Up­dat­ed: FDA re­mains silent on or­phan drug ex­clu­siv­i­ty af­ter last year's court loss

Since losing a controversial court case over orphan drug exclusivity last year, the FDA’s Office of Orphan Products Development has remained entirely silent on orphan exclusivity for any product approved since last November, leaving many sponsors in limbo on what to expect.

That silence means that for more than 70 orphan-designated indications for more than 60 products, OOPD has issued no public determination on the seven-year orphan exclusivity in the Orange Book, and no new listings of orphan exclusivity appear in OOPD’s searchable database, as highlighted recently by George O’Brien, a partner in Mayer Brown’s Washington, DC office.

Am­gen, years be­hind ri­vals, says PhI obe­si­ty drug shows dura­bil­i­ty signs

While NBC ran “The Biggest Loser” for 17 seasons, deemed toxic by critics for the reality show’s punishing exercise and diet upheavals, researchers in pharmaceutical labs have been attempting to create prescription drugs that induce weight loss — and one pharma betting it can require less frequent dosing is out with a new crop of data.

Amgen was relatively late to the game compared to its approved competitor Novo Nordisk and green light-approaching rival Eli Lilly. But early data suggested Amgen’s AMG 133 led to a 14.5% weight reduction in the first few months of dosing, buoying shares earlier this fall, and now the California pharma is out with its first batch of durability data showing that figure fell slightly to 11.2% about 150 days after the last dose. Amgen presented at the 20th World Congress on Insulin Resistance, Diabetes & Cardiovascular Disease on Saturday afternoon.

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Illustration: Assistant Editor Kathy Wong for Endpoints News

As mon­ey pours in­to dig­i­tal ther­a­peu­tics, in­sur­ance cov­er­age crawls

Talk therapy didn’t help Lily with attention deficit hyperactivity disorder, or ADHD. But a video game did.

As the 10-year-old zooms through icy waters and targets flying creatures on the snow-capped planet Frigidus, she builds attention skills, thanks to Akili Interactive Labs’ video game EndeavorRx. She’s now less anxious and scattered, allowing her to stay on a low dose of ADHD medication, according to her mom Violet Vu.

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Eli Lil­ly’s Alzheimer’s drug clears more amy­loid ear­ly than Aduhelm in first-ever head-to-head. Will it mat­ter?

Ahead of the FDA’s decision on Eli Lilly’s Alzheimer’s drug donanemab in February, the Big Pharma is dropping a first cut of data from one of the more interesting trials — but less important in a regulatory sense — at an Alzheimer’s conference in San Francisco.

In the unblinded 148-person study, Eli Lilly pitted its drug against Aduhelm, Biogen’s drug that won FDA approval but lost Medicare coverage outside of clinical trials. Notably, the study didn’t look at clinical outcomes, but rather the clearance of amyloid, a protein whose buildup is associated with Alzheimer’s disease, in the brain.

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Big week for Alzheimer’s da­ta; As­traZeneca buys cell ther­a­py start­up; Dig­i­tal ther­a­peu­tics hits a pay­er wall; 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.

You may start to notice more stories exclusively available to Premium subscribers. This week alone, paid subscribers can read our in-depth reporting on Alzheimer’s data, digital therapeutics and Allogene’s cell therapy for solid tumors, as well as scoops on Twitter ads and Catalent. With your support, we can keep growing our team and spend more time on quality work. We have both individual and company plans available — check them out to unlock the full Endpoints experience.

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Matt Gline, Roivant Sciences CEO (Photo by John Sciulli/Getty Images for GLG)

Pfiz­er and Roivant team up again for an­oth­er 'Van­t', set­ting up an­ti-in­flam­ma­to­ry show­down with Prometheus

Pfizer and Roivant are teaming up to launch a new ‘Vant’ aimed at bringing a mid-stage anti-inflammatory drug to market, the pair announced Thursday.

There’s no name for the startup yet, nor are there any employees. Thus far, the new company and Roivant can be considered “one and the same,” Roivant CEO Matt Gline tells Endpoints News. But Pfizer is so enthusiastic about the target that it elected to keep 25% of equity in the drug rather than take upfront cash from Roivant, Gline said.

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Chris Kim, Liminatus Pharma CEO

A fledg­ling biotech goes SPAC route to bankroll can­cer vac­cine, CAR-T and CD47

A relatively unknown clinical-stage biotech — backed by a Korean lighting company and focused on a cancer vaccine out of a Thomas Jefferson University lab — is headed to Nasdaq via the blank check route.

Liminatus Pharma will get about $316 million in proceeds from the SPAC combination to fund its ongoing Phase IIa study of a cancer vaccine, bring a CAR-T therapy into the clinic and prep a CD47 immune checkpoint inhibitor for human trials, the company said this week.

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