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.

At the In­flec­tion Point for the Next Gen­er­a­tion of Can­cer Im­munother­a­py

While oncology researchers have long pursued the potential of cellular immunotherapies for the treatment of cancer, it was unclear whether these therapies would ever reach patients due to the complexity of manufacturing and costs of development. Fortunately, the recent successful development and regulatory approval of chimeric antigen receptor-engineered T (CAR-T) cells have demonstrated the significant benefit of these therapies to patients.

All about Omi­cron; We need more Covid an­tivi­rals; GSK snags Pfiz­er’s vac­cine ex­ec; Janet Wood­cock’s fu­ture at FDA; 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.

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Usama Malik

Ex-Im­munomedics CFO charged with in­sid­er trad­ing, faces up to 20 years in prison af­ter al­leged­ly tip­ping off girl­friend and rel­a­tives of a PhI­II suc­cess

The former CFO of Immunomedics, who helped steer the company to its $21 billion buyout by Gilead last year, has been charged with insider trading, the Department of Justice announced Thursday.

Usama Malik tipped off his then-girlfriend and four others that a Phase III study for Trodelvy would be stopped early four days before Immunomedics publicly announced the result in April 2020, DoJ alleged in its complaint. The individuals then purchased Immunomedics shares, selling them after the news broke and Immunomedics’ stock price doubled.

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Merck's new antiviral molnupiravir (Quality Stock Arts / Shutterstock)

As Omi­cron spread looms, oral an­tivi­rals ap­pear to be one of the best de­fens­es — now we just need more

After South African scientists reported a new Covid-19 variant — dubbed Omicron by the WHO — scientists became concerned about how effective vaccines and monoclonal antibodies might be against it, which has more than 30 mutations in the spike protein.

“I think it is super worrisome,” Dartmouth professor and Adagio co-founder and CEO Tillman Gerngross told Endpoints News this weekend. Moderna CEO Stéphane Bancel echoed similar concerns, telling the Financial Times that experts warned him, “This is not going to be good.”

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Pfiz­er, Am­gen and Janssen seek fur­ther clar­i­ty on FDA's new ben­e­fit-risk guid­ance

Three top biopharma companies are seeking more details from the FDA on how the agency conducts its benefit-risk assessments for new drugs and biologics.

While Pfizer, Amgen and Janssen praised the agency for further spelling out its thinking on the subject in a new draft guidance, including a discussion of patient experience data as part of the assessment, the companies said the FDA could’ve included more specifics in the 20-page draft document.

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Vas Narasimhan, Novartis CEO (Thibault Camus/Pool via AP Images)

With gener­ic com­pe­ti­tion heat­ing up, Vas Narasimhan out­lines No­var­tis' growth plans at R&D day

Thursday marks Novartis’ annual R&D day, and with it comes CEO Vas Narasimhan’s attempt to spotlight the company’s pipeline strategy and emerging stars.

The biggest question entering Thursday’s presentation dealt with how the big biopharma will make up revenues from upcoming generic competition — Novartis says within the next five years, generics will eat away roughly $9 billion in sales. To offset this, Narasimhan outlined a strategy for 4% growth or higher until 2026, focusing on six key medicines he believes will see multibillion dollar profits during this time.

Watch out, Roche: No­var­tis inks $1.5B deal to chase down promi­nent Parkin­son’s tar­get

Novartis is plopping down $150 million in cash to pick up an experimental Parkinson’s drug and grab an option to another, a move that puts it on an increasingly popular path in the field’s search for disease-modifying therapies.

Belgium’s UCB is its partner of choice, supplying two small molecule alpha-synuclein misfolding inhibitors in a deal that can add up to nearly $1.5 billion.

Out of the pair, UCB0599 is already in Phase II trials, making Novartis confident enough to pull the trigger on co-development and commercialization, including to foot half of the R&D bill. The pharma giant will make a decision on UCB7853 once UCB wraps the ongoing Phase I program.

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Glax­o­SmithK­line, Ox­ford un­veil new part­ner­ship pit­ting buzzy R&D ad­vances against neu­ro­log­i­cal dis­ease

When GlaxoSmithKline trumpeted its return to neuroscience with a $700 million upfront deal with Alector this summer, it touted its early investments in functional genomics as a key guidepost for that deal. Now, the drug giant has partnered up with Oxford to hopefully add jet fuel to its hunt for breakthroughs in the brain.

GSK and Oxford have kickstarted a five-year collaboration aimed at spurring R&D breakthroughs across a range of hard-to-treat diseases like Alzheimer’s and Parkinson’s through the use of genomic testing and machine learning, the partners said Wednesday.

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In­cor­po­rat­ing Ex­ter­nal Da­ta in­to Clin­i­cal Tri­als: Com­par­ing Dig­i­tal Twins to Ex­ter­nal Con­trol Arms

Most drug development professionals are familiar with the nerve-racking wait for the read-out of a large trial. If it’s negative, is the investigational therapy ineffective? Or could the failure result from an unforeseen flaw in the design or execution of the protocol, rather than a lack of efficacy? The team could spend weeks analyzing data, but a definitive answer may be elusive due to insufficient power for such analyses in the already completed trial. These problems are only made worse if the trial had lower enrollment, or higher dropout than expected due to an unanticipated event like COVID-19. And if a trial is negative, the next one is likely to be larger and more costly — if it happens at all.