Emma Walmsley (GSK via investor day livestream)

Mul­ti­va­lent mR­NA vac­cines, I/O 3.0, long-act­ing HIV drugs: The $46B game is on for the new Glax­o­SmithK­line

To un­der­stand the new Glax­o­SmithK­line that Em­ma Walm­s­ley is promis­ing to ush­er in, one must see through the old GSK.

“We know this has been a com­pa­ny that has peren­ni­al­ly dis­ap­point­ed when you look at the first half of the last decade,” the CEO said in one of her fi­nal re­marks at the long-an­tic­i­pat­ed in­vestor day.

But af­ter four years of strate­gic trans­for­ma­tion and in­vest­ment, her top team is ready to take the wraps off a shiny new ver­sion of the Big Phar­ma that, among oth­er things, will strive to de­liv­er £33 bil­lion ($46 bil­lion) in an­nu­al sales by 2031. It will start with a planned de­merg­er of the con­sumer health busi­ness in­to a sep­a­rate com­pa­ny in the mid­dle of next year, while the new GSK re­tains a 20% stake as a “short-term fi­nan­cial in­vest­ment.”

Hal Bar­ron

The ma­jor­i­ty of the rev­enue should come from cur­rent late-stage projects, hand­picked by R&D chief Hal Bar­ron in con­sul­ta­tion with chief com­mer­cial of­fi­cer Luke Miels.

The end goal? A “step change” in per­for­mance to re­vi­tal­ize the busi­ness around new vac­cines and spe­cial­ty med­i­cines — two ar­eas that are now in­ter­twined as pre­ven­tion and treat­ment con­verge.

For any­one who’s been fol­low­ing the R&D roadmap that Bar­ron laid out in 2018 af­ter tak­ing over, it can sound like more of the same. Sure, the sep­a­ra­tion of the con­sumer health busi­ness might be the most sig­nif­i­cant change for GSK in two decades; yet the com­pa­ny is stub­born­ly con­sis­tent about what it will take to thrive in the fu­ture. It’s still about the sci­ence of the im­mune sys­tem, the un­der­stand­ing of func­tion­al ge­nomics, some ma­chine learn­ing or ar­ti­fi­cial in­tel­li­gence, as well as a healthy di­et of bolt-on and li­cens­ing deals to en­hance all those ar­eas.

The dif­fer­ence could be that the clock is tick­ing. Amid threats of an ac­tivist at­tack and calls for greater re­form, Walm­s­ley, Bar­ron and oth­ers are un­der se­ri­ous pres­sure to de­liv­er, and every blun­der or set­back could be mag­ni­fied.

But if they suc­ceed, the new GSK will make a mark and cre­ate last­ing fran­chis­es across in­fec­tious dis­ease, HIV, on­col­o­gy and im­munol­o­gy. And they’ve brought in fresh blood to ex­e­cute: Ac­cord­ing to Walm­s­ley, “85% of our top 125 lead­ers are new in roles since 2017, in­clud­ing 30% re­cruit­ed ex­ter­nal­ly.”

Now that they are wrap­ping up a dras­tic re­struc­tur­ing that led to mas­sive lay­offs, she added, there are no near-term plans for an­oth­er round. It’s show­time.

Be­yond Covid-19 vac­cines

GSK may have fall­en be­hind on the glob­al hunt for a Covid-19 vac­cine, but it’s very much look­ing to get in the next-gen game.

Roger Con­nor

Like ri­vals at Pfiz­er (and cur­rent part­ners at Sanofi), GSK wants to be at the fore­front of mR­NA, ac­cord­ing to Roger Con­nor, pres­i­dent of glob­al vac­cines — which is why they’ve re­cruit­ed more than 200 sci­en­tists to fo­cus on this area and teamed up with Ger­many’s Cure­Vac to cre­ate mul­ti­va­lent vac­cines not just for Covid-19 but al­so for in­fluen­za.

De­spite the dis­ap­point­ing re­sults around a Covid-19 vac­cine made from the first gen­er­a­tion of tech­nol­o­gy, the phar­ma clear­ly sees a fu­ture in the sec­ond-gen­er­a­tion ver­sion of the tech.

Said Bar­ron:

I think the key thing to re­mem­ber at least our view is that the fu­ture for mR­NA vac­cines are go­ing to be mul­ti­va­lent. Mul­ti­va­len­cy re­quires a low­er dose be­cause you can’t give a cer­tain amount of mR­NA with­out cre­at­ing re­ac­to­genic­i­ty. So if you’re go­ing to use mul­ti­va­lent vac­cines, you got­ta get the num­ber of mi­cro­grams per va­lence down. What Cure­Vac’s sec­ond-gen­er­a­tion, I think, is telling us is that when you op­ti­mize the un­trans­lat­ed re­gions on each side, the 5’, 3’, you get more ef­fi­cient trans­la­tion, more pro­tein pro­duced.

While Shin­grix cur­rent­ly head­lines the port­fo­lio, GSK is al­so hop­ing its vac­cine for RSV — cre­at­ed from the same plat­form — would do just as well, with broad reach to both old­er adults and preg­nant women (to pre­vent in­fec­tions for their new­borns). As with menin­gi­tis, Con­nor sug­gest­ed that these are ex­am­ples of pathogens where old­er tech­nolo­gies may still have a role to play over mR­NA.

HIV, can­cer, and some wild cards

One thing that the new GSK will like­ly do is to keep drop­ping hints about its in­ter­ests through deals.

Vi­iV Health­care, the HIV-fo­cused sub­sidiary, made its goal clear as it inked a deal with Halozyme ear­li­er this week: to cre­ate ul­tra long-act­ing reg­i­mens that will com­plete­ly change how peo­ple live with the virus.

The first drug to do that is cabote­gravir, the in­te­grase in­hibitor that Vi­iV hopes will be a back­bone for an­ti­retro­vi­ral ther­a­py for the next 10 years the same way that do­lute­gravir — which is los­ing patent pro­tec­tion around 2028 — has.

In can­cer, the most re­cent ex­am­ple came in a pact with iTeos, where GSK paid $625 mil­lion up­front to grab a TIG­IT drug. Even though they’ve en­tered the race lat­er than oth­ers, Bar­ron reck­ons their own ge­nomics da­ta and an­a­lyt­ics point them to things oth­ers can’t see. If TIG­IT plus PD-1 is I/O 2.0, they might be able to add a third ther­a­py tar­get­ing the CD226 ax­is to reach I/O 3.0.

“There’s first to ap­proval, but there’s al­so first in dis­ease,” he said. “We have a re­al­ly ro­bust set of stud­ies that we’re an­tic­i­pat­ing do­ing where we think while we may not be first to mar­ket in lung, where Roche and Mer­ck are prob­a­bly both ahead, we have some par­tic­u­lar­ly in­ter­est­ing in­sights about oth­er dis­eases that we might want to pur­sue, and we can use some da­ta from ex­ter­nal sources to un­gate those tri­als.”

Then there are the in­ter­nal projects you may have to squint hard­er to find, such as Ze­ju­la — which Bar­ron touts as a best-in-class PARP in­hibitor — or the mul­ti­ple myelo­ma drug Blenrep or otil­imab for rheuma­toid arthri­tis or a new an­tibi­ot­ic dubbed gepoti­dacin or dapro­du­s­tat for ane­mia as­so­ci­at­ed with chron­ic kid­ney dis­ease.

As for the lega­cy meds that don’t fit in­to the four new fo­cus ar­eas, mov­ing for­ward GSK will lump them in­to a gen­er­al med­i­cines group that will hap­pi­ly di­vest or part­ner out non-pri­or­i­ty drugs to in­vest in the vac­cines and spe­cial­ty can­di­dates.

“(H)aving the op­por­tu­ni­ty to al­lo­cate cap­i­tal across both phar­ma and vac­cines al­lows us to put mon­ey to where the great projects are,” Bar­ron not­ed.

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.

Alaa Halawaa, executive director at Mubadala’s US venture group

The ven­ture crew at Mubadala are up­ping their biotech cre­ation game, tak­ing care­ful aim at a new fron­tier in drug de­vel­op­ment

It started with a cup of coffee and a slow burning desire to go early and long in the biotech creation business.

Wrapping up a 15-year discovery stint at Genentech back in the summer of 2021, Rami Hannoush was treated to a caffeine-fueled review of the latest work UCSF’s Jim Wells had been doing on protein degradation — one of the hottest fields in drug development.

“Jim and I have known each other for the past 15 years through Genentech collaborations. We met over coffee, and he was telling me about this concept of the company that he was thinking of,” says Hannoush. “And I got immediately intrigued by it because I knew that this could open up a big space in terms of adding a new modality in drug discovery that is desperately needed in pharma.”

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'Band­ing to­geth­er': 50 fe­male biotech ex­ec­u­tives lay out plans for board di­ver­si­ty, new com­pa­nies and men­tor­ing founders

Earlier this month, during the Silicon Valley Bank meltdown, Angie You recalled the speed with which female biotech CEOs were helping each other connect with bankers, get their wires through and assuage concerns during a financial implosion.

This past weekend, 50 of about 125 women who are part of that Slack group and a broader coalition self-dubbed the Biotech Sisterhood met in person in Cancun for the second rendition of an annual summit connecting female biotech CEOs. The attendance list doubled that of the inaugural gathering in Arizona 12 months ago.

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No­var­tis touts sev­en years of dura­bil­i­ty da­ta for Zol­gens­ma

The same day that Roche touted positive durability and safety data for its spinal muscular atrophy drug Evrysdi, Novartis also made a splash with its multi-million dollar gene therapy for the disease.

Novartis rolled out interim data from two long-term follow-up studies Monday at the 2023 Muscular Dystrophy Association (MDA) Clinical and Scientific Conference. In the first study, LT-001, all children in the trial that were treated after showing symptoms of SMA “maintained all previously achieved motor milestones” up to 7.5 years after being dosed. The average time since Zolgensma was given was 6.86 years.

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Rohan Palekar, 89bio CEO

89bio’s PhII da­ta add to quick suc­ces­sion of NASH read­outs as field seeks turn­around

89bio said its drug was better than placebo at lessening fibrosis without worsening nonalcoholic steatohepatitis, or NASH, in two of three dose groups.

The San Francisco biotech said it thinks the Phase IIb data pave the way for a potential Phase III, following in the footsteps of another biotech in its drug class, Akero Therapeutics. To fund a late-stage study, CEO Rohan Palekar told Endpoints News 89bio “would need to raise additional capital,” with the company having about $188 million at the end of last year.

Flare Therapeutics biochemists Yong Li (L) and Valerie Vivat

A $123M Flare will get Third Rock on­col­o­gy biotech in­to the clin­ic this year

Flare Therapeutics will start its first human trial this year with an investigational urothelial cancer drug after pulling together a $123 million Series B from Big Pharmas, VCs and its incubator, Third Rock Ventures.

Launched in 2021 on the idea that a biotech could finally succeed at drugging the much-sought-after but stubborn transcription factor, Flare Therapeutics said Wednesday it is now primed for the clinic after closing its large financing haul earlier this year. The raise is a relatively stark figure in a tough startup financing environment but further buoys the upbeat signals coming out of other Third Rock biotechs in recent weeks, including the $200 million CARGO Therapeutics and $100 million Rapport Therapeutics rounds.

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Francesco Marincola, newly-appointed Sonata Therapeutics CSO

Kite's head of re­search leaves for Flag­ship start­up Sonata

Another leader is departing Kite Pharma, and will to spend the “last part” of his career exploring how cancer evades the immune system.

Kite’s senior VP and global head of cell therapy research Francesco Marincola left the Gilead CAR-T unit last week for Sonata Therapeutics. Flagship last May unveiled the startup, which was pieced together from two fledgling biotechs Inzen and Cygnal Therapeutics. As CSO, Marincola will lead Sonata’s push to reprogram cancer cells to make them more immunogenic.

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FDA in­di­cates will­ing­ness to ap­prove Bio­gen ALS drug de­spite failed PhI­II study

Ahead of Wednesday’s advisory committee hearing to discuss Biogen’s ALS drug tofersen, the FDA appeared open to approving the drug, newly released briefing documents show.

Citing the need for flexibility in a devastating disease like ALS, regulators signaled a willingness to consider greenlighting tofersen based on its effect on a certain protein associated with ALS despite a failed pivotal trial. The documents come after regulatory flexibility was part of the same rationale the agency expressed when approving an ALS drug last September from Amylyx Pharmaceuticals, indicating the FDA’s openness to approving new treatments for the disease.

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NIH re­jects an­oth­er at­tempt to 'march-in' on Astel­las' prostate can­cer drug over ex­ces­sive price

The National Institutes of Health has again declined to use so-called “march-in” rights to lower the price of Astellas and Pfizer’s prostate cancer drug Xtandi despite being invented at UCLA with grants from the US Army and NIH.

“Given the remaining patent life and the lengthy administrative process involved for a march-in proceeding, NIH does not believe that use of the march-in authority would be an effective means of lowering the price of the drug,” NIH told prostate cancer patients Robert Sachs and Clare Love, in a letter shared with Endpoints News. The institutes’ analyses found Xtandi “to be widely available to the public,” an indication that there was not a pressing need for the US to act.