Samit Hirawat. Bristol-Myers Squibb

Bris­tol-My­ers is mak­ing a bee-line to the FDA with pos­i­tive liso-cel da­ta — but is it too late in the CAR-T game?

Bris­tol-My­ers Squibb came to ASH this past week­end with a va­ri­ety of mes­sages on the new can­cer drugs they had ac­quired in the big Cel­gene buy­out, in­clud­ing liso-cel, the lead CAR-T pro­gram picked up in the $9 bil­lion Juno ac­qui­si­tion. And one of the most im­por­tant was that they had the piv­otal ef­fi­ca­cy and safe­ty da­ta need­ed to snag an ap­proval from the FDA next year, with the BLA on track for a fil­ing this month.

Pro­vid­ed there are no sna­fus or even mod­est stum­bles now, they should get an OK with­in the 2020 time­line lined out in their $9 CVR for Cel­gene — the first in a tri­fec­ta of ap­provals re­quired for a pay­out.

Whether they can go on to make it in­to a vi­able com­mer­cial ther­a­py, though, is a whole oth­er thing.

At first glance, there isn’t any­thing about the safe­ty and ef­fi­ca­cy da­ta that would force a CRL or de­lay. In a large tri­al of pa­tients with re­lapsed/re­frac­to­ry large B-cell lym­phomas in­ves­ti­ga­tors tracked an out­stand­ing 73% re­sponse rate and 53% com­plete re­sponse rate in heav­i­ly pre­treat­ed pa­tients with few op­tions.

That’s in line with Yescar­ta from Gilead’s Kite, which snagged an ap­proval more than 2 years ago, just af­ter No­var­tis’ Kym­ri­ah came through.

Then there’s safe­ty. To be sure, the drug has safe­ty is­sues. There were 4 pa­tients in the study who died af­ter treat­ment. Sev­er­al oth­ers died for un­re­lat­ed is­sues. But…with on­ly a 2% rate of cy­tokine re­lease syn­drome, Bris­tol-My­ers has a shot at a dif­fer­en­ti­at­ed safe­ty pro­file.

“There is a po­ten­tial these pa­tients can be treat­ed on an out­pa­tient ba­sis,” says Samit Hi­rawat, the chief med­ical of­fi­cer at Bris­tol-My­ers. He not­ed that 26% of pa­tients were nev­er ad­mit­ted, while 76% were ad­mit­ted 4 days or lat­er af­ter ther­a­py. The key is to treat them at a hos­pi­tal with the in­fra­struc­ture to pro­vide care 24/7, so a pa­tient can be ad­mit­ted at any time lat­er if need­ed.

How that will fly with pay­ers af­ter ri­vals have been on the mar­ket for about 3 years, with new ev­i­dence that ear­li­er use of steroids can dra­mat­i­cal­ly re­duce CRS and con­sid­er­able dura­bil­i­ty of re­sponse, will have to be seen.

But time is not on Bris­tol-My­ers’ side. Liso-cel is the long de­layed fol­lowup to the dis­as­trous JCAR015, which killed a num­ber of pa­tients. Their set­back threw them years off sched­ule. And ri­vals are ad­vanc­ing off-the-shelf al­ter­na­tives or oth­er new ap­proach­es that could knock the pi­o­neers com­plete­ly out of the game. In the mean­time, Bris­tol-My­ers is gath­er­ing its own dura­bil­i­ty da­ta and will grad­u­al­ly see if their mix of CD4 and CD8 cells can do bet­ter.

The main in­ter­est now is in the time­line around the ap­proval. Hi­rawat says they’ll ask for pri­or­i­ty re­view, and there’s every rea­son to be­lieve that reg­u­la­tors will move swift­ly — bar­ring a nasty sur­prise.

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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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.

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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Roche and Lil­ly team up to de­vel­op blood test to de­tect ear­ly signs of Alzheimer's

Eli Lilly is teaming up with Roche to help develop a blood test to detect early signs of Alzheimer’s disease and determine whether a patient should go for further confirmatory testing.

Roche’s Elecsys Amyloid Plasma Panel (EAPP) measures pTau 181 protein assay and APOE E4 assay in human blood plasma – elevations in pTau 181 are present in the early stages of Alzheimer’s, while the presence of APO E4 is the most common genetic risk factor for the disease.

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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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Vipin Garg, Altimmune CEO

Al­tim­mune’s shares halved af­ter in­ter­im look at PhII weight loss drug da­ta

Altimmune’s attempt to catch up to Novo Nordisk and Eli Lilly’s GLP-1 drugs hit an investor snag Tuesday after the biotech shared interim Phase II weight loss data.

The Maryland biotech’s pemvidutide is a GLP-1/glucagon dual receptor agonist meant to activate GLP-1 receptors to squash appetite and glucagon to ramp up energy use. The 2.4 mg dose showed a placebo-adjusted weight loss of 9.7% at week 24 of 48, which Jefferies analysts said would be comparable to Novo Nordisk’s semaglutide (Wegovy) and Eli Lilly’s tirzepatide (Mounjaro).

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Josep Bassaganya-Riera, NImmune Biopharma

Ex­clu­sive: Af­ter get­ting his drug back, Lan­dos founder as­sem­bles new start­up for the big PhI­II test

By the time Josep Bassaganya-Riera stepped down as founding CEO of Landos Biopharma in 2021, the company had racked up Phase II data for its top autoimmune program, completed what he called a positive end-of-Phase-II meeting with the FDA and plans to launch pivotal Phase III trials.

Since then, though, the new leaders at Landos have reshuffled their plans for the drug, omilancor, first announcing they will run a Phase IIb ahead of a Phase III and eventually shelving it altogether.

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