Nan­jing Leg­end wows AS­CO re­searchers with ear­ly da­ta on a BC­MA-tar­get­ed CAR-T con­tender

Chi­na’s Nan­jing Leg­end Biotech is a vir­tu­al un­known in the CAR-T field. But it gained mar­quee sta­tus at AS­CO to­day with stel­lar ear­ly mul­ti­ple myelo­ma da­ta on a drug tar­get­ed at B-cell mat­u­ra­tion pro­tein, or BC­MA.

Among 35 re­lapsed, drug-re­sis­tant pa­tients, 33 — 94% — demon­strat­ed clin­i­cal re­mis­sion rang­ing from a com­plete to a par­tial re­sponse. There was a 100% ob­jec­tive re­sponse rate, ac­cord­ing to in­ves­ti­ga­tors.

The in­ves­ti­ga­tors al­so flagged some promis­ing signs of dura­bil­i­ty.

Of the first 19 pa­tients to reach the four-month mark spec­i­fied in the tri­al, a re­mark­able 14 achieved a “strin­gent” com­plete re­sponse (sCR), which is as­so­ci­at­ed with longer sur­vival times. And 5 pa­tients who hit the one-year mark were still in the sCR stage.

Xi­ao­hu (Frank) Fan

Look­ing past the CD19 bio­mark­er, BC­MA of­fers one of the most promis­ing tar­gets in CAR-T. And this is, to date, the largest pa­tient group yet re­port­ed on. There are, how­ev­er, some sig­nif­i­cant caveats to point out. Its BC­MA ri­val blue­bird bio points out that these pa­tients ev­i­dent­ly weren’t as sick as the ones they treat­ed, with few­er ear­li­er drug fail­ures.

But you can be sure that there will be plen­ty of an­a­lysts pour­ing over the da­ta to­day to start cal­cu­lat­ing how the lead­ers all line up.

The same safe­ty is­sues that have plagued in­ves­ti­ga­tors for years now were ev­i­dent in the study. 85% of the pa­tients in the study ex­pe­ri­enced cy­tokine re­lease syn­drome, with two ex­pe­ri­enc­ing a grade 3 case that re­quired Actem­ra to tamp down on the in­flam­ma­to­ry re­sponse. All those pa­tients re­cov­ered.

And no mat­ter who is do­ing the work, CAR-T ap­pears to be more promis­ing than ever for this group of pa­tients.

“While it’s still ear­ly, these da­ta are a strong sign that CAR T-cell ther­a­py can send mul­ti­ple myelo­ma in­to re­mis­sion,” said AS­CO Ex­pert Michael S. Sabel in a state­ment. “It’s rare to see such high re­sponse rates, es­pe­cial­ly for a hard-to-treat can­cer. This serves as proof that im­munother­a­py and pre­ci­sion med­i­cine re­search pays off. We hope that fu­ture re­search builds on this suc­cess in mul­ti­ple myelo­ma and oth­er can­cers.”


Pic­tured: Nan­jing, Chi­na on April 27th, 2017 / Shut­ter­stock

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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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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'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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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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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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