CAR-T, Results

#ASH17: The BCMA-targeted CAR-T bb2121 from bluebird and Celgene takes a star turn in the spotlight

A few days ago, Celgene $CELG launched the pivotal Phase II/III study that will dictate the future of the BCMA-targeting bb2121, the CAR-T in-licensed from bluebird bio $BLUE. And if they come close to the data that bluebird has on offer at ASH this weekend, this drug will go on to blaze a big trail in the market as the developers move on to the next big focus in the field of engineered T cells.

Investigators in the Phase Ib study outlined a 94% overall response rate for bb2121, with a jaw-dropping 56% complete response rate among a small group of 18 patients in heavily pretreated active dose cohorts. Three of those 10 CRs, though, were unconfirmed at the time of the presentation in Atlanta.

The median progression-free response rate had yet to be reached. But the 6- and 9-month PFS marks were passed with 81% and 71% rates.

Nick Leschly

“This is what we all dream about,” says bluebird CEO Nick Leschly, “this kind of data for patients that are in a really, really tough position.”

Bluebird’s stock jumped 24% in pre-market trading Monday, with Celgene {$CELG) up close to 5%.

To be sure there was plenty of toxicity on the dose ranging study, with an advanced patient with a punishing tumor burden experiencing grade 4 neurotoxicity with cerebral edema and subarachnoid hemorrhage after getting the high dose: 450 x 106 CAR+ T cells. And there were a number of cases of cytokine release syndrome, a hallmark of these first generation CAR-Ts going through the clinic.

Celgene will now take 150 to 300 million cell doses into the landmark study with about 80 patients, looking to see largely if it can replicate anything similar. If they are successful, the partners can look forward to a blockbuster market for one of the most widely anticipated late-stage studies in oncology.

David Davidson

The data underscore the potential of BCMA as a target in the field, which is seeing the first pioneers hit the market by hitting CD19 with major effect. The data will certainly brighten the stage for bluebird and Celgene, the clear leaders in the field, as a bevy of rivals follow up in hopes of catching or leapfrogging the frontrunners.

Juno, making a CAR-T comeback with JCAR017, just bagged a gamma secretase inhibitor from Eli Lilly for its BMCA therapy, adding IP agreements with the Fred Hutch and OncoTracker on their combo approach.

But bluebird isn’t sitting still. It’s also snapping up new tech to keep its cutting edge, like the CAR-engineered gamma delta T cell pact it struck with Scotland’s TC BioPharm a few days ago.

“We’ve got all cylinders firing,” says David Davidson, the bluebird chief medical officer.

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Research Scientist - Immunology
Recursion Pharmaceuticals Salt Lake City, UT
Director of Operations
Atlas Venture Cambridge, MA

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