CAR-T

In wake of a CAR-T restructuring, Novartis says it’s ready to hunt a pioneering OK for lead program

Bruno Strigini, CEO Novartis Oncology

Bruno Strigini, CEO Novartis Oncology

Novartis is still in the race to win the world’s first approval for a pioneering CAR-T treatment.

The pharma giant $NVS pulled back the veil from their Phase II study of their CAR-T CTL019, registering an impressive 82% complete remission rate among the 50 children and young adults with B-cell acute lymphoblastic leukemia. And with no deaths, though significant evidence of toxicity, Novartis says it will be ready to file for an approval early on in a looming 2017.

Forty-one of 50 patients achieved complete remission or complete remission with incomplete blood count recovery three months after an infusion of CTL019, which reengineers patients’ T cells into a cancer therapy.

Close to half of the patients experienced a grade 3 or 4 case of cytokine release syndrome, not uncommon in the field. And 15% percent of patients experienced grade 3 neurological and psychiatric events including encephalopathy and delirium. But there were no grade 4 events and no one died.

That’s a solid enough benefit/safety profile to take to regulators as Novartis hunts up an accelerated approval.

Novartis managed to stun practically everyone in the field when it abruptly disbanded its cell and gene therapy unit three months ago, laying off 120 and integrating another 280 back into its regular R&D ranks as CAR-T chief Usman ‘Oz’ Azam hit the exit. That was widely regarded as a serious setback that could affect future programs, even if more advanced efforts like this one at the University of Pennsylvania stayed on track.

Juno has been thrown into utter disarray with multiple deaths derailing their lead effort for JCAR015. And Kite recently noted that it would need a little more time to complete a rolling submission for their top therapy, which should now be completed in Q1.

“This first-of-its-kind trial represents exciting progress toward our goal of helping children and young adults with relapsed or refractory B-cell ALL, a patient population with an urgent need for new treatment options,” said Bruno Strigini, CEO, Novartis Oncology. “We are committed to advancing CTL019 and look forward to working closely with the FDA and EMA in the coming months.”


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RAPS Regulatory Convergence 2017