So much for bureaucracy. In what may well be the shortest high-profile FDA clinical hold in the agency’s history, Juno reported today that the FDA has lifted the halt on its lead CAR-T program, satisfied that it found the cause for three patient deaths.
Juno confounded investors last week when it reported the deaths and the surprising hold, citing the recent addition of the chemotherapy agent fludarabine to its preconditioning of patients as a likely culprit. Some experts, who had used the drug in other studies to help clear the path for a followup therapy, publicly questioned the rationale. But the agency had no problem providing a quick green light to resume the pivotal study.
The Phase II ROCKET study will now continue in adult patients with relapsed or refractory B-cell acute lymphoblastic leukemia (r/r ALL), using only cyclophosphamide in pre-conditioning patients for JCAR015.
Not immediately clear is whether Juno will be able to gain an approval in 2017, as it had hoped before the hold, putting it back in a race with rival Kite.
CAR-T therapies like this take cells from patients and reengineer them with chimeric antigen receptors so they can be reinjected into patients, multiply and swarm cancer cells. Early studies have impressed investigators with their potential for blood cancers. But Juno roiled the field just days ago with news that a new kind of neurotoxicity had killed several cancer patients in a study.
Some investigators, though, note that fludaribine has been used in many other studies, without the kind of toxicity Juno’s therapy triggered. And because it was used to help amp up the effect of the drug, some wonder if Juno’s lead therapy may not measure up to Kite’s, which now appears to be in the lead for the first, pioneering approval.
Brad Loncar, who manages an index of the top 30 immunotherapy companies on the market, noted that Juno CEO Hans Bishop indicated that he would update investors on the timeline for JCAR015 in August. Kite, meanwhile, is pushing full steam ahead on their pivotal study. And Novartis indicated back in late May that it is on track to file an approval for CTL019 in early 2017.
— j l (@bio_clouseau) July 13, 2016
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