FDA clears Juno to resume CAR-T trial

Juno Therapeutics ($JUNO) was up by about 25% in after-hours trading on news that the FDA has already given it permission to resume the Phase II trial for its JCAR015 that it halted last week. The biotech will continue the trial, as it had hoped, with a modified preconditioning regimen that it expects will prevent future patient deaths.

The biotech is convinced that exclusion of chemotherapy drug fludarabine from the preconditioning regimen will obviate the problem that resulted in at least two deaths during a Phase II trial of its JCAR015 to treat adult patients with relapsed or refractory B cell acute lymphoblastic leukemia (r/r ALL).

And, evidently, the FDA agrees. The agency had up to 30 days to respond to Juno regarding its request to resume the ROCKET trial without fludarabine; the company had said that it would submit the requested information this week.

“Adding fludarabine to the preconditioning on the ROCKET trial, we have seen an increase in the incidences of severe neurotoxicity, which has unfortunately included two patient deaths that occurred last week from cerebral edema that appeared to be treatment-related,” summed up Juno President and CEO Hans Bishop on a call about the FDA hold.

The FDA’s prompt, go-ahead action suggests that the agency isn’t particularly concerned that this could be a safety issue beyond the combo with fludarabine. The trial will continue, as it was previously, with a preconditioning regimen consisting solely of another chemotherapy drug, cyclophosphamide.

Obviously, this is good news--at least for now--for the many companies working on chimeric antigen receptor T cell (CAR-T) treatments. Juno’s CAR-T technology takes the patient’s own T cells and genetically engineers them to recognize and combat cancer cells.

JCAR015 is Juno’s lead candidate, and the company had been hoping for an FDA approval next year. Last week, it removed that timeline. But with the quick trial resumption, that could be back on the table. It said it will offer an update on the regulatory expectations for JCAR015 on its next earnings call in August.

Investor confidence hasn’t returned completely, though, despite the rapid share price rebound. Prior to the clinical trial hold, Juno’s share price hovered around $40; now it’s closer to $35.

- here is the release

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