Juno's CAR-T contender comes through in another Phase I leukemia study

Illustration of CAR-T technology--Courtesy of Juno

Juno Therapeutics' ($JUNO) in-development cancer immunotherapy notched a 91% complete remission rate in a small trial on children with a form of leukemia, an incremental victory for the company as it rolls into late-stage trials.

The biotech's partners at Seattle Children's Hospital administered Juno's JCAR017 to 22 kids with acute lymphoblastic leukemia (ALL) who have failed on marrow transplants, and 20 of them have since achieved complete remission, the company said. JCAR017, a so-called CAR-T therapy, is crafted by removing a patient's T cells and rewiring them to bind to CD19, a protein expressed by cancer cells.

Children with relapsed ALL typically face survival odds of only 10% to 20%, according to Juno, and the latest JCAR017 results, presented at the American Association for Cancer Research conference this week, underscore cancer immunotherapies' potential to change the standard of care.

Oncologist Rebecca Gardner

"Some of our earliest treated patients are now a year post-therapy and are still in remission," lead investigator and Seattle Children's oncologist Dr. Rebecca Gardner said in a statement. "They've remained in remission without further chemotherapy or other treatments. This gives us hope that, eventually, we'll be able to use this therapy in patients who are newly diagnosed, reducing the need for toxic therapies and bone marrow transplants."

Juno last disclosed results from the open-label study in August, reporting a complete remission rate of 83% among the 6 patients then evaluable. Seattle Children's plans to add another 8 to 10 patients to the study over the next 12 weeks, kicking off a second stage of the trial later this year.

The data add to a growing body of evidence supporting JCAR017's potential in blood cancer, and now Juno is blueprinting trials in adult patients with B cell malignancies, including non-Hodgkin lymphoma, with hopes of getting to Phase III next year. Juno's lead CAR-T asset is JCAR015, an FDA breakthrough-designated ALL treatment, and the biotech is also running trials on the blood cancer treatment JCAR014.

The latest JCAR017 results nudged Juno's shares up a bit on Tuesday as investors moved on from a CAR-T panic that marred the value of Juno and its rival Kite Pharma ($KITE). On Sunday, CAR-T leader Novartis ($NVS) reported early results from study testing one such candidate against solid tumors, noting positive safety signals but little in the way of efficacy. Some investors read that as a sign CAR-T's promise might be limited to blood cancers, spurring debate among analysts and market watchers.

- read the results

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