Juno Therapeutics ($JUNO) and Kite Pharma ($KITE) have reiterated their ambition to bring CAR-T therapies to market in 2017 following the publication of fresh clinical trial data at the ASCO Annual Meeting 2016. The stage is now nearly set for a flurry of regulatory filings and decisions over the next year or so as Juno, Kite and Novartis ($NVS) all race to get their CAR-T therapies in front of FDA.
Executives at both Juno and Kite confirmed their intent to win approval for their products next year in interviews with Reuters. Kite is still aiming to have a submission with FDA before the year is over, a timeline that would set it up to win approval for KTE-C19 next year. And with Juno thinking the data from its ongoing mid-stage trial can support accelerated approval, it also has set its sights on winning a regulatory nod in 2017. Novartis, the third horse in the CAR-T race, has similar ambitions.
The comments from Juno and Kite follow the publication of clinical trial data updates at ASCO. Juno shared an update on a trial of JCAR15 in patients with acute lymphoblastic leukemia, a hard-to-treat population that is responding well to the therapy. Among the 25 patients with morphological disease at the time of infusion, JCAR15 was associated with a complete response rate of 75%. The rate shot up above 90% in the cohort who had minimal disease at the start of the study.
Kite had its own data to trumpet. In a NIH study that gave low-dose chemotherapy and Kite’s CAR-T to 19 patients with various subtypes of diffuse large B-cell lymphoma (DLBCL), 8 participants had a complete response and a further 5 experienced a partial response. The full dataset is expected to enable Kite to go to FDA later this year.
If Juno and Kite succeed in winning approval for their products next year, both companies will face a whole new set of challenges. Doubts about the ability of either firm to turn the complicated CAR-T production process into a cost-effective, commercial-scale operation remain. And the broader use of the therapies could uncover results that tip the risk-benefit balance.
Juno CEO Hans Bishop, who has firsthand experience of how a promising pipeline product can turn into a commercial calamity from his time as COO of Dendreon, is confident the therapeutic profile of JCAR15 will continue to look appealing as more data are generated.
“These are patients that are relapsed and refractory. They are going to die of their disease,” he told Reuters. “We can get 90 to 100% of them into remission, and a meaningful percentage of them have durable remission.”
While such efficacy in a hard-to-treat population will mean many patients are willing to endure the risk of severe side effects, Juno is working on a way to identify people who are likely to respond badly to its drug prior to starting treatment. Bishop teased the side effect-predicting assay in his interview with Reuters, but is yet to disclose details of the product.