- Juno Therapeutics continues to view 2018 as a pivotal year for its pipeline of CAR-T products, reiterating to analysts on an earnings call Wednesday that approvals for JCAR015 and JCAR017 in initial indications could come as early as that year.
- While Juno's original plans for JCAR015 were derailed by several patient deaths this summer, the cell therapy company said the trial had returned to a "normal pace" after progressing through treatment limitations imposed in response to the deaths.
- But CEO Hans Bishop chose to focus more on the progress of JCAR017, pointing to encouraging response rates and low nuerotoxicity seen in a Phase 1 trial. Juno plans to give greater detail on the results and its plans for JCAR017 at the upcoming annual meeting of the American Society of Hematology(ASH).
While JCAR015 remains Juno's most advanced program, the company clearly has high expectations for JCAR017.
"Our goal is to be on the market in its first indication for NHL [non-Hodgkin lymphoma] as early as 2018 with approvals projected in three additional indications, pediatric and adult ALL [acute lympoblastic leukemia] and CLL [chronic lymphocytic leukemia], as early as 2019," said Bishop on a call with investors.
As of August 1, 14 patients with non-Hodgkin lymphoma had been treated with JCAR017, according to an ASH abstract posted earlier this month. Eight of the 11 patients (73%) with diffuse large b-cell lymphoma (DLBCL) who had been assessed post-treatment saw a complete response, although only 29 days had elapsed following treatment at the time of assessment. Durability of response remains a concern with CAR-T treatments, as some patients relapse from an initial positive reaction.
Notably, no patients experienced severe cytokine release syndrome (CRS) — a serious side effect associated with CAR-T. Three patients (21%) had low-grade CRS and two (14%) experienced dose-limiting neurotoxicity.
"We continue to be very excited by the data, not just the encouraging response rates we are seeing in that Phase 1 trial, but also the encouraging low levels of toxicity," said Bishop. The company believes the observed safety profile of JCAR017 is both manageable and competitive to other CAR-T treatments.
Rival Kite Pharma is accelerating toward filing an application for U.S. approval of its KTE-C19 treatment in DLBCL, reporting 3-month follow-up interim data from its Phase 2 trial earlier this fall. Grade 3 CRS and neurological toxicity were observed in 18% and 34% of patients in that study, respectively.
While Kite could win approval for KTE-C19 in DLBCL as early as the first half of 2017, Juno looks set to remain a competitive threat as the pipelines of both companies continue to progress. Focus so far has been on which company is first to market, but development of second-generation candidates and improving safety profiles will be even more important as the drug class emerges.