Dive Brief:
- 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).
Dive Insight:
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.