December 2019 - inThought Research

December 18, 2019

Impact: Moderate

Gilead’s Kite subsidiary has announced that it has submitted a BLA for the investigational CAR-T KTE-X19 for the treatment of patients with relapsed or refractory mantle cell lymphoma (R/R MCL).
The BLA is based on data from the Phase 2 ZUMA-2 trial which demonstrated an ORR of 93% (CR rate = 67%) in 60 evaluable patients. Enrolled patients had MCL that was refractory to or had relapsed following up to 5 prior lines of therapy.
Grade 3 or higher cytokine release syndrome (CRS) and neurotoxicity were seen in 15% and 31% of patients, respectively. No Grade 5 CRS or neurological events were observed.
Detailed findings from ZUMA-2 were presented Monday at ASH 2019.
KTE-X19 has been granted FDA Breakthrough Therapy and EMA PRIME status for the MCL indication.
Kite anticipates submitting a European Marketing Authorization Application in early 2020.

December 5, 2019

Impact: High

CymaBay Therapeutics has terminated its phase 2b study of PPARδ agonist, seladelpar, in patients with NASH due to atypical histological findings.
Planned, blinded histological assessments of liver biopsies from trial participants revealed histology characterized as an interface hepatitis presentation, with or without biliary injury.
Notably, the atypical histological findings were observed in patients who demonstrated on-study improvement or stabilization of their biochemical measures of inflammation and liver injury and none of these patients experienced liver-related adverse events after 52 weeks of treatment. Evaluation is ongoing to better understand these findings.
The company also terminated its recently initiated phase 2 study of seladelpar in primary sclerosing cholangitis (PSC), and all studies in primary biliary cholangitis (PBC) have been placed on clinical hold.

Gilead Aims to Bring 2nd CAR-T Product, KTE-X19, to Market with BLA Submission for Mantle Cell Lymphoma