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.

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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.

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