FDA grants fast track designation to ME-401 and orphan drug designation to umbralisib for the treatment of FL

The United States Food & Drug Administration (FDA) has granted fast track designation to ME-401 for the treatment of patients with relapsed/refractory (R/R) follicular lymphoma (FL).¹ The FDA has also granted orphan drug designation to umbralisib for patients with FL.²

ME-401

Fast track designation has been granted to ME-401 for the treatment of adult patients with R/R FL who have received at least two prior systemic therapies. ME-401 is an oral, once-daily, selective inhibitor of phosphatidylinositol 3-kinase delta (PI3Kδ) and is currently in clinical development

• Phase Ib study (NCT02914938)³
  • ME-401 is being investigated as monotherapy or in combination with rituximab or zanubritinib in patients with R/R FL and other B-cell malignancies
  • Interim results reported in October 2019 show, in 73 patients with R/R FL, R/R chronic lymphocytic leukemia, or small lymphocytic leukemia, ME401 achieved an overall response rate (ORR) of 81%. In patients with FL (n = 55), this was 78%. No Grade 4/5 adverse events were reported. Patients treated with an intermittent dosing schedule showed significantly increased overall tolerability compared with patients treated on a continuous schedule
• Phase II “Trials of PI3K delta in Non-Hodgkin’s lymphoma” (TIDAL) study (NCT03768505)³
  • Investigating ME-401 as monotherapy in patients with R/R FL who have received ≥ 2 prior systemic therapies including chemotherapy and an anti-CD20 antibody
  • Two oral dosing schedules are under investigation, with approximately 150 patients randomized 1:1 to either
    • 60 mg once daily, continuously 
    • 60 mg once daily for two cycles (8 weeks) followed by an intermittent schedule of 60 mg once daily for the first 7 days of a 28-day cycle and three weeks of 60 mg placebo
  • Efficacy endpoint: objective response rate

Umbralisib²

Umbralisib, also known as TGR-1202, is a dual inhibitor of PI3Kδ and CK1ε under investigation in patients with FL. The FDA Orphan Drug Designation is based on the results of the phase IIb UNITY-NHL study (NCT02793583). In the FL arm, patients must have received ≥ 2 prior lines of therapy, including an anti-CD20 regimen and an alkylating agent. It was announced in October 2019 that the study had met the primary endpoint of ORR (target 40–50%) as assessed by independent review committee.  

The FDA previously granted umbralisib breakthrough therapy designation for the treatment of adult patients with marginal zone lymphoma. Read more here.