Phase III trial of Ninlaro (ixazomib) + lenalidomide + dexamethasone demonstrates improved survival in multiple myeloma- Takeda

Takeda has announced results from the TOURMALINE-MM1, an international, randomized, double-blind, placebo-controlled Phase III clinical trial, showing that treatment with Ninlaro (ixazomib) plus lenalidomide (Revlimid) and dexamethasone is effective in extending progression free survival (PFS) with a manageable tolerability profile in patients with relapsed and/or refractory multiple myeloma. Trial results demonstrate a statistically significant and clinically meaningful (35%) improvement in PFS, with patients treated in the ixazomib arm living for a significantly longer time without their disease worsening compared to patients in the control arm (20.6 months vs. 14.7 months in control group).

Overall response rate (ORR) was 78.3% in the ixazomib arm and median duration of response was 20.5 months, versus 71.5% and 15 months in the control group. Ixazomib also showed efficacy in patients with high risk disease and cytogenetic abnormalities. Results demonstrated that median PFS more than doubled in the overall high-risk population (21.4 months in the ixazomib regimen compared to 9.7 months in the placebo regimen). A subsequent exploratory analysis of PFS was conducted (median follow-up 23.3 and 22.9 months in the IRd and placebo-Rd arms); median PFS 20 vs 15.9 months.

The most common adverse events in the TOURMALINE-MM1 trial for patients receiving ixazomib included diarrhoea, constipation, thrombocytopenia, peripheral neuropathy, nausea, peripheral oedema, vomiting and back pain. These results were presented at the 57th American Society of Hematology Annual meeting.

Comment: Ninlaro has recently been approved for multiple myeloma by the FDA and is currently under review by the European Medicines Agency. It was granted an accelerated assessment by the Committee for Medicinal Products for Human Use.