New Drug Application Submitted for Multiple Myeloma Drug

Five pivotal phase 3 trial ongoing for oral proteasome inhibitor.

Takeda Pharmaceutical Company announced Tuesday it has filed a New Drug Application (NDA) for an investigational oral proteasome inhibitor for patients with relapsed and/or refractory multiple myeloma.

The NDA submission for the drug ixazomib is based on an ongoing randomized, double-blind, placebo controlled clinical trial including 722 patients. The trial, which is the first of a planned five phase 3 clinical trials, will examine ixazomib plus lenalidomide and dexamethasone over placebo plus lenalidomide and dexamethasone in adult relapsed and/or refractory multiple myeloma patients.

“The TOURMALINE-MM1 study is the first in a series of five Phase 3 trials within our ixazomib program, which is designed to evaluate whether sustained therapy with a proteasome inhibitor, delivered orally, improves the clinical outcomes of patients living with multiple myeloma or with systemic light-chain (AL) amyloidosis,” said Andrew Plump, MD, PhD, Takeda Chief Medical and Scientific Officer. “This submission marks an important step in Takeda’s ongoing commitment to innovation for patients living with multiple myeloma. We thank the patients and their families for placing their trust in us and in ixazomib as they continue to participate in the TOURMALINE program.”

Ixazomib (MLN9708) was granted orphan drug designation for multiple myeloma in 2011 and for AL amyloidosis in 2012 in both Europe and the United States. The FDA granted ixazomib Breakthrough Therapy designation for relapsed or refractory AL amyloidosis in 2014.

The drug is the first oral proteasome inhibitor to enter phase 3 clinical trials.

“Continuous treatment is emerging as a standard of care in multiple myeloma with demonstrable improvement in long-term outcomes,” said clinical program leader Paul Richardson, MD. “Proteasome inhibition has become an essential component of treatment, but there are logistical challenges for patients with both intravenous and subcutaneous approaches, and especially in the absence of an effective oral option. If approved, ixazomib, with the convenience of once-a-week oral administration as well as promising efficacy, should provide a very meaningful advance for our patients.”