Cambridge, MA and Osaka, Japan (Press Release) – Takeda Pharma­ceu­tical Company Limited (TSE:4502) today announced that the ran­dom­ized, double-blind, placebo-controlled TOURMALINE-MM1 pivotal Phase 3 trial eval­u­ating the safety and efficacy of ixazomib, the first oral pro­te­a­some inhibitor, conducted in patients with re­lapsed or refractory multiple myeloma (MM) achieved its pri­mary end­point of im­prov­ing pro­gres­sion-free survival at the first pre-specified interim analysis. In the trial, patients treated with inves­ti­ga­tional ixazomib plus lena­lido­mide and dexa­meth­a­sone lived without their disease worsening for a sig­nif­i­cantly longer time compared to patients who received placebo plus lena­lido­mide/dexamethasone.

Efficacy and safety data were reviewed by an Independent Data Monitoring Committee (IDMC). Takeda intends to submit these data to health author­i­ties globally for market­ing authori­za­tions.

"We are very pleased with the out­come of this interim analysis of the pivotal trial, and are excited about the poten­tial that inves­ti­ga­tional ixazomib holds for patients with multiple myeloma," said Dixie-Lee Esseltine, MD, FRCPC, Vice Pres­i­dent, Oncology Clinical Research, Takeda. "We thank the patients and investigators for their engagement and con­tinued par­tic­i­pa­tion in this ongoing clin­i­cal evaluation of ixazomib."

About the TOURMALINE-MM1 Study

The study (n=722) is an inter­na­tional, ran­dom­ized, double-blind, placebo controlled clin­i­cal trial designed to compare the efficacy and safety of two treat­ment regi­mens admin­istered until pro­gres­sion – ixazomib plus lena­lido­mide and dexa­meth­a­sone versus placebo plus lena­lido­mide and dexa­meth­a­sone – in adult patients with re­lapsed and/or refractory MM.

Subjects in­cluded in the study had a con­firmed diag­nosis of MM, received one to three prior ther­a­pies and meet other outlined eligibility criteria. Patients who were refractory to lena­lido­mide or pro­te­a­some inhibitor-based ther­apy were excluded.

About Ixazomib

Ixazomib (MLN9708) is an inves­ti­ga­tional oral pro­te­a­some inhibitor, which is being studied in multiple myeloma (MM), systemic light-chain (AL) amyloidosis and other malig­nan­cies. Ixazomib was granted orphan drug desig­na­tion in MM in both the U.S. and Europe in 2011, and for AL amyloidosis in both the U.S. and Europe in 2012. Ixazomib received Break­through Therapy status by the U.S. Food and Drug Admin­istra­tion (FDA) for re­lapsed and/or refractory AL amyloidosis in 2014. It is also the first oral pro­te­a­some inhibitor to enter Phase 3 clin­i­cal trials. Four global Phase 3 trials are ongoing: TOURMALINE-MM1, investigating ixazomib vs. placebo in com­bi­na­tion with lena­lido­mide and dexa­meth­a­sone in re­lapsed and/or refractory MM; TOURMALINE-AL1, investigating ixazomib plus dexa­meth­a­sone in patients with re­lapsed or refractory AL amyloidosis; TOURMALINE-MM2, investigating ixazomib vs. placebo in com­bi­na­tion with lena­lido­mide and dexa­meth­a­sone in patients with newly diag­nosed MM; and TOURMALINE-MM3, investigating ixazomib vs. placebo as main­te­nance ther­apy in patients with newly diag­nosed MM fol­low­ing induction ther­apy and au­tol­o­gous stem cell trans­plant. For addi­tional in­­for­ma­tion on the ongoing Phase 3 studies please visit www.clinicaltrials.gov.

About Takeda

Located in Osaka, Japan, Takeda is a research-based global com­pany with its main focus on pharma­ceu­ticals. As the largest pharma­ceu­tical com­pany in Japan and one of the global leaders of the industry, Takeda is committed to strive to­wards better health for people world­wide through leading inno­va­t in medicine. Additional in­­for­ma­tion about Takeda is avail­able through its corporate website, www.takeda.com.

Source: Takeda Pharma­ceu­ticals.