Phase 3 Trial of NINLAROTM (ixazomib) as First Line Maintenance Therapy Met Primary Endpoint in Multiple Myeloma Patients not treated with Stem Cell Transplantation

Takeda Pharmaceutical Company Limited (TSE: 4502/NYSE: TAK) ("Takeda") today announced the randomized, Phase 3 TOURMALINE-MM4 study met its primary endpoint of progression free survival (PFS). The trial evaluated the effect of single-agent oral NINLARO (ixazomib) as a first line maintenance therapy versus placebo in adult patients diagnosed with multiple myeloma not treated with stem cell transplantation. TOURMALINE-MM4 is the first industry sponsored Phase 3 trial to explore the concept of “switch” maintenance, the use of medicines not included in initial induction therapy, in this setting. NINLARO is currently not approved for this specific use.

“We are very encouraged by the results of the TOURMALINE-MM4 trial and continue our forward momentum in developing maintenance options for multiple myeloma patients. Importantly, this is the third positive Phase 3 readout from the TOURMALINE clinical trial program,” said Phil Rowlands, Ph.D., Head, Oncology Therapeutic Area Unit, Takeda. “We remain committed to bringing this convenient and well-tolerated treatment option to patients.”

The safety profile of NINLARO in the maintenance setting was consistent with previously reported results of single-agent NINLARO use, and there were no new safety signals identified in TOURMALINE-MM4.

Full data results will be submitted for presentation at an upcoming medical meeting.

About the TOURMALINE-MM4 Trial

TOURMALINE-MM4 is a randomized, placebo-controlled, double-blind Phase 3 study of 706 patients, designed to determine the effect of single-agent oral NINLARO^TM (ixazomib) maintenance therapy on progression-free survival (PFS), compared to placebo, in adult patients newly diagnosed with multiple myeloma not treated with stem cell transplantation, who have completed 6-12 months of initial therapy and achieved a partial response or better. For additional information, please visit https://clinicaltrials.gov/ct2/show/NCT02312258.

About Multiple Myeloma

Multiple myeloma is a life-threatening rare blood cancer that arises from the plasma cells, a type of white blood cell that is made in the bone marrow. These plasma cells become abnormal, multiply and release a type of antibody known as a paraprotein, which causes symptoms of the disease, including bone pain, frequent or recurring infections and fatigue, a symptom of anemia. These malignant plasma cells have the potential to affect many bones in the body and can cause a number of serious health problems affecting the bones, immune system, kidneys and red blood cell count. The typical multiple myeloma disease course includes periods of symptomatic myeloma followed by periods of remission. Nearly 230,000 people around the world live with multiple myeloma, with approximately 114,000 new cases diagnosed globally each year.