Cambridge, MA and Osaka, Japan (Press Release) – Takeda Pharma­ceu­tical Company Limited (TSE: 4502) today announced that a New Drug Application (NDA) has been submitted to the United States (U.S.) Food and Drug Admin­istra­tion (FDA) for ixazomib, an inves­ti­ga­tional oral pro­te­a­some inhibitor for the treat­ment of patients with re­lapsed and/or refractory multiple myeloma.

The NDA sub­mission was based on the pivotal Phase 3 trial TOURMALINE-MM1, an inter­na­tional, ran­dom­ized, double-blind, placebo controlled clin­i­cal trial of 722 patients designed to eval­u­ate the superiority of ixazomib plus lena­lido­mide and dex­a­meth­a­sone over placebo plus lena­lido­mide and dex­a­meth­a­sone in adult patients with re­lapsed and/or refractory multiple myeloma. Patients con­tinue to be treated in this trial and eval­u­ated for long-term out­comes.

“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, M.D., Ph.D., Takeda’s Chief Medical and Scientific Officer. “This sub­mis­sion marks an im­por­tant step in Takeda’s ongoing commitment to inno­va­t 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.”

“Continuous treat­ment is emerging as a standard of care in multiple myeloma with demonstrable im­prove­ment in long-term out­comes,” commented Paul Richardson, M.D., Clinical Program Leader and Director of Clinical Research, Jerome Lipper Multiple Myeloma Center, Institute Physician at Dana-Farber Cancer Institute. “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.”

This is the first regu­la­tory sub­mis­sion for ixazomib. Additional filings are planned to begin in Europe and other countries later this year.

About Multiple Myeloma

Multiple myeloma is a cancer of the plasma cells, which are found in the bone marrow. In multiple myeloma, a group of plasma cells, or myeloma cells, becomes can­cer­ous and multiplies, in­creas­ing the number of plasma cells to a higher than normal level. Because plasma cells circulate widely in the body, they have the poten­tial to affect many bones in the body, possibly resulting in compression fractures, lytic bone lesions and related pain. Multiple myeloma can cause a number of serious health problems affecting the bones, immune system, kidneys and an individual’s red blood cell count, with some of the more common symp­toms in­­clud­ing bone pain and fatigue, a sign of anemia. Multiple myeloma is a rare form of cancer with approx­i­mately 20,000 new cases in the U.S. and 114,000 new cases globally per year.

About Ixazomib

Ixazomib (MLN9708) is an inves­ti­ga­tional oral pro­te­a­some inhibitor which is being studied in multiple mye­lo­ma, AL amyloidosis, and other malig­nan­cies. Ixazomib was granted orphan drug desig­na­tion in multiple myeloma 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 or re­fractory AL amyloidosis in 2014. It is also the first oral pro­te­a­some inhibitor to enter Phase 3 clin­i­cal trials.

Ixazomib’s clin­i­cal devel­op­ment pro­gram further reinforces Takeda’s ongoing commitment to devel­op­ing inno­va­tive ther­a­pies for people living with multiple myeloma world­wide and the health­care professionals who treat them. Five global Phase 3 trials are ongoing:

• TOURMALINE-MM1, investigating ixazomib vs. placebo, in combination with lenalidomide and dex­a­meth­a­sone in relapsed and/or refractory multiple myeloma
• TOURMALINE-MM2, investigating ixazomib vs. placebo, in combination with lenalidomide and dex­a­meth­a­sone in patients with newly diagnosed multiple myeloma
• TOURMALINE-MM3, investigating ixazomib vs. placebo as maintenance therapy in patients with newly diagnosed multiple myeloma following induction therapy and autologous stem cell transplant (ASCT)
• TOURMALINE-MM4, investigating ixazomib vs. placebo as maintenance therapy in patients with newly diagnosed multiple myeloma who have not undergone ASCT
• TOURMALINE-AL1, investigating ixazomib plus dex­a­meth­a­sone vs. physician choice of selected regimens in patients with relapsed or refractory AL amyloidosis

For addi­tional in­­for­ma­tion on the ongoing Phase 3 studies please visit www.clinicaltrials.gov.

About Takeda Oncology

Takeda Oncology is a global business unit of Millennium Pharma­ceu­ticals Inc., a wholly owned sub­sid­i­ary of Takeda Pharma­ceu­tical Company Limited. Takeda aspires to cure cancer by delivering novel medicines to meet the unique and urgent needs of people living with cancer, their loved ones and the health care pro­viders who sup­port them around the world. Takeda is the 11th largest global on­col­ogy com­pany with a portfolio of paradigm-changing ther­a­pies and an extensive pipe­line of investi­ga­tional prod­ucts that have the poten­tial to sig­nif­i­cantly im­prove patient out­comes across a number of cancers. By combining the power of leading scientific minds with our agile and entrepreneurial spirit and the vast resources of a global pharma­ceuti­cal com­pany, Takeda Oncology is finding new and inno­va­tive ways to im­prove the treat­ment of cancer. Additional in­­for­ma­tion about Takeda Oncology is avail­able through its website, www.takedaoncology.com.

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.