Cambridge, MA and Osaka, Japan (Press Release) – Takeda Pharma­ceu­tical Company Limited (TSE: 4502) today announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Med­i­cines Agency (EMA) has granted an accelerated assess­ment to ixazomib, an inves­ti­ga­tional oral pro­te­a­some inhibitor for the treat­ment of patients with re­lapsed and /or refractory multiple myeloma. The EMA awards an accelerated assess­ment to those medicines deemed to be of major public health interest and, in particular, thera­peutic inno­va­t.

Takeda ex­pec­ts to submit a market­ing authori­za­tion appli­ca­tion for ixazomib in the European Union (EU) in the coming weeks. The sub­mission is based on the results of the first pre-specified interim analysis of the pivotal Phase 3 trial TOURMALINE-MM1. This study is 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 to pro­gres­sion in this trial and eval­u­ated for long-term out­comes.

“We are pleased that the CHMP has granted accelerated assess­ment for ixazomib, and believe that this desig­na­tion underscores the urgent need for thera­peutic inno­va­t in multiple myeloma treat­ments,” said Andrew Plump, M.D., Ph.D., Takeda’s Chief Medical and Scientific Officer. “We look forward to sharing the TOURMALINE-MM1 study data from the first pre-specified interim analysis at an upcoming medical meeting, and greatly appreciate the ongoing dedication and commitment from the patients and their families who have been participating in the ixazomib clin­i­cal devel­op­ment pro­gram.”

“Continuous ther­apy is emerging as a standard of care in multiple myeloma because it has dem­onstrated im­proved long-term out­comes,” said Philippe Moreau, MD, University Hospital of Nantes in France. “If ixazomib is approved, for the first time physicians will have the option of an all-oral pro­te­a­some inhibitor-based regi­men for the treat­ment of multiple myeloma, which could be a real advantage in delivering sus­tained ther­apy.”

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 red blood cell count, with some of the more common symp­toms in­­clud­ing bone pain and fatigue, a symp­tom of anemia. Multiple myeloma is a rare form of cancer with approx­i­mately 39,000 new cases in the EU 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, systemic light-chain (AL) amyloidosis, and other malig­nan­cies. Ixazomib was granted orphan drug desig­na­tion in multiple myeloma in both the United States 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 the brand for the global on­col­ogy business unit 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.