Takeda Pharmaceuticals Release: European Medicines Agency Grants Accelerated Assessment Of Ixazomib For Patients With Relapsed/Refractory Multiple Myeloma

CAMBRIDGE, Mass. & OSAKA, Japan--(BUSINESS WIRE)--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 innovation.

“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.”

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 innovation 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 11^th 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 innovation in medicine. Additional information about Takeda is available through its corporate website, www.takeda.com.