Takeda's new drug submission for ixazomib, a new treatment for patients with relapsed and/or refractory multiple myeloma, is accepted for priority review by Health Canada

Announcement comes as recent clinical results reveal ixazomib significantly extends progression-free survival for patients with relapsed/refractory Multiple Myeloma

OAKVILLE, ON, Feb. 16, 2016 /CNW/ - The New Drug Submission (NDS) for Takeda's ixazomib has been accepted for Priority Review by Health Canada. Ixazomib is the first investigational oral proteasome inhibitor for the treatment of patients with relapsed and/or refractory multiple myeloma. If approved, ixazomib will be the first oral proteasome inhibitor available in Canada, helping meet the urgent needs of patients living with multiple myeloma, a devastating, relapsing and incurable rare cancer. With once-weekly oral dosing, ixazomib enables the first all-oral triplet regimen including both a proteasome inhibitor and an immunomodulator for the treatment of relapsed and/or refractory multiple myeloma.

Health Canada may grant Priority Review status for drug submissions indicated for the treatment of a severely debilitating disease or condition wherein there exists an unmet medical need or for which a substantial improvement in the benefit/risk profile of the therapy is demonstratedⁱ. On November 20, 2015, the U.S. Food and Drug Administration (FDA) approved the New Drug Application (NDA) for ixazomib capsules under Priority Review status. In July 2015, ixazomib was granted Accelerated Assessment by the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA).

"It is extremely encouraging to hear of Health Canada's decision to grant Priority Review of a new treatment option for multiple myeloma," said Aldo Del Col, Co-founder and Chairman of the Board of Myeloma Canada. "For the many Canadians living with this incurable but increasingly treatable blood cancer, this news provides hope to patients for urgently-needed new treatment options that will increase survival and provide a higher quality of life."

The NDS for ixazomib was primarily based on the results of the final 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 will be evaluated for long-term outcomes.

"It's with great pleasure that we welcome Health Canada's decision to grant Priority Review for ixazomib. This is an important development for Canadians living with multiple myeloma" said Chatrick Paul, General Manager at Takeda Canada. "Health Canada has clearly shown their commitment to Canadian patients living with complex and challenging diseases."

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 anaemia. Multiple myeloma is a rare form of cancer. In Canada, it is estimated that approximately 7,500 people live with multiple myeloma, with 2,700 new cases estimated to be diagnosed in 2015ⁱⁱ.

About Ixazomib
Ixazomib 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. FDA for relapsed or refractory AL amyloidosis in 2014. It is also the first oral proteasome inhibitor to enter Phase 3 clinical trials. There are currently 22 Phase 3 clinical trial sites of ixazomib in Canada.

The TOURMALINE 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

In addition to the TOURMALINE program, a large number of investigator initiated studies are evaluating ixazomib for patients globally.

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 at takeda.com.

Takeda Canada, located in Oakville, Ontario, is the Canadian sales and marketing organization of Takeda Pharmaceutical Company Limited. Takeda Canada is transforming to become an agile specialty pharmaceutical company, focusing on gastroenterology and oncology, while continuing to meet a number of important primary care needs. Additional information about Takeda Canada is available at takedacanada.com.

ⁱ Health Canada. "Guidance for Industry - Priority Review of Drug Submissions" Available at http://www.hc-sc.gc.ca/dhp-mps/prodpharma/applic-demande/guide-ld/priorit/priordr-eng.php. Accessed January 2016.

ⁱⁱ Canadian Cancer Statistics 2015

SOURCE Takeda Pharmaceutical Company Limited

Media contact: Natacha Raphael, Corporate Communications, Takeda Canada Inc., Tel: [phone], [email protected]