First proteasome inhibitor and first investigational therapy for amyloidosis to receive FDA Breakthrough Therapy designation

CAMBRIDGE, Mass. & OSAKA, Japan - Monday, December 1st 2014 [ME NewsWire]

(BUSINESS WIRE) - Takeda Pharmaceutical Company Limited (TSE:4502) today announced that the U.S. Food & Drug Administration (FDA) has granted Breakthrough Therapy status to the company’s investigational, oral proteasome inhibitor, ixazomib (MLN9708), for the treatment of relapsed or refractory systemic light-chain (AL) amyloidosis. This is the first proteasome inhibitor and first investigational therapy for AL amyloidosis to receive Breakthrough Therapy designation.

The development program for ixazomib in this indication progressed directly from a Phase 1 to a Phase 3 clinical trial, TOURMALINE-AL1, which is currently evaluating ixazomib plus dexamethasone in patients with relapsed or refractory AL amyloidosis. This is the only Phase 3 trial for relapsed or refractory AL amyloidosis and it is recruiting globally.

Breakthrough Therapy designation is intended to expedite the development and review of new medicines to treat serious or life-threatening conditions. Breakthrough Therapy designation requires preliminary clinical evidence indicating that the drug may demonstrate substantial improvement on a clinically significant endpoint(s) over available therapies. The data used to support this designation will be presented at the American Society of Hematology (ASH) annual meeting to be held December 6-9, 2014 in San Francisco [Abstract 3450: Long-Term Outcome of a Phase 1 Study of the Investigational Oral Proteasome Inhibitor (PI) Ixazomib at the Recommended Phase 3 Dose (RP3D) in Patients (Pts) with Relapsed or Refractory Systemic Light-Chain (AL) Amyloidosis (RRAL)].

“Patients with AL amyloidosis face a debilitating disease that can affect many of their organs and tissues. The Breakthrough Therapy designation for ixazomib is a major milestone in the development of new treatment options for patients battling this rare and aggressive disease,” said Raymond L. Comenzo, M.D., Director, Blood Bank and Stem Cell Processing Laboratory and Professor, Tufts University School of Medicine. “We are encouraged by positive preliminary data evaluating use of ixazomib in the treatment of patients with relapsed or refractory AL amyloidosis.”

“Ixazomib is a breakthrough drug showing activity and organ improvement in most of these heavily pretreated AL amyloidosis patients,” said Professor Giampaolo Merlini, Director, Center for Research and Treatment of Systemic Amyloidosis, University of Pavia, Italy.

AL amyloidosis is a rare and aggressive protein misfolding disorder with fewer than 3,000 cases diagnosed in the U.S. every year. It is characterized by the deposition of amyloid in bodily organs and tissues. While AL amyloidosis can affect different organs in different people, it frequently affects the heart, kidneys, liver, spleen, nervous system, and gastrointestinal tract. There are no approved treatments in the U.S. or globally for AL amyloidosis, representing a significant unmet medical need.

“This Breakthrough Therapy designation is also an important recognition of the strength of our oncology development program and our commitment to extend proteasome inhibition to conditions that have not traditionally been the focus of research and development,” said Michael Vasconcelles, M.D., Head, Oncology Therapeutic Area Unit, Takeda. “We would like to acknowledge and thank the patients, study investigators, and institutions whose participation in this program have made this milestone possible.”

Compounds that receive Breakthrough Therapy status receive more intensive FDA guidance on an efficient drug development program and an enhanced agency commitment of senior personnel.

About Ixazomib

Ixazomib is an investigational oral proteasome inhibitor, which is being studied in multiple myeloma (MM), systemic light-chain (AL) amyloidosis and other malignancies. Ixazomib was granted orphan drug designation in MM in both the U.S. and Europe in 2011, and for AL amyloidosis in both the U.S. and Europe in 2012. It is the first oral proteasome inhibitor to enter Phase 3 clinical trials. Four global Phase 3 trials are ongoing: TOURMALINE-MM1, investigating ixazomib vs. placebo in combination with lenalidomide and dexamethasone in relapsed and/or refractory MM; TOURMALINE-AL1, investigating ixazomib plus dexamethasone in patients with relapsed or refractory AL amyloidosis; TOURMALINE-MM2, investigating ixazomib vs. placebo in combination with lenalidomide and dexamethasone in patients with newly diagnosed MM; and TOURMALINE-MM3, investigating ixazomib vs. placebo as maintenance therapy in patients with newly diagnosed MM following induction therapy and autologous stem cell transplant. For additional information on the ongoing Phase 3 studies please visit www.tourmalinetrials.com or www.clinicaltrials.gov.

About Amyloidosis

Amyloidosis is a disease that occurs when the body's antibody-producing cells do not function properly and produce abnormal protein fibers known as amyloids. These amyloids can form deposits in any organ in the body. For this reason, amyloidosis can affect people differently. There are different types of amyloidosis and some are inherited. AL amyloidosis, also known as light-chain or primary or systemic amyloidosis, is the most common form of the disease. AL amyloidosis most frequently affects the kidneys and heart. However, almost any other tissue can be affected, including the liver, nervous system, and soft tissues.

About Takeda Pharmaceutical Company Limited

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 better health for people worldwide through leading innovation in medicine. Additional information about Takeda is available through its corporate website, www.takeda.com.

Contacts

Takeda Pharmaceutical Company Limited

Elizabeth Pingpank,

+1-617-444-1495

elizabeth.pingpank@takeda.com

Corporate Communications Dept.

(PR/IR), +81-3-3278-2037