Cambridge, Mass., July 14, 2015 and Osaka, Japan, July 15, 2015 –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 innovation 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.
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.