Takeda's Proteasome Inhibitor Ninlaro Meets Primary Endpoint in Phase 3 Trial as First-Line Maintenance Therapy for Multiple Myeloma

Today, Takeda announced that its proteasome inhibitor Ninlaro (ixazomib), as a first-line maintenance therapy, significantly improved progression-free survival (PFS) in adult patients with multiple myeloma (MM) who had not undergone stem cell transplantation, meeting the primary endpoint of the Phase 3 trial.

Multiple myeloma is a malignant plasma cell disorder characterized by the abnormal proliferation of plasma cells in the bone marrow, accompanied by the overproduction of monoclonal immunoglobulins or light chains. These abnormal plasma cells accumulate in the bone marrow and can form tumors in multiple bone sites throughout the body. Not only are these cells unable to perform normal functions, but the antibodies they produce also impair the bone marrow’s ability to generate healthy blood cells. In addition, patients often present with multiple osteolytic lesions, hypercalcemia, anemia, and renal impairment. Globally, approximately 230,000 people are affected by this disease, with about 114,000 new cases diagnosed each year. Although multiple therapies are currently available for multiple myeloma (MM), many patients develop resistance to all approved treatments, underscoring the critical importance of developing innovative therapeutic approaches for MM.

Ninlaro is the first oral proteasome inhibitor approved by the FDA. It selectively binds to the PSMB5 subunit of the proteasome and inhibits its activity. Previously, it was approved by the U.S. FDA in combination with two other drugs for the treatment of patients with multiple myeloma (MM) who have received at least one prior therapy. Currently, Ninlaro is being evaluated in multiple pivotal clinical trials in combination with other agents for the treatment of patients with MM.

In the randomized, double-blind, placebo-controlled Phase 3 clinical trial TOURMALINE-MM4, 706 newly diagnosed adult patients who had not undergone stem cell transplantation completed 6 to 12 months of initial therapy prior to enrollment and subsequently received either Ninlaro or placebo after achieving a partial response. The trial results demonstrated that, compared with the placebo group, patients treated with Ninlaro showed a statistically significant improvement in progression-free survival (PFS), thereby meeting the primary endpoint of the study. This study aimed to validate the concept of switch maintenance therapy, wherein the maintenance therapy agent differs from the drugs used in the initial induction regimen. Detailed data from this trial will be presented at future medical conferences.

“The positive results from the TOURMALINE-MM4 trial provide significant momentum for our development of new therapies for multiple myeloma. This marks the third positive outcome from our TOURMALINE clinical trial program,” said Dr. Phil Rowlands, Head of Oncology Therapeutic Area at Takeda. “We remain committed to delivering convenient and well-tolerated treatment options for patients.”

References:

[1] Phase 3 Trial of NINLAROTM (ixazomib) as First Line Maintenance Therapy Met Primary Endpoint in Multiple Myeloma Patients not treated with Stem Cell Transplantation, Retrieved November 7, 2019, from https://www.businesswire.com/news/home/20191107005221/en/

[2] Overview | Understanding the R&D Status of Proteasome Inhibitors in 3 Minutes, Retrieved November 7, 2019, from https://www.wuximediaglobal.com/%e7%9b%98%e7%82%b9-3%e5%88%86%e9%92%9f%e8%af%bb%e6%87%82%e8%9b%8b%e7%99%bd%e9%85%b6%e4%bd%93%e6%8a%91%e5%88%b6%e5%89%82%e7%9a%84%e7%a0%94%e5%8f%91%e7%8e%b0%e7%8a%b6/

▽Follow [WuXi AppTecDe】WeChat Official Account