Takeda has announced the premature ending of its Phase 3 TOURMALINE-AL1 clinical trial in patients with relapsed or refractory systemic light-chain (AL) amyloidosis, due to the study not meeting the first of two primary endpoints.
The trial was testing Ninlaro (ixazomib) in combination with dexamethasone, and found that the combo did not demonstrate a significant improvement in overall hematologic response compared to physician’s choice of standard of care regimens, thus prompting the company to discontinue the trial.
Despite the company being understandably disappointed with the outcome, Phil Rowlands, head of the Takeda’s Oncology Therapeutic Area Unit said that they “aim to maximise our learnings from this trial and share findings with the community in hopes of helping to improve care for patients living with this devastating disease.”
He continued, “This has been one of the largest studies ever conducted in systemic light-chain AL amyloidosis and we are proud to have led it. This study demonstrated our dedication to this rare and traditionally difficult-to-enroll patient population and we thank the patients and investigators for their engagement and participation.
We remain optimistic about Ninlaro and continue to investigate Ninlaro in patient populations across the continuum of multiple myeloma care.”
Takeda also stated that an Independent Data Monitoring Committee (IDMC) did not raise any concerns about the safety profile of Ninlaro in this setting, and that patients are encouraged to consult their study investigators to address any questions.
Primary AL amyloidosis is a condition that falls under the umbrella of plasma cell dyscrasias, and arises from a clonal plasma cell that produces abnormal immunoglobulin light-chain fragments. The most common organs affected are the kidneys, heart, liver, and autonomic or peripheral nerves and there are currently no treatments approved for the treatment of AL amyloidosis, meaning it is an area of high unmet need.