Takeda's Ninlaro fails phase 3 amyloidosis trial

Takeda is discontinuing a trial of its drug Ninlaro in systemic light-chain AL amyloidosis after it did not meet the first of two primary endpoints.

The phase 3 TOURMALINE-AL1 trial was investigating patients with relapsed or refractory systemic light-chain (AL) amyloidosis. Treatment with Ninlaro (ixazomib) in combination with dexamethasone did not demonstrate a significant improvement in overall hematologic response compared to physician’s choice of standard of care regimens.

“While we are disappointed with this outcome, we 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,” said Phil Rowlands, head of the Oncology Therapeutic Area Unit at Takeda.

“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 added that an Independent Data Monitoring Committee did not raise any concerns about the safety profile of the drug in this setting.

AL amyloidosis is a rare and aggressive protein misfolding disorder. Fewer than 3,000 cases are diagnosed in the US each year. It is characterised by the deposition of amyloid in bodily organs and tissues. It frequently affects the heart, kidneys, liver, spleen, nervous system and gastrointestinal tract.

There are currently no treatments approved for the treatment of AL amyloidosis.

Ninlaro is an oral proteasome inhibitor, most commonly used to treat multiple myeloma – for which it was first approved in 2015.

Four other trials, investigating the drug in further indications for multiple myeloma, are still ongoing.