Takeda swings and misses with Ninlaro in AL amyloidosis, calls off phase 3 trial

With its once-biggest seller Velcade bracing for generic competition, Takeda has big plans for its oral multiple myeloma drug Ninlaro. But now, the med has suffered two setbacks in the span of a few months. After Ninlaro hit a snag at the FDA in an expanded use back in February, the company this week canned a phase 3 trial in another rare disease. Ninlaro had been in phase 3 testing for patients with systemic light-chain amyloidosis, but an analysis showed the med didn't meet a primary endpoint. Investigators were testing a combo of Ninlaro and dexamethasone in patients who'd relapsed or failed to respond to another treatment. Compared with a doctor’s choice of standard chemotherapy, Ninlaro didn’t improve overall hematologic response, so Takeda called the trial off. In patients with the disease, certain plasma cells produce abnormal immunoglobulin light-chain fragments. Patients develop amyloid deposits in organs and tissues, particularly the kidneys, heart, liver and nerves, Takeda said. That, in turn, can lead to organ failure and death.