ASH: Takeda details Ninlaro's miss in scrapped AL amyloidosis trial

ORLANDO, FLORIDA—Back in June, Takeda scrapped a Ninlaro trial in the rare disease light-chain (AL) amyloidosis. Saturday, it shared the data behind that decision—results exec say they’re excited to get into the public domain.

Ninlaro missed the primary endpoint in a phase 3 trial, failing to significantly top physician’s choice of treatment at spurring responses to therapy, Takeda showed Saturday at the American Society of Hematology annual meeting.

A combination of the Takeda drug plus dexamethasone provoked a response in 53% of patients, versus 51% for physician’s choice.

But the drug did hit some secondary endpoints that Takeda execs called “patient-relevant.” For one, hematologic benefits lasted for a median 46.5 months among those in the trial’s Ninlaro-plus-dexamethasone arm, compared with 20.2 months for those taking other treatments.

“It’s a rather unique position to be in: You power a study to show a primary endpoint, but then have what is really very encouraging data kind of on the back end,” Jenn Elliott, senior scientific director and medical lead at Takeda, said.

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

And those data are especially important considering AL amyloidosis patients’ lack of treatment options, Elliott said.

“This is a very underserved patient population. There’s nothing approved for these patients. They get bounced around a lot before they’re diagnosed; typically, they see about five specialists before they’re diagnosed, sometimes late in their disease,” she said. And in light of that, “we felt it was very important to make these data public.”

While Takeda has already canned the trial, dubbed Tourmaline-AL1, that doesn’t mean Ninlaro has no future in the disease. A handful of investigator-sponsored trials are looking at the drug in different combinations, including a pairing with Johnson & Johnson multiple myeloma star Darzalex that’s going through testing in the front-line and relapsed/refractory settings.

It hasn’t been a great year for Takeda’s Velcade follow-up. Before the AL amyloidosis slip, the Japanese company in February withdrew a multiple myeloma maintenance application from the FDA to await more survival data.