Takeda’s Narcolepsy Trials Stopped Over Unspecified Safety Signal

An unspecified safety signal has slammed the brakes on Takeda Pharmaceutical’s twin studies of TAK-994, an investigational oral orexin agonist intended for the treatment of excessive daytime sleepiness in patients with narcolepsy type 1 (NT1), a chronic neurological disorder that alters the sleep-wake cycle.

Orexin, a hormone produced in the pituitary, regulates the sleep/wake cycle and REM sleep. NT1 is characterized by a loss of orexin-producing neurons. TAK-994 and other orexin agonists increase neuronal uptake of the molecule.

As an “immediate precautionary measure,” Takeda has suspended dosing of patients and has decided to stop both phase 2 studies early,” to allow for “timely interpretation of the benefit /risk profile of TAK-994 and to determine next steps for the program, “the company said.

TAK-994 won FDA’s Breakthrough Therapy status in July based on preclinical and early-phase data suggesting that it significantly improved both objective and subjective measures of daytime wakefulness in patients with NT1.