Positive results in phase 2b trial of orexin receptor agonist for narcolepsy type 1

Key takeaways:

• TAK-861 was investigated in a trial of 112 individuals with narcolepsy type 1 over 8 weeks.
• Data showed significant improvements in primary, secondary endpoints, while efficacy was also sustained.

Takeda has reported positive results from a phase 2b clinical trial of an investigational, oral orexin receptor agonist for narcolepsy type 1.

In a press release, Takeda reported that TAK-861 was examined in a randomized, double-blind, placebo-controlled, multiple dose study, in which 112 patients with NT1 demonstrated statistically significant and clinically meaningful improvements across primary and secondary endpoints, with efficacy sustained over 8 weeks of treatment. Data from the trial were presented at the 38th annual meeting of the American Academy of Sleep Medicine and the Sleep Research Society.

Consistent results were also achieved in key secondary endpoints including the Epworth Sleepiness Scale and Weekly Cataplexy Rate, while a majority of the participants who completed the trial enrolled in its long-term extension study with some reaching 1 year of treatment, per the release.

Takeda said in the release that TAK-861 was generally safe and well-tolerated during the study, with no treatment-related serious treatment-emergent adverse events (TEAEs) or discontinuations due to TEAEs. No cases of hepatotoxicity or visual disturbances were reported in either the parent trial or LTE, while the most common TEAEs — which were reported as mild to moderate in severity — were insomnia, urinary urgency and frequency and salivary hypersecretion.

“In this trial, TAK-861’s profile balanced efficacy and safety with the potential to establish a new standard of care for people with [narcolepsy type 1],” Sarah Sheikh, MD, MSc, BM, BCh, MRCP, head of global development at Takeda, said in the release. “We are dedicated to investigating the full potential of orexin biology and advancing TAK-861 to late-stage clinical trials, with the ultimate goal of delivering a potential first-in-class treatment that can make a meaningful difference for patients.”

According to the release, Takeda intends to initiate global phase 3 clinical trials of TAK-861 in NT1 this year.