Phase 2 Data on Investigational Narcolepsy Drug Published

Key takeaways:

• Alixorexton demonstrated statistically significant and clinically meaningful improvements in wakefulness and cataplexy.
• At all doses tested, alixorexton demonstrated clinically meaningful improvements in patient-reported outcomes related to disease severity, fatigue, cognition, and health-related quality of life.
• Alixorexton was generally well tolerated at all doses tested.

Data from the Vibrance-1 phase 2 study of alixorexton in adults with narcolepsy type 1 (NT1) has been published in The Lancet Neurology. Alixorexton is an investigational, oral, selective orexin 2 receptor (OX2R) agonist in development by Alkermes plc for the treatment of NT1, narcolepsy type 2 (NT2), and idiopathic hypersomnia (IH).

The study showed that treatment with once-daily alixorexton led to statistically significant improvements from baseline versus placebo in both objective and subjective measures of excessive daytime sleepiness. The weekly cataplexy rate was also significantly decreased versus placebo in the 6 mg alixorexton group. The treatment was generally well tolerated, with most adverse events being mild to moderate.

“The data published in The Lancet Neurology highlight the robust efficacy of once-daily doses of alixorexton in patients with narcolepsy type 1 across measures of wakefulness and excessive daytime sleepiness. Along with a generally well-tolerated profile, alixorexton demonstrated improvements across a broad range of symptoms that affect daily functioning, including cataplexy, overall disease severity, cognition and fatigue,” says neurologist Giuseppe Plazzi, MD, PhD, director of the Narcolepsy Center at the IRCCS of the Neurological Sciences of Bologna and professor of childhood neuropsychiatry at the University of Modena and Reggio Emilia, in a release.

The phase 2 Vibrance-1 study was a randomized, double-blind, placebo-controlled study conducted in 92 adults with NT1. Participants received one of three doses of alixorexton (4 mg, 6 mg, or 8 mg) or a placebo once daily for six weeks. Based on the results, Alkermes has initiated the Brilliance Studies, a global phase 3 program evaluating alixorexton in adults with NT1 and NT2.

“In the Vibrance-1 study, alixorexton demonstrated substantial and clinically meaningful benefits across multiple dimensions of narcolepsy type 1,” says Craig Hopkinson, MD (MBChB), chief medical officer and executive vice president, research & development at Alkermes, in a release. “These findings highlight the potential of alixorexton to address a broad range of symptoms that continue to burden patients despite currently available therapies. With the global Brilliance phase 3 program now underway in both narcolepsy type 1 and type 2, we are excited to continue advancing alixorexton in this next stage of development.”

The US Food and Drug Administration has granted alixorexton Breakthrough Therapy designation for the treatment of NT1 and Orphan Drug Designation for the treatment of IH.

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