Alkermes PLC announced on Wednesday at a major sleep medicine conference in Baltimore that its investigational drug alixorexton significantly improved the ability to stay awake and reduced excessive daytime sleepiness in patients with type 2 narcolepsy.

This finding suggests the much-watched class of orexin receptor agonist drugs may benefit a broader patient population than initially anticipated. This study is among the first large clinical trials to demonstrate efficacy for such a drug in type 2 narcolepsy.

Current treatment options largely rely on stimulants or sleep-promoting medications, which often have limited efficacy and fail to fully alleviate all symptoms. This has spurred pharmaceutical companies to race toward developing more promising treatments that could address other sleep-wake disorders.

Alkermes PLC CEO Richard Pops stated in an interview, "This is the first time we have something that's not a 'blunt instrument' to treat narcolepsy. We're going directly into the neural circuitry in the brain that drives wakefulness."

The orexin system, discovered in the late 1990s, is a core mechanism in the brain for regulating wakefulness. Patients with classic type 1 narcolepsy lose the neurons that produce orexin. However, for years, developing drugs that could enter the brain and safely mimic this neuropeptide's action has been a technical challenge.

Today, the significant potential of orexin biology is fueling a high-stakes pharmaceutical race, with participants including Eli Lilly and Co and Takeda Pharmaceutical Co. Ltd. Earlier this year, Eli Lilly committed up to $7.8 billion to acquire Centessa Pharmaceuticals, betting that this scientific mechanism could ultimately be extended to a range of neurological disorders.

Takeda currently leads in this field, with its lead drug for another type of narcolepsy, oveporexton, having already delivered strong results in late-stage trials for type 1 narcolepsy. New data presented at the conference this week showed the drug's benefits extend beyond maintaining wakefulness to improvements in cognitive function, daily living activities, and nighttime sleep quality.

In February, oveporexton was accepted for priority review by the U.S. Food and Drug Administration, with a decision expected in the second half of this year.

The mid-stage study by Alkermes PLC enrolled 93 patients with type 2 narcolepsy—a type that does not involve the profound orexin deficiency typical of type 1. The company reported that after eight weeks of treatment, patients receiving alixorexton showed a significant increase in time awake during a standardized maintenance of wakefulness test and a notable reduction in self-reported daytime sleepiness compared to the placebo group.

Insomnia and frequent urination were the most common side effects.

Alkermes PLC is betting that this class of orexin-mimicking drugs could be used for a wider range of indications. The company is also conducting a clinical trial of alixorexton for idiopathic hypersomnia, another disorder characterized by excessive sleepiness.

Furthermore, the company is researching other drug candidates based on the orexin mechanism for conditions including attention deficit hyperactivity disorder (ADHD) and fatigue associated with multiple sclerosis and Parkinson's disease.

The 64-year-old Pops, who has served as CEO of Alkermes PLC since the early 1990s, is set to retire at the end of July, after which he will remain as Chairman of the Board. Pops was previously seen as a potential candidate for the role of FDA Commissioner—a position that has been vacant since the departure of former Commissioner Marty Makary in May.

When asked if he would consider a government role, Pops said he has "always been drawn to public service," noting his "25 years of deep involvement with FDA-related matters," but that his current focus remains on Alkermes.