Alkermes has announced positive results from a Phase 2 clinical trial of alixorexton, an investigational daily pill for type 1 narcolepsy that demonstrated statistically significant improvements in daytime wakefulness and achieved normal alertness levels in patients over a six-week treatment period.
Trial Results Show Broad Efficacy Across Doses
The mid-stage trial evaluated patients with narcolepsy type 1, a neurological condition characterized by excessive daytime sleepiness and cataplexy, the sudden loss of muscle control. According to the company's announcement on Monday, patients taking all doses tested of alixorexton experienced statistically significant improvements in wakefulness, reaching normal levels, as well as statistically significant decreases in daytime sleepiness.
The study assessed both the efficacy and safety of alixorexton in maintaining alertness during waking hours, with participants showing measurable improvements in their ability to stay awake compared to baseline levels.
Mixed Results on Cataplexy Control
While the wakefulness results were consistent across all dose levels, only patients receiving the middle dose tested showed a statistically significant decrease in the frequency of cataplexy episodes. CEO Richard Pops attributed this finding to methodological limitations rather than dose-dependent efficacy, explaining that patients self-report cases of cataplexy based on their memory.
"Those results were likely due to methodological limitations, as patients self-report cases of cataplexy based on their memory," Pops said. The company plans to refine the method for measuring cataplexy in future trials to address these reporting challenges.
Market Positioning and Future Development
The positive results position Alkermes to compete in what industry analysts predict will be a multibillion-dollar narcolepsy treatment market. The company did not release specific numerical data from the trial but emphasized the statistical significance of the wakefulness improvements across all tested doses.
The findings suggest potential benefits for managing symptoms associated with type 1 narcolepsy, though the company acknowledges that further research is required to confirm these outcomes and evaluate long-term effects. The success of alixorexton in achieving normal wakefulness levels represents a significant development for patients with this debilitating neurological condition.
