Axsome drug succeeds in narcolepsy symptoms trial

2024-03-25

临床结果临床3期临床2期

An experimental drug from Axsome Therapeutics was successful in a late-stage clinical trial evaluating it as a treatment for narcolepsy symptoms.

The trial found Axsome’s drug to be fast-acting and substantially better than a placebo at reducing excessive daytime sleepiness, improving cognition and memory, and combating cataplexy — a condition characterized by a sudden loss of muscle tone.

Axsome said its drug, named AXS-12, met the trial’s main goal by significantly reducing weekly cataplexy attacks. Study participants given AXS-12 were experiencing 56% fewer attacks one week into treatment, compared to a 31% reduction in the placebo arm. By the end of week 5, the rates were down 83% and 66% across the two groups.

Additionally, Axsome noted how its drug was better at inducing “cataplexy remission,” defined as being completely free of the condition. This was seen in 33% of participants on AXS-12 versus 9.5% of those on placebo. The trial enrolled 90 participants, all of whom were diagnosed with both narcolepsy and cataplexy.

According to Axsome, the drug was also better than a placebo at improving patient function and quality of life, the severity of their narcolepsy, as well as two common comorbidities associated with the sleep disorder: anxiety and depression.

As for safety, the company said participants tolerated its drug well. The most commonly reported adverse events among those who took it were dry mouth, nausea and constipation, and those were “overall mild to moderate.” Researchers did not observe any serious adverse events in the trial.

Axsome is currently running an open-label extension study to assess the long-term safety and effectiveness of its drug. On a Monday morning call with investors, the company said it expects to finish gathering safety data from that study in about six months. Once the database of safety information is complete, Axsome said it would be ready to submit an approval application within six months.

If ultimately approved, AXS-12 would add another sleep disorder therapy to Axsome’s portfolio. In 2022, the company bought rights to Sunosi, which the Food and Drug Administration approved a few years prior as a treatment to improve wakefulness in adults with excessive daytime sleepiness associated with narcolepsy or obstructive sleep apnea.

TD Cowen analyst Joseph Thome and his team see “substantial operational synergies” between Sunosi and AXS-12. “The potential of AXS-12 in narcolepsy has largely been falling under investor radars, and thus we are encouraged that the [late-stage] trial was successful across measures of disease,” Thome wrote in a Monday note to clients.

Importantly, though, the analyst pointed out that the placebo response rate seen on the cataplexy attacks measure was “higher than expectations.” It was also “elevated when compared to other datasets,” including a earlier-stage study of AXS-12 and trials of Jazz Pharmaceuticals’ Xyrem and Harmony Biosciences’ Wakix. According to Thome, Axsome management did not indicate any specific factors contributing to the placebo response during the call with investors.

Shares of Axsome were down more than 3% by late Monday morning, to trade just under $78 apiece.

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