On Tuesday, Axsome Therapeutics Inc (NASDAQ:AXSM) said AXS-12 statistically significantly reduced the frequency of cataplexy attacks compared to placebo (p=0.017, primary endpoint) in Phase 3 ENCORE trial.
AXS-12 was also well tolerated with long-term dosing, and its safety profile was consistent with that observed in previously completed trials.
The primary endpoint was achieved with patients randomized to switch to placebo experiencing a mean increase of 10.29 cataplexy attacks per week compared to a mean increase of 1.32 attacks per week for patients randomized to continue AXS-12 treatment at 3 weeks (p=0.017).
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AXS-12 resulted in statistically significant benefit in cognition compared to placebo.
A higher number of patients who switched to placebo experienced worsening on the NSAQ Ability to Concentrate item compared to those continuing on AXS-12 (52.6% versus 14.3%) at 3 weeks (p=0.011).
A statistically significant improvement in narcolepsy overall was observed compared to placebo.
More patients randomized to switch to placebo reported worsening of their narcolepsy compared to those continuing on AXS-12 (52.6% versus 16.7%) at 3 weeks (p=0.024).
During the long-term open-label treatment portion of the trial, patients experienced substantial and sustained improvement of cataplexy with AXS-12 treatment.
Patients experienced a 71% reduction from baseline in mean weekly cataplexy attacks at 1 month with AXS-12 treatment, which was sustained with long-term treatment, resulting in a 77% reduction at 6 months.
Long-term open-label treatment with AXS-12 resulted in substantial improvements in excessive daytime sleepiness. Mean scores were reduced by 5.6 points at 1 month, with this improvement maintained with long-term treatment, resulting in a mean reduction of 7.3 points at 6 months.
William Blair views the safety profile of AXS-12 as a key differentiator from Jazz Pharmaceuticals plc’s (NASDAQ:JAZZ) Xyrem. The analyst reiterates the Outperform rating on Axsome.
“Ultimately, we believe AXS-12 is both an approvable and effective therapy, with the data released today building on both the efficacy and long-term safety of the product,” the analyst writes.
William Blair conservatively models $440 million in peak AXS-12 sales, relative to growing Wakix sales of $582 million in 2023, and says the asset is undervalued with investor focus on data for AXS-05 in Alzheimer's disease agitation expected before year-end.
Price Action: AXSM stock is up 2.5% at $99.46 at last check Wednesday.
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