Swiss clinical-stage biopharmaceutical company NLS Pharmaceutics Ltd is set to begin the phase 3 clinical trial for mazindol ER, called AMAZE, following US Food and Drug Administration (FDA) review of the full protocol and approval from the independent Institutional Review Board (IRB) to evaluate the safety and efficacy of the drug in patients with narcolepsy type 1.
The AMAZE program will encompass two almost-identical double-blind phase 3 studies (N=50 each) investigating mazindol ER versus placebo in adult patients with narcolepsy, commencing this summer at multiple sites exclusively in the United States.
Based on the FDA’s recommendations, both phase 3 trials will measure the weekly cataplexy episodes as the primary endpoint over eight weeks of treatment and excessive daytime sleepiness as a secondary objective using the Patient-Reported Outcomes Measurement Information System and the Epworth Sleepiness Scale.
“In addition to IRB approval of the phase 3 study protocol for AMAZE obtained last week, with this regulatory milestone acheived, we can recruit US clinical sites quickly and efficiently, allowing us to move forward with providing mazindol ER to patients with narcolepsy type 1,” says George Apostal, MD, MS, chief medical officer of NLS, in a release.
Patients who complete these studies will be offered participation in a 12-month open-label extension (OLE) study. To be eligible for enrollment into the OLE study, patients must be at least 18 years of age and have been diagnosed with narcolepsy with cataplexy.
“We are pleased with the FDA’s review of the phase 3 protocol and now expect to move quickly to begin enrolling patients in the AMAZE program in centers across the US in the coming days,” says Alex Zwyer, CEO of NLS, in a release.
NLS previously reported on the phase 2 study results in narcolepsy in which mazindol ER met all primary and secondary endpoints. Patients treated with mazindol ER in the randomized phase 2 trial showed continued improvement after rolling over into the OLE study, and patients treated with placebo in the randomized phase 2 trial and who subsequently received mazindol ER in the OLE study showed similar efficacy with the mazindol ER-treated patients in the randomized trial.
Data from the Phase 2 studies were presented in June at SLEEP 2023, the annual meeting of the American Academy of Sleep Medicine and the Sleep Research Society.
Mazindol ER is a proprietary extended-release formulation of mazindol and is being developed for the treatment of narcolepsy and potentially other sleep-wake disorders such as idiopathic hypersomnia, for which NLS recently obtained Orphan Disease Designation from the FDA and the European Medicines Agency.
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