NLS Pharmaceutics Ltd. announced that the U.S. Food and Drug Administration (FDA) has reviewed the full protocol for the NLS-1031 study, part of the Phase 3 program for Mazindol ER, called AMAZE. In addition, the Company announced that the Phase 3 clinical trial protocol to evaluate the safety and efficacy of Mazindol ER in patients with narcolepsy type 1 received approval from the independent Institutional Review Board ("IRB"). 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 U.S. Based on the FDA's recommendations, both Phase 3 trials will measure the weekly cataplexy episodes as the primary endpoint over 8 weeks of treatment and excessive daytime sleepiness as a secondary objective using the Patient-Reported Outcomes Measurement Information System (PROMIS-SRI) and the Epworth Sleepiness Scale (ESS).

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. 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 early June at SLEEP 2023, the annual meeting of the American Academy of Sleep Medicine (AASM) and the Sleep Research Society (SRS). An IRB operates under FDA regulations and is an FDA registered constituted group that has been formally designated to review and monitor biomedical research involving human subjects. In accordance with FDA regulations, an IRB has the authority to approve, require modifications (to secure approval), or disapprove research.

The purpose of IRB review is to assure, both in advance and by periodic review, that appropriate steps are taken to protect the rights and welfare of humans participating as subjects in the research. To accomplish this purpose, IRBs use a group process to review research protocols and related materials (e.g., informed consent documents and investigator brochures) to ensure the protection of the rights and welfare of human subjects of research.