Pharma Focus Europe

NLS Pharmaceutics Receives FDA Review and IRB Approval for Full Study Protocol, Advances Phase 3 Clinical Program (AMAZE) for Mazindol Er in the Treatment of Narcolepsy

Tuesday, July 04, 2023

NLS Pharmaceutics Ltd. (Nasdaq: NLSP) has made significant announcements regarding the development of their drug Mazindol ER for narcolepsy type 1. The U.S. Food and Drug Administration (FDA) has thoroughly reviewed the NLS-1031 study protocol, which is part of the Phase 3 program known as AMAZE. Additionally, the FDA has given its approval for the Phase 3 clinical trial protocol to assess the safety and efficacy of Mazindol ER in adult patients with narcolepsy.

The AMAZE Program comprises two double-blind Phase 3 studies, each involving 50 adult patients, who will be given Mazindol ER or a placebo. The primary focus of these trials will be to measure weekly cataplexy episodes during an 8-week treatment period, while excessive daytime sleepiness will be a secondary objective, measured using the Patient-Reported Outcomes Measurement Information System (PROMIS-SRI) and the Epworth Sleepiness Scale (ESS).

Dr. George Apostal, NLS's Chief Medical Officer, expressed satisfaction with the FDA's recommendations and the independent Institutional Review Board (IRB) approval. This milestone allows them to quickly recruit U.S. clinical sites and offer Mazindol ER to patients with narcolepsy type 1. Following the completion of Phase 3 studies, patients may have the opportunity to participate in a 12-month open-label extension (OLE) study, provided they meet the eligibility criteria.

NLS CEO Alex Zwyer stated their enthusiasm for the FDA's review and their readiness to initiate patient enrollment in the AMAZE program across multiple U.S. centers shortly.

Earlier, NLS had reported positive results from the Phase 2 study in narcolepsy, where Mazindol ER demonstrated efficacy by meeting primary and secondary endpoints. Patients who continued to receive Mazindol ER in the OLE study after the Phase 2 trial showed sustained improvement, and those who switched from placebo to Mazindol ER in the OLE study demonstrated similar efficacy to those treated with the drug in the randomized trial. The Phase 2 study data was presented at the SLEEP 2023 conference.

In conclusion, NLS is progressing well with the development of Mazindol ER for narcolepsy type 1, with the FDA review of the Phase 3 protocol and the IRB approval. The company is now gearing up to enroll patients in the AMAZE program, with a possibility of extending participation in the study.

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