0593 Functional Outcomes of Sleep Questionnaire in a Phase 2 Study of Mazindol ER in Narcolepsy

Introduction Efficacy secondary outcome measure Functional Outcomes of Sleep Questionnaire, Short Version (FOSQ-10] was collected in the Phase 2 (POLARIS, NLS-1021), multicenter (n=21) study of mazindol ER in adult participants with narcolepsy type 1 (NT1) or type 2 (NT2) (ClinicalTrials.gov Identifier: NCT04923594). Methods Adults diagnosed with NT1 or NT2 who were washed out of narcolepsy medications at baseline were included into the study. Patients were treated with mazindol ER (NLS-2) at once-daily 2 mg for one week and once-daily 3 mg thereafter or matching placebo (PBO) on a 1:1 ratio. Throughout the study, FOSQ-10 was assessed and compared vs. PBO scores at baseline. Treatment emergent adverse events (TEAEs) were assessed. Results Sixty-seven participants were randomized (NT1 33.3% in NLS-2 group versus 35.3% in PBO). At baseline, the mean [SD] FOSQ-10 score was 23.1 (5.5) for the NLS-2 arm and 23.7 (7.1) for the PBO arm. After four weeks of treatment, there was an increase of 4.1 (9.0) on NLS-2 vs an increase of 0.4 (6.9) on PBO (p = 0.04). Similar magnitude of effect was seen in NT1 patients (D = +4.7, p = 0.08, n = 20) as well as NT2 patients (D = +3.0, p = 0.20, n = 44) No serious adverse events were reported. The most frequently reported TEAEs (>5%) were dry mouth, nausea, decreased appetite and tachycardia. Conclusion Mazindol ER resulted in significant improvements of functional outcome measure FOSQ-10 These secondary outcome results support the efficacy of mazindol ER on cataplexy attacks, in NT1 patients, and on sleepiness, in NT1 as well as in NT2 patients. Support (if any) NLS Pharmaceutics