CATAPLEXY RESPONSE WITH FT218, A ONCE-NIGHTLY SODIUM OXYBATE: POST-HOC RESPONDER ANALYSES FROM THE PHASE 3 REST-ON CLINICAL TRIAL

TOPIC: Sleep Disorders TYPE: Original Investigations PURPOSE: Sodium oxybate is an effective treatment for multiple narcolepsy symptoms, including cataplexy. Currently approved formulations require twice-nightly dosing. Efficacy and safety of FT218, investigational, extended-release formulation once-nightly sodium (ON-SXB), were analyzed in adults with the REST-ON clinical trial (NCT02720744). ON-SXB was associated significant improvements vs placebo on coprimary endpoints Maintenance Wakefulness Test, Clinical Global Impressions Improvement, weekly cataplexy attacks at all 3 tested doses (6, 7.5, 9 g; P<0.001). This a post hoc responder analysis data from that assessed improvement mean number attacks. METHODS: 13-week, phase 3, double-blind, placebo-controlled, multicenter, randomized trial. Participants type 1 (NT1) or 2, age ≥16 years, 1:1 to receive matching once nightly before bedtime. took 4.5 g week, 6 2 weeks, 7.5 5 then efficacy assessments completed end each dosing period. For participants NT1, recorded daily sleep symptom diary. The percentage arm who experienced ≥25%, ≥50%, ≥75%, 100% reductions baseline (ie, responders) determined. Two-sided P-values calculated based Fisher's exact test. RESULTS: In total, 212 enrolled (ON-SXB, n=107; placebo, n=105). Baseline (SD) 18.9 (8.7) 19.8 (8.9) arms, respectively. Reduction observed significantly greater proportion 6-g dose (week 3; ≥25% reduction, 68.5% 40.3%, P<0.001; ≥50% 38.4% 20.8%, P<0.05; ≥75% 23.3% 4.2%, P=0.001; 2.7% 0, P=NS), 7.5-g 8; 67.1% 43.1%, 53.4% 25.0%, 32.9% 8.3%, 6.8% P<0.05), 9-g 13; 58.9% 41.7%, 49.3% 26.4%, 15.3%, P<0.01; 11.0% 2.8%, P<0.05). CONCLUSIONS: A received 6, g. Importantly, ~10% patients taking highest had complete elimination their CLINICAL IMPLICATIONS: may be narcolepsy. DISCLOSURES: Advisory Committee Member relationship Avadel Please note: current Added 05/08/2021 by Akinyemi Ajayi, source=Admin input, value=Consulting feestockholder WaterMark Medical 05/12/2021 Richard Bogan, value=dividendsstockholder Healthy Humming, LLC value=dividends Board National Foundation fee Consultant Jazz Harmony Biosciences Takeda Oventus feegrantee value=Grant/Research Supportgrantee BresoTec Bayer ldorsia value=Grant/Researchgrantee Suven Balance Vanda Merc Eisai Philips Fresca Liva Nova Roche Sommectrics Support Speaker/Speaker's Bureau value=Honoraria 05/05/2021 Bruce Corser, Merck Employee Paragon Jordan Dubow, value=Salary Abbvie Removed source=Web Response Pharmaceuticals Curry Rockefeller Group 03/08/2021 Elizabeth Guida, Response, 8/19-present 05/04/2021 David Seiden, Stock holder value=Stock 3000 04/01/2021 Michael Thorpy,