SLEEP LATENCY 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 efficacious across multiple narcolepsy symptom domains, including excessive daytime sleepiness; currently approved formulations require twice-nightly dosing. The efficacy and safety of FT218, an investigational, extended-release formulation once-nightly sodium (ON-SXB), were analyzed in adults with the pivotal REST-ON trial (NCT02720744). ON-SXB treatment was associated significant improvements vs placebo on coprimary endpoints Maintenance Wakefulness Test (MWT), Clinical Global Impression-Improvement, weekly cataplexy attacks at all 3 tested doses (6, 7.5, 9 g; P<0.001). This post hoc responder analysis from assessed improvement mean sleep latency MWT. METHODS: a 13-week, phase 3, double-blind, placebo-controlled, multicenter, randomized clinical trial. Participants age ≥16 years 1:1 to receive (4.5 g for 1 week, 6 2 weeks, 7.5 5 then weeks) or matching once nightly before bedtime. Efficacy assessments completed end each dosing period. Mean measured using MWT trials up 30 minutes each. percentage participants arm whose improved ≥5, ≥10, ≥15, ≥20 min baseline MWT=30 determined. Two-sided P-values calculated (Fisher's exact test). RESULTS: In total, 212 enrolled (ON-SXB, n=107; placebo, n=105). Baseline (SD) 5.0 (3.1) 4.7 (2.6) arms, respectively. =30 5.7% 0% (P<0.05), 10.5% 1.3% 13.2% 5.1% (P=0.14) receiving 6, A significantly greater proportion group had increased 6-g dose (week 3; ≥5 min, 57.5% 25.0%, P<0.001; ≥10 35.6% 9.1%, ≥15 18.4% 3.4%, P=0.001; 10.3% 1.1%, P<0.01), 7.5-g 8; 63.2% 28.2%, 43.4% 11.5%, 31.6% 3.8%, 15.8% 1.3%, P<0.001), 9-g 13; 66.2% 37.2%, 50.0% 19.2%, 32.4% 10.3%, P<0.01; 17.6% 6.4%, P<0.05). CONCLUSIONS: as shown by MWT; more who received than experienced ranging between min. evident early week continued improve 7.5- 8 13, CLINICAL IMPLICATIONS: sleepiness narcolepsy. DISCLOSURES: stockholder relationship WaterMark Medical Please note: current Added 05/12/2021 Richard Bogan, source=Admin input, value=dividendsstockholder Healthy Humming, LLC value=dividends Board Member National Foundation value=Consulting fee Consultant Jazz Harmony Biosciences Takeda Avadel 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 Employee Paragon 05/05/2021 Jordan Dubow, value=Salary Abbvie Removed source=Web Response Pharmaceuticals feestockholder Curry Rockefeller Group 03/08/2021 Elizabeth Guida, Response, Advisory Committee Maurice Ohayon, 8/19-present 05/04/2021 David Seiden, Stock holder value=Stock Colin Shapiro, Avedel 3000 04/01/2021 Michael Thorpy, 01/01/2020-05/01/202 04/28/2021 John Winkelman,