Abstract Details

Title Long-term Safety and Efficacy of Extended-release Once-nightly Sodium Oxybate for Narcolepsy in Patients not Currently Taking an Oxybate

Topic Sleep

Presentation(s) P6 - Poster Session 6 (5:00 PM-6:00 PM)

Poster/Presentation
Number 14-004

Objective To evaluate safety/efficacy data of once-nightly sodium oxybate (ON-SXB) in the long-term, open-label RESTORE (NCT04451668) study for participants who were not taking an oxybate at study entry.

Background Treatment with ON-SXB met primary/secondary efficacy endpoints for excessive daytime sleepiness, cataplexy, overall condition, and disrupted nighttime sleep, with a safety profile consistent with twice-nightly oxybates (TN-OXB), in the phase 3 REST-ON trial.

Design/Methods Participants were aged ≥16 years with narcolepsy who completed REST-ON, were oxybate-naive, or switched from stable (≥1 mo) TN-OXB. If not taking oxybate at entry, ON-SXB was initiated at 4.5 g/night; doses were adjusted by ±1.5 g/night weekly as needed up to 9 g/night. Adverse drug reactions (ADRs; ie, adverse events [AEs] related to ON-SXB) were collected. Epworth Sleepiness Scale (ESS), daily diary (weekly cataplexy episodes, nighttime awakenings), and Clinical and Patient Global Impression of Improvement (CGI-I; PGI-I) data were collected for participants not taking an oxybate at study entry.

Results REST-ON completers/oxybate-naive participants (N=50) were mean age 32.7 (range, 16-72) years; 60% were female and 80% were white. Median ON-SXB exposure was 408.1 (range, 8-1098) days; 8 participants discontinued owing to an AE. Most common ADRs (≥4 participants) included nausea (24%), somnolence (12%), dizziness (10%), paresthesia (8%), and tremor (8%). In the modified intent-to-treat (mITT) population (n=47), baseline mean (SD) ESS score was 14.4 (4.5); least squares mean (LSM) change from baseline (CFB) was -7.9 (95% CI, -10.1, -5.7; P<0.0001). Cataplexy episodes were significantly reduced (mITT, n=31; baseline mean [SD], 13.4 [10.1]; LSM CFB, -8.3 [95% CI, -12.1, -4.6; P=0.0003]). Nighttime awakenings were significantly reduced (baseline mean [SD], 15.8 [10.9]; LSM CFB, -7.2 [95% CI, -9.8, -4.7; P<0.0001]). Overall condition improved for most participants (CGI-I, 80%; PGI-I, 80%).

Conclusions

In participants not taking an oxybate at study entry, ON-SXB treatment was well tolerated and led to significant improvements across narcolepsy symptoms.

Authors/Disclosures
Michael Sangobowale, PhD (Eisai) PRESENTER	Dr. Sangobowale has received personal compensation for serving as an employee of Avadel Pharmaceuticals .
Bruce C. Corser, MD	Dr. Corser has received personal compensation in the range of $500-$4,999 for serving on a Speakers Bureau for Axsome. Dr. Corser has received personal compensation in the range of $10,000-$49,999 for serving as an Expert Witness for Avadel. The institution of Dr. Corser has received research support from Alkermes, . The institution of Dr. Corser has received research support from Apnimed. The institution of Dr. Corser has received research support from Avadel. The institution of Dr. Corser has received research support from Axsome. The institution of Dr. Corser has received research support from Eisai. The institution of Dr. Corser has received research support from Eli Lilly. The institution of Dr. Corser has received research support from Harmony. The institution of Dr. Corser has received research support from Incannex. The institution of Dr. Corser has received research support from Jazz. The institution of Dr. Corser has received research support from Mineralys. The institution of Dr. Corser has received research support from Suven. The institution of Dr. Corser has received research support from Takeda. The institution of Dr. Corser has received research support from Centessa.
Anne M. Morse, DO (Geisinger Medical Center)	Dr. Morse has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Jazz Pharmaceuticals. Dr. Morse has received personal compensation in the range of $50,000-$99,999 for serving as a Consultant for Avadel. Dr. Morse has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Harmony Biosciences. Dr. Morse has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Alkermes. Dr. Morse has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Takeda. Dr. Morse has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Eisai. Dr. Morse has received personal compensation in the range of $10,000-$49,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Jazz Pharmaceuticals. Dr. Morse has received personal compensation in the range of $10,000-$49,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Avadel. Dr. Morse has received personal compensation in the range of $10,000-$49,999 for serving on a Speakers Bureau for Avadel. Dr. Morse has received personal compensation in the range of $10,000-$49,999 for serving on a Speakers Bureau for Harmony biosciences. Dr. Morse has received personal compensation in the range of $100,000-$499,999 for serving as an Expert Witness for Hayes solicitors. Dr. Morse has received publishing royalties from a publication relating to health care. Dr. Morse has received personal compensation in the range of $100,000-$499,999 for serving as a CEO with DAMM Good Sleep, LLC.
Thomas P. Stern, MD	The institution of Dr. Stern has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Avadel. The institution of Dr. Stern has received personal compensation in the range of $5,000-$9,999 for serving on a Speakers Bureau for Avadel. Dr. Stern has received personal compensation in the range of $10,000-$49,999 for serving as an Expert Witness for Avadel. The institution of Dr. Stern has received research support from Avadel.
Akinyemi Ajayi, MD	Dr. Ajayi has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Jazz pharmaceuticals. Dr. Ajayi has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for avadel. Dr. Ajayi has received personal compensation in the range of $500-$4,999 for serving as a Consultant for harmony biosciences. Dr. Ajayi has received personal compensation in the range of $10,000-$49,999 for serving on a Scientific Advisory or Data Safety Monitoring board for jazz pharmaceuticals. Dr. Ajayi has received personal compensation in the range of $10,000-$49,999 for serving on a Scientific Advisory or Data Safety Monitoring board for avadel . Dr. Ajayi has received personal compensation in the range of $10,000-$49,999 for serving on a Speakers Bureau for jazz pharmaceuticals. Dr. Ajayi has received personal compensation in the range of $10,000-$49,999 for serving on a Speakers Bureau for avadel. Dr. Ajayi has stock in avadel.
Clete A. Kushida, MD, PhD (Stanford Sleep Medicine Center)	Dr. Kushida has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Avadel. Dr. Kushida has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Genentech. Dr. Kushida has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for XW Pharma, Inc.. Dr. Kushida has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Samsung. Dr. Kushida has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Zephyr Sleep Technologies. Dr. Kushida has received personal compensation in the range of $10,000-$49,999 for serving as a Consultant for Vivos. Dr. Kushida has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Cerebra. Dr. Kushida has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for Merck & Co., Inc.. Dr. Kushida has received personal compensation in the range of $5,000-$9,999 for serving as an Expert Witness for State of Arizona. Dr. Kushida has received personal compensation in the range of $500-$4,999 for serving as an Expert Witness for Browne George Ross, LLP. The institution of Dr. Kushida has received research support from AmCAD, Philips-Respironics, Asate, Syneos Health, Inspire. Dr. Kushida has received publishing royalties from a publication relating to health care.
Brian Abaluck, MD	Dr. Abaluck has received personal compensation for serving as an employee of Avadel Pharmaceuticals.
Jennifer Gudeman (Avadel Pharmaceuticals)	Miss Gudeman has received personal compensation for serving as an employee of Avadel Pharmaceuticals. Miss Gudeman has stock in Avadel Pharmaceuticals.

��ɫ��

��ɫ��