Avadel Pharmaceuticals Announces Publication of Positive Secondary Endpoint Data from Pivotal Phase 3 REST-ON Trial

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Avadel Pharmaceuticals plc

  • FT218 once at bedtime demonstrated clinically significant improvement in ratings of disturbed nocturnal sleep compared to placebo in adults with narcolepsy

  • New Data Reinforce Positive Primary Endpoint Data from Completed Phase 3 REST-ON Trial and Indicate FT218 Positively Impacts Daytime and Nighttime Narcolepsy Symptoms

DUBLIN, Ireland, April 06, 2022 (GLOBE NEWSWIRE) — Avadel Pharmaceuticals plc (Nasdaq: AVDL), a biopharmaceutical company focused on transforming medicines to transform lives, today announced the release of positive endpoint data secondary assessment of its pivotal phase 3 REST-ON trial of FT218 in CNS drugs, a peer-reviewed medical journal focused on the treatment of psychiatric and neurological disorders. The article, titled “Effect of FT218, a once-a-night formulation of sodium oxybate, on disturbed nocturnal sleep in patients with narcolepsy: results from the randomized phase 3 REST-ON trial”, is accessible at https://link.springer. com/article/10.1007/s40263-022-00904-6. FT218, also referred to as ON-SXB in Avadel’s scientific publications, is the Company’s lead drug candidate, an investigational formulation of sodium oxybate designed to be taken once at bedtime for the treatment of excessive daytime sleepiness. (EDS) or cataplexy in adults with narcolepsy.

It is estimated that around 65% of people with narcolepsy suffer from nighttime sleep disturbances.1 Although people with narcolepsy can fall asleep easily, their sleep tends to include frequent awakenings and stage changes and is often not refreshing. Most narcolepsy treatments are taken in the morning to fight EDS; only oxybate treatments are taken at bedtime, with current immediate-release formulations being taken twice a night. To assess the potential for improvement in sleep architecture with a single bedtime dose of FT218, secondary endpoints of the double-blind, placebo-controlled REST-ON trial were evaluated, including measures polysomnographic studies of sleep stage changes and nocturnal awakenings. In addition, patient-reported ratings of sleep quality and refreshing nature of sleep, as measured on a visual analog scale, were analyzed. Here are the highlights of the analyses:

  • At all doses tested (6, 7.5, and 9 g), FT218 once at bedtime demonstrated a statistically significant decrease in the number of transitions from N1, N2, N3 and rapid eye movement (REM) sleep to awakening and from N2, N3 and paradoxical sleep to N1 (P

  • The quality of sleep and the refreshing nature of sleep were significantly improved with all doses evaluated compared to placebo (P

  • A post-hoc analysis showed that significant improvements in DNS were observed independent of concomitant stimulant use.

  • Additional post-hoc data also supported improvements in sleep architecture with increases in N3, or slow wave sleep, and increased REM latency.

“DNS is a common, bothersome, but often minimized symptom of narcolepsy,” said Thomas Roth, Ph.D., lead author and director of the Sleep Disorders and Research Center at Henry Ford Hospital. “These data show that the FT218 improved both objective and subjective ratings of DNS, as measured by polysomnographic recordings and participants’ own ratings. These results will be relevant for the sleep medicine community to consider in treatment selection, if FT218 is approved. »

Key safety and efficacy results for REST-ON were published in August 2020 in SLEEP by Kushida et al. The most common adverse events (incidence >5% and greater than placebo) in patients receiving FT218 were nausea, dizziness, enuresis, headache and vomiting. Additional secondary and post-hoc data were presented at SLEEP (the annual meeting of the Associated Sleep Professional Societies) and World Sleep.

“While treatment for narcolepsy often focuses on relieving daytime symptoms, such as EDS, these findings underscore the importance of a potential treatment option that also addresses nighttime symptoms,” said Douglas Williamson, MD, Avadel’s medical director. “This peer-reviewed publication marks the first time improvements in sleep quality, refreshing nature of sleep, and DNS have been demonstrated with oxybate in a segment of uninterrupted consolidated sleep. The body of evidence for FT218 once at bedtime, including previously published results demonstrating its positive impact on EDS and cataplexy, reinforces our confidence in its potential, if approved, as a major breakthrough for the treatment of narcolepsy.

About Narcolepsy
Narcolepsy is a chronic neurological disease that impairs the brain’s ability to regulate the sleep-wake cycle. The condition affects approximately one in 2,000 people in the United States with the cardinal symptom of EDS. Additional symptoms may vary from person to person and may include DNS, sudden loss of muscle tone usually triggered by strong emotion (cataplexy), sleep paralysis, and hypnagogic hallucinations.

About FT218
FT218 is an investigational formulation of sodium oxybate leveraging our proprietary drug delivery technology and designed to be taken once at bedtime for the treatment of EDS or cataplexy in adults with narcolepsy.

In March 2020, Avadel completed the REST-ON trial, a pivotal Phase 3 randomized, double-blind, placebo-controlled trial to evaluate the efficacy and safety of FT218 in adults with narcolepsy. Of the three co-primary endpoints, FT218 demonstrated statistically significant and clinically meaningful results in EDS, clinician’s global assessment of patient functioning, and reduction in cataplexy attacks for all three doses assessed by compared to placebo.

In January 2018, the FDA granted FT218 Orphan Drug Designation for the treatment of narcolepsy based on the plausible hypothesis that FT218 may be clinically superior to the twice-nightly formulation of sodium oxybate already approved by the FDA for people with narcolepsy due to consequences of middle-of-the-night dosing of the approved product. A marketing application for FT218 is currently under review by the FDA.

Avadel is currently evaluating the long-term safety and tolerability of FT218 in the RESTORE open-label clinical study. For more information, visit: www.restore-narcolepsy-study.com.

About Avadel Pharmaceuticals plc
Avadel Pharmaceuticals plc (Nasdaq: AVDL) is a biopharmaceutical company focused on transforming medicines to transform lives. Our approach includes applying innovative solutions to drug development that address the challenges patients face with current treatment options. Our current lead drug candidate, FT218, is an investigational formulation of sodium oxybate leveraging our proprietary drug delivery technology and designed to be taken once at bedtime for the treatment of EDS and cataplexy. in adults with narcolepsy. For more information, visit www.avadel.com.

Caution Regarding Forward-Looking Statements
This press release contains “forward-looking statements” within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934. These forward-looking statements relate to our expectations, beliefs, plans, strategies, objectives, results, conditions, financial performance, outlook or other events. These forward-looking statements include, but are not limited to, our expectations regarding the therapeutic benefits and tolerability of FT218, if approved. In some cases, forward-looking statements can be identified by the use of words such as “will”, “may”, “could”, “believe”, “expect”, “look into the future”, ” on the right track”, “orientation”. “, “anticipate”, “estimate”, “project”, “next steps” and similar expressions, as well as their negative aspects (where applicable).

Our forward-looking statements are based on estimates and assumptions that are made within the limits of our knowledge of our business and operations and that we believe to be reasonable. However, our business and operations are subject to significant risks and, accordingly, there can be no assurance that the actual results and results of our business and operations will not differ materially from the results contemplated in these forward-looking statements. Factors that could cause actual results to differ from expectations in our forward-looking statements include the risks and uncertainties described in the “Risk Factors” section of Part I, Item 1A of our Annual Report on Form 10-K for the fiscal year ended December 31. , 2021, which we filed with the Securities and Exchange Commission on March 16, 2022, and subsequent filings with the SEC.

Forward-looking statements speak only as of the date they are made and are not guarantees of future performance. Accordingly, you should not place undue reliance on forward-looking statements. We undertake no obligation to publicly update or revise our forward-looking statements, except as required by law.

Investor contacts:
Courtney Turiano
Stern Investor Relations, Inc.
[email protected]
(212) 698-8687

Media Contact:
Nicole Raisch Goelz
real chemistry
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(408) 568-4292

The references:

  1. Basseti et al. European guidelines and expert opinions on the management of narcolepsy in adults and children. Eur J Neurol. 2021 Sep;28(9):2815-2830.

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