Avadel Pharmaceuticals plc (Nasdaq: AVDL), a company focused on transforming medicines to transform lives, today announced new and encore data supporting the clinical profile for LUMRYZ™ (sodium oxybate) for extended-release oral suspension (CIII) and patient preference for a once-nightly oxybate in 15 poster presentations and two oral presentations, at World Sleep 2023, being held from October 20-25, 2023 in Rio de Janeiro, Brazil. LUMRYZ (previously known as FT218), is a U.S. Food and Drug Administration (FDA) approved extended-release formulation of sodium oxybate indicated to be taken once at bedtime for the treatment of cataplexy or excessive daytime sleepiness (EDS) in adults with narcolepsy.

“We are excited to be at this year’s World Sleep meeting with another robust display of data that supports both the established clinical benefit of LUMRYZ,” said Jennifer Gudeman, PharmD, Senior Vice President, Medical and Clinical Affairs of Avadel. “Quality sleep is a foundational pillar of health for people with narcolepsy, yet ~65% of patients with narcolepsy experience poor, fragmented sleep in addition to their daytime symptoms.* LUMRYZ is the only FDA-approved oxybate treatment that provides clinically proven symptom improvement for excessive daytime sleepiness (EDS) or cataplexy while its novel once-at-bedtime formulation removes the need for forced awakenings to take a middle of the night oxybate dose.”

LUMRYZ has a boxed warning as a central nervous system depressant, and for its potential for abuse and misuse. LUMRYZ is available only through a restricted program under a Risk Evaluation and Mitigation Strategy called the LUMRYZ REMS. Most common adverse reactions (incidence > 5% and greater than placebo) reported for all doses of LUMRYZ combined were nausea, dizziness, enuresis, headache, and vomiting.

Highlights from the presentations include:

  • Post-hoc analyses reinforcing data from the completed pivotal Phase 3 REST-ON trial, demonstrating that treatment with LUMRYZ resulted in statistically significant and clinically meaningful improvement in EDS as measured by the Epworth Sleepiness Scale, a patient reported outcome, with the median score in the range considered normal at the end of the study.
  • A poster from the open-label RESTORE study demonstrating long-term tolerability and clinically significant improvement in symptoms, including EDS, cataplexy, nighttime awakenings and severity of narcolepsy, with LUMRYZ, providing insight into its real-world use.
  • Two posters including new post-hoc analyses that demonstrate the robust clinical efficacy of LUMRYZ and provide further insight into the improvements on measures of EDS and cataplexy compared with placebo in different demographic and clinical subgroups based on age, sex, race, body mass index, narcolepsy subtype and alerting agent use.
  • A poster presenting demographic differences between patients with narcolepsy subtypes NT1 (with cataplexy) and NT2 (without cataplexy), with data from objective measures of EDS showing identical sleep latency, comparable subjective assessments of sleep quality and refreshing nature of sleep, with some other baseline parameters suggestive of more severe disease in NT1.
  • A poster with results from a survey of people living with narcolepsy describing their significant daytime and nighttime symptoms and primary focus on treatment of daytime symptoms in discussions between clinicians and patients, highlighting the need for comprehensive discussion and understanding of the full range of symptoms experienced by people with narcolepsy.

Presentation details are as follows:

Title Session Presenter Date & Time (BRT)
Oral Presentations
Improvement in Sleep Latency With Once-Nightly Sodium Oxybate: Analysis From the Phase 3 REST-ON Clinical Trial O02 Michael J. Thorpy October 23; 09:52 - 10:05 a.m.
Sodium Oxybate Treatment Patterns in Narcolepsy Patients: A Propensity Score–Matched Cohort Study Subanalysis O02 Lois Krahn October 23; 10:05 - 10:18 a.m.
Poster Presentations
Long-Term Safety and Efficacy of Extended-Release Once-Nightly Sodium Oxybate for Narcolepsy Poster #105 Clete A. Kushida October 22; 5:00 - 6:00 p.m.
Characterization of Patients With Narcolepsy Treated vs Not Treated With Sodium Oxybate: A Propensity Score–Matched Cohort Study Poster #108 Lois Krahn October 22; 5:00 - 6:00 p.m.
Understanding Daytime and Nighttime Treatment Needs From the Patient’s Perspective: A Survey of People Living With Narcolepsy Poster #110 Luis E. Ortiz October 22; 5:00 - 6:00 p.m.
Patient Preferences and Nocturnal Experiences With Oxybate Therapy for Narcolepsy: RESTORE Study Interim Analysis Poster #112 Akinyemi O. Ajayi October 22; 5:00 - 6:00 p.m.
Cataplexy Response With Once-Nightly Sodium Oxybate: Post Hoc Responder Analysis From the Phase 3 REST-ON Clinical Trial Poster #114 Michael J. Thorpy October 22; 5:00 - 6:00 p.m.
Magnitude of Improvement in Excessive Daytime Sleepiness With the Once-at-Bedtime Oxybate for Narcolepsy Poster #109 Clete Kushida October 23; 6:00 - 7:00 p.m.
Path to Diagnosis and Impact of Narcolepsy on Quality of Life: A Survey of People Living With Narcolepsy Poster #116 Anne Marie Morse October 23; 6:00 - 7:00 p.m.
Understanding Narcolepsy Treatments From the Patient’s Perspective: A Survey of People Living With Narcolepsy Poster #117 Luis E. Ortiz October 23; 6:00 - 7:00 p.m.
Application of AASM Clinical Significance Thresholds to Once-Nightly Sodium Oxybate for Improvement in Narcolepsy Symptoms Poster #119 Yves Dauvilliers October 23; 6:00 - 7:00 p.m.
Dose Titration of Once-Nightly Sodium Oxybate:Analysis of Interim Data From RESTORE Poster #120 Jennifer Gudeman October 23; 6:00 - 7:00 p.m.
Consistent Efficacy of Once-Nightly Sodium Oxybate Regardless of Patient Demographic and Baseline Disease Characteristics Poster #123 Michael J. Thorpy October 23; 6:00 - 7:00 p.m.
Comparison of Demographics and Baseline Narcolepsy SymptomsBetween Participants With NT1 and NT2 From the Phase 3 REST-ON Clinical Trial Poster #096 Yves Dauvilliers October 24; 6:00 - 7:00 p.m.
Composite Response With Once-Nightly Sodium Oxybate: Symptom Improvement in Participants With Narcolepsy Type 1 in REST-ON Poster #103 Luis E. Ortiz October 24; 6:00 - 7:00 p.m.
Characterization of Patients Who Had ≥5% Weight Loss With Once- Nightly Sodium Oxybate: Post Hoc Analysis From REST-ON Poster #105 Anne Marie Morse October 24; 6:00 - 7:00 p.m.
Long-Term Safety of Once-Nightly Sodium Oxybate for Narcolepsy: RESTORE Study Interim Analysis of Data Poster #106 Akinyemi O. Ajayi October 24; 6:00 - 7:00 p.m.

In addition to Avadel’s oral and poster presentations, the Company will support a symposium for U.S. healthcare professionals titled “Evolving the Therapeutic Landscape for Narcolepsy” on Tuesday, October 24, 12:30 p.m. – 2:00 p.m. BRT at the Windsor Convention & Expo Center.

The symposium will feature Anne Marie Morse, D.O., Director of Child Neurology and Pediatric Sleep Medicine at Geisinger Medical Center at Janet Weis Children’s Hospital; Yves Dauvilliers, M.D., Ph.D., Director of the Sleep and Wake Disorders Centre in the Department of Neurology at the Gui de Chauliac Hospital in Montpellier, France; Michael J. Thorpy, M.D., Director of the Sleep-Wake Disorders Center at Montefiore and Professor of Neurology at Albert Einstein College of Medicine; Clete Kushida, M.D., Ph.D., Division Chief and Medical Director of Stanford Sleep Medicine, neurologist and Professor in the Department of Psychiatry and Behavioral Sciences at Stanford University Medical Center and Director of the Stanford Center for Human Sleep Research at Stanford University.

About LUMRYZ™ (sodium oxybate) for extended-release oral suspensionLUMRYZ, is an extended-release sodium oxybate medication approved by the FDA on May 1, 2023, as the first and only once-at-bedtime oxybate treatment for cataplexy or excessive daytime sleepiness (EDS) in adults with narcolepsy.  

The FDA approval of LUMRYZ was supported by results from REST-ON, a randomized, double-blind, placebo-controlled, pivotal Phase 3 trial in adults with narcolepsy. LUMRYZ demonstrated statistically significant and clinically meaningful improvements in the three co-primary endpoints: EDS, clinicians’ overall assessment of patients’ functioning (CGI-I) and cataplexy attacks, for all three evaluated doses when compared to placebo.  

With its approval, the FDA also granted seven years of Orphan Drug Exclusivity to LUMRYZ for the treatment of cataplexy or EDS in adults with narcolepsy due to a finding of clinical superiority of LUMRYZ relative to currently available oxybate treatments. In particular, the FDA found that LUMRYZ makes a major contribution to patient care over currently available, twice-nightly oxybate products by providing a once-nightly dosing regimen that avoids nocturnal arousal to take a second dose.

IMPORTANT SAFETY INFORMATION

WARNING: Taking LUMRYZ™ (sodium oxybate) with other central nervous system (CNS) depressants, such as medicines used to make you fall asleep, including opioid analgesics, benzodiazepines, sedating antidepressants, antipsychotics, sedating anti-epileptic medicines, general anesthetics, muscle relaxants, alcohol or street drugs, may cause serious medical problems, including trouble breathing (respiratory depression), low blood pressure (hypotension), changes in alertness (drowsiness), fainting (syncope) and death.
 
The active ingredient of LUMRYZ (sodium oxybate) is a form of gamma hydroxybutyrate (GHB), a controlled substance. Abuse or misuse of illegal GHB alone or with other CNS depressants (drugs that cause changes in alertness or consciousness) have caused serious side effects. These effects include seizures, trouble breathing (respiratory depression), changes in alertness (drowsiness), coma and death. Call your doctor right away if you have any of these serious side effects.
 
Because of these risks, LUMRYZ is available only by prescription and filled through certified pharmacies in the LUMRYZ REMS. You must be enrolled in the LUMRYZ REMS to receive LUMRYZ. Further information is available at www.LUMRYZREMS.com or by calling 1-877-453-1029.

INDICATIONSLUMRYZ (sodium oxybate) for extended-release oral suspension is a prescription medicine used to treat the following symptoms in adults with narcolepsy:

  • sudden onset of weak or paralyzed muscles (cataplexy)
  • excessive daytime sleepiness (EDS)

It is not known if LUMRYZ is safe and effective in people less than 18 years of age.

Do not take LUMRYZ if you take other sleep medicines or sedatives (medicines that cause sleepiness), drink alcohol or have a rare problem called succinic semialdehyde dehydrogenase deficiency.

Keep LUMRYZ in a safe place to prevent abuse and misuse. Selling or giving away LUMRYZ may harm others and is against the law. Tell your doctor if you have ever abused or been dependent on alcohol, prescription medicines or street drugs.

Anyone who takes LUMRYZ should not do anything that requires them to be fully awake or is dangerous, including driving a car, using heavy machinery or flying an airplane, for at least six (6) hours after taking LUMRYZ. Those activities should not be done until you know how LUMRYZ affects you.

Falling asleep quickly, including while standing or while getting up from the bed, has led to falls with injuries that have required some people to be hospitalized.

LUMRYZ can cause serious side effects, including the following:

  • Breathing problems, including slower breathing, trouble breathing and/or short periods of not breathing while sleeping (e.g., sleep apnea). People who already have breathing or lung problems have a higher chance of having breathing problems when they take LUMRYZ.
  • Mental health problems, including confusion, seeing or hearing things that are not real (hallucinations), unusual or disturbing thoughts (abnormal thinking), feeling anxious or upset, depression, thoughts of killing yourself or trying to kill yourself, increased tiredness, feelings of guilt or worthlessness and difficulty concentrating. Tell your doctor if you have or had depression or have tried to harm yourself. Call your doctor right away if you have symptoms of mental health problems or a change in weight or appetite.
  • Sleepwalking. Sleepwalking can cause injuries. Call your doctor if you start sleepwalking.

Tell your doctor if you are on a salt-restricted diet or if you have high blood pressure, heart failure or kidney problems. LUMRYZ contains a lot of sodium (salt) and may not be right for you.

The most common side effects of LUMRYZ in adults include nausea, dizziness, bedwetting, headache and vomiting. Your side effects may increase when you take higher doses of LUMRYZ. LUMRYZ can cause physical dependence and craving for the medicine when it is not taken as directed. These are not all the possible side effects of LUMRYZ.

For more information, ask your doctor or pharmacist. Call your doctor for medical advice about side effects.

You are encouraged to report negative side effects of prescription drugs to the FDA.

Visit www.fda.gov/medwatch, or call 1-800-FDA-1088.

Please see full Prescribing Information, including BOXED Warning.

About Avadel Pharmaceuticals plcAvadel Pharmaceuticals plc (Nasdaq: AVDL) is a biopharmaceutical company focused on transforming medicines to transform lives. Our approach includes applying innovative solutions to the development of medications that address the challenges patients face with current treatment options. Avadel’s commercial product, LUMRYZ, was approved by the U.S. Food & Drug Administration (FDA) as the first and only once-at-bedtime oxybate for the treatment of cataplexy or excessive daytime sleepiness (EDS) in adults with narcolepsy. For more information, please visit www.avadel.com.

Cautionary Disclosure Regarding Forward-Looking StatementsThis press release includes “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 future expectations, beliefs, plans, strategies, objectives, results, conditions, financial performance, prospects or other events. Such forward-looking statements include, but are not limited to, expectations regarding the potential therapeutic benefit of LUMRYZ and the potential market preference for LUMRYZ. In some cases, forward-looking statements can be identified by the use of words such as “will,” “may,” “could,” “believe,” “expect,” “look forward,” “on track,” “guidance,” “anticipate,” “estimate,” “project,” “next steps” and similar expressions and the negatives thereof (if applicable).

The Company’s forward-looking statements are based on estimates and assumptions that are made within the bounds of our knowledge of our business and operations and that we consider reasonable. However, the Company’s business and operations are subject to significant risks, and, as a result, there can be no assurance that actual results and the results of the company’s business and operations will not differ materially from the results contemplated in such forward-looking statements. Factors that could cause actual results to differ from expectations in the Company’s forward-looking statements include the risks and uncertainties described in the “Risk Factors” section of Part I, Item 1A of the Company’s Annual Report on Form 10-K for the year ended December 31, 2022, which was filed with the Securities and Exchange Commission (SEC) on March 29, 2023, and subsequent SEC filings.

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. The Company does not undertake any obligation to publicly update or revise our forward-looking statements, except as required by law.

Investor Contact:Courtney TurianoStern Investor Relations, Inc.Courtney.Turiano@sternir.com(212) 698-8687

Media Contact:Lesley StanleyReal Chemistrylestanley@realchemistry.com(609) 273-3162

References*Basseti CLA, Kallweit U, Vignatelli L, et al. European guideline and expert statements on the management of narcolepsy in adults and children. Euro J Neurol. 2012;28(9):2815-2830.

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