Cataplexy

Avadel Pharmaceuticals Provides Corporate Update and Reports Third Quarter 2023 Financial Results

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Wednesday, November 8, 2023
FDA, Policy, Narcolepsy, Food, Cataplexy, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Poster, EDS, Caregiver, Avadel Pharmaceuticals, Marketing, CVS, NDA, Patient, Shanda, Video game, Nursing, Pharmaceutical industry, Cryptocurrency

-- Management to host a conference call today at 8:30 a.m. ET --

Key Points: 
  • ET --
    DUBLIN, Ireland, Nov. 08, 2023 (GLOBE NEWSWIRE) -- Avadel Pharmaceuticals plc (Nasdaq: AVDL), a biopharmaceutical company focused on transforming medicines to transform lives, today provided a corporate update and announced its financial results for the third quarter ended September 30, 2023.
  • R&D expenses were $2.8 million in the quarter ended September 30, 2023, compared to $2.9 million for the same period in 2022.
  • SG&A expenses were $39.2 million in the quarter ended September 30, 2023, compared to $14.1 million for the same period in 2022.
  • The Company drew the first $30.0 million tranche from a $75.0 million royalty financing arrangement on August 1, 2023.

Centessa Pharmaceuticals Announces Preclinical Data Supporting ORX750’s Potential as a Best-in-Class Oral OX2R Agonist for the Treatment of Narcolepsy and Other Sleep-Wake Disorders

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Wednesday, October 25, 2023
IH, BRT, Wakefulness, Sleep, Cataplexy, IND, NT2, Human, Congress, Narcolepsy, Electromyography, DTA, Idiopathic hypersomnia, OX2R, OX1R, PK, EC50, EMG, EEG, CHO, Patient, CNTA, Pharmaceutical industry, Aroma compound, Surat Bus Rapid Transit System, NT1

BOSTON and LONDON, Oct. 25, 2023 (GLOBE NEWSWIRE) -- Centessa Pharmaceuticals plc (Nasdaq: CNTA), a clinical-stage pharmaceutical company that aims to discover and develop medicines that are transformational for patients, today announced a robust set of new preclinical data from in vivo and in vitro studies of its investigational, novel orexin receptor 2 (OX2R) agonist, ORX750, that support its potential best-in-class profile for the treatment of narcolepsy and other sleep-wake disorders.

Key Points: 
  • “The preclinical data showed ORX750 achieved maximal wake times and suppressed cataplexy at 0.1 mg/kg, the lowest oral dose tested in the DTA mouse model.
  • Notably, this activity was observed in both the DTA and Atax mouse models that recapitulate NT1 symptoms in humans.
  • Fluorescent imaging plate reader (FLIPR) assay with Chinese hamster ovary (CHO) cells stably expressing recombinant human OX1R or OX2R; OXA EC50 at hOX2R = 0.035 nM.
  • As measured by electroencephalogram (EEG) and electromyogram (EMG) with concurrent video in DTA and Atax mouse models.

Avadel Pharmaceuticals Announces Data Supporting Clinical Profile for LUMRYZ™ (sodium oxybate) for extended-release oral suspension (CIII) at World Sleep 2023

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Friday, October 20, 2023
FDA, Headache, Narcolepsy, Incidence, NT2, Food, Health, Vomiting, Dizziness, Epworth Sleepiness Scale, Cataplexy, Nausea, Poster, Senior, EDS, Disease, Enuresis, Avadel Pharmaceuticals, World Sleep Day, Sodium oxybate, Doctor of Pharmacy, Body mass index, Sleep, CIII, Mitigation, Patient, Central nervous system, Pharmaceutical industry, Dietary supplement, NT1

DUBLIN, Ireland, Oct. 20, 2023 (GLOBE NEWSWIRE) -- 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.

Key Points: 
  • “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 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.

Jazz Pharmaceuticals Presents Data Highlighting the Continued Need for Low-Sodium Treatment Option Xywav® (calcium, magnesium, potassium, and sodium oxybates) Oral Solution and Real-World, Evidence-Based Approaches for Individualized Treatment in Adults w

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Monday, October 23, 2023
MB, Jazz Pharmaceuticals, Sodium, Mercury (element), Literature, Blood pressure, Congress, Narcolepsy, MD, Observational study, American Heart Association, Cataplexy, Disease, Idiopathic hypersomnia, Systematic review, Gamma-Hydroxybutyric acid, Sleep, Magnesium, American Heart, Risk, Somnolence, Colorado Ultraviolet Transit Experiment, Patient, Calcium, MRCPCH, Potassium, Medicine, Diagnosis, Cardiovascular disease, Mercury, Salt, Pharmaceutical industry, Person, Sodium oxybate, Xywav

DUBLIN, Oct. 23, 2023 /PRNewswire/ -- Jazz Pharmaceuticals plc (Nasdaq: JAZZ) today announced new data that examine the comorbid risk of cardiovascular disease in patients with narcolepsy and idiopathic hypersomnia as well as the effect of sodium intake on cardiovascular health. The Company will also present results from the real-world TENOR study on individualized dosing regimens for adults with type 1 or type 2 narcolepsy transitioning to low-sodium Xywav® (calcium, magnesium, potassium, and sodium oxybates) oral solution from high-sodium Xyrem® (sodium oxybate) oral solution. The study will be presented today at the World Sleep 2023 Congress in an oral session on evidence-based approaches for optimizing pharmacologic treatment for narcolepsy.

Key Points: 
  • The Company will also present results from the real-world TENOR study on individualized dosing regimens for adults with type 1 or type 2 narcolepsy transitioning to low-sodium Xywav® (calcium, magnesium, potassium, and sodium oxybates) oral solution from high-sodium Xyrem® (sodium oxybate) oral solution.
  • The study will be presented today at the World Sleep 2023 Congress in an oral session on evidence-based approaches for optimizing pharmacologic treatment for narcolepsy.
  • "Real-world data regarding individualized dosing regimens for the treatment of narcolepsy are limited.
  • The TENOR study was a patient-centric, prospective, observational study of U.S. adults with type 1 or type 2 narcolepsy.

Alkermes to Present First Clinical Data for Orexin 2 Receptor Agonist ALKS 2680 at World Sleep Congress

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Tuesday, October 10, 2023
Congress, Neuroscience, BRT, Pharmacokinetics, Idiopathic hypersomnia, Cataplexy, Risk factor, ALKS, Safety, Wakefulness, Risk, Signs and symptoms, Model, OX2R, Investigational New Drug, Diagnosis, Pharmaceutical industry, Alkermes, Narcolepsy, Patient

DUBLIN, Oct. 10, 2023 /PRNewswire/ -- Alkermes plc (Nasdaq: ALKS) today announced plans to present new data related to ALKS 2680, the company's novel, investigational orexin 2 receptor (OX2R) agonist in development for the treatment of narcolepsy, at World Sleep Congress, taking place Oct. 20-25, 2023, in Rio de Janeiro. In an oral presentation, the company will present initial data from its ongoing phase 1 study evaluating the safety, tolerability, pharmacokinetics and pharmacodynamics of ALKS 2680 in healthy volunteers and patients with narcolepsy or idiopathic hypersomnia. This presentation will include initial clinical proof-of-concept data from the first cohort of four patients with narcolepsy type 1.

Key Points: 
  • — Oral Presentation to Include Initial Clinical Proof-of-Concept Data in Patients With Narcolepsy Type 1 for ALKS 2680, the Company's Investigational Orexin 2 Receptor Agonist —
    DUBLIN, Oct. 10, 2023 /PRNewswire/ -- Alkermes plc (Nasdaq: ALKS) today announced plans to present new data related to ALKS 2680, the company's novel, investigational orexin 2 receptor (OX2R) agonist in development for the treatment of narcolepsy, at World Sleep Congress, taking place Oct. 20-25, 2023, in Rio de Janeiro.
  • In an oral presentation, the company will present initial data from its ongoing phase 1 study evaluating the safety, tolerability, pharmacokinetics and pharmacodynamics of ALKS 2680 in healthy volunteers and patients with narcolepsy or idiopathic hypersomnia.
  • This presentation will include initial clinical proof-of-concept data from the first cohort of four patients with narcolepsy type 1.
  • We look forward to presenting data at the World Sleep Congress, including our first clinical data for ALKS 2680 in patients with narcolepsy."

Alkermes to Present First Clinical Data for Orexin 2 Receptor Agonist ALKS 2680 at World Sleep Congress

Retrieved on: 
Tuesday, October 10, 2023
Congress, Neuroscience, BRT, Pharmacokinetics, Idiopathic hypersomnia, Cataplexy, Risk factor, ALKS, Safety, Wakefulness, Risk, Signs and symptoms, Model, OX2R, Investigational New Drug, Diagnosis, Pharmaceutical industry, Alkermes, Narcolepsy, Patient

DUBLIN, Oct. 10, 2023 /PRNewswire/ -- Alkermes plc (Nasdaq: ALKS) today announced plans to present new data related to ALKS 2680, the company's novel, investigational orexin 2 receptor (OX2R) agonist in development for the treatment of narcolepsy, at World Sleep Congress, taking place Oct. 20-25, 2023, in Rio de Janeiro. In an oral presentation, the company will present initial data from its ongoing phase 1 study evaluating the safety, tolerability, pharmacokinetics and pharmacodynamics of ALKS 2680 in healthy volunteers and patients with narcolepsy or idiopathic hypersomnia. This presentation will include initial clinical proof-of-concept data from the first cohort of four patients with narcolepsy type 1.

Key Points: 
  • — Oral Presentation to Include Initial Clinical Proof-of-Concept Data in Patients With Narcolepsy Type 1 for ALKS 2680, the Company's Investigational Orexin 2 Receptor Agonist —
    DUBLIN, Oct. 10, 2023 /PRNewswire/ -- Alkermes plc (Nasdaq: ALKS) today announced plans to present new data related to ALKS 2680, the company's novel, investigational orexin 2 receptor (OX2R) agonist in development for the treatment of narcolepsy, at World Sleep Congress, taking place Oct. 20-25, 2023, in Rio de Janeiro.
  • In an oral presentation, the company will present initial data from its ongoing phase 1 study evaluating the safety, tolerability, pharmacokinetics and pharmacodynamics of ALKS 2680 in healthy volunteers and patients with narcolepsy or idiopathic hypersomnia.
  • This presentation will include initial clinical proof-of-concept data from the first cohort of four patients with narcolepsy type 1.
  • We look forward to presenting data at the World Sleep Congress, including our first clinical data for ALKS 2680 in patients with narcolepsy."

Avadel Pharmaceuticals Provides Corporate Update and Reports Second Quarter 2023 Financial Results

Retrieved on: 
Wednesday, August 9, 2023
Sodium oxybate, Cataplexy, NT2, Pharmacy, EDS, Narcolepsy, Marketing, Anxiety, Sleep, Sleep Research Society, American depositary receipt, NDA, Prescription, American Academy, U.S. FDA, Avadel Pharmaceuticals, American Academy of Sleep Medicine, Patient, NT1, ESI, Somnolence, Growth, FDA, Health, Pharmaceutical industry, Nursing, Fine chemical

DUBLIN, Ireland, Aug. 09, 2023 (GLOBE NEWSWIRE) -- Avadel Pharmaceuticals plc (Nasdaq: AVDL), a biopharmaceutical company focused on transforming medicines to transform lives, today provided a corporate update and announced its financial results for the second quarter ended June 30, 2023.

Key Points: 
  • ET --
    DUBLIN, Ireland, Aug. 09, 2023 (GLOBE NEWSWIRE) -- Avadel Pharmaceuticals plc (Nasdaq: AVDL), a biopharmaceutical company focused on transforming medicines to transform lives, today provided a corporate update and announced its financial results for the second quarter ended June 30, 2023.
  • Recognized $1.5 million in net product revenues for the second quarter 2023.
  • R&D expenses were $4.2 million in the quarter ended June 30, 2023, compared to $4.5 million for the same period in 2022.
  • SG&A expenses were $46.8 million in the quarter ended June 30, 2023, compared to $21.8 million for the same period in 2022.

The New England Journal of Medicine Publishes Data from Phase 2 Study of First Oral Orexin Receptor 2 Agonist TAK-994 in Patients with Narcolepsy Type 1

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Wednesday, July 26, 2023
Health, Other Health, Clinical Trials, General Health, Pharmaceutical, Biotechnology, Hepatotoxicity, Cataplexy, The New England Journal of Medicine, TAK, Narcolepsy, Wakefulness, Patient, OX2R, Somnolence, Disease, Pharmaceutical industry, Takeda, NT1

Takeda ( TSE:4502/NYSE:TAK ) today announced that data from the Phase 2 study of orexin receptor 2 (OX2R) agonist TAK-994 in patients with narcolepsy type 1 (NT1) was published in The New England Journal of Medicine.

Key Points: 
  • Takeda ( TSE:4502/NYSE:TAK ) today announced that data from the Phase 2 study of orexin receptor 2 (OX2R) agonist TAK-994 in patients with narcolepsy type 1 (NT1) was published in The New England Journal of Medicine.
  • These data represent the first-ever clinical data to be published of an oral orexin agonist in NT1.
  • The article appears in the July 27 issue of the journal and is titled, “Oral Orexin Receptor 2 Agonist in Narcolepsy Type 1.”
    “There is significant unmet need for patients with narcolepsy, a chronic neurologic disease caused by severe loss or absence of the neuropeptide orexin, also known as hypocretin.
  • Today, most patients with NT1 require multiple therapies that only manage excessive daytime sleepiness and cataplexy.

HARMONY BIOSCIENCES ANNOUNCES PLANS TO INITIATE PHASE 3 REGISTRATIONAL STUDY OF PITOLISANT IN PRADER-WILLI SYNDROME FOLLOWING POSITIVE END-OF-PHASE 2 MEETING WITH THE U.S. FOOD AND DRUG ADMINISTRATION

Retrieved on: 
Thursday, July 20, 2023
Prader–Willi syndrome, Cataplexy, Narcolepsy, EDS, Pitolisant, DSM-IV codes, Harmony, MD, Patient, Somnolence, PWS, FDA, Safety, Pharmaceutical industry

Harmony plans to initiate a Phase 3 registrational study in the fourth quarter of 2023.

Key Points: 
  • Harmony plans to initiate a Phase 3 registrational study in the fourth quarter of 2023.
  • Based on a positive signal observed from the Phase 2 proof-of-concept study, Harmony aligned with the FDA on the proposed Phase 3 study design elements to support further investigation of pitolisant for children, adolescents and adults with PWS experiencing EDS.
  • Pitolisant is marketed as WAKIX® in the U.S. and is FDA approved to treat EDS or cataplexy in adult patients with narcolepsy.
  • Pitolisant is not approved for use in patients with PWS and is currently being evaluated as an investigational agent in this patient population.

China NMPA approves RareStone's pitolisant (Wakix) for the treatment of narcolepsy

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Tuesday, July 4, 2023
Narcolepsy, Cataplexy, EDS, Sleep paralysis, European Medicines Agency, Pitolisant, Approximation error, Government, EMA, Pharmacotherapy, Hypnagogia, Patient, Peking University, Diagnosis, FDA, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, NMPA, Food, Disease, Pharmaceutical industry

SHANGHAI, July 4, 2023 /PRNewswire/ -- RareStone Group, a rare disease-focused company aiming to establish the first rare disease ecosystem in China, announced that on June 30, the Chinese National Medical Products Administration (NMPA)  approved pitolisant (Wakix) for the treatment of excessive daytime sleepiness (EDS) or cataplexy in adult patients with narcolepsy. In China, pitolisant is the first approved innovative drug for narcolepsy and the only non-scheduled drug with narcolepsy indication.

Key Points: 
  • -- pitolisant is the first and only treatment drug approved in mainland China for narcolepsy.
  • Pitolisant has been listed in the Chinese guidelines for diagnosis and treatment for narcolepsy (2022) as a first-line treatment therapy.
  • According to , published by CHARD & Peking University, more than half of the narcolepsy patients in China have experienced misdiagnosis.
  • The fruitful and positive collaboration contributes a lot to bring this promising treatment drug to narcolepsy patients in China.