Cataplexy

Axsome Therapeutics Announces AXS-12 Achieves Primary Endpoint in the SYMPHONY Phase 3 Trial in Narcolepsy

Retrieved on: 
Monday, March 25, 2024
Diagnosis, Neurology, Week, Narcolepsy, Somnolence, Memory, Cataplexy, Central nervous system, Patient, CNS, Montefiore Medical Center, EDS, Concentrate, SYMPHONY, Epworth Sleepiness Scale, Quality of life, Therapy, Cognition, Concurrency, ESS, Reboxetine, Pharmaceutical industry

AXS-12 also reduced excessive daytime sleepiness (EDS) severity, improved cognitive function, and reduced overall narcolepsy severity as compared to placebo.

Key Points: 
  • AXS-12 also reduced excessive daytime sleepiness (EDS) severity, improved cognitive function, and reduced overall narcolepsy severity as compared to placebo.
  • SYMPHONY was a Phase 3 multicenter, randomized, double-blind, placebo-controlled trial in which 90 patients with a diagnosis of narcolepsy with cataplexy were randomized to treatment with AXS-12 or placebo for 5 weeks.
  • “The SYMPHONY Phase 3 trial results confirm the promise and potential of AXS-12 for the treatment of narcolepsy,” said Dr. Herriot Tabuteau, CEO of Axsome Therapeutics.
  • Axsome will host a conference call and webcast today at 8:00 AM Eastern to discuss the topline SYMPHONY study results in narcolepsy.

Axsome Therapeutics Announces Results of CRESCENDO Narcolepsy Patient Survey Demonstrating Unmet Needs in Treated Narcolepsy Type 1 Patients

Retrieved on: 
Monday, March 25, 2024
Diagnosis, Neurology, Sleep, Depression, Somnolence, Deficit spending, Thomas Jefferson University, Partnership, Central nervous system, NT1, Cataplexy, Patient, CNS, Sleep disorder, Cognitive deficit, Ageing, EDS, Columbia, Anxiety, Hallucination, Narcolepsy Network, Epworth Sleepiness Scale, Life, Therapy, Sleep paralysis, ESS, Medical director, Idiopathic hypersomnia, FDA, Human, Nursing

NEW YORK, March 25, 2024 (GLOBE NEWSWIRE) -- Axsome Therapeutics, Inc. (NASDAQ: AXSM), a biopharmaceutical company developing and delivering novel therapies for the management of central nervous system (CNS) disorders, today announced topline results from the CRESCENDO (Characterizing Patient Perspectives on Unmet Needs in Narcolepsy) survey of patients with narcolepsy type 1 (NT1, i.e., narcolepsy with cataplexy) receiving treatment, demonstrating high rates of persistent symptoms and significant patient burden, despite being on current treatments. CRESCENDO was conducted in partnership with Narcolepsy Network, a national non-profit patient support organization for people with narcolepsy, idiopathic hypersomnia, and related sleep disorders.

Key Points: 
  • CRESCENDO was conducted in partnership with Narcolepsy Network, a national non-profit patient support organization for people with narcolepsy, idiopathic hypersomnia, and related sleep disorders.
  • All patients taking part in the survey were currently undergoing treatment for NT1.
  • The most common treatments were wake promoting agents (about 53% of surveyed patients), oxybates (47%), and stimulants (42%).
  • Axsome plans to present the detailed results of the CRESCENDO survey at upcoming scientific meetings.

HARMONY BIOSCIENCES INITIATES GLOBAL PHASE 3 REGISTRATIONAL TRIAL (TEMPO STUDY) OF PITOLISANT IN PATIENTS WITH PRADER-WILLI SYNDROME

Retrieved on: 
Wednesday, April 3, 2024
ODD, Narcolepsy, Prader–Willi syndrome, TEMPO, Cataplexy, Patient, Ageing, EDS, Safety, Harmony, Plasma protein binding, FDA, PWS, Pitolisant, Pharmaceutical industry

Approximately 15,000 – 20,000 people in the U.S. are living with PWS, the majority experiencing behavioral symptoms and more than half with EDS.

Key Points: 
  • Approximately 15,000 – 20,000 people in the U.S. are living with PWS, the majority experiencing behavioral symptoms and more than half with EDS.
  • "Our progress in expanding the clinical applications of pitolisant in PWS and in other rare diseases, remains promising.
  • 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.

Avadel Pharmaceuticals Provides Corporate Update and Reports Fourth Quarter and Full Year 2023 Financial Results

Retrieved on: 
Monday, March 4, 2024
Idiopathic hypersomnia, Caregiver, Narcolepsy, Marketing, FDA, Food, System, Shanda, Cataplexy, UnitedHealth Group, Food and Drug Administration, Webcast, EDS, Patient, PBM, Avadel Pharmaceuticals, Trial of the century, Nursing

-- Management to host a conference call today at 7:30 a.m. ET --

Key Points: 
  • ET --
    DUBLIN, Ireland, March 04, 2024 (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 fourth quarter ended December 31, 2023.
  • “We are carrying significant momentum into 2024 following the successful launch of LUMRYZ and are pleased with the strong early launch results we have seen.
  • Recognized $19.5 million and $28.0 million in net product revenue for the quarter and year ended December 31, 2023, respectively.
  • Net product revenue consists of LUMRYZ product sales, which was launched in the U.S. on June 5, 2023.

Global Narcolepsy Drugs Market Research Report 2024: Pipeline Analysis, Key Company Profiles and Recent Developments 2023-2030 - ResearchAndMarkets.com

Retrieved on: 
Tuesday, January 30, 2024
General Health, Pharmaceutical, Health, Narcolepsy, Jazz Pharmaceuticals, CIII, FDA, Central nervous system, Somnolence, Consciousness, Quality of life, DSM-IV codes, Mazindol, Takeda Pharmaceutical Company, Tobacco, Organization, Cataplexy, EDS, Prevalence, American Academy, CNS, Selective serotonin reuptake inhibitor, Growth, Patient, Diagnosis, Marketing, Disease, U.S. FDA, NINDS, Research, Therapy, Teva Pharmaceuticals, Investment, Avadel Pharmaceuticals, European Medicines Agency, Pharmaceutical industry

The "Global Narcolepsy Drugs Market (By Therapeutic Type, Disease Type, End User, Regional Analysis), Pipeline Analysis, Key Company Profiles and Recent Developments - Forecast to 2030" report has been added to ResearchAndMarkets.com's offering.

Key Points: 
  • The "Global Narcolepsy Drugs Market (By Therapeutic Type, Disease Type, End User, Regional Analysis), Pipeline Analysis, Key Company Profiles and Recent Developments - Forecast to 2030" report has been added to ResearchAndMarkets.com's offering.
  • The global narcolepsy drugs market has witnessed significant growth in recent years owing to the rising prevalence of narcolepsy and increased awareness about the condition.
  • Latin America and Middle East & Africa are competing closely to grab the maximum share of the global narcolepsy drugs market.
  • Global Narcolepsy Drugs Market - Key Company Profiles, Recent Developments, Financial Insights

Human medicines European public assessment report (EPAR): Wakix, pitolisant, Date of authorisation: 31/03/2016, Revision: 16, Status: Authorised

Retrieved on: 
Tuesday, January 2, 2024
Medical Subject Headings, Marketing, UNS, INN, Neuron, Fatigue, Heartburn, Woman, MWT, Sleep, ATC, Cataplexy, Insomnia, Sleep disorder, Depression, International, Tremor, Epworth Sleepiness Scale, Headache, Wakefulness, ESS, Nausea, Language, Brain, Muscle weakness, Mouth, Dizziness, Vertigo, Breakfast, Anxiety, Irritability, Observational study, European Medicines Agency, Indigestion, Tablet, Histamine, Select, Patient, Safety, Anatomy, Medicine, Somnolence, Vomiting, IIA, Pharmaceutical industry

Human medicines European public assessment report (EPAR): Wakix, pitolisant, Date of authorisation: 31/03/2016, Revision: 16, Status: Authorised

Key Points: 


Human medicines European public assessment report (EPAR): Wakix, pitolisant, Date of authorisation: 31/03/2016, Revision: 16, Status: Authorised

Narcolepsy Drugs Market, Size, Global Forecast 2024-2030: Industry Trends, Share, Growth, Insight, Impact of Inflation, Company Analysis - ResearchAndMarkets.com

Retrieved on: 
Friday, December 29, 2023
Health, Pharmaceutical, Novartis, Teva Pharmaceuticals, Quality of life, Sodium oxybate, Food, Risk, Drug development, Takeda Pharmaceutical Company, Roche, Research, Cataplexy, FDA, Instability, Prevalence, NINDS, Incidence, Mazindol, GHB, Stimulant, EDS, NMPA, Narcolepsy, Tobacco, DSM-IV codes, Investment, Patient, Diagnosis, Organization, Somnolence, Neurological disorder, Pharmaceutical industry

The "Narcolepsy Drugs Market, Size, Global Forecast 2024-2030, Industry Trends, Share, Growth, Insight, Impact of Inflation, Company Analysis" report has been added to ResearchAndMarkets.com's offering.

Key Points: 
  • The "Narcolepsy Drugs Market, Size, Global Forecast 2024-2030, Industry Trends, Share, Growth, Insight, Impact of Inflation, Company Analysis" report has been added to ResearchAndMarkets.com's offering.
  • The Global Narcolepsy Drugs Market is poised for substantial growth, with a projected value of US$5.95 billion by 2030
    Several factors contribute to the expansion of the narcolepsy drugs market.
  • The Narcolepsy Drugs Market is expected to grow steadily, with a compound annual growth rate (CAGR) of 8.01% from 2023 to 2030.
  • Combined, these elements make the U.S. narcolepsy drugs market a leader in global healthcare.

HARMONY BIOSCIENCES ANNOUNCES POSITIVE TOPLINE DATA FROM PHASE 2 SIGNAL DETECTION STUDY EVALUATING PITOLISANT IN ADULT PATIENTS WITH MYOTONIC DYSTROPHY TYPE 1

Retrieved on: 
Thursday, December 7, 2023
Harmony, Safety, ESS, EDS, Narcolepsy, Fatigue, Data, FSS, Patient, DM1, OLE, Cataplexy, FDA, Muscle weakness, DSS, Pitolisant, Epworth Sleepiness Scale, Myotonic dystrophy, Myotonia, News, Somnolence, Pharmaceutical industry, Nursing, Medicinal plants

PLYMOUTH MEETING, Pa., Dec. 7, 2023 /PRNewswire/ -- Harmony Biosciences Holdings, Inc. ("Harmony" or the "Company") (Nasdaq: HRMY) today announced positive topline results from its Phase 2 signal detection study evaluating the safety and efficacy of pitolisant in adult patients with myotonic dystrophy type 1 (DM1).

Key Points: 
  • PLYMOUTH MEETING, Pa., Dec. 7, 2023 /PRNewswire/ -- Harmony Biosciences Holdings, Inc. ("Harmony" or the "Company") (Nasdaq: HRMY) today announced positive topline results from its Phase 2 signal detection study evaluating the safety and efficacy of pitolisant in adult patients with myotonic dystrophy type 1 (DM1).
  • "In addition, a positive signal for pitolisant has been demonstrated for fatigue, suggesting it could be a potential new treatment option for this symptom as well.
  • "The positive signals from this Phase 2 study are very encouraging, and support pitolisant as a potential treatment option for DM1."
  • This Phase 2 signal detection study was a randomized, double-blind, placebo-controlled study in adults ages 18-65 with DM1.

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

Retrieved on: 
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

Retrieved on: 
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.