Epworth Sleepiness Scale

Zevra Therapeutics Reports Fourth Quarter and Full Year 2023 Financial Results and Corporate Updates

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목요일, 3월 28, 2024
Narcolepsy, Exercise, Somnolence, New Drug Application, Patient, Visual analogue scale, Sale, NDA, G&A, Arimoclomol, Nature, IND, Bank statement, Microsoft Access, Investment, Therapy, Priority review, Corporation, Audit committee, Research, Niemann–Pick disease, Medical science liaison, Sleep disorder, Brain, Trial of the century, Safety, Interim, Food, Sleep, QuickStart, Disease, FY, Acquisition, Emetine, UCD, COL3A1, Celiprolol, Syndrome, FDA, Marketing, Conference call, Conference, EAP, SDX, NPC, Epworth Sleepiness Scale, Idiopathic hypersomnia, Physician, PDUFA, Pharmaceutical industry

CELEBRATION, Fla., March 28, 2024 (GLOBE NEWSWIRE) -- Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra, or the Company), a rare disease therapeutics company, today provided corporate updates and reported its financial results for the fourth quarter and year ended December 31, 2023. 

Key Points: 
  • ET
    CELEBRATION, Fla., March 28, 2024 (GLOBE NEWSWIRE) -- Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra, or the Company), a rare disease therapeutics company, today provided corporate updates and reported its financial results for the fourth quarter and year ended December 31, 2023.
  • “We made solid progress on our key priorities in 2023,” said Neil F. McFarlane, President and Chief Executive Officer of Zevra.
  • Overview of Q4 2023 and FY 2023 Financial Results:
    Net revenue for Q4 2023 was $13.2 million, compared to prior year Q4 net revenue of $2.2 million.
  • ET, to discuss its corporate and financial results for Q4 and FY 2023.

Zevra Therapeutics Announces Top-Line Data from the Phase 2 Clinical Trial of KP1077 for Idiopathic Hypersomnia

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화요일, 3월 26, 2024
Brain, Disease, Somnolence, Appetite, Partnership, Headache, Visual analogue scale, Patient, Insomnia, EDS, Anxiety, SDX, Doctor of Philosophy, Epworth Sleepiness Scale, Nausea, Clinical trial, Therapy, Safety, Narcolepsy, ESS, IHSS, Idiopathic hypersomnia, Clouding of consciousness, BFS, Food, Pharmaceutical industry

CELEBRATION, Fla., March 26, 2024 (GLOBE NEWSWIRE) -- Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra, or the Company) a rare disease therapeutics company, today announced top-line data from its placebo-controlled, double-blind Phase 2 clinical trial (NCT05668754) evaluating the safety and tolerability of KP1077 (serdexmethylphenidate, or SDX) in patients with idiopathic hypersomnia (IH). This proof-of-concept study was not powered to demonstrate statistical significance.   The data gathered for several secondary and exploratory endpoints, including the Epworth Sleepiness Scale (ESS), Idiopathic Hypersomnia Severity Scale (IHSS) and Sleep Inertia Visual Analog Scale (SIVAS) will inform the Phase 3 study design.

Key Points: 
  • CELEBRATION, Fla., March 26, 2024 (GLOBE NEWSWIRE) -- Zevra Therapeutics, Inc. (NasdaqGS: ZVRA) (Zevra, or the Company) a rare disease therapeutics company, today announced top-line data from its placebo-controlled, double-blind Phase 2 clinical trial ( NCT05668754 ) evaluating the safety and tolerability of KP1077 (serdexmethylphenidate, or SDX) in patients with idiopathic hypersomnia (IH).
  • The data gathered for several secondary and exploratory endpoints, including the Epworth Sleepiness Scale (ESS), Idiopathic Hypersomnia Severity Scale (IHSS) and Sleep Inertia Visual Analog Scale (SIVAS) will inform the Phase 3 study design.
  • The results from the completed Phase 2 trial provide key information for the design of a potentially pivotal Phase 3 trial of KP1077 in patients with IH.
  • The Company plans to request an end-of-Phase 2 (EOP2) meeting with the U.S. Food and Drug Administration to seek guidance on the Phase 3 clinical trial design.

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

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월요일, 3월 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

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월요일, 3월 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.

Takeda Intends to Rapidly Initiate the First Global Phase 3 Trials of TAK-861, an Oral Orexin Agonist, in Narcolepsy Type 1 in First Half of Fiscal Year 2024

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목요일, 2월 8, 2024
Biotechnology, Neurology, Health, Pharmaceutical, Clinical Trials, MRCP, Hepatotoxicity, NT2, Neurological disorder, Narcolepsy, WCR, Fiscal, Wakefulness, Caregiver, Head, Patient, ESS, DSM-IV codes, Medicine, Neuron, OX2R, Disease, Epworth Sleepiness Scale, MWT, Pharmaceutical industry

It is currently classified into two different types: narcolepsy type 1 (NT1) caused by significant loss of orexin neurons with resulting lack of orexin, and narcolepsy type 2 (NT2) where orexin levels are generally normal.

Key Points: 
  • It is currently classified into two different types: narcolepsy type 1 (NT1) caused by significant loss of orexin neurons with resulting lack of orexin, and narcolepsy type 2 (NT2) where orexin levels are generally normal.
  • Stimulating the orexin receptor 2 in NT1 patients targets the underlying pathophysiology of the disease to restore orexin signaling.
  • Based on these results, and in consultation with global health authorities, Takeda plans to initiate global Phase 3 trials of TAK-861 in NT1 rapidly in the first half of its fiscal year 2024.
  • Results from the Phase 2b trials have no impact on the full year consolidated reported forecast for the fiscal year ending March 31, 2024 (Fiscal Year 2023).

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

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화요일, 1월 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

Nyxoah Announces Real World Case Series Demonstrating Positive Results in Treating CCC Patients with Genio®

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월요일, 12월 11, 2023
Mont-Saint-Guibert, Conference, Obstructive sleep apnea, FDA, Epworth Sleepiness Scale, ESS, Euronext, Patient, AHI, CCC, Medical device

The investigator-sponsored case series was presented by Dr. Christian Plettenberg from the Universitätsklinikum Düsseldorf on December 8, 2023.

Key Points: 
  • The investigator-sponsored case series was presented by Dr. Christian Plettenberg from the Universitätsklinikum Düsseldorf on December 8, 2023.
  • Results showed an average apnea-hypopnea index (AHI) decrease of 73% and Epworth Sleepiness Scale (ESS) decrease of 58%.
  • “These data further validate Genio’s bilateral stimulation approach in treating CCC patients, who represent approximately 30% of HGNS eligible-to-treat OSA patients and are contraindicated to commercially available HGNS therapy in the US.
  • “I want to thank Dr. Plettenberg and his colleagues for their important work which reinforces Genio as a treatment solution for both non-CCC and CCC patients.”

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

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목요일, 12월 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.

Pharma Two B Announces Publication of Positive Phase 3 Study Results of P2B001, an Investigational Treatment for Early Parkinson’s Disease, in Movement Disorders

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목요일, 11월 9, 2023
UPDRS, Incidence, PD, III, Neurology, University of South Florida, Physician, Emeritus, Pramipexole, Epworth Sleepiness Scale, FRCPC, LSM, ESS, Somnolence, University, Department of Neurology, Xuanwu hospital, Mount Sinai School, NDA, Safety, Patient, PPX, Movement disorder, Dietary supplement, Pharmaceutical industry, Surveying

The study included 544 untreated patients with Parkinson’s disease who were randomized (2:2:2:1) to treatment with P2B001, its individual components (pramipexole-ER 0.6 mg or rasagiline-ER 0.75 mg), or marketed PramiER individually titrated to optimal dose (1.5-4.5 mg).

Key Points: 
  • The study included 544 untreated patients with Parkinson’s disease who were randomized (2:2:2:1) to treatment with P2B001, its individual components (pramipexole-ER 0.6 mg or rasagiline-ER 0.75 mg), or marketed PramiER individually titrated to optimal dose (1.5-4.5 mg).
  • Further, he stated that “P2B001 is a once-a-day treatment that requires no titration.
  • P2B001 was generally well tolerated and showed fewer sleep-related and dopaminergic adverse events in comparison to commercially used doses of pramipexole ER.
  • “We look forward to moving P2B001 forward towards an NDA submission to make this potential treatment option available to patients.”

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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금요일, 10월 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.