Idiopathic hypersomnia

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

Retrieved on: 
Thursday, March 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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Tuesday, March 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 Results of CRESCENDO Narcolepsy Patient Survey Demonstrating Unmet Needs in Treated Narcolepsy Type 1 Patients

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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.

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

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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.

Illuminate Hypersomnia Initiative Shines Light on What It's Like to Live with Idiopathic Hypersomnia

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Thursday, December 7, 2023
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WEST PALM BEACH, Fla., Dec. 7, 2023 /PRNewswire/ -- The Sleep Consortium, in collaboration with sleep-related patient advocacy organizations worldwide, announces the Illuminate Hypersomnia initiative, a groundbreaking effort to highlight the lived experience of idiopathic hypersomnia (IH). This initiative aims to fully describe the impact of IH on the lives of those affected, their current management strategies, and the treatment benefits that matter most to them as new therapies are developed.

Key Points: 
  • The meeting will be streamed live, bringing members of the global IH community together to share their experiences and perspectives.
  • The Illuminate Hypersomnia initiative also aims to expand collaboration and support within the IH community to help individuals learn from and empower one another.
  • "Our mission with the Illuminate Hypersomnia initiative is clear: bring to light the hidden struggles of idiopathic hypersomnia and unite in the quest for better treatments," remarked CEO of Sleep Consortium Lindsay Jesteadt, PhD.
  • Organizations generously supporting the Illuminate Hypersomnia initiative with unrestricted educational grants include Alkermes, Avadel Pharmaceuticals, Harmony Biosciences, Jazz Pharmaceuticals, Takeda, and Zevra Therapeutics.

Project Sleep Launches the Sleep Helpline™ to Provide Free and Personalized Support for People Facing Sleep Issues and Sleep Disorders

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Wednesday, December 6, 2023
Sleep, JD, Kleine–Levin syndrome, DSM-IV codes, REM, Narcolepsy, Sleep disorder, Patient, LOS, Obstructive sleep apnea, Idiopathic hypersomnia, Uncertainty, Sleep-related breathing disorder, Central sleep apnea, Eastern Time Zone, Caregiver, University, Upper airway resistance syndrome, Parasomnia, Diagnosis, Social, Doctor of Philosophy, Sleepwalking, Restless legs syndrome, Non-24-hour sleep–wake disorder, Pharmaceutical industry, Nursing

LOS ANGELES, Dec. 6, 2023 /PRNewswire/ -- Project Sleep, a nonprofit dedicated to raising awareness of sleep health, sleep equity, and sleep disorders, is thrilled to announce the official launch of the Sleep Helpline™, a new, professionally-staffed national helpline providing timely and trusted resources to help individuals navigate sleep issues and sleep disorders at every step of the journey.

Key Points: 
  • "An estimated 50-70 million Americans live with a chronic sleep disorder, yet the majority are undiagnosed," said Julie Flygare , JD, President & CEO of Project Sleep.
  • "We know that the path to finding accurate sleep disorder diagnosis, treatment, and support is often long, lonely, and inefficient.
  • This is why the new Sleep Helpline will meet people where they are in moments of uncertainty, to provide free and personalized one-to-one support and resources."
  • Special thanks to Jazz Pharmaceuticals and Harmony Biosciences for generously providing sponsorship funding to support the development of Project Sleep's new Sleep Helpline to support millions of people facing sleep issues and sleep disorders.

Zevra Therapeutics Reports Corporate Updates and Third Quarter 2023 Financial Results

Retrieved on: 
Tuesday, November 7, 2023
IH, Priority review, Vipul Ved Prakash, FDA, Sale, IND, Narcolepsy, Niemann–Pick disease, Food, G&A, Corporation, Conference, Emilie Christaller, Acquisition, Idiopathic hypersomnia, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Exercise, NPC, R, UCD School of Medicine, EDS, Disease, Retirement, Arimoclomol, Somnolence, Brain, NDA, Conference call, Investment, Therapy, Patient, Sleep inertia, Syndrome, Broadcasting, Pharmaceutical industry, Fine chemical, Zebra, Acer

Ended Q3 2023 with $83.4M in cash, cash equivalents, and investments, supporting our forecasted cash runway into 2026

Key Points: 
  • Ended Q3 2023 with $83.4M in cash, cash equivalents, and investments, supporting our forecasted cash runway into 2026
    Conference call and live audio webcast scheduled for today,
    CELEBRATION, Fla., Nov. 07, 2023 (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 quarter ended September 30, 2023.
  • Overview of Q3 2023 Financial Results:
    Net revenue for Q3 2023 was $2.9 million compared to prior year Q3 net revenue of $2.9 million.
  • As of September 30, 2023, these assets are included in secured corporate notes in the unaudited condensed consolidated balance sheet and totaled $42.0 million as of September 30, 2023.
  • ET, to discuss its corporate and financial results for Q3 2023.

Harmony Biosciences Reports Strong Third Quarter 2023 Financial Results

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Tuesday, October 31, 2023
IH, DM1, FDA, Research, Acquisition, Prader–Willi syndrome, Continue, Pivotal, LCA, Fragile X syndrome, Narcolepsy, FXS, Share repurchase, Conference, GAAP, Idiopathic hypersomnia, Bank statement, Development, Common stock, Commercialization, Growth, DSM-IV codes, Research and development, Benchmarking, PWS, Total, Myotonic dystrophy, Patient, Administration, Marketing, Shanda, Video game, Pharmaceutical industry, Fine chemical, Car rental, Silviculture, Cryptocurrency, Bank, Jean-François Zygel, Webcast, Harmony

ET

Key Points: 
  • ET
    PLYMOUTH MEETING, Pa., Oct. 31, 2023 (GLOBE NEWSWIRE) -- Harmony Biosciences Holdings, Inc. (“Harmony” or the “Company”) (Nasdaq: HRMY), a pharmaceutical company dedicated to developing and commercializing innovative therapies for patients with rare neurological diseases, today reported financial results and business updates for the quarter ended September 30, 2023.
  • The average number of patients on WAKIX increased by approximately 350 sequentially to approximately 5,800 for the quarter ended September 30, 2023.
  • Reconciliations of applicable GAAP financial measures to Non-GAAP financial measures are included at the end of this press release.
  • ET
    We are hosting our third quarter 2023 financial results conference call and webcast today, beginning at 8:30 a.m. Eastern Time.

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.

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

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Monday, October 23, 2023
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DUBLIN, Oct. 23, 2023 /PRNewswire/ -- Alkermes plc (Nasdaq: ALKS) today announced preliminary results, including initial proof-of-concept data, from a phase 1 study evaluating ALKS 2680, the company's novel, investigational orexin 2 receptor (OX2R) agonist in development for the treatment of narcolepsy. The ongoing phase 1 study is evaluating the safety, tolerability, pharmacokinetics and pharmacodynamics of ALKS 2680 in healthy volunteers and patients with narcolepsy or idiopathic hypersomnia via once-daily, oral administration. Initial data from the single- and multiple-ascending dose evaluations in healthy volunteers and the first cohort of four patients with narcolepsy type 1 (NT1) will be presented today at the 2023 World Sleep Congress in Rio de Janeiro.

Key Points: 
  • In the four patients with NT1, treatment with ALKS 2680 demonstrated improved sleep latency compared to placebo at all doses tested, with a clear dose response.
  • The differences between ALKS 2680 and placebo were statistically significant for all doses: 1 mg (p
  • Treatment with ALKS 2680 resulted in clinically meaningful improvements in MWT from baseline at all doses tested.
  • "These initial data support our design rationale for ALKS 2680 as a highly potent, orally bioavailable, selective orexin 2 receptor agonist designed to address the underlying pathology of narcolepsy.