Narcolepsy

KemPharm Supports Niemann-Pick Awareness Month During October and Global Niemann-Pick Awareness Day on October 19th

Retrieved on: 
Tuesday, October 4, 2022

CELEBRATION, Fla., Oct. 04, 2022 (GLOBE NEWSWIRE) -- KemPharm, Inc.(NasdaqGS: KMPH) (KemPharm, or the Company), a biotechnology company focused on the discovery, development and commercialization of novel treatments for rare diseases, announced its support for Niemann-Pick Awareness Month, which is the month of October, and Global Niemann-Pick Awareness Day, which is October 19th.

Key Points: 
  • CELEBRATION, Fla., Oct. 04, 2022 (GLOBE NEWSWIRE) -- KemPharm, Inc.(NasdaqGS: KMPH) (KemPharm, or the Company), a biotechnology company focused on the discovery, development and commercialization of novel treatments for rare diseases, announced its support for Niemann-Pick Awareness Month, which is the month of October, and Global Niemann-Pick Awareness Day, which is October 19th.
  • KemPharm supports and unites with these groups in advocating for those affected by NPC and their families and in seeking new therapeutic options for NPC where no satisfactory alternative treatments currently exist.
  • KemPharm is a biotechnology company focused on the discovery, development and commercialization of novel treatments for rare diseases.
  • KemPharm has a diverse product portfolio, combining a clinical-stage development pipeline with NDA-stage and commercial assets.

KemPharm Announces Positive Topline Data from Phase 1 Clinical Trial Evaluating Cardiovascular Safety of Serdexmethylphenidate (SDX)

Retrieved on: 
Wednesday, September 28, 2022

CELEBRATION, Fla., Sept. 28, 2022 (GLOBE NEWSWIRE) -- KemPharm, Inc. (NasdaqGS: KMPH) (KemPharm, or the Company), a specialty pharmaceutical company focused on the discovery, development and commercialization of novel treatments for rare central nervous system (CNS), neurodegenerative and lysosomal storage diseases, today announced topline data from its exploratory Phase 1 clinical trial confirming the relative cardiovascular effects and pharmacokinetics of serdexmethylphenidate (SDX) compared to immediate-release and long-acting formulations of Ritalin® (racemic methylphenidate), a commonly prescribed CNS stimulant. SDX, KemPharm’s proprietary prodrug of d-methylphenidate (d-MPH), is the sole active pharmaceutical ingredient (API) in KP1077, a potential treatment for idiopathic hypersomnia (IH), a rare sleep disorder.

Key Points: 
  • KemPharm expects to initiate a Phase 2 clinical trial of KP1077 in patients with IH prior to year-end 2022 and a second trial in patients with narcolepsy in 2023.
  • We are pleased with the initial results from the Phase 1 cardiovascular safety trial of SDX, which demonstrated the potential for higher dose formulations of SDX to be safe and well-tolerated.
  • Overall, the data suggest that SDX can be safely dosed at levels higher than current products, which is expected to result in improved efficacy.
  • Maximal plasma concentrations (Cmax) of d-MPH were similar for both Ritalin treatments that were administered at equal doses of 80mg.

PM360 Magazine Announces Winners of 2022 Trailblazer Awards

Retrieved on: 
Friday, September 23, 2022

NEW YORK, Sept. 23, 2022 /PRNewswire/ -- PM360, a leading life sciences marketing industry trade publication, announced the winners of their 14th annual Trailblazer Awards during an event held at Gotham Hall in New York City on Thursday, September 22, 2022. The awards recognize outstanding companies, CEOs, products, marketers, brand managers, and cutting-edge initiatives within the pharmaceutical, biotech, medical device, and diagnostics industries. In total, 72 winners were named across eight overall categories: Companies of the Year, CEOs of the Year, Products of the Year, Marketer of the Year, Marketing Team of the Year, Lifetime Achievement, Brand Champions, and Initiatives.

Key Points: 
  • NEW YORK, Sept. 23, 2022 /PRNewswire/ -- PM360, a leading life sciences marketing industry trade publication, announced the winners of their 14th annual Trailblazer Awards during an event held at Gotham Hall in New York City on Thursday, September 22, 2022.
  • The awards recognize outstanding companies, CEOs, products, marketers, brand managers, and cutting-edge initiatives within the pharmaceutical, biotech, medical device, and diagnostics industries.
  • Each of the winners were judged and selected by the PM360 Editorial Advisory Board, a cross section of experts from across the industry.
  • PM360 will publish profiles of this year's Trailblazer winners in its October issue and on its website.

WINNERS OF THE 2023 BREAKTHROUGH PRIZES IN LIFE SCIENCES, MATHEMATICS AND FUNDAMENTAL PHYSICS ANNOUNCED

Retrieved on: 
Thursday, September 22, 2022

The Breakthrough Prize in Mathematics goes to Daniel A. Spielman, for multiple discoveries in theoretical computer science and mathematics.

Key Points: 
  • The Breakthrough Prize in Mathematics goes to Daniel A. Spielman, for multiple discoveries in theoretical computer science and mathematics.
  • The Breakthrough Prize in Fundamental Physics is shared by Charles H. Bennett, Gilles Brassard,David Deutsch and Peter Shorfor their foundational work in quantum information.
  • And important contributions from early-career scientists are also recognized, with six New Horizons Prizes in Physics and Mathematics and three Maryam Mirzakhani New Frontiers Prizes awarded for women mathematicians who have recently completed their doctorates.
  • Each of the five main prizes is $3 million, and the addition of the early-career awards brings this year's total prizes to $15.75 million.

WINNERS OF THE 2023 BREAKTHROUGH PRIZES IN LIFE SCIENCES, MATHEMATICS AND FUNDAMENTAL PHYSICS ANNOUNCED

Retrieved on: 
Thursday, September 22, 2022

The Breakthrough Prize in Mathematics goes to Daniel A. Spielman, for multiple discoveries in theoretical computer science and mathematics.

Key Points: 
  • The Breakthrough Prize in Mathematics goes to Daniel A. Spielman, for multiple discoveries in theoretical computer science and mathematics.
  • The Breakthrough Prize in Fundamental Physics is shared by Charles H. Bennett, Gilles Brassard,David Deutsch and Peter Shorfor their foundational work in quantum information.
  • And important contributions from early-career scientists are also recognized, with six New Horizons Prizes in Physics and Mathematics and three Maryam Mirzakhani New Frontiers Prizes awarded for women mathematicians who have recently completed their doctorates.
  • Each of the five main prizes is $3 million, and the addition of the early-career awards brings this year's total prizes to $15.75 million.

HARMONY BIOSCIENCES ANNOUNCES 2022 FUNDING RECIPIENTS OF PATIENTS AT THE HEART AND PROGRESS AT THE HEART AWARD PROGRAMS

Retrieved on: 
Thursday, September 22, 2022

PLYMOUTH MEETING, Pa., Sept. 22, 2022 /PRNewswire/ -- Harmony Biosciences Holdings, Inc. ("Harmony") (NASDAQ: HRMY), a pharmaceutical company dedicated todeveloping and commercializing innovative therapies for patients with rare neurological diseases, today announced on the fourth annual World Narcolepsy Day, the funding recipients of its Patients at the Heart and Progress at the Heart award programs.

Key Points: 
  • PLYMOUTH MEETING, Pa., Sept. 22, 2022 /PRNewswire/ -- Harmony Biosciences Holdings, Inc. ("Harmony") (NASDAQ: HRMY), a pharmaceutical company dedicated todeveloping and commercializing innovative therapies for patients with rare neurological diseases, today announced on the fourth annual World Narcolepsy Day, the funding recipients of its Patients at the Heart and Progress at the Heart award programs.
  • This award funds innovative programs that address health disparities and inequities in rare neurological disease and sleep disorder communities.
  • Combined with the Patients at the Heart awards, total funding for these two programs has now increased to $380,000 in over the last four years.
  • "In recognition of World Narcolepsy Day, we are pleased to announce this year's recipients of the Patients at the Heart and Progress at the Heart award programs.

Wake Up Narcolepsy Physician Survey Shows Significant Challenges in Successful Diagnosis of Narcolepsy

Retrieved on: 
Thursday, September 22, 2022

WORCESTER, Mass., Sept. 22, 2022 /PRNewswire/ -- Wake Up Narcolepsy (WUN), a national leader for narcolepsy research and awareness, today announced the results of a survey of 100 physicians who treat narcolepsy in children and adolescents in the Greater Boston Area. These findings, which coincide with World Narcolepsy Day, highlight the challenges of identifying, managing and treating narcolepsy in this patient population.

Key Points: 
  • These findings, which coincide with World Narcolepsy Day , highlight the challenges of identifying, managing and treating narcolepsy in this patient population.
  • "These survey results are illuminating, as they highlight improved physician knowledge about narcolepsy compared to previously published data.
  • There are two main types of narcolepsy: narcolepsy with cataplexy (Type 1) and narcolepsy without cataplexy (Type 2).
  • Wake Up Narcolepsy (WUN) is a 501(c)(3) not-for-profit organization founded in 2008 to promote narcolepsy research and awareness.

Avadel Pharmaceuticals to Present at the Ladenburg Thalmann Healthcare Conference

Retrieved on: 
Thursday, September 22, 2022

DUBLIN, Ireland, Sept. 22, 2022 (GLOBE NEWSWIRE) -- Avadel Pharmaceuticals plc (Nasdaq: AVDL), a biopharmaceutical company focused on transforming medicines to transform lives, announced today that members of management will participate in a fireside chat at the Ladenburg Thalmann Healthcare Conference on Thursday, September 29 at 12:30 p.m. E.T.

Key Points: 
  • DUBLIN, Ireland, Sept. 22, 2022 (GLOBE NEWSWIRE) -- Avadel Pharmaceuticals plc (Nasdaq: AVDL), a biopharmaceutical company focused on transforming medicines to transform lives, announced today that members of management will participate in a fireside chat at the Ladenburg Thalmann Healthcare Conference on Thursday, September 29 at 12:30 p.m. E.T.
  • A live webcast of the fireside chat, as well as an archived recording, will be available on Avadels Investor Relations website, investors.avadel.com , for 90 days following the conference.
  • Avadel 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.

Centessa Pharmaceuticals Announces Poster Presentation Featuring Novel Orexin Receptor 2 (OX2R) Agonist Discovery Pipeline at the 26th Conference of the European Sleep Research Society (Sleep Europe 2022)

Retrieved on: 
Tuesday, September 20, 2022

BOSTON and LONDON, Sept. 20, 2022 (GLOBE NEWSWIRE) -- Centessa Pharmaceuticals plc (Nasdaq: CNTA), today announced that non-clinical data from its oral orexin receptor 2 (OX2R) agonist discovery pipeline have been accepted for poster presentation at the 26th Conference of the European Sleep Research Society meeting (Sleep Europe 2022) being held on September 27-30, 2022, in Athens, Greece. Centessa’s OX2R agonists are designed to directly target the underlying pathophysiology of orexin neuron loss in narcolepsy type 1 (NT1), with potential expansion into narcolepsy type 2 (NT2), and other sleep disorders.

Key Points: 
  • These novel OX2R agonist compounds showed high potency in activating recombinant human and endogenous mouse OX2Rs, with more than a thousand-fold selectivity for OX2R compared to OX1R.
  • Our innovative pipeline is both a testament to their expertise and strong validation of our approach to structure-based drug design.
  • We are encouraged by the non-clinical data and are rapidly progressing several potentially best-in-class oral small-molecule OX2R agonists through our discovery pipeline.
  • We are excited to showcase some of our small-molecule OX2R agonist discovery pipeline and share these data at Sleep Europe 2022, said Mario Alberto Accardi, PhD, Head, Centessa Orexin Program.

US Narcolepsy Market and Competitive Landscape 2022: Valuations, Forecasts, Sales and Competitive Landscape - ResearchAndMarkets.com

Retrieved on: 
Thursday, September 8, 2022

The "US Narcolepsy Market and Competitive Landscape - 2022" report has been added to ResearchAndMarkets.com's offering.

Key Points: 
  • The "US Narcolepsy Market and Competitive Landscape - 2022" report has been added to ResearchAndMarkets.com's offering.
  • The report US Narcolepsy Market and Competitive Landscape Highlights - 2022, provides comprehensive insights into Narcolepsy pipeline products, Narcolepsy epidemiology, Narcolepsy market valuations and forecast, Narcolepsy drugs sales and competitive landscape in the US.
  • The research is classified into seven sections - Narcolepsy treatment options, pipeline products, market analysis comprising of epidemiology, key products marketed, market valuations and forecast, drugs sales and market shares.
  • Find out how the market advanced from 2019 and forecast to 2027
    Evaluate commercial market opportunity assessment, positioning, and segmentation for Narcolepsy products