Spinocerebellar ataxia

Orphan designation: rovatirelin Treatment of spinocerebellar ataxia, 11/11/2022 Positive

Retrieved on: 
Mardi, avril 9, 2024
Community, Civil service commission, Spinocerebellar ataxia, Diagnosis, Brain, Prevalence, Disease, BBB, Clinical, Locus coeruleus, Patient, Committee, Thyrotropin-releasing hormone, Spinocerebellar tract, Traffic barrier, EMA, IRIS, Neuron, TRH, Agency, European, COMP, European Commission, Marketing, Medicine, Pharmaceutical industry

Overview

Key Points: 
  • Overview
    This medicine was designated as an orphan medicine for the treatment of spinocerebellar ataxia in the European Union on 11 November 2022.
  • All medicines, including designated orphan medicines, must be authorised before they can be marketed and made available to patients in the EU.
  • The full list of orphan designations is available in the Community register of orphan medicinal products for human use.
  • EU register of orphan medicines
    The list of medicines that have received an orphan designation in the EU is available on the European Commission's website:

Vico Therapeutics Announces $60 million (€54 million) Series B Financing to Advance VO659 Clinical-Stage Program for Rare Neurological Diseases and Expand Pipeline

Retrieved on: 
Vendredi, janvier 5, 2024
DSM-IV codes, Spinocerebellar ataxia, Biotechnology, Research, SCA1, HD, Therapy, BNP Paribas Fortis, Investment, SCA3, Patient, Safety, Ackermans & van Haaren, Huntington's disease, Pharmaceutical industry

"This financing positions us to accelerate our efforts to advance our lead program VO659, currently in a Phase 1/2a clinical study of patients with spinocerebellar ataxia types 3 and 1 and Huntington's disease," said Micah Mackison, chief executive officer at Vico.

Key Points: 
  • "This financing positions us to accelerate our efforts to advance our lead program VO659, currently in a Phase 1/2a clinical study of patients with spinocerebellar ataxia types 3 and 1 and Huntington's disease," said Micah Mackison, chief executive officer at Vico.
  • The financing will also help support discovery and research efforts to further expand the company's pipeline in other genetic neurological diseases.
  • The company also announced that Jeroen Vangindertael, Ph.D., investment manager at AvH, will join Vico's board of directors.
  • "We are proud to support Vico in this financing round as they continue to advance their lead clinical program and further develop a portfolio of novel antisense oligonucleotides," said Dr. Vangindertael.

Seelos Therapeutics to Present a Poster on SLS-005 in Alzheimer's Disease at Neuroscience 2023

Retrieved on: 
Vendredi, octobre 27, 2023
Washington Convention Center, Alzheimer's disease, AFL, Rare, Spinocerebellar ataxia, HD, Autophagy, Mouse, Tauopathy, Research fellow, Walter, Huntington's disease, Society, Therapy, Dietary supplement, ALS, Neuroscience

NEW YORK, Oct. 27, 2023 /PRNewswire/ -- Seelos Therapeutics, Inc. (Nasdaq: SEEL) ("Seelos"), a clinical-stage biopharmaceutical company focused on the development of therapies for central nervous system disorders and rare diseases, today announced that it has been selected to present a poster from a study of SLS-005 (trehalose injection, 90.5 mg/mL for intravenous infusion) in a tauopathy model of Alzheimer's disease at the Society for Neuroscience's Neuroscience 2023 meeting, to be held on November 11-15, 2023, at the Walter E. Washington Convention Center in Washington, D.C.

Key Points: 
  • In this model, tauopathy was induced in older non-human primates (NHPs) through double-tau mutations introduced in entorhinal cortex bilaterally.
  • NfL is a non-specific biomarker for several neurodegenerative conditions, including Alzheimer's disease, Huntington's disease, and amyotrophic lateral sclerosis (ALS).
  • This has now been demonstrated in two aggressive and fast progressing neurodegenerative disease models of Huntington's disease and Alzheimer's disease across two species, mice and non-human primates, respectively," said Krishna Subramanian, Ph.D., Vice President, Non-Clinical Development and Translational Science at Seelos.
  • Seelos will present a poster on Monday, November 13, 2023 at 9:00 a.m.

Avenue Therapeutics Reports First Quarter 2023 Financial Results and Recent Corporate Highlights

Retrieved on: 
Vendredi, mai 12, 2023
Epilepsy, DSM-IV codes, IV, Symantec Endpoint Protection, Addiction, Tramadol, Type, SBMA, Food and Drug Administration Amendments Act of 2007, Panic, Patient, Division, Risk, Hypoventilation, Spinal and bulbar muscular atrophy, Neuro, FDA, Therapy, Research, Spinocerebellar ataxia, Food, Safety, D, Pharmaceutical industry, Cryptocurrency, Spine, Pain management, Avenue

MIAMI, May 12, 2023 (GLOBE NEWSWIRE) -- Avenue Therapeutics, Inc. (Nasdaq: ATXI) (“Avenue” or the “Company”), a specialty pharmaceutical company focused on the development and commercialization of therapies for the treatment of neurologic diseases, today reported financial results and recent corporate highlights for the first quarter ended March 31, 2023.

Key Points: 
  • MIAMI, May 12, 2023 (GLOBE NEWSWIRE) -- Avenue Therapeutics, Inc. (Nasdaq: ATXI) (“Avenue” or the “Company”), a specialty pharmaceutical company focused on the development and commercialization of therapies for the treatment of neurologic diseases, today reported financial results and recent corporate highlights for the first quarter ended March 31, 2023.
  • In addition, Jay Kranzler, M.D., Ph.D., a current Director of Avenue, was appointed as the Chairman of Avenue.
  • R&D Expenses: Research and development expenses for the first quarter of 2023 were $1.2 million, compared to $1.8 million for the first quarter of 2022.
  • G&A Expenses: General and administrative expenses for the first quarter of 2023 were $1.0 million, compared to $1.0 million for the first quarter of 2022.

Avenue Therapeutics Reports Full Year 2022 Financial Results and Recent Corporate Highlights

Retrieved on: 
Jeudi, mars 30, 2023
Epilepsy, DSM-IV codes, Research, IV, Fortress Biotech, Symantec Endpoint Protection, Disease, Clinical trial, Tramadol, Type, Anterior grey column, SBMA, Acquisition, Panic, Patient, Risk, Hypoventilation, Spinal and bulbar muscular atrophy, Orphan, Neuro, FDA, Therapy, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Spinocerebellar ataxia, Food, Safety, D, Pharmaceutical industry, Cryptocurrency, Spine, Avenue

MIAMI, March 30, 2023 (GLOBE NEWSWIRE) -- Avenue Therapeutics, Inc. (Nasdaq: ATXI) (“Avenue” or the “Company”), a specialty pharmaceutical company focused on the development and commercialization of therapies for the treatment of rare and neurologic diseases, today reported financial results and recent corporate highlights for the year ended December 31, 2022.

Key Points: 
  • MIAMI, March 30, 2023 (GLOBE NEWSWIRE) -- Avenue Therapeutics, Inc. (Nasdaq: ATXI) (“Avenue” or the “Company”), a specialty pharmaceutical company focused on the development and commercialization of therapies for the treatment of rare and neurologic diseases, today reported financial results and recent corporate highlights for the year ended December 31, 2022.
  • In September 2022, Avenue received the official meeting minutes from the FDA regarding a meeting conducted in August 2022, for IV Tramadol.
  • In addition, Jay Kranzler, M.D., Ph.D., a current Director of Avenue, was appointed as the Chairman of Avenue.
  • G&A Expenses: General and administrative expenses for the full year 2022 were $5.3 million, compared to $2.5 million in 2021.

AnnJi Pharmaceutical Announced A Licensing Agreement With Avenue Therapeutics To Develop And Commercialize AJ201, A First-In-Class Clinical Asset For The Treatment Of Kennedy's Disease

Retrieved on: 
Vendredi, mars 10, 2023
Sale, Disease, ODD, Spinal and bulbar muscular atrophy, Cell, Spinocerebellar ataxia, Orphan, SBMA, Safety, Human, European Medicines Agency, Anterior grey column, Oxidative stress, Patient, AR, Huntington's disease, Pharmaceutical industry, Avenue

In addition, AnnJi will retain the manufacturing right to provide clinical and commercial supply of AJ201 to Avenue under agreed sale price and will be compensated for any Avenue's sublicense activity.

Key Points: 
  • In addition, AnnJi will retain the manufacturing right to provide clinical and commercial supply of AJ201 to Avenue under agreed sale price and will be compensated for any Avenue's sublicense activity.
  • This agreement is one of the largest license deals for a small molecule drug in early Phase 1b/2a clinical development in the past twenty-four months.
  • AJ201 is currently being investigated in a multicenter, randomized, double-blind clinical trial in six clinical sites across the U.S.
  • The study aims to evaluate the safety and clinical response of AJ201 in Kennedy's Disease patients.

Avenue Therapeutics Enters into a Transformational License Agreement with AnnJi Pharmaceutical to Develop and Commercialize AJ201, a First-in-Class Clinical Asset for the Treatment of Spinal and Bulbar Muscular Atrophy

Retrieved on: 
Jeudi, mars 2, 2023
Safety, Walking, DSM-IV codes, Spinal and bulbar muscular atrophy, NFE2L2, Cell death, Patient, Spinocerebellar ataxia, Choking, Speech, Literature, Therapy, Anterior grey column, Regulation of food and dietary supplements by the U.S. Food and Drug Administration, Muscle atrophy, Oxidative stress, Orphan, SBMA, Food, AR, Pharmaceutical industry, Avenue

“The license for AJ201 brings a cutting-edge asset into Avenue’s pipeline that is the lead molecule in the clinic to treat Kennedy’s Disease, a debilitating rare neuromuscular disorder.

Key Points: 
  • “The license for AJ201 brings a cutting-edge asset into Avenue’s pipeline that is the lead molecule in the clinic to treat Kennedy’s Disease, a debilitating rare neuromuscular disorder.
  • SBMA is a rare, inherited, X-linked genetic neuromuscular disease primarily affecting men.
  • The weakening of the bulbar muscles affects chewing, speech and swallowing, with patients prone to choking or inhaling foods or liquids, resulting in airway infection.
  • This study aims to evaluate the safety and clinical response of AJ201 in patients suffering from SBMA.

Ataxin 3 Drugs in Development by Therapy Areas and Indications, Stages, MoA, RoA, Molecule Type and Key Players - ResearchAndMarkets.com

Retrieved on: 
Jeudi, décembre 8, 2022
Genetics, Pharmaceutical, Health, Molecule, Spinocerebellar ataxia, Therapy, Research and development, R, Key, EC, Ataxin 3, Product description, ATXN3, Research, Machado–Joseph disease, Mall of America, Route of administration, History, Administration, News, SCA3, Pharmaceutical industry, Medical device

The "Ataxin 3 Drugs in Development by Therapy Areas and Indications, Stages, MoA, RoA, Molecule Type and Key Players" report has been added to ResearchAndMarkets.com's offering.

Key Points: 
  • The "Ataxin 3 Drugs in Development by Therapy Areas and Indications, Stages, MoA, RoA, Molecule Type and Key Players" report has been added to ResearchAndMarkets.com's offering.
  • This report provides in depth analysis on Ataxin 3 (Machado Joseph Disease Protein 1 or Spinocerebellar Ataxia Type 3 Protein or SCA3 or ATXN3 or EC 3.4.19.12) targeted pipeline therapeutics.
  • The report provides comprehensive information complete with Analysis by Indications, Stage of Development, Mechanism of Action (MoA), Route of Administration (RoA) and Molecule Type.
  • The report also covers the descriptive pharmacological action of the therapeutics, its complete research and development history and latest news and press releases.

BGV co-leads €10 million seed investment in neuroscience company FundaMental Pharma

Retrieved on: 
Jeudi, novembre 17, 2022
Spinocerebellar ataxia, Department of Neurobiology, Harvard Medical School, Neurotoxicity, Patient, Neuroscience, Fundamental, Heidelberg University, Interdisciplinary Center Herzliya, Amyotrophic lateral sclerosis, VC, Neurodegeneration, BGV, ALS, Neuropharmacology, Therapy, Pharmaceutical industry, Management, Research

Amsterdam, The Netherlands, 17 November 2022 - BioGeneration Ventures (BGV), a leading early-stage VC in European biopharma today announces that it has co-led, a 10 million Seed investment in FundaMental Pharma (FundaMental), a preclinical neuroscience company spun out of Heidelberg University with Thuja Capital.

Key Points: 
  • Amsterdam, The Netherlands, 17 November 2022 - BioGeneration Ventures (BGV), a leading early-stage VC in European biopharma today announces that it has co-led, a 10 million Seed investment in FundaMental Pharma (FundaMental), a preclinical neuroscience company spun out of Heidelberg University with Thuja Capital.
  • BGV invested from its Fund IV and following the financing, Dr Keno Gutierrez, Partner at BGV, has been appointed Chair of the Board of FundaMental.
  • FundaMental is built on the pioneering research of Professor Hilmar Bading and Dr. Jing Yan.
  • In conjunction with the financing, FundaMental also announced several key senior management appointments and the Board of Directors of the company.

Seelos Therapeutics to Sponsor and Participate in Wonderland, the World's Leading Psychedelic Event

Retrieved on: 
Jeudi, octobre 27, 2022
MDD, Suicide, SCA, Central nervous system, Private Securities Litigation Reform Act, Agency, Nature, Company, Student, Program, Neuroscience, U.S. Securities and Exchange Commission, Sanfilippo syndrome, Policy, Agency for Healthcare Research and Quality, Ketamine, Annual report, Senior management, FDA, Therapy, Major depressive disorder, Brain, Spinocerebellar ataxia, CEO, Parkinson's disease, PTSD, World, Breakfast, Meanings of minor planet names: 8001–9000, Degenerative disease, Mental health, ASIB, Industry, COVID-19, Nasdaq, Trial of the century, Disorder, ALS, Amyotrophic lateral sclerosis, Depression, Science, Education, NEW, Writing, Post-traumatic stress disorder, Movement disorders, Risk, Music, CNS, Psychiatry, Safety, Pharmaceutical industry, Nightclub, Online shopping, Behavior, Patient

NEW YORK, Oct. 27, 2022 /PRNewswire/ -- Seelos Therapeutics, Inc. (Nasdaq: SEEL), a clinical-stage biopharmaceutical company focused on the development of therapies for central nervous system disorders and rare diseases, today announced it will sponsor and participate in Wonderland, the World's Leading Psychedelic Event, organized by Microdose Psychedelics Insights, to be held at the Mana Wynwood Convention Center in Miami, FL from November 3rd to the 5th.

Key Points: 
  • -Seelos Senior Management will sponsor the Press Breakfast on Friday November 4th at 8:30am and take part in 2 panel discussions on Saturday November 5th.
  • Additionally, Seelos will participate in a panel discussion titled: Route of Administration Dose; Does Dissociation Correlate with Antidepressant/Analgesic Effects on Saturday, November 5th at 10:40am ET.
  • Wonderland Miami offers the psychedelic medicine industry a unique in-person convergence of business, science, music, and emerging technologies.
  • The event is a thought-leadership platform and an opportunity to connect with influential leaders in science, policy, and business.