Cardiomyopathy

EQS-News: Berlin Cures Expands Research Capabilities and Scientific Expertise with New Advisory Board

Retrieved on: 
Wednesday, April 10, 2024
Research, Glaucoma, COVID, ME/CFS, Diabetes, Patient, Biomarker, OBE, University, Long, Heart failure, Chronic kidney disease, Maastricht University, Cardiomyopathy, Pharmaceutical industry

Berlin, Germany, 3 April 2024 – Berlin Cures, a biotechnology company in Phase II clinical trial for a treatment against Long COVID, appoints six distinguished scientists to a new advisory board.

Key Points: 
  • Berlin, Germany, 3 April 2024 – Berlin Cures, a biotechnology company in Phase II clinical trial for a treatment against Long COVID, appoints six distinguished scientists to a new advisory board.
  • The aim of the board is to advise on industry developments and new growth potentials, as well as to support research and development that will further strengthen the portfolio of Berlin Cures and thus the successful market launch of new therapies.
  • “We are very pleased to now work with six renowned scientists who have achieved great success in their respective fields of research.
  • Berlin Cures has been known for innovative and effective research for years.

Alnylam Presents Positive Results from the KARDIA-2 Phase 2 Study of Zilebesiran Added to Standard of Care Antihypertensives in Patients with Inadequately Controlled Hypertension

Retrieved on: 
Sunday, April 7, 2024
Research, FDA, Clinical Trials, Cardiology, Biotechnology, Managed Care, Health, Pharmaceutical, Science, Blood pressure, Medication, Indapamide, Risk, Patient, Month, SBP, ABPM, American College, Olmesartan, Injection, Safety, RNA interference, Therapy, AGT, ACC, Amlodipine, Cardiomyopathy, Hypertension, Pharmaceutical industry

Zilebesiran is an investigational RNAi therapeutic targeting liver-expressed angiotensinogen (AGT) in development for the treatment of hypertension with the potential for biannual dosing.

Key Points: 
  • Zilebesiran is an investigational RNAi therapeutic targeting liver-expressed angiotensinogen (AGT) in development for the treatment of hypertension with the potential for biannual dosing.
  • The results were presented today as a late-breaking clinical trial at the 2024 American College of Cardiology (ACC) Annual Scientific Session.
  • The Company previously announced positive topline results from the KARDIA-2 study in March 2024.
  • “Although many effective oral treatments are available, a large proportion of patients with hypertension are not managed to guideline-recommended targets.

Cytokinetics Announces Four Upcoming Presentations at the American College of Cardiology 73rd Annual Scientific Session

Retrieved on: 
Monday, March 25, 2024
Cardiology, Pharmacology, Internal medicine, Safety, Associate, SAN, Cytokinetics, University, Patient, American College, Cardiomyopathy, Heart failure, Pharmaceutical industry

SOUTH SAN FRANCISCO, Calif., March 25, 2024 (GLOBE NEWSWIRE) -- Cytokinetics, Incorporated (Nasdaq: CYTK) today announced four presentations at the American College of Cardiology 73rd Annual Scientific Session (ACC.24) taking place in Atlanta, GA from April 6, 2024 – April 8, 2024.

Key Points: 
  • SOUTH SAN FRANCISCO, Calif., March 25, 2024 (GLOBE NEWSWIRE) -- Cytokinetics, Incorporated (Nasdaq: CYTK) today announced four presentations at the American College of Cardiology 73rd Annual Scientific Session (ACC.24) taking place in Atlanta, GA from April 6, 2024 – April 8, 2024.
  • Title: 1075-11 - Efficacy and Safety of Aficamten in the First Cohort of Patients with Symptomatic Obstructive Hypertrophic Cardiomyopathy Completing 1-Year Follow-Up: Findings From the FOREST-HCM Study
    Presenter: Sara Saberi, M.D., M.S., Associate Professor of Internal Medicine, University of Michigan Health Frankel Cardiovascular Center
    Session: 1226 - Heart Failure and Cardiomyopathies: Special Populations 02
    Session: 1343 - Heart Failure and Cardiomyopathies: Pharmacology 08

Tenaya Therapeutics Announces Publication of TN-401 Gene Therapy Preclinical Data in Nature Communications Medicine

Retrieved on: 
Monday, March 18, 2024
PKP2, Arrhythmogenic cardiomyopathy, Arrhythmia, Survival, Disease, Risk, Patient, SAN, Hope, Quality of life, Protein, ACM, Clinical trial, Therapy, Heart, Cardiomyopathy, Safety, Vaccine

SOUTH SAN FRANCISCO, Calif., March 18, 2024 (GLOBE NEWSWIRE) -- Tenaya Therapeutics, Inc. (NASDAQ: TNYA), a clinical-stage biotechnology company with a mission to discover, develop and deliver potentially curative therapies that address the underlying causes of heart disease, today announced the publication of its preclinical research related to its gene therapy candidate, TN-401, in the current issue of Nature Communications Medicine.

Key Points: 
  • TN-401 is an adeno-associated virus serotype 9 (AAV9)-based gene therapy being developed for the treatment of arrhythmogenic right ventricular cardiomyopathy (ARVC) caused by Plakophilin-2 (PKP2) gene mutations.
  • TN-401 is designed to deliver a functional PKP2 gene to heart cells where it works to restore normal protein levels in order to halt or even reverse disease after a single dose.
  • “Following a single infusion of our AAV9-based PKP2 gene therapy in a severe knock-out mouse model of the disease, PKP2 protein levels were restored.
  • Tenaya is currently also conducting the RIDGE™ global non-interventional natural history and serotype study of PKP2-associated ARVC.

Intellia Therapeutics Announces First Patient Dosed in the Phase 3 MAGNITUDE Study of NTLA-2001 as a Single-Dose CRISPR-Based Treatment for Transthyretin Amyloidosis with Cardiomyopathy

Retrieved on: 
Monday, March 18, 2024
Pharmacology, Royal Free Hospital, NTLA, Disease, TTR, Richmond Pharmacology, Patient, Amyloidosis, Cardiomyopathy, Intellia Therapeutics, Heart failure, Therapy, CRISPR, Safety, Phase 1, ATTR, Pharmaceutical industry

NTLA-2001 is an investigational in vivo CRISPR-based therapy designed as a single-dose treatment to inactivate the TTR gene and thereby prevent the production of TTR protein for the treatment of transthyretin (ATTR) amyloidosis.

Key Points: 
  • NTLA-2001 is an investigational in vivo CRISPR-based therapy designed as a single-dose treatment to inactivate the TTR gene and thereby prevent the production of TTR protein for the treatment of transthyretin (ATTR) amyloidosis.
  • The MAGNITUDE trial is evaluating the efficacy and safety of NTLA-2001 in patients with ATTR amyloidosis with cardiomyopathy.
  • We look forward to evaluating the efficacy and safety of NTLA-2001 in patients with cardiomyopathy in the pivotal Phase 3 trial.
  • There is remarkable interest from the ATTR amyloidosis patient community for a potential single-dose treatment, and we look forward to contributing to the advancement of novel treatment approaches.”

Edgewise Therapeutics Announces Upcoming Podium Presentation at the American College of Cardiology's 2024 Annual Scientific Session

Retrieved on: 
Thursday, March 28, 2024
Biotechnology, Pharmaceutical, Health, Cardiology, Pharmacology, Congress Center, Edgewise, Patient, Heart, Heart failure, American College, HCM, Therapy, ACC, Cardiomyopathy, Pharmaceutical industry

Edgewise Therapeutics, Inc. (Nasdaq: EWTX), a leading muscle disease biopharmaceutical company, today announced that the Company will present data on EDG-7500 at the American College of Cardiology's Annual Scientific Session (ACC.24).

Key Points: 
  • Edgewise Therapeutics, Inc. (Nasdaq: EWTX), a leading muscle disease biopharmaceutical company, today announced that the Company will present data on EDG-7500 at the American College of Cardiology's Annual Scientific Session (ACC.24).
  • The conference will take place in Atlanta, GA at the Georgia World Congress Center from April 6-8, 2024.
  • “EDG-7500 is a novel investigational therapy for the treatment of HCM with a differentiated mechanism of action,” said Kevin Koch, Ph.D., President and Chief Executive Officer, Edgewise Therapeutics.
  • “Importantly, in our preclinical studies, EDG-7500 has shown potent gradient reduction, normalization of hyperdynamic contractility, and significant improvements in ventricular filling.

Atamyo Therapeutics Obtains Regulatory Authorization in Europe to Initiate a Clinical Trial for ATA-200, its Gene Therapy to Treat Limb-Girdle Muscular Dystrophy Type 2C/R5

Retrieved on: 
Tuesday, March 26, 2024
Health, Genetics, Clinical Trials, Pharmaceutical, Cardiology, Biotechnology, CNRS, Neurology, AAV, Motivation, Environment, Scientific method, Patient, CTA, DSM-IV codes, Biomarker, Research, ANSM, University, EMA, European Medicines Agency, Clinical trial, Therapy, Muscular dystrophy, Safety, Pathology, Weakness, Cardiomyopathy, Pharmaceutical industry

This authorization was first granted by the Italian Medicines Agency (AIFA), then by the French Medicines Agency (ANSM).

Key Points: 
  • This authorization was first granted by the Italian Medicines Agency (AIFA), then by the French Medicines Agency (ANSM).
  • Giacomo Comi, Full Professor of Neurology at the University of Milan (Italy), and principal investigator of this trial.
  • ATA-200, a gene therapy candidate for LGMD2C/R5, delivers a normal copy of the gene for production of γ-sarcoglycan protein.
  • In addition to its LGMD2C/R5 gene therapy, Atamyo is developing a clinical trial with ATA-100 gene therapy for LGMD2I/R9, related to deficiencies in FKRP; and is in IND-enabling studies for LGMD2A/R1, related to deficiencies in calpain protein.

Alnylam to Webcast Investor Event to Discuss Results from KARDIA-2 Phase 2 Study of Zilebesiran at American College of Cardiology Scientific Session

Retrieved on: 
Wednesday, March 20, 2024
Science, Cardiology, Biotechnology, Research, Pharmaceutical, Managed Care, Health, Clinical Trials, Q&A, Medication, Webcast, Alnylam Pharmaceuticals, Angiotensin, American College, RNA interference, Therapy, AGT, ACC, Cardiomyopathy, Hypertension

The event will include presentations and a Q&A session with Alnylam management and Akshay Desai, M.D., Director of the Cardiomyopathy and Heart Failure Program, Brigham and Women’s Hospital.

Key Points: 
  • The event will include presentations and a Q&A session with Alnylam management and Akshay Desai, M.D., Director of the Cardiomyopathy and Heart Failure Program, Brigham and Women’s Hospital.
  • A replay of the call will be available two hours after the call and archived on the same webpage for six months.
  • A live audio webcast of the call will be available on the Investors section of the Company’s website at www.alnylam.com/events .
  • An archived webcast will be available on the Company’s website approximately two hours after the event.

Stealth BioTherapeutics Presents Data of Novel Compound, SBT-589, in Friedreich's Ataxia Cardiac Models at the Wellcome Trust Mitochondrial Medicine Conference

Retrieved on: 
Tuesday, March 19, 2024
Discovery, Friedreich's ataxia, Death, Hypertrophy, Conditional sentence, Conference, Disease, Mitochondrion, The Company, Mouse, Mortality, Cardiomyopathy, Senior, Heart failure, Mitochondrial disease, Pathology, Stealth, Pharmaceutical industry

NEEDHAM, Mass., March 19, 2024 /PRNewswire/ -- Stealth BioTherapeutics Inc. (the "Company" or "Stealth"), a clinical-stage biotechnology company focused on the discovery, development, and commercialization of novel therapies for diseases involving mitochondrial dysfunction, announced today the presentation of new SBT-589 data demonstrating cardioprotective effects across pre-clinical models of Friedreich's ataxia (FA). The data were presented at the Wellcome Trust Conference on Mitochondrial Medicine – Therapeutic Development, held March 18-20, 2024, in Cambridge, England.

Key Points: 
  • The data were presented at the Wellcome Trust Conference on Mitochondrial Medicine – Therapeutic Development, held March 18-20, 2024, in Cambridge, England.
  • SBT-589 is a promising novel molecule that acts on mitochondrial pathways essential for cellular health and energy production that are impaired in FA cardiomyopathy.
  • "Mitigating heart disease in FA is central to Stealth's ongoing efforts to improve the lives of individuals with FA.
  • The data were presented by Dr. Laura E. Kropp, Senior Manager of Discovery Biology at Stealth, and recipient of the Keith Michael Andrus Cardiac Award from the Friedreich's Ataxia Research Alliance.

Intellia Therapeutics Announces Fourth Quarter and Full-Year 2023 Financial Results and Highlights Recent Company Progress

Retrieved on: 
Thursday, February 22, 2024
Research, Intellia Therapeutics, Regeneron Pharmaceuticals, Fatigue, Haemophilia, NK, CTA, Liver, Cystic fibrosis, ATM, A1AT, Amyloidosis, HAE, Therapy, European Medicines Agency, ATTR, Haemophilia B, DNA, European Commission, Hereditary angioedema, Recode, Conference, Cardiomyopathy, AATD, Civil service commission, Clinical trial, Facial muscles, CRISPR, Gene editing, Food, NTLA, KLKB1, Lung, NEJM, TTR, Patient, Medicine, Disease, Tissue, Pharmaceutical industry

Collaboration Revenue: Collaboration revenue decreased by $15.5 million to negative $1.9 million during the fourth quarter of 2023, compared to $13.6 million during the fourth quarter of 2022.

Key Points: 
  • Collaboration Revenue: Collaboration revenue decreased by $15.5 million to negative $1.9 million during the fourth quarter of 2023, compared to $13.6 million during the fourth quarter of 2022.
  • R&D Expenses: Research and development expenses increased by $9.0 million to $109.0 million during the fourth quarter of 2023, compared to $100.0 million during the fourth quarter of 2022.
  • G&A Expenses: General and administrative expenses increased by $5.4 million to $29.0 million during the fourth quarter of 2023, compared to $23.6 million during the fourth quarter of 2022.
  • Net Loss: The Company’s net loss was $132.2 million for the fourth quarter of 2023, compared to $113.4 million during the fourth quarter of 2022.