Moyamoya disease

NewAmsterdam Pharma Enrolls Over 9,000 Patients in Pivotal Phase 3 PREVAIL Global Cardiovascular Outcome Trial Evaluating the Effect of Obicetrapib in Patients with Established Atherosclerotic Cardiovascular Disease

Retrieved on: 
Tuesday, April 9, 2024
Disability, Cardiovascular disease, Myocardial infarction, CVD, Risk, Patient, Stroke, NAMS, History, Mortality, Death, FESC, Moyamoya disease, Phase 3, Monash University, MACE, ASCVD, Hypercholesterolemia, Pharmaceutical industry

Driven by strong patient and physician interest globally, NewAmsterdam will extend enrollment to the end of April where we expect to randomize over 9,000 patients.

Key Points: 
  • Driven by strong patient and physician interest globally, NewAmsterdam will extend enrollment to the end of April where we expect to randomize over 9,000 patients.
  • “PREVAIL, our pivotal CVOT, will potentially demonstrate that obicetrapib’s lowering of LDL-C will reduce major adverse cardiac events (“MACE”).
  • The primary objective of the study is to evaluate the effect of obicetrapib compared to placebo on MACE, including cardiovascular death, myocardial infarction, stroke and non-elective coronary revascularization.
  • “Despite widespread availability of statin therapies, CVD-related deaths are on the rise, and many patients are not at their risk-based LDL-C goals.

iCAD’s ProFound AI Suite Uncovers Hidden Heart Disease Risk According to New Data Presented at American College of Cardiology Meeting

Retrieved on: 
Monday, April 8, 2024
Prevalence, Cardiovascular disease, Sudden death, Radiology, Blood, Risk, Physician, ICAD, Patient, Ageing, Stroke, Research, Breast, Angiography, Death, Woman, American College, Breast cancer, Literature, Prognosis, Moyamoya disease, ACC, Medical imaging

The 15-site prospective study included 2D screening mammograms from women ages 20 to 100, with a median age of 56.

Key Points: 
  • The 15-site prospective study included 2D screening mammograms from women ages 20 to 100, with a median age of 56.
  • “The ProFound BAC AI algorithm may provide a critical surrogate biomarker for women at risk of heart disease or stroke,” said Dana Brown, president and CEO of iCAD.
  • The study also suggests the ProFound Heart Health AI algorithm can standardize BAC detection, potentially improving efficiency and reducing variability among observers.
  • Early cardiovascular disease detection is key, as among asymptomatic women, the first manifestation of underlying coronary heart disease is often acute myocardial infarction (MI) or sudden death.

Verve Therapeutics Announces Updates on its PCSK9 Program

Retrieved on: 
Tuesday, April 2, 2024
LDLR, Cardiovascular disease, Gene editing, PCSK9, ALT, Patient, RNA, Health care, Health Canada, IND, N-Acetylgalactosamine, MHRA, Asialoglycoprotein receptor, Clinical trial, CTAS, Therapy, Bleeding, Safety, Moyamoya disease, DSMB, FDA, Liver, ASCVD, LNP, Medsafe, Hypercholesterolemia, Food, Pharmaceutical industry

BOSTON, April 02, 2024 (GLOBE NEWSWIRE) -- Verve Therapeutics, a clinical-stage biotechnology company pioneering a new approach to the care of cardiovascular disease with single-course gene editing medicines, today announced updates from the Heart-1 Phase 1b clinical trial of VERVE-101 and clearance of its Clinical Trial Applications (CTAs) by the U.K. Medicines and Healthcare products Regulatory Agency (MHRA) and Health Canada for VERVE-102, with the Heart-2 Phase 1b clinical trial expected to initiate in the second quarter of this year.

Key Points: 
  • Six participants have been dosed at 0.45 mg/kg of VERVE-101, with a total of 13 participants dosed in the study.
  • Verve is conducting an investigation into the laboratory abnormalities and based on those results, expects to work with regulatory authorities to define a path forward for VERVE-101.
  • Verve is now prioritizing the development of VERVE-102 and the initiation of the Heart-2 clinical trial.
  • VERVE-102 uses the same base editor and guide RNA for PCSK9 but a different lipid nanoparticle (LNP) delivery system than VERVE-101.

Exponential Health Partners with Cardio Diagnostics to Elevate Cardiovascular Disease Prevention in Michigan

Retrieved on: 
Tuesday, April 9, 2024
Cardiology, Biotechnology, Technology, Health, General Health, Pharmaceutical, Health Technology, Other Technology, Medical Devices, Artificial Intelligence, Genetics, Diagnosis, Exponential, Cardio, Genomics, Cardiovascular disease, Growth, Entrepreneurship, Partnership, Risk, Patient, Artificial intelligence, Splenic infarction, Death, CHD, Medicine, Concierge medicine, Epigenomics, Moyamoya disease, USD, Medical device

As a result, Exponential Health is expanding access to a new chapter in the proactive management of heart disease.

Key Points: 
  • As a result, Exponential Health is expanding access to a new chapter in the proactive management of heart disease.
  • Concierge practices such as Exponential Health are leading the way in adopting epigenetic cardiovascular solutions.
  • "We are thrilled to partner with Dr. Baer and Exponential Health," said Meesha Dogan, Ph.D., CEO and Co-Founder of Cardio Diagnostics.
  • Integrating Cardio Diagnostics' advanced tests into Exponential Health's offerings is a proactive step toward mitigating this global health challenge.

Silence Therapeutics Announces JAMA Publication of Additional Phase 1 Data for Zerlasiran in Subjects with Elevated Lipoprotein(a)

Retrieved on: 
Monday, April 8, 2024
Research, FDA, Genetics, Clinical Trials, Cardiology, Biotechnology, Health, Pharmaceutical, Science, JAMA, American Medical Association, Cardiovascular disease, Serum, Patient, Journal, RNA, MD, Therapy, Moyamoya disease, Safety, Small RNA, APOLLO, ASCVD, Pharmaceutical industry

“Positive phase 1 data published in JAMA demonstrate treatment with zerlasiran produced sustained reductions in Lp(a) concentrations with a well-tolerated profile using varying dosing regimens,” said Curtis Rambaran, MD, Chief Medical Officer at Silence and senior author of the publication.

Key Points: 
  • “Positive phase 1 data published in JAMA demonstrate treatment with zerlasiran produced sustained reductions in Lp(a) concentrations with a well-tolerated profile using varying dosing regimens,” said Curtis Rambaran, MD, Chief Medical Officer at Silence and senior author of the publication.
  • Results from the single ascending dose portion of the trial in healthy participants were previously published in the April 2022 issue of JAMA, linked here .
  • Secondary outcomes included the serum levels of zerlasiran and effects on Lp(a) serum concentrations.
  • Zerlasiran is currently being evaluated in the ALPACAR-360 phase 2 study in subjects with baseline Lp(a) levels at or over 125 nmol/L at high risk of ASCVD events.

Leading Cardiologists from NYU Langone Heart Present Latest Clinical Findings & Research at American College of Cardiology 73rd Annual Scientific Session

Retrieved on: 
Friday, April 5, 2024
Diagnosis, Internal medicine, Ozone, Cardiovascular disease, Eugene Braunwald, System, N95, Blood, Digital, Assistant, Memory, Risk, Diabetes, Inflammation, Patient, Biomarker, Research, Moyamoya disease, Cardiovascular Research, Splenic infarction, NYU, Ischemia, IMR, Particulates, Woman, Heart failure, American College, MPH, CFR, NYU Langone Health, EHR, American Red Cross, Bangladesh Technical Education Board, Air pollution, Data, Osteoarthritis, ACC, CCD, Work, Physician, Medical device

NEW YORK, April 5, 2024 /PRNewswire/ -- Experts from NYU Langone Heart are presenting their latest clinical findings and research discoveries during the American College of Cardiology (ACC) 2024 Annual Scientific Session & Expo, held April 6 to 8 in Atlanta.

Key Points: 
  • NEW YORK, April 5, 2024 /PRNewswire/ -- Experts from NYU Langone Heart are presenting their latest clinical findings and research discoveries during the American College of Cardiology (ACC) 2024 Annual Scientific Session & Expo, held April 6 to 8 in Atlanta.
  • "The ACC conference serves as a place for our faculty to explore, learn, and share all the latest advancements in the field.
  • They will also be available for media interviews to discuss innovative and noteworthy scientific developments emerging from the conference.
  • Her research is focused on understanding what makes heart attacks in women unique and the best methods of detection and treatment.

NewAmsterdam Pharma to Present New Analysis from the Phase 2 ROSE2 Trial Evaluating Obicetrapib in Combination with Ezetimibe at 2024 American College of Cardiology Congress

Retrieved on: 
Monday, March 25, 2024
Moyamoya disease, Cardiovascular disease, Congress, Risk, Patient, NAMS, American College, Ezetimibe, Clinical trial, FDC, ACC, ASCVD

ROSE2 evaluated obicetrapib in combination with ezetimibe as an adjunct to high-intensity statin therapy; full results from the trial were previously presented at the National Lipid Association Scientific Sessions in June 2023.

Key Points: 
  • ROSE2 evaluated obicetrapib in combination with ezetimibe as an adjunct to high-intensity statin therapy; full results from the trial were previously presented at the National Lipid Association Scientific Sessions in June 2023.
  • At ACC, NewAmsterdam will share new data demonstrating obicetrapib and ezetimibe’s impact on small dense LDL-C, a robust predictor of residual risk of future cardiovascular events.
  • NewAmsterdam recently initiated TANDEM, a pivotal Phase 3 clinical trial to evaluate obicetrapib and ezetimibe FDC (‘fixed-dose combination”) in adult patients with Heterozygous Familial Hypercholesterolemia (“HeFH”) and/or Atherosclerotic Cardiovascular Disease (“ASCVD”) or multiple risk factors for ASCVD, whose LDL-C is not adequately controlled despite being on maximally tolerated lipid-modifying therapies.
  • Details of the presentation are as follows:
    Presentation at the 2024 American College of Cardiology (ACC) Congress:

CHMP Issues Positive Opinions For Both Bempedoic Acid And The Bempedoic Acid / Ezetimibe Fixed-Dose Combination Tablet As Treatments For Hypercholesterolemia And Significantly Reducing Cardiovascular Events

Retrieved on: 
Friday, March 22, 2024
Urinary tract infection, Anaphylaxis, Bronchitis, European Commission, Tendon rupture, Patient, European Medicines Agency, Bempedoic acid, Ezetimibe, Therapy, Citrate synthase, Anemia, Pain, Sinusitis, Abdominal pain, Risk, Gout, Ageing, Committee, ACL, Spasm, EMA, Back pain, ATP, Safety, Fatigue, CHMP, Arthralgia, Angioedema, Tablet, Constipation, Agent handling, Death, Pregnancy, CLEAR, Rash, Kidney, Moyamoya disease, Rupture, Apolipoprotein, Hypercholesterolemia, Hypersensitivity, Health, CVD, Incidence, Diarrhea, Fetus, Esperion Therapeutics, Diet, Upper respiratory tract infection, FDC, Uric acid

ANN ARBOR, Mich. and MUNICH, Germany, March 22, 2024 (GLOBE NEWSWIRE) -- Daiichi Sankyo Europe GmbH (hereafter, ‘Daiichi Sankyo’) and Esperion Therapeutics, Inc. jointly announced today, that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted positive opinions for the label update of both bempedoic acid (marketed as NILEMDO®▼) and the bempedoic acid / ezetimibe fixed dose combination (FDC) (marketed as NUSTENDI®▼ ), recommending their approval as treatments to reduce low-density lipoprotein cholesterol (LDL-C) and cardiovascular risk.2 The existing label of bempedoic acid (NILEMDO®▼) provides authorisation for adults with primary hypercholesterolaemia (heterozygous familial and non-familial) or mixed dyslipidaemia, as an adjunct to diet:2

Key Points: 
  • “We are thrilled with the positive CHMP opinion, which reflects the significant cardiovascular risk reduction benefit that the bempedoic acid global franchise brings to patients worldwide,” said Sheldon Koenig, President and CEO, Esperion.
  • The CHMP is a scientific committee of the EMA that reviews medical product applications on their scientific and clinical merit.
  • Hyperuricemia: Bempedoic acid, a component of NEXLIZET and NEXLETOL, may increase blood uric acid levels which may lead to gout.
  • CLEAR Outcomes is part of the CLEAR clinical research program for NEXLETOL® (bempedoic acid) Tablet and NEXLIZET® (bempedoic acid and ezetimibe) Tablet.

Tourmaline Bio Reports Fourth Quarter and Full Year 2023 Financial Results and Recent Business Highlights

Retrieved on: 
Tuesday, March 19, 2024
Research, Moyamoya disease, Quarter, Cardiovascular disease, Spirited, Growth, Exercise, Entrepreneurship, IL-6, Inflammation, Patient, SVP, Business development, Commercial, NASDAQ Biotechnology Index, MD, IND, Investment, Clinical trial, NBI, Investigational New Drug, Therapy, TRML, Medical research, Graves' ophthalmopathy, FDA, Food, Pharmaceutical industry

In October 2023, Tourmaline become a publicly-traded company via reverse merger with Talaris Therapeutics, including a concurrent private placement of $75.0 million.

Key Points: 
  • In October 2023, Tourmaline become a publicly-traded company via reverse merger with Talaris Therapeutics, including a concurrent private placement of $75.0 million.
  • Fourth Quarter and Full Year 2023 Financial Results:
    Cash, cash equivalents and investments were $203.0 million as of December 31, 2023, as compared to $8.3 million as of December 31, 2022.
  • Research and development expenses were $8.0 million for the fourth quarter of 2023, as compared to $3.8 million for the fourth quarter of 2022.
  • General and administrative expenses were $6.9 million for the fourth quarter of 2023, as compared to $1.1 million for the fourth quarter of 2022.

NewAmsterdam Pharma Doses First Patient in Phase 3 TANDEM Clinical Trial Evaluating Fixed-Dose Combination of Obicetrapib and Ezetimibe in Patients with HeFH and/or ASCVD

Retrieved on: 
Tuesday, March 12, 2024
Consensus, Moyamoya disease, Cardiovascular disease, Myocardial infarction, Dyslipidemia, CVD, Cardiology, Risk, Patient, Apolipoprotein B, Consensus decision-making, Stroke, NAMS, ASCVD, Plaque, Artery, Diet, Cleveland Clinic, Ezetimibe, Clinical trial, Safety, FDC, Apolipoprotein, Genetic disorder, Physician, Pharmaceutical industry

NAARDEN, The Netherlands and MIAMI, March 12, 2024 (GLOBE NEWSWIRE) -- NewAmsterdam Pharma Company N.V. (Nasdaq: NAMS or “NewAmsterdam” or the “Company”), a late-stage, clinical biopharmaceutical company developing oral, non-statin medicines for patients at risk of cardiovascular disease (“CVD”) with elevated low-density lipoprotein cholesterol (“LDL-C”), for whom existing therapies are not sufficiently effective or well-tolerated, today announced the dosing of the first patient and initiation of TANDEM, a pivotal Phase 3 clinical trial to evaluate obicetrapib and ezetimibe FDC in adult patients with Heterozygous Familial Hypercholesterolemia (“HeFH”) and/or Atherosclerotic Cardiovascular Disease (“ASCVD”) or multiple risk factors for ASCVD, whose LDL-C is not adequately controlled despite being on maximally tolerated lipid-modifying therapies.

Key Points: 
  • The ACC Expert Consensus set LDL-C goals of below the 70mg/dl for patients with ASCVD and below the 55mg/dl goal for ASCVD patients with very high risk.
  • The primary objective of the placebo-controlled, double-blind, four-arm, randomized TANDEM trial is to evaluate the effect of 10mg obicetrapib and 10mg ezetimibe FDC on LDL-C levels, compared to both ezetimibe 10mg and obicetrapib 10mg monotherapy and to placebo.
  • NewAmsterdam anticipates enrolling approximately 400 patients on maximally tolerated lipid-modifying therapies with HeFH, ASCVD or ASCVD risk equivalents and who have a baseline LDL-C of at least 70 mg/Dl.
  • Patients who have multiple risk factors for ASCVD are at high risk of experiencing a cardiovascular event in the near future.