PCSK9

LIB Therapeutics Announces Positive Lerodalcibep Results from Two Phase 3 LIBerate Studies at the 92nd European Atherosclerosis Society Congress

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水曜日, 5月 29, 2024
Other Health, Research, Managed Care, Pharmaceutical, Clinical Trials, Science, Cardiology, Biotechnology, FDA, Other Science, Health, Diabetes, Patient, Cardiovascular disease, Doctor of Philosophy, EAS, Safety, PCSK9, Ezetimibe, MD, Week, CVD, Apolipoprotein, Concentration ratio, History, Liberation, Female, Pharmaceutical industry

LIB Therapeutics Inc. (LIB), a privately-held, late-stage biopharmaceutical company advancing Lerodalcibep, a novel, once-monthly, LDL-cholesterol lowering, third-generation PCSK9 inhibitor, today announced positive results from two studies in the recently completed Phase 3 LIBerate registration-enabling program were presented during the 92nd European Atherosclerosis Society Congress in Lyon, France, May 26-29, 2024.

Key Points: 
  • LIB Therapeutics Inc. (LIB), a privately-held, late-stage biopharmaceutical company advancing Lerodalcibep, a novel, once-monthly, LDL-cholesterol lowering, third-generation PCSK9 inhibitor, today announced positive results from two studies in the recently completed Phase 3 LIBerate registration-enabling program were presented during the 92nd European Atherosclerosis Society Congress in Lyon, France, May 26-29, 2024.
  • The co-primary efficacy endpoints were the percent change from baseline in LDL-C at Week 52 and the mean of Weeks 50 and 52.
  • Secondary outcomes included achievement of EAS / ESC LDL-C targets, and other lipid and apolipoproteins changes.
  • Patients were randomized 2:1 to a single 300 mg once-monthly, subcutaneous dose of Lerodalcibep or placebo for 52 weeks.

New Campaign from The Trial for #ClinicalEquality – “Worth Less” –Spotlights the Cost of Clinical Inequality

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月曜日, 5月 20, 2024
Medicine, Diabetes, Disease, Patient, Doctor of Philosophy, Native Americans, Trial, Clinical trial, National academy, PCSK9, Hypertension, Research, Mortality, Physician, Pharmaceutical industry

“While there has been progress among clinical trial sponsors since we launched in 2020 – with many influential partners joining us to bring this conversation to some of the largest stages in the world – there’s still more work to be done, particularly as we look at the full burden, or cost, that a lack of diversity in clinical research has.”

Key Points: 
  • “While there has been progress among clinical trial sponsors since we launched in 2020 – with many influential partners joining us to bring this conversation to some of the largest stages in the world – there’s still more work to be done, particularly as we look at the full burden, or cost, that a lack of diversity in clinical research has.”
    Based on the National Academies report, health disparities in heart disease, hypertension and diabetes and the resulting mortality, morbidity and loss of work will cost society more than $5 trillion through to 2050.
  • Better representation in clinical trials would lead to reductions in these health disparities, which would save all US taxpayers billions of dollars.1
    When clinical trials are not inclusive, clinical trial sponsors fail to acquire vital scientific data from a diverse patient population, which could lead to important discoveries for the broader population.1 For instance, PCSK9 inhibitors, a class of cholesterol-lowering medicines expected to hit a $2 billion valuation in 2030, owe their discovery in part to diverse clinical trials.
  • In Black patients, scientists discovered a variant of the PCSK9 gene that is associated with lower cholesterol and as a result identified this gene as an important target for treatment.2,3
    Patients of color are disproportionately impacted by diseases such as diabetes and heart disease and yet poorly represented in clinical trials for those conditions.
  • 4–7 For example, 0% of clinical trial participants are Native American, yet diabetes hits their communities the hardest, depriving at-risk groups of life-improving and/or life-saving therapies, which increase the disease burden on their families and communities.4,5
    A lack of diverse representation in research further degrades trust among underrepresented groups and healthcare professionals and has the potential to further worsen healthcare disparities.8,9 More than 70% of physicians in the US are being asked a question by their patients that they cannot answer for all: Will this new medicine work in people like me?9

LIB Therapeutics Announces Lerodalcibep Abstracts Accepted for Presentation at the 92nd European Atherosclerosis Society Congress

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木曜日, 5月 23, 2024
Research, Diabetes, Clinical Trials, Cardiology, Biotechnology, Health, Pharmaceutical, Other Science, Science, High Risk, PCSK9, Public, CVD, Safety, Abstract, Moyamoya disease, Liberation, Pharmaceutical industry

LIB Therapeutics Inc. (LIB), a privately-held, late-stage biopharmaceutical company advancing Lerodalcibep, a novel, LDL-cholesterol lowering, third-generation PCSK9 inhibitor, today announced acceptance of two abstracts from the recently completed Phase 3 registration-enabling LIBerate program for presentation at the 92nd European Atherosclerosis Society Congress in Lyon, France on May 26-29, 2024.

Key Points: 
  • LIB Therapeutics Inc. (LIB), a privately-held, late-stage biopharmaceutical company advancing Lerodalcibep, a novel, LDL-cholesterol lowering, third-generation PCSK9 inhibitor, today announced acceptance of two abstracts from the recently completed Phase 3 registration-enabling LIBerate program for presentation at the 92nd European Atherosclerosis Society Congress in Lyon, France on May 26-29, 2024.
  • Oral Presentation: Science at a Glance Session - Late-Breaking Lipids; Moderated Poster Station 8; “Randomized, Open-Label, Study Comparing Efficacy and Safety of Lerodalcibep to Inclisiran in Patients with CVD or at High Risk for CVD Requiring Additional LDL-Cholesterol Reduction”; presented by Prof. Ulrich Laufs (Germany), May 28, 1:30 PM – 1:37 PM CET
    Oral Presentation: Late Breaker Session 2 - New therapeutic agents; René Leriche Hall - Forum 2; “Long Term Efficacy and Safety of Lerodalcibep in Patients with Atherosclerotic Cardiovascular Disease (LIBerate-CVD)”; presented by Dr. Evan Stein, May 29, 11:00 AM – 11:15 AM CET

BioAge Labs Announces Inaugural Scientific Advisory Board to Support Advancement of Novel Therapeutics Targeting Metabolic Aging

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水曜日, 5月 15, 2024
Cardiology, Biotechnology, Other Health, Health, General Health, Seniors, Pharmaceutical, Research, Genetics, Consumer, Science, GLP1R, Diabetes, Biology, MASH, Translational medicine, Obesity, Metformin, Drug development, GSK, FACP, Senior, Harvard Medical School, Body composition, Inflammation, Geriatrics, Type 2 diabetes, The Obesity Society, DSM-IV codes, Sarcopenia, Publication, Ageing, Duke University, FGF21, Internal medicine, Clinical trial, PCSK9, Associate, Human nutrition, Weight loss, SAB, Obesity medicine, Patient, UC, Amgen, Exercise, Therapy, Longevity, Apelin, University of California, Berkeley, Pharmaceutical industry

BioAge Labs, Inc. ("BioAge"), a clinical-stage biotechnology company developing therapeutic candidates for metabolic diseases, such as obesity, by targeting the biology of aging, today announced the formation of its Scientific Advisory Board (SAB).

Key Points: 
  • BioAge Labs, Inc. ("BioAge"), a clinical-stage biotechnology company developing therapeutic candidates for metabolic diseases, such as obesity, by targeting the biology of aging, today announced the formation of its Scientific Advisory Board (SAB).
  • The SAB is composed of world-renowned leaders with expertise spanning metabolic disease biology, obesity medicine, and clinical development of blockbuster therapeutics.
  • The SAB will work closely with BioAge's leadership team to advance the company's pipeline of novel treatments for age-related chronic metabolic diseases into mid- and late-stage clinical development.
  • “We will also leverage their broad expertise in drug development for metabolic diseases to guide the continued advancement of the other programs in our metabolic disease pipeline.”

Arbor Biotechnologies Presents Data Supporting Clinical Development of ABO-101 and Robust Potential of Platform to Enable Therapeutic Programs at the American Society of Gene and Cell Therapy (ASGCT) 27th Annual Meeting

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木曜日, 5月 9, 2024
Mouse, CRISPR, Impact, Compact, Gene editing, Cell, Artificial intelligence, CGT, PCSK9, IND, CNS, TDP43, PH1, Therapy, Patient, Liver, Gene, Society, ALS, Brain cell, Central nervous system, Messenger, HAO1, NHP, Annual general meeting, Pharmacology, Engineering, Medical device

“This approach has fueled our robust pipeline of genetic medicines which address a range of liver and CNS indications, including PH1 and ALS.

Key Points: 
  • “This approach has fueled our robust pipeline of genetic medicines which address a range of liver and CNS indications, including PH1 and ALS.
  • The data show highly specific targeting of the HAO1 gene in the liver and preservation of genomic integrity upon editing.
  • Together, this strong preclinical data package and the ongoing IND-enabling studies support continued advancement of ABO-101 toward clinical evaluation.
  • Details for the presentations are as follows:
    Oral Presentation Title: Identification and Engineering of ABR-004, a Compact, High-fidelity Nuclease for Therapeutic Gene Editing

NewAmsterdam Pharma Provides Corporate Update and Reports First Quarter Financial Results

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木曜日, 5月 9, 2024
Congress, American College, Investment, Risk, ASCVD, CVD, Research, Moyamoya disease, Research and development, Partnership, Conference, PCSK9, Hypercholesterolemia, History, Growth, Administration, Cardiology, Patient, Physician, LDL, Cardiovascular disease, NAMS, Standard of care, Phase 3, Clinical trial, Biomarker, Drug discovery, Partner, Pharmaceutical industry

-- Strong financial position; ending the quarter with $481.1 million in cash --

Key Points: 
  • Driven by strong patient and physician interest globally, NewAmsterdam extended enrollment to the end of April and randomized over 9,500 patients.
  • In January 2024, NewAmsterdam appointed William H. Lewis, J.D., M.B.A as Chair of its Board of Directors.
  • NewAmsterdam currently expects to achieve the following upcoming milestones:
    Announce topline data from the Phase 3 BROOKLYN trial for obicetrapib monotherapy in the third quarter of 2024.
  • Revenue: NewAmsterdam recognized $1.4 million in revenue for the quarter ended March 31, 2024, compared to $8.6 million in the same period in 2023.

Verve Therapeutics Announces Pipeline Progress and Reports First Quarter 2024 Financial Results

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水曜日, 5月 8, 2024
LDLR, Verve, Safety, Pharmacokinetics, ASCVD, LNP, Eli Lilly, Moyamoya disease, N-Acetylgalactosamine, RNA, Aortic stenosis, Stroke, Cell, Conference, Research, Webcast, PCSK9, IND, Administration, Potential, Research and development, Patient, Liver, Gene, CAD, Society, Medicine, ANGPTL3, Lilly, Cardiovascular disease, CTAS, Therapy, Asialoglycoprotein receptor, G&A, LPA, Pharmaceutical industry

BOSTON, May 08, 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 reported pipeline updates and financial results for the quarter ended March 31, 2024.

Key Points: 
  • Cash Position: Verve ended the first quarter of 2024 with $606.4 million in cash, cash equivalents, and marketable securities.
  • Collaboration Revenue: Collaboration revenue was $5.7 million for the first quarter of 2024, compared to $1.4 million for the first quarter of 2023.
  • General & Administrative (G&A) Expenses: G&A expenses were $14.2 million for the first quarter of 2024, compared to $12.6 million for the first quarter of 2023.
  • Stock-based compensation expense included in G&A expenses was $4.7 million and $3.5 million for the first quarter of 2024 and 2023, respectively.

Verve Therapeutics Announces Dosing of First Patient in Heart-2 Phase 1b Clinical Trial Evaluating VERVE-102

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火曜日, 5月 7, 2024
LDLR, Patient, Clinical trial, Risk, Safety, Woman, Pharmacokinetics, Blood, ASCVD, Gene, Moyamoya disease, Gene editing, RNA, PCSK9, Life, Ageing, Liver, CAD, Cardiovascular disease, Therapy, Asialoglycoprotein receptor, DSM-IV codes, Apolipoprotein, Sudden death, Pharmaceutical industry

BOSTON, May 07, 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 that the first patient has been dosed with VERVE-102 in the Heart-2 Phase 1b clinical trial. The Heart-2 trial is enrolling adult patients with heterozygous familial hypercholesterolemia (HeFH) or premature coronary artery disease (CAD), two patient populations who require deep reductions of low-density lipoprotein cholesterol (LDL-C) levels in the blood for an extended period of time. Patients living with HeFH have an inherited disorder characterized by elevated blood levels of LDL-C starting early in life. Patients living with premature CAD experience cholesterol-driven blockage of coronary arteries early in life and are at high risk of further complications. A lack of durable control of LDL-C levels in both HeFH and premature CAD patients carries high lifetime risks for cardiovascular events, including heart attack and sudden death.

Key Points: 
  • Patients living with HeFH have an inherited disorder characterized by elevated blood levels of LDL-C starting early in life.
  • Patients living with premature CAD experience cholesterol-driven blockage of coronary arteries early in life and are at high risk of further complications.
  • “Dosing the first patient in the Heart-2 Phase 1b clinical trial for VERVE-102 is an important step in the continued progress of our pipeline,” said Sekar Kathiresan, M.D., co-founder and chief executive officer of Verve Therapeutics.
  • Clinical Trial Applications for the Heart-2 trial have been cleared in Canada and the United Kingdom.

Arbor Biotechnologies to Present Data Supporting Therapeutic Programs in PH1 and ALS, and the Discovery of a Novel Type V Nuclease at the American Society of Gene and Cell Therapy (ASGCT) 27th Annual Meeting

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月曜日, 4月 22, 2024
CRISPR, Impact, Compact, Cell, CGT, PCSK9, PH1, STMN2, Liver, Gene, Society, ALS, Messenger, HAO1, NHP, Engineering, Medical device

CAMBRIDGE, Mass., April 22, 2024 (GLOBE NEWSWIRE) -- Arbor Biotechnologies®, a biotechnology company discovering and developing the next generation of genetic medicines, today announced four upcoming presentations at the 2024 American Society of Gene and Cell Therapy (ASGCT) 27th Annual Meeting, taking place May 7-11 in Baltimore, Maryland.

Key Points: 
  • Arbor will present in vivo NHP data supporting clinical development of ABO-101, its most advanced gene editing therapeutic candidate designed to address primary hyperoxaluria type 1 (PH1) through inactivation of the HAO1 gene in the liver.
  • The company also will present data supporting its end-to-end nuclease development platform in a third presentation outlining the discovery and optimization of a unique, compact nuclease, termed ABR-004.
  • Proof-of-concept data in non-human primates show potent, therapeutically relevant silencing of PCSK9 in vivo with the novel nuclease, signaling opportunities for broader therapeutic applications.
  • Together, the suite of presentations will demonstrate the utility of Arbor’s proprietary discovery and development approach for enabling efficient identification and optimization of novel nucleases.

Verve Therapeutics Announces Updates on its PCSK9 Program

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火曜日, 4月 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.