Latest news with #LDL-C
Yahoo
2 days ago
- Business
- Yahoo
FDA approves YolTech's YOLT-101 for familial hypercholesterolemia
YolTech Therapeutics has received the US Food and Drug Administration (FDA) approval for its investigational new drug (IND) application for YOLT-101 to treat heterozygous familial hypercholesterolemia (HeFH). YOLT-101 is an in vivo base editing therapy designed to target proprotein convertase subtilisin/kexin type 9 (PCSK9). Its innovative approach lies in its potential to durably reduce blood low-density lipoprotein cholesterol (LDL-C) levels through a single-dose treatment. The technology behind YOLT-101 incorporates YolTech's adenine base editor, known as hpABE5, which consists of nCas and a new deaminase evolved from Hafnia paralvei. To deliver this therapeutic agent, the company employs an advanced lipid nanoparticle (LNP) delivery system. hpABE5 can perform precise A•T to G•C base conversions without inducing DNA double-strand breaks, thus minimising risks associated with chromosomal abnormalities and off-target effects. YolTech Therapeutics co-founder and CEO Yuxuan Wu stated: 'The FDA IND clearance marks a significant milestone for YolTech. In vivo gene editing represents a new generation of therapeutics — offering one-time, durable solutions for chronic and genetic diseases. 'We are committed to advancing breakthrough gene editing solutions that offer transformative benefits for patients living with severe genetic and cardiovascular diseases.' Currently under evaluation in an ongoing investigator-initiated trial (IIT), YOLT-101 has shown promising results with a favourable safety profile and substantial LDL-C–lowering effects. Familial hypercholesterolemia is a genetic disorder stemming from mutations affecting LDL metabolism-related genes such as LDLR, Apolipoprotein B (APOB) and PCSK9. Individuals suffering from FH typically experience elevated LDL-C levels, which contribute to a heightened risk of developing early-onset atherosclerotic cardiovascular diseases. "FDA approves YolTech's YOLT-101 for familial hypercholesterolemia" was originally created and published by Pharmaceutical Technology, a GlobalData owned brand. The information on this site has been included in good faith for general informational purposes only. It is not intended to amount to advice on which you should rely, and we give no representation, warranty or guarantee, whether express or implied as to its accuracy or completeness. You must obtain professional or specialist advice before taking, or refraining from, any action on the basis of the content on our site.
Yahoo
2 days ago
- Business
- Yahoo
FDA approves YolTech's YOLT-101 for familial hypercholesterolemia
YolTech Therapeutics has received the US Food and Drug Administration (FDA) approval for its investigational new drug (IND) application for YOLT-101 to treat heterozygous familial hypercholesterolemia (HeFH). YOLT-101 is an in vivo base editing therapy designed to target proprotein convertase subtilisin/kexin type 9 (PCSK9). Its innovative approach lies in its potential to durably reduce blood low-density lipoprotein cholesterol (LDL-C) levels through a single-dose treatment. The technology behind YOLT-101 incorporates YolTech's adenine base editor, known as hpABE5, which consists of nCas and a new deaminase evolved from Hafnia paralvei. To deliver this therapeutic agent, the company employs an advanced lipid nanoparticle (LNP) delivery system. hpABE5 can perform precise A•T to G•C base conversions without inducing DNA double-strand breaks, thus minimising risks associated with chromosomal abnormalities and off-target effects. YolTech Therapeutics co-founder and CEO Yuxuan Wu stated: 'The FDA IND clearance marks a significant milestone for YolTech. In vivo gene editing represents a new generation of therapeutics — offering one-time, durable solutions for chronic and genetic diseases. 'We are committed to advancing breakthrough gene editing solutions that offer transformative benefits for patients living with severe genetic and cardiovascular diseases.' Currently under evaluation in an ongoing investigator-initiated trial (IIT), YOLT-101 has shown promising results with a favourable safety profile and substantial LDL-C–lowering effects. Familial hypercholesterolemia is a genetic disorder stemming from mutations affecting LDL metabolism-related genes such as LDLR, Apolipoprotein B (APOB) and PCSK9. Individuals suffering from FH typically experience elevated LDL-C levels, which contribute to a heightened risk of developing early-onset atherosclerotic cardiovascular diseases. "FDA approves YolTech's YOLT-101 for familial hypercholesterolemia" was originally created and published by Pharmaceutical Technology, a GlobalData owned brand. The information on this site has been included in good faith for general informational purposes only. It is not intended to amount to advice on which you should rely, and we give no representation, warranty or guarantee, whether express or implied as to its accuracy or completeness. You must obtain professional or specialist advice before taking, or refraining from, any action on the basis of the content on our site. Sign in to access your portfolio
Yahoo
7 days ago
- Business
- Yahoo
NewAmsterdam Pharma to Host R&D Day on June 11, 2025
NAARDEN, The Netherlands and MIAMI, June 05, 2025 (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 that it will host an R&D Day event for analysts and investors on June 11, 2025 beginning at 9:00 a.m. ET in New York City. Please join members of our management team, including: Michael Davidson, M.D., Chief Executive Officer, John Kastelein, M.D., Ph.D., FESC, Founder and Chief Scientific Officer, BJ Jones, Chief Commercial Officer, Ian Somaiya, Chief Financial Officer, and Matthew Philippe, Executive Vice President. A live webcast of the R&D event will be available and those who intend to join virtually can pre-register for the webcast through the link here. The live webcast and supporting presentation materials will be available on the Events section of the Investor Relations page of the NewAmsterdam website at at the time of the live event. An archived replay will be available on the NewAmsterdam website. Please note advanced registration is required for in-person attendance. About ObicetrapibObicetrapib is a novel, oral, low-dose CETP inhibitor that NewAmsterdam is developing to overcome the limitations of current LDL-lowering treatments. In each of the Company's Phase 2 trials, ROSE2, TULIP, ROSE, and OCEAN, as well as the Company's Phase 3 BROOKLYN, BROADWAY and TANDEM trials, evaluating obicetrapib as monotherapy or combination therapy, the Company observed statistically significant LDL-lowering combined with a side effect profile similar to that of placebo. The Company commenced the Phase 3 PREVAIL CVOT in March 2022, which is designed to assess the potential of obicetrapib to reduce occurrences of MACE. The Company completed enrollment of PREVAIL in April 2024 and randomized over 9,500 patients. Commercialization rights of obicetrapib in Europe, either as a monotherapy or as part of a fixed-dose combination with ezetimibe, have been exclusively granted to the Menarini Group, an Italy-based, leading international pharmaceutical and diagnostics company. About NewAmsterdamNewAmsterdam Pharma (Nasdaq: NAMS) is a late-stage, clinical biopharmaceutical company whose mission is to improve patient care in populations with metabolic diseases where currently approved therapies have not been adequate or well tolerated. We seek to fill a significant unmet need for a safe, well-tolerated and convenient LDL-lowering therapy. In multiple Phase 3 trials, NewAmsterdam is investigating obicetrapib, an oral, low-dose and once-daily CETP inhibitor, alone or as a fixed-dose combination with ezetimibe, as LDL-C lowering therapies to be used as an adjunct to statin therapy for patients at risk of CVD with elevated LDL-C, for whom existing therapies are not sufficiently effective or well tolerated. Company ContactMatthew PhilippeP: Media ContactSpectrum Science on behalf of NewAmsterdamJaryd LeadyP: 1-856-803-7855jleady@ Investor ContactPrecision AQ on behalf of NewAmsterdamAustin MurtaghP:
Yahoo
09-05-2025
- Health
- Yahoo
LEQVIO Strengthens Position as Leader in Cholesterol-Lowering Therapies Across Seven Major Markets
LEQVIO marks a significant advancement in hypercholesterolemia management, particularly for patients not achieving target LDL-C levels despite maximally tolerated statin therapy. As the first FDA-approved siRNA therapy for LDL-C reduction, it introduces a novel mechanism of action with sustained efficacy and biannual dosing, enhancing patient adherence. LAS VEGAS, May 8, 2025 /PRNewswire/ -- DelveInsight's "LEQVIO Market Size, Forecast, and Market Insight Report" highlights the details around LEQVIO, which is a PCSK9 Inhibitor. The report provides product descriptions, patent details, and competitor products (marketed and emerging therapies) of LEQVIO. The report also highlights the historical and forecasted sales from 2020 to 2034 segmented into 7MM [the United States, the EU4 (Germany, France, Italy, and Spain), the United Kingdom, and Japan]. Novartis/Alnylam Pharmaceuticals' LEQVIO (inclisiran) Overview LEQVIO (inclisiran) is a first-of-its-kind therapy developed by Novartis based on small interfering RNA (siRNA) technology. It targets the mRNA of PCSK9 (proprotein convertase subtilisin/kexin type 9), reducing the production of this protein in the liver. Unlike traditional treatments, LEQVIO lowers LDL-C levels by enhancing the liver's ability to absorb and eliminate it from the bloodstream. In the US, LEQVIO is approved for use alongside diet and statin therapy in adults with primary hyperlipidemia, including those with heterozygous familial hypercholesterolemia (HeFH), to help reduce low-density lipoprotein cholesterol (LDL-C). The recommended LEQVIO dosage is 284 mg, given via subcutaneous injection initially, followed by a dose at 3 months, and then every 6 months thereafter, in combination with statins. In 2034, the market size of inclisiran is expected to be USD 2.2 billion in the US. Drug Name LEQVIO (inclisiran) Molecule type Small interfering Ribonucleic Acid (siRNA) Developer Novartis/Alnylam Pharmaceuticals Primary Indication Primary hyperlipidemia, clinical ASCVD or HeFH, hypercholesterolemia, and others Mechanism of action Proprotein Convertase Subtilisin Kexin 9 (PCSK9) Inhibitor Route of administration Subcutaneous Learn more about LEQVIO projected market size for PCSK9 inhibitors @ LEQVIO Market Potential Proprotein convertase subtilisin/kexin type 9 (PCSK9) is a serine protease essential to cholesterol metabolism, primarily by controlling the breakdown of low-density lipoprotein (LDL) receptors. This mechanism decreases the removal of LDL particles from the blood, thereby influencing LDL cholesterol (LDL-C) levels. There is an inverse relationship between PCSK9 activity and LDL-C levels: gain-of-function mutations in the PCSK9 gene lead to increased LDL-C and a higher cardiovascular risk, as seen in familial hypercholesterolemia, while loss-of-function mutations are associated with lower LDL-C levels and reduced atherosclerotic cardiovascular disease (ASCVD) risk. In 2023, approximately 640,000 cases of familial hypercholesterolemia were diagnosed across the 7MM, with homozygous forms being extremely rare. PCSK9 inhibitors have emerged as a vital therapeutic option for hypercholesterolemia management, especially for patients at elevated cardiovascular risk or those unresponsive to standard treatments like statins. These drugs inhibit PCSK9, a protein that promotes LDL receptor degradation. By blocking PCSK9, these therapies enhance the presence of LDL receptors on liver cells, improving LDL-C clearance from the bloodstream. According to DelveInsight, the PCSK9 inhibitor market in the 7MM was valued at USD 2 billion in 2023. In summary, the PCSK9 inhibitor market is projected to witness substantial growth, driven by their use in statin-intolerant patients, expanding applications of PCSK9 inhibition, potential in broader therapeutic areas, and their role in preventive strategies to help reduce the overall cardiovascular disease burden. Discover more about the PCSK9 inhibitors market in detail @ PCSK9 Inhibitors Market Report Emerging Competitors of LEQVIO Several drugs are currently under development in the PCSK9 inhibitor pipeline, including Lerodalcibep (LIB Therapeutics), MK-0616 (Merck), VERVE-101 and VERVE-102 (Verve Therapeutics), and CiVi 008 (CiVi Biopharma), among others. Lerodalcibep is a third-generation PCSK9 inhibitor designed to overcome the limitations of traditional LDL-C-lowering therapies like statins and ezetimibe. It aims to help patients reach the more aggressive LDL-C targets recommended by recent cardiovascular guidelines. The drug is being developed as a once-monthly, low-volume injection with long shelf stability at room temperature. Currently in Phase III clinical trials, LIB Therapeutics intends to file a Biologics License Application (BLA) by year-end, with a potential PDUFA decision expected in the latter half of 2025. MK-0616, an oral PCSK9 inhibitor from Merck, is a novel macrocyclic peptide that blocks the interaction between PCSK9 and LDL receptors, thus reducing LDL cholesterol. It is currently in Phase III trials and could become the first oral treatment in its class. Verve Therapeutics is advancing two gene-editing candidates—VERVE-101 and VERVE-102—targeting PCSK9. These one-time treatments are designed to permanently switch off the PCSK9 gene in the liver, thereby lowering LDL-C levels. VERVE-102 is being evaluated in the Heart-2 Phase Ib trial for patients with heterozygous familial hypercholesterolemia (HeFH) or early-onset coronary artery disease. Meanwhile, enrollment in the Heart-1 trial for VERVE-101 has been paused due to lab abnormalities, and an investigation is ongoing. Based on the results, Verve will work with regulators to decide the next steps for VERVE-101. The company plans to initiate a randomized, placebo-controlled Phase II trial based on data from both the Heart-1 and Heart-2 studies. As these next-generation therapies move closer to approval, they have the potential to significantly disrupt and redefine the PCSK9 inhibitor market, ultimately giving fierce competition to LEQVIO. To know more about the number of competing drugs in development, visit @ LEQVIO Market Positioning Compared to Other Drugs Key Milestones of LEQVIO In July 2023, the US FDA approved an expanded indication for Novartis LEQVIO to include treatment of adults with high LDL-C and who are at increased risk of heart disease. The updated indication for primary hyperlipidemia allows for the expanded use of LEQVIO as an adjunct to diet and statin therapy beyond the previously approved ASCVD and HeFH patient populations. In 2023, LEQVIO (inclisiran) was approved by MHLW for familial and non-familial hypercholesterolemia and for patients who are at a high risk of developing cardiovascular events. In August 2022, Novartis selected Soleo Health as a Limited Drug Distribution Partner for the Administration of LEQVIO. Under this partnership, Soleo Health will administer the drug to patients in their homes or at one of the Company's Ambulatory Infusion Centers (AICs) in the US. In 2021, Novartis reached an agreement with the National Health Service (NHS) in England to implement a first-of-its-kind population health management approach designed to provide faster and broader access to LEQVIO for certain high-risk patients with ASCVD. In December 2020, Novartis announced that the European Commission (EC) approved LEQVIO to treat adults with hypercholesterolemia or mixed dyslipidemia, two common forms of elevated cholesterol. Discover how LEQVIO is shaping the PCSK9 inhibitors treatment landscape @ LEQVIO Injection LEQVIO Market Dynamics LEQVIO, as a first-in-class small interfering RNA (siRNA) that helps maintain bad cholesterol levels and improve mortality rates, strengthens its market position with a value-based price per dose and promising market access and reimbursement. Ongoing trials for CVRR and Pediatric Hyperlipidemia expansion pave the way for label expansion, addressing diverse medical needs. The escalating patient population presents a favorable trend for expanding LEQVIO's market reach and driving growth. However, its market is currently constrained by its specific indication for HeFH in adults, potentially limiting its reach compared to competitors like REPATHA and PRALUENT, which are approved for both HoHF and HeHF in adults and pediatrics. The market is expected to face heightened competition post-2027, which may impact LEQVIO's market share, and similar annual treatment costs across PCSK9 inhibitors may pose a challenge, affecting LEQVIO's competitive edge despite better reimbursement policies. Dive deeper to get more insight into LEQVIO's strengths & weaknesses relative to competitors @ LEQVIO Market Drug Report Table of Contents 1 Report Introduction 2 LEQVIO: Novartis/Alnylam Pharmaceuticals 2.1 Product Overview 2.2 Other Development Activities 2.3 Clinical Development 2.4 Clinical Trials Information 2.5 Safety and Efficacy 2.6 Product Profile 2.7 Market Assessment 2.7.1 The 7MM Analysis 2.7.1.1 Cost Assumptions and Rebate 2.7.1.2 Pricing Trends 2.7.1.3 Analogue Assessment 2.7.1.4 Launch Year and Therapy Uptake 2.7.2 The United States Market Analysis 2.7.3 EU4 and the United Kingdom Market Analysis 2.7.3.1 Germany 2.7.3.2 France 2.7.3.3 Italy 2.7.3.4 Spain 2.7.3.5 UK 2.7.4 Japan Market Analysis 2.8 Market Drivers 2.9 Market Barriers 2.10 SWOT Analysis 3 Key Cross of Marketed Competitors of LEQVIO 4 Key Cross of Emerging Competitors of LEQVIO Related Reports PCSK9 Inhibitor Market PCSK9 Inhibitor Market Size, Target Population, Competitive Landscape & Market Forecast – 2034 report deliver an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key PCSK9 inhibitor companies, including LIB Therapeutics, Merck, Verve Therapeutics, CiVi Biopharma, among others. PCSK9 Inhibitors Pipeline PCSK9 Inhibitors Pipeline Insight – 2025 report provides comprehensive insights about the pipeline landscape, pipeline drug profiles, including clinical and non-clinical stage products, and the key PCSK9 inhibitors companies, including Ionis Pharmaceuticals, Akeso Biopharma, Amgen, Novo Nordisk, Civi BioPharma, among others. Hypercholesterolemia Market Hypercholesterolemia Market Insights, Epidemiology, and Market Forecast – 2034 report delivers an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key hypercholesterolemia companies, including Novartis Pharmaceuticals, Merck Sharp & Dohme LLC, Esperion Therapeutics, Inc., Arrowhead Pharmaceuticals, LIB Therapeutics LLC, Medpace, Inc., AstraZeneca, NewAmsterdam Pharma, among others. Homozygous Familial Hypercholesterolemia Market Homozygous Familial Hypercholesterolemia Market Insights, Epidemiology, and Market Forecast – 2034 report delivers an in-depth understanding of the disease, historical and forecasted epidemiology, as well as the market trends, market drivers, market barriers, and key homozygous familial hypercholesterolemia companies, including Arrowhead Pharmaceuticals, Novartis, Alnylam Pharmaceuticals, LIB Therapeutics, among others. About DelveInsight DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. It supports pharma companies by providing comprehensive end-to-end solutions to improve their performance. Get hassle-free access to all the healthcare and pharma market research reports through our subscription-based platform PharmDelve. Contact UsShruti Thakur info@ +14699457679 Logo: View original content: SOURCE DelveInsight Business Research, LLP Error in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
The Guardian
01-04-2025
- Health
- The Guardian
Lowering bad cholesterol may cut risk of dementia by 26%, study suggests
Lowering your levels of bad cholesterol could reduce the risk of dementia by 26%, a study suggests. People with low levels of low-density lipoprotein cholesterol (LDL-C) in their blood have a lower overall risk of dementia, and a reduced risk of Alzheimer's disease specifically, according to research published in the Journal of Neurology Neurosurgery & Psychiatry. Taking statins also provided an 'additional protective effect' against the condition for those people with low levels of bad cholesterol, researchers found. The number of people living with dementia worldwide is forecast to nearly triple to 153 million by 2050, but evidence suggests almost half of cases could be prevented or delayed. LDL-C is often referred to as bad cholesterol and can cause plaque to build in arteries, leading to cardiovascular disease, which can increase the risk of strokes, heart attacks and death. However, until recently, the relationship between LDL-C levels and dementia has been less clear. Last year, a Lancet report found 7% of cases of dementia were linked to high levels of bad cholesterol in midlife. Now a new study suggests having low levels of LDL-C could reduce the risk of dementia by a quarter. Researchers collected data on 571,000 people in South Korea who had not been diagnosed with dementia – 192,213 people with LDL-C levels less than 1.8 mmol/L and 379,006 patients with LDL-C levels higher than 3.4 mmol/L (>130mg/dL). Analysis of subsequent diagnoses of dementia showed that LDL-C levels below 1.8 mmol/L were associated with a 26% reduction in the risk of dementia and a 28% cut in the risk of Alzheimer's disease specifically, compared with LDL-C levels above 3.4 mmol/L. Statins appeared to offer additional protection against dementia in the presence of low LDL-C levels. Among people with LDL-C levels below 1.8 mmol/L, statin use was linked with a 13% reduction in dementia risk and a 12% cut in risk of Alzheimer's disease, compared with non-users. This was an observational study, and no firm conclusions can be drawn about cause and effect. The authors also acknowledged several limitations, including the focus on baseline LDL-C levels when lipid profiles could change over time. Sign up to First Edition Our morning email breaks down the key stories of the day, telling you what's happening and why it matters after newsletter promotion Nevertheless, they concluded: 'Low LDL-C levels … are significantly associated with a reduced risk of dementia, including Alzheimer's disease-related dementia, with statin therapy providing additional protective effects.' Dr Francesco Tamagnini, a neurophysiologist at the University of Reading, who was not involved with the study, said: 'There is clearly more to the story of Alzheimer's than we first thought. 'This paper looks at the correlation and potential causal relationship between high levels of bad cholesterol and dementia risk. The results give a convincing argument for researchers to consider LDL cholesterol in addition to the classic approaches.' Dr Julia Dudley, head of research at Alzheimer's Research UK, said: 'The use of statins seemed to offer a protective effect – even in those who already had cholesterol levels within a lower range. 'However, dementia risk is complex and influenced by many factors. Without a detailed picture of what's going on in the brain, we do not know if there is a direct link between lower cholesterol and reduced dementia risk. 'Clinical trials will be key to understand what effects statins might be having on disease processes in the brain.'
