Latest news with #PCSK9
The Print
26-05-2025
- Health
- The Print
Experimental drug promising to bring down bad cholesterol by up to 69% completes early stage trial
The potential breakthrough medicine developed by US-based biotechnology firm Verve Therapeutics is a novel, in vivo (delivered directly into the body), base editing medicine that permanently turns off the PCSK9 gene (a protein that plays a role in regulating cholesterol levels in the blood) in the liver, thereby reducing disease-causing bad cholesterol. High levels of low-density lipoprotein cholesterol (LDL-C), commonly referred to as bad cholesterol, are among the leading causes of heart attacks and strokes, the two top reasons as per the World Health Organisation (WHO) for mortality worldwide as well as India. New Delhi: Would you believe it if someone tells you that the risk of you suffering a heart attack or a stroke could be dramatically reduced by a single injection once in your lifetime? That is exactly the promise that results from an early stage trial of an experimental drug in the US has shown, bringing down bad cholesterol by up to 69 percent after just one shot. The results from its stage 1b clinical trial show that intravenous infusion of the drug, named VERVE 102 currently, led to a mean LDL reduction of 53 percent among patients receiving the highest dose, with the maximum individual reduction reaching 69 percent, the company said in a statement. The drug suppressed PCSK9 levels by an average of 60 percent at the highest dose. Among the 14 participants across three dose levels, the drug was well-tolerated, with no treatment-related serious adverse events and no clinically significant laboratory abnormalities observed, the company said. Specialists, however, insist results from a large group of patients would be crucial. The drug has triggered a buzz in the global pharmaceutical industry and is now set to enter a larger trial across five countries to test the efficacy and safety. 'These initial results from the early trial of the drug are very interesting and promise, for the first time, a lifetime treatment of a condition that is main risk factor for several serious cardiovascular events,' Dr Ambuj Roy, a senior cardiologist at the All India Institute of Medical Sciences (AIIMS), New Delhi, told ThePrint. The clinician-researcher also underlined that data from the drug's trial on a larger group of people would be pivotal before hopes are raised. The company has maintained that the initial data are promising with respect to both safety and efficacy, and suggest the potential for a new era of cardiovascular disease treatment where a single dose 'might lead to lifelong control of LDL-C'. Verve has tied up with pharma giant Eli Lilly and Company to co-commercialise the drug following successful trials and regulatory approvals. WHO defines raised total cholesterol or dyslipidemia—which includes LDL-C—over 5 millimole or 190 mg/dL and it is estimated that nearly 39 percent of adults worldwide suffer from dyslipidemia. A large survey carried out by the Indian Council of Medical Research (ICMR) and the Madras Diabetes Research Foundation, published in The Lancet in 2023, had shown 81.2 percent Indians have dyslipidemia and 20.9 percent—or 18.5 crore—have high levels of bad cholesterol. Also Read: India gets 1st guidelines for managing lipid levels, the biggest risk factor for heart attack, stroke What sets the drug apart As of now, statins are the most commonly used pharmacotherapies to lower cholesterol levels and work by inhibiting an enzyme called HMG-CoA reductase that is involved in the synthesis of cholesterol in the liver. But experts say that while statins and various lipid-lowering medications have greatly diminished cardiovascular risks in many patients, there is an urgent requirement for improved, more specific drugs to manage cholesterol. Such need, explained Dr Amit Bhushan Sharma, the director and unit head of cardiology at Paras Health in Gurugram, is particularly evident in patients with statin intolerance, genetic dyslipidemias such as familial hypercholesterolemia (or high cholesterol), and in those who fail to reach target lipid levels despite receiving optimal treatment. New therapies like PCSK9 inhibitors, inclisiran—a small interfering RNA (siRNA) therapy that offers sustained LDL-C reduction with just two doses a year launched in India last year—and bempedoic acid have created new possibilities, but they remain underused because of cost and access challenges, experts say. Amid this, there is an increasing interest in RNA-based and gene-editing treatments that may offer lasting cholesterol management with reduced side-effects. 'Improved cholesterol-lowering medications could help reduce the residual cardiovascular risk in high-risk populations, prevent recurrent cardiac events, and potentially offer better safety profiles for patients with comorbidities,' Sharma added. The cardiologist also said that a more personalised approach to cholesterol management—driven by advanced pharmacological options—could be the next big step in preventive cardiology. Verve has said the drug trial has been aimed at evaluating its result in patients with heterozygous familial hypercholesterolemia (HeFH) and/or premature coronary artery disease (CAD), two populations that require deep and durable reductions of bad cholesterol levels in the blood. (Edited by Ajeet Tiwari) Also Read: A new AI tool can predict if you'll have a heart attack 10 years later, shows 1st global trial
Mint
23-05-2025
- Health
- Mint
Strokes and heart attacks kill. New drugs are coming to prevent them.
Forty years after the first statin drug started lowering levels of artery-choking cholesterol, heart attacks and strokes remain the world's biggest killers. This year, we'll see test results on some drugs that cut cholesterol even more. Statins are cheap and effective drugs for reducing blood levels of the 'bad" cholesterol known as LDL, which gunks up arteries over time. A quarter of Americans have high LDL. But only about half of patients stay on their prescribed statins, and the drugs don't get LDL low enough in some 10% of patients. A pack of new cardiovascular drugs are being tried as pills, biweekly or semiannual shots, and even as once-for-a-lifetime infusions. And their benefits all seem additive to those of statins and each other. Successful trial results in the next few years could save thousands of lives and generate billions in new annual revenue for drugmakers. The companies working on them range from pharmaceutical companies—like Eli Lilly, Merck, and AstraZeneca—to biotechs—like Amgen and Regeneron Pharmaceuticals—to gene-editing specialists—like Crispr Therapeutics and Verve Therapeutics. Regeneron and Sanofi took the first big step beyond statins in 2015 with Praluent, a biweekly injection of antibodies that sop up the enzyme called PCSK9, which strongly effects blood levels of LDL. Amgen followed just weeks later, with the PCSK9 antibody Repatha. In 2021, Novartis launched Leqvio, which blocks the genetic instructions for making PCSK9 and can be injected just twice a year. The PCSK9 drugs are lifesavers for families with genetically-high LDL levels. But they require regular injections and can cost $30,000 a year for the rest of a patient's life. The cost and hassle has led to sales that are good, but not as great as expected. Regeneron and Sanofi's Praluent sales were $585 million in 2024. For the same year, Amgen's Repatha pulled in $2.2 billion, up 15% year over year. Last year's sales of Leqvio for Novartis were $260 million, up 80%. To address these drugs' cost and convenience issues, Merck is testing a pill treatment that it code-named MK-0616. The company is betting the drug will help replace some of the revenue it will lose after patents expire on its huge-selling cancer drug, Keytruda. On April 7, Merck finished the once-daily pill's Phase 3 trial in about 300 people with inherited high-LDL cholesterol. In August, it expects to complete the broader study among some 2,800 patients with serious cardiovascular problems. Both studies measure reductions in LDL—which regulators accept as good-enough proof that cardiovascular problems will also be prevented. Analysts hope the company will report results at the November 2025 meeting of the American Heart Association. Merck aims for a bigger market than just high-LDL families, so a third Phase 3 for MK-0616 will use the gold-standard test of whether those on the pill actually have fewer heart attacks, strokes, or other cardiovascular calamities. That study, which is enrolling over 14,000 people, won't finish until 2029. While Merck's pill could find its way to a larger population than today's PCSK9 injections, it still has some convenience issues. Patients have to take MK-0616 on an empty stomach and shouldn't be on certain other drugs. That's why AstraZeneca is pushing ahead with its own PCSK9 pill, dubbed AZD0780, which requires no fasting. In March, the company said the pill achieved deep reductions in LDL in a Phase 2 study on patients whose statin treatment wasn't doing the job. The size of the cardio market and the current PCSK9 drugs' issues have also caught the eye of the gene-editing industry. These companies' Crispr technology can zero in on a particular, troublesome stretch in the 3 billion links of our DNA, and then permanently inactivate it, or make a repair. So far, genetic-medicine firms have struggled to convince investors that there's real money to be made from the Nobel Prize-winning technology. Companies like Crispr Therapeutics initially focused on dire, but relatively uncommon diseases like sickle cell disorder. Revenue has been slow to come. Verve Therapeutics, however, focused on cardiovascular disease from the start. It licensed a second-generation Crispr technology called base-editing, from the company Beam Therapeutics. Base-editing gently changes a single letter at a time in DNA genetic code. By permanently disrupting the code for PCSK9, Verve's one-time treatment could lower LDL for life. The stock perked up in April when the company reported initial Phase 1 data that showed no serious side effects among 14 patients—with LDL reductions of around 60% even two years after the one-time treatment. That reduction is comparable to today's PCSK9 injectables. Eli Lilly has an option to partner on Verve's cardio programs, and could opt in this year. Other genetic-medicine approaches to PCSK9 are exploring nonpermanent, but long-lasting fixes. The privately held Scribe Therapeutics has reported intriguing results in monkeys from 'epigenetic" edits of the molecules that turn genes on and off. And PCSK9 isn't the only strong lever on cholesterol levels. Another that our body uses to control blood levels of cholesterol is a protein called CETP. Early in May, NewAmsterdam Pharma reported on a couple of Phase 3 trials of obicetrapib, its antibody that blocks CETP. By itself, the antibody cut LDL levels by a third. Combined with a statin, LDL levels fell by half. Another lever on LDL is ANGPTL3, a protein controlling blood levels of LDL and harmful triglycerides. Since 2021, Regeneron has marketed Evkeeza, an antibody that inhibits ANGPTL3. Sales of the drug to patients with inherited high cholesterol were $126 million in 2024. Lilly, Arrowhead Pharmaceuticals, and Regeneron are all testing so-called siRNA drugs that interfere with cellular production of ANGPTL3. They are injections that work for as long as a year at a time. Verve began Phase 1 trials in November 2024 on a base-editing treatment that permanently disrupts the genetic instructions for ANGPTL3. It hopes to report some data later this year. Crispr Therapeutics, meanwhile, is further along in trials of a one-and-done edit that blocks ANGPTL3. In April, it reported that LDL dropped by two-thirds, among the first 10 patients treated. No serious side effects surfaced in the first months after infusion. For rare but deadly disorders, gene-editing fixes are priced above $1 million. The Crispr crowd thinks it can find a price that is attractive to the healthcare payers that cover the expensive siRNA injections now given to patients with inherited high levels of cholesterol. 'If you're talking about siRNA, that is $30,000 a year for 50 years," said Crispr Therapeutics Chief Executive Samarth Kulkarni at an April conference. 'We could spend one-tenth of that and charge $150,000 for a single-shot therapy. For the payer, the economic argument is very clear." Write to Bill Alpert at
Yahoo
15-05-2025
- Business
- Yahoo
Regeneron Prevails over Amgen in Antitrust PCSK9 Lawsuit Protecting Biotech Innovation and Patient Access to Life-Saving Treatments
Federal court jury found Amgen liable for violating antitrust and tort laws by using cross-therapeutic bundled rebates to prevent Praluent® (alirocumab) from competing in the market Jury awarded Regeneron $135.6 million dollars of compensatory damages and $271.2 million dollars in punitive damages TARRYTOWN, N.Y., May 15, 2025 (GLOBE NEWSWIRE) -- Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) applauds the jury verdict in the U.S. District Court for the District of Delaware that found that Amgen Inc. violated antitrust and tort laws by creating a bundling scheme that illegally leveraged its blockbuster anti-inflammatory drugs Enbrel® (etanercept) and Otezla® (apremilast) to convince pharmacy benefit managers (PBMs) to select Repatha® (evolocumab) as the exclusive PCSK9 category product over Praluent® (alirocumab). The jury found that Amgen violated the Clayton Act, the Sherman Act, the New York State Donnelly Act, the California Cartwright Act and Delaware tort law. Enbrel and Otezla are therapeutically different medicines that do not treat the heart conditions that Praluent or Repatha address. Amgen threatened to withhold rebates unless PBMs preferred Repatha and excluded Praluent. Amgen prevented Regeneron from competing on a level playing field and unfairly denied patients access to Praluent. This anticompetitive practice shut out an innovative therapy from the PCSK9 marketplace, not based on clinical merit or price. Because of Amgen's size and unrelated product portfolio, their anticompetitive actions ultimately hurt competition and patients. The jury awarded Regeneron $135.6 million dollars of compensatory damages. The jury also awarded Regeneron $271.2 million dollars in punitive damages. Punitive damages may be awarded to punish a party for outrageous conduct and deter a party, and others like it, from engaging in similar conduct in the future. "Patients rely on the biotech industry to find solutions for their most urgent medical conditions. At Regeneron, we take this responsibility very seriously and are committed to delivering breakthrough therapies that improve patient lives. To achieve this, companies must be able to compete fairly based on the clinical and economic value of their products. Larger companies should not be allowed to use anticompetitive tactics to push competitors out of the market," said Leonard S. Schleifer, M.D., Ph.D., Board co-Chair, President and Chief Executive Officer of Regeneron. 'Fair competition is critical as it expands patient access to life-changing therapies and drives innovation forward so we can continue to address the medical challenges of tomorrow.' 'Since the FDA approved Praluent in 2015, Amgen has tried to remove and exclude Praluent from the market. After a failed patent litigation campaign, they pivoted toward an anticompetitive bundling scheme that created a dangerous precedent that virtually eliminated all competition,' said Joseph J. LaRosa, Executive Vice President, General Counsel and Secretary of Regeneron. 'Today's verdict validates Regeneron's efforts to help protect patient access to our innovations and provides a clear sign that anticompetitive efforts will not remain unchecked.' Regeneron thanks the jury for their time and thoughtful consideration of the facts of this case. Regeneron's lead counsel was Jonathan D. Polkes of White & Case LLP. About PraluentPraluent inhibits the binding of PCSK9 to the LDL receptor and thereby increases the number of available LDL receptors on the surface of liver cells to clear LDL, which lowers LDL-C levels in the blood. Praluent was developed by Regeneron and Sanofi under a global collaboration agreement and invented by Regeneron using the company's proprietary VelocImmune® technology that yields optimized fully-human monoclonal antibodies. In addition to the U.S., Praluent is approved in 60 countries, including the European Union, Japan, Canada, Switzerland and Brazil. About Regeneron's TechnologyRegeneron's VelocImmune technology utilizes a proprietary genetically engineered mouse platform endowed with a genetically humanized immune system to produce optimized fully human antibodies. When Regeneron's co-Founder, President and Chief Scientific Officer George D. Yancopouloswas a graduate student with his mentor Frederick W. Alt in 1985, they were the first to envision making such a genetically humanized mouse, and Regeneron has spent decades inventing and developing VelocImmune and related VelociSuite® technologies. Dr. Yancopoulos and his team have used VelocImmune technology to create a substantial proportion of all original, FDA-approved fully human monoclonal antibodies. This includes Dupixent® (dupilumab), Libtayo® (cemiplimab-rwlc), Praluent® (alirocumab), Kevzara® (sarilumab), Evkeeza® (evinacumab-dgnb), Inmazeb® (atoltivimab, maftivimab and odesivimab-ebgn) and Veopoz® (pozelimab-bbfg). In addition, REGEN-COV®(casirivimab and imdevimab) had been authorized by the FDA during the COVID-19 pandemic until 2024. Indications and Important Safety Information PRALUENT is an injectable prescription medicine used: in adults with cardiovascular disease to reduce the risk of heart attack, stroke, and certain types of chest pain conditions (unstable angina) requiring hospitalization. along with diet, alone or together with other cholesterol-lowering medicines in adults with high blood cholesterol levels called primary hyperlipidemia (including a type of high cholesterol called heterozygous familial hypercholesterolemia [HeFH]), to reduce low-density lipoprotein cholesterol (LDL-C) or bad cholesterol. along with other LDL-lowering treatments in adults with a type of high cholesterol called homozygous familial hypercholesterolemia, who need additional lowering of LDL-C. along with diet and other LDL-C lowering treatments in children aged 8 years and older with HeFH to reduce LDL-C. It is not known if PRALUENT is safe and effective in children who are younger than 8 years of age or in children with other types of high cholesterol (hyperlipidemias). Important Safety InformationDo not use PRALUENT if you are allergic to alirocumab or to any of the ingredients in PRALUENT. Before you start using PRALUENT, tell your healthcare provider about all of your medical conditions, including allergies, and if you are pregnant or plan to become pregnant or if you are breastfeeding or plan to breastfeed. Tell your healthcare provider or pharmacist about any medicines you take, including prescription and over-the-counter medicines, vitamins, or herbal supplements. PRALUENT can cause serious side effects, including allergic reactions that can be severe and require treatment in a hospital. Stop using PRALUENT and call your healthcare provider or go to the nearest hospital emergency room right away if you have any symptoms of an allergic reaction including a severe rash, redness, hives, severe itching, trouble breathing, or swelling of the face, lips, throat, or tongue. The common side effects of PRALUENT include: redness, itching, swelling, or pain/tenderness at the injection site; flu or flu-like symptoms; diarrhea; muscle pain; muscle spasms; and bruising. Tell your healthcare provider if you have any side effect that bothers you or that does not go away. Talk to your doctor about the right way to prepare and give yourself a PRALUENT injection and follow the 'Instructions For Use' that comes with PRALUENT. In children aged 12 to 17 years, it is recommended that PRALUENT be given by or under the supervision of an adult. In children aged 8 to 11 years, PRALUENT should be given by a caregiver. You are encouraged to report negative side effects of prescription drugs to the FDA. Visit or call 1-800-FDA-1088. Please click here for full Prescribing Information. About RegeneronRegeneron (NASDAQ: REGN) is a leading biotechnology company that invents, develops and commercializes life-transforming medicines for people with serious diseases. Founded and led by physician-scientists, our unique ability to repeatedly and consistently translate science into medicine has led to numerous approved treatments and product candidates in development, most of which were homegrown in our laboratories. Our medicines and pipeline are designed to help patients with eye diseases, allergic and inflammatory diseases, cancer, cardiovascular and metabolic diseases, neurological diseases, hematologic conditions, infectious diseases, and rare diseases. Regeneron pushes the boundaries of scientific discovery and accelerates drug development using our proprietary technologies, such as VelociSuite, which produces optimized fully human antibodies and new classes of bispecific antibodies. We are shaping the next frontier of medicine with data-powered insights from the Regeneron Genetics Center® and pioneering genetic medicine platforms, enabling us to identify innovative targets and complementary approaches to potentially treat or cure diseases. For more information, please visit or follow Regeneron on LinkedIn, Instagram, Facebook or X. Forward-Looking Statements and Use of Digital MediaThis press release includes forward-looking statements that involve risks and uncertainties relating to future events and the future performance of Regeneron Pharmaceuticals, Inc. ('Regeneron' or the 'Company'), and actual events or results may differ materially from these forward-looking statements. Words such as 'anticipate,' 'expect,' 'intend,' 'plan,' 'believe,' 'seek,' 'estimate,' variations of such words, and similar expressions are intended to identify such forward-looking statements, although not all forward-looking statements contain these identifying words. These statements concern, and these risks and uncertainties include, among others, risks associated with litigation and other proceedings and government investigations relating to the Company and/or its operations (including without limitation the antitrust litigation discussed in this press release), risks associated with intellectual property of other parties and pending or future litigation relating thereto, the ultimate outcome of any such proceedings and investigations, and the impact any of the foregoing may have on Regeneron's business, prospects, operating results, and financial condition; the outcome of any post-trial motions or other appeals relating to the jury verdict discussed in this press release; the nature, timing, and possible success and therapeutic applications of products marketed or otherwise commercialized by Regeneron and/or its collaborators or licensees (collectively, 'Regeneron's Products') and product candidates being developed by Regeneron and/or its collaborators or licensees (collectively, 'Regeneron's Product Candidates') and research and clinical programs now underway or planned, including without limitation Praluent® (alirocumab); the likelihood, timing, and scope of possible regulatory approval and commercial launch of Regeneron's Product Candidates and new indications for Regeneron's Products; uncertainty of the utilization, market acceptance, and commercial success of Regeneron's Products and Regeneron's Product Candidates and the impact of studies (whether conducted by Regeneron or others and whether mandated or voluntary) on any of the foregoing; the ability of Regeneron's collaborators, licensees, suppliers, or other third parties (as applicable) to perform manufacturing, filling, finishing, packaging, labeling, distribution, and other steps related to Regeneron's Products and Regeneron's Product Candidates; the ability of Regeneron to manage supply chains for multiple products and product candidates and risks associated with tariffs and other trade restrictions; safety issues resulting from the administration of Regeneron's Products (such as Praluent) and Regeneron's Product Candidates in patients, including serious complications or side effects in connection with the use of Regeneron's Products and Regeneron's Product Candidates in clinical trials; determinations by regulatory and administrative governmental authorities which may delay or restrict Regeneron's ability to continue to develop or commercialize Regeneron's Products and Regeneron's Product Candidates; ongoing regulatory obligations and oversight impacting Regeneron's Products, research and clinical programs, and business, including those relating to patient privacy; the availability and extent of reimbursement or copay assistance for Regeneron's Products from third-party payors and other third parties, including private payor healthcare and insurance programs, health maintenance organizations, pharmacy benefit management companies, and government programs such as Medicare and Medicaid; coverage and reimbursement determinations by such payors and other third parties and new policies and procedures adopted by such payors and other third parties; changes in laws, regulations, and policies affecting the healthcare industry; competing drugs and product candidates that may be superior to, or more cost effective than, Regeneron's Products and Regeneron's Product Candidates (including biosimilar versions of Regeneron's Products); the extent to which the results from the research and development programs conducted by Regeneron and/or its collaborators or licensees may be replicated in other studies and/or lead to advancement of product candidates to clinical trials, therapeutic applications, or regulatory approval; unanticipated expenses; the costs of developing, producing, and selling products; the ability of Regeneron to meet any of its financial projections or guidance and changes to the assumptions underlying those projections or guidance; the potential for any license, collaboration, or supply agreement, including Regeneron's agreements with Sanofi and Bayer (or their respective affiliated companies, as applicable), to be cancelled or terminated; and the impact of public health outbreaks, epidemics, or pandemics on Regeneron's business. A more complete description of these and other material risks can be found in Regeneron's filings with the U.S. Securities and Exchange Commission, including its Form 10-K for the year ended December 31, 2024 and its Form 10-Q for the quarterly period ended March 31, 2025. Any forward-looking statements are made based on management's current beliefs and judgment, and the reader is cautioned not to rely on any forward-looking statements made by Regeneron. Regeneron does not undertake any obligation to update (publicly or otherwise) any forward-looking statement, including without limitation any financial projection or guidance, whether as a result of new information, future events, or otherwise. Regeneron uses its media and investor relations website and social media outlets to publish important information about the Company, including information that may be deemed material to investors. Financial and other information about Regeneron is routinely posted and is accessible on Regeneron's media and investor relations website ( and its LinkedIn page ( Contacts: Media RelationsMary HeatherTel: +1 Investor RelationsMark HudsonTel: +1
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. 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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
Miami Herald
02-05-2025
- Health
- Miami Herald
New Heart Scan Review Finds Hidden Disease in 'Low-Risk' Patients - 83% Show Signs of Treatable Heart Problems
BELLEVUE, WA / ACCESS Newswire / May 2, 2025 / A new study by Cardia Vision, LLC has revealed that a commonly used heart scan may be missing signs of early heart disease in many patients who appear "low risk." Using a more advanced imaging method, the research team discovered that 83% of patients had signs of treatable heart disease-even when traditional tests showed no cause for concern. The study focused on 100 patients who underwent both a calcium score test, which looks for hardened plaque in the arteries, and a more detailed scan called a coronary CT angiography (CCTA), which can detect softer, more dangerous plaque that doesn't show up on the more standard test. Key findings include: 69.8% of patients with a "zero" calcium score actually had soft plaque, which can lead to heart findings challenge the idea that a calcium score of zero means a person is heart-disease soft plaque found is highly treatable with medications like statins and PCSK9 inhibitors-if it's caught in time. "This is potentially life-saving information," said Dr. Kimberly Brockenbrough, Radiologist and CEO of Cardia Vision. "Many people assume they're in the clear based on a single test, but we now know that dangerous plaque can be hiding undetected. CCTA gives us a clearer, more complete picture." Heart disease remains the leading cause of death in the U.S., and early detection is key. This research adds to a growing number of studies suggesting that advanced heart scans could play a crucial role in catching disease early and preventing serious events like heart attacks. "It's time to rethink how we screen for heart disease," added Dr. Brockenbrough. "We have tools that can see the full picture-we just need to use them." About Cardia Vision Cardia Vision is a preventative cardiovascular imaging center based in Bellevue, Washington, committed to diagnosing heart disease before a heart attack or sudden death. Media Contact: Dr. Kimberly Brockenbrough425-998-8627info@ Vision, LLCBellevue, WA Contact Information: Kimberly Brockenbrough CEO, Radiologist, and Founderkim@ SOURCE: Cardia Vision 83% Treatable Heart press release
