This is an open-label study designed to evaluate the long-term safety and efficacy of evinacumab, a fully human ANGPTL3 antibody, in patients with homozygous familial hypercholesterolemia (HoFH), in a real-life setting in Canada.
Eligible patients for this study are male and female adult patients with HoFH. Evinacumab will be added on top of the patient's background lipid-modifying therapy (LMT), including statins, ezetimibe, PCSK9 inhibitors, lomitapide or other lipid lowering therapies. This study will be conducted using an hybrid (on-site, foldable sites) approach. Patients will enter the current study, in an open-label treatment period, following their screening. This study will continue until reimbursement of evinacumab in Canada or for a maximum of 24 months. The end of study (EoS) visit will be scheduled 4 weeks after the last dose has been injected and will be followed by a 52-week follow-up.

Start Date21 Feb 2023

Sponsor / Collaborator

Ultragenyx Pharmaceutical, Inc.

NCT04863014

/ TerminatedPhase 2

A Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of Evinacumab in Patients With Severe Hypertriglyceridemia for the Prevention of Recurrent Acute Pancreatitis

The primary objective of the study is to determine the proportion of patients with elevated triglycerides (TG), without familial chylomicronemia syndrome (FCS) due to loss of function (LoF) mutations in lipoprotein lipase (LPL), and a history of hypertriglyceridemia (HTG)-associated acute pancreatitis (AP) who experience a recurrent episode of AP after treatment with evinacumab versus placebo.
The secondary objectives of the study are:
To determine the change in the standard lipid profile after therapy with evinacumab versus placebo
To determine the changes in specialty lipoprotein parameters (ApoC3, ApoB48, ApoB100, and nuclear magnetic resonance [NMR] lipid profile) after therapy with evinacumab versus placebo
To measure the number of AP episodes per patient
To assess the safety and tolerability of evinacumab
To assess the potential immunogenicity of evinacumab
To assess the concentrations of total evinacumab and total angiopoietin-like 3 (ANGPTL3)

Start Date12 Jul 2021

Sponsor / Collaborator

Regeneron Pharmaceuticals, Inc.

100 Clinical Results associated with Evinacumab-dgnb

100 Translational Medicine associated with Evinacumab-dgnb

100 Patents (Medical) associated with Evinacumab-dgnb

139

Literatures (Medical) associated with Evinacumab-dgnb

01 May 2025·Molecular Diagnosis & Therapy

Molecular Therapeutics in Development to Treat Hyperlipoproteinemia

Review

Author: Ahmad, Maud ; Hegele, Robert A

Clinical endpoints caused by hyperlipoproteinemia include atherosclerotic cardiovascular disease and acute pancreatitis. Emerging lipid-lowering therapies targeting proprotein convertase subtilisin/kexin 9 (PCSK9), lipoprotein(a), apolipoprotein C-III, and angiopoietin-like protein 3 represent promising advances in the management of patients with hyperlipoproteinemia. These therapies offer novel approaches for lowering pathogenic lipid and lipoprotein species, particularly in patients with serious perturbations who are not adequately controlled with conventional treatments or who are unable to tolerate them. Molecular targets for these novel therapeutic agents were identified and validated through genetic epidemiology studies. Proprotein convertase subtilisin/kexin 9 inhibitors (e.g., monoclonal antibodies and small interfering RNA) have revolutionized hypercholesterolemia management by significantly reducing both low-density lipoprotein cholesterol levels and major cardiovascular events. Genome editing of PCSK9 promises to provide a potential cure for patients with familial hypercholesterolemia. Several investigational lipoprotein(a)-targeting therapies aim to reduce the risk of atherosclerotic cardiovascular disease and aortic valve disease, although definitive clinical endpoint studies remain to be completed. Inhibition of APOC3 messenger RNA expression by olezarsen and plozasiran significantly lowers plasma triglyceride levels and markedly reduces pancreatitis risk in patients with familial chylomicronemia syndrome. Finally, angiopoietin-like protein 3 inhibition by the monoclonal antibody evinacumab has transformed management of patients with homozygous familial hypercholesterolemia. Together, these novel agents expand the therapeutic cache, offering personalized lipid-lowering strategies for high-risk patients with hyperlipoproteinemia, improving clinical outcomes and addressing previously unmet medical needs.

01 May 2025·ADVANCES IN THERAPY

Evinacumab for Homozygous Familial Hypercholesterolemia: The Italian Cohort of the ELIPSE HoFH Study

Article

Author: Di Taranto, Maria Donata ; Di Minno, Matteo ; Gentile, Marco ; De Ruberto, Francesca ; Fortunato, Giuliana ; Iannuzzo, Gabriella ; Stanzione, Claudia ; Calcaterra, Ilenia

INTRODUCTION:

Homozygous familial hypercholesterolemia (HoFH) is a severe rare genetic disorder characterized by elevated plasma low-density lipoprotein (LDL) cholesterol levels. Here, we report data from the Italian cohort of the Evinacumab Lipid Studies in Patients with Homozygous Familial Hypercholesterolemia (ELIPSE HoFH) trial.

METHODS:

ELIPSE HoFH was conducted at 30 sites in 11 countries, with 2-10 patients enrolled per country. The study included patients aged ≥ 12 years with LDL cholesterol ≥ 70 mg/dl (1.8 mmol per liter) at screening despite stable maximally tolerated lipid-lowering therapy. Patients were randomly assigned evinacumab (15 mg/kg every 4 weeks) or matching placebo for 24 weeks, with an option for a 24-week open-label extension or follow-up period thereafter. The Italian cohort included seven patients assigned to evinacumab.

RESULTS:

Five patients (3 males and 2 females) received evinacumab and were included in this report. Substantial and consistent reductions in LDL cholesterol from baseline levels were observed in all patients at all follow-up time points. Overall, an 84.5% decrease in median (range) LDL cholesterol was observed, from 323 (203-587) mg/dl in 2016 to 50.0 (13-103) mg/dl (P = 0.043) in 2019, with LDL cholesterol levels stable through 2023. Total cholesterol, high-density lipoprotein (HDL) cholesterol, non-HDL cholesterol, and triglycerides decreased markedly over time. Evinacumab was well tolerated, with no treatment-related adverse events reported.

CONCLUSION:

Evinacumab substantially lowered LDL cholesterol levels in patients with HoFH regardless of the degree of LDL receptor function, with low levels sustained over 5 years of follow-up.

TRIAL REGISTRATION:

ClinicalTrials.gov identifier NCT03399786 registered 16 January 2018.

26 Mar 2025·JOURNAL OF PEDIATRIC ENDOCRINOLOGY & METABOLISM

Dramatic response to Evinacumab in a North Indian girl with homozygous familial hypercholesterolemia

Article

Author: George, Arun ; Arora, Sonali ; Bala, Anju ; Banerjee, Sayan ; Rohit, Manoj Kumar ; Thingnam, Shyam Kumar Singh ; Dayal, Devi

Abstract:

Objectives:

Homozygous familial hypercholesterolemia (HoFH) is a rare inherited disorder of lipoprotein metabolism associated with significant morbidity and early mortality. The conventional management with lipid-lowering drugs and lipoprotein apheresis is unable to consistently achieve guidelines recommended low-density lipoprotein cholesterol (LDL-C). We aim to describe the efficacy of Evinacumab, a recently approved monoclonal antibody, in lowering LDL-C in an Indian girl with HoFH.

Case presentation:

A 16-year-old girl was diagnosed with HoFH at age two years after the appearance of cutaneous and tendo-calcaneus xanthomas. Genetic testing revealed a pathogenic homozygous deletion in the LDL receptor (LDLR) gene. Despite lifestyle measures, a low-fat diet, and maximum doses of atorvastatin and ezetimibe, her average LDL-C level remained 320.3 mg/dL over the past decade. Her caregivers did not accept LDL-apheresis. She also did not respond to Evolocumab therapy. The patient developed progressive calcific aortic stenosis and concentric left ventricular hypertrophy, necessitating aortic valve replacement surgery at age 16 years. The recent addition of Evinacumab to her lipid-lowering drug regimen, resulted in a significant LDL-C reduction of 76.16 %, bringing levels down to 82 mg/dL. These levels were sustained over the last four months.

Conclusions:

Evinacumab offers a promising option for managing high-risk and difficult-to-treat HoFH patients. This is the first Indian child receiving Evinacumab for HoFH.

147

News (Medical) associated with Evinacumab-dgnb

15 May 2025

·investor.regeneron.com

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 Praluent Praluent 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 VelocImmune Technology Regeneron'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 Information Do 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 www.fda.gov/medwatch or call 1-800-FDA-1088. Please click here for full Prescribing Information. About Regeneron Regeneron (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 www.Regeneron.com or follow Regeneron on LinkedIn, Instagram, Facebook or X. Forward-Looking Statements and Use of Digital Media This 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 (https://investor.regeneron.com) and its LinkedIn page (https://www.linkedin.com/company/regeneron-pharmaceuticals). Contacts: Source: Regeneron Pharmaceuticals, Inc.

Drug ApprovalPatent Infringement

07 May 2025

·endpts.com

CRISPR Therapeutics' gene editing therapy reduces bad cholesterol and triglycerides by as much as 80%

CRISPR Therapeutics has revealed promising but early data from its first clinical study of a gene editing therapy to lower the risk of heart disease, setting the stage for competition with other drugmakers working on similar treatments. The therapy, dubbed CTX310, uses lipid nanoparticles (LNPs) to deliver the CRISPR therapy into the liver, where it cuts a gene called ANGPTL3. The approach aims to mimic the effects of natural mutations that cause some people to have unusually low levels of cholesterol and triglycerides. The Phase 1 trial tested four dose levels in 10 people, but the most dramatic results came from the sole patient who got the highest dose, which reduced their triglyceride levels by 82% and low-density lipoprotein (LDL) — often called “bad” cholesterol — by 65% a month after the infusion. Three patients who got the second-highest dose had their triglycerides reduced by an average of 56% and their LDL cholesterol by an average of 29%, although one of those patients had an 81% reduction. The six people who got the smallest two doses had only minor triglyceride reduction. The topline results were announced Tuesday afternoon in a press release . It was the first time that CRISPR Therapeutics shared results from a study testing its namesake technology as an infusion directly into the body. “We’re seeing dramatic reductions in triglycerides,” CRISPR Therapeutics CEO Samarth Kulkarni told Endpoints News in an interview. “Gene editing works.” Much larger studies will be needed to prove the safety and efficacy of the drug, but the preliminary results build on positive data from Beam Therapeutics, Intellia Therapeutics and Verve Therapeutics, which have also successfully used lipid nanoparticles to deliver gene editing therapies to the liver. “Once we establish an LNP platform for the liver, we can keep adding targets and developing more drugs in a derisked fashion,” Kulkarni said. “And the mRNA we make is applicable to all the programs, so our costs are going to go down over time, too.” CRISPR Therapeutics hopes that its treatment may one day lower the risk of heart disease in many people. But first, it is testing the drug in patients with severe conditions, including familial hypercholesterolemia, severe hypertriglyceridemia and mixed dyslipidemias. The company said the two highest doses of its therapy reduced ANGPTL3 by up to 75%, but didn’t say what the average reduction was. There were no severe adverse reactions to the drug. And there were no meaningful changes to liver enzyme levels or platelets, two potential problems caused by some gene therapies. CRISPR’s triglyceride reduction appears roughly on par with the 52% lowering seen in a Phase 2 study of solbinsiran, an siRNA drug made by Eli Lilly that also targets ANGPTL3. But CRISPR Therapeutics is quick to note its potential edge in LDL cholesterol, which Lilly’s drug only reduced by about 12% to 17%. “The LDL reduction seems to be a lot higher than some of the siRNA therapies. So this could end up being a best-in-class therapy,” Kulkarni said. “So from that perspective, we’re absolutely thrilled.” Regeneron already sells an antibody drug called Evkeeza that also targets ANGPTL3. In a study of people with familial hypercholesterolemia, the drug reduced LDL cholesterol by 49% and triglycerides by 50%. By targeting a different gene called PCSK9, Verve has reduced LDL cholesterol levels by about 53% in a recent study . The company also began a clinical trial of a therapy that targets ANGPTL3 last year, and it expects to share updates in the second half of 2025. CRISPR Therapeutics expects to present more details at a medical conference in the second half of 2025. The company is also planning to share data from a Phase 1 study of a similar therapy that aims to reduce lipoprotein(a), another lipid linked to heart disease, in the second quarter this year. On Tuesday, the company also shared an update on the commercial launch of Casgevy, its CRISPR-edited cell therapy for sickle cell disease and beta thalassemia. More than 90 patients have had their bone marrow stem cells collected to begin the process of making the therapy, and the company expects that number will “grow significantly” this year, the company said in its press release. Kulkarni told Endpoints that eight patients received Casgevy in the first quarter of 2025, a notable uptick from previous quarters but still a far cry from the roughly 300 patients the company needs to treat each year to break even on the therapy, according to one analyst . CRISPR’s stock fell more than 11% on Tuesday, coinciding with the appointment of controversial scientist Vinay Prasad as the new director of the Center for Biologics Evaluation and Research, the division of the FDA responsible for regulating gene editing. Kulkarni wouldn’t comment on the potential impact on CRISPR Therapeutics, but he did say that other recent shakeups at the agency haven’t affected the company yet. “There are a lot of people who are worried about how the FDA is functioning, and so far, for us, we’ve had interactions where it’s been business as usual. So we have not, at the ground level, felt any disruptions,” Kulkarni said. Editor’s note: A previous version of this story incorrectly said that Verve had not begun testing its ANGPTL3 therapy. The drug is in a clinical trial.

Clinical ResultPhase 1Gene TherapyPhase 2Executive Change

07 May 2025

·www.biospace.com

CRISPR’s Casgevy on the Rise With More Gene Therapy Proof of Concept To Come in 2025

iStock, ArtemisDiana CRISPR Therapeutics’ partner Vertex reported that more than 65 treatment centers have been activated for the gene therapy Casgevy. While Vertex handles the market, CRISPR has been focused on its clinical program. The outlook for CRISPR Therapeutics’ approved gene therapy Casgevy is starting to look up, with more treatment centers added over the past quarter. The positive trend comes just as the gene editing biotech reported that its cholesterol-lowering gene therapy dropped lipoprotein and triglycerides in a Phase I trial, showcasing the potential of gene therapy if the space can overcome its commercial hurdles . CRISPR’s partner Vertex reported earlier this week that more than 65 treatment centers have been activated for Casgevy, which is approved for sickle cell disease and beta-thalassemia . The revelation provided a glimmer of hope for the gene therapy space, which has been battered by bad news. Vertex reported revenue of $14.2 million for Casgevy in the first quarter, up a modest but encouraging 4%, according to William Blair analysts. This growth signals that “the ramp in patient starts is beginning to convert to a ramp in cell infusions,” the group wrote in a note to investors Wednesday morning. In September 2024, patients finally started to receive infusions of Casgevy, 10 months after its December 2023 approval. It’s been a slow build for a modality that could have profound impacts on the lives of patients. The treatments have struggled to gain market share; they are expensive and challenging for patients to undergo. The companies involved have also had to build out a network of treatment centers capable of supporting patients through the process. As CRISPR and Vertex have charged ahead, rival bluebird bio has fallen behind. Once a promising gene therapy company, bluebird sold itself to private equity earlier this year for a mere $30 million after failing to reach profitability in time to make up a looming cash gap . But CRISPR has the backing of a much larger pharma company in Vertex, which has been leading the marketing efforts. Vertex has set a goal of activating 75 treatment centers globally, and has hit 65 already, Chief Operating Officer Stuart Arbuckle explained on Vertex’s first quarter earnings report Monday. “Casgevy truly does have the potential to be a multibillion-dollar product for Vertex,” Arbuckle said. About 90 patients have undergone the initial cell collection process, according to Arbuckle, and more than twice that number have been referred to begin treatment. A total of eight patients have completed treatment. “It’s been inspiring to hear that Casgevy patients now feel able to live their lives in ways they never have before,” Arbuckle said. “Whether that means having the energy to play with their kids, taking up snowboarding without fear that the cold might bring on a pain crisis or investing in their education and careers given expectations now for a longer and healthier life. It is a privilege to be part of their journey.” Laser-Focused While Vertex handles the market, CRISPR has been cleared to focus on its clinical program, such as the in vivo liver editing program CTX310. On Tuesday, the company reported a small batch of data from just 10 patients with elevated lipoprotein (LDL) and triglyceride (TG) levels in a first-in-human trial of the gene therapy. A single dose of the treatment decreased expression of the ANGPTL3 gene, which is involved in the regulation of LDL and TG levels. Both LDL and TG decreased as well. One patient with severe hypertriglyceridemia had an 82% reduction in TG. Another with heterozygous familial hypercholesterolemia had an 81% reduction in LDL-C, a measure of the cholesterol carried by LDL particles. William Blair said the data “looks competitive” with LDL-C reductions seen with Arrowhead Therapeutics ARO-ANG3 and Regeneron’s approved therapy Evkeeza. CTX310 was well tolerated and there were no severe adverse events reported. CRISPR also noted that liver enzymes were not elevated at any dose level. CRISPR heralded the results as a “significant milestone” for its proprietary lipid nanoparticle (LNP) delivery technology for gene editing in the liver. More data from the Phase I trial will be presented at a medical meeting later this year. CRISPR is also developing CTX320 for cardiovascular disease, with a Phase I readout expected in the second quarter. “We also think the safety pro CTX310 de-risks the upcoming readout from CTX320, and we highlight that CRISPR is the only company with an in vivo gene-editing candidate targeting Lp(a) in the clinic,” William Blair said. The company is also working on two preclinical programs, CTX340 for refractory hypertension and CTX450 for acute hepatic porphyrias. In oncology, CRISPR will have a readout for a Phase I trial of CTX131 in solid tumors and blood cancers sometime this year. An update for the regenerative medicine diabetes treatment CTX211 is also expected before the end of 2025. “We view CRISPR as having a catalyst-rich 12-month period ahead as it advances candidates across its ex vivo and in vivo platforms through the clinic,” William Blair wrote.

Phase 1Clinical ResultGene Therapy

100 Deals associated with Evinacumab-dgnb

External Link

KEGG	Wiki	ATC	Drug Bank
D11753	Evinacumab-dgnb	C10	DB15354

R&D Status

Approved

10 top approved records.

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Indication	Country/Location	Organization	Date
Homozygous familial hypercholesterolemia	United States	Regeneron Pharmaceuticals, Inc.	11 Feb 2021

Developing

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Hypertriglyceridemia	Phase 2	United States	Regeneron Pharmaceuticals, Inc.	07 Jun 2018
Hypertriglyceridemia	Phase 2	Canada	Regeneron Pharmaceuticals, Inc.	07 Jun 2018
Hypertriglyceridemia	Phase 2	Italy	Regeneron Pharmaceuticals, Inc.	07 Jun 2018
Hypertriglyceridemia	Phase 2	United Kingdom	Regeneron Pharmaceuticals, Inc.	07 Jun 2018
Pancreatitis	Phase 2	United States	Regeneron Pharmaceuticals, Inc.	07 Jun 2018
Pancreatitis	Phase 2	Canada	Regeneron Pharmaceuticals, Inc.	07 Jun 2018
Pancreatitis	Phase 2	Italy	Regeneron Pharmaceuticals, Inc.	07 Jun 2018
Pancreatitis	Phase 2	United Kingdom	Regeneron Pharmaceuticals, Inc.	07 Jun 2018
Hypercholesterolemia	Phase 2	United States	Regeneron Pharmaceuticals, Inc.	10 Nov 2017
Hypercholesterolemia	Phase 2	Japan	Regeneron Pharmaceuticals, Inc.	10 Nov 2017

Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT04863014 (CTgov)	Phase 2	Hypertriglyceridemia \| Pancreatitis	21	Placebo (Placebo)	kioevwyquy = pepncdjxmh gueiismrvy (amxdvfmhct, euiyemvmud - pehdeseiln) View more	-	22 May 2024
				evinacumab (Evinacumab)	kioevwyquy = nztslayxej gueiismrvy (amxdvfmhct, vstarjeunh - xmqorgyrsj) View more
NCT04233918 (Pubmed) Manual	Phase 3	Homozygous familial hypercholesterolemia	14	evinacumab 15 mg/kg	wuwjbxubmp(wxnecwmcwx) = rdvejdvtco tgfbnhokyt (ttamzkablh ) View more	Positive	30 Jan 2024
NCT04233918 (phase 3, part B, CTgov)	Phase 3	Homozygous familial hypercholesterolemia	20	Evinacumab (Part A: Evinacumab 15mg/Kg IV)	icsqnjthma(rjkllnswbf) = lduldpkjlc imdwnualtn (ppucndgxqw, 90.8) View more	-	07 Jun 2023
				Evinacumab (Part B: Evinacumab 15mg/Kg IV Q4W)	lafkluprew(oakjncnbzu) = romrylpbeu fmrqgdnfpo (rgmcpaqzwr, jfevtstxqd - xoslwzykss) View more
NCT04233918 (Corporate Publications) Manual	Phase 3	Hypercholesterolemia	14	EVINACUMAB	iahrclloze(lchnzffrpg) = msyeompykk ejamdxndwh (kpteiujaen ) View more	Positive	21 May 2022
NCT03399786 (ELIPSE HoFH, CTgov)	Phase 3	Homozygous familial hypercholesterolemia	65	Placebo (Placebo IV Q4W)	skptwwagjr(hzhmiuutoo) = htzikuqvql pmjnkuswfr (ieauzyieyl, 6.5) View more	-	18 May 2021
				Evinacumab (Evinacumab 15 mg/kg IV Q4W)	skptwwagjr(hzhmiuutoo) = vfhcjbjhtj pmjnkuswfr (ieauzyieyl, 4.6) View more
NCT03399786 (Pubmed \| Br Dent J \| N Engl J Med) Manual	Phase 3	Homozygous familial hypercholesterolemia	65	Evinacumab	zvizuesxpu(mqwvtkqqqw) = threlwbbwd yqfkprebne (bwmxrqmofv )	Positive	20 Aug 2020
				Placebo	zvizuesxpu(mqwvtkqqqw) = xdeqokxlbv yqfkprebne (bwmxrqmofv )
NCT02265952 (CTgov)	Phase 2	Homozygous familial hypercholesterolemia	9	REGN1500 (REGN1500 250 mg SC/15 mg/kg IV/450 mg SC)	zlccopqfmi(jisjpnfoso) = rgyqqhfxso thcvapoudj (iyyfeweych, 23.136) View more	-	09 Dec 2019
				REGN1500 (REGN1500 300 mg SC/20 mg/kg IV)	jmtiypsrrc(nzvndspmpo) = cyfauehlyt pxmfhvpcit (yamkpseswi, 178.792) View more
NCT02265952 (Pubmed) Manual	Phase 2	Homozygous familial hypercholesterolemia	9	evinacumab	ovtaneqcrh(fugpardbxt) = dmrkskimjp tipkhvfkla (vgssqdrafj, 18)	Positive	01 Nov 2019
NCT02265952 (ESC2017)	Phase 2	Homozygous familial hypercholesterolemia LDL-receptor mutations \| ANGPTL3	9	Evinacumab	ugkspbhqxq(qrqmaktngc) = mnodwtmpio fvwmnleyxv (bzflkvxniu ) View more	-	29 Aug 2017

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