Thurston G.,Regeneron Pharmaceuticals |
Daly C.,Regeneron Pharmaceuticals
Cold Spring Harbor Perspectives in Medicine | Year: 2012
The angiopoietin-Tie signaling system is a vascular-specific receptor tyrosine kinase pathway that is essential for normal vascular development. Although the basic functioning of the pathway is understood, many uncertainties remain about the role of certain members of the pathway, particularly angiopoietin-2 (Ang2), in pathological vascular remodeling and angiogenesis. We summarize the components of the angiopoietin-Tie pathway and then focus on studies that highlight the role of Ang2 in disease settings, including cancer and inflammation. The expression of Ang2 is elevated in many cancers and types of inflammation, which prompted the development of specific reagents to block its interaction with the Tie2 receptor. The application of these reagents in preclinical models of inflammation and cancer has begun to elucidate the role of Ang2 in vascular remodeling and disease pathogenesis and has led to emerging clinical tests of Ang2 inhibitors. © 2012 Cold Spring Harbor Laboratory Press.
Regeneron Pharmaceuticals, Institute For Research In Biomedicine Irb and Yale University | Date: 2013-11-05
Genetically modified non-human animals are provided that may be used to model human hematopoietic cell development, function, or disease. The genetically modified non-human animals comprise a nucleic acid encoding human IL-6 operably linked to an IL-6 promoter. In some instances, the genetically modified non-human animal expressing human IL-6 also expresses at least one of human M-CSF, human IL-3, human GM-CSF, human SIRPa or human TPO. In some instances, the genetically modified non-human animal is immunodeficient. In some such instances, the genetically modified non-human animal is engrafted with healthy or diseased human hematopoietic cells. Also provided are methods for using the subject genetically modified non-human animals in modeling human hematopoietic cell development, function, and/or disease, as well as reagents and kits thereof that find use in making the subject genetically modified non-human animals and/or practicing the subject methods.
Regeneron Pharmaceuticals, Institute For Research In Biomedicine Irb and Yale University | Date: 2013-10-14
A mouse with a humanization of the mIL-3 gene and the mGM-CSF gene, a knockout of a mRAG gene, and a knockout of a mIl2rg subunit gene; and optionally a humanization of the TPO gene is described. A RAG/Il2rg KO/hTPO knock-in mouse is described. A mouse engrafted with human hematopoietic stem cells (HSCs) that maintains a human immune cell (HIC) population derived from the HSCs and that is infectable by a human pathogen, e.g., S. typhi or M. tuberculosis is described. A mouse that models a human pathogen infection that is poorly modeled in mice is described, e.g., a mouse that models a human mycobacterial infection, wherein the mouse develops one or more granulomas comprising human immune cells. A mouse that comprises a human hematopoietic malignancy that originates from an early human hematopoietic cells is described, e.g., a myeloid leukemia or a myeloproliferative neoplasia.
News Article | February 27, 2017
TARRYTOWN, N.Y. and CAMBRIDGE, Mass., Feb. 27, 2017 /PRNewswire/ -- Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) and Sanofi today announced that detailed results from the Phase 3 CHRONOS study will be presented as a late-breaking abstract at this year's Annual Meeting of the American...
News Article | March 1, 2017
NEW YORK, Feb. 28, 2017 /PRNewswire/ -- Summary This report provides all the information you require to better understand Regeneron Pharmaceuticals and its partnering interests and activities since 2010.Read the full report:...
News Article | February 27, 2017
CAMBRIDGE, Mass. and TARRYTOWN, N.Y., Feb. 27, 2017 /PRNewswire/ -- Sanofi and Regeneron Pharmaceuticals, Inc. today announced that detailed results from the Phase 3 CHRONOS study will be presented as a late-breaking abstract at this year's Annual Meeting of the American Academy of...
News Article | February 21, 2017
MENLO PARK, Calif., Feb. 21, 2017 (GLOBE NEWSWIRE) -- Adverum Biotechnologies, Inc. (Nasdaq:ADVM) a gene therapy company committed to discovering and developing novel medicines for patients suffering from diseases with few or burdensome treatment options, announced today that Amber Salzman, Ph.D., president and chief executive officer of Adverum, will present at two upcoming investor conferences: The audio portions of the corporate presentations will be accessible live through the investors’ section of the Company’s website at www.adverum.com. A replay will be available for 14 days following the presentations. About Adverum Biotechnologies, Inc. Adverum is a gene therapy company committed to discovering and developing novel medicines that can offer life-changing benefits to patients living with rare diseases or diseases of the eye who currently have limited or burdensome treatment options. Adverum has a robust pipeline that includes product candidates to treat wet age-related macular degeneration (wAMD), alpha-1 antitrypsin (A1AT) deficiency, and hereditary angioedema (HAE). Leveraging a next-generation adeno-associated virus (AAV)-based directed evolution platform, the Company generates product candidates designed to provide durable efficacy by inducing sustained expression of a therapeutic protein. Adverum has collaboration agreements with Regeneron Pharmaceuticals to research, develop, and commercialize gene therapy products for ophthalmic diseases and Editas Medicine to explore the delivery of genome editing medicines for the treatment of inherited retinal diseases. Adverum has clinical development expertise and core capabilities in vector optimization, process development, manufacturing, and assay development. For more information please visit www.adverum.com.
News Article | November 17, 2016
Sanofi and Regeneron Announce Praluent® (alirocumab) Cardiovascular Outcomes Trial will Continue as Planned Following Interim Analysis - ODYSSEY OUTCOMES trial1 is designed to prospectively assess the effect of Praluent on cardiovascular events in high-risk patients - Paris, France and Tarrytown, New York - November 17, 2016 - Sanofi and Regeneron Pharmaceuticals, Inc. today announced that the ongoing Praluent® (alirocumab) ODYSSEY OUTCOMES trial will continue as planned, based on the recommendation of an independent Data Monitoring Committee (DMC) after it completed a second pre-specified interim analysis. The DMC will continue to monitor the ongoing safety and efficacy of Praluent as planned. The Phase 3, multi-center, randomized, double-blind, placebo-controlled ODYSSEY OUTCOMES trial involves more than 18,000 patients from 57 countries. All patients who entered the trial had experienced a heart attack or unstable angina requiring hospitalization within a year of entering the trial, and were unable to control their LDL cholesterol despite receiving maximally-tolerated statins and potentially other lipid-lowering therapies. Patients receiving maximally-tolerated statin therapy were randomized to receive either Praluent 75 milligrams (mg) every two weeks or placebo. Patients on Praluent had their dose increased to 150 mg every two weeks at week 8 if their LDL cholesterol remained above 50 milligrams/deciliter (mg/dL).1 About Praluent Praluent is a human monoclonal antibody that inhibits the binding of PCSK9 (proprotein convertase subtilisin/kexin type 9) to the LDL receptor and thereby increases the number of available LDL receptors on the surface of liver cells, which results in lower LDL cholesterol levels in the blood. Praluent is the only PCSK9 inhibitor available in two dosages with two levels of efficacy (75 mg and 150 mg), allowing physicians to select the dose based on a patient's LDL cholesterol lowering needs. Praluent is currently approved in approximately 40 countries worldwide, including the U.S., Japan, Canada, Switzerland, Mexico, Brazil and the European Union (EU). In the U.S., Praluent is approved for use as adjunct to diet and maximally tolerated statin therapy for the treatment of adults with heterozygous familial hypercholesterolemia (HeFH) or clinical atherosclerotic CV disease, who require additional lowering of LDL cholesterol. In the E.U., Praluent is approved for the treatment of adult patients with primary hypercholesterolemia (HeFH and non-familial) or mixed dyslipidemia as an adjunct to diet: a) in combination with a statin, or statin with other lipid-lowering therapies in patients unable to reach their LDL cholesterol goals with the maximally-tolerated statin or b) alone or in combination with other lipid-lowering therapies for patients who are statin intolerant, or for whom a statin is contraindicated. The effect of Praluent on CV morbidity and mortality has not yet been determined. This medicinal product is subject to additional monitoring. This will allow quick identification of new safety information. Healthcare professionals are asked to report any suspected adverse reactions. About Sanofi Sanofi, a global healthcare leader, discovers, develops and distributes therapeutic solutions focused on patients' needs. Sanofi is organized into five global business units: Diabetes and Cardiovascular, General Medicines and Emerging Markets, Sanofi Genzyme, Sanofi Pasteur and Merial. Sanofi is listed in Paris (EURONEXT: SAN) and in New York (NYSE: SNY). About Regeneron Pharmaceuticals, Inc. Regeneron (NASDAQ: REGN) is a leading science-based biopharmaceutical company that discovers, invents, develops, manufactures and commercializes medicines for the treatment of serious medical conditions. Regeneron commercializes medicines for eye diseases, high LDL cholesterol and a rare inflammatory condition and has product candidates in development in other areas of high unmet medical need, including rheumatoid arthritis, atopic dermatitis, asthma, pain, cancer and infectious diseases. For additional information about the company, please visit www.regeneron.com or follow @Regeneron on Twitter. Sanofi Forward-Looking Statements This press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, as amended. Forward-looking statements are statements that are not historical facts. These statements include projections and estimates regarding the marketing and other potential of Praluent, or regarding potential future revenues from Praluent. Forward-looking statements are generally identified by the words "expects", "anticipates", "believes", "intends", "estimates", "plans" and similar expressions. Although Sanofi's management believes that the expectations reflected in such forward-looking statements are reasonable, investors are cautioned that forward-looking information and statements are subject to various risks and uncertainties, many of which are difficult to predict and generally beyond the control of Sanofi, that could cause actual results and developments to differ materially from those expressed in, or implied or projected by, the forward-looking information and statements. These risks and uncertainties include among other things, unexpected regulatory actions or delays, or government regulation generally, that could affect the availability or commercial potential of Praluent; the absence of guarantee that Praluent will be commercially successful, the uncertainties inherent in research and development, including future clinical data and analysis of existing clinical data relating to Praluent, including post marketing, unexpected safety, quality or manufacturing issues, competition in general, risks associated with intellectual property and any related pending or future litigation and the ultimate outcome of such litigation, volatile economic conditions, and the impact of cost containment initiatives and subsequent changes thereto, as well as those risks discussed or identified in the public filings with the SEC and the AMF made by Sanofi, including those listed under "Risk Factors" and "Cautionary Statement Regarding Forward-Looking Statements" in Sanofi's annual report on Form 20-F for the year ended December 31, 2015. Other than as required by applicable law, Sanofi does not undertake any obligation to update or revise any forward-looking information or statements. Regeneron Forward-Looking Statements and Use of Digital Media This news 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, the nature, timing, and possible success and therapeutic applications of Regeneron's products, product candidates, and research and clinical programs now underway or planned, including without limitation Praluent® (alirocumab) Injection; unforeseen safety issues and possible liability resulting from the administration of products (including without limitation Praluent) and product candidates in patients; serious complications or side effects in connection with the use of Regeneron's products and product candidates in clinical trials, such as the ODYSSEY OUTCOMES trial discussed in this news release, which is prospectively assessing the potential of Praluent to demonstrate cardiovascular benefit; the timing of completion of the ODYSSEY OUTCOMES trial; coverage and reimbursement determinations by third-party payers, including Medicare, Medicaid, and pharmacy benefit management companies; ongoing regulatory obligations and oversight impacting Regeneron's marketed products (such as Praluent), research and clinical programs, and business, including those relating to the enrollment, completion, and meeting of the relevant endpoints of post-approval studies (such as the ODYSSEY OUTCOMES trial); 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 product candidates; risks associated with intellectual property of other parties and pending or future litigation relating thereto; the likelihood, timing, and scope of possible regulatory approval and commercial launch of Regeneron's late-stage product candidates and new indications for marketed products; competing drugs and product candidates that may be superior to Regeneron's products and product candidates; uncertainty of market acceptance and commercial success of Regeneron's products and product candidates and the impact of studies (whether conducted by Regeneron or others and whether mandated or voluntary) on the commercial success of Regeneron's products and product candidates; the ability of Regeneron to manufacture and manage supply chains for multiple products and product candidates; unanticipated expenses; the costs of developing, producing, and selling products; the ability of Regeneron to meet any of its sales or other financial projections or guidance and changes to the assumptions underlying those projections or guidance; and the potential for any license or collaboration agreement, including Regeneron's agreements with Sanofi and Bayer HealthCare LLC (or their respective affiliated companies, as applicable), to be cancelled or terminated without any further product success. A more complete description of these and other material risks can be found in Regeneron's filings with the United States Securities and Exchange Commission, including its Form 10-K for the year ended December 31, 2015 and its Form 10-Q for the quarterly period ended September 30, 2016. 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 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 (http://newsroom.regeneron.com) and its Twitter feed (http://twitter.com/regeneron).
News Article | October 27, 2016
The Science and Science Careers' 2016 annual Top Employers Survey polled employees in the biotechnology, biopharmaceutical, pharmaceutical, and related industries to determine the 20 best employers in these industries as well as their driving characteristics. Respondents to the web-based survey were asked to rate companies based on 23 characteristics, including financial strength, easy adaptation to change, and a research-driven environment. This year, Regeneron Pharmaceuticals of Tarrytown, New York, receives the top honor in a ranking of the world's most respected biopharmaceutical employers. Regeneron Pharmaceuticals tops this year's list, reinforcing its dominance as it has been ranked first or second place for the last six years. It received this accolade due to its focus on innovation, teamwork, and proper maintenance of a work-life balance. The rankings, determined from a study conducted by an independent research firm commissioned by the AAAS Office of Publishing, will appear in the 28 October 2016 issue of Science and online at ScienceCareers.org on October 27 at 2:00 p.m. US Eastern Time. Like Science and Science Careers' previous rankings of biopharma employers, the 2016 survey sought to identify the companies with the best reputations as employers, based on 5,984 survey responses from readers of Science and other survey invitees. Survey participants came from North America (56%), Europe (28%), and Asia/Pacific Rim (12%); 93% work in biotechnology, biopharmaceutical, and pharmaceutical companies. Survey responses were analyzed by The Brighton Consulting Group, which used a mathematical process to assign a unique score to rate each company's employer reputation. Each company received a ranking, for example, on the basis of whether it treats its employees with respect, whether its work-culture values align with employees' personal values, and other factors. For the complete feature along with individual company rankings, go to sciencecareers.org/TopEmployers2016, which will be live beginning 2:00 p.m. US Eastern Time on October 27, 2016. The American Association for the Advancement of Science (AAAS) is the world's largest general scientific society and publisher of the journal Science as well as Science Translational Medicine, Science Signaling, a digital, open-access journal, Science Advances, Science Immunology, and - coming soon - Science Robotics. AAAS was founded in 1848 and includes nearly 250 affiliated societies and academies of science, serving 10 million individuals. Science has the largest paid circulation of any peer-reviewed general science journal in the world. The non-profit AAAS is open to all and fulfills its mission to "advance science and serve society" through initiatives in science policy, international programs, science education, public engagement, and more. For the latest research news, log onto EurekAlert!, the premier science-news Web site, a service of AAAS. See http://www. .
News Article | February 15, 2017
NEW YORK--(BUSINESS WIRE)--In its latest Cardiology Report, BioPharm Insight (BPI) reported that Amgen’s Repatha Phase III FOURIER trial will be deemed successful by experts if it shows a 35% reduction in major cardiovascular events (MACE). The report from BPI, the most comprehensive life science news and analytical solution, highlights recent editorial coverage of cardiology therapies in development that have potentially market-moving clinical events expected in the next few months. It also provides analysis of sales forecasts and licensing deals. “Amgen’s announcement at the start of February that it met its primary and key secondary composite endpoints was a hugely anticipated event for a drug predicted to have peak sales in excess of $7 billion,” said Peter Murphy, BPI senior editorial analyst. Amgen management has reiterated the cardiovascular outcomes trial (CVOT) trial is powered to show a 15% risk reduction but did not provide further details on FOURIER outcomes, which the market widely expects could be a game-changer for the lipid-lowering treatment space. While equity analysts think a 20% reduction has been hit, experts BPI spoke to said that anything lower than a 35% MACE reduction may mean that the cost outweighs the treatment benefit. “Although Amgen’s news has significant implications for the entire PCSK9-inhibitor class, experts expect to see similar results between Repatha and its competitor Praluent,” said BPI reporter Alexandra Thompson. Amgen’s (NASDAQ:AMGN) Repatha and Praluent, from Sanofi (EPA:SAN) and Regeneron Pharmaceuticals (NASDAQ:REGN), were approved within one month of each other in 2015 and are currently the only two anti-PCSK9s on the market. BPI reports, though, that so far physicians and payers alike are not satisfied that PCSK9 inhibitors successfully lower LDL-c (bad cholesterol) levels. The market is looking for evidence that the reduction in LDL also drives a reduction in the likelihood of patients suffering potentially catastrophic cardiovascular events. Praluent’s Phase III CVOT is due in late 2017, and Alnylam Therapeutics (NASDAQ:ALNY)/The Medicines Company’s (NASDAQ:MDCO)/inclisiran is due to start its own Phase III trials soon. BPI’s Cardiology Report also highlights articles covering experts’ dubious expectations for Merck’s (NYSE:MRK) hypercholesterolemia drug anacetrapib’s Phase III CV trial following termination of three drugs of the same CETP inhibitor class. Pfizer’s (NYSE:PFE) torcetrapib, Roche’s (VTX:ROG) dalcetrapib and Eli Lilly’s (NYSE:LLY) evacetrapib all suffered high-profile clinical trial failures over the past decade. Additionally, the report offers insights on several ongoing therapies under development to prevent heart failure (HF), including Novartis’ (VTX:NOVN) Entresto (sacubitril/valsartan) and Mesoblast’s (ASX:MSB) MPC-150-IM. The perforation risk of MPC-150-IM’s transendocardial delivery could possibly limit its administration to specialized cardiology cell injection centers or warrant cardiologist training, but the significant positive outcomes of the earlier Phase II trial have analysts optimistic, forecasting peak sales of the HF therapy to be $5.2 billion if successful. Learn more about expected market catalyst events in Cardiology Indications with BioPharm Insight’s full report: BioPharm Insight is the most comprehensive life science market intelligence and analytics solution, featuring a team of investigative journalists writing exclusive news and thousands of healthcare data points, aggregated into one centralized source. In addition to the proprietary articles, BioPharm Insight is currently tracking 250,000 management and R&D contacts, 7,500 biopharma companies with full pipeline data, 120,000 investigational and approved drug profiles, 28,000 M&A and licensing deals, 10,000 extended sales forecasts and epidemiology profiles for hundreds of indications.