News Article | March 2, 2017
(New York, NY - March 2, 2017) --Mount Sinai researchers have discovered that a rheumatoid arthritis drug can block a metabolic pathway that occurs in tumors with a common cancer-causing gene mutation, offering a new possible therapy for aggressive cancers with few therapeutic options, according to a study to be published in Cancer Discovery. Ramon Parsons, MD, PhD, Ward-Coleman Chair in Cancer Research and Chair of the Department of Oncological Sciences at the Icahn School of Medicine at Mount Sinai, led a team that studied how a mutation of the PTEN gene rewires a metabolic pathway in tumors, channeling increased amounts of the amino acid glutamine into the pathway, speeding up DNA production, and causing uncontrolled growth of the tumor. The team discovered that leflunomide, an oral rheumatoid arthritis drug approved by the U.S. Food and Drug Administration, blocks an enzyme in this pathway and damages the DNA created through the pathway, killing PTEN mutant cancer cells while leaving healthy cells untouched. Parsons and his team transplanted human breast cancer cells into mice to test leflunomide's efficacy. Leflunomide drastically reduced the breast cancer tumors in the mice. "Finding successful targeted therapies for cancer is a challenging but important goal in the face of insufficient treatment options," said Dr. Parsons, who discovered the PTEN gene. "Targeted therapies that are tumor-specific are much needed, and identifying changes based on specific tumor suppressor or oncogene alterations will facilitate this effort. Due to the high mutation rate of PTEN in cancer, the effects of PTEN could be at the heart of targeted therapy." This discovery has implications in aggressive cancers with the PTEN mutation and few treatment options such as triple negative breast cancer, prostate cancer, endometrial cancer, and glioblastoma, a brain cancer. Dr. Parsons hopes to create a clinical trial to further test leflunomide in patients with breast and colon cancer. This research was funded by NCI R01CA082783, R01CA155117, and P01CA97403 (R.P.), and partially supported by NIH grants 5P01CA120964 and 5P30CA006516 (J.MA.), and R01 GM041890 and the Breast Cancer Research Foundation (L.C.). The Mount Sinai Health System is an integrated health system committed to providing distinguished care, conducting transformative research, and advancing biomedical education. Structured around seven hospital campuses and a single medical school, the Health System has an extensive ambulatory network and a range of inpatient and outpatient services--from community-based facilities to tertiary and quaternary care. The System includes approximately 7,100 primary and specialty care physicians; 12 joint-venture ambulatory surgery centers; more than 140 ambulatory practices throughout the five boroughs of New York City, Westchester, Long Island, and Florida; and 31 affiliated community health centers. Physicians are affiliated with the renowned Icahn School of Medicine at Mount Sinai, which is ranked among the highest in the nation in National Institutes of Health funding per investigator. The Mount Sinai Hospital is on the "Honor Roll" of best hospitals in America, ranked No. 15 nationally in the 2016-2017 "Best Hospitals" issue of U.S. News & World Report. The Mount Sinai Hospital is also ranked as one of the nation's top 20 hospitals in Geriatrics, Gastroenterology/GI Surgery, Cardiology/Heart Surgery, Diabetes/Endocrinology, Nephrology, Neurology/Neurosurgery, and Ear, Nose & Throat, and is in the top 50 in four other specialties. New York Eye and Ear Infirmary of Mount Sinai is ranked No. 10 nationally for Ophthalmology, while Mount Sinai Beth Israel, Mount Sinai St. Luke's, and Mount Sinai West are ranked regionally. Mount Sinai's Kravis Children's Hospital is ranked in seven out of ten pediatric specialties by U.S. News & World Report in "Best Children's Hospitals." For more information, visit http://www. or find Mount Sinai on Facebook, Twitter and YouTube.
News Article | February 28, 2017
The Colon Cancer Foundation, a New York-based nonprofit dedicated to the fight against colorectal cancer, today announced a massive new public awareness campaign called “Protect Your Butt!” The new campaign will focus on the Colon Cancer Foundation’s commitment to saving lives through colon cancer awareness, prevention, and translational research programs focused on a cure and optimal care for those most affected by this disease. The campaign also will break through all stigmas and fears around colon cancer and screening, and significantly increase awareness of the disease. Over 50,000 Americans die from colon cancer each year making colon cancer the #2 cancer in the USA. The kickoff event for the Protect Your Butt! Campaign is the “World’s Largest Booty Shake,” scheduled for Wednesday, March 1, in Central Park. The World’s Largest Booty Shake is expected to draw thousands of colon cancer survivors, those passionate about the cause, and more. “Our kick-off booty shake event is just the beginning,” said Dr. Thomas Weber, the foundation’s founder. “Too many people avoid talking about colon cancer because they are unaware or scared. Our new multifaceted Protect Your Butt! Campaign will reach millions of Americans through large events, social media, email marketing, and television. We are literally going to shake this up, raise money for the cure, and save lives.” The World’s Largest Booty Shake is sponsored by the Mount Sinai Health System, and partners of the Colon Cancer Foundation, Epigenomics and Bracco. These three leading health providers focus on colon cancer prevention, screening, and research. “We are proud to be the presenting sponsor for the World’s Largest Booty Shake,” said David Greenwald, MD, Director of Clinical Gastroenterology and Endoscopy at The Mount Sinai Hospital. “Colon cancer is the No. 2 cancer killer in the United States. By raising awareness and encouraging screening, we can save more lives and change this statistic.” The World’s Largest Booty Shake event starts at noon on Wednesday, March 1, at the Central Park Band Shell, south of Bethesda Terrace between 66th and 72nd streets. Headlining the World’s Largest Booty Shake will be rising R&B star Mark MK and DJ Theo, who will debut the new R&B single: “PYB – Protect Your Butt!” The new single can be downloaded on iTunes, Spotify, Tidal, Amazon, and other services, and part of the proceeds will go towards colon cancer research. All money raised during the Protect Your Butt! Campaign helps to raise awareness of the nation’s second leading cancer killer and provides funds for colorectal cancer research, education, and prevention programs for the underserved. For more information about the Colon Cancer Foundation, visit http://www.coloncancerchallenge.org. The Colon Cancer Foundation (coloncancerchallenge.org) is a 501(c)(3) not-for-profit organization registered in New York state and listed by the Federal IRS as a public charity dedicated to reducing colorectal cancer incidence and death. Its mission includes supporting research into the causes and cures for colorectal cancer, increasing public awareness, educating the public about the importance of early detection and forming strategic partnerships in the fight against colorectal cancer. The Mount Sinai Health System is an integrated health system committed to providing distinguished care, conducting transformative research, and advancing biomedical education. Structured around seven hospital campuses and a single medical school, the Health System has an extensive ambulatory network and a range of inpatient and outpatient services—from community-based facilities to tertiary and quaternary care. The System includes approximately 7,100 primary and specialty care physicians; 12 joint-venture ambulatory surgery centers; more than 140 ambulatory practices throughout the five boroughs of New York City, Westchester, Long Island, and Florida; and 31 affiliated community health centers. Physicians are affiliated with the renowned Icahn School of Medicine at Mount Sinai, which is ranked among the highest in the nation in National Institutes of Health funding per investigator. The Mount Sinai Hospital is in the “Honor Roll” of best hospitals in America, ranked No. 15 nationally in the 2016-2017 “Best Hospitals” issue of U.S. News & World Report. The Mount Sinai Hospital is also ranked as one of the nation’s top 20 hospitals in Geriatrics, Gastroenterology/GI Surgery, Cardiology/Heart Surgery, Diabetes/Endocrinology, Nephrology, Neurology/Neurosurgery, and Ear, Nose & Throat, and is in the top 50 in four other specialties. New York Eye and Ear Infirmary of Mount Sinai is ranked No. 10 nationally for Ophthalmology, while Mount Sinai Beth Israel, Mount Sinai St. Luke's, and Mount Sinai West are ranked regionally. Mount Sinai’s Kravis Children’s Hospital is ranked in seven out of ten pediatric specialties by U.S. News & World Report in "Best Children's Hospitals." For more information, visit http://www.mountsinai.org, or find Mount Sinai on Facebook, Twitter and YouTube. Epigenomics is a molecular diagnostics company focused on blood-based detection of cancers using its proprietary DNA methylation biomarker technology. The company develops and commercializes diagnostic products across multiple cancer indications with high medical need. Epigenomics' lead product, Epi proColon, is a blood-based screening test for the detection of colorectal cancer. Epi proColon has received approval from the U.S. Food and Drug Administration (FDA) and is currently marketed in the United States, Europe, and China and selected other countries. Epigenomics’ second product, Epi proLung®, is in development as a blood-based test for lung cancer detection. Bracco Imaging S.p.A., part of the Bracco Group, is one of the world’s leading companies in the diagnostic imaging business. Headquartered in Milan, Italy, Bracco Imaging develops, manufactures and markets diagnostic imaging agents and solutions that meet medical needs. Bracco Imaging offers a product and solution portfolio for all key diagnostic imaging modalities: X-ray Imaging (including Computed Tomography-CT, Interventional Radiology, and Cardiac Catheterization), Magnetic Resonance Imaging (MRI), Contrast Enhanced Ultrasound (CEUS), and Nuclear Medicine through radioactive tracers. The diagnostic imaging portfolio is completed by a range of medical devices and advanced administration systems for contrast imaging products.
News Article | February 28, 2017
The InteloMed CVInsight® Patient Monitoring & Informatics System will be featured at booth 303 PITTSBURGH, PA--(Marketwired - Feb 28, 2017) - InteloMed, Inc., a leader in the development of advanced non-invasive systems for intelligent patient monitoring, will exhibit at the upcoming National Association of Nephrology Technicians (NANT) 34th Annual Symposium. InteloMed will feature the CVInsight® Patient Monitoring & Informatics System for continuous monitoring of cardiovascular stress to assess dialysis treatment tolerance. The NANT mission is to promote the highest quality of care for End Stage Renal Disease (ESRD) and Chronic Kidney Disease (CKD) patients through education and professionalism. The symposium will focus on quality care with innovation, education, and motivation. This year's event is being held February 21 - 22 in Las Vegas, NV. InteloMed Clinical Operations Manager Lori Poole MSN, RN will join RJ Picciano BA, CHT, OCDT, CHBT on the podium on Wednesday, February 22, highlighting the topic "Fluid Management: How the Nephrology Clinical Technician Can Help." Picciano serves as Fluid Management Coordinator for the Centers for Dialysis Care, Cleveland Ohio. "The CVInsight System provides new insight on assessing treatment tolerance, and we look forward to demonstrating that at the NANT Symposium," said InteloMed CEO Jill Schiaparelli. The CVInsight Monitoring System will be available at the InteloMed booth, number 303. For more information about the event, visit http://www.dialysistech.net/education/2017-annual-symposium. About InteloMed InteloMed is redefining the standard of care in patient monitoring and informatics through its intelligent, multi-dimensional, non-invasive CVInsight® Patient Monitoring & Informatics System. Providing an unprecedented level of physiologic insight, the CVInsight® Monitoring System is an easy-to-use, multi-parameter system for monitoring cardiovascular stress. It empowers clinicians with non-invasive, real-time, dynamic, and actionable information about a patient's dialysis tolerance. Based on patented and proprietary algorithms, the system provides clinician set alerts to notify healthcare providers of changes in the patient's tolerance to dialysis treatment, allowing them to intervene to avoid dialysis interruptions that can have serious patient consequences. The CVInsight® Patient Monitoring and Informatics System is FDA-cleared and CE-marked and has launched commercially in the United States. InteloMed is headquartered in Wexford, PA. For more information, visit us at www.InteloMed.com. Safe Harbor Statement Certain statements in this release are "forward-looking" and as such are subject to numerous risks and uncertainties. Actual results may vary significantly from the results expressed or implied in such statements. Factors that could cause actual results to materially differ from forward-looking statements include, but are not limited to, the need to obtain additional capital in order to grow our business, our ability to engage qualified employees, the acceptance of new products by doctors and hospitals, changes in the regulatory environment and healthcare legislation, the need to keep pace with technological changes, our dependence on third party manufacturers to produce components of our technology platform on time and to our specifications, and the successful implementation of our commercial strategies.
News Article | March 1, 2017
VICTORIA, British Columbia--(BUSINESS WIRE)--Aurinia Pharmaceuticals Inc. (NASDAQ:AUPH / TSX:AUP) (“Aurinia” or the “Company”), a clinical stage biopharmaceutical company focused on the global immunology market, today announced top-line results from its Phase IIb AURA-LV (AURA) study in lupus nephritis (LN). At 48 weeks, the trial met the complete and partial remission (“CR”/ “PR”) endpoints, demonstrating statistically significantly greater CR and PR in patients in both low dose (23.7mg of voclosporin twice daily (p<.001)) and high dose (39.5mg twice daily (p=.026)) cohorts versus the control group. Each arm of the study included the current standard of care of mycophenolate mofetil (MMF) as background therapy and a forced steroid taper to 5mg/day by week 8 and 2.5mg by week 16. No unexpected safety signals were observed and there were no additional deaths in the voclosporin treated patients; however, there were three deaths and one malignancy reported in the control arm after completion of the study treatment period. Additional data analyses for the AURA study at 48 weeks will be released at future corporate, medical and scientific meetings. “ Lupus nephritis (LN) is one of the most severe complications of systemic lupus erythematosus. The current treatments of LN are toxic and the complete renal response rates are unacceptably low. For the last several years the community of lupus researchers in collaboration with the pharmaceutical industry have been engaged in finding more effective therapies for LN, but success has been difficult to achieve,” said Brad Rovin, MD, FASN, Director of Nephrology and Vice Chairman of Research for the Department of Internal Medicine at the Ohio State University Wexner Medical Center. “ The AURA trial’s long-term results convincingly demonstrate that the addition of voclosporin to standard of care treatment is superior to standard of care alone. These data are not only statistically significant, but clinically important. Twice as many patients given 23.7mg voclosporin twice daily achieved a complete renal response compared to those treated with placebo. This is an impressive renal response rate and these results may shift the treatment paradigm of LN. Based on these encouraging data, I am looking forward to the Phase III trial of voclosporin in LN.” The 24 and 48-week top-line efficacy results are summarized below: *All p-values are vs control “ We are grateful to both the patients and investigators who have worked with us on this groundbreaking program. Voclosporin is the first and only treatment candidate that has demonstrated such a clear treatment effect for LN patients,” said Neil Solomons, MD, Aurinia’s Chief Medical Officer. “ These data provide us with tremendous confidence that we can execute a successful Phase III program and make a meaningful impact on patients’ lives.” “ Lupus nephritis carries with it life-threatening complications, including kidney failure. The treatment of the disease is challenging, and steroid side-effects are often difficult for patients to endure. The National Kidney Foundation supports the development of steroid-sparing treatment options, and we look forward to following the results of the Phase III trial,” said Joseph Vassalotti, MD, Chief Medical Officer, National Kidney Foundation. Conference Call and Webcast Details Aurinia will host a conference call and webcast tomorrow, March 2, 2017 at 8:30am Eastern Standard Time to provide an overview of the AURA 48-week top-line results. In order to participate in the conference call, please dial +1-877-407-9170 (Toll-free U.S. & Canada). An audio webcast can be accessed under "News/Events” through the “Investors” section of the Aurinia corporate website at www.auriniapharma.com. A replay of the webcast will be available on Aurinia’s website. About AURA-LV The AURA–LV study (Aurinia Urinary protein Reduction in Active Lupus with Voclosporin) is a 48-week study comparing the efficacy of two doses of voclosporin added to current standard of care of MMF against standard of care with placebo in achieving CR in patients with active LN. All arms also received low doses of corticosteroids as background therapy. 265 patients were enrolled at centers in 20 countries worldwide. On entry to the study, patients were required to have a diagnosis of LN according to established diagnostic criteria (American College of Rheumatology) and clinical and biopsy features indicative of highly active nephritis. The 24-week primary and secondary endpoints were released in Q3 2016 where the primary and all secondary endpoints were met. CR is a composite endpoint that includes: confirmed UPCR of ≤0.5 mg/mg; normal, stable renal function (≥60 mL/min/1.73m2 or no confirmed decrease from baseline in eGFR of ≥20%); presence of sustained, low dose steroids (≤10mg prednisone from week 16-24); and no administration of rescue medications. PR in the trial is measured by a ≥50% reduction in UPCR with no concomitant use of rescue medication. About Voclosporin Voclosporin, an investigational drug, is a novel and potentially best-in-class calcineurin inhibitor (“CNI”) with clinical data in over 2,200 patients across indications. Voclosporin is an immunosuppressant, with a synergistic and dual mechanism of action that has the potential to improve near- and long-term outcomes in LN when added to standard of care (MMF). By inhibiting calcineurin, voclosporin blocks IL-2 expression and T-cell mediated immune responses. It is made by a modification of a single amino acid of the cyclosporine molecule which has shown a more predictable pharmacokinetic and pharmacodynamic relationship, an increase in potency, an altered metabolic profile, and potential for flat dosing. The Company anticipates that upon regulatory approval, patent protection for voclosporin will be extended in the United States and certain other major markets, including Europe and Japan, until at least October 2027 under the Hatch-Waxman Act and comparable laws in other countries. About Lupus Nephritis (LN) LN in an inflammation of the kidney caused by Systemic Lupus Erythematosus (“SLE”) and represents a serious progression of SLE. SLE is a chronic, complex and often disabling disorder and affects more than 500,000 people in the United States (mostly women). The disease is highly heterogeneous, affecting a wide range of organs & tissue systems. It is estimated that as many as 60% of all SLE patients have clinical LN requiring treatment. Unlike SLE, LN has straightforward disease outcomes where an early response correlates with long-term outcomes, measured by proteinuria. In patients with LN, renal damage results in proteinuria and/or hematuria and a decrease in renal function as evidenced by reduced estimated glomerular filtration rate (eGFR), and increased serum creatinine levels. LN is debilitating and costly and if poorly controlled, LN can lead to permanent and irreversible tissue damage within the kidney, resulting in end-stage renal disease (ESRD), thus making LN a serious and potentially life-threatening condition. About Aurinia Aurinia is a clinical stage biopharmaceutical company focused on developing and commercializing therapies to treat targeted patient populations that are suffering from serious diseases with a high unmet medical need. The company is currently developing voclosporin, an investigational drug, for the treatment of LN. The company is headquartered in Victoria, BC and focuses its development efforts globally. www.auriniapharma.com Forward Looking Statements This press release contains forward-looking statements, including statements related to Aurinia’s ability to execute a successful Phase III program and voclosporin potentially shifting the treatment paradigm for LN, Aurinia's analysis, assessment and conclusions of the results of the AURA-LV clinical study. It is possible that such results or conclusions may change based on further analyses of these data. Words such as "plans," "intends," “may,” "will," "believe," and similar expressions are intended to identify forward-looking statements. These forward-looking statements are based upon Aurinia’s current expectations. Forward-looking statements involve risks and uncertainties. Aurinia’s actual results and the timing of events could differ materially from those anticipated in such forward-looking statements as a result of these risks and uncertainties, which include, without limitation, the risk that Aurinia’s analyses, assessment and conclusions of the results of the AURA-LV clinical study set forth in this release may change based on further analyses of such data, and the risk that Aurinia’s clinical studies for voclosporin may not lead to regulatory approval. These and other risk factors are discussed under "Risk Factors" and elsewhere in Aurinia’s Annual Information Form for the year ended December 31, 2015 filed with Canadian securities authorities and available at www.sedar.com and on Form 40-F with the U.S. Securities Exchange Commission and available at www.sec.gov, each as updated by subsequent filings, including filings on Form 6-K. Aurinia expressly disclaims any obligation or undertaking to release publicly any updates or revisions to any forward-looking statements contained herein to reflect any change in Aurinia's expectations with regard thereto or any change in events, conditions or circumstances on which any such statements are based, except as required by law.
News Article | February 28, 2017
CAMBRIDGE, Mass. & TOKYO--(BUSINESS WIRE)--Affinivax, Inc. ("Affinivax") and Astellas Pharma Inc. ("Astellas") today announced they have entered into an exclusive worldwide license agreement to develop and commercialize a vaccine targeting Streptococcus pneumoniae (pneumococcus). The partnership will utilize Affinivax’s proprietary vaccine technology platform – Multiple Antigen Presenting System (MAPS) – to advance a novel MAPS vaccine targeted to prevent and reduce the spread of pneumococcal disease. “I am pleased to enter into this agreement and partner with Affinivax to develop a pneumococcal vaccine using their novel MAPS technology.” said Kenji Yasukawa, Ph.D., Senior Vice President and Chief Strategy Officer of Astellas. “Astellas expects to provide a new option to prevent pneumococcal disease with high unmet medical needs by developing innovative MAPS vaccine as part of our commitment to serve patients interests worldwide.” Astellas will lead and fully fund the development program and will obtain worldwide rights to commercialize the MAPS vaccine for pneumococcal disease. Affinivax will receive an initial upfront payment of $10 million from Astellas and will be eligible to receive a range of development milestones and commercial milestones. In addition, Affinivax will receive tiered royalties on any future product sales. The MAPS vaccine platform is designed to enable the high affinity binding of protective polysaccharides and proteins in a single vaccine, offering the potential to provide broader protection against invasive disease1) than currently available vaccines, as well as the potential to reduce nasopharyngeal colonization (the first step in disease transmission). To date, Affinivax has advanced its novel vaccine targeting pneumococcus through nonclinical proof-of-concept studies. "We are very pleased that Astellas has recognized the significant potential of our MAPS pneumococcal vaccine," said Mr. Steven B. Brugger, CEO of Affinivax, Inc. “We are excited to join forces with Astellas to advance a vaccine which can offer such an important global benefit for pneumococcal disease and contribute to Astellas’ impact with its global vaccine franchise” There is no impact of this transaction on Astellas' financial results for the fiscal year ending March 31, 2017. 1)Invasive disease: Invasive disease is defined as an infection confirmed by the isolation of bacteria from a normally sterile site (eg, blood or cerebrospinal fluid but not sputum). About the Global Impact of Pneumococcal Disease Pneumococcus is a bacterium frequently found in the upper respiratory tract of healthy children and adults, and can cause serious infections ranging from pneumonia, meningitis, and sepsis, representing a major global health problem. The World Health Organization estimates that more than 1.6 million people, including more than 800,000 children under 5 years old, die every year from pneumococcal infections, with most of these deaths occurring in low-resource countries. The U.S. Centers for Disease Control and Prevention estimates that about 900,000 cases occur in the U.S. alone, resulting in up to 400,000 hospitalizations and 50,000 deaths annually. MAPS uses proprietary chemistry that capitalizes on the highly specific and durable non-covalent, affinity binding between biotin and rhizavidin, a biotin-binding protein. The highly stable complex created by this affinity binding contributes to a simple, modular, and efficient approach to the development of MAPS vaccines. In stark contrast to the highly complex chemistry of conventional vaccine conjugation (which is optimized to induce protective antibody responses to only the polysaccharide antigen with the protein antigen serving primarily as a carrier), a MAPS vaccine presents both the polysaccharide antigen and the protein antigen to induce an immune response. Affinivax is advancing a next generation vaccine technology platform to enable the development of vaccines that provide the highest level of protection against challenging infectious diseases. Backed by an investment from the Bill & Melinda Gates Foundation, and working with world experts in vaccine discovery and development, Affinivax is focused on creating a pipeline of vaccines for children and adults in both the developed and developing worlds. The company’s proprietary vaccine platform, called Multiple Antigen Presentation System (MAPS), enables the high affinity binding of protective polysaccharides and proteins in a single vaccine and uniquely induces a broad and protective immune response. The MAPS technology provides a highly stable, modular, and efficient approach to develop vaccines against a wide range of diseases. The company has achieved preliminary preclinical proof-of-concept for several MAPS vaccines and is currently advancing its lead vaccine candidate against Streptococcus pneumoniae. Affinivax initiated a collaboration with PATH in early 2015 to help support the advancement of its lead MAPS vaccine candidate for Streptococcus pneumoniae (pneumococcal disease), and since September 2015, Affinivax has collaborated with Astellas and its R&D partner, ClearPath Development Company to research and develop MAPS vaccines to prevent bacterial nosocomial infections. For more information, visit www.affinivax.com. Astellas Pharma Inc., based in Tokyo, Japan, is a company dedicated to improving the health of people around the world through the provision of innovative and reliable pharmaceutical products. We focus on Urology, Oncology, Immunology, Nephrology and Neuroscience as prioritized therapeutic areas while advancing new therapeutic areas and discovery research leveraging new technologies/modalities. We are also creating new value by combining internal capabilities and external expertise in the medical/healthcare business. Astellas is on the forefront of healthcare change to turn innovative science into value for patients. For more information, visit www.astellas.com/en. The safety and efficacy of the agent discussed herein are under investigation and have not been established. There is no guarantee that the agent will receive regulatory approval and become commercially available for uses being investigated. Information about pharmaceutical products (including products currently in development) which is included in this press release is not intended to constitute an advertisement or medical advice. In this press release, statements made with respect to current plans, estimates, strategies and beliefs and other statements that are not historical facts are forward-looking statements about the future performance of Astellas. These statements are based on management’s current assumptions and beliefs in light of the information currently available to it and involve known and unknown risks and uncertainties. A number of factors could cause actual results to differ materially from those discussed in the forward-looking statements. Such factors include, but are not limited to: (i) changes in general economic conditions and in laws and regulations, relating to pharmaceutical markets, (ii) currency exchange rate fluctuations, (iii) delays in new product launches, (iv) the inability of Astellas to market existing and new products effectively, (v) the inability of Astellas to continue to effectively research and develop products accepted by customers in highly competitive markets, and (vi) infringements of Astellas’ intellectual property rights by third parties.
News Article | February 21, 2017
Markers of Acute Kidney Injury to be Monitored in Forthcoming Clinical Trials BOSTON, MA and MORRISTOWN, NJ--(Marketwired - February 21, 2017) - BioAegis Therapeutics Inc., a privately held biotechnology company exploiting plasma gelsolin's (pGSN) role in immune function announced that it has expanded its clinical advisory board to include John Kellum, M.D. -- noted critical care physician and acute kidney injury expert at the University of Pittsburgh Medical Center. John Kellum, M.D. is a clinician scientist whose research interests span various aspects of Critical Care Medicine, but center in critical care nephrology (including acid-base, and renal replacement therapy), sepsis and multi-organ failure (including blood purification), and clinical epidemiology. His research has received continuous funding from the National Institutes of Health since 2001, and he has active funding from multiple different NIH Institutes. Dr. Kellum has authored more than 300 publications and has also edited several major textbooks including Critical Care Nephrology and Stewart's Textbook of Acid-Base. Steven Opal, M.D. Professor of Medicine, Brown University School of Medicine; Chief, Infectious Disease Division, Memorial Hospital of RI Atul Malhotra, M.D. Division Chief, Pulmonary and Critical Care Medicine, Kenneth M. Moser Professor, Department of Medicine University of California-San Diego. Vice President, American Thoracic Society (2015-16 President) Acute Kidney Injury (AKI) is known to be a common occurrence in Severe Community-Acquired Pneumonia patients (SCAP). Data from a recent study of 1,800 community-acquired pneumonia patients (GenIMS Study) indicated that approximately one-third of SCAP patients were diagnosed with AKI. Patients with AKI exhibited longer stay in the hospital and had approximately ten times the risk of death at discharge (11% versus 1.3%). Plasma Gelsolin Plasma gelsolin (pGSN) becomes depleted in a wide range of inflammatory conditions, and critically low levels associate with significant morbidity and mortality in animals and humans. Data from many independent laboratories documenting that plasma gelsolin administration produces striking recoveries from lethal infections in experimental animals -- in the absence of antibiotic therapy -- strongly supports the rationale for pGSN repletion therapy. Also in support of this approach is the fact that no adverse effects have emerged from extensive preclinical toxicity studies with plasma gelsolin nor from the administration of recombinant gelsolin by different routes to healthy and diseased humans in three separate clinical studies. Based on recent breakthrough findings by Dr. Lester Kobzik at the TH Chan Harvard School of Public Health, plasma gelsolin replacement therapy markedly decreased mortality of murine pneumonia due to its stimulation of macrophages' ability to ingest and kill bacteria. Based on prior studies demonstrating rhu-pGSN's protective effect in models of infection and organ failure, it is expected that this therapeutic will reduce the incidence of acute kidney injury and its longterm consequences. The company is planning to conduct studies to treat severe community-acquired pneumonia, a leading cause of death in the US and around the world despite antibiotics and improved levels of care. Susan Levinson, Ph.D., Chief Executive Officer of BioAegis stated, "We are delighted to add John Kellum to our prestigious clinical advisory board as we advance our development efforts towards our next clinical study." John Kellum commented, "The incidence of AKI in severe pneumonia is well-known, but the long-term adverse effects are often underappreciated. A therapeutic agent such as plasma gelsolin may have the potential to reduce acute kidney injury in these patients." About BioAegis Therapeutics Founded by a group of highly experienced pharmaceutical and business executives, BioAegis' mission is to harness the body's innate immune system to address adverse outcomes in diseases driven by inflammation and infection. This press release contains express or implied forward-looking statements, which are based on current expectations of management. These statements relate to, among other things, our expectations regarding management's plans, objectives, and strategies. These statements are neither promises nor guarantees, but are subject to a variety of risks and uncertainties, many of which are beyond our control, and which could cause actual results to differ materially from those contemplated in these forward- looking statements. BioAegis assumes no obligation to update any forward-looking statements appearing in this press release in the event of changing circumstances or otherwise, and such statements are current only as of the date they are made. Image Available: http://www.marketwire.com/library/MwGo/2017/2/21/11G130819/Images/Kellum-7b172312af54b2bbaf806fb1170e815f.jpg Image Available: http://www.marketwire.com/library/MwGo/2017/2/21/11G130819/Images/opal-e781fc12c841954da7ae7091ec80947d.jpg Image Available: http://www.marketwire.com/library/MwGo/2017/2/21/11G130819/Images/smalleratul-22c4d7ef766cbc0f858147a4442e2f42.jpg Image Available: http://www.marketwire.com/library/MwGo/2017/2/21/11G130819/Images/thompson-f7ddd39b3c3c930e8ee9e15dea473d19.jpg Image Available: http://www.marketwire.com/library/MwGo/2017/2/21/11G130819/Images/vincent-d5210971e55a8524803fa4c69bd36fe1.jpg
News Article | February 22, 2017
An early-stage clinical trial has found that, compared to a placebo, a novel medication significantly reduces potentially life-threatening episodes of swelling of the airway as well as the hands, feet, and abdomen of patients affected by a rare genetic disorder. The findings were published in the New England Journal of Medicine. Although the small study was designed to test safety of the drug, the findings were so obviously promising that an expedited phase III clinical study has just started enrolling patients at the Icahn School of Medicine at Mount Sinai and other locations. The study's co-lead author, Paula Busse, MD, is an allergist-immunologist at Mount Sinai and an associate professor at the Icahn School of Medicine. The disorder, hereditary angioedema (HAE), causes recurrent and unpredictable attacks of swelling throughout the body. What triggers these attacks is not known. The multicenter, double-blind, placebo-controlled phase 1b study of 37 patients with type 1 or 2 HAE found that all participants that used a 300-mg dose of the drug, lanadelumab, were attack-free, as were 82 percent who took 400 mg. Only 27 percent of participants who used a placebo were incident-free. "Lanadelumab appears to stop attacks of HAE before they start, and this prophylactic approach may represent an exciting new advance for patients," says Dr. Busse, who treats dozens of patients with the disorder. "Given the uncertainty of when an attack may happen, some HAE patients don't want to travel," she says. "The disease can produce such swelling for three to five days that a face is disfigured, hands can't be used, it is difficult to walk, and the abdomen can become very painful. Having a drug that can provide an ongoing buffer against HAE will be wonderful." Current therapies for preventing HAE attacks work, but there are some drawbacks, Dr. Busse adds. A drug known as a C-1 inhibitor needs to be given intravenously every three or four days, and another approach, the use of pills called androgens (a modified testosterone), can produce unwanted hair growth in women, affect mood, cause weight gain, affect lipid levels and liver function, and inhibit growth in children. Lanadelumab, on the other hand, is a monoclonal antibody that is injected every two weeks and was found to be well tolerated by patients in this trial. HAE is an autosomal, dominantly inherited blood disorder caused by a deficiency or dysfunction of a regulatory gene known as the C1 inhibitor. The C1 inhibitor protein normally suppresses the production of another protein called bradykinin, which is the cause of swelling. Without a C1 inhibitor that functions properly, a person produces more bradykinin and consumes less of it. Bradykinin causes the swelling (angioedema) by making vessels dilate and leak fluid. The study included extensive blood analysis, which closely tracked how the drug successfully suppressed production of bradykinin. "If the phase III clinical study confirms the benefit we have seen, lanadelumab will make life much easier for HAE patients," says Dr. Busse. "Many patients who don't like and therefore don't use current treatments will likely opt for this new therapy, which will hopefully reduce morbidity and mortality, and improve the quality of life of patients with HAE." Dr. Busse worked with researchers from a number of universities nationally, and with a research team from Dyax, the developer of Lanadelumab and supporter of the trial. The Mount Sinai Health System is an integrated health system committed to providing distinguished care, conducting transformative research, and advancing biomedical education. Structured around seven hospital campuses and a single medical school, the Health System has an extensive ambulatory network and a range of inpatient and outpatient services--from community-based facilities to tertiary and quaternary care. The System includes approximately 7,100 primary and specialty care physicians; 12 joint-venture ambulatory surgery centers; more than 140 ambulatory practices throughout the five boroughs of New York City, Westchester, Long Island, and Florida; and 31 affiliated community health centers. Physicians are affiliated with the renowned Icahn School of Medicine at Mount Sinai, which is ranked among the highest in the nation in National Institutes of Health funding per investigator. The Mount Sinai Hospital is in the "Honor Roll" of best hospitals in America, ranked No. 15 nationally in the 2016-2017 "Best Hospitals" issue of U.S. News & World Report. The Mount Sinai Hospital is also ranked as one of the nation's top 20 hospitals in Geriatrics, Gastroenterology/GI Surgery, Cardiology/Heart Surgery, Diabetes/Endocrinology, Nephrology, Neurology/Neurosurgery, and Ear, Nose & Throat, and is in the top 50 in four other specialties. New York Eye and Ear Infirmary of Mount Sinai is ranked No. 10 nationally for Ophthalmology, while Mount Sinai Beth Israel, Mount Sinai St. Luke's, and Mount Sinai West are ranked regionally. Mount Sinai's Kravis Children's Hospital is ranked in seven out of ten pediatric specialties by U.S. News & World Report in "Best Children's Hospitals." For more information, visit http://www. , or find Mount Sinai on Facebook, Twitter and YouTube.
News Article | February 16, 2017
(New York, NY - February 16, 2017) --Mount Sinai researchers have created a novel model that shows the step-by-step progression from normal blood cells to leukemia and its precursor diseases, creating replicas of the stages of the disease to test the efficacy of therapeutic interventions at each stage, according to a study to be published in Cell Stem Cell. This research marked the first time scientists have been able to transplant leukemia from humans to a test tube and then into mice for study, a landmark feat that will allow for valuable research to help find therapies for blood cancer patients in the future. "The new model will empower investigation into the cellular and molecular events underlying the development of leukemia in ways that were not possible before," said Eirini P. Papapetrou, MD, PhD, Associate Professor of Oncological Sciences, Medicine, Hematology, and Medical Oncology at the Icahn School of Medicine at Mount Sinai. "These findings provide a framework to aid investigation into disease mechanisms, drug responses, and the cellular and molecular events driving leukemia progression." Scientists used CRISPR (Clustered Regularly Interspaced Short Palindromic Repeat), a new, cutting-edge genome editing technology, to convert blood cells from patients with myelodysplastic syndrome (MDS) and acute myeloid leukemia (AML) to particular stem cells (called induced pluripotent stem cells) that can mimic all stages of disease progression, from a healthy state to pre-malignancy and finally full-blown leukemia. Though scientists believe that cancer develops through a step-by-step process by which a normal cell transforms to a fully malignant cell through intermediate stages, recreating the steps was challenging with previous tools. Scientists were able to manipulate the leukemia in a test tube environment, both by genetically modifying the disease-ridden stem cells at certain stages to revert to a pre-cancerous state, and by altering them so they would either progress to a severe or mild form of MDS. The ability to manipulate the leukemia to regress or progress will allow future researchers to test therapies that may be most potent at a particular stage, thus saving or extending a patient's life. Memorial Sloan Kettering Cancer Center was a valuable collaborator in this research. The lab of Michael G. Kharas, PhD, performed some of the mouse transplantation experiments in the study. "We are encouraged by the discovery that it was possible to generate potent engraftable leukemia derived from AML induced pluripotent stem cells," said Dr. Kharas, the co-corresponding author. "This work shows that integrated patient cell reprogramming and cancer genetics is a powerful way to dissect cancer progression." The progression model created through this research could also be used to develop models for more complex cancers, including solid tumors, the researchers said. The research was funded by grants from the National Institutes of Health from the National Heart, Lung, and Blood Institute and the National Institute of Diabetes and Digestive and Kidney Diseases (R01HL121570 and R00DK087923); the Damon Runyon Cancer Research Foundation, the Edward P. Evans Foundation, the Ellison Medical Foundation, the Henry and Marilyn Taub Foundation, the Babich Family Foundation and Alex's Lemonade Stand Foundation. The Mount Sinai Health System is an integrated health system committed to providing distinguished care, conducting transformative research, and advancing biomedical education. Structured around seven hospital campuses and a single medical school, the Health System has an extensive ambulatory network and a range of inpatient and outpatient services--from community-based facilities to tertiary and quaternary care. The System includes approximately 7,100 primary and specialty care physicians; 12 joint-venture ambulatory surgery centers; more than 140 ambulatory practices throughout the five boroughs of New York City, Westchester, Long Island, and Florida; and 31 affiliated community health centers. Physicians are affiliated with the renowned Icahn School of Medicine at Mount Sinai, which is ranked among the highest in the nation in National Institutes of Health funding per investigator. The Mount Sinai Hospital is on the "Honor Roll" of best hospitals in America, ranked No. 15 nationally in the 2016-2017 "Best Hospitals" issue of U.S. News & World Report. The Mount Sinai Hospital is also ranked as one of the nation's top 20 hospitals in Geriatrics, Gastroenterology/GI Surgery, Cardiology/Heart Surgery, Diabetes/Endocrinology, Nephrology, Neurology/Neurosurgery, and Ear, Nose & Throat, and is in the top 50 in four other specialties. New York Eye and Ear Infirmary of Mount Sinai is ranked No. 10 nationally for Ophthalmology, while Mount Sinai Beth Israel, Mount Sinai St. Luke's, and Mount Sinai West are ranked regionally. Mount Sinai's Kravis Children's Hospital is ranked in seven out of ten pediatric specialties by U.S. News & World Report in "Best Children's Hospitals." For more information, visit http://www. or find Mount Sinai on Facebook, Twitter and YouTube.
News Article | February 23, 2017
If a new, personalized medical treatment saves you from a stroke or kidney failure in the future, you may have to thank Flipper and his friends. NIST research chemist Ben Neely and his team have just finished creating a detailed, searchable index of all the proteins found in the bottlenose dolphin genome. While the dolphin genome was first compiled in 2008, recent technology allowed Neely to develop a more exhaustive map of all the proteins produced by the marine animal’s DNA. Since dolphins and humans are such similar mammals, this proteomics approach has wide applications. “Once you can identify all of the proteins and know their amounts as expressed by the genome, you can figure out what’s going on in the bottlenose dolphin’s biological systems in this really detailed manner,” Neely explained. In addition to improving care for bottlenose dolphins in captivity as well as assessment of wild populations, dolphin proteomics can yield useful data on the safety and health of the world’s oceanic food web. The generated proteomic data can also be compared to human’s proteins, providing researchers with new information about how the body works, leading to new medical treatment options. Specifically, researchers like Neely are exploring the dolphins vanin-1 protein, which as a human counterpart, but in much smaller amounts. Scientists believe the vanin-1 protein is the key to how dolphins quickly descend in water without damaging their organs. When marine mammals swim toward the ocean floor, they actually shut off blood flow to their organs. During dives that can last as long as 90 minutes, marine mammals restrict blood floor to their kidneys, liver, heart and lungs to shunt more oxygen to the brain. In contrast, if blood stops flowing to the organs of a human’s body for even a few minutes, the result is often catastrophic, including stroke, kidney failure and even death. So, could elevated levels of the vanin-1 protein in humans actually protect our kidneys? “There’s this gap in the knowledge about genes and the proteins [dolphins] make. We are missing a huge piece of the puzzle in how these animals do what they do,” said Mike Janech, director of the Nephrology Proteomics Laboratory at the Medical University of South Carolina. Janech, a kidney researcher and expert in proteomics, draws from the field of biomimicry, where researchers look to nature for creative solutions to human problems. In Janech’s case, his nature inspiration comes from dolphins who seem to have protective proteins that may contain clues to treatments for aging-associated diseases in humans. During a study to determine why captive dolphins were living longer in the wild and developing an insulin resistance, Janech discovered that excessively high vanin-1 levels were correlated with decreased liver function in wild dolphins, which suggests they provide a protective effect in avoiding metabolic syndrome. But the researchers weren’t done just yet. Janech and his colleagues also noticed another potential use for the special protein. The function of vanin-1 is to make vitamin B5 and in doing so it releases an antioxidant that has been shown to protect tissues from injury, like that which occurs after the hypoxia and re-oxygenation of diving and resurfacing in marine mammals. So, again, the question becomes, could this be the key to helping humans resist the hypoxia that causes acute kidney injury? While Janech has applied for a grant to study that very question, Neely and his team will continue to apply newer, high-tech analytical techniques to the study of marine animals. The protein map Neely developed was done so using an ultra-high resolution tribrid mass spectrometer, which is currently the most powerful tool available to identify and quantify proteins.