News Article | February 17, 2017
SALT LAKE CITY, Feb. 17, 2017 (GLOBE NEWSWIRE) -- Myriad Genetics, Inc. (NASDAQ:MYGN), a global leader in personalized medicine, today announced new data demonstrating the utility of the Prolaris® test to more accurately classify mortality risk and guide the management of newly diagnosed men with prostate cancer. The data are being presented at the 2017 Genitourinary Cancers Symposium (ASCO GU) meeting in Orlando, Fla. “Myriad is pioneering personalized medicine for prostate cancer and is committed to helping men achieve their treatment goals,” said Michael Brawer, M.D., vice president of Medical Affairs, Myriad Genetic Laboratories. “We are excited about the new data on prostate cancer reclassification being presented at ASCO GU, which adds to the growing body of evidence supporting the Prolaris test and will help urologists to match treatment options with patients’ risk profiles.” This study evaluated the prognostic information provided by the Prolaris test plus CAPRA (i.e., clinical features) to generate an estimate of prostate cancer mortality within 10 years of diagnosis versus NCCN risk category as determined by clinical features alone. The analysis included data from 16,442 men who received the Prolaris test. Based on clinical features alone, men were classified according to NCCN guidelines as low (n=8,695), favorable intermediate (n=3,347), intermediate (n=3,086) or high risk (n=1,224). After recalculating the risk of prostate cancer mortality using the Prolaris test plus CAPRA, approximately one third of patients were reassigned to a different 10-year mortality risk category. The specific reclassifications by NCCN category were as follows: “Clinical features alone are useful, but as this study illustrates, and was demonstrated by our numerous prior clinical validation studies, the Prolaris test is a powerful and independent predictor of clinical outcome that can substantially improve the risk classification of newly diagnosed men with prostate cancer,” said Brawer. Follow Myriad on Twitter via @MyriadGenetics and stay informed about symposium news and updates by using the hashtag #GU17. About Prolaris® Prolaris is a novel 46-gene RNA-expression test that directly measures tumor cell growth characteristics for stratifying the risk of disease-specific mortality in patients with prostate cancer. Prolaris provides a quantitative measure of the RNA expression levels of genes involved in the progression of tumor growth. Low gene expression is associated with a low risk of disease-specific mortality in men who may be candidates for active surveillance and high gene expression is associated with a higher risk of disease-specific mortality in patients who may benefit from additional therapy. For more information visit: www.prolaris.com. About Myriad Genetics Myriad Genetics Inc., is a leading personalized medicine company dedicated to being a trusted advisor transforming patient lives worldwide with pioneering molecular diagnostics. Myriad discovers and commercializes molecular diagnostic tests that: determine the risk of developing disease, accurately diagnose disease, assess the risk of disease progression, and guide treatment decisions across six major medical specialties where molecular diagnostics can significantly improve patient care and lower healthcare costs. Myriad is focused on three strategic imperatives: transitioning and expanding its hereditary cancer testing markets, diversifying its product portfolio through the introduction of new products and increasing the revenue contribution from international markets. For more information on how Myriad is making a difference, please visit the Company's website: www.myriad.com. Myriad, the Myriad logo, BART, BRACAnalysis, Colaris, Colaris AP, myPath, myRisk, Myriad myRisk, myRisk Hereditary Cancer, myChoice, myPlan, BRACAnalysis CDx, Tumor BRACAnalysis CDx, myChoice HRD, EndoPredict, Vectra, GeneSight and Prolaris are trademarks or registered trademarks of Myriad Genetics, Inc. or its wholly owned subsidiaries in the United States and foreign countries. MYGN-F, MYGN-G. Safe Harbor Statement This press release contains “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995, including statements relating to data being presented at the at the 2017 Genitourinary Cancers Symposium; the ability of the Prolaris test to more accurately classify mortality risk and guide the management of newly diagnosed men with prostate cancer; the ability to help urologists to match treatment options with patients’ risk profiles; and the Company’s strategic directives under the caption “About Myriad Genetics.” These “forward-looking statements” are based on management’s current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those described or implied in the forward-looking statements. These risks include, but are not limited to: the risk that sales and profit margins of our existing molecular diagnostic tests and pharmaceutical and clinical services may decline or will not continue to increase at historical rates; risks related to our ability to transition from our existing product portfolio to our new tests; risks related to changes in the governmental or private insurers’ reimbursement levels for our tests or our ability to obtain reimbursement for our new tests at comparable levels to our existing tests; risks related to increased competition and the development of new competing tests and services; the risk that we may be unable to develop or achieve commercial success for additional molecular diagnostic tests and pharmaceutical and clinical services in a timely manner, or at all; the risk that we may not successfully develop new markets for our molecular diagnostic tests and pharmaceutical and clinical services, including our ability to successfully generate revenue outside the United States; the risk that licenses to the technology underlying our molecular diagnostic tests and pharmaceutical and clinical services tests and any future tests are terminated or cannot be maintained on satisfactory terms; risks related to delays or other problems with operating our laboratory testing facilities; risks related to public concern over our genetic testing in general or our tests in particular; risks related to regulatory requirements or enforcement in the United States and foreign countries and changes in the structure of the healthcare system or healthcare payment systems; risks related to our ability to obtain new corporate collaborations or licenses and acquire new technologies or businesses on satisfactory terms, if at all; risks related to our ability to successfully integrate and derive benefits from any technologies or businesses that we license or acquire, including but not limited to our acquisition of Assurex, Sividon and the Clinic; risks related to our projections about the potential market opportunity for our products; the risk that we or our licensors may be unable to protect or that third parties will infringe the proprietary technologies underlying our tests; the risk of patent-infringement claims or challenges to the validity of our patents; risks related to changes in intellectual property laws covering our molecular diagnostic tests and pharmaceutical and clinical services and patents or enforcement in the United States and foreign countries, such as the Supreme Court decision in the lawsuit brought against us by the Association for Molecular Pathology et al; risks of new, changing and competitive technologies and regulations in the United States and internationally; the risk that we may be unable to comply with financial operating covenants under our credit or lending agreements; the risk that we will be unable to pay, when due, amounts due under our credit or lending agreements; and other factors discussed under the heading “Risk Factors” contained in Item 1A of our Annual report on Form 10-K for the fiscal year ended June 30, 2016, which has been filed with the Securities and Exchange Commission, as well as any updates to those risk factors filed from time to time in our Quarterly Reports on Form 10-Q or Current Reports on Form 8-K.
News Article | February 23, 2017
ALACHUA, FL--(Marketwired - February 23, 2017) - CTD Holdings, Inc. ( : CTDH), a clinical stage biotechnology company that develops cyclodextrin-based products for the treatment of disease, today announced that it has closed a private placement of its securities with a group of accredited investors that included several directors of the Company and members of management. Investors in the private placement purchased 5,754,832 units at a price per unit of $0.35. Each unit consisted of one share of common stock and one seven-year warrant to purchase one share of common stock at an exercise price of $0.35. "The proceeds of this transaction will support CTD's Trappsol® Cyclo™ drug development program for the treatment of Niemann-Pick Type C (NPC), a rare genetic disease that causes neurologic, liver and lung dysfunction, and is ultimately fatal," said N. Scott Fine, CTD Chairman and CEO. "This private placement will provide us with the necessary resources to move forward with our clinical development program aimed at obtaining regulatory approval for Trappsol® Cyclo™ in the United States and the European Union," added Dr. Sharon Hrynkow, CTD's Senior Vice President for Medical Affairs. "We look forward to enrolling the initial patients into our U.S. Phase I and EU Phase I/II studies shortly." Scarsdale Equities acted as CTD's financial advisor in the financing. The securities sold in the private placement have not been registered under the Securities Act of 1933, as amended, and may not be offered or sold in the United States in the absence of an effective registration statement or exemption from registration requirements. This press release shall not constitute an offer to sell or the solicitation of an offer to buy, nor shall there be any sale of these securities in any state in which such offer, solicitation or sale would be unlawful prior to the registration or qualification under the securities laws of any such state. About CTD Holdings: CTD Holdings, Inc. is a clinical-stage biotechnology company that develops cyclodextrin-based products for the treatment of disease. The company's Trappsol® Cyclo™, an orphan drug designated product in the United States and Europe, is used to treat Niemann-Pick Disease Type C, a rare and fatal genetic disease. Additional indications for the active ingredient in Trappsol® Cyclo™, are in development. For additional information, visit the company's website: www.ctd-holdings.com Safe Harbor Statement: This press release contains "forward-looking statements" about the company's current expectations about future results, performance, prospects and opportunities. Statements that are not historical facts, such as "anticipates," "believes" and "expects" or similar expressions, are forward-looking statements. These statements are subject to a number of risks, uncertainties and other factors that could cause actual results in future periods to differ materially from what is expressed in, or implied by, these statements. The factors which may influence the company's future performance include the company's ability to obtain additional capital to expand operations as planned, success in achieving regulatory approval for clinical protocols, enrollment of adequate numbers of patients in clinical trials, unforeseen difficulties in showing efficacy of the company's biopharmaceutical products, success in attracting additional customers and profitable contracts, and regulatory risks associated with producing pharmaceutical grade and food products. These and other risk factors are described from time to time in the company's filings with the Securities and Exchange Commission, including, but not limited to, the company's reports on Forms 10-K and 10-Q. Unless required by law, the company assumes no obligation to update or revise any forward-looking statements as a result of new information or future events.
News Article | February 21, 2017
(Vienna, February 21, 2017) The lung is an important interface between the body and the outside environment: with each breath, a surface of roughly 100 square meters exchanges oxygen for carbon dioxide. More than 10,000 liters of air pass adult lungs every day and with this come numerous viruses, bacteria and pollutants, which need to be prevented from entering the body. To defend the organism from these intruders, the lungs harbor their own arsenal of highly specialized immune cells that are equipped to maintain the balance between host defense and tissue quiescence. However, how this balanced immune homeostasis in lungs emerged after birth, was largely unexplored. Now, for the first time, the group of Sylvia Knapp, Director of Medical Affairs at CeMM Research Center for Molecular Medicine of the Austrian Academy of Sciences and Professor of Infection Biology at the Medical University of Vienna showed with the help of mouse models that the very first breath of a newborn releases crucial signals that shape the lifelong immunological milieu of lungs. The study, published in Cell Reports (DOI:10.1016/j.celrep.2017.01.071), reveals that the mechanical forces of spontaneous ventilation at birth lead to the release of interleukin (IL)-33, a cytokine with a wide-range of effects: So-called "type 2 innate lymphoid cells" (ILC2s) follow the IL-33 signal and migrate into the lung tissue, where they release IL-13, another cytokine. This second signal determines the faith of alveolar macrophages by inducing the anti-inflammatory M2 phenotype. "ILC2-cells are crucial in defending the lungs against parasites or influenza viruses, but little was known about their role in lung homeostasis", first author Simona Saluzzo, PhD Student at CeMM and the Medical University of Vienna, explains. "Now we understand that right after birth, ILC2 are responsible for the differentiation of alveolar macrophages into specialized cells that keep the immune system in check and ensure that the lungs stay calm and healthy to ensure proper gas exchange." These ILC2-induced effects protect the lungs from excessive inflammation to daily encountered environmental triggers - but there is a catch, senior author Sylvia Knapp emphasizes: "We could show in our study that the described mechanisms are crucial in achieving lung quiescence after the first contact with the outside world. However, these processes at the same time increase the susceptibility to bacterial infections, such as caused by pneumococci. In other words: The mechanism that maintains the lung function of gas exchange at the same time explains why bacterial pneumonia is the primary cause of death by an infectious disease in Western countries." Attached pictures: 1. Fluorescent staining of IL-33 in a newborn mouse lung; 2. Graphical abstract of the study; 3. Senior author Sylvia Knapp and first author Simona Saluzzo The study "First-breath induced type-2 pathways shape the lung immune environment" was published in Cell Reports on February 21, 2017. DOI:10.1016/j.celrep.2017.01.071 The study was supported by the Austrian Science Funds (FWF), the Vienna Science and Technology Fund (WWTF) and grants from the Medical Research Council and the Wellcome Trust. Sylvia Knapp is Director of Medical Affairs at CeMM and Professor of Infection Biology at the Medical University of Vienna. She studied Medicine in Vienna and Berlin, is a board-certified Internist and obtained her PhD at the University of Amsterdam. Sylvia's research focuses on the innate immune response to bacterial infections in general, focusing specifically on the comprehensive repertoire of macrophage functions in health, development and disease. The mission of CeMM Research Center for Molecular Medicine of the Austrian Academy of Sciences is to achieve maximum scientific innovation in molecular medicine to improve healthcare. At CeMM, an international and creative team of scientists and medical doctors pursues free-minded basic life science research in a large and vibrant hospital environment of outstanding medical tradition and practice. CeMM's research is based on post-genomic technologies and focuses on societally important diseases, such as immune disorders and infections, cancer and metabolic disorders. CeMM operates in a unique mode of super-cooperation, connecting biology with medicine, experiments with computation, discovery with translation, and science with society and the arts. The goal of CeMM is to pioneer the science that nurtures the precise, personalized, predictive and preventive medicine of the future. CeMM trains a modern blend of biomedical scientists and is located at the campus of the General Hospital and the Medical University of Vienna. http://www. The Medical University of Vienna is one of the most traditional medical education establishments with nearly 7,500 students and approximately 5,500 staff members, and one of the most important top-level biomedical research institutions in Europe. Its international outlook is one of its most important pillars and the research focus is centered on immunology, cancer research, imaging, brain research and cardiovascular diseases. https:/ For further information please contact Mag. Wolfgang Däuble Media Relations Manager CeMM Research Center for Molecular Medicine of the Austrian Academy of Sciences Lazarettgasse 14, AKH BT 25.3 1090 Vienna, Austria Phone +43-1/40160-70 057 Fax +43-1/40160-970 000 email@example.com http://www.
News Article | February 22, 2017
BOSTON, Feb. 22, 2017 /PRNewswire/ -- Mitra Biotech, Inc., a global leader in advancing the personalization of cancer treatment, today announced the appointment of Anton Ehrhardt, Ph.D., as Vice President of Medical Affairs. "Anton Ehrhardt is a respected leader in Medical Affairs,...
News Article | February 28, 2017
San Diego, CA, February 28, 2017 --( Elite began operations in 1997, focused on the rapidly growing biotechnology market in San Diego, California. Within the first year, the company expanded operations nationally, and within 2 years had several global clients. As a niche, boutique agency, Elite has achieved success by delivering stellar experiences for clients and candidates throughout the entire recruitment process. Elite has achieved goals and milestones through its ongoing commitment to excellence coupled with strong client loyalty. Lisa De Benedittis, Elite Executive Search’s President, reflected on the company’s 20th anniversary by stating “Twenty years has flown by, I can recall the excitement of going out on my own and wanting to be different from other search firms that were mostly generalists. From day one, Elite was focused on the life sciences sector. I started by crafting a corporate philosophy based on ethics, rolling out the red carpet for clients and candidates, and always going the extra mile a.k.a. putting a cherry on top. That philosophy has remained our paramount priority, and has served Elite well. It has become our brand.” Offering Retained, Engaged, and Contingent search terms; Elite partners with exceptional life science organizations, uniting them with high caliber leaders and scientific experts. The company’s clients range from start-up ventures to Fortune 100 pharmaceutical companies. Specialty divisions include Clinical, Commercial, Research and Development, Regulatory Affairs, Drug Safety/Pharmacokinetics, Diagnostics, Engineering, Market Access, Reimbursement, Laboratory, Medical Affairs, Marketing, Manufacturing, Quality, Physicians, Nurses, and Sales. Lisa De Benedittis added: “We’ve weathered challenging market cycles, clients going out of business, and an ever-changing economic landscape. Thanks to our dedicated employees, loyal clients, and exceptional candidates, we’ve been able to thrive and grow. When you build teams that drive value and results for your clients, they reward you with more business, it’s a win-win.” About Elite Executive Search: Elite Executive Search is a national executive search firm specializing exclusively in the life science industries. Offering Retained, Engaged, and Contingent search terms, Elite is the partner of choice for some of the world’s leading biotechnology, pharmaceutical, medical device, and healthcare companies throughout the US. Elite is focused on sourcing scientific and healthcare experts including Directors, Vice Presidents, M.D.'s and C-Suite candidates, for all departments. Elite has an enormous life sciences network and approaches recruitment with unmatched passion, focused on delivering results. If it exits, Elite will find it. www.EliteExecSearch.com San Diego, CA, February 28, 2017 --( PR.com )-- Elite Executive Search is proud to announce its twentieth year as a leading national search firm, specializing in biotechnology, pharmaceutical, medical device and healthcare. In a highly competitive market, Elite has achieved two decades of successful placements, long-term contract /consultant assignments and steady growth. The company has been commemorating the occasion by offering a promotional discount for the month of February, 2017, for new and current clients.Elite began operations in 1997, focused on the rapidly growing biotechnology market in San Diego, California. Within the first year, the company expanded operations nationally, and within 2 years had several global clients. As a niche, boutique agency, Elite has achieved success by delivering stellar experiences for clients and candidates throughout the entire recruitment process. Elite has achieved goals and milestones through its ongoing commitment to excellence coupled with strong client loyalty.Lisa De Benedittis, Elite Executive Search’s President, reflected on the company’s 20th anniversary by stating “Twenty years has flown by, I can recall the excitement of going out on my own and wanting to be different from other search firms that were mostly generalists. From day one, Elite was focused on the life sciences sector. I started by crafting a corporate philosophy based on ethics, rolling out the red carpet for clients and candidates, and always going the extra mile a.k.a. putting a cherry on top. That philosophy has remained our paramount priority, and has served Elite well. It has become our brand.”Offering Retained, Engaged, and Contingent search terms; Elite partners with exceptional life science organizations, uniting them with high caliber leaders and scientific experts. The company’s clients range from start-up ventures to Fortune 100 pharmaceutical companies. Specialty divisions include Clinical, Commercial, Research and Development, Regulatory Affairs, Drug Safety/Pharmacokinetics, Diagnostics, Engineering, Market Access, Reimbursement, Laboratory, Medical Affairs, Marketing, Manufacturing, Quality, Physicians, Nurses, and Sales.Lisa De Benedittis added: “We’ve weathered challenging market cycles, clients going out of business, and an ever-changing economic landscape. Thanks to our dedicated employees, loyal clients, and exceptional candidates, we’ve been able to thrive and grow. When you build teams that drive value and results for your clients, they reward you with more business, it’s a win-win.”About Elite Executive Search:Elite Executive Search is a national executive search firm specializing exclusively in the life science industries. Offering Retained, Engaged, and Contingent search terms, Elite is the partner of choice for some of the world’s leading biotechnology, pharmaceutical, medical device, and healthcare companies throughout the US. Elite is focused on sourcing scientific and healthcare experts including Directors, Vice Presidents, M.D.'s and C-Suite candidates, for all departments.Elite has an enormous life sciences network and approaches recruitment with unmatched passion, focused on delivering results.If it exits, Elite will find it.www.EliteExecSearch.com Click here to view the list of recent Press Releases from Elite Executive Search
News Article | February 23, 2017
Nexstim Plc (NXTMH:HEX, NXTMS:STO) ("Nexstim" or "Company"), a medical technology company with a pioneering navigated non-invasive brain stimulation system, today presented the final results of a multi-center Phase III trial in stroke rehabilitation (NICHE trial) assessing the Company's Navigated Brain Therapy system (NBT®) at the International Stroke Conference in Houston, TX. The presentation, by the trial Principal Investigator, Richard L. Harvey, MD, Rehabilitation Institute of Chicago, summarised the main safety and efficacy results of the NICHE trial, previously announced in March 2016. The safety results were good with no serious adverse events (SAE) related to the use of Nexstim NBT® system. The primary outcome measure of significant clinical improvement of upper extremity motor function, defined as an increase of at least 5 points on the Upper Extremity Fugl-Meyer scale (UEFM) between baseline and six months after end of trial therapy, was reached by 67% of subjects in the active Nexstim NBT® trial arm and 65% in the sham NBT® trial arm. In the full trial population, the average improvement on UEFM was 8.2 points. The secondary outcome measures including Action Research Arm Test; Wolf-Motor Function Test; Stroke Impact Scale; National Institute of Health Stroke Scale; Chedoke McMaster Stroke Assessment for Hand and Arm and the quality of life instrument ED-5Q, demonstrated a statistically significant improvement from baseline to 6 months after end of trial therapy in both trial arms (p<0.001 for each trial arm). As previously announced, the difference between trial arms was not statistically significant. A journal article describing the full trial results is in preparation. Commenting on the results, Jarmo Laine, Vice President Medical Affairs stated: "The efficacy results obtained in NICHE show functional improvement in both trial arms. As previously announced by Nexstim, we believe the sham trial arm was not a true sham. Nexstim has now agreed a new 60 patient supplementary clinical trial with the FDA. We remain confident, given the results from the active arm of the NICHE study, that we can provide an important improvement in treatment for post-acute stroke patients as they undergo rehabilitation from this debilitating disease." In line with the FDA recommendation the data from the ongoing trial will be combined with data from the NICHE study in order to supplement the de novo 510(k) submission. Nexstim estimates it will receive efficacy results from the on-going 60 patient trial in Q1 2018. The combined results are then expected to be used to support a de novo 510(k) response in the summer or the fall of 2018. Clearance of this de novo 510(k) would allow Nexstim to start marketing and selling its NBT® system for stroke rehabilitation in the USA. Further information is available on the website www.nexstim.com or by telephone: About Nexstim Plc Nexstim is a medical technology company which has pioneered its technology in brain diagnostics with the Navigated Brain Stimulation (NBS) system. It is the first and only FDA cleared and CE marked system based on navigated Transcranial Magnetic Stimulation (nTMS) for pre-surgical mapping of the speech and motor cortices of the brain. Based on the same technology platform, the Company has developed a system called Navigated Brain Therapy (NBT®) which is CE marked for the treatment of stroke, chronic neuropathic pain, and depression. Nexstim shares are listed on the Nasdaq First North Finland and Nasdaq First North Sweden. For more information please visit www.nexstim.com
News Article | February 28, 2017
CAMBRIDGE, Mass.--(BUSINESS WIRE)--Alnylam Pharmaceuticals, Inc. (Nasdaq:ALNY), the leading RNAi therapeutics company, today marked the 10th annual Rare Disease Day by underscoring its commitment to enabling diagnosis for people and caregivers impacted by specific rare diseases, such as hereditary ATTR amyloidosis (hATTR amyloidosis). As part of this effort, Alnylam sponsors free third-party genetic counseling and testing through Alnylam Act™ (previously known as Alnylam Assist), a program created to empower patients with the knowledge and tools to make informed decisions about their health and facilitate an early, accurate diagnosis, potentially leading to improved care. The services, currently available in the United States, are provided by independent third parties. hATTR amyloidosis is an inherited, rapidly progressive life-threatening disease impacting 50,0001 people worldwide. It is caused by a mutation in the transthyretin (TTR) gene that results in misfolded TTR proteins accumulating as amyloid fibrils in multiple tissues including the nerves, heart and gastrointestinal tract. The degree and severity of symptoms vary from person to person but can lead to morbidity, disability and mortality within two to 15 years of symptom onset.1,2 “hATTR amyloidosis is significantly under-diagnosed and often misdiagnosed because of its constellation of symptoms that may overlap with other diseases, leading many patients to experience inappropriate medical intervention, such as unnecessary medicine and surgery,” said Sami L. Khella, M.D., Chief, Department of Neurology, Penn Presbyterian Medical Center and Professor of Clinical Neurology, University of Pennsylvania School of Medicine. “For my patients who have symptoms consistent with hATTR amyloidosis, Alnylam Act helps to make an accurate diagnosis.” “As of January 2017 approximately one thousand people have been tested via Alnylam Act, and nearly 16 percent of these tests were positive for a pathogenic mutation in the TTR gene, demonstrating the need to increase awareness and improve diagnosis rates,” said Pritesh Gandhi, Vice President, Medical Affairs at Alnylam. “Alnylam Act is a reflection of our commitment to the hATTR amyloidosis community, and we are proud to make these complimentary third-party services available to the people at risk for, or impacted by, this progressive rare disease.” In addition to genetic testing that can be ordered by a healthcare professional, Alnylam Act allows patients and their families to connect with genetic counselors who provide education and support, serving as an advocate to help guide them through the diagnostic journey. “It can be beneficial to meet with a genetic counselor prior to undergoing testing to understand the benefits and risks involved, including life and health insurance implications and how to work through a diagnosis,” said Shawna Feely, MS, CGC, Genetic Counselor, University of Iowa. “It is our goal to help enable those at risk for hATTR amyloidosis to obtain the answers and support they need to make more informed decisions about their health and the health of their family.” For physicians interested in ordering free hATTR amyloidosis genetic testing for their patients, or for people interested in scheduling a genetic counseling session to discuss the benefits, risks and limitations of genetic testing, visit Alnylam Act. About hATTR Amyloidosis Hereditary ATTR amyloidosis (hATTR amyloidosis) is an inherited, rapidly progressive, life-threatening disease. hATTR amyloidosis is a multisystemic disease with a heterogeneous clinical presentation that includes sensory and motor, autonomic (e.g., diarrhea, erectile dysfunction, hypotension) and cardiac symptoms. hATTR amyloidosis can lead to significant morbidity, disability and mortality within two to 15 years. The disease continuum of hATTR amyloidosis includes patients who present with predominantly polyneuropathy symptoms, historically known as familial amyloidotic polyneuropathy (FAP), as well as patients who present with predominantly cardiomyopathy symptoms, historically known as familial amyloidotic cardiomyopathy (FAC). However, many patients suffer from both polyneuropathy and cardiomyopathy symptoms. hATTR amyloidosis represents a major unmet medical need, affecting approximately 50,000 people worldwide. The only approved treatment options for early stage disease are liver transplantation and tafamidis (approved in Europe, certain countries in Latin America and Japan, where it is approved for all stages of the disease). There is a significant need for novel therapeutics to treat patients with ATTR amyloidosis. About RNAi RNAi (RNA interference) is a revolution in biology, representing a breakthrough in understanding how genes are turned on and off in cells, and a completely new approach to drug discovery and development. Its discovery has been heralded as "a major scientific breakthrough that happens once every decade or so," and represents one of the most promising and rapidly advancing frontiers in biology and drug discovery today which was awarded the 2006 Nobel Prize for Physiology or Medicine. RNAi is a natural process of gene silencing that occurs in organisms ranging from plants to mammals. By harnessing the natural biological process of RNAi occurring in our cells, the creation of a major new class of medicines, known as RNAi therapeutics, is on the horizon. Small interfering RNA (siRNA), the molecules that mediate RNAi and comprise Alnylam's RNAi therapeutic platform, target the cause of diseases by potently silencing specific mRNAs, thereby preventing disease-causing proteins from being made. RNAi therapeutics have the potential to treat disease and help patients in a fundamentally new way. About Alnylam Pharmaceuticals Alnylam is a biopharmaceutical company developing novel therapeutics based on RNA interference, or RNAi. The company is leading the translation of RNAi as a new class of innovative medicines. Alnylam's pipeline of investigational RNAi therapeutics is focused in 3 Strategic Therapeutic Areas (STArs): Genetic Medicines, with a broad pipeline of RNAi therapeutics for the treatment of rare diseases; Cardio-Metabolic Disease, with a pipeline of RNAi therapeutics toward genetically validated, liver-expressed disease targets for unmet needs in cardiovascular and metabolic diseases; and Hepatic Infectious Disease, with a pipeline of RNAi therapeutics that address the major global health challenges of hepatic infectious diseases. In early 2015, Alnylam launched its "Alnylam 2020" guidance for the advancement and commercialization of RNAi therapeutics as a whole new class of innovative medicines. Specifically, by the end of 2020, Alnylam expects to achieve a company profile with 3 marketed products, 10 RNAi therapeutic clinical programs - including 4 in late stages of development - across its 3 STArs. The company's demonstrated commitment to RNAi therapeutics has enabled it to form major alliances with leading companies including Ionis, Novartis, Roche, Takeda, Merck, Monsanto, The Medicines Company, and Sanofi Genzyme. In addition, Alnylam holds an equity position in Regulus Therapeutics Inc., a company focused on discovery, development, and commercialization of microRNA therapeutics. Alnylam scientists and collaborators have published their research on RNAi therapeutics in over 200 peer-reviewed papers, including many in the world's top scientific journals such as Nature, Nature Medicine, Nature Biotechnology, Cell, New England Journal of Medicine, and The Lancet. Founded in 2002, Alnylam maintains headquarters in Cambridge, Massachusetts. For more information about Alnylam's pipeline of investigational RNAi therapeutics, please visit www.alnylam.com. Alnylam Forward Looking Statements Various statements in this release concerning Alnylam's future expectations, plans and prospects, including without limitation, Alnylam's views with respect to the potential for RNAi therapeutics, its expectations regarding its STAr pipeline growth strategy, and its “Alnylam 2020” guidance for the advancement and commercialization of RNAi therapeutics, constitute forward-looking statements for the purposes of the safe harbor provisions under The Private Securities Litigation Reform Act of 1995. Actual results and future plans may differ materially from those indicated by these forward-looking statements as a result of various important risks, uncertainties and other factors, including, without limitation, Alnylam's ability to discover and develop novel drug candidates and delivery approaches, successfully demonstrate the efficacy and safety of its product candidates, the pre-clinical and clinical results for its product candidates, which may not be replicated or continue to occur in other subjects or in additional studies or otherwise support further development of product candidates for a specified indication or at all, actions or advice of regulatory agencies, which may affect the design, initiation, timing, continuation and/or progress of clinical trials or result in the need for additional pre-clinical and/or clinical testing, delays, interruptions or failures in the manufacture and supply of our product candidates, obtaining, maintaining and protecting intellectual property, Alnylam's ability to enforce its intellectual property rights against third parties and defend its patent portfolio against challenges from third parties, obtaining and maintaining regulatory approval, pricing and reimbursement for products, progress in establishing a commercial and ex-United States infrastructure, competition from others using technology similar to Alnylam's and others developing products for similar uses, Alnylam's ability to manage its growth and operating expenses, obtain additional funding to support its business activities, and establish and maintain strategic business alliances and new business initiatives, Alnylam's dependence on third parties for development, manufacture and distribution of products, the outcome of litigation, the risk of government investigations, and unexpected expenditures, as well as those risks more fully discussed in the "Risk Factors" filed with Alnylam's most recent Annual Report on Form 10-K filed with the Securities and Exchange Commission (SEC) and in other filings that Alnylam makes with the SEC. In addition, any forward-looking statements represent Alnylam's views only as of today and should not be relied upon as representing its views as of any subsequent date. Alnylam explicitly disclaims any obligation, except to the extent required by law, to update any forward-looking statements.
News Article | February 28, 2017
RESEARCH TRIANGLE PARK, NC--(Marketwired - February 28, 2017) - Roughly 70% of life science firms have altered their medical communications with patients in response to newer technologies, such as the Internet and social media, according to a study by industry research firm Cutting Edge Information. The study, Medical Affairs Management: Igniting Innovative Global Strategies, revealed that due to the increasingly patient-centric market environment, 84% of surveyed teams either believe and react to patients increased digital resources that they consult before visiting their healthcare practitioners. In anticipation of the challenges associated with inaccurate information that may exist on social media and other online platforms, many life science teams work to reduce misinformation by correcting any inaccurate data they find and maintaining constant lines of communication with physicians. To help direct consumers obtain accurate and relevant medical information, life science teams may also elect to work hand-in-hand with patient advocacy groups. "The challenge that uncontrolled media presents for the life sciences industry does not stop with patient audiences," said Adam Bianchi, senior director of research at Cutting Edge Information. "For example, physicians may consult secondary resources for sensitive scientific information about companies' products or diagnostics because they are time convenient." Transparency continues to be a key trend within the drug and device industries. Regarding medical publications, 75% of surveyed executives believe that open access journals have successfully increased clinical trial data transparency. Another developing trend within the medical education space is the push toward online programs, including self-study training modules and webinars conducted in real time. Cutting Edge Information's study found that 29% of surveyed medical affairs teams agree and another 3% of teams strongly agree that online programs have improved the quality of medical education programs. Similar percentages of teams also agree that use of online programs may decrease medical education costs. However, while online solutions may represent a convenient alternative for time-constrained physicians, these programs should not completely replace in-person continuing medical education (CME). Instead, teams should consider online CME options as complements to existing, in-person programs. Medical Affairs Management: Igniting Innovative Global Strategies, available at: https://cuttingedgeinfo.com/product/medical-affairs/, explores world-class medical affairs teams' evolving role in corporate strategy, as well as department innovations, including internal and external trends affecting medical affairs. Other highlights include: For more information about Medical Affairs Management: Igniting Innovative Global Strategies please visit: https://cuttingedgeinfo.com/preview/medical-affairs-data/.
Germain M.,Medical Affairs
Transfusion | Year: 2016
BACKGROUND In most industrialized countries, men who had sex with men (MSM) are permanently deferred from blood donation. Some countries have adopted a temporary deferral after the MSM behavior, thus avoiding donations made during the window period of human immunodeficiency virus (HIV) infection. However, one concern with this approach is the possible increase in the number of HIV-positive donations obtained from unknowingly infected, abstinent MSM; such donations might inadvertently be made available for transfusion, a risk that was previously estimated through mathematical modeling. STUDY DESIGN AND METHODS Model predictions were compared to the actual donor rate of HIV in three countries that went from a permanent to a temporary deferral: Australia, the United Kingdom, and Canada. RESULTS Depending on the model, a temporary deferral should have increased the rate of HIV in the male donor pool by 73% to more than 3400%. In reality, the very low baseline rate of HIV before the change in these three countries (22 cases/year) remained unchanged 2 years after the revised policy (16 cases/year). CONCLUSION These observations strongly suggest that a temporary deferral for MSM incurs zero risk to recipients, at least in jurisdictions where HIV epidemiology is comparable to that of countries where the change happened. © 2016 AABB.
Rundell K.W.,Medical Affairs
British Journal of Sports Medicine | Year: 2012
Unfavourable effects on the respiratory and the cardiovascular systems from short-term and long-term inhalation of air pollution are well documented. Exposure to freshly generated mixed combustion emissions such as those observed in proximity to roadways with high volumes of traffic and those from ice-resurfacing equipment are of particular concern. This is because there is a greater toxicity from freshly generated whole exhaust than from its component parts. The particles released from emissions are considered to cause oxidative damage and inflammation in the airways and the vascular system, and may be related to decreased exercise performance. However, few studies have examined this aspect. Several papers describe deleterious effects on health from chronic and acute air pollution exposure. However, there has been no research into the effects of long-term exposure to air pollution on athletic performance and a paucity of studies that describe the effects of acute exposure on exercise performance. The current knowledge of exercising in the high-pollution environment and the consequences that it may have on athlete performance are reviewed.