AveXis hit as Biogen becomes first to market SMA med

AveXis was down 6% today as Biogen and partner Ionis got an early present in the form of the first-ever approval for a drug that helps children with spinal muscular atrophy (SMA). Spinraza (nusinersen), which had FDA priority review and fast-track tags, was approved (much earlier than analysts predicted) on Friday for the treatment of SMA in both pediatric and adult patients. It has however come with safety warnings, including risk of thrombocytopenia and renal toxicity.  Analysts at Leerink said they thought the drug wouldn’t see an approval until halfway through next year. They believe it could make around $2 billion by 2020, much more than it initially forecast, which it says could boost Biogen’s top line by 3% a year by the end of the decade.

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Bridges Home Healthcare is a full service home health care agency, offering both medical and non-medical home care throughout Southeastern Wisconsin. Our service area covers the counties of Dodge, Washington, Ozaukee, Milwaukee, Waukesha, Racine and Kenosha. At Bridges Home Healthcare, we strive to bridge the gap between personal health, financial resource, and health care. Our primary goal is to help create a community of caring, safety, and positive health outcomes while maintaining a holistic and cohesive continuum of care for the patient caregiver healthcare provider relationship.

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MedTech

How to Choose a Reliable Biotech Clinical Trial Management System?

Article | July 13, 2022

Introduction The medical and life-science industries are experiencing a robust transformation with the increasing prevalence of various types of diseases, including infectious diseases, chronic disorders, and acute conditions around the world. As a result, a significant rise in demand for more effective therapeutic drugs and bionics is being witnessed, leading to a swift increase in the number of clinical trials. For a successful trial, it is important for biotech companies to ensure the data submitted to regulatory bodies regarding clinical trials is accurate, reliable, and definitive from an ethical point of view. A reliable clinical trial management system plays a vital role in collecting, monitoring, and managing clinical data. The availability of high-quality clinical data also helps clinical research institutions make efficient treatment decisions and provide proper patient care. Hence, a number of biotech companies and research organizations are focusing on leveraging innovative clinical trial management solutions to handle a large amount of data, particularly in multi-center trials, and generate reliable, high-quality, and statistically sound data from clinical trials. However, selecting the most appropriate and reliable clinical trial management system is vital for the clinical trial's success. Let's see some of the steps that will assist these firms in choosing the right CTMS. Key Steps for Selecting Right Biotech Clinical Trial Management System Prioritize Study Needs Considering and prioritizing study needs is a crucial step in choosing the most reliable clinical trial management system for biotech companies. Prioritizing helps them to identify a solution that improves the study's quality and removes uncertainty for researchers when faced with difficult choices. Hence, biotech and life-science organizations should choose a clinical trial system that is simple to use, well-organized, and suitably designed to minimize the number of clicks required to complete a task. Select CTMS with Multiple Integrations Integrated clinical trial management systems provide the best value for the companies’ funds as they guarantee the smooth functioning of research protocols. In addition, integrations are necessary to fully understand the importance and advantages of clinical trial management software for ensuring smooth transitions between site management and data collection. Biotech and clinical research should look for CTMS platforms that can integrate with electronic medical record (EMR) platforms and clinical research process content (CRPC) billing grids. This will allow them to use the same billing designations and ensure compliance while minimizing the need for duplicate processes. Ensure System Compliance and Security Clinical research organizations need to adhere to a plethora of complex regulations in order to ensure compliance with one of the most challenging environments of principles, which is information security and privacy. Security and system compliance are vital aspects of choosing the right CTMS solutions for biotech firms as they assist in building trust and form a part of the system’s duties. While selecting CTMS systems, it is essential for companies engaged in clinical research to ensure that these platforms are able to configure both, group and individual permissions, along with having a data backup and recovery plan for hosted systems. This will allow companies to assess the privacy and security implications of research and anticipate complications that may arise in each phase of the project. Assess the Scalability Choosing a scalable CTMS that can accommodate various types of fluctuations and expansions enables biotech and clinical firms to quickly adapt to fast-changing trends and demand spikes while reducing maintenance costs and enhancing user agility. As scalability also means secure and expanded data storage, these businesses should instead use SaaS solutions than manually manage an ever-growing collection of hard drives. The right CTMS ensures accommodating the firm’s availability requirements without incurring the capital costs associated with expanding a physical infrastructure. The Closing Thought A well-executed and successful clinical trial involves multiple stages and processes. Several quality controls and stringent adherence to regulations are essential for the steps, along with efficient cross-departmental processes and procedures. Incorporating the right CTMS paves the way for paperless data collection, regulatory filing, and fiscal management tools for biotech researchers and administrative personnel.

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Research

Data Analytics: A Groundbreaking Technology in Biotech

Article | July 11, 2022

Biotechnology is a vast discipline of biology that employs diverse biological systems to create solutions that can significantly alter the ways in which they operate across various domains. That said, biotechnology is not a new notion. It has existed for millennia, with ancient civilizations using its earliest incarnations to cultivate crops and create alcoholic beverages. Today, the biotechnology industry has developed by leaps and bounds and has amassed a vast quantity of scientific data through study and research. Given the importance of data in the biotechnology business, it is not difficult to understand why biotech companies utilize data analytics. Modern data analytics tools have made it possible for researchers in the biotech industry to build predictive analytics models and gain knowledge about the most efficient approaches to accomplish their desired goals and objectives. Data analytics is increasingly being adopted by biotech businesses to better understand their industry and foresee any problems down the road. How is Data Analytics Revolutionizing Fields in Biotechnology? Today's business and scientific fields greatly benefit from data. Without the analysis of vast information libraries that provide new insights and enable new innovations, no industry can really advance. Being highly reliant on big data analytics, biotech is not an exception in this regard. With the tools and methods that help scientists systematize their findings and speed up their research for better and safer results, data analytics is making deeper inroads into the biotechnology industry. It is emerging as a crucial link between knowledge and information and is extensively being used for purposes other than just examining the information that is already available. The following are a few of the cutting-edge biotechnology applications of data analytics Genomics and Disease Treatment Pharmaceutical Drug Discovery Drug Recycling and Safety Agriculture and Agri-products Environmental Damage Mitigation Data Analytics Possibilities in Biotechnology With data analytics becoming an integral part of how biotech businesses operate, biotechnologists and related stakeholders need to understand its emergence and crucial role. Data analytics has opened new frontiers in the realm of biotechnology. Thanks to developments in data analytics, research and development activities that once took years may now be accomplished in a matter of months. Also, now scientists have access to biological, social, and environmental insights that can be exploited to create more effective and sustainable products. By understanding the importance of data-related tools and techniques applications, biotech companies are aiming to invest in the popularizing technology to stay updated in the fast-paced biotechnology industry.

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Medical

Immunology: A New Frontier in Medical Science

Article | August 16, 2022

Introduction Recent developments in the bioengineering of monoclonal antibodies (mAbs) have revolutionized the treatment of numerous rheumatic and immunological disorders. Currently, several immunological disorders are successfully being targeted and treated using innovative medical techniques such as immunotherapy. Leading companies are increasingly investing in research activities to expand the usage and application of immunology for the treatment of various infectious diseases, including multiple sclerosis, inflammatory bowel disorders, lupus, and psoriasis, leading companies are increasingly investing in research activities. Today, the efforts of researchers in immunology, with a long history of study and research, have borne fruit, as bioengineered mAbs are now being employed in clinical practices. Accelerating Investments: Paving the Way for Immunology The increasing prevalence of infectious diseases, cancer, and immune-mediated inflammatory disorders (IMIDs) is raising the need for more precise classification and an in-depth understanding of the pathology underlying these ailments. Numerous leaders in the biotechnology domain are thus focusing on undertaking numerous strategies, such as new facility launches and collaborations, to address the need by finding deeper inroads into immunology and its use in disease treatments. For instance, in 2022, the University of Texas MD Anderson Cancer Center announced the launch of a visionary research and innovation hub, the James P. Allison Institute, to find new roads in immunotherapy, develop new treatments, and foster groundbreaking science. These developments will result in better diagnosis through the use of selective biomarkers, and early detection of fatal diseases and their treatment, which will prevent complications from happening. Also, the identification of high-risk populations through a deeper understanding of genetic and environmental factors can assist in the prevention of disease through immunotherapy. The Way Forward Immunology has led to the development of biotechnology, making it possible to develop novel drugs and vaccines, as well as diagnostic tests, that can be used to prevent, diagnose, and treat a wide range of autoimmune, infectious, and cancerous diseases. With the rapid advancement in technology and the integration of artificial intelligence, immunology is finding its way into an array of domains and industries, encompassing several research areas including medicine, pharmaceuticals, agriculture, and space. Today, not only researchers but also leading biotech and pharmaceutical companies have recognized that conventional therapies with pharmaceutical and chemical products are being replaced by products derived from immunology. This is because they work well for health problems, are environmentally friendly, and are also emerging as a wealth-generating business in the medical field.

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5 Biotech Stocks Winning the Coronavirus Race

Article | April 13, 2020

There are quite a few companies that have found ways to grow their business during the ongoing COVID-19 pandemic. This is especially true for a number of biotechs now working on developing a potential treatment for, or vaccine against, the virus; shares of such companies have largely surged over the past couple of months. Although many of these treatments and vaccines are still have quite a way to go before they're widely available, it's still worth taking some time to look through what's going on in the COVID-19 space right now. Here are five biotech stocks that are leading the way when it comes to addressing COVID-19. Regeneron Pharmaceuticals (NASDAQ:REGN) wasn't among the initial wave of companies to announce a potential COVID-19 drug. However, investor excitement quickly sent shares surging when the company announced that its rheumatoid arthritis drug, Kevzara, could help treat COVID-19 patients.

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Spotlight

Bridges Home Healthcare

Bridges Home Healthcare is a full service home health care agency, offering both medical and non-medical home care throughout Southeastern Wisconsin. Our service area covers the counties of Dodge, Washington, Ozaukee, Milwaukee, Waukesha, Racine and Kenosha. At Bridges Home Healthcare, we strive to bridge the gap between personal health, financial resource, and health care. Our primary goal is to help create a community of caring, safety, and positive health outcomes while maintaining a holistic and cohesive continuum of care for the patient caregiver healthcare provider relationship.

Related News

Biogen’s Experimental Drug Shows Promise for Genetic Form of ALS

biospace | May 15, 2019

Amyotrophic lateral sclerosis (ALS) is a devastating disease with no approved treatments. Diagnosis is tantamount to a death sentence and researchers are desperately searching for some kind of therapy. Although there have been numerous failed treatments, Biogen believes it might be on the right track. “ALS is a disease you really want to make a difference in,” Toby Ferguson, M.D., Ph.D, Senior Medical Director for Neuromuscular Research and Early Development at Biogen told BioSpace. Earlier this month, Biogen announced interim results from a Phase I/II study of tofersen, an antisense oligonucleotide the company licensed from Ionis Pharmaceuticals that is being studied as a potential treatment of ALS patients who have a confirmed superoxide dismutase 1 (SOD1) mutation. The interim results showed both a proof-of-biology and proof-of-concept, which supported the start of a Phase III clinical trial to confirm the efficacy and safety of tofersen in that patient population, Ferguson said. Ferguson said this was exciting news as it showed the potential of tofersen to target the genetic driver of this form of ALS.

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Biogen and Ionis Pharmaceuticals Announce SPINRAZA (nusinersen) Meets Primary Endpoint at Interim Analysis of Phase 3

Biogen | November 07, 2016

Biogen (NASDAQ:BIIB) and Ionis Pharmaceuticals (NASDAQ:IONS) announced that SPINRAZATM (nusinersen), an investigational treatment for spinal muscular atrophy (SMA), met the primary endpoint at the interim analysis of CHERISH, the Phase 3 study evaluating SPINRAZA in later-onset (consistent with Type 2) SMA. The analysis found that children receiving SPINRAZA experienced a highly statistically significant improvement in motor function compared to those who did not receive treatment. SPINRAZA demonstrated a favorable safety profile in the study.

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Nature Publishes Results from Pre-Clinical Research and Phase 1b Study of Biogen’s Investigational Alzheimer’s Disease Treatment Aducanumab

Biogen | August 31, 2016

Results from pre-clinical research and PRIME, the Phase 1b study of Biogen’s (NASDAQ: BIIB) investigational treatment for early Alzheimer’s disease (AD), aducanumab, were published today in Nature. The full manuscript titled, “The Antibody Aducanumab Reduces Aβ Plaques in Alzheimer’s Disease,” can be found in the 1 September, 2016 issue of Nature

Read More

Biogen’s Experimental Drug Shows Promise for Genetic Form of ALS

biospace | May 15, 2019

Amyotrophic lateral sclerosis (ALS) is a devastating disease with no approved treatments. Diagnosis is tantamount to a death sentence and researchers are desperately searching for some kind of therapy. Although there have been numerous failed treatments, Biogen believes it might be on the right track. “ALS is a disease you really want to make a difference in,” Toby Ferguson, M.D., Ph.D, Senior Medical Director for Neuromuscular Research and Early Development at Biogen told BioSpace. Earlier this month, Biogen announced interim results from a Phase I/II study of tofersen, an antisense oligonucleotide the company licensed from Ionis Pharmaceuticals that is being studied as a potential treatment of ALS patients who have a confirmed superoxide dismutase 1 (SOD1) mutation. The interim results showed both a proof-of-biology and proof-of-concept, which supported the start of a Phase III clinical trial to confirm the efficacy and safety of tofersen in that patient population, Ferguson said. Ferguson said this was exciting news as it showed the potential of tofersen to target the genetic driver of this form of ALS.

Read More

Biogen and Ionis Pharmaceuticals Announce SPINRAZA (nusinersen) Meets Primary Endpoint at Interim Analysis of Phase 3

Biogen | November 07, 2016

Biogen (NASDAQ:BIIB) and Ionis Pharmaceuticals (NASDAQ:IONS) announced that SPINRAZATM (nusinersen), an investigational treatment for spinal muscular atrophy (SMA), met the primary endpoint at the interim analysis of CHERISH, the Phase 3 study evaluating SPINRAZA in later-onset (consistent with Type 2) SMA. The analysis found that children receiving SPINRAZA experienced a highly statistically significant improvement in motor function compared to those who did not receive treatment. SPINRAZA demonstrated a favorable safety profile in the study.

Read More

Nature Publishes Results from Pre-Clinical Research and Phase 1b Study of Biogen’s Investigational Alzheimer’s Disease Treatment Aducanumab

Biogen | August 31, 2016

Results from pre-clinical research and PRIME, the Phase 1b study of Biogen’s (NASDAQ: BIIB) investigational treatment for early Alzheimer’s disease (AD), aducanumab, were published today in Nature. The full manuscript titled, “The Antibody Aducanumab Reduces Aβ Plaques in Alzheimer’s Disease,” can be found in the 1 September, 2016 issue of Nature

Read More

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