Novartis crizanlizumab wins speedy FDA review, putting it closer to blockbuster sickle cell launch

fiercepharma | July 16, 2019

Novartis  plan to launch over 10 potential blockbuster drugs or new indications by 2021 is quickly unfolding. The FDA has accepted a biologics application for the pharma giant's sickle cell disease candidate crizanlizumab (SEG101) and put the drug under priority review, which slashes four months off its evaluation time to just six months in total, the company said Tuesday. If approved, it could sit alongside multiple sclerosis drug Mayzent, spinal muscular atrophy gene therapy Zolgensma and age-related macular degeneration candidate brolucizumab—which also under priority review—among the Swiss drugmaker's most important launches these days. But several competitors are not far behind. Crizanlizumab, a drug Novartis got via its acquisition of Selexys Pharmaceuticals in 2016, is designed to prevent vaso-occlusive crises (VOCs), the painful events resulting from blood vessel blockage in sickle cell disease patients. The drug reduces clusters of cells that block blood flow by targeting the P-selectin protein. In a phase 2 study, crizanlizumab, given once monthly, cut the median rate of VOCs that required healthcare visits by 45.3% in a year versus placebo. What’s more, more than double the number of crizanlizumab patients didn’t experience any VOCs at all. The reductions were seen in patients with or without the use of the old standard treatment hydroxyurea, according to Novartis.

Spotlight

From our understanding today, there are many environmental chemicals that carry the potential to affect the endocrine system. Known as endocrine disrupting chemicals (EDCs), these compounds alter drug and xenobiotic metabolic processes and can interfere with the production, release, metabolism, and elimination of natural hormones.


Other News
PHARMA TECH

Zynex Submits FDA Application for its Next Generation Fluid and Blood Volume Monitor

Zynex | January 04, 2022

Zynex, Inc. an innovative medical technology company specializing in the manufacture and sale of non-invasive medical devices for pain management, rehabilitation, and patient monitoring, announced that it has submitted a 510(k) application to the U.S. Food and Drug Administration for the CM-1600, its next generation fluid monitoring system. "I am thrilled to announce that we have submitted a 510(k) premarket notification to the FDA for our CM-1600. We have worked diligently at adding key enhancements to our FDA-cleared CM-1500 Fluid Monitoring System, including wireless connectivity to the non-invasive wrist wearable. This 510(k) submission is an important step in the evolution of our fluid monitoring system, which we believe will become a vital tool to ensure optimal fluid management and quality care for patients at risk for hemorrhagic events." said Thomas Sandgaard, CEO "We are pleased to bring a meaningful treatment improvement to market through the submission of the CM-1600 to the FDA for 510(k) clearance," said Donald Gregg, Vice President, Zynex Monitoring Solutions. "The improved fluid and blood volume monitor will provide more accurate patient observance before, during, and after surgical procedures. We look forward to working closely with the FDA throughout the submission and clearance process to bring this next generation, first of its kind technology to the market." The Zynex Fluid Monitoring System is a 100% non-invasive solution for monitoring fluid changes throughout patient care environments. Patient fluid status is determined using an algorithm that combines the trends of several physiological parameters to generate a single Relative IndexTM (RI) value, allowing for fast interpretation of changes in fluid volume. About Zynex, Inc. Zynex, founded in 1996, markets and sells its own design of electrotherapy medical devices used for pain management and rehabilitation as well as developing noninvasive patient fluid, pulse oximetry and sepsis monitoring systems.

Read More

BUSINESS INSIGHTS

GNS Healthcare and Arvinas Enter into Neuroscience Drug Discovery Collaboration

GNS Healthcare | May 10, 2022

GNS Healthcare, the leader in the use of "Virtual Patients", Causal AI and simulation technology for biopharmaceutical companies, today announced a collaboration with Arvinas, Inc. (Nasdaq: ARVN), a clinical-stage biotechnology company creating a new class of drugs based on targeted protein degradation, to generate insights to help accelerate drug development for neurodegenerative diseases. Every year more than six million patients in the United States alone are diagnosed with Alzheimer's, Parkinson's, and Huntington's diseases. These diseases are chronic, debilitating and there is substantial heterogeneity in the presentation of the symptoms and the rates of their progression among patients. The GNS Gemini Virtual Patient models help discover and unravel the complex biological circuitry of the diseases and predict patient response, which can help biopharmaceutical companies accelerate the discovery and development of new therapeutic options for patients. Under the terms of the agreement, Arvinas will leverage GNS' Gemini Virtual Patient models to better understand the biology of the underlying disease, how it progresses, and how potential drugs might perform at the individual patient level. This will help Arvinas researchers discover and prioritize novel drug targets and drug candidates, simulate clinical trials to better understand drivers of diseases for various clinical outcomes, better select patients for clinical trials, and help determine which treatments are most effective for given patient types. "We believe this collaboration will provide novel insights and will help us understand the underlying mechanisms of complex neurodegenerative diseases to inform our discovery and development efforts with the aim of improving patient outcomes." Angela Cacace, Ph.D., Senior Vice President of Neuroscience & Platform Biology at Arvinas "We are committed to supporting the critical work Arvinas is doing to discover and develop breakthrough therapies that help patients who suffer from these conditions," said Joseph F. Donahue, Chief Business Officer at GNS Healthcare. "These diseases impact so many families and we are proud to be able to help contribute to potential treatments that lead to better outcomes for them." About Arvinas Arvinas is a clinical-stage biotechnology company dedicated to improving the lives of patients suffering from debilitating and life-threatening diseases through the discovery, development, and commercialization of therapies that degrade disease-causing proteins. Arvinas uses its proprietary PROTAC® Discovery Engine platform to engineer proteolysis targeting chimeras, or PROTAC® targeted protein degraders, that are designed to harness the body's own natural protein disposal system to selectively and efficiently degrade and remove disease-causing proteins. In addition to its robust preclinical pipeline of PROTAC® protein degraders against validated and "undruggable" targets, the company has three clinical-stage programs: bavdegalutamide and ARV-766 for the treatment of men with metastatic castration-resistant prostate cancer; and ARV-471 for the treatment of patients with locally advanced or metastatic ER+/HER2- breast cancer. About GNS Healthcare GNS Healthcare is the leader in the application of Causal AI and simulation technology to help pharmaceutical and biotech companies discover and develop new medicines faster. GNS' patented AI uncovers new insights from multi-omic and real-world data leading to the discovery and prioritization of novel biological targets, more efficient clinical trials, and patients who are likely to respond to therapies. The Gemini Virtual Patient models across oncology, auto-immune diseases, and neurology allow researchers and data scientists to simulate clinical trials, disease progression and drug response at the individual patient level in diverse patient cohorts. GNS' partners include seven out of the top ten pharmaceutical companies, leading research centers, medical societies, and patient advocacy groups globally, and our advisory board consists of a renowned group of scientific and medical experts.

Read More

BUSINESS INSIGHTS

Bruker Acquires Optimal Companies for Pharma PAT Software and Biopharma Manufacturing Automation Expertise and Technologies

Bruker Corporation | April 06, 2022

Bruker Corporation announced the acquisition of Optimal Industrial Automation and Technologies, a leader in pharma and biopharma process analytical technology pharma manufacturing automation and Quality Assurance (QA) software and systems integration, based in the United Kingdom. The Optimal biopharma tools acquisition strengthens Bruker as a key software and solutions provider for small molecule, biologics and new drug modalities pharma companies. Financial details of the acquisition were not disclosed. Optimal’s 2022 revenue is expected to be approximately $10 million, and Optimal’s revenue growth rate and operating margins are projected to be accretive to Bruker in 2023 and beyond. The Optimal biopharma tools and automation capabilities complement Bruker’s innovative and differentiated high-value NMR, mass spectrometry, SPR, molecular spectroscopy and X-ray scientific instruments, and life-science solutions for biopharma drug discovery and development, as well as for PAT analytical and QA solutions. Together these biopharma tools enable Bruker’s global biopharma customers to bring efficacious and safe medicines to market more quickly. Optimal has a focus on the automation and optimization of batch and continuous processes in the biopharma industry – from R&D to manufacturing. As an integrated PAT solutions provider, Optimal offers development, testing, deployment and support of quality-centric pharmaceutical production systems. Optimal has more than 30 years of experience in successfully delivering regulatory-compliant biopharma solutions, ranging from the development of automated control systems for process control to the market-leading, vendor-agnostic PAT knowledge management software synTQ®. The synTQ PAT software can interface between multiple analytical instruments (spectral and univariate), multi-variate analysis (MVA) packages, manufacturing control systems and a range of adjacent software solutions to ensure quality in real-time. As a 2021 Pharma Innovation Award winner, synTQ is a proven enabler of quality-by-design (QbD) via PAT. For users of synTQ, this can significantly increase productivity and quality, while reducing waste, time-to-manufacture and time-to-market, with proven Optimal use cases tripling biopharma productivity. While remaining a vendor-agnostic biopharma solutions provider, Optimal can integrate bioanalytical technologies such as nuclear magnetic resonance (NMR), mass spectrometry (MS) and FTIR/NIR/Raman systems into innovative drug discovery, development, PAT and quality control workflows. Combining next-generation, high-performance technologies with Optimal solutions eases the transition from development to biopharma manufacturing, while remaining in regulatory compliance. “The acquisition of Optimal is a very exciting step for Bruker as it supports more comprehensive solutions for the biopharmaceutical industry, now adding the capabilities to integrate cutting-edge systems into biopharma manufacturing processes and workflows. We welcome the Optimal team with their strong PAT and automation expertise and, together, are eager to support biotech and pharmaceutical companies in increasing their manufacturing efficiency and flexibility – a global need highlighted by the response to the pandemic crisis.” Dr. Falko Busse, President of the Bruker BioSpin Group Mr. Martin Gadsby, the CEO and Co-Founder of Optimal Industrial Automation and Technologies, added: “We are delighted to become part of the Bruker family. Optimal and Bruker together are a perfect fit – we share the common ethos of innovation with integrity, together with our industry recognition as a high-quality solutions provider delivering tangible benefits to our customers. The whole Optimal team is excited by this new chapter in our history and looks forward to delivering benefits to even more biopharma customers and ultimately to patients around the world.” About Optimal Industrial Technologies and Automation A well-established system integrator with more than 30 years of experience, at Optimal we specialize in automating a client’s R&D or manufacturing system to ensure that their processes remain at the highest level of efficiency. The demands being placed on manufacturers in relation to production costs, product quality and business sustainability are ever increasing, and our primary aim within Optimal is to assist you in being the most competitive supplier of your product to the highest level of quality in the worldwide marketplace, and to ensure that you continue to maintain your lead in the years ahead. We have a rare and wide-ranging skill set covering a multitude of automation technologies, manufacturing processes and industry types, with very specific knowledge in highly regulated environments. In addition, we manufacture the world leading PAT Data Management software package - synTQ® and the widely used integrated Print and Inspect system - synTI®. The combination of our PAT implementation knowledge and synTQ® product together with our automation experience positions us with unique skills that can be leveraged to great advantage by our customers. About Bruker Corporation Bruker is enabling scientists to make breakthrough discoveries and develop new applications that improve the quality of human life. Bruker’s high-performance scientific instruments and high-value analytical and diagnostic solutions enable scientists to explore life and materials at molecular, cellular and microscopic levels. In close cooperation with our customers, Bruker is enabling innovation, improved productivity and customer success in life science molecular research, in applied and pharma applications, in microscopy and nanoanalysis, and in industrial applications, as well as in cell biology, preclinical imaging, clinical phenomics and proteomics research and clinical microbiology.

Read More

BUSINESS INSIGHTS

Pneuma Systems collaborates with West Pharmaceutical Services, Inc. to improve the patient experience

Pneuma Systems Corporation | April 08, 2022

Medical technology company, Pneuma Systems Corporation, has entered a multi-faceted business collaboration with West Pharmaceutical Services, Inc. with the goal of improving the patient's experience with drug therapy. West is a leading global manufacturer in the design and production of technologically advanced, high-quality, integrated containment and delivery systems for injectable medicines. Pneuma Systems Corporation has developed a family of fluid flow technologies that are aimed at scientific and medical applications. The global intravenous pump market segment is expected to reach USD 20.5 billion over the next five years. Pneuma will provide a novel closed-loop fluid flow control platform that West will use to develop drug delivery solutions for pharmaceutical companies and clinical end users. West will provide technical and financial support to Pneuma and access to the emerging West Digital Platform, designed to improve transmission of data and other information. "West's almost century-long commitment to the development of drug delivery systems dovetails with our cutting-edge flow control technology. West's latest foray into a broad digital architecture is very important to us. When you improve both fluid flow and information flow, the result is an improvement in workflow, which results in higher levels of patient safety and lower costs," said Pneuma CEO Jeffrey Carlisle. "We are aiming at disruptive improvements in workflow." "We are excited about our collaboration with Pneuma and the potential of their technologies to address a variety of flow control challenges to drive improvements in healthcare. At West our mission is to improve patient lives not only through our products and services but also by investing in small innovative companies and offering our deep technical and commercial expertise to help them bring their transformation technologies to the market," Scott Young, VP Venture & External Collaboration at West Pneuma Systems, together with its strategic partners, plans to install PneumaDrive™ technology-based infusion devices in infusion centers, home care, and acute care settings around the world. The low cost PneumaDrive™ technology can replace existing infusion pumps and automate "gravity drip" infusions, providing patients with a safer and easier experience. About Pneuma Pneuma Systems is a New Hampshire based company founded by Jeffrey Carlisle, also Founder of the recently acquired Ivenix. Pneuma is dedicated to improving fluid flow, information flow, and workflow for the IV therapy process. About West West Pharmaceutical Services, Inc. is a leading provider of innovative, high-quality injectable solutions and services. As a trusted partner to established and emerging drug developers, West helps ensure the safe, effective containment and delivery of life-saving and life-enhancing medicines for patients. With approximately 10,000 team members across 50 sites worldwide, West helps support our customers by delivering over 45 billion components and devices each year.

Read More

Spotlight

From our understanding today, there are many environmental chemicals that carry the potential to affect the endocrine system. Known as endocrine disrupting chemicals (EDCs), these compounds alter drug and xenobiotic metabolic processes and can interfere with the production, release, metabolism, and elimination of natural hormones.

Resources