Prostate cancer mutation found to triple risk of death

pharmatimes | May 07, 2019

The largest ever study of advanced prostate cancer genomics has revealed that men with mutations in the retinoblastoma gene in their tumours were more than three times as likely to die and nearly seven times as likely to relapse on standard treatments as those without the gene. Scientists identified the gene mutation in the tumours of men, which could help to pick out patients for more intensive treatment in the future. The gene in question – the retinoblastoma gene – is known as RB1 because mutations in it cause a rare children’s eye cancer of the same name and is known to play a central role in stopping healthy cells from dividing uncontrollably. Professor Johann de Bono, Regius Professor of cancer research at The Institute of Cancer Research, London, and consultant medical oncologist at The Royal Marsden NHS Foundation trust, said that the study “really got under the bonnet of prostate cancer to understand the ‘engine’ driving tumour growth and explore how a wide range of genes affect the disease and its response to treatment. "We identified one particular genetic mutation that seems to indicate that tumours are going to be very aggressive, and that the affected men need the most intensive treatment we have available. “Our research could also open up various new approaches to prostate cancer treatment, and offers the intriguing suggestion that some patients could benefit from immunotherapy alongside an existing breast cancer drug. That’s a great example of how genetic research can find the common links between cancers, and ensure research into one cancer type can also benefit patients with other tumours.”

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On October 12, 2017, through Executive Order 13813, you directed the Administration, to the extent consistent with the law, to facilitate the development and operation of a health care system that provides high-quality care at affordable prices for the American people by promoting choice and competition. We are pleased to provide you with this report, prepared by the Department of Health and Human Services (HHS) in collaboration with the Departments of the Treasury and Labor, the Federal Trade Commission, and several offices within the White House


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BUSINESS INSIGHTS

Qualigen Therapeutics Acquires Majority Stake in Infectious Disease Diagnostics Technology Company NanoSynex

Qualigen Therapeutics, Inc. | June 03, 2022

Qualigen Therapeutics, Inc. a diversified life sciences company focused on developing treatments for adult and pediatric cancers with potential for Orphan Drug Designation, while also commercializing diagnostics, today announces it has completed the acquisition of a majority stake in NanoSynex Ltd., an Israeli-based developer of next generation diagnostics technology. NanoSynex’s award winning technology is an Antimicrobial Susceptibility Testing (AST) platform that aims to provide clinical laboratories worldwide with a rapid, accurate and personalized test for bacterial infections, especially difficult-to-treat drug resistant strains, with the goal of quickly matching the correct antibiotics at the correct concentration to treat a patient’s specific infection. Antibiotic misuse and overuse have given significant rise to more frequent and more deadly antibiotic resistant bacteria, such as Methicillin-resistant Staphylococcus aureus (MRSA). Multi-drug resistant pathogens such as these have been declared a substantial threat to U.S. public health and national security by the Institute of Medicine and a federal Interagency Task Force on Antimicrobial Resistance. According to some estimates, the global infectious disease diagnostics market is expected to reach ~$39.8 Billion by 2026 from ~$28.1 Billion in 2021. “Living through two and a half years of COVID-19 has taught us the critical role diagnostics plays in combating infectious disease, and the need to be prepared for the next wave. NanoSynex’s technology shows great promise in rapidly and accurately matching antibiotics to the target bacteria, and in doing so, quickly equipping healthcare providers with the data needed to treat their patients with the right antibiotic at the right dose at the right time. We strongly believe in the potential of this platform, and our ability to leverage our long-standing diagnostics development, regulatory and commercial expertise to help bring the NanoSynex diagnostics technology to market sooner.” Michael Poirier, Qualigen's Chairman and CEO “We are excited about beginning a new journey with Qualigen and joining forces in a common mission to develop innovative diagnostics. This partnership takes the NanoSynex AST platform to the next level," added NanoSynex Co-Founder and Chief Executive Officer, Diane Abensur Bessin. NanoSynex is led by a dynamic team of healthcare subject matter experts, microbiologists, and engineers, including its co-founders – Diane Abensur Bessin and Michelle Heymann, who were selected for Forbes’ 2020 Tech 30 Under 30 list. Qualigen’s diagnostics team, led by Shishir Sinha, Senior VP and Chief Operating Officer, will work closely alongside the Israeli-based NanoSynex team on technology development, regulatory path, and commercial preparedness. Qualigen also envisions potential synergies with its proprietary FastPack® diagnostics platform to further strengthen the Company’s diagnostics business which has seen a post-COVID-19 resurgence. Qualigen’s purchase of the controlling interest in NanoSynex was primarily accomplished via a stock-for-stock acquisition with a controlling shareholder of NanoSynex. Qualigen also provided an investment in NanoSynex at closing and will provide future milestone-based funding leading to the commercialization of this technology. NanoSynex’s Board will initially be comprised of two current NanoSynex directors – NanoSynex co-founders - and two Qualigen appointees – Mr. Poirier and Mr. Sinha. About Qualigen Therapeutics, Inc. Qualigen Therapeutics, Inc. is a diversified life sciences company focused on developing treatments for adult and pediatric cancer, as well as maintaining and expanding its core FDA-cleared FastPack® System, which has been used successfully in diagnostics for over 20 years. Our investigational QN-302 compound is a small molecule selective transcription inhibitor with strong binding affinity to G4s prevalent in cancer cells; such binding could, by stabilizing the G4s against “unwinding,” help inhibit cancer cell proliferation. Our investigational QN-247 compound inhibits nucleolin, a key multi-functional regulatory protein that is overexpressed in cancer cells; QN-247 may thereby be able to inhibit the cells’ proliferation. QN-247 has shown promise in preclinical studies for the treatment of acute myeloid leukemia (AML). The investigational compounds within Qualigen’s RAS-F family of RAS oncogene protein-protein interaction inhibitor small molecules are believed to inhibit or block the binding of mutated RAS genes’ proteins to their effector proteins, thereby leaving the proteins from the mutated RAS unable to cause further harm. In theory, such mechanism of action may be effective in the treatment of about one quarter of all cancers, including certain forms of pancreatic, colorectal, and lung cancers. In addition to its oncology drug pipeline, Qualigen has an established diagnostics business which manufactures and distributes proprietary and highly accurate rapid blood testing systems to physician offices and small hospitals for the management of prostate cancer and other diseases and health conditions. About NanoSynex NanoSynex is a MedTech company that aims at providing new solutions to improve testing quality, patient outcomes, and reduce healthcare costs by speeding up diagnostic processes. NanoSynex is focused on the development and commercialization of a rapid innovative Antimicrobial Susceptibility Test (AST). The technology is based on a purely phenotypic approach and uses a microfluidic disposable test card platform and method that optimizes bacterial growth. This disruptive development was born from exciting research discoveries at the lab of Professor Shulamit Levenberg, former Dean of the Technion Institute of Technology – Biomedical Engineering Faculty.

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BUSINESS INSIGHTS

GoodRx Closes Acquisition of vitaCare, A Tech-Enabled Pharmacy Services Platform

GoodRx | April 18, 2022

GoodRx, a leading consumer-focused digital healthcare platform announced the completion of its previously announced acquisition of vitaCare for $150 million in cash, with an additional $7 million of consideration contingent payment upon vitaCare’s financial performance through 2023. VitaCare is a pharmacy services platform that expands GoodRx’s offering to pharmaceutical manufacturers while helping to improve patient access and adherence to affordable brand drugs. The acquisition will enable GoodRx to help more patients receive their prescriptions in an efficient, affordable, and transparent manner, and stay on their prescribed therapies as long as appropriate. In addition, the acquisition is expected to further strengthen our rapidly growing Pharma Manufacturer Solutions offerings which span medication awareness, access, and adherence with unique capabilities. “The vitaCare acquisition gives us unique capabilities to facilitate the brand prescription process from start to finish, expanding our capabilities beyond our digital platform and into the patient’s pharmacy journey. We look forward to growing our reach across consumers and providers, along with our established relationships with manufacturers, to help more patients access the brand drugs they need." Doug Hirsch, co-CEO and co-founder of GoodRx GoodRx plans to provide an update on the impact of vitaCare to its 2022 financial guidance during its first quarter 2022 earnings conference call scheduled for Monday, May 9, 2022. About GoodRx GoodRx is a leading consumer-focused digital healthcare platform. Our technology delivers strong savings, trusted information and access to care to make healthcare affordable and convenient for all Americans. Since 2011, we have helped consumers save over $35 billion and are one of the most downloaded medical apps over the past decade.

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PHARMA TECH

ADDING MULTIMEDIA Entos Pharmaceuticals Expands R&D Operations into the US

Entos Pharmaceuticals | June 08, 2022

Entos Pharmaceuticals, a clinical-stage biotechnology company developing genetic medicines with its Fusogenix proteolipid vehicle (PLV) nucleic acid delivery platform, announced today the opening of its new 9,600 square foot research and development facility in the Torrey Pines community of San Diego, California. This collaborative space will be shared with Entos’ sister companies, Oisín Biotechnologies, OncoSenX, and Aegis Life, allowing the companies to expand their team and infrastructure and support continued growth. Entos’ new office and lab space is located at the MUSE in Torrey Pines, a redeveloped 186,000 square foot life sciences property completed in 2021. Featuring three buildings, the campus was designed with walking paths, a locally-sourced restaurant, and conference space with indoor and outdoor seating. The US location complements Entos’ existing Canadian headquarters and R&D facilities in Edmonton, Canada, and new offices in London, UK. “We’re excited to move into our new lab space in San Diego, which is becoming one of the largest biotechnology hubs in the world for pharmaceutical research and innovation. This is a significant milestone for Entos as we continue developing genetic medicines for some of our most challenging diseases.” John D. Lewis, Ph.D., founder, and CEO of Entos Pharmaceuticals Entos’ proprietary Fusogenix PLV drug delivery system is formulated with FAST proteins to enable the delivery of nucleic acid cargo, such as DNA and RNA, directly into target cells. This technology is applicable to a wide range of genetic medicines. Unlike conventional lipid-based and viral-based delivery systems, Fusogenix PLVs have demonstrated, in multiple studies, to be well tolerated at high systemic doses, redosable, and able to target multiple tissues in the body. This announcement comes on the heels of two significant collaborations that began in late 2021, granting exclusive rights to Entos’ Fusogenix PLV nucleic acid delivery technology to research, develop and commercialize therapeutic genetic medicines. The first agreement was with BioMarin to target certain rare diseases. The second was a license agreement with Eli Lilly for multiple programs for central and peripheral nervous system diseases and came with an initial payment of $50M and $400M in milestones per program. About Entos Pharmaceuticals A new reality in genetic medicine lies ahead, one that will be ushered in with the advent of safe, effective, and redosable nucleic acid delivery technologies. At Entos, we develop next generation genetic medicines using our proprietary Fusogenix proteolipid vehicle (PLV) drug delivery system. Fusogenix PLVs are formulated with FAST proteins to enable the delivery of nucleic acid into target cells through direct fusion.

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BUSINESS INSIGHTS

Clinical research and drug development accelerated via analytics

SAS | June 22, 2022

Bringing life-changing therapies to market requires pharmaceutical companies to efficiently design and run clinical trials, gather and analyze massive amounts of data, and manage a complicated regulatory review process. SAS, a leader in clinical research analytics, delivers powerful technologies to support the life sciences industry – including machine learning, AI and analytics for IoT – through the SAS® Life Science Analytics Framework on Azure. The advanced analytics platform – a single, open, cloud-native statistical computing environment for clinical trial analysis and submission – helps pharmaceutical companies navigate the heavily regulated world of clinical research analytics and bring new therapies to the world faster. Delivering the power of analytics in the cloud From drug discovery to regulatory approval, developing a new medicine can take over a decade with a price tag in the billions. The clinical research required to achieve approval from the US Food and Drug Administration (FDA), European Medicines Agency (EMA), and other regulatory authorities involves massive amounts of data that can be difficult to manage, share and analyze, creating delays that affect patients' well-being. The cloud-based SAS Life Science Analytics Framework on Azure features an integrated and collaborative environment to manage and analyze clinical trial information as well as deliver trial results for the FDA and other global regulatory agencies' review. With the analytic solution, life sciences organizations can extract valuable insights from clinical data, mitigate risk, increase efficiency, and speed time to market for lifesaving pharmaceuticals. "The primary goal is to get drugs approved so that they can reach patients. The faster you can do that, the greater benefit for the patient and to help save lives." Bhawna Goel, CEO of Gunvatta Gunvatta USA, Inc, a contract research organization (CRO) headquartered in Washington, DC, helps clients collect, manage, analyze and visualize the terabytes of clinical and healthcare data produced by pharmaceutical and biotechnology companies, hospitals, and provider groups. With the SAS Life Science Analytics Framework, Gunvatta has modernized the way it approaches clinical trials. The CRO helps life sciences organizations reduce risk with secure data access, ensure data and results are validated, maintain an audit trail, and report their data to health authorities like the FDA. "The biggest advantage of the SAS Life Science Analytics Framework is that the analytics platform is cloud-based," said Goel. "I feel like SAS has jumped 15 to 20 steps ahead of where the market is." For more about Gunvatta's experience, see the customer story Helping pharmaceutical companies bring new therapies to the world faster using analytics for life sciences. Developing cutting-edge clinical research tools SAS life science experts will attend the DIA Global Annual Meeting this week in Chicago to share next generation approaches for driving excellence and innovation in clinical trial operations. At DIA, SAS will preview SAS Clinical Enrollment Simulation Cloud – a cloud-native, solution based in SAS® Viya® 4 – that enables life sciences and contract research organizations to simulate the outcome of the complex clinical trial enrollment process in a virtual world, resulting in faster, more strategic clinical trial enrollment plans. The new SaaS offering – available later this year on the Microsoft Azure Marketplace – uses a powerful discrete event simulation analytical engine to model the clinical trial enrollment process as it evolves over time, facilitating improved insights to guide enrollment strategy and meet contracted patient targets. "SAS Clinical Enrollment Simulation Cloud is highly differentiated from traditional methodologies for predicting trial enrollment," said Jim Box, Life Sciences Principal Data Scientist at SAS. "The technology helps life sciences organizations reduce timelines, minimize costs, and gain greater insight to inform clinical enrollment planning and rescue." Modernizing clinical trials for the future While the current clinical development research model can be an obstacle to the creation of safe, cost-effective therapies, the COVID-19 pandemic and other events set the stage for disruption in clinical trials. "The silver lining of the pandemic for the life sciences industry is the acceleration of digital transformation and modernization of clinical research," said Simon Tilley, Health and Life Sciences Product Director at SAS. "We experienced widespread adoption of innovative approaches, such as decentralized clinical trials for the development of new vaccines." "While the business benefits of modernizing clinical research include significant cost savings, efficiency improvements, accelerated time to regulatory approval and faster time to market, it is patients who stand to gain the most from clinical trial innovation." About SAS SAS is the leader in analytics. Through innovative software and services, SAS empowers and inspires customers around the world to transform data into intelligence. SAS gives you THE POWER TO KNOW®. SAS and all other SAS Institute Inc. product or service names are registered trademarks or trademarks of SAS Institute Inc. in the USA and other countries. ® indicates USA registration. Other brand and product names are trademarks of their respective companies. Copyright © 2022 SAS Institute Inc. All rights reserved.

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Spotlight

On October 12, 2017, through Executive Order 13813, you directed the Administration, to the extent consistent with the law, to facilitate the development and operation of a health care system that provides high-quality care at affordable prices for the American people by promoting choice and competition. We are pleased to provide you with this report, prepared by the Department of Health and Human Services (HHS) in collaboration with the Departments of the Treasury and Labor, the Federal Trade Commission, and several offices within the White House

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