Gilead rescinds orphan status request for COVID-19 drug

pharma news | March 26, 2020

Gilead has rescinded its request for orphan status for its potential COVID-19 antiviral remdesivir amid a furore over its pricing should it get to market. By asking for orphan status the US pharma sparked concerns that it would charge a premium price for remdesivir, which is being trialled against the disease. Remdesivir has been described by the World Health Organisation as the best hope for a therapy, at least until a vaccine is developed. Gilead decided to rescind the request for orphan status after drug pricing campaigners accused the company of gaming US drug laws to maximise any profits made from remdesivir should it get approved.

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The firm’s founding mission statement reflected our focus on health and wellness as a commitment to the alleviation of suffering, to the regeneration of spirit, and to the endless unfolding of the human heart.

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The firm’s founding mission statement reflected our focus on health and wellness as a commitment to the alleviation of suffering, to the regeneration of spirit, and to the endless unfolding of the human heart.

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Cadence Expands into Molecular Simulation with Acquisition of OpenEye Scientific, a Pioneering Leader in Computational Molecular Design

Cadence | July 26, 2022

Cadence Design Systems, Inc. announced that it has entered into a definitive agreement to acquire privately held OpenEye Scientific Software, Inc., a leading provider of computational molecular modeling and simulation software being widely and increasingly used by pharmaceutical and biotechnology companies for drug discovery. The addition of OpenEye’s technologies and experienced team with its deep scientific expertise accelerates Cadence’s Intelligent System Design™ strategy and expands its total addressable market bringing Cadence’s computational software expertise to apply proven algorithmic, simulation and solver advances to life sciences. Technological advancements, coupled with the rising demand for new drugs across a range of diseases, are driving increased demand for computational drug design. The growing utilization of computational techniques in drug discovery for pharmacology, chemical synthesis and drug screening to study the 3D structure of molecules is a major factor fostering the growth of the global biosimulation market, which has an estimated TAM of $2B and predicted to have a compound annual growth rate (CAGR) of approximately 15% over the next five years. Biosimulations are a critical tool for pharmaceutical research as they provide atomic-level insight into molecular interactions, and there’s a growing demand for high-performance simulation of larger biological systems over longer time scales. Cadence continues executing its Intelligent System Design strategy and has successfully leveraged its computational software expertise to expand into the system analysis space to address the electromagnetics, thermal and computational fluid dynamics (CFD) challenges in the high-tech electronics, aerospace and defense, and automotive sectors. Cadence is now extending that computational software core competency to molecular modeling and simulation that is targeted to life sciences. OpenEye, an industry leader in computational molecular design, has pioneered physics-based approaches and the cloud-native Orion® software platform to accelerate advances in human health. The acquisition allows pharmaceutical and biotechnology companies to benefit from more robust drug discovery solutions that combine OpenEye’s innovative molecular modeling and simulation software solutions for drug discovery with Cadence’s algorithmic and solver expertise, efficient large data management infrastructure, and leading AI/ML and cloud solutions. OpenEye’s products are used by 19 of the top 20 pharmaceutical companies globally—including Pfizer and AstraZeneca—as well as numerous biotechnology companies and academic institutions. Orion, OpenEye’s market-leading cloud-native software-as-a service (SaaS) platform, is growing rapidly as pharmaceutical and biotechnology users embrace its flexibility and scalability to perform complex molecular calculations on a large scale. “Drug discovery is an increasingly complex process that requires significant investment in research and development. The pending acquisition of OpenEye, with its scientifically tested methodologies and expertise, accelerates Cadence’s Intelligent System Design strategy with our entry into a new system domain of life sciences. Cadence’s deep computational software expertise drives further innovation in algorithms that enhance the reliability, efficiency and speed of molecular simulations. We look forward to welcoming such an accomplished team and are delighted to accelerate innovation and improve research and development productivity in the pharmaceutical and biotechnology industry.” Dr. Anirudh Devgan, president and CEO of Cadence “We founded OpenEye to stretch the boundaries of what’s possible in computational drug discovery,” said Dr. Anthony Nicholls, CEO of OpenEye Scientific. “Our 3D search technology, programming toolkits, and more recently, our cloud platform, Orion, have led the industry for more than 25 years, and this opportunity to join with Cadence to pursue even greater scale and accuracy is simply amazing. We foresee a time when pharmaceutical design, no matter the modality, is predominantly computational. The realization of that goal with our skills and Cadence’s resources and expertise is going to have a profound impact on human health.” Under the terms of the definitive agreement, Cadence will pay approximately $500 million in cash. The acquisition is expected to contribute immaterial revenue this year and about $40 million in fiscal 2023. It is expected to close in the third quarter of 2022, subject to completion of Hart-Scott-Rodino regulatory review and customary closing conditions. About Cadence Cadence is a pivotal leader in electronic systems design, building upon more than 30 years of computational software expertise. The company applies its underlying Intelligent System Design strategy to deliver software, hardware and IP that turn design concepts into reality. Cadence customers are the world’s most innovative companies, delivering extraordinary products from chips to boards to complete systems for the most dynamic market applications, including hyperscale computing, 5G communications, automotive, mobile, aerospace, consumer, industrial and healthcare. For eight years in a row, Fortune magazine has named Cadence one of the 100 Best Companies to Work For. About OpenEye Scientific OpenEye Scientific is an industry leader in computational molecular design through rapid, robust and scalable software, toolkits, technology and design services, and Orion®, the only cloud-native fully integrated software-as-a-service molecular modeling platform that runs on Amazon Web Services. Combining unlimited computation and storage with powerful tools for data sharing, visualization and analysis in an open development platform, Orion offers unprecedented capabilities for the advancement of pharmaceuticals, biologics, agrochemicals, and flavors and fragrances. Founded in 1997, OpenEye Scientific is a privately held company headquartered in Santa Fe, N.M., with offices in Boston, Mass.; Cologne, Germany; and Tokyo, Japan.

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BUSINESS INSIGHTS

AbbVie Secures Positive CHMP Opinion for Risankizumab (SKYRIZI®) for the Treatment of Adults with Moderate to Severe Crohn's Disease

AbbVie | September 19, 2022

AbbVie announced the European Medicines Agency's Committee for Medicinal Products for Human Use adopted a positive opinion recommending the approval of risankizumab for the treatment of adults with moderately to severely active Crohn's disease who have had inadequate response, lost response or were intolerant to conventional or biologic therapy. "We continue to raise the bar in researching treatments for immune-mediated gastroenterological conditions through more stringent and innovative clinical trial design, such as the first time a Phase 3 trial has reported co-primary endpoints, endoscopic response and clinical remission. With the recent CHMP recommendation for risankizumab in Crohn's disease, we're getting closer toward helping patients find sufficient disease control for this disruptive condition." Neil Gallagher, M.D., Ph.D., vice president, development, chief medical officer, AbbVie AbbVie's application for the approval of risankizumab in Crohn's disease is supported by data from three Phase 3 clinical trials — ADVANCE, MOTIVATE (induction studies) and FORTIFY (maintenance study).1,2 Across all three studies, significantly more patients treated with risankizumab achieved the co-primary endpoints of endoscopic response and clinical remission.1,2,5-10 This includes statistically significant improvements in these endpoints at week 12 compared to placebo with 600 mg intravenous infusions for the ADVANCE and MOTIVATE induction studies.1,8,9 Additionally, a significantly greater proportion of patients treated with risankizumab 360 mg subcutaneous injections achieved endoscopic response and clinical remission at 52 weeks compared to placebo (patients treated with placebo after risankizumab induction) in the FORTIFY maintenance study.2,10 Safety results of risankizumab in Crohn's disease were consistent with the known safety profile of risankizumab, with no new safety risks observed.1,2,8-13 "Living with Crohn's disease can pose many challenges and significantly impact a patient's health-related quality of life," said Marc Ferrante, M.D., Ph.D., Department of Gastroenterology and Hepatology, University Hospitals Leuven, Belgium. "Risankizumab could be an encouraging option for adult patients that continue to have moderately to severely active Crohn's disease and I look forward to the European Commission's final decision." Use of risankizumab in Crohn's disease is not approved in the European Union, and its safety and efficacy remain under evaluation. Risankizumab (SKYRIZI) is part of a collaboration between Boehringer Ingelheim and AbbVie, with AbbVie leading development and commercialization globally. About Crohn's Disease Crohn's disease is a chronic, systemic disease that manifests as inflammation within the gastrointestinal tract, causing persistent diarrhea and abdominal pain.3,4 It is a progressive disease, meaning it gets worse over time in a substantial proportion of patients or may develop complications that require urgent medical care, including surgery.3,4 Because the signs and symptoms of Crohn's disease are unpredictable, it causes a significant burden on people living with the disease—not only physically, but also emotionally and economically.14,15 About the ADVANCE and MOTIVATE Inductions Studies, and the FORTIFY Maintenance Study1,2,8-10 The three Phase 3 studies are multicenter, randomized, double-blind, placebo-controlled studies to evaluate the efficacy and safety of risankizumab 600 mg and 1200 mg as induction therapy, and risankizumab 180 mg and 360 mg as maintenance therapy in subjects with moderately to severely active Crohn's disease. Topline results of the ADVANCE and MOTIVATE induction studies were announced in January 2021 and topline results of the FORTIFY maintenance study were announced in June 2021. About SKYRIZI® (risankizumab) SKYRIZI is an interleukin-23 (IL-23) inhibitor that selectively blocks IL-23 by binding to its p19 subunit.11,16 IL-23, a cytokine involved in inflammatory processes, is thought to be linked to a number of chronic immune-mediated diseases, including Crohn's disease.11,16 The approved dose for SKYRIZI for moderate to severe plaque psoriasis and active psoriatic arthritis in the European Union is 150 mg (either as two 75 mg prefilled syringe injections or one 150 mg prefilled pen or prefilled syringe) administered by subcutaneous injections at week 0 and 4 and every 12 weeks thereafter.11 The use of risankizumab in Crohn's disease is not approved in the European Union and its safety remains under review with the global regulatory authorities. Phase 3 trials of SKYRIZI in psoriasis, psoriatic arthritis, Crohn's disease and ulcerative colitis are ongoing.12,13,17 EU Indications and Important Safety Information about SKYRIZI® (risankizumab)11 SKYRIZI is indicated for the treatment of moderate to severe plaque psoriasis in adults who are candidates for systemic therapy. SKYRIZI, alone or in combination with methotrexate is indicated for the treatment of active psoriatic arthritis in adults who have had an inadequate response or who have been intolerant to one or more disease-modifying antirheumatic drugs. SKYRIZI is contraindicated in patients with hypersensitivity to the active substance or to any of the excipients. SKYRIZI may increase the risk of infection. In patients with a chronic infection, a history of recurrent infection, or known risk factors for infection, SKYRIZI should be used with caution. Treatment with SKYRIZI should not be initiated in patients with any clinically important active infection until the infection resolves or is adequately treated. Prior to initiating treatment with SKYRIZI, patients should be evaluated for tuberculosis (TB) infection. Patients receiving SKYRIZI should be monitored for signs and symptoms of active TB. Anti-TB therapy should be considered prior to initiating SKYRIZI in patients with a history of latent or active TB in whom an adequate course of treatment cannot be confirmed. Prior to initiating therapy with SKYRIZI, completion of all appropriate immunizations should be considered according to current immunization guidelines. If a patient has received live vaccination (viral or bacterial), it is recommended to wait at least 4 weeks prior to starting treatment with SKYRIZI. Patients treated with SKYRIZI should not receive live vaccines during treatment and for at least 21 weeks after treatment. The most frequently reported adverse reactions were upper respiratory infections. Commonly (greater than or equal to 1/100 to less than 1/10) reported adverse reactions included tinea infections, headache, pruritus, fatigue and injection site reactions. About AbbVie in Gastroenterology With a robust clinical trial program, AbbVie is committed to cutting-edge research to drive exciting developments in inflammatory bowel diseases (IBD), like ulcerative colitis and Crohn's disease. By innovating, learning and adapting, AbbVie aspires to eliminate the burden of IBD and make a positive long-term impact on the lives of people with IBD. About AbbVie AbbVie's mission is to discover and deliver innovative medicines that solve serious health issues today and address the medical challenges of tomorrow. We strive to have a remarkable impact on people's lives across several key therapeutic areas: immunology, oncology, neuroscience, eye care, virology, women's health and gastroenterology, in addition to products and services across our Allergan Aesthetics portfolio.

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Mission Bio Announces Pharma Assay Development Services for Solid Tumor Research

Mission Bio | July 13, 2022

Mission Bio, Inc., the pioneer in high-throughput single-cell DNA and multi-omics analysis, announced the launch of solid tumor assays through its Pharma Assay Development services. The availability of this new service offering will help to accelerate the development of cancer therapeutics by reducing the time and cost associated with the characterization of solid tumor cells. Precision therapeutics for solid tumors have been historically bottlenecked due to technical challenges associated with the ability to deeply profile thousands of individual cells for a rich characterization of tumor evolution and heterogeneity. This must be done at a much higher resolution compared to bulk next-generation sequencing to enable detection of rare events, at a much earlier stage before progression. Utilizing Mission Bio's Tapestri™ platform, the company's PAD services for solid tumors partner with researchers to provide high-resolution data to unmask the underlying genetic diversity across cell populations. Insights into the clonal landscape and co-occurrence of mutations enable improved patient stratification for clinical trials and the identification of druggable targets for precision therapeutics. Researchers can also monitor treatment resistance by analyzing the acquisition of rare mutations driving tumor progression over the course of treatment. The expansion of Mission Bio's PAD services to solid tumor research comes just three months after the launch of the Solid Tumor Solution on the Tapestri™ Platform, demonstrating Mission Bio's commitment to continuously developing innovative single-cell technologies for its pharma customers. "We have seen tremendous uptake and interest from top-tier pharma companies for our blood cancer Pharma Assay Development program, and we expect the same momentum for our solid tumor services. With our solid tumor services, Mission Bio partners with pharma customers to elucidate the mechanisms of drug resistance through mutation acquisition, determine how cells transform from benign to malignant states, and reveal the genomic changes enabling cancer cells to metastasize – without having to bring single-cell technology or resources in-house. From a customer's perspective, it is as easy as shipping out samples and getting a fully analyzed report back." Todd Druley, MD, PhD, Chief Medical Officer of Mission Bio Mission Bio's PAD services deliver comprehensive support across the therapeutic development process. The company has a dedicated team that works with researchers to develop assays, identify high-impact samples, and analyze the data. As part of the service, pharma partners have access to Mission Bio's innovative technology, assay development team, R&D organization, and bioinformatics support. About Mission Bio Mission Bio is a life sciences company that accelerates discoveries and cures for a wide range of diseases by equipping researchers with the tools they need to better measure and predict our resistance and response to new therapies. Mission Bio's multi-omics approach improves time-to-market for new therapeutics, including innovative cell and gene therapies that provide new pathways to health. Founded in 2014, Mission Bio has secured investment from Novo Growth, Cota Capital, Agilent Technologies, Mayfield Fund, and others. The company's Tapestri™ platform gives researchers around the globe the power to interrogate every molecule in a cell together, providing a comprehensive understanding of activity from a single sample. Tapestri™ is the only commercialized multi-omics platform capable of analyzing DNA and protein simultaneously from the same sample at single-cell resolution. The Tapestri™ Platform is being utilized by customers at leading research centers, pharmaceutical, and diagnostics companies worldwide to develop treatments and eventually cures for cancer.

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