Acceleron abandons muscular dystrophy drug

PharmaTimes | September 18, 2019

Acceleron has announced the discontinuation of the development of its drug intended for facioscapulohumeral muscular dystrophy (FSHD), ACE-083. The drug did not achieve statistically significant improvements in functional endpoints relative to placebo in a Phase II trial, which means that Acceleron will not conduct further clinical trials. Although the drug demonstrated a robust, statistically significant increase in mean total muscle volume, the primary endpoint of the trial, the increase failed to translate to statistically significant improvements in functional tests. The company says that it is “disappointed” with the results, and that it had “stated consistently, for ACE-083 to become an important new therapy for patients with FSHD, it would have to deliver a meaningful functional benefit on top of an ability to grow muscle,” said Habib Dable, President and chief executive officer of Acceleron. He continued, “Unfortunately, in this case, the data show no evidence of such a benefit and, therefore, do not support further development of ACE-083 for FSHD. We’re grateful to the patients, families, caregivers, and investigators who participated in this research.” The company also said it is “looking forward to the first quarter of next year”, as it expects topline results from the placebo-controlled Phase II trial of ACE-083 in patients with Charcot-Marie-Tooth disease—a neuromuscular disorder of different pathophysiology.

Spotlight

Nearly 19,000 people in the United States died from overdoses that involved prescription pain relievers, also known as opioids, in 2014. This represents a 16 percent increase from the previous year and is the highest number recorded—equivalent to 52 deaths each day.1 Prescription drug monitoring programs, or PDMPs, are state-run electronic databases that collect information on controlled substances, including opioids, dispensed to patients. These systems are designed to help health care providers safely prescribe and monitor the use of these drugs, which can help prevent overdose deaths. However, PDMPs are not achieving their full potential, in part because they can be difficult or inconvenient to use. For example, in a 2014 survey, primary care physicians reported that retrieving data from PDMPs takes too much time and the format is not user-friendly.

Spotlight

Nearly 19,000 people in the United States died from overdoses that involved prescription pain relievers, also known as opioids, in 2014. This represents a 16 percent increase from the previous year and is the highest number recorded—equivalent to 52 deaths each day.1 Prescription drug monitoring programs, or PDMPs, are state-run electronic databases that collect information on controlled substances, including opioids, dispensed to patients. These systems are designed to help health care providers safely prescribe and monitor the use of these drugs, which can help prevent overdose deaths. However, PDMPs are not achieving their full potential, in part because they can be difficult or inconvenient to use. For example, in a 2014 survey, primary care physicians reported that retrieving data from PDMPs takes too much time and the format is not user-friendly.

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PHARMACY MARKET

GluCare.Health Partners with Novo Nordisk for Innovative Weight Management Patient Program

Glucare.Health and NovoNordisk: | June 09, 2022

GluCare.Health, one of the Middle East's leading health tech startups, has partnered with global pharmaceutical company Novo Nordisk, market leader in Diabetes & Obesity, to introduce a blueprint for the future of healthcare. The partnership will see a 150-patient weight management pilot program that will combine new digital bio-markers, using wearable technology and devices for continuous data monitoring, with existing therapeutics. It introduces the Middle East's first value-based reimbursement model, and will be the first relationship of its kind for Novo Nordisk globally. This innovative partnership will put accountability for outcomes vs. costs on the provider, rather than on the insurers or patients. The 6-month program, which begins in September, is open to people with obesity who meet certain criteria. Patients will receive an unparalleled level of in-patient and data-driven remote care, with a multidisciplinary clinic team having continuous access to all patient data through an intelligent analytical platform. If patients do not lose at least 10% of their body weight by the end of the program, GluCare.Health and Novo Nordisk will cover all or a portion of the cost under the value-based reimbursement pilot. GluCare.Health is also committed to transparency when it comes to reporting results of the program. With one in three adults affected by obesity in the UAE[1], GluCare.Health's Co-Founder and Managing Director, Dr. Ihsan AlMarzooqi, says that the blueprint for care has the potential to not only save many lives but the UAE economy billions of dollars in costs and lost productivity. "When patients with chronic conditions are not supported properly, it can have a devastating impact on those individuals and their families. Additionally, the cost of treating the health consequences of obesity to the UAE economy alone is approximately $3.6 billion annually[2]. Traditional models of care remain woefully limited when it comes to outcomes. This partnership allows us to embed Novo Nordisk's advanced therapeutics into a companion platform like GluCare.Health, which enhances its efficacy dramatically." About Glucare.Health The first hybrid Digital Therapeutics (DTx)+Human vertically integrated diabetes platform of its kind in the world, GluCare.Health is reinventing metabolic care and transforming lives. Conceptualized in the UAE and opening the doors to its state-of-the-art 10,000 sq foot Dubai clinic in September 2020, it is the world's first healthcare provider to empower both clinicians and patients through Remote Continuous Data Monitoring as part of its standard model of care, an innovative and highly personalized 'continuous healthcare' approach that provides a comprehensive and real-time view of patients. As part of its unique model, GluCare is also an early adopter of - and the region's first clinic to use - Digital Therapeutics (DTx), combining it with wearable and smart technology, data analytics, unique in-clinic workflows, and a caring and connected expert care team. Applying technology as a humanizing force, GluCare.Health's team partners with patients and their families, giving them the tools, knowledge, and continuous support to live longer, healthier, and more fulfilling lives. About NovoNordisk Novo Nordisk is a leading global healthcare company, founded in 1923 and headquartered in Denmark. Our purpose is to drive change to defeat diabetes and other serious chronic diseases such as obesity and rare blood and endocrine disorders. We do so by pioneering scientific breakthroughs, expanding access to our medicines, and working to prevent and ultimately cure disease. Novo Nordisk employs about 49,300 people in 80 countries and markets its products in around 170 countries. For more information, visit Novo Nordisk UAE.

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PHARMA TECH

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

ERS Genomics and Lepton Pharmaceuticals LTD Enter CRISPR/Cas9 License Agreement

ERS Genomics | July 27, 2022

ERS Genomics Limited is pleased to announce a new license agreement with Lepton Pharmaceuticals LTD. This is a non-exclusive licensing agreement granting Lepton access to the ERS CRISPR/Cas9 patent portfolio for certain applications. ERS, co-founded by Dr. Emmanuelle Charpentier, 2020 Nobel Prize winner for gene-editing, provides access to the foundational CRISPR/Cas9 intellectual property. Over 90 patents are held in over eighty countries. Lepton Pharmaceutical LTD is a biotechnology company developing a proprietary, breakthrough technology to substantially improve adaptive cell-mediated immune response. "We are very pleased to establish this relationship and license agreement with ERS Genomics. It will greatly assist us to develop our innovative Castling technology to significantly improve immune cells efficiency and longevity to fight cancer." Dr. Daniel Zurr, CEO of Lepton Eric Rhodes, CEO at ERS Genomics added: "Lepton's Castling technology has great potential to impact the field of immunotherapies and we are pleased to help enable Lepton in pursuing this exciting approach." The Castling technology is a proprietary, miRNA-based general technology platform, essentially consisting of the manipulation of miRNA expression to achieve enhanced efficacy and longevity (reduced exhaustion) of cell-based therapies. miRNAs are noncoding RNAs that each affects the expression of hundreds of coding genes. The Castling technology allows, in a single gene editing event in the immune cell (either allogeneic or autologous), to impact the expression of a multitude of genes in the immune cells, once such miRNAs penetrate the microenvironment of cancer cells. This technology aims to revolutionize the immune cell cancer therapy field. About ERS Genomics ERS Genomics is a biotechnology company based in Dublin, Ireland. The company was formed to provide broad access to the foundational CRISPR/Cas9 intellectual property held by Dr. Emmanuelle Charpentier. Non-exclusive licenses are available for research and sale of products and services across multiple fields including: research tools, kits, reagents; discovery of novel targets for therapeutic intervention; cell lines for discovery and screening of novel drug candidates; GMP production of healthcare products; companion animal and livestock health; production of industrial materials such as enzymes, biofuels and chemicals; and synthetic biology. About Lepton Pharmaceuticals Lepton Pharmaceuticals LTD. is based in Israel. Lepton focuses on subtle and precise silencing of coding RNA expression by small non-coding RNA molecules, working via the DROSHA/DICER/RISC pathways, small interfering RNA and microRNA (miRNA), to develop products and services to, ultimately, improve therapies for underserved diseases and improve and change patients' lives.

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