Discovery of mechanism behind Alexander disease may lead to enhanced drug development

LabRoots | November 25, 2019

Researchers have long known that the cause behind Alexander disease is a genetic culprit—mainly a mutation leading to the production of a defective protein called GFAP. Alexander disease (AxD) is a rare neurological condition involving myelin destruction and is present during many stages of life: infancy, adolescence, or adulthood. Many of those diagnosed with condition pass away with the first years of diagnosis. Although, some can survive for several decades, babies born to the condition experience seizures and developmental delays. Now, a new research study at UNC School of Medicine examined the differences in severe and milder forms of AxD for the first time. Through their studies, they discovered that the mutant form of GFAP undergoes diverse chemical modifications depending on the time of when symptoms appear, and these modifications can be manipulated by scientists using an in-vitro system taken from AxD patient cells. The research could potential provide the basis of drug discovery and development.

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Teva Announces Unique Collaboration with HealthSnap to Expand the Reach of its Respiratory Digital Health Platform

Teva Pharmaceuticals | November 21, 2022

Teva Pharmaceuticals, a U.S. affiliate of Teva Pharmaceutical Industries Ltd. announced a new collaboration with HealthSnap, a leading full-service Virtual Care Management Platform for chronic disease management. The collaboration will enable more consumers to have access to Teva’s Digihaler® system, a digital health platform designed to enable patients to have more informed treatment discussions with their healthcare providers through personalized care. The collaboration will enable healthcare providers to better integrate patient data into their workflows, supporting proactive remote care across large populations. The platform can be used to monitor and manage large populations by a network of health systems. It can capture objective and actionable data for asthma patients, thereby creating the mechanism for personalized care. “Digital health platforms will succeed only by seamlessly integrating multiple data sources into workflows and systems like electronic medical records (EMR). We believe in building an ecosystem and are delighted to partner with HealthSnap as we seek to expand the reach of our platform, and continue to shape digital therapeutics and the integrated care landscape,” said Manny Montalvo, Senior Vice President, Head of Digital Health & Innovation at Teva. “We believe that the future of healthcare is in predictive and preventative care, which is a significant paradigm shift from the standard of care today. That is why we will focus our collaboration and our services on facilitating this change, to help providers deliver better care and help patients manage their condition.” “With a rich heritage of delivering innovative medicines, alongside technology development capabilities, Teva is uniquely positioned to help the healthcare ecosystem move toward greater integration of objective health data, and create meaningful population-scale solutions to help patients and providers. We envision broad utilization of our platform by consumers, healthcare providers and other technology organizations. In respiratory care, our long-term vision is ambitious and clear: harnessing the power of technology and therapeutics to predict asthma attacks.” Montalvo continued, “Over the past few years, through a talented and continuously evolving in-house team we have built a robust technology arm within Teva to continue exploring ways to improve patient outcomes. Through our vision for connected therapy, we strive to be at the nexus between patients, healthcare providers, technology, and the future of medicine, exploring new ways for digital health to serve as an economically sustainable solution for small and large healthcare provider organizations, offering timely, data-driven solutions to benefit patients. Our hope is that by working with HealthSnap and other capable companies in this space, we will fulfill our mission of improving the lives of patients, and do so by improving the health and effectiveness of our healthcare system – and we’re just getting started.” “By partnering with Teva, HealthSnap is expanding its reach to patients with respiratory diseases, allowing them to receive personalized care based on their data. HealthSnap is continuously seeking partnerships and we look forward to delivering quality at home programs with this new and exciting collaboration with Teva,” Samson Magid, Co-Founder and CEO of HealthSnap About Teva Teva Pharmaceutical Industries Ltd. has been developing and producing medicines to improve people’s lives for more than a century. We are a global leader in generic, biosimilar and specialty medicines with a portfolio consisting of over 3,500 products in nearly every therapeutic area. Around 200 million people around the world take a Teva medicine every day, and are served by one of the largest and most complex supply chains in the pharmaceutical industry. Along with our established presence in generics, we have significant innovative research and operations supporting our growing portfolio of specialty and biopharmaceutical products. Learn more at www.tevapharm.com About Teva Digital Health Teva aims to be a global leader in personalized, predictive care, continuously investing in platforms and regulatory-compliant systems that will help change the nature of digital health as we know it. Teva’s proprietary software platform Digihaler® – developed and maintained in-house by a team of research specialists and programmers – is built into a series of FDA-approved inhalers, currently marketed in the U.S., and with plans to expand to Europe in 2023. About HealthSnap HealthSnap is an integrated Virtual Care Management Platform that helps healthcare organizations improve patient outcomes, reduce utilization, and diversify revenue streams. From chronic disease-agnostic Remote Patient Monitoringand Chronic Care Management to AI-guided care coordination, virtual care delivery, automated care management billing, population analytics - and so much more, HealthSnap is the simplest way to manage chronic conditions remotely.

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BUSINESS INSIGHTS, PHARMACY MARKET

Annogen Announces Research Project with Pfizer to Test Non-coding Genetic Sequences

Annogen | December 14, 2022

Annogen, the Amsterdam based biotech company behind the SuRE™ technology for the functional annotation of the non-coding part of the genome, today announced it has begun a research project with Pfizer to functionally test tens of thousands of disease-related non-coding sequence variants for their effect on gene regulation. The results may contribute to drug discovery by identifying functionally relevant non-coding variants that play a central role in diseases. More than 95 percent of disease- and trait-related variants are found in the non-coding genome. However, identifying the important causal variants amongst the thousands of non-functional ones is a major challenge, as non-coding variant functionality cannot be deduced from sequence alone. Using its SuRE™ methodology, Annogen can obtain a functional read-out for up to millions of non-coding variants in parallel. “We are very proud of this research project with Pfizer. We believe the non-coding part of the genome represents a huge opportunity for drug discovery that has been largely overlooked. We are now working with several top-tier biopharma companies on a broad array of projects, which demonstrates the interest in the SuRE™ platform and the AIM™ service for gene & cell therapy, drug discovery, and recombinant protein production”. Joris van Arensbergen, Annogen’s founder and CEO, is pleased to establish another project in this field About Annogen At Annogen we use our SuRE™ technology to identify regulatory DNA elements to be used for controlled gene expression valuable for gene & cell therapy, as well as for recombinant protein production. In addition, we offer the AIM™ service to identify favorable gene insertions and their expression levels for more than 100,000 integrations in parallel. These approaches enable researchers to qualitatively interpret the non-coding genome in humans, animals and plants.

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BUSINESS INSIGHTS, PHARMA TECH

Aequus Announces Health Canada Notice of Compliance (NOC) for Zimed® Preservative Free (Bimatoprost 0.03%)

Aequus Pharmaceuticals | December 21, 2022

Aequus Pharmaceuticals Inc. is pleased to announce that Health Canada has approved Zimed® PF for the reduction of elevated intraocular pressure in patients with open-angled glaucoma or ocular hypertension. Affecting more than 700,000 people in Canada, glaucoma is one of the leading causes of blindness in North America. Zimed® PF is the first prostaglandin analog that is preservative free and offered in an easy-to-use multi-dose bottle in Canada. PGAs are commonly prescribed first-line to reduce IOP. The vast majority of medications currently available include preservatives such as Benzalkonium Chloride. Chronic exposure to BAK has been found to increase the likelihood of experiencing Ocular Surface Disease and reduce patient adherence. “We have heard from the eyecare professionals that there is a need for preservative-free alternatives. The PGA market continues to grow year-over-year and we are excited to provide a new option for Canadian health care providers and their patients.” Grant Larsen, CCO of Aequus Pharma Zimed PF is contained in a unique multi-dose bottle that leverages a unidirectional valve and air filter technology to eliminates the need for a preservatives. In addition, this packaging format significantly reduces the amount of plastic used compared to single-use alternatives. “Medicom is excited by this news and looks forward to launching Zimed® PF in the Canadian market with Aequus,” says Simon Martin, CEO of Medicom. “Globally we have seen the strong uptake for Zimed® PF and pleased it will soon be available to Canadian patients.” Doug Janzen, CEO of Aequus Pharma said, “We want to thank Medicom for their support through the approval process with Health Canada. This is our third product we’ve brought to Canada in this partnership. We look forward to launching Zimed® PF in 2023 and providing Canadians with more treatment options.” ABOUT AEQUUS PHARMACEUTICALS INC. Aequus Pharmaceuticals Inc. is a specialty pharmaceutical company focused on developing and commercializing high quality, differentiated products. Aequus has grown its sales and marketing efforts to include several commercial products in ophthalmology and optometry. Aequus plans to build on its Canadian commercial platform through the launch of additional products that are either created internally or brought in through an acquisition or license, remaining focused on highly specialized therapeutic areas.

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insitro Appoints Philip Tagari, Industry-Leading Scientist and Drug Hunter, as Chief Scientific Officer

insitro | December 02, 2022

insitro, a machine learning-powered drug discovery and development company announced that Philip Tagari has been appointed as chief scientific officer. Tagari joins insitro following a 24-year career at Amgen, where he has led the organization's research platforms for over a decade as vice president, research - therapeutic discovery. “I am deeply excited to partner with Philip on insitro’s journey to build a transformative biology platform for the efficient discovery and development of effective medicines for patients in need. Philip brings unparalleled experience in drug development, including building and utilizing cutting-edge platforms, profound scientific expertise across therapeutic areas and modalities, and most importantly, the humility and courage to transcend the drug discovery status quo and do things differently.” Daphne Koller, Ph.D., founder and CEO of insitro Tagari joins a cross-functional team of experts in biology and technology at insitro, part of a uniquely collaborative culture that spans the boundaries of both fields. “Philip’s inspiring track record of fusing science and technology to build innovative platforms and leading therapeutic programs from bench to approval will guide us in the next step of insitro’s journey, as we deploy our expertise to identify exciting new targets and design therapeutic molecules,” said Koller. “insitro is completely redefining our understanding of disease biology and developing novel therapeutic strategies for a variety of grievous illnesses. Using machine learning on genetics and multi-modal phenotypic data at scale from human cohorts and cellular systems, their cutting edge laboratories are rapidly generating differentiated approaches to currently intractable unmet needs,” Tagari said. “I’m thrilled to partner with Daphne and insitro’s world-class scientists and technologists in achieving a patient-driven vision brought to life in a different kind of drug company.” Tagari joins insitro in early 2023 from Amgen, where he has held a variety of roles since 1998, and served as vice president, research - therapeutic discovery, since 2012. During his time at Amgen, Tagari built and led a global organization of over 600 scientists that delivered numerous experimental and marketed therapies in neurology, inflammation, cardiovascular disease and oncology. Throughout his career, which includes a decade at Merck and research positions at McGill University and Oxford University, Tagari made significant contributions toward multiple first-in-class, disease-modifying, life-changing therapies including Lumakras, Repatha, Singulair, Aimovig, Evenity, Tarlatamab, Acapatamab, Efavaleukin alfa, and AMG 133. With more than 30 years of research experience, he brings expertise across neurobiology, metabolic disease, hematology/oncology, immunology and inflammation, cell and molecular biology, antibody discovery and biologics engineering, medicinal, peptide and oligonucleotide chemistry, analytical chemistry and biochemistry, structural biology, pharmacokinetics, safety pharmacology, laboratory automation and information technologies. Tagari has published or contributed to more than 70 peer-reviewed publications. “I had the privilege of working with Philip for more than 15 years, first at Merck, and thereafter at Amgen, and can say unequivocally that he ranks among the most accomplished, and most innovative, scientific leaders in the industry,” said Roger M. Perlmutter, M.D., Ph.D., currently president, chief executive officer, and chairman of Eikon Therapeutics, Inc., (formerly EVP R&D both at Amgen and at Merck), and is a member of insitro’s Board of Directors. “With Philip as chief scientific officer and partner to Daphne, insitro will be well positioned to ensure that insights from its machine learning-enabled discovery platform contribute to the development of important new medicines that will make a meaningful difference for patients.” About insitro insitro is a data-driven drug discovery and development company using machine learning and data at scale to transform the way that drugs are discovered and developed for patients. insitro is developing predictive machine learning models to discover underlying biologic states based on human cohort data and in-house generated cellular data at scale. These predictive models are being brought to bear on key bottlenecks in pharmaceutical R&D to advance novel targets and patient biomarkers, design therapeutics and inform clinical strategy. insitro is advancing a wholly owned and partnered pipeline of biologic insights and molecules in metabolism and neuroscience. Since formation in mid 2018, insitro has raised over $700 million from top tech, biotech, and crossover investors, and from collaborations with pharmaceutical partners.

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