PHARMA TECH

Xeris Pharmaceuticals Enters Collaboration Agreement With Merck

Xeris | October 25, 2021

Xeris Biopharma Holdingsa biopharmaceutical company developing and commercializing unique therapies for patient populations in endocrinology, neurology, and gastroenterology, today announced a collaboration agreement with Merck , with an option to license Xeris

We are quite excited to be working closely with Merck on this opportunity using our novel platform technology, XeriJect The XeriJect platform has broad applicability in improving the delivery of large molecules, such as monoclonal antibodies. This is another in a series of collaborations Xeris has undertaken with top 10 pharma companies evaluating the XeriJect technology.”

Paul R. Edick, Chairman and CEO of Xeris Biopharma
 
Mr. Edick continued, "These collaborations are important to validating our technology. We recognize they take time to complete and thus, they are not immediately critical to our near-term core business. However, should Merck exercise the negotiated license agreement, they could be very valuable to Xeris. It is the continued growth of Gvoke® and Keveyis®, the potential for the approval and launch of Recorlev, a pipeline supported by our novel technologies, our strong cash balance, and an expected $50 million in synergies from the Strongbridge acquisition that puts Xeris in a solid financial position.”

About Xeris Biopharma Holdings
Xeris (Nasdaq: XERS) is a biopharmaceutical company developing and commercializing unique therapies for patient populations in endocrinology, neurology, and gastroenterology. Xeris has two commercially available products, Gvoke®, a ready-to-use liquid glucagon for the treatment of severe hypoglycemia and Keveyis®, the first and only FDA-approved therapy for primary periodic paralysis. Xeris also has a robust pipeline of development programs to extend the current marketed products into important new indications and uses and bring new products forward using its proprietary formulation technology platforms, XeriSol™ and XeriJect™, supporting long-term product development and commercial success.

Forward-Looking Statements
Any statements in this press release about future expectations, plans and prospects for Xeris Biopharma Holdings, Inc. including statements regarding the market and therapeutic potential of its products and product candidates, expectations regarding clinical data or results from planned clinical trials, estimates and projections about the potential benefits of the Strongbridge Biopharma acquisition, estimates and expectations regarding potential collaborations, the timing or likelihood of regulatory approval and commercialization of its product candidates, the timing or likelihood of expansion into additional markets, the timing or likelihood of identifying potential development and commercialization partnerships, the potential utility of its formulation platforms and other statements containing the words “will,” “would,” “continue,” and similar expressions, constitute forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. These forward-looking statements are subject to risks and uncertainties that may cause actual results to differ materially from those indicated in the forward-looking statements. Such risks and uncertainties include, but are not limited to, failure to realize the expected benefits of the acquisition, failure to promptly and effectively integrate Strongbridge’s businesses, general economic and business conditions that affect the combined companies following the consummation of the acquisition, the impact of the COVID-19 pandemic on the combined businesses following the consummation of the transaction, changes in global, political, economic, business, competitive, market and regulatory forces, future exchange and interest rates, changes in tax laws, regulations, rates and policies, future business acquisitions or disposals and competitive developments. These forward-looking statements are based on numerous assumptions and assessments made in light of Xeris’ experience and perception of historical trends, current conditions, business strategies, operating environment, future developments, and other factors it believes appropriate. By their nature, forward-looking statements involve known and unknown risks and uncertainties because they relate to events and depend on circumstances that will occur in the future. The factors described in the context of such forward-looking statements in this communication could cause Xeris’ plans with respect to Strongbridge, Xeris’ plans with respect to XeriJect, Xeris’ actual results, performance or achievements, industry results and developments to differ materially from those expressed in or implied by such forward-looking statements. Although it is believed that the expectations reflected in such forward-looking statements are reasonable, no assurance can be given that such expectations will prove to have been correct and persons reading this communication are therefore cautioned not to place undue reliance on these forward-looking statements which speak only as at the date of this communication. Additional information about economic, competitive, governmental, technological, and other factors that may affect Strongbridge is set forth in Item 1A, “Risk Factors,” in Strongbridge’s 2020 Annual Report on Form 10-K, which has been filed with the SEC, the contents of which are not incorporated by reference into, nor do they form part of, this communication. Any forward-looking statements in this communication are based upon information available to Xeris, as of the date of this communication and, while believed to be true when made, may ultimately prove to be incorrect. Subject to any obligations under applicable law, Xeris does not undertake any obligation to update any forward-looking statement whether as a result of new information, future developments or otherwise, or to conform any forward-looking statement to actual results, future events, or to changes in expectations. All subsequent written and oral forward-looking statements attributable to Xeris or any person acting on behalf of any of them are expressly qualified in their entirety by this paragraph.


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Automated image analysis systems that provide detailed morphological information are rapidly replacing manual microscopy as the most effective method of characterizing both particle shape and size. These sophisticated new instruments use the latest technology to provide useful and actionable insight. Until recently, these instruments had been regarded as rather niche, due to their previously limited functionality. However, significant advances in terms of both their capabilities and performance, along with the new insights into particle morphology that these instruments provide, have opened up exciting new avenues for research and development and helped optimize manufacturing and production techniques to make them a much more generally attractive proposition.

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Inozyme Pharma Announces Partnership with Rady Children’s Institute for Genomic Medicine to Advance Newborn Screening for Genetic Diseases

Inozyme Pharma Inc. | June 17, 2022

Inozyme Pharma, Inc. a clinical-stage rare disease biopharmaceutical company developing novel therapeutics for the treatment of abnormal mineralization, today announced a partnership with Rady Children’s Institute for Genomic Medicine (RCIGM) to advance and evaluate a novel newborn screening technology to facilitate diagnosis of genetic diseases. The partnership includes several leading genomics, biotechnology companies and patient advocacy groups and focuses specifically on a diagnostic and precision medicine guidance tool called BeginNGS™, which incorporates rapid Whole Genome Sequencing (rWGS®) to currently screen newborns for approximately 400 genetic diseases. “Newborn screening will be essential to identifying and initiating timely intervention in children with rare genetic disorders like GACI (generalized arterial calcification of infancy) as we advance INZ-701 through clinical testing. We look forward to working with Rady Children’s Institute for Genomic Medicine, and with the BeginNGS consortium, to advance the use of this promising screening technology.” Catherine Nester, vice president, physician and patient strategies at Inozyme Pharma RCIGM is in a pilot evaluation that aims to supplement existing newborn screening protocols at birthing hospitals throughout the United States. The pilot program’s goal is for BeginNGS to become the genetic disease screening standard, with testing expanding to approximately 1,000 [disorders] and sequencing of 3.7 million newborns annually. Founding members of the public-private BeginNGS consortium include Inozyme, Alexion, Travere Therapeutics, and several patient advocacy groups that are helping to advance this program. “RCIGM helped pioneer the use of rWGS for diagnosis of genetic disease in intensive care settings,” said Stephen Kingsmore, MD, DSc, president and CEO of RCIGM. “With the proven clinical utility of diagnostic rWGS , we are using that experience to screen, diagnose, and help treat genetic conditions at or before onset of symptoms. Through a public-private consortium of leading organizations such as Inozyme, and advocacy groups in pediatrics, genetics, biopharma, biotech, and information technology, we aim to scale newborn sequencing to every life-threatening childhood genetic disease, RCIGM believes now is the time to end the diagnostic and therapeutic odyssey for all children with treatable genetic diseases.” BeginNGS developed through a research collaboration with Alexion; AstraZeneca’s Rare Disease group; Illumina, Inc.; TileDB; Fabric Genomics; and Genomemon, which uses rWGS to diagnose and identify treatment options for genetic conditions before symptoms begin. This approach represents an advance over current pediatric uses of rWGS that focus mainly on children who are already critically ill. Once a diagnosis is made, BeginNGS uses Genome-to-Treatment (GTRx™), a tool that provides immediate treatment guidelines to help physicians understand genetic conditions and their available treatment options. Addressing the Need for Enhanced Newborn Screening Tools Traditional newborn screening is one of the most successful public health programs in the United States. Of nearly 4 million babies born annually, 98 percent are tested in the first days of life. The BeginNGS test identifies serious childhood diseases that have effective treatments. States currently screen for only 31 to 76 of the hundreds of severe, childhood genetic diseases that have available treatments. Adding a new condition to the screening protocol is slow (5 to 6 years per condition), laborious, and costly. In the last decade, WGS has increased in speed, diagnostic performance, and scalability. BeginNGS will not replace the current biochemical newborn screening paradigm; rather, it is designed to complement the newborn screening processes and infrastructure that are already in place. “We are thrilled at the prospect of newborn screening to assist in early identification of infants affected by ENPP1 Deficiency and ABCC6 Deficiency via Inozyme’s collaboration with Rady Children’s Institute for Genomic Medicine. Early diagnosis is crucial to improving a baby’s chances of survival and long-term health if they have these rare and devastating diseases,” said Christine O’Brien and Liz Molloy, co-presidents of GACI Global. About Rady Children’s Institute for Genomic Medicine Rady Children’s Institute for Genomic Medicine is transforming pediatric critical care by advancing disease-specific healthcare for infants and children with rare disease. Discoveries at the Institute are enabling rapid diagnosis and targeted treatment of critically ill newborns and pediatric patients at Rady Children’s Hospital-San Diego and a growing network of more than 60 children’s hospitals nationwide. The vision is to expand delivery of this life-changing technology to enable the practice of Rapid Precision Medicine™ at children’s hospitals across the nation and the world. RCIGM is a non-profit, research institute embedded within Rady Children’s Hospital and Health Center. About Inozyme Pharma Inozyme Pharma, Inc. is a clinical-stage rare disease biopharmaceutical company developing novel therapeutics for the treatment of diseases of abnormal mineralization impacting the vasculature, soft tissue, and skeleton. Through our in-depth understanding of the biological pathways involved in mineralization, we are pursuing the development of therapeutics to address the underlying causes of these debilitating diseases. It is well established that two genes, ENPP1 and ABCC6, play key roles in a critical mineralization pathway and that defects in these genes lead to abnormal mineralization. We are initially focused on developing a novel therapy, INZ-701, to treat the rare genetic diseases of ENPP1 and ABCC6 Deficiencies. INZ-701 is currently in Phase 1/2 clinical trials for the treatment of ENPP1 Deficiency and ABCC6 Deficiency.

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Labcorp Enhances Clinical Trial and Drug Development Capabilities Through Real World Data Collaboration With HealthVerity

Labcorp® | June 23, 2022

Labcorp® a leading global life sciences company, announced a new collaboration with HealthVerity, Inc., the leader in Identity, Privacy, Governance, and Exchange (IPGE) for real-world data that will expand Labcorp’s comprehensive, end-to-end drug development and clinical trial programs. HealthVerity’s IPGE platform, an integrated technology and RWD infrastructure, enables Labcorp Drug Development and other participating companies to access fully interoperable, HIPAA-compliant data from the U.S.’s largest ecosystem1 of health care and consumer data. “This collaboration allows Labcorp to expand existing end-to-end solutions for drug and diagnostics development, commercialization and clinical trial efforts to include large-scale access to real-world data for research applications. By applying advanced analytics, Labcorp can help its clients improve their processes and reach better outcomes. Our substantial repository of test results can also help study sponsors more quickly and accurately assess patient eligibility for clinical trials, enroll patients faster and accelerate the availability of new medicines.” Dr. Paul Kirchgraber, CEO of Labcorp Drug Development Now more than ever, study sponsors are seeing the potential of RWD to yield longitudinal patient insights before, during and after trials. With more predictive analytics and artificial intelligence applications requiring comprehensive and fully interoperable RWD, Labcorp can align de-identified patient data with ten times greater accuracy2 than industry alternatives by using the HealthVerity IPGE platform. In addition, access to HealthVerity’s RWD with on-demand de-identification and data linkage capabilities reinforces Labcorp’s ability to be a trusted source of information for its clients. "Fragmentation of patient data is at an all-time high, and the goal is no longer just connecting this data," said Andrew Kress, CEO of HealthVerity. "Rather, the goal is making data more accessible and useful for gaining a detailed understanding of patient journeys. With analytics and applications requiring more frictionless access to the data itself, the HealthVerity IPGE platform stands alone in offering the ability to combine transaction-level patient data in a de-identified, fully interoperable manner, and delivering it directly into the client's applications of choice." About Labcorp Labcorp is a leading global life sciences company that provides vital information to help doctors, hospitals, pharmaceutical companies, researchers, and patients make clear and confident decisions. Through our unparalleled diagnostics and drug development capabilities, we provide insights and accelerate innovations to improve health and improve lives. With over 75,000 employees, we serve clients in more than 100 countries. Labcorp (NYSE: LH) reported revenue of $16 billion in FY2021. About HealthVerity Pharmaceutical manufacturers, payers, and government organizations have partnered with HealthVerity to solve some of their most complicated use cases through transformative technologies and real-world data infrastructure. The HealthVerity IPGE platform, based on the foundational elements of Identity, Privacy, Governance, and Exchange, enables the discovery of real-world data across the broadest healthcare data ecosystem, the building of more complete and accurate patient journeys and the ability to power best-in-class analytics and applications with flexibility and ease. Together with our partners, HealthVerity has built the modern way to data for the health insights economy.

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C-Path and Replica Analytics Collaboration Aims to Accelerate Rare Disease Research

Replica Analytics | June 30, 2022

Critical Path Institute and Replica Analytics, an Aetion company, announced a new partnership that involves leveraging synthetic data to further catalyze the generation of actionable solutions to accelerate drug development for rare diseases. Replica Analytics will help generate synthetic datasets across rare and orphan indications in which patient-level datasets are often quite small, which heightens considerations regarding data privacy and accessibility. Together with real data, these synthetic data will help maximize the utility of C-Path's Rare Disease Cures Accelerator-Data and Analytics Platform which provides a centralized and standardized infrastructure to support and accelerate rare disease characterization targeted to accelerate drug development. Additionally, the platform includes a framework that supports the rigorous conduct of natural history studies, with attention to established data quality standards, to be most useful to clinical trial design and regulatory review. It includes a robust, integrated database and analytics hub that allows for the aggregation of rare disease data from various sources and the efficient and effective interrogation of that data. Once added to RDCA-DAP, the synthetic data will also represent valuable real-world, electronic health record (EHR) based data available on the platform, opening doors to modeling and research based on longitudinal focused analyses. "Our collaboration with Replica Analytics is important because synthetic data can add value to real-world data while observing data privacy considerations, which will help accelerate overall rare disease drug development. The goal of RDCA-DAP is to provide a centralized and standardized infrastructure to support and accelerate rare disease characterization and therapy development, this collaboration is in line with our efforts." RDCA-DAP Scientific Director Alexandre Betourne, Pharm.D., Ph.D Synthetic data generation is a privacy enhancing technology that has been gaining rapid adoption, particularly in the life sciences sector. SDG uses AI to create machine learning models that learn the statistical patterns and properties of real datasets to generate data that retain the same characteristics as the original dataset, but with no one-to-one mapping back to an identifiable person. SDG can help amplify small datasets, simulate virtual patients to augment patients in existing datasets, and optimize the design of small sample clinical trials. "We are certainly seeing a growing opportunity to partner with organizations like C-Path for generating datasets that are fit-for-purpose," says Khaled El Emam, Ph.D., SVP and GM of Replica Analytics. "Synthetic data, which preserves the integrity and utility of source data, as well as being privacy-protective, can be a very valuable tool to enable this collaboration." In May 2022, C-Path hosted a webinar in which the Replica Analytics team introduced SDG and discussed the role the technology can play in researching and treating rare diseases. The event was recorded and can be viewed on C-Path's YouTube channel. About C-Path Critical Path Institute is an independent, non-profit organization established in 2005 as a public and private partnership. C-Path's mission is to catalyze the development of new approaches that advance medical innovation and regulatory science, accelerating the path to a healthier world. An international leader in forming collaborations, C-Path has established numerous global consortia that currently include more than 1,600 scientists from government and regulatory agencies, academia, patient organizations, disease foundations, and hundreds of pharmaceutical and biotech companies. C-Path U.S. is headquartered in Tucson, Arizona, C-Path in Europe is headquartered in Amsterdam, Netherlands and C-Path Ltd. operates from Dublin, Ireland with additional staff in multiple other locations. Critical Path Institute is supported by the Food and Drug Administration of the U.S. Department of Health and Human Services and is 54.2% funded by the FDA/HHS, totaling $13,239,950, and 45.8% funded by non-government sources, totaling $11,196,634. The contents are those by the author(s) and do not necessarily represent the official views of, nor an endorsement by, FDA/HHS or the U.S. Government. About Replica Analytics, an Aetion company Replica Analytics is the premier science-based SDG technology provider to the healthcare industry. The company a pioneer in the development of unique technologies for generating privacy-protective synthetic data that maintain the statistical properties of real-world data. The company was acquired in late 2021 by Aetion, the leading regulatory-grade real-world evidence technology provider. Replica Synthesis software provides a full suite of synthetic data generation and evaluation capabilities that can solve multiple grand challenges facing the life sciences industry, and health research in general. About Aetion Aetion is a healthcare analytics company that delivers real-world evidence for the manufacturers, purchasers, and regulators of medical treatments and technologies. The Aetion Evidence Platform® analyzes data from the real world to produce transparent, rapid, and scientifically validated answers on safety, effectiveness, and value. Founded by Harvard Medical School faculty members with decades of experience in epidemiology and health outcomes research, Aetion informs healthcare's most critical decisions—what works best, for whom, and when—to guide product development, commercialization, and payment innovation.

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