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.
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.
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.
assisTek Inc | June 21, 2022
assisTek, based in Doylestown, PA, has announced its latest partnership with Philadelphia based Strados Labs. Strados Labs is known for their creation of cutting-edge smart technology medical devices for respiratory illnesses.
assisTek, has been a longtime leader in the eSource industry across various therapeutic areas, over 900+ Global clinical Trials and offers an array of services for the electronic collection of clinical data. Their technology offerings include eCOA, ePRO, eConsent, BYOD modules and an extensive reputation for their custom solutions to support clinical trial teams worldwide. "We are very excited to announce this partnership and are looking forward to working together with Strados to provide another way for clinicians and patients to gather novel information during their trials. By making the integration seamless into our current eDiary offerings, this paired technology will allow for better patient engagement and more efficient information gathering, with the ultimate goal of improved data for cures and treatment of these conditions," explains Richard Gastineau, CEO of assisTek.
Strados Labs is a medical technology company developing smart sensor platforms that utilize machine learning and proprietary algorithms to detect and predict compounding conditions associated with serious, chronic respiratory diseases to improve the lives of patients globally. "We are grateful and proud to solidify our partnership with assisTek. They have been a leader in integrating technology and data collection in clinical trials for over 20 years," said Strados Labs Director of Business Development, Shane Krauss. "We look forward to working with their experienced and knowledgeable team in clinical trials by helping them scale their digital therapeutics using our clinically validated measures for wheeze, cough, and other adventitious breath sounds, or CABS—which is something no other cleared device can measure." The Company developed a first-of-its-kind smart sensor platform for respiratory health, RESP®. Strados recently received FDA Class II 510(k) clearance for home use of its first product, RESP®, its second FDA clearance in less than 18 months. RESP® was also granted a CE Mark late last year. The Company is developing a robust pipeline algorithm to support and address other chronic diseases, as well as chronic disease management pathways. Strados will continue to target asthma, COPD and infectious respiratory diseases (COVID, RSV, and more)—with plans to add heart failure and pediatrics later this year. The Company is based in Philadelphia and is a privately held company.
Sutro Biopharma, Inc.; Astellas Pharma Inc. | June 28, 2022
Astellas Pharma Inc. and Sutro Biopharma, Inc. announced a worldwide, strategic collaboration and licensing agreement focused on the discovery and development of novel immunostimulatory antibody-drug conjugates. The collaboration leverages the unique cancer-fighting potential of iADCs as a novel modality, enabled by Sutro’s ability to engineer complex conjugated antibodies, and Astellas’ global oncology R&D expertise.
“We are delighted to work with Astellas, a premier biopharmaceutical company with substantial expertise in immuno-oncology, on this novel modality. iADCs hold promise well beyond the existing success of ADCs. Sutro’s unique conjugation technology enables dual conjugations that site-specifically incorporate a potent cytotoxin that can directly kill tumor cells together with an immunostimulatory component that has the potential to locally prime an immune response to the patient’s particular tumor cells. We look forward to collaboratively exploring the potential of this approach to treat cold tumors and bring new drug therapies to patients who do not respond to existing immunotherapies.”
William J. Newell, Sutro’s Chief Executive Officer
Naoki Okamura, Chief Strategy Officer, at Astellas commented, "Astellas considers Immuno-Oncology as one of the Primary Focuses of its R&D strategy; our goal is to bring effective drugs to patients who do not respond to existing immune checkpoint inhibitors. Sutro is a leading company in the area of iADCs, a new modality, and has its own original iADC technologies. The strategic partnership with Sutro will help us expand our pipeline and widen the choice of cancer immunotherapies.”
A key challenge with cancer immunotherapies, including immune checkpoint inhibitors, is the tumor microenvironment*1 within cold tumors creating barriers to immune cell infiltration and thus preventing the cure. The immune checkpoint inhibitors approved to date are efficacious as a monotherapy in only about 20% of cancer types, which vary widely*1.
This strategic partnership will engage in the development of iADCs, a next generation modality with the potential for effective and efficient approaches for treatment of cold tumors so as to bring new drug therapies to patients who do not respond to existing therapies. An iADC, which combines an antibody with a small molecule compound that induces immunogenic cell death*2 in addition to an immune activating molecule, has the potential to boost the anti-cancer action.
This partnership will enable Astellas and Sutro to mutually leverage strengths in their respective fields to accelerate iADC development for three distinct biological targets; Sutro will engage in research and preclinical studies to identify candidate compounds and then Astellas will pursue clinical development. Sutro has advanced technologies for linking drugs to antibodies and proprietary component parts, including candidate antibodies and linkable cytotoxins and immunostimulatory molecules. For development of iADCs, Astellas will utilize the strength of its global R&D and commercialization capabilities in the area of antibodies and the small molecular components. These iADCs may have the potential to provide new therapeutic options for treatment of cancers for which no broadly effective therapy is currently available.
Under the terms of the agreement, Sutro will receive an upfront cash payment of US$90 million to develop iADCs for three biological targets and may be eligible to receive up to US$422.5 million in development, regulatory and commercial milestones for each product candidate, and tiered royalties ranging from low double-digit to mid-teens on worldwide sales of any commercial products that may result from the collaboration, subject to Sutro’s cost and profit sharing option for the United States.
Sutro has the option to share in the costs and profits for developing and commercializing product candidates in the United States. If Sutro exercises this option for a particular product candidate, Astellas and Sutro will equally share the costs of such co-development and co-commercialization, with the resulting profits/losses from co-commercialization also shared equally in the United States.