CHARLOTTESVILLE, Va., February 28, 2023 -- AMPEL BioSolutions today announced a genomic precision medicine test for chronic kidney diseases, such as Lupus Nephritis, that could save lives by predicting kidney damage before it is irreversible and providing decision support for prophylactic therapies. Genes that drive the inflammatory pathways characteristic of different stages of kidney damage are revealed in the peer-reviewed journal Frontiers in Immunology. AMPEL's breakthrough approach identified markers in kidney biopsy samples that drive pathways that can be targeted by drugs. The NephroGENE® lab test, only a concept for the last few years, is now ready for development for practical use as a decision support biomarker test to manage kidney disease.
AMPEL's innovation is crucial to provide decision support for day-to-day patient care as well as effective drug development. Currently, physicians rely on microscopic examination of kidney biopsies to assess the severity of disease and there is no current way to predict drug options. Today's publication identifies the molecular pathways at every stage of damage leading to end stage renal disease as well as targets of medications to slow down the immune driven damage. And translation of key genes in animal models used for Lupus Nephritis drug development with human patients is essential to increase the success rate of clinical trials and provide earlier confirmation of the applicability of pre-clinical work to human disease.
Working with colleagues at the UVA Health and Virginia Tech, AMPEL found unique gene signatures for stages of kidney disease. In both these studies, AMPEL’s Genomic Platform allowed the breakthrough connection between human lupus nephritis and several mouse models of Lupus Nephritis used by Pharma for drug development. The team effort of two Virginia university mouse labs and the bioinformatics group at AMPEL BioSolutions has accelerated the commercialization of precision medicine testing for Lupus patients, addressing the unmet need of getting the right drug to the right person at the right time. The importance of decision support is emphasized by the observation that it takes five years on average, if ever, for a Lupus Nephritis patient to be prescribed medications that stabilize disease activity and reduce flares.
NephroGENE®'s initial focus is slowing patient progression to kidney failure that requires dialysis and transplantation but the test can be used for the 40-60% of Lupus patients diagnosed with nephritis every year, many of whom are young with an average age of thirty. Kidney involvement happens more often in women of color for a variety of reasons including genetics and health disparities. According to the Johns Hopkins Lupus Center, a major challenge of lupus nephritis is that progression to end stage renal disease occurs even when patients are on standard-of-care medications, many of which have significant side effects. And the Lupus Foundation of America notes that life-threatening flares in people with lupus nephritis were more frequent and severe compared to those without kidney involvement.
One application of NephroGENE® is to assist the pharmaceutical companies who have 45+ drugs in development for Lupus Nephritis and face the challenge of enrolling patients in clinical trials that have the best potential to respond to the treatment being tested. Enrolling the "wrong" patients can result in trial failure, often leading to cancellation of a drug's development towards FDA approval that may have benefit in a sub-group of the overall patient population. AMPEL's technology helps pharmaceutical companies connect mouse models to human disease and proactively identify the patients most likely to respond to specific treatments, thereby helping improve outcomes in clinical trials and quality of life for patients in need.
“I am thrilled to announce a genomic approach for decision support and drug development in Lupus," said Dr. Amrie Grammer, AMPEL Co-Founder, President and Chief Scientific Officer. “AMPEL's collaboration with UVA and Virginia Tech has attracted talent to the Virginia ecosystem and generated peer-reviewed publications that address the unmet need of understanding the molecular pathways driving Lupus that is essential for personalized precision medicine (the right drug for the right person at the right time)."
"Despite the advances in new drug development, the frequency with which lupus patients progress to end stage renal disease has not changed, emphasizing the need for better delineation of disease processes, development of new ways to identify those processes accurately and the means to communicate them precisely to health care professionals", said Dr. Shu Man Fu, Professor Medicine, Rheumatology and Immunology, University of Virginia School of Medicine, "Precisely translating and connecting mouse models of lupus to human disease is a step forward to identifying targeted therapies for Lupus patients."
"Virginia Tech's collaboration with AMPEL has allowed the translation of our lupus mouse model work to the clinic," said Dr. Chris Reilly, Associate Professor, Department Biomedical Sciences and Pathobiology, Virginia Polytechnic Institute and State University, "Dr. Xin Luo and I are thrilled to see our work focused on molecular pathways driving the immune system to produce damaging autoantibodies being actively translated into personalized precision medicine testing for Lupus patients."
"The ability to characterize each lupus nephritis patient accurately and provide meaningful information should provide important decision support to health care professionals and also speed up the development of new, effective targeted therapies for lupus nephritis" said Dr. Peter Lipsky, AMPEL Co-Founder, CEO and Chief Medical Officer.
"As both the leader of a national lupus organization that represents individuals struggling daily to manage their lupus nephritis and as a person who knows firsthand the limited number of biomarkers and therapies for diseases of unmet need such as lupus, I am thrilled to see AMPEL’s positive results for NephroGENE®,” stated Kathleen A. Arntsen, President & CEO of Lupus and Allied Diseases Association, “Thanks to AMPEL we are now one step closer to the day when research study enrollment and treatment decisions are based on a person’s genomics, making true personalized precision medicine a reality."
"AMPEL's work connecting mouse and human gene expression has clarified that there are initial acute and transitional stages of Lupus Nephritis that may be reversible with targeted intervention," said Dr. George Tsokos, Professor of Medicine, Harvard and Rheumatology Chief, Beth Israel Deaconess Medical Center, "Decision support to treat the inflammatory environment may prevent progression to the chronic stage that has a high conversion rate to kidney failure requiring dialysis and transplantation."
"This interesting paper examined the molecular pathology of lupus nephritis at different stages of disease evolution which is likely to be more useful than routine histology under the microscope to stage patients for clinical trials and match patients to the most effective therapy for their own disease," said Dr. Brad Rovin, Nephrology Division Director, Internal Medicine Research Vice-Chair and Lee A. Hebert Distinguished Professor of Nephrology, Ohio State Medical Center, "Importantly the molecular gene expression signatures for acute, transitional and chronic kidney disease effectively translated mouse models of lupus nephritis to human disease."
"The wonderful thing about NephroGENE® is the impact it will have on patients. As a patient and researcher, I understand the importance of new approaches to determine which therapies have the potential to make a difference in a patient's disease," said Carly Harrison, Co-Host and Chief Research/Innovation Officer of Lupus Chat, "Focusing now at the molecular level helps researchers develop more targeted approaches to how we study AND treat the different stages of Lupus. For many Lupus Nephritis patients like myself, until now our disease progression severely limited whether or not we had potential treatment options. Now, although still limited, there may be some hope for future advancements and ability to receive treatment. This is a win for the Lupus community and scientific community as a whole."
“These new findings have leveraged mouse models to develop a new molecular signature of human lupus nephritis. This could provide a new way to think about the distinct stages of lupus nephritis pathogenesis in the glomerular and interstitial compartments and perhaps be useful for selecting patients for clinical trials or improving our ability to match patients with the most appropriate therapies in the clinic,” said Dr. Maria Dall’Era, UCSF Chair Rheumatology, Director of Lupus Nephritis Clinic and Jean S. Engelman Distinguished Professor as well as Director Lupus Clinical Investigator Network, Lupus Research Alliance, “This is exactly where Lupus Nephritis needs to be going. I look forward to the day when we can place microscopic classification on the back burner and instead rely on molecular classification to select patients for specific trials and assist physicians with rational therapeutic decision making in the clinic.”
AMPEL BioSolutions is a precision medicine company commercializing a development pipeline of CLIA-certified gene expression tests for blood or tissue samples that provide clinical decision support by determining disease status, identifying molecular pathway and predicting drug options. AMPEL’s technology is a cloud-based platform that hosts proprietary DNA/RNA analytic tools and machine learning algorithms covered by 25+ filed/pending patents and 90+ peer-reviewed publications. Machine learning predictions are fueled by coverage of 95% of all known genes and an exclusive curated database of >20,000 gene expression profiles with rich clinical data. Disease areas for AMPEL’s precision medicine test portfolio include Systemic Lupus Erythematosus, Fibromyalgia, Psoriasis, Scleroderma, Atopic Dermatitis, Cardiovascular, Wellness, Autism, Sjogren’s Syndrome, Lung Cancer and COVID (SARS-Cov2). In 2022, AMPEL BioSolutions was elected to the Coalition for 21st Century Precision Medicine. And in 2023, AMPEL was ranked by Nature in the top five Precision Medicine Companies with respect to the impact of published science.