PDGFA x PDGFB x TGF-β

MELBOURNE, Australia, Feb. 19, 2024 (GLOBE NEWSWIRE) -- Certa Therapeutics (Certa), a biotechnology company developing innovative precision therapies for patients with inflammatory and fibrotic diseases, today announces that the U.S. Food and Drug Administration (FDA) has granted Fast Track Designation for its investigational therapy FT011 for the treatment of systemic sclerosis (scleroderma), having previously granted Orphan Drug Designation.[1] The Fast Track Designation was granted based on results of the previously announced Phase 2 study which indicated that treatment of scleroderma patients with FT011 for 12 weeks resulted in a clinically meaningful improvement in 60% of patients treated with FT011 400mg and 20% of patients in the FT011 200mg group compared with 10% in the placebo group.[2] The FDA’s Fast Track program is designed to facilitate the expedited development and review of new drugs that are intended to treat serious or life-threatening conditions and demonstrate the potential to address unmet medical needs.[3] FT011 is a novel, first-in-class oral therapy for the treatment of chronic fibrosis in multiple organs. It targets a previously important but undrugged membrane GPCR receptor, GPCR68, with an extensive body of data demonstrating promising efficacy in multiple models of fibrotic disease. Transcriptomic research has validated the mechanism of action and demonstrated that treatment with FT011 results in reversal in the activation of genetic markers associated with fibrosis, providing potential for a precision therapy.[4] Professor Darren Kelly, Certa Therapeutics CEO and founder said, “We are thrilled to have received Fast Track Designation which supports further acceleration of the FT011 clinical development program. It also provides validation of FT011’s potential to offer patients with scleroderma the first anti-fibrotic and disease modifying treatment of this type.” “We know that this debilitating and life-threatening disease can severely impact the lives of patients and to date existing treatments only focus on the relief and management of symptoms, whereas FT011 precisely targets the root cause of fibrosis and has the potential to offer treatment across multiple organs within these patients.” Under the Fast Track Designation, the FT011 development program for scleroderma is eligible for various expedited regulatory review processes, including generally more frequent FDA interactions, potential eligibility for rolling review of a New Drug Application (NDA) and accelerated approval and priority review of an NDA. Certa is progressing preparations toward a pivotal clinical trial of FT011 as a treatment for scleroderma. Supported by global clinical experts, the clinical trial design and associated development plans will be discussed with the FDA as soon as possible in 2024, with complementary scientific advice sought from the EMA mid-2024, with the aim of starting the pivotal study in late-2024. C Denton, W Stevens, N Kruger, M Papadimitriou, F Khong, M Bradney, D Kelly, R Lafyatis “FT011 for the Treatment of Systemic Sclerosis. Results from a Phase II Study” Arthritis Rheumatol. 2023;75(suppl 9). Abstract 2593. U.S. Food and Drug Administration. Fast Track. Available at: Accessed January 23, 2022. S Eddy, et al., “Identification of Non-Invasive Surrogates as Predictors of Response to FT011 in Kidney Disease” ASN Kidney Week 2022, abstract 3767783. Media – Australia Kirrily Davis, E: kdavis@bcpvc.com M: +61 (0)401 220228 Media - International Sue Charles, Charles Consultants E: sue.charles@charles-consultants.com M: +44 (0)7968 726585 About Systemic Sclerosis (Scleroderma) Scleroderma is an extremely debilitating, potentially life-threatening autoimmune condition characterised by inflammation and fibrosis of the skin and other organs (commonly the lungs, kidneys, and heart). This condition results in high morbidity with substantial detriment on quality of life, with patients commonly experiencing loss of mobility and function, pain, fatigue, often accompanied with a significant impact to their mental health. Scleroderma has the highest mortality among rheumatic diseases. About Certa Therapeutics Certa Therapeutics is a clinical-stage biotechnology company focused on improving lives by treating patients with debilitating diseases via novel targeted therapies. Certa Therapeutics has designed a platform of candidate drugs and validated the role of GPR68, a defined G protein-coupled receptor (GPCR) receptor which mediates signalling pathways associated with inflammation and fibrosis. GPR68 is silent in healthy tissue but activated following injury or disease. Evidence demonstrates the role of GPR68 on multiple downstream pathways causing inflammation and fibrosis. These targeted drug candidates have established proof of concept as potential treatments for multiple fibrotic diseases including serious and chronic conditions impacting the kidney, lung, eye, skin, and heart. The morbidity and mortality impact of fibrotic diseases is substantial, ultimately causing 45% of all deaths globally. Significant breakthroughs are urgently needed in this field, addressing a market worth more than US$15B annually. The company is seeking to combine its innovative therapeutics with biomarkers and genetic analysis to identify those patients most likely to benefit from treatment, providing potential for a precision therapy. FT011 is an investigational product which has not received marketing authorisation or approval by any regulatory agency, including the US Food and Drug Administration, the European Medicines Agency, or the Australian Therapeutic Goods Agency. The investigational drug products being developed by Certa Therapeutics are undergoing clinical studies to evaluate the safety and effectiveness in humans. In the company’s lead program, FT011 is being developed as a novel, first-in-class oral therapy for the treatment of systemic sclerosis (scleroderma) and has successfully completed a multi-national, double-blinded randomised controlled trial. It has been awarded Orphan Drug Designation and Fast Track by the FDA. Follow us on LinkedIn :

Research presented today by University of Michigan Medical School highlights FT011’s ground-breaking treatment potential from human expression profiles in chronic kidney disease study supported by animal model data The research demonstrated that treatment with FT011 results in a remarkable reversal in the activation of genetic markers associated with fibrosis Fibrosis is responsible for 45 percent of all deaths globally - FT011 is a novel drug with potential to treat chronic fibrosis in multiple organs such as the kidneys, skin, lungs, heart, and eyes Certa Therapeutics is a clinical stage biotechnology company based in Melbourne, Australia, developing innovative therapies for the treatment of fibrotic diseases Michigan, USA, 4th November 2022 – New research presented today by the University of Michigan reveals ground-breaking results for Certa Therapeutics’ FT011, a novel potential treatment of serious inflammatory and fibrotic diseases. The results in a chronic kidney disease (CKD) study presented at Kidney Week 2022, the annual scientific meeting of the American Society of Nephrology, concluded FT011 highly effective in reversing the activation of molecular markers associated with fibrosis[1]. Certa Therapeutics CEO, Professor Darren Kelly, one of Australia’s leading life science entrepreneurs and a foremost expert in the study of fibrosis, said the results are a pivotal breakthrough in the development of treatments for fibrosis - an unmet clinical need responsible for 45 percent of all deaths globally in the industrialised world. “These findings indicate significant promise for FT011 as a potential novel therapeutic for the treatment of fibrotic diseases and support our plans to advance this investigational drug into later stage clinical trials,” says Professor Kelly. “The precision and insight gained from this transformative and disruptive research could change the way we identify and treat patients with chronic kidney diseases.” Comparative transcriptomic analysis with human kidney data identified potential predictive clinical and biomarker profiles of patients most likely to respond to FT011 treatment for advanced kidney diseases, including Focal Segmental Glomerulosclerosis (FSGS) and Membranous Nephropathy (MN). This analogous pattern of elevated gene activation is also present in an animal model of chronic kidney disease. Research showed that treatment with FT011 over 12 weeks in this model significantly reduced the fibrosis-associated gene response characteristic of the human disease. Consequently, the study indicates a likely positive response in human patients with chronic kidney diseases. The data was presented by Dr Matthias Kretzler’s research team. Dr Kretzler is a world-renowned nephrologist, University of Michigan Medical School Professor, and principal investigator for the Nephrotic Syndrome Study Network (NEPTUNE) – a leading research consortium of physician scientists at 26 sites in the United States and Canada which develops innovative, investigational strategies to improve the diagnosis, management and treatment of kidney diseases. NEPTUNE has recruited a prospective, longitudinal, observational cohort of more than 650 kidney disease patients with proteinuric and glomerular disease. NEPTUNE holds one of the world’s largest clinical cohorts which enables analysis of clinical, histological, genetic and molecular patient information, combined with profiles of patients’ long-term outcomes under standard of care. “Using this data set, there is a strong indication that treatment of patients with FT011 could mirror the positive impacts observed in the animal study in reversing the activation of molecular markers associated with fibrosis,” says Dr Kretzler. “FT011 has demonstrated potential to be highly effective in the treatment of fibrotic disease. In my many decades of research, I have never seen such a significant result in our model in relation to the potential of a drug to treat fibrosis in CKD.” Certa Therapeutics’ fibrosis research program has been in development for over 15 years. The company has identified and understood the gene pro for fibrosis, a significant scientific advancement in the study of fibrotic diseases. FT011 is a novel drug with potential to treat chronic fibrosis in multiple organs such as the kidneys, skin, lungs, heart, and eyes. “We have identified the biological receptor related to fibrosis which gives us the potential to target fibrosis in a wide range of disease states,” Professor Kelly continues. "To our knowledge, there is no other drug on the market that does this.” Top line data from Certa Therapeutics’ phase II trial in scleroderma patients is expected to be released in early 2023. ENDS Note to Editors: For further information or to arrange an interview, please contact: Damon Birrell, Mana Communications, db@manacommunications.com, +61 (0) Ciara Byrne, Mana Communications, cb@manacommunications.com, +61 (0) 41 3519 430 About Certa Therapeutics & FT011 Certa Therapeutics is a biotechnology company focused on improving lives by developing innovative precision treatments for inflammatory and fibrotic diseases. Certa Therapeutics has designed a platform of candidate drugs and validated the role of a novel receptor which mediates signalling pathways associated with inflammation and fibrosis. These targeted drug candidates have established proof of concept as potential treatments for multiple fibrotic diseases including serious and chronic conditions impacting the kidney, lung, eye, skin, and heart. The morbidity and mortality impact of fibrotic diseases is substantial, ultimately causing 45 percent of all deaths globally. Certa Therapeutics is seeking to combine these innovative therapeutics with biomarkers and genetic analysis to identify those patients most likely to benefit from treatment. Significant breakthroughs are urgently needed in this field, addressing a market worth more than US$15B annually. Breakthrough research conducted by Certa Therapeutics has identified a novel biologic target in the fibrosis mechanism as a defined G protein-coupled receptor (GPCR). This GPCR is silent in healthy tissue but activated following injury or disease. Evidence demonstrates the role this GPCR has on multiple downstream pathways causing inflammation and fibrosis. FT011 is a novel drug which inhibits this GPCR, offering potential to treat chronic fibrosis in multiple organs. University of Michigan Medical School Chronic Kidney Disease Initiative (CKDI) As a research consortium of physician scientists at 33 sites in the United States and Canada, along with patient advocacy groups NephCure Kidney International, the Alport Syndrome Foundation, and the Halpin Foundation, NEPTUNE strives to bring the latest advances in research to patients diagnosed with Focal Segmental Glomerulosclerosis (FSGS), Minimal Changes Disease (MCD), and Membranous Nephropathy (MN), with an overarching goal of utilizing precision medicine for rare diseases. NEPTUNE aims to understand what causes Nephrotic Syndrome (NS) and has captured information derived from clinical data, patient reported outcomes, and information from kidney biopsy tissue, blood, and urine samples. With this rich knowledge base, NEPTUNE will now transition from observing how NS impacts patients to finding new and effective ways to treat the disease. Most importantly, NEPTUNE will match the disease triggers that are active in an individual patient’s kidney, to targeted clinical trials in pre-competitive partnerships with leading companies developing novel treatments for NS. NEPTUNE is a member of the Rare Disease Clinical Research Network (RDCRN). The RDCRN, a National Center for Advancing Translational Sciences (NCATS) initiative, includes 20 rare disease consortia which all strive to identify the causes, risks of progression, and potential new therapies for a broad spectrum of diseases. [1] S Eddy, et al., “Identification of Non-Invasive Surrogates as Predictors of Response to FT011 in Kidney Disease” ASN Kidney Week 2022, abstract 3767783