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July 22, 2024 -- Certa Therapeutics (Certa), a biotechnology company developing innovative precision therapies for patients with inflammatory and fibrotic diseases, today announces that the European Medicines Agency (EMA) has granted Orphan Drug Designation (ODD) for its lead drug candidate, FT011, as a potential treatment of Systemic Sclerosis (SSc). Certa has previously also received ODD and Fast Track designation for FT011 as a potential treatment of SSc from the U.S. Food and Drug Administration (FDA). FT011 is a novel, first-in-class oral therapy for the treatment of chronic fibrosis in multiple organs. It targets an important but previously undrugged membrane GPCR receptor, GPR68, a master switch of fibrosis. An extensive body of data demonstrates promising efficacy in multiple in vitro and in vivo models of inflammatory and fibrotic disease. Phase I and IIa clinical studies in patients with SSc have also demonstrated favourable efficacy, safety and pharmacokinetics. SSc is a chronic, progressive, autoimmune disease characterised by inflammation and fibrosis (scar tissue formation) in the skin and in various internal organs - commonly lungs, kidneys and heart. Skin fibrosis is the distinguishing feature of SSc and is associated with significantly reduced function and disability in patients. Currently, there are no treatments on the market that effectively stop or reverse scarring in the skin and organs. Professor Darren Kelly Certa Therapeutics CEO and founder said, “GPR68 inhibition modulates the biological pathways causing inflammation and fibrosis to the skin in patients with SSc. With limited treatment options available for patients with SSc, the EMA Orphan Drug Designation and FDA Orphan Drug and Fast Track Designations reflect the potential for FT011 to address a critical need for people living with this debilitating and life-threatening condition. “These are significant validations for the company and highlight the unmet need for novel medicines to reduce inflammation and scarring in chronic fibrotic diseases, which are often progressive and can have a poor prognosis.” Certa is planning for a Phase IIb confirmatory SSc clinical trial with FT011, bringing the company closer to providing better treatment options for patients with fibrosis. It is also developing biomarkers and gene signatures to identify patients most likely to respond to treatment to ensure the best outcomes for those with fibrosis. The FDA and EMA grant orphan status to products intended to treat, diagnose, or prevent life-threatening rare diseases or conditions that affect fewer than five in 10,000 people in Europe, or under 200,000 people in the US, and with either no currently approved method of diagnosis, prevention, or treatment, or with significant benefit to those affected by the disease. Orphan drug designation provides certain benefits, including the potential for extensive marketing exclusivity following regulatory approval, reduction in regulatory fees and, in the case of EU, a centralised approval process. 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. 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. In addition to being granted EMA Orphan Drug Designation, FT011 has been awarded Orphan Drug Designation and Fast Track Designation from the FDA. 内容来源于网络，如有侵权，请联系删除。

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 :