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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 :

Not only is skin hardened in systemic sclerosis, but internal organs are also affected too. Image credit: Shutterstock/Numstocker. AnaMar has received orphan drug designations for its systemic sclerosis candidate AM1476 from the FDA and the EMA. The Swedish biotech is now eligible for several incentives in the two regions, according to the designations. Both agencies will provide regular feedback during the application process and waive certain fees. The FDA also offers seven years of market exclusivity while the EMA promises ten years of protection from competition. AnaMar’s AM1476 is an oral antagonist of the 5-hydroxytryptamine receptor (5-HT2B) receptor, which is also known as a serotonin receptor. The company says its candidate halts key signalling pathways associated with fibrosis in a 5 February press release. Systemic sclerosis , also known as scleroderma, is a progressive autoimmune disease characterised by inflammation and fibrosis. Not only is skin hardened in this condition, but internal organs can be affected too. Interstitial lung disease (ILD) is one of the most common disease consequences, occurring in approximately 80% of patients. Though no approved therapies are treating the underlying cause of the disease, the FDA has approved drugs that manage associated complications. Boehringer Ingelheim’s Ofev (nintedanib) became the first drug available to treat systemic sclerosis-associated ILD when the FDA approved the tyrosine kinase inhibitor in September 2019. Genentech’s subcutaneous injection Actemra (tocilizumab) followed as the first biologic with FDA approval in March 2021. See Also: Contentious Medicare price negotiations underway as US drug prices rise Inmagene to secure licence for two HUTCHMED drug candidates The market for systemic sclerosis is expected to be worth $2.05bn in 2030, as per GlobalData’s Pharma Intelligence Centre. GlobalData is the parent company of Pharmaceutical Technology . AnaMar says that there are no treatments on the market that stop or reverse scarring in both lung and skin tissue. The biotech says its candidate has a dual-action approach to treat both these aspects of the disease. AM1476 has established pharmacokinetic and safety profiles based on Phase I data. An already-designed Phase II trial will evaluate the treatment effects of AM1476 in 60 patients with systemic sclerosis-associated ILD. Patients’ lung function and skin thickness will be assessed over a year. Australian biotech Certa Therapeutics gained FDA orphan drug designation for its systemic sclerosis candidate. Certa’s FT011 is also an oral anti-fibrotic agent. The company has already unveiled positive results from a Phase II trial assessing patients’ skin, with pivotal trial plans underway. GSK is also targeting scleroderma treatment with its B-cell inhibiting monoclonal antibody Benlysta (belimumab). Its candidate received an FDA orphan drug designation in February 2023. Though the company said it planned to initiate a Phase II/III trial in the first half of 2023, no updates have been issued since.