San Diego, California--(Newsfile Corp. - November 4, 2024) - Thiogenesis Therapeutics, Corp. (TSXV: TTI) ("Thiogenesis" or the "Company") a clinical-stage biotechnology company developing novel thiol compounds, including precursors to cysteamine that have potent antioxidant and anti-inflammatory properties for mitochondrial diseases and related metabolic conditions; is pleased to announce that one of its core patents titled, "Methods For The Treatment of Cysteamine Sensitive Disorders," has been allowed by the European Patent Office (European Patent No. 18858763.8). The patent is scheduled to expire in September 2037. The new patent allowance in Europe is in addition to patents granted in this same patent family in United States in November 2021 and in Japan in 2023.
About TTI-0102 and Cysteamine
Thiogenesis' lead compound, TTI-0102, is a new chemical entity (NCE) that is an asymmetric disulfide consisting of two different thiols, moreover it is a prodrug that is metabolized into the thiol cysteamine. Thiols, like TTI-0102 and cysteamine, have a functional SH group (containing sulfur and hydrogen) and are versatile bio-active molecules that are known to be involved in a number of biochemical reactions and metabolic processes, making them promising candidates for a number of therapeutic applications.
In particular, certain thiols like cysteamine are precursors to the critical antioxidant glutathione. Glutathione is considered one of the most important antioxidants because it: is uniquely transported into the mitochondria, effectively neutralizes free radicals in the mitochondria and enhances the overall antioxidant defense system. As a result, TTI-0102 has the potential to significantly reduce oxidative stress in the mitochondria - the 'powerplant' of the cell that produces both ATP energy to power cells and volatile free radicals as a by-product. Oxidative stress occurs when there is an imbalance of too many free radicals versus antioxidants in the mitochondria, which can damage cells and exacerbate disease. Oxidative stress is a hallmark of many chronic diseases such as inherited mitochondrial diseases and related metabolic disorders such as fatty liver disease, diabetes and obesity.
TTI-0102 was developed to address the challenges facing thiol-based drugs, including their short half live, adverse side effects and dosing limitations. As a prodrug, it only becomes active after oral administration, leading to a well-tolerated and sustained release of cysteamine that can last over 24 hours. Its active moiety has been previously approved for the lysosomal disease nephropathic cystinosis. As a result, TTI-0102 is eligible for the accelerated 505 (b)(2) regulatory pathway in the U.S. and a similar hybrid pathway in the European Union.
"We are extremely pleased to have this important European patent allowed in addition to our previously granted US and Japanese patents. They provide the Company with significant long term IP protection of our novel thiol compounds, just as we are anticipating the initiation of Phase 2 clinical trials in MELAS, Leigh syndrome and pediatric MASH," said Patrice Rioux, MD, Ph.D., Chief Executive Officer of Thiogenesis. "TTI-0102 has the potential to be the best-in-class method of generating intracellular glutathione and has the promise of yielding meaningful clinical benefits for patients with diseases that have a strong oxidative stress component."
About MELAS
Mitochondrial encephalomyopathy with lactic acidosis and stroke-like episodes ("MELAS") is a rare, inherited mitochondrial disorder caused by a mutation in mitochondrial DNA. Initial symptoms include seizures, vomiting, muscle weakness, and fatigue; longer term the disease may cause a loss of motor skills and intellectual disability. Oxidative stress plays an important role in mitochondria and is a potential pathological mechanism of mitochondrial disease, making it a viable target for the treatment of MELAS and other mitochondrial diseases. The prevalence of MELAS is not well understood; however, it has been estimated to occur in approximately 15,000 patients in the US and up to 20,000 in the EU.
About Leigh Syndrome
Leigh syndrome is a rare inherited genetic disease that results from the disruption of normal mitochondrial function, it is usually diagnosed in infancy and affects an estimated 1/40,000 births. Initial symptoms for Leigh syndrome include impaired or weak sucking/breastfeeding capability, loss of motor and communication skills, respiratory issues, poor muscle development, loss of appetite and seizures. There is currently no cure for Leigh syndrome, oxidative stress is a distinguishing feature of the disease.
About Pediatric MASH
Pediatric Metabolic Dysfunction-Associated Steatohepatitis (MASH) is a disease of the liver in children and is a more severe form of pediatric Nonalcoholic Fatty Liver Disease ("NAFLD"). It is often linked to obesity and characterized by inflammation, accumulation of fat and scarring (fibrosis) in the liver. There are estimated to be well-over 1,000,000 children in the U.S. with pediatric MASH, for which there are no approved drugs or treatments.
About Thiogenesis
Thiogenesis Therapeutics, Corp. (TSXV: TTI) is a clinical-stage biopharmaceutical company operating through its wholly owned subsidiary based in San Diego, CA. The Company is publicly traded on the TSX Venture Exchange. Thiogenesis is developing sulfur-containing prodrugs that act as precursors to previously approved thiol compounds, with the potential to treat serious pediatric diseases with unmet medical needs. Prodrugs are drugs that contain previously approved active ingredients and are modified so that they only become active when metabolized. For regulatory purposes prodrugs can use existing third-party safety data in regulatory submissions in the streamlined 505 (b)(2) regulatory pathway in the U.S., and its equivalent hybrid system in Europe, to proceed into human efficacy trials with regulatory approval. Prodrugs may enhance the profile of the active ingredient to increase its bioavailability and reduce side effects. The Company's initial target indications include MELAS, Leigh syndrome, pediatric MASH and Rett syndrome.
Forward Looking Statements
This news release contains certain forward-looking statements and forward-looking information (collectively referred to herein as "forward- looking statements") within the meaning of Canadian securities laws including, without limitation, statements with respect to the future investments by the Company. All statements other than statements of historical fact are forward-looking statements. Undue reliance should not be placed on forward-looking statements, which are inherently uncertain, are based on estimates and assumptions, and are subject to known and unknown risks and uncertainties (both general and specific) that contribute to the possibility that the future events or circumstances contemplated by the forward-looking statements will not occur. Although the Company believes that the expectations reflected in the forward-looking statements contained in this press release, and the assumptions on which such forward-looking statements are made, are reasonable, there can be no assurance that such expectations will prove to be correct. Readers are cautioned not to place undue reliance on forward-looking statements included in this document, as there can be no assurance that the plans, intentions, or expectations upon which the forward-looking statements are based will occur. By their nature, forward-looking statements involve numerous assumptions, known and unknown risks and uncertainties that contribute to the possibility that the predictions, forecasts, projections and other forward-looking statements will not occur, which may cause the Company's actual performance and results in future periods to differ materially from any estimates or projections of future performance or results expressed or implied by such forward-looking statements. The forward-looking statements contained in this news release are made as of the date hereof and the Company does not undertake any obligation to update publicly or to revise any of the included forward-looking statements, except as required by applicable law. The forward-looking statements contained herein are expressly qualified by this cautionary statement.
Neither the TSX Venture Exchange nor its Regulation Services Provider (as that term is defined in the policies of the TSX Venture Exchange) accepts responsibility for the adequacy or accuracy of this news release.
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