San Diego, California--(Newsfile Corp. - September 17, 2024) - Thiogenesis Therapeutics, Corp. (TSXV: TTI) ("Thiogenesis" or the "Company") a clinical-stage biotechnology company developing disulfides that are precursors to thiol-active compounds and potent antioxidants targeting unmet pediatric diseases, today announced that Patrice Rioux, M.D., Ph.D., the Company's Chief Executive Officer, has been invited to present this week at the Cure Mito Foundation's "Empower and Inspire: 3rd Annual Leigh Syndrome Symposium." The Cure Mito Foundation is a global non-profit patient advocacy organization led by parents who volunteer their time to change the future for families affected by Leigh syndrome.
Dr. Rioux's presentation is titled, "A New Chemical Entity (TTI-0102) for the Treatment of Glutathione-Deficient Mitochondrial Diseases Like Leigh Syndrome and MELAS."
Leigh Syndrome and TTI-0102
Leigh syndrome is a rare inherited genetic disease that results from the disruption of normal mitochondrial function. It is usually diagnosed in infancy and occurs in an estimated 1/40,000 live births. Initial symptoms of Leigh syndrome include impaired or weak sucking/breastfeeding capability, loss of motor and communication skills, significant respiratory issues, poor muscle development, loss of appetite and seizures. There is currently no cure for Leigh syndrome, and treatment is primarily supportive, focusing on managing symptoms and complications. TTI-0102 has been engineered to increase the intracellular levels of the critical antioxidant glutathione to combat abnormally high levels of mitochondrial oxidative stress, a key characteristic of Leigh syndrome, and thereby potentially help restore normal mitochondrial function to improve clinical outcomes for these patients.
About MELAS
Mitochondrial encephalomyopathy with lactic acidosis and stroke-like episodes ("MELAS") is a rare, inherited mitochondrial disorder, most often caused by genetic mutations in mitochondrial DNA. Initial symptoms usually include fatigue, loss of appetite, muscle weakness, headaches and seizures. Longer term the disease may cause a loss of motor skills and intellectual disability. Oxidative stress plays an important role in mitochondrial dysfunction and is a pathological mechanism of mitochondrial disease, making TTI-0102 a potential treatment of MELAS and other mitochondrial diseases. There are no approved treatments for MELAS and there are an estimated 15,000 patients in the US and 20,000 in the EU.
"It is an honor to be given the opportunity to present at the Cure Mito Symposium," said Dr. Rioux. "TTI-0102 has the potential to increase intracellular glutathione and combat oxidative stress which is prevalent in mitochondrial diseases and provide a much-needed therapeutic option in these unmet pediatric diseases. We recently announced clinical program updates in Leigh syndrome and MELAS and anticipate the initiation of our first Phase 2 clinical trial shortly."
About TTI-0102
Thiogenesis' lead compound, TTI-0102, is a new chemical entity that is an asymmetric disulfide and a prodrug that acts as a precursor to the thiol-active compound cysteamine. Thiols, which have a functional SH group (containing sulfur and hydrogen) are versatile bio-active molecules that are known to be involved in key biochemical reactions and metabolic processes, making them promising candidates for several therapeutic applications. Thiols are known to be precursors to important antioxidants such as glutathione, and to further reduce inflammation, as a result they have the potential to significantly reduce oxidative stress in the mitochondria. The oral prodrug TTI-0102 was developed to address the challenges of first-generation thiol-active drugs, including their short half live, adverse gastrointestinal side effects and dosing limitations.
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-active 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, Rett syndrome and pediatric NASH.
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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