San Diego, California--(Newsfile Corp. - June 25, 2025) - Thiogenesis Therapeutics, Inc. (TSXV: TTI) (OTCQX: TTIPF), a clinical-stage biopharmaceutical company developing next-generation thiol-based therapies for mitochondrial disease and pediatric metabolic conditions, today announced that the Committee for Medicinal Products for Human Use ("CHMP") of the European Medicines Agency ("EMA") has provided scientific advice supporting the company's submission of an Investigational Medicinal Product Dossier ("IMPD") for a Phase 2a clinical trial in pediatric patients with metabolic dysfunction-associated steatohepatitis ("MASH").
This exploratory trial will evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamic effects of TTI-0102, a third-generation disulfide prodrug of cysteamine, in children ages 10 to 17 with biopsy-confirmed MASH - and with a relevant level of disease activity, including at least fibrosis stage 2. The proposed open-label, single-arm design will assess changes in liver enzymes and mitochondrial biomarkers as exploratory endpoints. EMA guidance acknowledged the high unmet medical need in this pediatric population and confirmed that initiating pediatric studies may be justified based on the clinical data available for cysteamine, the active moiety of TTI-0102.
"This EMA clearance marks another milestone in our clinical development strategy, enabling us to expand our TTI-0102 program to pediatric MASH in Europe," said Dr. Patrice Rioux, MD, Ph.D., Chief Executive Officer of Thiogenesis. "Combined with our recently initiated Phase 2 MELAS trial and IND clearance for Leigh syndrome spectrum in the U.S., we are positioned to demonstrate clinical benefit across multiple serious pediatric conditions linked to mitochondrial dysfunction."
In parallel with the clinical advancement of TTI-0102, Thiogenesis has recently been granted a European Union patent for its asymmetric disulfide prodrugs of cysteamine, titled "Compositions for Controlled Release of Cysteamine and Systemic Treatment of Cysteamine Sensitive Disorders - PCT Pub No. WO2017161318." This intellectual property establishes the company's competitive position in both mitochondrial and metabolic disease indications until 2038. The same patent has been previously granted in the U.S.
About Pediatric MASH
Pediatric Metabolic dysfunction-associated steatohepatitis ("MASH") is a severe form of fatty liver disease in children, marked by liver inflammation and damage due to fat buildup, along with scarring. It's an advanced stage of Metabolic dysfunction-associated steatotic liver disease ("MASLD"), previously known as NAFLD. Roughly 7-14% of children and adolescents in the EU have MASLD, equating to 5-10 million patients. Oxidative stress is a crucial factor in the progression of MASH, transitioning from simple steatosis to steatohepatitis and fibrosis. It causes liver damage and inflammation by creating an imbalance between reactive oxygen species (ROS) production and the body's antioxidant defenses.
About TTI-0102
Thiogenesis' lead product candidate, TTI-0102, is an asymmetric disulfide and a prodrug that acts as a precursor to the thiol 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 to treat several diseases. Cysteamine drives the production of a significant amount of intracellular cysteine, which is a precursor to important antioxidants such as glutathione and the conditionally essential amino acid taurine, both providing the potential to address oxidative stress and restore mitochondrial function. Thiogenesis seeks to develop the prodrug TTI-0102 to address the challenges of earlier thiol-based drugs, including their short half-life, adverse side effects, and dosing limitations.
About Prodrugs
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 the EU, to proceed into human efficacy trials with regulatory clearance. Prodrugs may enhance the profile of the active ingredient to increase its bioavailability and reduce side effects.
About Thiogenesis
Thiogenesis Therapeutics, Corp. (TSXV: TTI) (OTCQX: TTIPF) is a clinical-stage biopharmaceutical company with operations based in San Diego, CA. The Company is publicly traded on the TSX Venture Exchange and in the U.S. on the OTCQX. 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. Thiogenesis' lead product candidate, TTI-0102 has an active Phase 2 clinical trial in Mitochondrial Encephalopathy Lactic Acidosis and Stroke ("MELAS") an IND cleared Phase 2a clinical trial in Leigh syndrome spectrum ("LSS") and is planning clinical trials in Rett syndrome and pediatric Metabolic Dysfunction-Associated Steatohepatitis ("MASH").
For further information, please contact:
Brook Riggins, Director, and CFO
Email: info@thiogenesis.com
Tel.: (888) 223-9165
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.
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SOURCE: Thiogenesis Therapeutics, Corp.
