Thiogenesis Announces FDA Clearance of its Investigational New Drug Application ("IND") for a Phase 2a Clinical Trial in Leigh Syndrome Spectrum
- FDA clearance received for IND application of TTI-0102
- Partnership with prestigious Children's Hospital of Philadelphia (CHOP) for clinical trials
- Advancement to Phase 2a clinical trial without requiring Phase 1
- Targeting an unmet medical need in rare pediatric disease
- None.
San Diego, California--(Newsfile Corp. - June 11, 2025) - Thiogenesis Therapeutics, Corp. (TSXV: TTI) (OTCQX: TTIPF) ('Thiogenesis' or the 'Company') a clinical-stage biotechnology company developing disulfides that drive the production of intracellular cysteine, an amino acid that is a critical precursor to the generation of the master antioxidant glutathione and other important therapeutic compounds, today announced that the U.S. Food and Drug Administration ("FDA") has cleared the Company's Investigational New Drug application ("IND") for TTI-0102, for the treatment of the rare pediatric mitochondrial disease Leigh syndrome spectrum ("LSS"). The Company expects to initiate the Phase 2a clinical trial in the second half of 2025.
As a New Chemical Entity ("NCE") but also a prodrug, TTI-0102 has been cleared to start a Phase 2a clinical trial that was designed, and is to be conducted, in collaboration with Children's Hospital of Philadelphia ("CHOP"), with Zarazuela Zolkipli-Cunningham, MBChB, acting as the primary site for the trial. CHOP is a recognized leader in the field of pediatric mitochondrial medicine.
"The FDA clearance marks a critical milestone for Thiogenesis and underscores the potential of TTI-0102 to address the significant unmet needs in Leigh syndrome spectrum," said Patrice Rioux, MD, Ph.D., Chief Executive Officer of Thiogenesis. "We are honored to collaborate with Zarazuela Zolkipli-Cunningham, MBChB, Marni Falk, MD, and their expert clinical research team at CHOP to advance this promising therapy."
About the Clinical Trial:
The Phase 2a clinical trial in Leigh syndrome spectrum will be conducted in two-stages:
Stage 1: A randomized, double-blind, placebo-controlled trial enrolling 9 patients, with 6 receiving TTI-0102 and 3 receiving placebo. This stage will evaluate safety, tolerability, efficacy, and pharmacokinetics / pharmacodynamics ("PK/PD") over a 3-month period, in adults and adolescent patients with Leigh syndrome spectrum.
Stage 2: An open-label extension of the trial, enrolling 6 pediatric patients with Leigh syndrome spectrum, 5 years and older, all being treated with TTI-0102 for 3 months, to further assess safety, tolerability, efficacy, and PK/PD endpoints.
In the event that the trial yields positive results and receives FDA clearance, Thiogenesis intends to advance TTI-0102 to a pivotal Phase 2b/3 clinical trial in pediatric Leigh syndrome spectrum.
"TTI-0102 has the potential to significantly enhance intracellular glutathione levels, which is critical in combatting oxidative stress," said Marni Falk, MD, Executive Director of the Mitochondrial Medicine Frontier Program at CHOP. "Based on our extensive preclinical studies of cysteamine effects in mitochondrial disease models, innovative approaches like TTI-0102 may provide meaningful clinical benefits and improve quality of life for patients with mitochondrial diseases, possibilities which we are excited to begin to study in rigorous clinical trials in the Leigh syndrome spectrum population."
About Leigh Syndrome Spectrum ("LSS")
Mitochondria are critical intracellular "powerplants" that provide the cell with the energy it needs to function normally; the disruption of mitochondrial function can result in a range of complex and life-threatening conditions. LSS is one such rare inherited genetic disease manifestation that results from the disruption of normal mitochondrial function, which is usually diagnosed in infancy and affects an estimated 1/40,000 births. Initial symptoms for LSS include impaired feeding capability, loss of motor and communication skills, respiratory and gastrointestinal problems, poor muscle function, and seizures. There is currently no cure for LSS, and treatment is primarily supportive, focusing on managing symptoms and complications. LSS is highly heterogeneous, caused by pathogenic variants in over 113 mitochondrial DNA ("mtDNA") and nuclear DNA ("nDNA") genes that adversely affect mitochondrial respiratory chain function. TTI-0102 has been engineered to combat abnormally high levels of mitochondrial oxidative stress, a key characteristic of LSS (Enns et al., 2014) and thereby help to ameliorate mitochondrial function and potentially improve clinical outcomes for these patients.
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 first-generation 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.
Neither the TSX Venture Exchange nor its Regulation Services Provider (as that term is defined in the policies of the TSX Venture Exchange) nor the OTC Markets Group Inc. (OTCQX: OTCM) accepts responsibility for the adequacy or accuracy of this news release.
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FAQ
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