Thiogenesis Announces Second Site Begins Enrolling in Phase 2 MELAS Clinical Trial and Provides Update

Rhea-AI Summary

Thiogenesis Therapeutics announced the activation of its second clinical site at Angers University Hospital Center in France for its Phase 2 MELAS clinical trial of TTI-0102. The trial has begun enrolling patients, with the first French patient dosed last week. The study is a multicenter, randomized, double-blind, placebo-controlled trial evaluating TTI-0102 in 12 MELAS patients across the Netherlands and France, with 8 receiving the treatment and 4 receiving placebo. The company has already reached the enrollment threshold required for a three-month interim analysis, expected in September 2025. The trial will assess safety, tolerability, PK/PD, and efficacy over six months, with key endpoints including the 12-Minute Walk Test, Fatigue Severity Scale, and WHOQOL-BREF Quality of Life assessment.

Positive

• Trial has reached enrollment threshold for interim analysis ahead of schedule
• Expansion to second clinical site in France demonstrates trial progress
• Strong enrollment momentum at first site (Radboud University Medical Center)
• Clear timeline for interim analysis set for September 2025

Negative

• None.

San Diego, California--(Newsfile Corp. - June 17, 2025) - Thiogenesis Therapeutics, Corp. (TSXV: TTI) (OTCQX: TTIPF) ("Thiogenesis" or the "Company") a clinical-stage biopharmaceutical company developing sulfur-based therapeutics for serious pediatric and inherited mitochondrial diseases, today announced the recent activation of its French clinical site for Thiogenesis' ongoing Phase 2 clinical trial evaluating TTI-0102 in patients with Mitochondrial Encephalomyopathy with Lactic Acidosis and Stroke-like Episodes ("MELAS").

The first patient at Angers University Hospital Center ("CHU Angers") was dosed last week, marking a key expansion milestone in the Company's multicenter, randomized, double-blind, placebo-controlled MELAS Phase 2 clinical trial. This trial is designed to assess the safety, tolerability, PK/PD, and efficacy of oral TTI-0102 over a six-month treatment period.

The clinical trial is enrolling 12 MELAS patients across leading institutions in the Netherlands and France, with 8 patients receiving TTI-0102 and 4 receiving placebo. Driven by robust enrollment at Radboud University Medical Center in Nijmegen, Netherlands, the Company has already surpassed the threshold of patients required for a three-month interim analysis - now planned for September 2025.

"We are excited to activate our second clinical site and to accelerate our Phase 2 MELAS program with strong momentum," said Dr. Patrice Rioux, MD, Ph.D., Chief Executive Officer of Thiogenesis. "We are especially encouraged by the pace of enrollment and are looking forward to our interim analysis, which we anticipate will evaluate both safety and early signals of efficacy. This is a significant step toward our mission to deliver novel thiol-based drugs as therapies for patients with unmet medical needs in inherited mitochondrial disease."

Key clinical endpoints in the Phase 2 clinical trial includes the 12-Minute Walk Test ("12-MWT"), Fatigue Severity Scale ("FSS"), and the WHOQOL-BREF Quality of Life assessment - important indicators of functional and symptomatic improvement in this patient population.

About MELAS
Mitochondrial encephalomyopathy with lactic acidosis and stroke-like episodes ("MELAS") is an inherited mitochondrial disorder, most often caused by a mutation of m.3243A>G in the MT-TL1 gene in mitochondrial DNA. Initial symptoms usually include seizures, vomiting, headaches, muscle weakness, loss of appetite and fatigue. Longer term the disease may cause a loss of motor skills and intellectual disability. MELAS usually presents itself before the age of 20. Oxidative stress, including deficiencies in glutathione and taurine, play an important role in mitochondria dysfunction and are potential pathological mechanisms of mitochondrial disorders, making for viable targets for the treatment of MELAS and other mitochondrial diseases. Although it is one of the most prevalent inherited mitochondrial diseases, MELAS is still considered an orphan disease. There are estimated to be approximately 4.1/100,000 of the population (Ryytty et al. 2023) with MELAS worldwide.

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. Thiols are known to be precursors to important antioxidants such as glutathione and amino acids like taurine, providing the potential to restore mitochondrial function. The prodrug TTI-0102 was developed to address the challenges of first-generation thiol-based drugs, including their short half live, 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") and is planning clinical trials in Leigh syndrome, Rett syndrome and pediatric 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.

To view the source version of this press release, please visit https://www.newsfilecorp.com/release/255687

FAQ

What is the status of Thiogenesis (TTIPF) Phase 2 MELAS clinical trial?

The trial is actively enrolling patients across two sites in France and Netherlands, with the first French patient recently dosed at Angers University Hospital Center. The trial has already reached the threshold for interim analysis.

How many patients will be enrolled in Thiogenesis MELAS Phase 2 trial?

The trial will enroll 12 MELAS patients total, with 8 patients receiving TTI-0102 and 4 receiving placebo across institutions in the Netherlands and France.

When will Thiogenesis (TTIPF) release interim results for its MELAS trial?

The company plans to release the three-month interim analysis in September 2025, which will evaluate both safety and early efficacy signals.

What are the key endpoints in Thiogenesis Phase 2 MELAS trial?

The key clinical endpoints include the 12-Minute Walk Test (12-MWT), Fatigue Severity Scale (FSS), and the WHOQOL-BREF Quality of Life assessment.

What is TTI-0102 being developed for by Thiogenesis?

TTI-0102 is being developed for treating Mitochondrial Encephalomyopathy with Lactic Acidosis and Stroke-like Episodes (MELAS), a serious pediatric and inherited mitochondrial disease.