Thiogenesis Initiates Phase 2 Clinical Trial in Mitochondrial Encephalomyopathy with Lactic Acidosis and Stroke-like Episodes ("MELAS")
Thiogenesis Therapeutics (OTCQX: TTIPF) has initiated its Phase 2 clinical trial for TTI-0102, its lead product candidate targeting MELAS (Mitochondrial Encephalomyopathy with Lactic Acidosis and Stroke-like Episodes). The first two patients were dosed on May 12th at Radboud University Medical Center in Netherlands.
The multi-center trial will be conducted in the Netherlands and France, enrolling 12 patients total (8 receiving TTI-0102, 4 receiving placebo) in a randomized, double-blind, placebo-controlled study over 6 months. Key clinical endpoints include the 12-Minute Walking Test, Fatigue Severity Scale, and Quality of Life Assessment, with an interim analysis planned at 3 months.
TTI-0102 is designed to increase intracellular antioxidant glutathione and amino acid taurine, both deficient in MELAS patients. Currently, there are no approved drugs for MELAS in the EU or U.S.
Thiogenesis Therapeutics (OTCQX: TTIPF) ha avviato la fase 2 della sperimentazione clinica per TTI-0102, il suo principale candidato farmaco destinato a trattare la MELAS (Encefalomiopatia Mitocondriale con Acidosi Lattica ed Episodi simili a Ictus). I primi due pazienti sono stati trattati il 12 maggio presso il Radboud University Medical Center nei Paesi Bassi.
Lo studio multicentrico si svolgerà nei Paesi Bassi e in Francia, arruolando 12 pazienti in totale (8 riceveranno TTI-0102, 4 il placebo) in uno studio randomizzato, in doppio cieco e controllato con placebo della durata di 6 mesi. Gli endpoint clinici principali includono il Test di Camminata di 12 Minuti, la Scala di Gravità della Fatica e la Valutazione della Qualità della Vita, con un'analisi intermedia prevista dopo 3 mesi.
TTI-0102 è progettato per aumentare i livelli intracellulari di glutatione antiossidante e taurina, un amminoacido entrambi carenti nei pazienti con MELAS. Attualmente non esistono farmaci approvati per la MELAS né nell'UE né negli Stati Uniti.
Thiogenesis Therapeutics (OTCQX: TTIPF) ha iniciado su ensayo clínico de fase 2 para TTI-0102, su principal candidato a medicamento dirigido a MELAS (Encefalomiopatía Mitocondrial con Acidosis Láctica y Episodios similares a un Accidente Cerebrovascular). Los primeros dos pacientes recibieron la dosis el 12 de mayo en el Radboud University Medical Center en los Países Bajos.
El ensayo multicéntrico se llevará a cabo en los Países Bajos y Francia, inscribiendo a 12 pacientes en total (8 recibirán TTI-0102, 4 recibirán placebo) en un estudio aleatorizado, doble ciego y controlado con placebo durante 6 meses. Los principales puntos finales clínicos incluyen la Prueba de Caminata de 12 Minutos, la Escala de Severidad de Fatiga y la Evaluación de la Calidad de Vida, con un análisis intermedio planificado a los 3 meses.
TTI-0102 está diseñado para aumentar el glutatión antioxidante intracelular y el aminoácido taurina, ambos deficientes en pacientes con MELAS. Actualmente, no existen medicamentos aprobados para MELAS en la UE ni en EE. UU.
Thiogenesis Therapeutics (OTCQX: TTIPF)는 MELAS(젖산산증 및 뇌졸중 유사 증상을 동반한 미토콘드리아 뇌병증)를 표적으로 하는 주력 후보물질 TTI-0102에 대한 2상 임상시험을 시작했습니다. 첫 두 환자는 5월 12일 네덜란드 라드바우드 대학교 의료센터에서 투여를 받았습니다.
이 다기관 임상시험은 네덜란드와 프랑스에서 진행되며, 총 12명의 환자(8명은 TTI-0102, 4명은 위약)를 무작위 배정, 이중 맹검, 위약 대조 방식으로 6개월 동안 등록할 예정입니다. 주요 임상 평가 지표로는 12분 걷기 검사, 피로 심각도 척도, 삶의 질 평가가 포함되며, 3개월 시점에 중간 분석이 계획되어 있습니다.
TTI-0102는 MELAS 환자에게 결핍된 세포 내 항산화제 글루타티온과 아미노산 타우린을 증가시키도록 설계되었습니다. 현재 EU나 미국에서는 MELAS 치료제로 승인된 약물이 없습니다.
Thiogenesis Therapeutics (OTCQX: TTIPF) a lancé son essai clinique de phase 2 pour TTI-0102, son principal candidat médicament ciblant la MELAS (encéphalomyopathie mitochondriale avec acidose lactique et épisodes de type AVC). Les deux premiers patients ont été traités le 12 mai au Radboud University Medical Center aux Pays-Bas.
L'essai multicentrique se déroulera aux Pays-Bas et en France, recrutant 12 patients au total (8 recevant TTI-0102, 4 recevant un placebo) dans le cadre d'une étude randomisée, en double aveugle, contrôlée par placebo sur 6 mois. Les principaux critères cliniques incluent le test de marche de 12 minutes, l'échelle de gravité de la fatigue et l'évaluation de la qualité de vie, avec une analyse intermédiaire prévue à 3 mois.
TTI-0102 est conçu pour augmenter le glutathion antioxydant intracellulaire et l'acide aminé taurine, tous deux déficients chez les patients atteints de MELAS. Actuellement, aucun médicament n'est approuvé pour la MELAS ni dans l'UE ni aux États-Unis.
Thiogenesis Therapeutics (OTCQX: TTIPF) hat seine Phase-2-Studie für TTI-0102, den führenden Produktkandidaten zur Behandlung von MELAS (Mitochondriale Enzephalomyopathie mit Laktatazidose und Schlaganfall-ähnlichen Episoden), gestartet. Die ersten zwei Patienten wurden am 12. Mai im Radboud University Medical Center in den Niederlanden dosiert.
Die multizentrische Studie wird in den Niederlanden und Frankreich durchgeführt und umfasst insgesamt 12 Patienten (8 erhalten TTI-0102, 4 Placebo) in einer randomisierten, doppelblinden, placebokontrollierten Studie über 6 Monate. Wichtige klinische Endpunkte sind der 12-Minuten-Gehtest, die Fatigue-Schweregrad-Skala und die Lebensqualitätsbewertung, mit einer geplanten Zwischenanalyse nach 3 Monaten.
TTI-0102 ist darauf ausgelegt, das intrazelluläre Antioxidans Glutathion und die Aminosäure Taurin zu erhöhen, die bei MELAS-Patienten beide vermindert sind. Derzeit gibt es keine zugelassenen Medikamente für MELAS in der EU oder den USA.
- First patients dosed in Phase 2 trial for TTI-0102, targeting an unmet medical need
- Trial design includes interim analysis at 3 months, potentially accelerating development timeline
- Addressing MELAS, which has no approved treatments in EU or US, representing a significant market opportunity
- Small trial size of only 12 patients may limit statistical significance
- Early-stage clinical trial with no guarantee of success
- Multi-country trial coordination could potentially lead to delays or complications
San Diego, California--(Newsfile Corp. - May 14, 2025) - Thiogenesis Therapeutics, Corp. (TSXV: TTI) (OTCQX: TTIPF) ("Thiogenesis" or the "Company") a clinical-stage biotechnology company developing disulfides that drive the production of critically important intracellular antioxidants and other therapeutic compounds, today announced that it has dosed its first two patients in its Phase 2 clinical trial of TTI-0102, Thiogenesis' lead product candidate for the potential treatment of the inherited mitochondrial disease MELAS. The first two patients were dosed on May 12th, at the Radboud University Medical Center in Nijmegen, Netherlands, where the Phase 2 clinical trial has been activated and is currently screening and recruiting patients.
The Company's Phase 2 MELAS clinical trial is a multi-country, multi-center trial that will be conducted at leading institutions in the Netherlands and France. The trial is a randomized, double-blind, placebo-controlled study to assess the safety, tolerability, efficacy, and pharmacokinetics / pharmacodynamics of oral TTI-0102 for the treatment of patients with MELAS over a 6-month period. The trial will enroll a total of 12 patients, 8 patients will receive TTI-0102, and 4 patients will receive placebo. After 3 months, there will be an interim analysis of safety and clinical efficacy.
The significant clinical endpoints being assessed in the clinical trial will include:
- A 12-Minute Walking Test ("12-MWT")
- Fatigue Severity Scale ("FSS")
- Quality of Life Assessment ("WHOQOL-BREF")
"The dosing of the first two patients in our Phase 2 clinical trial is the culmination of a lot of planning and dedicated work from the Thiogenesis team and a major milestone for an emerging biotech company," said Patrice Rioux, MD, Ph.D., Chief Executive Officer of Thiogenesis. "We are excited to test our lead product candidate TTI-0102 in MELAS, and bring a potential treatment to one of the most prevalent and debilitating of the inherited mitochondrial diseases for which there are no approved drugs in the EU or the U.S. TTI-0102 is a precursor to the thiol cysteamine and has been engineered to be well-tolerated and to intracellularly increase the antioxidant glutathione and the amino acid taurine, both of which are known to be deficient in MELAS patients and contribute to the disease."
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 glutathione and taurine deficiency, 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.
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.
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FAQ
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