Thiogenesis Receives Final EU Clearance of CTA to Initiate its Phase 2 Clinical Trial in MELAS
Thiogenesis Therapeutics (TSXV: TTI) has received final EMA clearance for its Clinical Trial Application (CTA) Part II to begin a Phase 2 clinical trial of TTI-0102 for MELAS treatment. The trial will be conducted in France and the Netherlands as a randomized, double-blind, placebo-controlled study over 6 months.
The study will involve 12 patients total, with 8 receiving TTI-0102 and 4 receiving placebo. An interim analysis will be conducted after 3 months to assess safety data, pharmacokinetics/pharmacodynamics, and biomarkers. The trial's efficacy endpoints include the 12-Minute Walking Test, Fatigue Severity Scale, and Quality of Life Assessment.
TTI-0102 is designed to increase intracellular antioxidant glutathione and amino acid taurine levels, which are typically deficient in MELAS patients. Currently, there are no approved drugs for MELAS treatment.
Thiogenesis Therapeutics (TSXV: TTI) ha ricevuto l'approvazione finale dall'EMA per la sua domanda di sperimentazione clinica (CTA) Parte II per avviare una sperimentazione clinica di Fase 2 di TTI-0102 per il trattamento della MELAS. Lo studio sarà condotto in Francia e nei Paesi Bassi come uno studio randomizzato, in doppio cieco e controllato con placebo della durata di 6 mesi.
Lo studio coinvolgerà un totale di 12 pazienti, con 8 che riceveranno TTI-0102 e 4 che riceveranno un placebo. Un'analisi intermedia sarà condotta dopo 3 mesi per valutare i dati di sicurezza, la farmacocinetica/farmacodinamica e i biomarcatori. Gli endpoint di efficacia della sperimentazione includono il Test di Camminata di 12 Minuti, la Scala di Severità della Fatigue e la Valutazione della Qualità della Vita.
TTI-0102 è progettato per aumentare i livelli dell'antiossidante intracellulare glutathione e dell'amminoacido taurina, che sono tipicamente deficienti nei pazienti con MELAS. Attualmente non esistono farmaci approvati per il trattamento della MELAS.
Thiogenesis Therapeutics (TSXV: TTI) ha recibido la autorización final de la EMA para su Solicitud de Ensayo Clínico (CTA) Parte II, para iniciar un ensayo clínico de Fase 2 de TTI-0102 para el tratamiento de MELAS. El ensayo se llevará a cabo en Francia y los Países Bajos como un estudio aleatorizado, doble ciego y controlado con placebo durante 6 meses.
El estudio involucrará un total de 12 pacientes, con 8 recibiendo TTI-0102 y 4 recibiendo placebo. Se realizará un análisis intermedio después de 3 meses para evaluar los datos de seguridad, farmacocinética/farmacodinamia y biomarcadores. Los objetivos de eficacia del ensayo incluyen la Prueba de Caminata de 12 Minutos, la Escala de Severidad de la Fatiga y la Evaluación de la Calidad de Vida.
TTI-0102 está diseñado para aumentar los niveles del antioxidante intracelular glutatión y del aminoácido taurina, que suelen estar deficientes en los pacientes con MELAS. Actualmente, no existen medicamentos aprobados para el tratamiento de MELAS.
Thiogenesis Therapeutics (TSXV: TTI)는 MELAS 치료를 위한 TTI-0102의 2상 임상 시험을 시작하기 위해 임상 시험 신청(CTA) 2부에 대한 최종 EMA 승인을 받았습니다. 이 시험은 프랑스와 네덜란드에서 무작위 이중 맹검, 위약 대조 연구로 6개월 동안 진행될 예정입니다.
이 연구에는 총 12명의 환자가 참여하며, 8명은 TTI-0102를 받고 4명은 위약을 받을 것입니다. 3개월 후 안전성 데이터, 약물 동태학/약리학 및 바이오마커를 평가하기 위한 중간 분석이 수행됩니다. 시험의 효능 지표에는 12분 걷기 테스트, 피로 심각도 척도, 삶의 질 평가가 포함됩니다.
TTI-0102는 MELAS 환자에서 일반적으로 결핍된 세포내 항산화제 글루타티온 및 아미노산 타우린의 수치를 증가시키도록 설계되었습니다. 현재 MELAS 치료를 위한 승인된 약물이 없습니다.
Thiogenesis Therapeutics (TSXV: TTI) a reçu l'approbation finale de l'EMA pour sa demande d'essai clinique (CTA) Partie II afin de commencer un essai clinique de Phase 2 de TTI-0102 pour le traitement du MELAS. L'essai sera réalisé en France et aux Pays-Bas sous forme d'une étude randomisée, en double aveugle et contrôlée par placebo pendant 6 mois.
L'étude impliquera un total de 12 patients, avec 8 recevant TTI-0102 et 4 recevant un placebo. Une analyse intermédiaire sera effectuée après 3 mois pour évaluer les données de sécurité, la pharmacocinétique/pharmacodynamie et les biomarqueurs. Les critères d'efficacité de l'essai comprennent le Test de Marche de 12 Minutes, l'Échelle de Sévérité de la Fatigue et l'Évaluation de la Qualité de Vie.
TTI-0102 est conçu pour augmenter les niveaux de l'antioxydant intracellulaire glutathion et de l'acide aminé taurine, qui sont généralement déficients chez les patients atteints de MELAS. Actuellement, il n'existe aucun médicament approuvé pour le traitement du MELAS.
Thiogenesis Therapeutics (TSXV: TTI) hat die endgültige Genehmigung der EMA für seinen klinischen Prüfungsantrag (CTA) Teil II erhalten, um eine Phase-2-Studie zu TTI-0102 zur Behandlung von MELAS zu beginnen. Die Studie wird in Frankreich und den Niederlanden als randomisierte, doppelblinde, placebokontrollierte Studie über einen Zeitraum von 6 Monaten durchgeführt.
Die Studie umfasst insgesamt 12 Patienten, von denen 8 TTI-0102 erhalten und 4 ein Placebo. Nach 3 Monaten wird eine Zwischenanalyse durchgeführt, um Sicherheitsdaten, Pharmakokinetik/Pharmakodynamik und Biomarker zu bewerten. Die Wirksamkeitsendpunkte der Studie umfassen den 12-Minuten-Gehtest, die Fatigue-Skala und die Lebensqualitätsbewertung.
TTI-0102 soll die intrazellulären Antioxidantien Glutathion und die Aminosäure Taurin erhöhen, die bei MELAS-Patienten typischerweise defizitär sind. Derzeit gibt es keine zugelassenen Medikamente zur Behandlung von MELAS.
- Final regulatory clearance received from EMA for Phase 2 clinical trial
- Trial design completed with clear endpoints and measurement criteria
- TTI-0102 addresses specific deficiencies in MELAS patients with no current approved treatments
- Small trial size of only 12 patients may limit statistical significance
- 6-month trial duration may not be sufficient to demonstrate long-term efficacy
San Diego, California--(Newsfile Corp. - January 27, 2025) - Thiogenesis Therapeutics, and Corp. (TSXV: TTI) ("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 the European Medicines Agency ("EMA") has accepted its Clinical Trial Application ("CTA") Part II - National and Patient Level Documentation, for Thiogenesis' lead compound TTI-0102. It is the final regulatory clearance required for Thiogenesis to commence its Phase 2 clinical trial for the treatment of mitochondrial encephalomyopathy with lactic acidosis and stroke-like episodes ("MELAS"). The Company previously announced that its CTA Part I - Scientific and Medicinal Product Documentation, had been accepted by EMA.
Thiogenesis anticipates initiating the Phase 2 clinical trial in MELAS shortly, subject to the activation of the clinical trial sites.
The Phase 2 MELAS clinical trial is a multi-country, multi-center trial that will be conducted at leading institutions in France and the Netherlands. The trial is a randomized, double-blind, placebo-controlled study to assess the safety, tolerability, pharmacokinetics / pharmacodynamics and efficacy 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. There will be an interim analysis of safety data, pharmacokinetics / pharmacodynamics and biomarkers (cysteamine, glutathione, taurine etc.) after 3 months.
In addition to assessing several significant biomarkers, the efficacy endpoints for the clinical trial are:
12-Minute Walking Test ("12-MWT")
Fatigue Severity Scale ("FSS")
Quality of Life Assessment ("WHOQOL-BREF")
"We are thrilled to be nearing the dosing of a patient with MELAS," said Patrice Rioux, MD, Ph.D., Chief Executive Officer of Thiogenesis. "As a well-tolerated precursor to cysteamine, TTI-0102 is engineered to intracellularly increase the antioxidant glutathione and the amino acid taurine, both of which are well understood to be deficient in MELAS patients. As a result, TTI-0102 has the potential to be a viable therapeutic compound for the treatment of MELAS, for which there is no currently approved drugs."
About MELAS
Mitochondrial encephalomyopathy with lactic acidosis and stroke-like episodes ("MELAS") is a rare, 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 and taurine deficiency play an important role in mitochondria and are potential pathological mechanisms of mitochondrial disorders, making for viable targets for the treatment of MELAS and other mitochondrial diseases. MELAS is an orphan disease, there are estimated to be approximately 4.4/100,000 patients with MELAS relative to the population (Chakrabarty S, et al.2020).
About TTI-0102
Thiogenesis' lead compound, 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 for several therapeutic applications. 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 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 US, and its equivalent hybrid system in Europe, 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. The Company's initial target indications include Mitochondrial Encephalopathy Lactic Acidosis and Stroke ("MELAS"), Leigh's 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) 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/238463
FAQ
What is the purpose of Thiogenesis' Phase 2 MELAS trial for TTI-0102 (TTIPF)?
How many patients will be enrolled in Thiogenesis' TTIPF Phase 2 MELAS trial?
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When will Thiogenesis (TTIPF) conduct the interim analysis for the MELAS trial?
Where will Thiogenesis conduct its Phase 2 MELAS trial for TTI-0102 (TTIPF)?
