Thiogenesis Receives Confirmatory Guidance on IMPD for Pediatric MASH Phase 2a Clinical Trial in Europe and Core Patent Granted in EU
Thiogenesis Therapeutics (OTCQX: TTIPF) received positive guidance from the EMA's CHMP supporting their IMPD submission for a Phase 2a clinical trial of TTI-0102 in pediatric MASH patients. The trial will evaluate the company's third-generation disulfide prodrug of cysteamine in children aged 10-17 with biopsy-confirmed MASH.
The open-label, single-arm trial will assess safety, tolerability, pharmacokinetics, and pharmacodynamic effects, focusing on changes in liver enzymes and mitochondrial biomarkers. Additionally, Thiogenesis secured a crucial European Union patent for its asymmetric disulfide prodrugs of cysteamine, extending protection until 2038.
Thiogenesis Therapeutics (OTCQX: TTIPF) ha ricevuto un parere positivo dal CHMP dell'EMA a supporto della loro domanda IMPD per uno studio clinico di Fase 2a su TTI-0102 in pazienti pediatrici con MASH. Lo studio valuterà il prodrug disolfuro di terza generazione della cisteamina sviluppato dall'azienda in bambini di età compresa tra 10 e 17 anni con MASH confermato da biopsia.
Lo studio, in aperto e a braccio singolo, analizzerà sicurezza, tollerabilità, farmacocinetica ed effetti farmacodinamici, concentrandosi sui cambiamenti degli enzimi epatici e dei biomarcatori mitocondriali. Inoltre, Thiogenesis ha ottenuto un importante brevetto nell'Unione Europea per i suoi prodrug disolfuro asimmetrici della cisteamina, estendendo la protezione fino al 2038.
Thiogenesis Therapeutics (OTCQX: TTIPF) recibió una orientación positiva del CHMP de la EMA que respalda su presentación IMPD para un ensayo clínico de Fase 2a de TTI-0102 en pacientes pediátricos con MASH. El ensayo evaluará el profármaco disulfuro de tercera generación de cisteamina de la compañía en niños de 10 a 17 años con MASH confirmado por biopsia.
El ensayo abierto y de un solo brazo evaluará la seguridad, tolerabilidad, farmacocinética y efectos farmacodinámicos, enfocándose en los cambios en las enzimas hepáticas y biomarcadores mitocondriales. Además, Thiogenesis aseguró una patente clave en la Unión Europea para sus profármacos disulfuro asimétricos de cisteamina, extendiendo la protección hasta 2038.
Thiogenesis Therapeutics (OTCQX: TTIPF)는 EMA의 CHMP로부터 소아 MASH 환자를 대상으로 한 TTI-0102 2a상 임상시험 IMPD 제출에 대한 긍정적인 지침을 받았습니다. 이 임상시험은 생검으로 확인된 MASH를 가진 10~17세 어린이들을 대상으로 회사의 3세대 시스테아민 이황화 프로드럭을 평가할 예정입니다.
이 공개라벨 단일군 시험은 안전성, 내약성, 약동학 및 약력학적 효과를 평가하며, 간 효소와 미토콘드리아 바이오마커의 변화를 중점적으로 살펴봅니다. 또한 Thiogenesis는 비대칭 이황화 시스테아민 프로드럭에 대해 중요한 유럽 연합 특허를 확보하여 2038년까지 보호를 연장했습니다.
Thiogenesis Therapeutics (OTCQX: TTIPF) a reçu un avis favorable du CHMP de l'EMA soutenant sa soumission IMPD pour un essai clinique de phase 2a de TTI-0102 chez des patients pédiatriques atteints de MASH. L'essai évaluera le prodrogue disulfure de troisième génération de cystéamine de la société chez des enfants âgés de 10 à 17 ans avec un MASH confirmé par biopsie.
L'essai ouvert, en simple bras, évaluera la sécurité, la tolérance, la pharmacocinétique et les effets pharmacodynamiques, en se concentrant sur les modifications des enzymes hépatiques et des biomarqueurs mitochondriaux. De plus, Thiogenesis a obtenu un brevet clé dans l'Union européenne pour ses prodrogues disulfures asymétriques de cystéamine, prolongeant la protection jusqu'en 2038.
Thiogenesis Therapeutics (OTCQX: TTIPF) erhielt eine positive Empfehlung vom CHMP der EMA zur Unterstützung ihrer IMPD-Einreichung für eine Phase-2a-Studie mit TTI-0102 bei pädiatrischen MASH-Patienten. Die Studie wird das disulfidische Prodrug der dritten Generation von Cysteamins bei Kindern im Alter von 10 bis 17 Jahren mit bioptisch bestätigtem MASH evaluieren.
Die offene, einarmige Studie wird Sicherheit, Verträglichkeit, Pharmakokinetik und pharmakodynamische Effekte untersuchen, mit Fokus auf Veränderungen der Leberenzyme und mitochondrialer Biomarker. Zusätzlich sicherte sich Thiogenesis ein wichtiges Patentrecht in der Europäischen Union für seine asymmetrischen Disulfid-Prodrugs von Cysteamins, das den Schutz bis 2038 verlängert.
- EMA acknowledgment of high unmet medical need in pediatric MASH population
- Expansion of TTI-0102 clinical program to include pediatric MASH in Europe
- EU patent granted for cysteamine prodrugs, providing protection until 2038
- Strategic advancement across multiple pediatric conditions (MELAS, Leigh syndrome, MASH)
- None.
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
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.
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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