Business
Thiogenesis Receives Confirmatory Guidance on IMPD for Pediatric MASH Phase 2a Clinical Trial in Europe and Core Patent Granted in EU
San Diego, California--(Newsfile Corp. - June 25, 2025) - Thiogenesis Therapeutics, Inc. (TSXV: ...
About this update from Thiogenesis Therapeutics Corp
[{"type":"text","content":"Thiogenesis Receives Confirmatory Guidance on IMPD for Pediatric MASH Phase 2a Clinical Trial in Europe and Core Patent Granted in EUSan 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 i...