Thiogenesis Therapeutics Announces Investigator-Initiated Study in Nephropathic Cystinosis and Provides Program Update

Executive Summary

• Thiogenesis Therapeutics announced a new investigator‑initiated study (IIS) with Dr. Larry Greenbaum at Emory University/Children’s Healthcare of Atlanta to evaluate lead candidate TTI‑0102 in nephropathic cystinosis patients.
• The IIS will generate dosing, tolerability, and white‑blood‑cell cystine control data to support dose optimization for the planned Phase 3 pivotal trial.
• Thiogenesis is scaling up manufacturing of a newly patented salt formulation of TTI‑0102 and preparing IND filing work to enable the upcoming Phase 3 non‑inferiority study under FDA 505(b)(2) pathway.

Key Details

• Study Collaboration: Dr. Larry Greenbaum (Chief of Pediatric Nephrology, Children’s Healthcare of Atlanta; Professor, Emory University) will lead the IIS evaluating TTI‑0102 in a representative cystinosis patient cohort.
• Study Objectives: Assess once‑daily dosing feasibility, tolerability, and WBC cystine control; data to inform dose optimization for Phase 3 program.
• Candidate Profile (TTI‑0102):
• Novel cysteamine‑based disulfide prodrug delivering sustained exposure with lower peak plasma levels.
• Designed for once‑daily oral dosing, targeting ~50 % of daily cysteamine base dose used by existing therapies.
• Demonstrated 24‑hour sustained exposure, reduced GI adverse events, and dual activity (cystine depletion + antioxidant pathways).
• Manufacturing & Regulatory Progress:
• Initiating scale‑up of a newly patented salt formulation required for sufficient clinical material and formal stability testing.
• Stability testing to run in parallel with finalization of IND application for cystinosis.
• Phase 3 Plan:
• Non‑inferiority pivotal trial versus an approved cysteamine therapy under FDA 505(b)(2) pathway.
• Primary endpoint: WBC cystine concentration (validated surrogate).
• Secondary endpoints: simplified dosing, improved tolerability, patient‑reported outcomes.
• Market Context: Nephropathic cystinosis affects ~2,000–2,500 patients worldwide; market opportunity > $300 M. Current therapies limited by frequent dosing and GI intolerance.

Notable Quotes

“This investigator‑initiated study allows us to evaluate TTI‑0102 in a real‑world cystinosis population under the leadership of one of the most experienced investigators in the field,” said Dr. Patrice Rioux, CEO of Thiogenesis Therapeutics.

All forward‑looking statements are subject to risks and uncertainties; actual results may differ.