Thiogenesis Therapeutics Inc.

(TSXV: TTI | OTCQB: TTIPF)

A clinical-stage biopharmaceutical company developing next-generation, sulfur-based therapeutics, classified as thiols, to address oxidative stress and inflammation in inherited and acquired mitochondrial diseases.
TTI-0102, a novel cysteamine prodrug, is designed to replace a burdensome daily regimen of multiple capsules with oral dosing in a powder format; potentially improving tolerability, bioavailability, and dosing burden relative to legacy cysteamine therapies (which are thiols). TTI-0102 has been well tolerated at high doses in Phase 1 testing.

With an experienced leadership team in orphan drug development, the Company follows a capital-efficient strategy:

• 505(b)(2) regulatory pathway in the U.S.
• Small, focused human efficacy trials
• Substantial addressable orphan markets with unmet need

TTI-0102 is in late-stage development for nephropathic cystinosis, a rare, life-threatening lysosomal disease with a validated regulatory pathway and an established commercial precedent, building on an investigator-initiated study ahead of a planned Phase 3 IND filing.

Beyond cystinosis, the Company holds an FDA-cleared IND for Leigh syndrome spectrum, a rare pediatric mitochondrial disease with no approved therapies, and will be starting an important Phase 2 clinical trial in the indication with a leading U.S. children's hospital in 2026.

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