Nephropathic cystinosis is a rare, autosomal recessive disorder caused by CTNS gene mutations, leading to toxic cystine buildup inside cells. Diagnosed in infancy, the disease is fatal in childhood if untreated; treated, it becomes a lifelong condition where consistent dosing of cysteamine-based drugs helps patients function normally and preserves their vulnerable kidneys.

The current standard of care, Procysbi®, consists of delayed-release, enteric-coated cysteamine capsules, dosed twice daily and associated with GI side effects and a pervasive sulfur body odor. That burden drives real non-adherence, and missed doses let toxic cystine spike, resuming organ damage. TTI-0102 is a next-generation cysteamine prodrug built to remove that burden: a sustained-release oral powder with no enteric coating, with the potential for once-daily dosing and improved tolerability.

There are an estimated 2,000–2,500 patients worldwide, an addressable market of up to ~$350 million annually. Development rests on established precedent, WBC cystine depletion, the surrogate endpoint that approved Procysbi® in 2012, is the basis for TTI-0102’s own plan. The Company is advancing an investigator-initiated study at Emory University under Dr. Larry Greenbaum to generate patient proof-of-concept data ahead of a planned pivotal Phase 3 trial.

Leigh Syndrome (“LS”) is a rare, inherited mitochondrial disorder that typically begins in infancy or early childhood, caused by mutations in mitochondrial or nuclear genes that impair cellular energy production. Symptoms include developmental regression, muscle weakness, and breathing difficulties, with characteristic brain lesions visible on MRI. The condition is progressive and often life-threatening; there are currently no approved therapies for LS.

TTI-0102’s rationale in LS rests on sustained cysteamine exposure supporting glutathione production, the antioxidant most severely deficient in Leigh patients among mitochondrial disease populations studied, while also increasing taurine and coenzyme A. The Company holds an FDA-cleared IND for a Phase 2a trial evaluating TTI-0102 in LS with a leading children’s hospital in the US, planned to start in 2026.

On July 13, 2026, the FDA granted TTI-0102 Rare Pediatric Disease designation for Leigh Syndrome, providing the potential for a Priority Review Voucher upon future approval — historically a valuable strategic asset for biotechnology companies. Classical Leigh Syndrome affects approximately 1 in 40,000 newborns; a positive result would extend TTI-0102’s relevance to the broader family of primary mitochondrial diseases, affecting an estimated 75,000 patients in the US.
‍

Mitochondrial encephalomyopathy, lactic acidosis, and stroke-like episodes (MELAS) is a progressive
mitochondrial disorder typically diagnosed in childhood or adolescence, causing seizures, muscle weakness,
fatigue, and stroke-like episodes. It is part of the broader family of primary mitochondrial diseases (PMD),
which together affect an estimated 75,000 patients in the US. There are currently no approved treatments.

The Company’s Phase 2 study of TTI-0102 in MELAS, initiated in the EU in May 2025, is now complete. The
exploratory study characterized how drug exposure varies with body weight and tolerability, and showed
biomarker changes consistent with reduced oxidative stress, findings informing dosing in the Leigh syndrome
and cystinosis programs.

Pediatric MASH (Metabolic dysfunction-associated steatohepatitis) is a serious, escalating liver disease in children marked by fat accumulation,
inflammation, and fibrosis. It is the advanced stage of MASLD, closely linked to metabolic syndrome, with oxidative stress central to its
progression.

TTI-0102 has the potential to restore redox balance by boosting intracellular cysteine and glutathione, and may reduce liver scarring by inhibiting hepatic stellate cell (“HSC”) activation. There are currently no approved treatments for pediatric MASH. The Company has received EMA guidance supporting an Investigational Medicinal Product Dossier (“IMPD”) submission for a Phase 2a trial in Europe.