TTI–0102 Pipeline

Indications
Preclinical
Phase 1
(Australia)
Phase 2
Phase 3
MELAS
Phase 2
Phase 1
Leigh Syndrome
Phase 2
Phase 1
MASH
(Pediatric)
Phase 2
Phase 1
Rett Syndrome
Phase 1
Phase 1

Regulatory

The Food and Drug Administration (FDA) and the European Medicines Agency (EMA) will govern Thiogenesis’ clinical trials in their respective jurisdictions. As a prodrug, TTI-0102 is eligible to use the expedited 505(b)(2) regulatory pathway in the US and its equivalent hybrid pathway in Europe. The 505(b)(2) pathway provides certain regulatory advantages, as some of the clinical safety data from the active compound may be provided by referencing previous studies not affiliated with Thiogenesis (in this case, safety data from Cystagon®), which can save substantial time and money in progressing to human efficacy trials.

MELAS

MELAS- Trial

Mitochondrial encephalomyopathy, lactic acidosis, and stroke-like episodes (MELAS) is a progressive, multisystem disorder that typically begins in childhood or adolescence. Caused by mutations in mitochondrial DNA—most often m.3243A>G in the MT-TL1 gene—it leads to seizures, muscle weakness, hearing loss, fatigue, and stroke-like episodes.

Despite its severity and an estimated prevalence of 4.1 per 100,000 people, there are currently no approved treatments for MELAS. A Phase 2 clinical trial in the EU was initiated on May 14, 2025.

TTI-0102 Key Mechanisms of Action

  • Precursor to glutathione
  • Metabolizes into taurine
  • Increases the production of CoA

About Leigh Syndrome Spectrum

LSS - Trial


Leigh syndrome spectrum (“LSS”) is a rare, inherited mitochondrial disorder that typically begins in infancy or early childhood. It is caused by mutations in either mitochondrial DNA or nuclear genes that impair energy production in cells. Symptoms often include developmental delay, loss of motor skills, muscle weakness, breathing difficulties, and lesions in the brainstem and basal ganglia visible on MRI. The condition is progressive and often life-threatening.

The estimated prevalence of LSS is approximately 1 in 40,000 newborns. There are currently no approved therapies for LSS.  The Company has received FDA clearance to initiate a Phase 2a clinical trial in the U.S. for the treatment of LSS with TTI-0102.

TTI-0102 Key Mechanisms of Action

  • Precursor to glutathione
  • Metabolizes into taurine
  • Increases the production of CoA

About Pediatric MASH

MASH - Trial


Pediatric MASH (Metabolic dysfunction-associated steatohepatitis) is a serious liver disease in children that is rapidly escalating; marked by fat accumulation, inflammation, and fibrosis. It is the advanced stage of MASLD (Metabolic dysfunction-associated liver disease) and is closely linked to metabolic syndrome. Oxidative stress plays a central role in its progression by disrupting the balance between reactive oxygen species (ROS) and the liver’s antioxidant defenses.

TTI‑0102 has the potential to restore redox balance by boosting intracellular cysteine and glutathione. It may also reduce liver scarring by inhibiting hepatic stellate cell (“HSC”) activation, which drives fibrosis. There are currently no approved treatments for pediatric MASH. Thiogenesis plans to submit an Investigational Medicinal Product Dossier (IMPD) in 2025 to initiate a Phase 2 clinical trial in Europe.

TTI-0102 Key Mechanisms of Action

  • Precursor to glutathione
  • Blocks HSC proliferation and fibrosis
  • Restores mitochondrial redox balance

About Rett Syndrome

Rett syndrome (RS) is a rare neurodevelopmental disorder that primarily affects young girls and is caused by mutations in the MECP2 gene, which plays a critical role in brain development. RS is characterized by slowed growth, loss of motor and communication skills, seizures, and distinct repetitive hand movements.

In addition to mitochondrial oxidative stress, a hallmark of the disease is the reduced expression of brain-derived neurotrophic factor (BDNF)—a protein essential for neuron development, synaptic connectivity, and cell survival. Thiogenesis has received Orphan Drug Designation for TTI-0102 in Rett syndrome in the European Union.

    TTI-0102 Key Mechanisms of Action

  • Promotes the increase of BDNF
  • Maintains the thiol/disulfide balance, and healthy oxidative status in the mitochondria
  • Precursor to glutathione
  • Metabolizes into taurine 

Coronaviruses

Coronaviruses are abundant in nature and normally cause mild to moderate upper-respiratory infections. In the last 20 years there has been an increase in coronaviruses that have been much more impactful on humans. COVID-19 is caused by a novel coronavirus identified in Wuhan, China in 2020, the SARS-CoV-2 virus. It can trigger extreme respiratory tract infection, cytokine storm and cytokine release syndrome. Previous coronaviruses that have jumped from animals to humans include SARS (severe acute respiratory syndrome) in 2002 and MERS (Middle East respiratory syndrome) in 2012.

Thiogenesis is in the process of submitting an IND application with FDA for human efficacy trials in coronaviruses, with TTI-0102, including COVID-19 and its variants. The Company is also evaluating partnering opportunities for SARS and other infectious disease applications, both in the US and internationally.

TTI-0102 Key Mechanisms of Action - Coronaviruses

  • Antiviral, binds to ACE2 receptor
  • Anti-oxidant, precursor to glutathione
  • Anti-inflammatory
  • Mucolytic, mucus thinning