Key Mechanisms for TTI's Lead Product Key Mechanisms for Thiogenesis Therapeutics' (TTI.v) Lead Product Targeting Unmet Medical Needs Capitalizing on a $9.8 billion market opportunity within the global rare disease and antioxidant therapies space, Thiogenesis Therapeutics (TTI.v) is focused on the development of thiol-based drugs with its lead product TTI-0102, targeting unmet medical needs including rare conditions such as MELAS, Leigh Syndrome, Rett Syndrome, and Pediatric MASH.
Regulatory Pathways
- TTI-0102, a prodrug, will use the expedited 505(b)(2) pathway in the US and its European equivalent hybrid pathway.
- These pathways allow referencing prior clinical safety data (e.g., from Cystagon®), reducing the time and cost needed to progress to human efficacy trials.
MELAS (Mitochondrial Encephalomyopathy, Lactic Acidosis, and Stroke-Like Episodes)
- Prevalence: Affects 1 in 10,000 people, with an estimated 30,000 patients in the US.
- Symptoms: Neurological impairment, seizures, stroke-like episodes, fatigue, and weakness.
- Current Status: No approved treatments, representing a significant unmet medical need.
- TTI-0102 Mechanisms: Restores mitochondrial oxidative balance & acts as an anti-oxidant and precursor to taurine and glutathione.
Rett Syndrome
- Prevalence: Primarily affects girls, with 1 in 10,000 impacted and an estimated 25,000 cases in Europe.
- Symptoms: Slowed growth, motor skill loss, seizures, and abnormal hand movements linked to decreased Brain Derived Neurotropic Factor (BDNF).
- Current Status: Symptom-focused therapies only; no approved treatment in Europe.
- TTI-0102 Mechanisms: Promotes BDNF increase for nerve cell health and communication & enhances mitochondrial oxidative balance and acts as an anti-oxidant.
NASH (Non-Alcoholic Steatohepatitis)
- Prevalence: Approximately 7 million children in the US with pediatric NASH.
- Symptoms: Liver inflammation and damage leading to cirrhosis, liver failure, or cancer.
- Current Status: No approved treatments; Thiogenesis targets pediatric NASH as its initial focus.
- TTI-0102 Mechanisms: Anti-inflammatory and anti-oxidant properties & restores healthy oxidative balance in mitochondria.
TTI-0102 leverages its proven safety profile (from cysteamine's pediatric use) to target critical unmet medical needs in MELAS, Rett Syndrome, and NASH. By addressing mitochondrial dysfunction through innovative mechanisms, Thiogenesis presents a promising therapeutic approach for these challenging conditions.
Source:
https://thiogenesis.webflow.io/pipeline Deep Dive:
https://www.reddit.com/r/wallstreetsmallcaps/comments/1hxs2xe/biotech_thiogenesis_therapeutics_ttiv_advances/ Posted on Behalf of Thiogenesis Therapeutics Corp.