Treating the Untreatable

OUR SCIENCE

TTX targets two critical pathways that drive brain diseases: the NLRP3 Inflammasome (which triggers destructive inflammation) and the Integrated Stress Response (which determines cell survival). By modulating both pathways simultaneously, we are setting out to address the root causes of neurodegeneration and brain cancer.

NLRP3 Inflammasome

The NLRP3 inflammasome is a cellular danger sensor that triggers inflammation when activated. In chronic diseases, it becomes overactive, creating destructive inflammation that damages brain tissue and promotes tumor growth.

In neurodegeneration, persistent NLRP3 activation accelerates protein aggregation, damages neurons, and triggers neuroinflammation in Alzheimer's, Parkinson's, and multiple sclerosis. In brain cancer, it creates an immunosuppressive tumor microenvironment that helps tumors evade immune detection.

TTX compounds directly bind to the NLRP3 protein, disrupting inflammasome assembly and blocking the inflammatory cascade at its source.

Integrated Stress Response (ISR)

The ISR is a cellular adaptation system that helps cells survive stress from protein misfolding, nutrient loss, and oxidative damage. In disease, this system becomes dysregulated.

In neurodegeneration, the ISR becomes hyperactive, triggering excessive stress signaling that can damage neurons rather than protect them. Cancer cells exploit the ISR to adapt to hostile tumor microenvironments, resist therapy, and maintain aggressive growth.

TTX compounds modulate the ISR by activating protective protein clearance mechanisms, boosting cellular antioxidant defenses, and preventing inflammatory gene expression. This protects neurons while disrupting cancer cell survival mechanisms.

TTX-01 modulates the ISR through three mechanistic actions:

TTX-01 restores balance to the stress response through three coordinated actions:

  • Activates Nrf2: Boosts cellular antioxidant defenses

  • Activates UPR: Clears toxic proteins that accumulate in disease

  • Suppresses NF-κB: Reduces chronic inflammation

The Synergistic Advantage

These mechanisms work together by reducing inflammation while enhancing cellular protection and protein clearance and addressing multiple disease drivers with a single therapeutic.