Our core technology is unique and has defined mechanisms of action.
PLX-200 binds to the retinoid X receptor-α (RXRα) which binds to PPARα thereby up-regulating the expression of TPP1 mRNA in brain cells via the PPARα/RXRα heterodimer. PLX-200 also activates PPARα which enhances production of transcription factor EB (TFEB) in brain cells. TFEB then binds to the promoter of genes involved in lysosome biogenesis and activates their production. TFEB can regulate lysosomes due to its effects on the expression of lysosomal genes.
Our treatment potentially prolongs lifespan, delays loss of mobility, and increases certain genes involved in anti-inflammation and in anti-apoptosis in CLN2/TPP1 deficient animals.
Our treatment potentially prolongs lifespan, delays loss of mobility, and protects patient quality of life.
PLX-100 is composed of both PLX-200 and all trans retinoic acid.
PLX-100 lowers the doses of both compounds for boosting TPP1 mRNA expression in patients with a residual copy of the CLN2 gene and it enhances lysosome biogenesis in brain cells.
PLX-100 also prolongs the lifespan of CLN2 deficient animals, delays the loss of mobility, and increases certain genes involved in anti-inflammation and in anti-apoptosis.
Taken together, the neuroprotective effects together with the potential to increase both TPP1 expression and lysosome biogenesis strongly suggest that our drug candidates may have a benefit for early stage patients and possibly for other NCL forms, such as infantile and juvenile NCL.
Our drug candidates could also be used alone or combination with some of the emerging treatments for this devastating diseases.