Biotech company Polaryx Therapeutics reported on Tuesday the receipt of approval from the US Food and Drug Administration (FDA) for Investigational New Drug Application (IND) of PLX-200 for the treatment of Juvenile Neuronal Ceroid Lipofuscinosis (JNCL or CLN3).

JNCL or CLN3 is reportedly a rare and fatal genetic lysosomal storage disorder caused by a Cln3 gene mutation. CLN3 patients suffer from vision loss leading to blindness, seizures, progressive neurological deterioration, severe motor and cognitive declines and eventually death.

The company said PLX-200 is a repurposed drug that 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 activates PPARα, which enhances production of transcription factor EB (TFEB) in brain cells. PLX-200 also reduces inflammation and preventing cell death (apoptosis).

No drug is currently available to halt and/or delay the progression of the disease, concluded the company.