Research & Development
Polaryx Therapeutics wins FDA IND approval for PLX-200 to treat Late Infantile Neuronal Ceroid Lipofuscinosis
21 January 2020 -

Biotech company Polaryx Therapeutics revealed on Monday the receipt of US Food and Drug Administration (FDA) Investigational New Drug Application (IND) approval for PLX-200 for the treatment of LINCL (Late Infantile Neuronal Ceroid Lipofuscinosis or CLN2).

Neuronal Ceroid Lipofuscinoses are a group of rare autosomal recessive neurodegenerative lysosomal storage disorders. Among them, LINCL is caused by mutations leading deficiency or loss of function of tripeptidyl peptidase 1 (TPP1). Patients suffer from vision loss, severe seizures and declining motor function, leading to premature 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 reduces inflammation and prevents cell death (apoptosis).

According to the company, it has advanced a unique repurposing drug development strategy to provide patients with a safe and effective oral treatment option for LINCL.

Additionally, the company is developing its PLX-200 candidate to target this devastating disease and for securing approval from the FDA to proceed with clinical studies to assess the safety, tolerability and efficacy of PLX-200 for children with CLN2 disease.