Clinical Trials

Our Clinical Trials

Polaryx is approved to examine our lead candidate PLX-200, a novel and patient-friendly therapy, through two trials: a single pivotal trial for Late Infantile Neuronal Ceroid Lipofuscinosis (LINCL; CLN2); and a single pivotal trial for Juvenile Neuronal Ceroid Lipofuscinosis (JNCL, i.e., CLN3).

In preclinical models of both CLN2 and CLN3, PLX-200 has demonstrated the potential to treat the underlying factors associated with these conditions by decreasing lysosomal storage materials, blocking neuronal cell death, improving locomotor function, and increasing life span.​

Single Pivotal Trial For CLN2

Polaryx will perform a safety, tolerability, and efficacy study of PLX-200 in subjects with mild-to-moderate Neuronal Ceroid Lipofuscinosis Type 2 (CLN2) Disease. The main study will consist of a 96-week treatment period followed by a 30-day safety follow-up visit. PLX-200 represents a safe, superior, and excellent alternative to the currently available standard of care administered via an invasive, intraventricular infusion.

Single Pivotal Trial For CLN3

Polaryx will also perform a safety, tolerability, and efficacy study of PLX-200 in participants with mild-to-moderate Juvenile Neuronal Ceroid Lipofuscinosis (CLN3) Disease. There is currently no drug available to halt or delay CLN3 disease progression and the standard of care only manages symptoms. Polaryx has explored relevant drug candidates and identified PLX-200 to treat CLN3 disease. More information on the trial can be found at Clinicaltrials.gov NCT04637282.

If you need more information about the clinical trial, please contact Dr. Luis Rojas, M.Sc., Ph.D. at luis.rojas@polaryx.com.