Clinical Trials

Our Clinical Trials

Polaryx is approved to examine our lead candidate PLX-200, a novel and patient-friendly therapy, through three trials: a Ph.2, open-label, proof-of-concept basket trial for CLN2, CLN3, Krabbe Disease and Sandhoff Disease; a single pivotal trial for CLN2; and a single pivotal trial for 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.​

SOTERIA Basket Trial

Polaryx will initiate a Ph.2, open-label, proof-of-concept basket trial of PLX-200 in patients with 2 of the 13 ODD-approved NCL sub-types as well as Krabbe Disease and Sandhoff Disease. Polaryx believes that positive read-outs from this basket trial will enable the company to initiate a number of potentially single pivotal studies and support future regulatory approvals.

Single Pivotal Trial For CLN2

Polaryx plans to 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 plans to 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 us at info@polaryx.com.