Juvenile Batten, also known as Juvenile Neuronal Ceroid Lipofuscinosis (JNCL) or CLN3 disease, is caused by mutations in the CLN3 gene, which codes for a protein called battenin. This protein is believed to be involved in the functioning of lysosomes (the ârecycling machineâ of a cell).
mutations in CLN3 lead to the accumulation of protein fragments within lysosomes, causing damage to cells and, in particular, to neurons (nerve cells). Currently, there is no approved treatment for juvenile Batten disease.
PLX-200 is a reused drug, originally developed to lower cholesterol, which by binding to its receptor (X-alpha retinoid receptor, abbreviated RXRa), triggers a chain of molecular events believed to promote the formation and function of lysosomes.
It can also reduce cell death (apoptosis) and inflammation. It has shown beneficial effects, including increased lifespan, in mouse modes of Batten disease.
Polaryx has reported receiving Request for an investigational new drug (IND) FDA approval, allowing the company to move forward with trials of PLX-200 in young Batten.
âWe are very excited about our CLN3 IND approval by the FDA as we can continue the CLN3 clinical studies with PLX-200. We also recently received CLN2 IND approval with PLX-200 from the FDA, âHahn-Jun Lee, MSc, PhD, President and CEO of Polaryx, said in a Press release.
“We are very pleased with the recent FDA approvals to effectively conduct clinical human efficacy studies on a number of indications for Batten disease,” added Alex yang, President and CEO of Mstone Hong Kong partners and Chairman of the Board of Directors of Polaryx.