FDA Favors Clinical Trial of Potential Juvenile Batten Treatment, PLX-200

Polaryx Therapeutics has received approval from the U.S. Food and Drug Administration (FDA) to open a clinical trial into PLX-200 as a potential treatment for juvenile Batten disease.

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 ‘recycling machinery’ of a cell).

Mutations in CLN3 lead to the accumulation of protein fragments within lysosomes, causing damage to cells and, particularly, to neurons (nerve cells). Currently, there are no approved treatments for juvenile Batten disease.

PLX-200 is a repurposed medicine, originally developed to lower cholesterol, that upon binding to its receptor (retinoid X receptor-alpha, abbreviated RXRa), sets in motion a chain of molecular events that are believed to promote the formation and functioning of lysosomes.

It may also reduce cell death (apoptosis) and inflammation. It has shown beneficial effects, including an increased lifespan, in mouse modes of Batten disease.

Polaryx reported receiving Investigational New Drug Application (IND) approval from the FDA, allowing the company to move forward with trials of PLX-200 in juvenile Batten.

A similar approval to study PLX-200 as a potential treatment of late infantile Batten disease (also known as CLN2 disease) was given in January.

“We are very excited about our CLN3 IND approval from the FDA, as we can go ahead with CLN3 clinical studies with PLX-200. We also recently received a CLN2 IND approval with PLX-200 from the FDA,” Hahn-Jun Lee, MSc, PhD, president and chief executive officer of Polaryx, said in a press release.

“We are quite excited with the recent FDA approvals to efficiently proceed with human efficacy clinical studies on a number of Batten disease indications,” added Alex Yang, president and CEO of Mstone Partners Hong Kong and chair of the Board at Polaryx.