Polaryx Therapeutics, a biotech company developing patient-friendly, small molecule therapeutics for lysosomal storage disorders, announced that the company has received an Investigational New Drug Application (IND) approval from the US Food and Drug Administration (FDA) to study PLX-200 treatment on Juvenile Neuronal Ceroid Lipofuscinosis (JNCL or CLN3) patients.
JNCL or CLN3 is a rare and fatal genetic lysosomal storage disorder caused by a Cln3 gene mutation. It is the most prevalent among the NCL diseases occurring in 3 out of 100,000 births. The function of the mutated protein, called battenin, remains unknown. CLN3 patients suffer from vision loss leading to blindness, seizures, progressive neurological deterioration, severe motor and cognitive declines, and eventually death in the second decade of life. No drug is currently available to halt and/or delay the progression of the disease.
"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. Our team has made tremendous efforts to move forward in preparing clinical trials in order to start studies as soon as possible," says Dr. Hahn-Jun Lee, M.Sc., Ph.D., president and CEO of Polaryx Therapeutics, Inc.
Alex Yang, J.D., LLM, president and CEO of Mstone Partners Hong Kong and Chair of the Board at Polaryx Therapeutics jointly stated that "We are quite excited with the recent US FDA approvals to efficiently proceed with human efficacy clinical studies on a number of Batten disease indications. We may also look for potential partnering opportunities to expand into other lysosomal storage disorder areas to help bring effective treatment for many patients with unmet needs."
PLX-200 is a repurposed drug that binds to the retinoid X receptor-a (RXRa), which binds to PPARa thereby up-regulating the expression of TPP1 mRNA in brain cells via the PPARa/RXRa heterodimer. PLX-200 also activates PPARa, which enhances production of transcription factor EB (TFEB) in brain cells. TFEB then binds to the promoter of genes involved in lysosome biogenesis and activates their production. Thus, TFEB regulates lysosomes due to its effects on the expression of lysosomal genes. PLX-200 also has additional important activities, such as reducing inflammation and preventing cell death (apoptosis).