Polaryx Therapeutics issued the following announcement on Jan. 20.
Polaryx Therapeutics, a biotech company developing patient-friendly small molecule therapeutics for lysosomal storage disorders announced today that the Company has received an Investigational New Drug Application (IND) approval from the U.S. Food and Drug Administration (FDA) for the treatment of LINCL patients with PLX-200.
Neuronal Ceroid Lipofuscinoses are a group of rare autosomal recessive neurodegenerative lysosomal storage disorders. Among them, LINCL is caused by mutations leading deficiency or loss of function of tripeptidyl peptidase 1 (TPP1). Intracellular accumulation of auto-fluorescent lipoproteins is one of the typical characteristics of LINCL resulting in deterioration of neurons in both the brain and other organs, such as the retina. Patients suffer from vision loss, severe seizures, and declining motor function, leading to premature death. Polaryx has advanced a unique repurposing drug development strategy to provide patients with a safe and effective oral treatment option.
"We are very excited about the IND approval from the FDA, as this will allow us to advance PLX-200 clinical studies for LINCL patients. We will prepare the clinical trial with PLX-200 as soon as possible," says Dr. Hahn-Jun Lee, M.Sc., Ph.D., President and CEO of Polaryx Therapeutics, Inc.
Dr. Raymond Wang, M.D., Ph.D., the Foundation of Caring Director of the Multidisciplinary Lysosomal Storage Disorder Program at CHOC Children's Hospital in Orange, California, also stated that "CLN2 disease is a rare progressive and fatal neurodegenerative disorder affecting young children. There is a serious unmet need for non-invasive therapies targeted against CLN2 disease. I congratulate Polaryx Therapeutics for developing its PLX-200 candidate to target this devastating disease and for securing approval from the FDA to proceed with clinical studies to assess the safety, tolerability, and efficacy of PLX-200 for children with CLN2 disease."
Polaryx Therapeutics, Inc
Polaryx Therapeutics, Inc is dedicated to developing drug candidates for lysosomal storage disorders, for which there is currently no safe and patient-friendly treatment option available. Lysosomal storage disorders are a group of rare inherited genetic disorders caused by the dysfunction of lysosomal enzymes and/or molecules important in the function of the enzymes. Young children are victims of these devastating diseases and die at an early age due to lack of treatment options. Polaryx is repurposing existing safe oral medications and/or developing new drugs, so that the treatment is patient-friendly for a prolonged use.
PLX-200 is a repurposed drug that binds to the retinoid X receptor-α (RXRα), which binds to PPARα thereby up-regulating the expression of TPP1 mRNA in brain cells via the PPARα/RXRα heterodimer. PLX-200 also activates PPARα, 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).
Late Infantile Neuronal Ceroid Lipofuscinosis
Late Infantile Neuronal Ceroid Lipofuscinosis (LINCL or CLN2) is caused by deficiency and/or loss of TPP1 caused by mutations in the Cln2 gene. TPP1, a soluble 46 kDa acid protease found in the lysosome, removes tripeptides from the N-terminus of peptides. Cln2 gene mutations result in a deficiency and/or loss of function of the TPP1 enzyme, which leads to the intralysosomal accumulation of auto-fluorescent lipofuscin. Neurons and other cells of the body accumulate auto-fluorescent lipofuscin that is highly enriched in ATP synthase subunit C. This leads to seizures, regression in developmental milestones, ataxia, visual impairment, motor deterioration, dementia, and early death between the age of six and the early teenage years.
Original source can be found here.