PARAMUS, N.J., Aug. 20, 2020 /PRNewswire/ — Polaryx Therapeutics Inc., a biotech company developing small molecule therapeutics for lysosomal storage disorders, announced today that the U.S. Food and Drug Administration (FDA) has granted Fast Track Designation to PLX-200 for the treatment of Juvenile Neuronal Ceroid Lipofuscinosis (JNCL/CLN3) patients.
JNCL/CLN3 is a rare and fatal genetic lysosomal storage disorder caused by a Cln3 mutation. It is the most prevalent among the NCL (neuronal ceroid lipofuscinosis) diseases and occurs in 3 out of 100,000 births. 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 this disease.
The FDA Fast Track Designation program is intended to facilitate the development and review process of drug candidates that treat serious and/or life-threatening conditions with an unmet medical need. Fast Track Designation enables a drug product greater access to the FDA to expedite development through more frequent review and correspondence from the FDA and priority review and accelerated approval if the requisite criteria are met.
“We are very excited about the FDA Fast Track Designation to PLX-200 for CLN3 treatment. This Fast Track Designation will expedite PLX-200 development process for the treatment of CLN3 patients with more frequent accesses to the FDA. Our team is currently preparing diligently to start clinical 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 “The Fast Track Designation suggests highly unmet medical needs in CLN3 patients and we are exceptionally motivated to proceed with clinical trials to bring patient-friendly drugs as quickly as possible. Our company also has programs to target CLN2 and other lysosomal storage disorders. We are committed to work to deliver effective drugs for patients.”
Polaryx Therapeutics, Inc
Polaryx Therapeutics, Inc is 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.
PLX-200 is a repurposed drug that binds to the retinoid X receptor-α (RXRα), which binds to PPARα. PLX-200 also activates PPARα, which enhances production of transcription factor EB (TFEB). TFEB then binds to the promoter of genes involved in lysosome biogenesis and activates their production. PLX-200 also has additional activities, such as reducing inflammation and preventing cell death (apoptosis).
Juvenile Neuronal Ceroid Lipofuscinosis
JNCL or CLN3 disease is an autosomal recessive genetic lysosomal storage disorder, where the CLN3 gene, which encodes battenin, is mutated, resulting in an accumulation of lipofuscins in neurons and in other cell types including the retina, an increase in apoptosis, and an increase in inflammation, which result in neurodegeneration. Life expectancy for patients with CLN3 ranges from the early teenage years to 30 years of age.