Polaryx Therapeutics Receives Both Rare Pediatric Disease and Orphan Drug Designations for the Treatment of Krabbe Disease With PLX-300

Polaryx Therapeutics, Inc., a biotech company developing small molecule therapeutics for lysosomal storage disorders, announced today that it has received both Rare Pediatric Disease and Orphan Drug Designations for the treatment of Krabbe Disease (a.k.a. Globoid Cell Leukodystrophy) with PLX-300 from the U.S. Food and Drug Administration (FDA).
Krabbe disease (KD) is a rare, heritable metabolic genetic disorder that affects approximately one in 100,000 individuals in the United States. KD is caused by deficiency of the lysosomal enzyme, galactocerebrosidase (GALC). The function of GALC is to catabolize the cytotoxic lipid, galactosylsphingosine (also known as psychosine). When either the level of GALC or its activity is compromised, galactosylsphingosine accumulates in the nervous system, ultimately leading to neurodegeneration and demyelination in patients. KD is a devastating neurological disease that typically leads to a premature death in affected children within the first two years of life. Therefore, it is critical to develop novel therapeutics for this rare and severe neurological disease. 
Under the FDA’s rare pediatric disease designation program, the FDA grants the Rare Pediatric Disease Designation for serious or life-threatening diseases affecting fewer than 200,000 patients aged from birth to 18 years in the U.S. If a new drug application (NDA) for PLX-300 is approved, the Company is eligible to receive a priority review voucher that may be sold or transferred. In addition, the Company can receive the FDA’s expedited review and approval process because Orphan Drug Designation has been granted to PLX-300 for Krabbe disease.
“Granting both Rare Pediatric Disease and Orphan Drug Designations to PLX-300 for Krabbe disease by the FDA demonstrates the broad potential of applying PLX-300 to various lysosomal storage disorders. The FDA has previously granted Rare Pediatric Disease and Orphan Drug Designations to PLX-300 for both GM2 Gangliosidosis and Acid Sphingomyelinase Deficiency. Polaryx is now performing IND-enabling studies to enter into the clinical stage 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, Inc., stated, “We are very proud that we have received three Orphan Drug Designations in rare indications with PLX-300, along with corresponding Rare Pediatric Disease Designations. In connection with our efforts in other lysosomal storage disorders, such as Batten disease with PLX-200, we will also strive to quickly and successfully move towards clinical trials with PLX-300 for the children with these highly unmet orphan diseases.”
Polaryx Therapeutics, Inc.
Polaryx Therapeutics, Inc. is developing drug candidates for lysosomal storage disorders, for which there are currently no safe and patient-friendly treatment options 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 these enzymes. Young children are victims of these devastating diseases and die at an early age due to lack of treatment options.
PLX-300
PLX-300 is a novel, small molecule found in many plants as a deaminated product of phenylalanine. It is widely used as a spice or flavoring for food. It 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-300 also has additional activities that prevent lysosomal storage disease progression, such as reducing inflammation and preventing cell death (apoptosis).
Media ContactHahn-Jun Lee, M.Sc., Ph.D.201-724-1786hahnjun7@polaryx.com
Source: Polaryx Therapeutics, Inc.