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Polaryx Therapeutics Receives Both Rare Pediatric Disease and Orphan Drug Designations for the Treatment of GM2 Gangliosidosis With PLX-300

PARAMUS, N.J. - November 25, 2020 - (

​​Polaryx Therapeutics, Inc., a biotech company developing small molecule therapeutics for lysosomal storage disorders, announced today that it has received from the U.S. Food and Drug Administration (FDA) both Rare Pediatric Disease and Orphan Drug designations for the treatment of GM2 gangliosidosis with PLX-300.

GM2 gangliosidosis, also known as Tay-Sachs and Sandhoff diseases, are ultra-rare and fatal pediatric neurodegenerative disorders caused by defects in Hexosaminidase A (HEXA) and Hexosaminidase B (HEXB), key enzymes in the lysosome, respectively. These genetic defects lead to abnormal accumulation of gangliosides, resulting in severe progressive neurodegeneration, seizures, loss of mobility, hearing, and vision, and early death. There is no cure for these diseases and the only treatment is supportive care.

Under the FDA's rare pediatric disease designation program, the FDA grants Rare Pediatric Disease designation for serious or life-threatening diseases with patients aged from birth to 18 years which affects fewer than 200,000 people 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 to others. In addition, because orphan drug designation has been granted to PLX-300 for GM2 gangliosidosis from the FDA, the Company can receive the FDA’s expedited review and approval process.

“We are very excited to receive both rare pediatric disease and orphan drug designations from the FDA for the treatment of GM2 gangliosidosis with PLX-300. These designations clearly demonstrate the translational excellence of the PLX-300 from bench to bedside. We are now doing required preclinical studies in order to enter into Phase1/2 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, stated, “We are making tremendous steps towards developing several promising drugs to treat a number of highly unmet diseases affecting the lysosomal enzymes in the brain. On top of the recent commencement of other lysosomal storage disorders, we will also make every effort to bring the effective drugs for children suffering from these life-threatening 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 is a novel, small molecule found in many plants as a deaminated product of phenylalanine. It is widely used as a spice or flavoring material 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, such as reducing inflammation and preventing cell death (apoptosis).

GM2 gangliosidosis

The GM2 gangliosidosis is caused by mutations in the HEXA and HEXB genes encoding subunits of ganglioside β-hexosaminidase (Hex), the lysosomal enzyme that normally degrades GM2. As a result, GM2 ganglioside accumulates in the lysosomes of nerve cells resulting in distended neurons engorged with swollen lysosomes (membranous cytoplasmic bodies; MCB) throughout the nervous system. There are two major forms of Hex: HEXA, a heterodimer composed of one α and one β subunit, and HEXB, composed of two β subunits. Tay–Sachs disease is caused by mutations in the HEXA gene encoding the α subunit of HEXA. The much rarer Sandhoff disease, a more severe form of Tay-Sachs disease, is caused by mutations in the HEXB gene encoding the β subunit, leading to deficiency of both HEXA and HEXB activities.​

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Hahn-Jun Lee, M.Sc., Ph.D.

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Original Source: Polaryx Therapeutics Receives Both Rare Pediatric Disease and Orphan Drug Designations for the Treatment of GM2 Gangliosidosis With PLX-300

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Last Updated: 07-Jan-2021