Product Description
PLX-300 is a new drug isolated from cinnamon. It is a PPARalpha agonist. PLX-300 and its bioactive metabolites are also abundantly present in the human diet, including vegetables, fruits, honey, and whole grains. It may have therapeutic and/or prophylactic potential for Late Infantile Neuronal Ceroid Lipofuscinosis (LINCL or CLN2) and for other NCLs, such as Juvenile Neuronal Ceroid Lipofuscinosis (JNCL or CLN3). Animal Proof of Concept studies for Niemann Pick Disease Types A and B, Tay-Sachs/Sandhoff Disease, and Krabbe disease have been fully completed. Both Rare Pediatric Disease and Orphan Drug designations for GM2 Gangliosidosis Acid Sphingomyelinase Deficiency, and Krabbe Disease. (Sourced from: https://www.polaryx.com/pipeline.html)
Mechanisms of Action: PPAR Agonist
Novel Mechanism: No
Modality: Small Molecule
Route of Administration: Oral
FDA Designation:
Orphan Drug - Gangliosidosis, GM2
Orphan Drug - Krabbe Disease
Orphan Drug - Niemann-Pick Disease, Type A *
Approval Status: Not Approved
Approved Countries: None
Approved Indications: None
Known Adverse Events: None
Company: Polaryx Therapeutics
Company Location:
Company CEO:
Additonal Commercial Interests: None
Clinical Description
Countries in Clinic:
Active Clinical Trial Count:
Highest Development Phases
Phase 0: Niemann-Pick Disease, Type A
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