The Pipeline

PLX-100

PLX-100 is a combination of PLX-200 and a supplement. The safety of each component has been well established. PLX-100 may have a therapeutic effect and/or prophylactic potential for Late Infantile Neuronal Ceroid Lipofuscinosis (LINCL or CLN2) patients and for other NCLs, such as Juvenile Infantile Neuronal Ceroid Lipofuscinosis (JNCL or CLN3). A neuroprotective effect has been demonstrated with murine LINCL and JNCL disease models. PLX-100 treatment extended the life span of a murine LINCL disease model and reduced the amounts of brain storage materials (lipofuscin). Orphan drug designation has been obtained from the FDA for all subtypes of neuronal ceroid lipofuscinosis.

PLX-200

PLX-200 is a repurposed drug that has been used to treat other diseases in both adults and children. It is a PPARα agonist that boosts lysosome biogenesis via TFEB upregulation. It may have therapeutic and/or prophylactic potential for Late Infantile Neuronal Ceroid Lipofuscinosis (LINCL or CLN2) and for other NCLs, such as Juvenile Infantile Neuronal Ceroid Lipofuscinosis (JNCL or CLN3). A neuroprotective effect has been demonstrated in murine LINCL and JNCL disease models. The treatment extended the life span of a murine LINCL disease model and reduced the level of storage materials (lipofuscin) in the brain. Orphan drug designation has been obtained from both the FDA and EMA for all subtypes of neuronal ceroid lipofuscinosis. INDs for LINCL and JNCL with PLX-200 were approved in January and April 2020 from the FDA, respectively. Fast track designation for JNCL with PLX-200 was also granted from the FDA in August 2020. We are preparing a clinical trial for JNCL with PLX-200, and its detailed clinical trial information is as follows.

PLX-300

PLX-300 is a new drug isolated from cinnamon. It is a PPARα 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.