The Pipeline

Polaryx® has a diversified pipeline for lysosomal storage disorders. Our drug candidates enhance the cellular clearance pathway via TFEB-dependent lysosome biogenesis.

Our mechanism of action is unique and involves upregulation of TPP1 mRNA expression and increased lysosome biogenesis.

They also have a neuroprotective effect that prolongs the life span of a CLN2-deficient murine disease model.

Pipeline

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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 safely used to treat other diseases in both adults and children. It is a PPARα agonist that boosts lysosome biogenesis via TFEB upregulation. It has 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. An IND filing for a pivotal trial is expected to be made in 1Q 2019.

PLX-300

PLX-300 is a new drug isolated from an edible plant. 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 has 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). Currently, PoC studies on animal models of Krabbe Disease, Sandhoff disease, and Niemann Pick Type A/B disease are being performed. PLX-300 also attenuated the accumulation of amyloid beta plaque and improved memory deficit in 5XFAD mice in a PPARα-dependent manner. PLX-300 may have therapeutic potential for lysosomal storage disorders and for other neurodegenerative disorders in which cellular aggregates are detrimental to the cell.

Batten disease presents as seizures and / or vision failure followed by progressive motor dysfunction and cognitive decline.

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