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

Product
Indication
Preclinic
PI
PII
PIII
Market
505(b)2
plx-100
LSD
LINCL
JNCL
Niemann Pick Disease Types A/B
plx-200
LSD
LINCL
JNCL
Krabbe Disease
Tay-Sachs/Sandhoff Disease

Clinical trial information for JNCL with PLX-200 is https://www.clinicaltrials.gov/ct2/show/NCT04637282?term=Polaryx&draw=2&rank=1

505(b)1
plx-300
LSD
Krabbe Disease
Tay-Sachs/Sandhoff Disease
Niemann Pick Disease Types A/B
  • LSD: Lysosomal Storage Disorders
  • LINCL: Late Infatile Neuronak Ceroid Lipofuscinosis
  • JNCL: Juvenile Neuronal Ceroid Lipofuscinosis

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 PPARĪ± 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. 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 an edible plant. It is a PPARĪ± 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). 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 were granted from the FDA in November 2020. Rare Pediatric Disease Designation for Acid Sphingomyelinase Deficiency was also granted from the FDA in November 2020.

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

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