Our Pipeline

Candidate

Preclinical

Ph.1

Ph. 2/3

Commercialization

505(b)2 – Repurposed Therapies

PLX – 200

CLN2 / Single Pivotal Trial

CLN3 / Single Pivotal Trial1

Krabbe Disease

Tay-Sachs / Sandhoff Disease

1Clinical trial information for CLN3 with PLX-200 is available here: (Link)

PLX – 100

CLN2

CLN3

Niemann Pick Disease Type A / B

505(b)1 – New Chemical Entity

PLX – 300

Krabbe Disease

Tay-Sachs / Sandhoff Disease

Niemann Pick Disease Type A / B

Gene Therapy

PLX – 400

CLN2

CLN3

PLX-100

PLX-100 is a combination of PLX-200 and a supplement. The safety of each component has been well established. As a therapeutic option for NCL conditions, it was demonstrated to have a neuroprotective effect in murine Late Infantile Neuronal Ceroid Lipofuscinosis (LINCL or CLN2) and JNCL/CLN3 disease models, 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. Due to its PPARα agonistic properties that boost lysosome biogenesis via TFEB upregulation, it has been demonstrated to reduce inflammation in the brain and promote neuronal survival in murine LINCL and JNCL disease models. The treatment has also been shown to extend the life span of a murine LINCL disease model. 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 by the FDA in January (Link) and April 2020 (Link), respectively. Fast-track designation for JNCL with PLX-200 was also granted from the FDA in August 2020.

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 Tay-Sachs/Sandhoff Disease have been fully completed. Both Rare Pediatric Disease and Orphan Drug designations have been granted for GM2 Gangliosidosis Acid Sphingomyelinase Deficiency and Krabbe Disease.

PLX-400

PLX-400 is an early-stage gene therapy. Polaryx is in the process of completing pre-clinical work and plans to move quickly towards an IND filing with the FDA. PLX-400 works by reconstituting back the missing protein activity to its fully functional state in lysosomal storage disorders. Polaryx is initially focusing PLX-400 on treating CLN2 and CLN3.

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

Kim et al., 2017

We’re seeking partners to help bring Polaryx therapies to patients and families fighting lysosomal storage disorders.
Interested?