The Pipeline

We have repurposed thoroughly tested drugs used for other indications that have been proven safe in adults.

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 CLN2-deficient murine disease model.

The Pipeline

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PLX-100

PLX-100 is a combination of PLX-200 and ATRA (All Trans Retinoic Acid). Both compounds are safe and have been approved for other indications. PLX-100 may have a therapeutic and/or prophylactic potential for Late Infantile Neuronal Ceroid Lipofuscinosis (LINCL or CLN2) patients with Cln2 gene mutations.

Both compounds are safe and well-known drugs that have been used for other indications, and are safe for use in adult patients.

PLX-200

PLX-200 is a repurposed drug that has been safely used in adults.

It has a therapeutic and/or prophylactic potential for Late Infantile Neuronal Ceroid Lipofuscinosis (CLN2) with Cln2 gene (TPP1) mutations.

We are seeking partners to help bring Polaryx's®
medications to children in need. Interested?

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