The Pipeline

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

The Pipeline

PLX-100

PLX-100 is a combination of PLX-200 and ATRA (All Trans Retinoic Acid), and both components are safe and well-known drugs that have been used 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.

The mechanism of action is unique and involves upregulation of TPP1 mRNA expression when a residual copy of the Cln2 gene exists in cells and increased lysosome biogenesis. Because PLX-100 can be used with lower doses of both compounds due to a synergistic effect, it is safe for use in pediatric patients. It also has a neuroprotective effect and prolongs the life span of CLN2 deficient animals.

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

PLX-200

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

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

Its mechanism of action is unique and involves upregulation of TPP1 mRNA expression when a residual copy of the Cln2 gene exists in cells and increased lysosome biogenesis.

It also has a neuroprotective effect that prolongs the life span of CLN2 deficient animals.

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

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