Our Pipeline

PLX-100 is a preclinical stage orally administrable combination therapy comprised of our PPARα agonist, PLX-200, and vitamin A, a RXRα agonist. 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 CLN2 and CLN3 disease models, extended the life span of a murine CLN2 disease model and reduced the amounts of brain storage materials (lipofuscin). Orphan drug designation (ODD) has been obtained from the FDA for all subtypes of neuronal ceroid lipofuscinosis (NCL).

PLX-200, our most advanced drug candidate, is a repurposed oral small molecule for the treatment of LSDs. We are pursuing PLX-200 through a 505(b)(2) regulatory pathway and is designed to be administered through a novel and proprietary oral solution. 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.

Polaryx plan to advance PLX-200 through an IND-approved Phase 2 SOTERIA basket trial for the treatment of certain LSDs, which we believe represent approximately one quarter of the LSD population, including CLN2, and CLN3 subtypes of NCL, Krabbe disease, and Sandhoff disease. We have also received authorization under two separate INDs to initiate potentially single pivotal trials in CLN2 and CLN3, the most prevalent subtypes of NCLs, which we filed on December 20, 2019 and March 6, 2020 and received authorization for on January (Link) and April 2020 (Link), respectively. The FDA has granted three orphan drug designations (ODDs) to PLX-200, for the treatment of all 13 subtypes of NCLs, GM2 gangliosidoses, such as Tay-Sachs and Sandhoff diseases, and Krabbe disease. PLX-200 has also received fast track designation (FTD) for the treatment of CLN3.

PLX-300 is a preclinical stage orally administrable combination therapy comprised of unsaturated carboxylic acid and a PPARα agonist that occurs naturally in several plants as a deaminated product of phenylalanine. Cinnamic acid, the main component of PLX-300, has been documented to have antioxidant and anti-inflammatory activities that are important for protecting brain cells from neurodegeneration, a common pathology in LSDs. PLX-300 has received orphan drug designation (ODD) and rare pediatric drug designation (RPDD) status for the treatment of GM2 gangliosidosis, Krabbe disease, and NPD type A and type B. We have completed preclinical animal model studies for PLX-300 and are evaluating the potential for clinical advancement.

PLX-400 is a preclinical stage novel gene therapy candidate. It is designed to deliver lysosomal genes in appropriate adeno-associated viral vectors. Initial development of PLX-400 is focused on the treatment of CLN2 and CLN3, and additional preclinical animal studies are ongoing with the goal of advancing the program toward clinical development. The drug candidate is also being explored as a monotherapy or in combination with oral administration of PLX-200 and expect to determine any clinical development plans for PLX-400 at a later date.