Estimated readout windows: registry primary-completion date + 6-12 weeks. Windows already open are shown from "Now". Drug names link to ClinicalTrials.gov.
Phase 2 · ActiveModality: small molecule (oral; PPARα agonist, repurposed)
Aliases gemfibrozil
Target PPARα
Partnerships Rush University Medical Center: 2016 exclusive license for LSD use (3.5% royalties; $100K approval milestone)
Indications
CLN2 and CLN3 neuronal ceroid lipofuscinosis (NCLs)(Phase 2)
Krabbe disease(Phase 2)
Sandhoff disease(Phase 2)
Catalysts
— H2 2026
Status note Lead. SOTERIA open-label multi-indication master study; IND cleared Oct 2025; recruitment opened Jul 2026 (sentinel safety group). FDA orphan designations for all 13 NCL subtypes, GM2 gangliosidoses, Krabbe.
PLX-300
Preclinical · ActiveModality: small molecule (oral; novel)
Aliases cinnamic acid
Target PPARα
Indications
GM2 gangliosidosis(Preclinical)
Krabbe disease(Preclinical)
Niemann-Pick disease type A and B(Preclinical)
Status note IND-enabling studies. FDA orphan + rare pediatric drug designations for GM2, Krabbe, NPD A/B.
Status note Preclinical combination therapy. FDA orphan designation for CLN2.
PLX-400
Preclinical · ActiveModality: gene therapy
Target CLN2
Partnerships Rush University Medical Center: exclusive gene therapy patent license (Jan 2025; 2022 Rush License Agreement milestones up to $75K orphan / $650K non-orphan; 1.75% royalties)
Indications
Lysosomal storage disorders(Preclinical)
Status note Preclinical novel gene therapy; being explored as monotherapy or with oral PLX-200.
Per-asset detail extracted from the 10-Q filed 2026-08-13. Fields the filing does not state are marked "not disclosed" rather than filled from other sources.
Sponsor-matched active trials from ClinicalTrials.gov (1 as sponsor, 0 as collaborator); "Sponsor" = company is the lead sponsor, "Collaborator" = company is a collaborator. The BP column flags big-pharma involvement as lead sponsor or collaborator (excluding the company itself). The Mgmt guide column shows what management guided for readout timing when a guided program matches the trial (hover for the quote, click for the source). Trial listings are not a company pipeline slide.