Business
Polaryx Therapeutics Receives FDA Fast Track Designation for PLX‑200 for Late‑Infantile Neuronal Ceroid Lipofuscinosis (CLN2 Disease)
Regulatory milestone supports advancement of the SOTERIA Phase 2 basket trial evaluating PLX‑200 across multiple lysosomal storage disorders PARAMUS, NJ,
About this update from Polaryx Therapeutics, Inc.
[{"type":"text","content":"Regulatory milestone supports advancement of the SOTERIA Phase 2 basket trial evaluating PLX‑200 across multiple lysosomal storage disorders PARAMUS, NJ, March 17, 2026 (GLOBE NEWSWIRE) -- Polaryx Therapeutics, Inc. (Nasdaq: PLYX), a clinical-stage biotechnology company developing disease-modifying therapies for rare pediatric lysosomal storage disorders (“LSDs”), today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track Designation to PLX-200 for the treatment of Late-Infantile Neuronal Ceroid Lipofuscinosis (LINCL/CLN2 disease). The Fast Track program is designed to facilitate and expedite the review of therapies intended to treat serious or life-threatening conditions with unmet medical need. Companies receiving Fast Track designation may benefit from more frequent interactions with the FDA and the potential for rolling review of a future marketing application. “Receiving Fast Track designation represents an important regulatory milestone as we prepare to initiate the SOTERIA Phase 2 basket trial evaluating PLX‑200 across multiple lysosomal storage disorders” said Alex Yang, Chair and Chief Executive Officer of Polaryx Therapeutics. “We appreciate the FDA’s recognition of the potential of PLX‑200 to address the significant unmet medical need in CLN2 disease and look forward to continued engagement with the agency as we advance development of this program.” Polaryx continues to advance the development of PLX-200 through SOTERIA, a phase 2, open-label, single-arm basket trial designed to evaluate the safety, tolerability, and clinical activity of PLX-200 across four rare lysosomal storage disorders: CLN2, CLN3, Krabbe disease, and Sandhoff disease. About PLX-200 Polaryx’s lead drug candidate, PLX-200, is an orally available compound comprised of gemfibrozil. Gemfibrozil is an FDA-approved lipid regulating agent in the fibrate family which has only been approved in a capsule form for adult patients with very high elevations of serum triglyceride levels to decrease serum triglycerides and very low-density lipoprotein cholesterol and increase high density lipoprotein cholesterol. The ability of gemfibrozil to cross the blood-brain barrier (“BBB”) has also been documented in third-party preclinical trials and safe use of gemfibrozil in adults has also been well-established over several decades of clini...