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A Phase II, open-label, single arm basket trial to assess the safety, tolerability, and clinical activity of PLX-200, in Neuronal Ceroid Lipofuscinosis (CLN2, CLN3), Krabbe disease, and Sandhoff disease

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Trial Profile

A Phase II, open-label, single arm basket trial to assess the safety, tolerability, and clinical activity of PLX-200, in Neuronal Ceroid Lipofuscinosis (CLN2, CLN3), Krabbe disease, and Sandhoff disease

Status: Planning
Phase of Trial: Phase II

Latest Information Update: 29 Apr 2026

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At a glance

  • Drugs Gemfibrozil (Primary)
  • Indications Globoid cell leukodystrophy; Neuronal ceroid lipofuscinosis; Sandhoff disease
  • Focus Therapeutic Use
  • Acronyms SOTERIA
  • Sponsors Polaryx Therapeutics

Most Recent Events

  • 21 Apr 2026 According to a Polaryx Therapeutics media release, the U.S. Food and Drug Administration (FDA) has granted Fast Track Designation (FTD) to PLX-200 for the treatment of Juvenile Neuronal Ceroid Lipofuscinosis (JNCL/CLN3 disease), Krabbe disease (globoid cell leukodystrophy), and Sandhoff disease (GM2 gangliosidosis Type II).
  • 17 Mar 2026 According to Polaryx Therapeutics media release, company plans to initiate the trial in the third quarter of 2026.
  • 17 Feb 2026 According to Polaryx Therapeutics media release, Polaryx received a safe to proceed letter from the FDA in October 2025 and continues to actively work with its contract research organization (CRO) to initiate the trial in the first half of 2026.

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