Polaryx Therapeutics raised $10 million and is moving PLX-200, an oral reformulation of gemfibrozil, into an open-label phase II basket trial spanning four ultra-rare pediatric lysosomal storage disorders. The company is conducting the trial across multiple indications within a single study structure. Polaryx’s strategy follows a model often used in ultra-rare pediatrics, where limited patient populations make conventional randomized trials difficult. The basket format aims to evaluate activity signals across related diseases. The company is “fresh off” a Nasdaq listing, tying capital access to near-term execution. Lawyer-turned biotech financier Alex Yang founded Polaryx after prior experience including the sale of Epygenix Therapeutics to Harmony Biosciences. For investors and clinicians, the move updates how reformulated small molecules are being repurposed for pediatric rare diseases using trial designs optimized for scarce cohorts.