Polaryx Therapeutics raised $10 million after a Nasdaq listing and is moving PLX-200—an oral gemfibrozil reformulation—into an open-label Phase 2 basket trial across four ultra-rare pediatric lysosomal storage disorders. The program is built for diseases where small, heterogeneous populations make trial design difficult and where single-arm approaches are common. The study follows Polaryx’s portfolio strategy rooted in repurposing and reformulating existing chemistry rather than starting from scratch, aiming to provide a scalable path through early development. Founder Alex Yang drew on prior rare-disease experience, including the 2024 sale of Epygenix Therapeutics to Harmony Biosciences. For investors and sponsors, the key signal is continued appetite for rare pediatric indications delivered through trial frameworks that can manage regulatory and endpoint constraints.