Prizm Pharmaceuticals

PriZm Therapeutics is a late-stage clinical biotechnology company dedicated to developing therapies for ultra-rare pediatric orphan diseases. The company's lead program, SRW101 (tiratricol), is a thyroid hormone analog designed to treat Allan-Herndon-Dudley Syndrome (AHDS), a severe neurodevelopmental disorder caused by mutations in the MCT8 thyroid hormone transporter. SRW101 works by bypassing the defective transporter to deliver thyroid hormone activity directly to the brain and peripheral tissues, aiming to normalize thyroid signaling. Having demonstrated significant clinical benefit in compassionate use studies, PriZm is currently advancing SRW101 through registration-enabling trials to provide the first approved treatment for AHDS patients.