Liberyx Therapeutics (“Liberyx”) is a clinical-stage drug development company focused on developing treatment for rare diseases (particularly metabolic disorders) primarily affecting children, such as Glycine Encephalopathy, also known as Non-Ketonic Hyperglycinemia (NKH) and Urea Cycle Disorders (UCD) followed by expansion into other metabolic rare indications. Liberyx currently has three drug candidates, including its lead drug, LBX-100 and 200, both of which are new chemical entities (Ph2 ready by 1H 2022) and a NKH is a rare, genetic, metabolic disorder caused by a defect in the enzyme system that breaks down the amino acid glycine, resulting in an accumulation of glycine in the body’s tissues and fluids. No curative treatment for NKH currently available. However, there are certain diet treatments that can improve outcomes.
Individuals with UCDs have deficiencies in enzymes involved in the urea cycle, the body’s main mechanism for removing ammonia from blood. UCD belong to inborn errors of metabolism that are associated with fatal brain damage and death in newborns.