Charlottesville, Va., June 24, 2020 – HemoShear Therapeutics, a clinical stage company developing treatments for rare metabolic disorders, has received clearance from the U.S. Food and Drug Administration (FDA) for its Investigational New Drug (IND) application to conduct a phase 2 clinical trial of HST5040, an oral small molecule drug for the treatment of patients with methylmalonic acidemia (MMA) and propionic acidemia (PA). MMA and PA are rare genetic disorders caused by the deficiency of certain enzymes required to metabolize amino acids. The diseases result in the rapid buildup of life-threatening metabolites that can lead to severe organ damage, seizures, developmental deficits, and premature death.
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