HemoShear Therapeutics, Inc., a Charlottesville, Va.-based clinical stage company developing treatments for rare metabolic disorders, has raised $40m in Series A financing.

The round was led by Suvretta Capital with participation from Janus Henderson Investors, Adage Capital Management LP and other private investors. In conjunction with the financing, David Friedman, MD, Managing Director at Suvretta Capital and John Tilton will join the board of HemoShear. Reginald F. Woods, a founding investor and board member of HemoShear, will retire from the board.

Led by Jim Powers, Chairman and CEO, HemoShear is a clinical stage company developing treatments for rare metabolic disorders with significant unmet patient need. The company’s drug discovery platform, REVEAL-Tx™, enables its scientists to create biologically relevant human disease models to uncover the underlying mechanisms of disease, translate those discoveries into drug targets, and select candidates that may treat patients successfully. In addition to the proprietary rare disease programs, HemoShear has exclusive partnerships to identify novel therapeutic approaches in nonalcoholic steatohepatitis (NASH) and an undisclosed rare liver disease with Takeda, and in gout with Horizon Therapeutics.

The company intends to use the funds to complete a phase 2 study of its lead compound, HST5040, an investigational oral small molecule therapy, for the treatment of methylmalonic acidemia (MMA) and propionic acidemia (PA), as well as fund future clinical studies and earlier stage programs. The FDA has granted the company’s HST5040 Orphan Drug, Fast Track and Rare Pediatric Disease designations to treat MMA and PA. HemoShear’s HERO (HElp Reduce Organic acids) phase 2 clinical study of HST5040 will enroll at least 12 patients aged 2 and older with MMA or PA at select children’s hospitals in the United States.