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CHARLOTTESVILLE, Va., Dec. 16, 2020 /PRNewswire/ HemoShear Therapeutics, a clinical stage company developing treatments for rare metabolic disorders, is pleased to announce that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designations to HemoShear s HST5040 oral small molecule for treatment of methylmalonic acidemia (MMA) and propionic acidemia (PA). HST5040 was granted Fast Track and Rare Pediatric Disease designations to treat MMA and PA earlier in 2020.
This past June, HemoShear received clearance from the FDA for its Investigational New Drug application to conduct a phase 2 clinical study of HST5040 in patients with MMA and 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 buildup of toxic metabolites that can lead to frequent metabolic decompensations, severe organ damage, seizures, developmental deficits, and pr
HemoShear Therapeutics Identifies Novel Drug Target to Treat NASH in Drug Discovery Partnership with Takeda
Drug Target Will Advance into Takeda s Discovery Portfolio as Part of Ongoing Collaboration
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CHARLOTTESVILLE, Va., Dec. 10, 2020 /PRNewswire/ HemoShear Therapeutics, LLC, a privately held clinical stage biotechnology company, has achieved a research milestone in its discovery and development collaboration with Takeda Pharmaceutical Company Limited ( Takeda ) to advance its first nonalcoholic steatohepatitis (NASH) target into Takeda s discovery portfolio. Using the company s
REVEAL-Tx™ human disease modeling platform, HemoShear is collaborating with Takeda to identify and validate novel drug targets for treating NASH. In accordance with the collaboration agreement executed in October 2017, HemoShear will receive an undisclosed milestone payment.