HemoShear Therapeutics is advancing drug discovery programs in a multi-target approach for rare metabolic disorders and non-alcoholic steatohepatitis (NASH).



Propionic acidemia (PA) and methylmalonic acidemia (MMA) are inborn errors of metabolism in the branched-chain amino acid catabolism pathways, characterized by the build-up of toxic organic acids that result in pathophysiologic effects leading to significant morbidity and mortality in afflicted children.  The prevalence of PA and MMA in the US and Europe is estimated to be ~2000 and ~4000, respectively.  Children afflicted with these diseases are identified through newborn screening, yet very little is available in terms of treatment other than an extremely restrictive diet.  Unfortunately, life expectancy is brief with nearly all patients dying in the first quartile of life. 


HemoShear Therapeutics is developing treatments for PA and MMA.  We have recapitulated the biology of these diseases using tissue from patients and our REVEAL-Tx™ platform, allowing us to interrogate the disease pathways, and select and test meaningful drug targets to combat the diseases.   Chemistry programs were initiated against our targets in 2016 and clinical trials are expected to start enrolling in late 2018. 


Further information about these diseases and the families affected can be found at http://www.oaanews.org/



We are additionally focusing efforts on drug discovery in Nonalcoholic Steatohepatitis (NASH), a serious, chronic, progressive liver disease that is estimated to impact over 16 million people in the U.S. alone. The disease is one of the fastest-growing global diseases driven by poor diet, obesity, type 2 diabetes, and metabolic syndrome, characterized by inflammation, dysregulation of the body’s metabolism, and excessive fat accumulation in the liver that can progress to fibrosis, cirrhosis, and eventually liver failure. 


With REVEAL-Tx™, we believe that HemoShear has the only human tissue-based platform that can interrogate this disease at a fundamental biological level and improve our probability of successful drug discovery.  Currently, we are studying a wide range of drug targets being developed by other companies to identify treatment gaps and opportunities, as well as our own novel target hypotheses. Our collaborator is NASH expert Dr. Arun Sanyal, the Charles Caravati Professor and chair of the Division of Gastroenterology, Hepatology and Nutrition, in the Department of Internal Medicine at Virginia Commonwealth University.