HemoShear has an innovative drug discovery team focused on a portfolio of proprietary research programs for several rare diseases with unmet medical needs. In addition to HST5040, now in a phase 2 clinical trial for MMA and PA, our REVEAL-TxTM drug discovery platform has enabled us to advance three additional programs. We also are conducting exploratory work to identify new opportunities for our pipeline.
We have also utilized REVEAL-TxTM to identify novel treatment approaches for our partners’ programs in NASH, gout and an undisclosed rare liver disease.
There are currently no effective treatments for many inborn errors of metabolism, which can lead to severe organ damage, developmental deficits and premature death.
“When they looked at my son’s blood work, they thought he drank nail polish remover or antifreeze. They accused me of poisoning my kid. They decided to admit him, and his toxicity kept going up.”
Propionic Acidemia (PA) is a rare inborn error of metabolism caused by an enzymatic defect that results in a build-up of harmful acids that can damage the heart, liver, brain, and bones. The disease can cause seizures, delays to normal development like walking and talking, and other health problems. Although usually diagnosed soon after birth, there is currently no medical treatment for PA. Instead, patients are treated by managing the symptoms and by following a very restrictive diet.
“We worry about the implications of not being diagnosed. What did all those decompensations do to his brain and where is he going to go?”
Methylmalonic acidemia (MMA) is a rare inborn error of metabolism caused by an enzymatic defect in metabolism that results in an abnormally high level of acid in the blood and body tissues. In the acute form of the disease, drowsiness, coma, and seizures may occur. Mental retardation is a long-term consequence. Although usually diagnosed soon after birth, there is currently no medical treatment for MMA. Instead, patients are treated by managing the symptoms and by following a very restrictive diet.
Maple syrup urine disease (MSUD) is a rare inborn error of metabolism caused by the deficiency of an enzyme required to metabolize the chemical "building blocks" of proteins which leads to the buildup of life-threatening toxins in the body. If left untreated, it can result in brain damage and death. The condition gets its name from the characteristic odor, reminiscent of maple syrup that can be detected in the urine and earwax. While some symptoms can be controlled by dietary restrictions, there are currently no effective treatments for MSUD.
Urea cycle disorders (UCD) are rare inborn errors of metabolism resulting in a deficiency of the enzymes in the urea cycle that are responsible for removing toxic ammonia from the bloodstream. Infants with a urea cycle disorder often appear normal initially but rapidly develop brain swelling and potentially irreversible brain damage, coma and death. The treatment of urea cycle disorders consists of dietary management to limit ammonia production in conjunction with medical interventions, medications and/or supplements to remove of ammonia from the bloodstream.
HemoShear has successfully utilized our REVEAL-Tx™ drug discovery platform to identify novel drug targets for nonalcoholic steatohepatitis (NASH) and gout through exclusive collaborations with Takeda Pharmaceutical Company and Horizon Therapeutics respectively.
In 2021, HemoShear successfully completed a four-year exclusive collaboration with Takeda resulting in two novel drug targets for nonalcoholic steatohepatitis (NASH) advancing into Takeda’s Discovery Portfolio. In accordance with the agreement, Takeda assumed drug discovery and development activities, while HemoShear will continue to earn milestone payments as the programs advance.
The collaboration delivered on HemoShear’s promise to uncover disease drivers and identified and validated two novel drug targets that modulate these processes. HemoShear’s REVEAL-Tx™ platform was utilized to create a model of NASH that recapitulates hallmark features of liver fat build up, increased cell stress and inflammation, extracellular matrix signaling and fibrosis that was published in the Journal of Clinical Investigation Insight. In addition, Nature Scientific Reports published the company’s research analyzing gene expression at various stages of nonalcoholic fatty liver disease. Under the agreement, HemoShear is eligible to receive milestone payments of potentially $470 million plus royalties, and Takeda will receive the right to license and commercialize any therapeutic that results from the partnership. More information can be found in the press release.
HemoShear is also developing a new human tissue-based model of a rare liver disease using the company’s REVEAL-Tx™ Platform. This partnership will enable Takeda to interrogate their drug targets in the genetic background of human disease and accelerate the selection of lead candidates. “This new collaboration gives us the opportunity to build on [NASH] momentum and expand the reach of our combined expertise to a challenging, severe rare liver disease of high unmet need,” said Gareth Hicks, Ph.D., GI Drug Discovery Unit Head at Takeda. More information can be found in the press release here.
HemoShear has achieved multiple milestones in an exclusive collaboration established in January 2019 with Horizon Therapeutics to discover new drugs for gout. HemoShear is advancing several potential lead drug candidates for the treatment of gout after identifying and validating two novel gout drug targets in accordance with the agreement. The partnership combines HemoShear’s REVEAL-Tx™ platform and drug discovery capabilities with Horizon’s rheumatology development and commercialization expertise. Under the terms of the agreement, HemoShear receives upfront payments and R&D funding, and Horizon receives exclusive access to the company’s platform to discover new therapeutics for gout. Successful development and commercialization of multiple therapies by Horizon will make HemoShear eligible to receive milestone payments of potentially more than $500 million plus royalties. More information can be found in the press release.
HemoShear Therapeutics, Inc.
501 Locust Avenue
Charlottesville, Virginia 22902