Tackling Rare Diseases

Pipeline

HemoShear has an innovative drug discovery team focused on a portfolio of proprietary research programs for several metabolic diseases with unmet medical needs. We also are conducting exploratory work to identify new opportunities for our pipeline.

We have utilized REVEAL-TxTM to identify novel treatment approaches for our partners’ programs in NASH, gout and an undisclosed rare liver disease.

New Hope Needed

There are currently limited treatments for cholestasis disorders and hepatitis D, which can lead to severe liver damage and potentially liver transplant.

Cholestasis is a devastating medical condition characterized by the impairment or obstruction of the normal flow of bile from the liver to the small intestine. Bile is a digestive fluid produced by the liver that plays a crucial role in the digestion and absorption of fats in the small intestine. When the flow of bile is disrupted, it can lead to a buildup of bile acids and other substances in the liver, which can have dangerous health consequences. Cholestasis can lead to a range of complications and health issues, including liver damage that can lead to liver transplant, malabsorption of fat-soluble vitamins, and discomfort from symptoms like itching and jaundice.

Read more about Cholestasis >>

Hepatitis D is a viral infection that attacks the liver, which can lead to lifelong damage and even death. Hepatitis D can only occur as a co-infection with HBV or as a superinfection in individuals already infected with HBV. There is no cure for hepatitis D, the management usually involves supportive care to relieve symptoms and prevent complications. The prognosis for hepatitis D varies. Some individuals may clear the infection spontaneously, while others may develop chronic liver disease, including cirrhosis or liver failure. The severity of the disease often depends on factors such as the age at infection, the presence of other liver diseases, and the immune status of the individual.

 Read more about Hepatitis D >>

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.

Read more at NORD >>

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.

Read more at NORD>>

Partnerships

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.

Takeda Pharmaceutical Company
Fibrotic Liver Diseases, Rare Liver Disease

Takeda Pharmaceutical Company

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.

Horizon Therapeutics
Gout

Horizon Therapeutics

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.

Contact

info@hemoshear.com

HemoShear Therapeutics, Inc.
501 Locust Avenue
Suite 301
Charlottesville, Virginia 22902