At HemoShear we are driven to apply our scientific insights to develop medicines that improve the lives of patients with rare metabolic diseases. We are committed to understanding and helping to meet the needs of patients and their families.
We are currently focused on developing a treatment for methylmalonic acidemia (MMA) and propionic acidemia (PA). These rare genetic diseases are caused by inherited metabolic defects that result in the build-up of harmful toxins that can lead to severe organ damage, seizures, developmental deficits, and premature death. Although usually diagnosed soon after birth, there are currently no medical treatments for MMA or PA that improve quality of life or extend lifespan. Patients are treated by managing symptoms and following very restrictive diets.
HemoShear has developed an experimental drug treatment for MMA and PA called HST5040. Our laboratory research shows that HST5040 has the potential to reduce toxins that impact the entire body. The drug is designed to be conveniently administered daily at home as a liquid taken either orally or through a gastric feeding tube.
HemoShear has been cleared by the Food and Drug Administration to conduct a clinical study assessing our experimental drug HST5040 in patients with MMA and PA. Our first clinical study, called HERO (HElp Reduce Organic Acids), will be conducted with at least 12 patients with MMA and PA. The study, which will start with patients age 12 and over and then expand to age 2 and older, will be conducted at select leading children’s hospitals around the United States.
We will post information on ClinicalTrials.gov when the study starts. If there is a study site located near you, then you can speak with your physician or contact the site about participating. Please sign up if you would like to receive information from HemoShear about our clinical trial or other company developments.
With MMA and PA often causing developmental delays, there is a need for validated ways to measure cognitive and behavioral function of patients. HemoShear is sponsoring preliminary research conducted with Children’s National Medical Center to evaluate how different assessment tools can be used to measure the impact of MMA and PA on verbal communication, processing speed, life skills and sleep. This research is being conducted online and does not involve in-person clinic visits.
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HemoShear Therapeutics is committed to developing therapies that improve the lives of patients with rare genetic disorders with high unmet need, such as methylmalonic acidemia (MMA) and propionic acidemia (PA). We are working with a sense of urgency as we focus on designing and conducting clinical trials to assess safety and efficacy of our oral drug candidate HST5040 for patients with MMA and PA.
We are dedicated to expeditiously developing potential new therapies and believe that the most effective strategy for making them widely available to patients around the world is to maintain our focus on conducting the necessary research and development, including efficient enrollment and completion of the clinical trials required to support global regulatory approvals.
Clinical trials are required to establish whether an investigational medical product is safe and effective, and participants in clinical trials play a critical role in informing the understanding and treatment of serious diseases. We believe that participation in one of our clinical trials is the most appropriate way to access investigational drugs like HST5040 and we are not currently providing the drug for expanded access.
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