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HemoShear - Model HST5040 - Investigational Oral Small Molecule Therapy for MMA and PA
HST5040 is an investigational small molecule therapy being developed by HemoShear to reduce the levels of toxins associated with methylmalonic acidemia (MMA) and propionic acidemia (PA), rare genetic disorders caused by the de?ciency of certain enzymes required to metabolize amino acids. HST5040 is formulated for convenient daily administration at home as a liquid taken either orally or through a gastrostomy tube. The FDA has granted HST5040 Orphan Drug, Fast Track and Rare Pediatric Disease designations for the treatment of MMA and PA.
HemoShear’s HERO (HElp Reduce Organic Acids) Phase 2 clinical study of HST5040 is enrolling at least 12 patients aged 2 and older with MMA or PA at select children’s hospitals in the United States. The study design includes 3 parts – starting with an open label dose escalation period, then progressing to a randomized, double-blind, placebo-controlled crossover period, followed by an open-label long-term extension. More information can be found on the HERO study website or at clinicaltrials.gov (NCT04732429).
Watch this short video to see how HST5040 may redistribute the coenzyme A (CoA) pool away from toxic propionyl-CoA to lower toxin levels and improve metabolic state.
The biopharmaceutical industry’s drug development success is impeded by a lack of physiological models that reflect the complexity of disease. HemoShear’s proprietary REVEAL-Tx™ platform combines biological and computational models of human disease to accelerate discovery of novel targets and successful new drug treatments.
Advanced Disease Models
We have developed transformational physiological models of human diseases by applying principles of physiological blood flow to tissue from patients to recapitulate their disease. Our models enable valuable insights into complex disease pathways and drug responses in a more meaningful human-relevant context.
Computational Insight
We leverage powerful computational science to extract signaling pathways from our disease models and identify novel therapeutic targets for treating diseases. We then assess our computational target hypotheses in our biological disease models.
Accelerated Discovery
The REVEAL-Tx™ platform enables HemoShear and our partners to gain unprecedented insight into the underlying mechanisms of disease, rapidly translate those discoveries into drug candidates, and reduce risk of failure by reliably assessing which drug candidates will be safer and more effectively treat patients.