Hillhurst Biopharmaceuticals Receives Grant to Develop Sickle Cell Disease Lead
US based Hillhurst Biopharmaceuticals received a Phase I Small Business Innovation Research (SBIR) grant of $300,000 from the National Heart, Lung and Blood Institute of Maryland, USA for the preclinical development of lead candidate, HBI 002 which is an oral formulation to prevent vaso-occlusive crises experienced by sickle cell disease (SCD) patients.
HBI 002 is an orally available formulation of the best studied molecules to up regulate heme oxygenase for the treatment of conditions and symptoms associated with sickle cell disease (SCD).
It is being developed to deal with the vaso-occlusive crisis related to the SCD. The upregulation of heme oxygenase has been shown to reduce inflammation in sickle cell disease as well as a number of other conditions like multiple sclerosis. The heme oxygenase pathway also prevents cell death (apoptosis). HBI 002 is an oral therapeutic drug and apart from the above mentioned conditions is also found useful in patients undergoing kidney transplantation and suffering acute cerebral injuries.
Figure 1. The heme oxygenase pathway
According to Hillhurst, this enzyme and its associated gasotransmitter have been shown to reduce inflammation and vaso-occlusion in a number of transgenic sickle cell mouse studies and also in vitro research. In addition, clinical studies have shown a reduction in sickling and increase in red blood cell survival after inhalation.
This developmental study of HBI 002 by Hillhurst is expected to enter IND (investigational new drug) enabling studies by the next year.
“With this award, the NHLBI recognizes the therapeutic potential of HBI-002 to address a significant unmet medical need in sickle cell disease,” said Andrew Gomperts, Hillhurst’s Chief Executive Officer. “Hillhurst is grateful to the NHLBI for recognizing the value of advancing the development of HBI-002. We believe our novel approach of targeting multiple mechanisms to prevent vaso-occlusive crises, including reducing inflammation, apoptosis, and sickling, holds great promise, which we believe is validated by this award.”
Sickle cell disease is an inherited blood disease and is known to cause a varied line of symptoms and conditions involving various vital organs like brain, heart, lungs, kidney, liver, bones, spleen. Severe pain in SCD is an emergency and is called as acute sickle cell crisis.
Figure 2. Appearance and characteristics of normal and sickle cell anemia blood cells
SCD affects nearly 90000 people in the US and the average life expectancy of the SCD patients is only 42 years.
It may be noted that hydroxyurea is the only FDA approved medication that prevents painful episodes in SCD.
Featured image credit: Red blood cells of sickle cell anaemia disease and normal cells © exty (Stock Photo ID: 80070536)