SOUTH PLAINFIELD, N.J., May 2, 2017 /PRNewswire/ -- SANGUINATE™, the only investigational biopharmaceutical product currently in clinical development for the treatment of multiple comorbidities of sickle cell disease (SCD), is effective in returning red blood cells to a more normal morphology (shape) in patients experiencing vaso-occlusive crisis (VOC) related to SCD, according to research presented at the 11th Annual Symposium of the Foundation for Sickle Cell Disease Research (FSCDR) in Fort Lauderdale, Fla.
Sickle cell disease is a group of chronic genetic disorders that affects an estimated 70,000 to 80,000 Americans.1 Vaso-occlusive crisis, the most common complication of SCD, is an extremely painful, potentially deadly condition characterized by abnormal hemoglobin, a protein in red blood cells that transports oxygen from the lungs to tissues and organs in the body and carries carbon dioxide back to the lungs.2,3 Cells affected by VOC become sickle- or crescent-shaped, a deformity that prevents them from flowing freely through blood vessels, potentially causing excruciating pain and other symptoms that may lead to death.4 VOC results in approximately 197,000 emergency department visits each year in the U.S. and costs an estimated $356 million annually for pain management alone.5
"SANGUINATE is a dual-gas transfer agent that has been shown to revert sickled red blood cells to a more normal shape. These latest findings are consistent with prior in vitro study results showing that SANGUINATE may offer a prolonged therapeutic effect on cell morphology," said Ronald Jubin, Ph.D., vice president of research and development at Prolong Pharmaceuticals. "We look forward to advancing our clinical program to further evaluate SANGUINATE's potential benefits for people suffering from sickle cell disease."
At the FSCDR symposium, researchers presented preliminary findings from an ongoing Phase II clinical trial evaluating the safety and efficacy of SANGUINATE in the treatment of VOC. The trial seeks to determine whether infusing SANGUINATE during an acute VOC episode can reduce the need for intravenous (IV) opiates and prevent hospitalization for patients experiencing VOC. The investigators collected whole blood samples from 10 patients prior to SANGUINATE infusion, at the time of patient discharge, and 72 hours post-infusion. The samples treated with SANGUINATE showed a reduction in the number of abnormally shaped cells. The beneficial effect of SANGUINATE was evident within hours of infusion, and was sustained at the 72-hour sampling, indicative of a prolonged duration of effect.
"Vaso-occlusive crisis is a debilitating, painful condition caused by sickling of the red blood cells," states Gershwin Blyden, M.D., Ph.D., principal investigator at the FSCDR. "These findings add to the mounting clinical evidence supporting the effects of SANGUINATE to ameliorate a painful crisis in the acute phase."
"Clinical research underway to evaluate SANGUINATE is just one example of the cutting-edge research advanced and discussed at the annual symposium of the Foundation for Sickle Cell Disease Research," noted Lanetta Bronté, M.D., MPH, MSPH, president and founder of the FSCDR. "It's a privilege to bring this innovative research to eligible and interested patients with sickle cell disease at our innovative outpatient center."
About the FSCDR
The Foundation for Sickle Cell Disease Research (FSCDR) is a comprehensive, non-profit organization that provides a platform for researchers, healthcare providers, and those living with sickle cell disease. The FSCDR works collaboratively with academia, pharmaceutical, biotechnology and community organizations to identify best practices to help with the management and future care for sickle cell patients. In 2015, the FSCDR opened the Sickle Cell Care and Research Network in South Florida, the nation's first standalone outpatient center completely devoted to sickle cell care and services.
SANGUINATE is the only biological product currently in clinical development for the multiple comorbidities of SCD, and has received an Orphan Drug Designation from the U.S. Food and Drug Administration. SANGUINATE facilitates the transfer of oxygen to oxygen-deprived cells and tissues and has also been shown to down-regulate the inflammatory response in blood samples of patients with SCD. Many of the comorbidities of SCD are caused by a spiraling cycle of sickling, hemolysis and blood vessel inflammation. These comorbidities include VOC, acute chest syndrome, leg ulcers and pediatric and adult stroke. By correcting oxygen levels and down-regulating inflammation, SANGUINATE may be effective in treating many of the debilitating, acute comorbidities associated with SCD.
Phase I studies in healthy volunteers and patients with stable SCD have been completed. Phase II trials of SANGUINATE are also planned for VOC and leg ulcers secondary to SCD as well as for ischemia.
About Prolong Pharmaceuticals
Headquartered in South Plainfield, New Jersey, Prolong Pharmaceuticals, LLC, is developing products to treat several diseases and their debilitating comorbidities associated with reduced quality of life, increased medical cost and significant mortality. Prolong's senior management team includes inventors of the most successful drug delivery technology in pharmaceutical history, PEGylation, now responsible for the development of a dozen drugs improving the quality of life for sufferers of hepatitis, kidney disease, and a number of other life-threatening diseases.
1Genetics Home Reference. Sickle cell disease. 2016. https://ghr.nlm.nih.gov/condition/sickle-cell-disease#diagnosis.
2National Institutes of Health, National Heart, Lung, and Blood Institute. Evidence-based management of sickle cell disease. Expert panel report. 2014. https://www.nhlbi.nih.gov/health-pro/guidelines/sickle-cell-disease-guidelines.
3National Institutes of Health, National Cancer Institute. NCI Dictionary of Cancer Terms. 2017. https://www.cancer.gov/publications/dictionaries/cancer-terms?cdrid=45108.
4National Institutes of Health, National Heart, Lung, and Blood Institute. What is sickle cell disease? 2016. https://www.nhlbi.nih.gov/health/health-topics/topics/sca.
5Wilson BH, Nelson J. Sickle cell disease pain management in adolescents: a literature review. Pain Manag Nurs. 2015;16(2):146–151.
SOURCE Prolong Pharmaceuticals, LLC