Prolong Pharmaceuticals Announces Completion of Enrollment in Phase II Study of SANGUINATE® in the Treatment of Sickle Cell Disease Patients with Vaso-Occlusive Crisis in an Ambulatory Setting

SOUTH PLAINFIELD, N.J., Dec. 13, 2017 /PRNewswire/ -- Prolong Pharmaceuticals announced today that it has completed enrollment in its Phase II study to evaluate SANGUINATE^® in the treatment of vaso-occlusive crisis in patients with sickle cell disease (SCD). SANGUINATE^® is a first-in-class oxygen-delivery agent that is in development to treat localized and systemic hypoxia. Vaso-occlusive crisis (VOC) is a complication of sickle cell disease that results in severe, debilitating pain that impacts the quality of life for patients. 

The Phase II study entitled, "A Phase II, Multicenter, Single-Blind, Randomized Study of the Safety and Effectiveness of SANGUINATE^® Versus Normal Saline in Adult Sickle Cell Disease Patients With Vaso-Occlusive Crisis (VOC)" is a clinical study evaluating the safety and efficacy of SANGUINATE^® for the treatment of VOC. The company expects data from all patients to be available in the first half of 2018. Preliminary results from a subset of patients were presented earlier this week in a poster session at the 59^th Annual Meeting & Exposition of the American Society of Hematology (ASH) in Atlanta, and included an exploratory assessment of patient samples for the "un-sickling" effect on red blood cells (RBCs) and its association with pain score after SANGUINATE^® treatment. 

"The potential for restoration of normal RBC shape is important because VOC occurs when sickle-shaped RBCs – the hallmark of SCD – block blood vessels and restrict blood flow, thereby depriving body tissues and organs of oxygen. This complication can damage the tissues and organs, causing severe pain," explained Ronald Jubin, PhD, Vice President of Research and Development at Prolong Pharmaceuticals. "The data interpretation presented at ASH is preliminary, and the full data set from the Phase II trial of SANGUINATE^® remains to be analyzed. Further nonclinical and clinical evaluations are required to explore these findings in more detail."

Additionally, in a separate ASH poster session, researchers presented data from a novel rat model of acute VOC in which SANGUINATE^® promoted VOC resolution by improving blood flow and restoring tissue oxygenation to near-baseline levels. Bjorn K. Song, PhD, and William H. Nugent, PhD, of Song BioTechnologies in Baltimore, Maryland, reported that in their novel animal model, SANGUINATE^® appeared to promote VOC resolution, as evidenced by improved blood flow and concomitant improved tissue oxygenation. Importantly, SANGUINATE^® not only acutely resolved VOC, but provided the added benefit of restoring tissue oxygenation to near-baseline levels, according to the investigators. Song and Nugent also reported that SANGUINATE^® improved microvascular perfusion, as indicated both by visual inspection and by measurements of tissue oxygenation; by contrast, rats in the saline and sham groups remained hypoxic.

"The sickle cell community continues to follow SANGUINATE^® with great interest, as the latest research appears to support the rationale for further clinical assessment of this agent to manage acute VOC in individuals with SCD," commented Lanetta Bronté, MD, MPH, MSPH, President and Founder of the Foundation for Sickle Cell Disease Research (FSCDR) and a co-investigator in the Phase II trial. "We look forward to the final data analysis from the SANGUINATE^® clinical program."

About VOC

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. It is caused by abnormal hemoglobin, the 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, which severely limits blood flow and oxygen delivery to tissues, thereby 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

About SANGUINATE^®

SANGUINATE^® is an investigational drug, and Prolong Pharmaceuticals plans to consult with the U.S. Food and Drug Administration (FDA) on a future clinical development program in VOC. SANGUINATE^® has been designed to act as an oxygen-transfer agent to deliver oxygen to hypoxic, or oxygen-deprived, cells. Prolong is developing SANGUINATE^® for the treatment of VOC in patients with SCD, and has received an Orphan Drug Designation from the FDA for the multiple comorbidities of 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 syndromes, and both pediatric and adult stroke.

About Prolong Pharmaceuticals

Headquartered in South Plainfield, New Jersey, Prolong Pharmaceuticals, LLC, is clinical stage biopharmaceutical company focusing on 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, which is 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.

References

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. http://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. http://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