National Heart, Lung, and Blood Institute / National Institutes of Health Signs Clinical Trial Agreement with Vascarta Inc. to Perform Phase 1 Clinical Study of VAS-101 in Sickle Cell Disease

SUMMIT, N.J., July 7, 2025 /PRNewswire/ -- Under the agreement, the National Heart, Lung, and Blood Institute / National Institutes of Health will perform a human phase I clinical trial of Vascarta's VAS-101 (Vasceptor®; topical curcumin gel) in Sickle Cell Disease (SCD). The clinical trial is anticipated to be initiated later this year by NHLBI under the medical supervision of principal investigator, Dr. Swee Lay Thein, Chief, Laboratory of Sickle Cell Genetics and Pathophysiology and Chief, Sickle Cell Branch.

The trial will evaluate safety, tolerability, and the effects of topically applied VAS-101 on biomarkers of inflammatory activity and red cell metabolism in SCD patients. Patients will be treated at the NIH Clinical Center with laboratory testing conducted at NHLBI.

Dr. Richard Prince, Chairman, CEO and President of Vascarta Inc., stated the following:

"NHLBI contacted us earlier this year indicating their interest in funding and performing a VAS-101 SCD clinical trial. NHLBI's interest in performing their own Vasceptor^® (VAS-101) SCD clinical trial, under a soon-to-be-submitted FDA Investigational New Drug Application, was sparked following their review of our recently co-authored publication with scientists from the United States FDA Department of Biochemistry and Vascular Biology and the University of California (Irvine; San Diego) that demonstrated that Vasceptor^® enhances red blood cell health in vivo and ex vivo. This latest Vasceptor^® SCD development following last month's attainment of FDA orphan drug designation further substantiates our regulatory strategy to pursue FDA approval of our curcumin-based topical/transdermal asset in SCD."

Dr. Joel Friedman, Professor, Department of Microbiology & Immunology, Albert Einstein College of Medicine, and Vascarta Scientific Founder & Head, Scientific Advisory Board, stated, "The preclinical results to date support a mechanism of action resulting from the rapid and sustained transdermal delivery of curcumin both into the plasma and on to the circulating blood cells. Curcumin loaded red blood cells can act as stealth delivery vehicles that bypass the immune system and first pass metabolism by the liver. This unique pathway accounts for the i) stabilization of red blood cells and ii) effective and rapid onset of anti-inflammatory and antioxidative stress activity that includes protection from neuroinflammatory processes that result in chronic pain. Most recently, preclinical SCD efficacy results show that the topically applied curcumin gel rapidly enhances both tissue perfusion and tissue oxygenation. The combination of these pleiotropic properties strongly suggest that Vasceptor^® (VAS-101) can safely and effectively manage the many clinical sequelae associated with SCD and other chronic inflammatory conditions."

About Vasceptor^® (VAS-101)

VAS-101, developed by Vascarta Inc. (Summit, NJ, USA), is a patented topical curcumin formulation that is delivered using patented transdermal technology and that is designed to overcome the limited bioavailability and suboptimal effectiveness of oral dosing of curcumin. A study in PNAS Nexus suggests VAS-101 may reduce chronic pain, stabilize red blood cells, and lower inflammation in sickle cell disease (SCD) based on a study in a humanized mouse model of SCD. It is currently in a Phase 1 clinical trial at the Foundation for Sickle Cell Disease Research (FSCDR) in Hollywood, Florida. While curcumin is known for its safety and anti-inflammatory, antioxidant, and anti-sickling properties, its clinical utility has been limited by poor oral bioavailability.

About Sickle Cell Disease

Sickle Cell Disease is the most common inherited genetic disorder that affects primarily African American and non – Hispanic Black individuals in the United States. SCD associated complications include anemia, acute and chronic pain, infections, pneumonia and acute chest syndrome, stroke, kidney, liver, and heart disease.

Current estimates indicate there are 175,000 cases of SCD in the USA and 45,000 in European Union countries. The estimated life expectancy of those with sickle cell disease in the USA is more than 20 years shorter than the average expected lifespan.

SCD is caused by a single point mutation in the globin gene leading to sickling of red blood cells. It is characterized by severe pain, inflammation, oxidative stress, and organ damage, which contributes to the poor quality of life and reduced survival. Recently approved SCD therapies do not mitigate pain, and patients are often on multiple drugs which often have undesirable side effects. 

The unmet medical need for most SCD sufferers is significant. Better therapies that are safe, improve outcomes, optimize compliance for patients of all ages, minimize the need for blood transfusions, and reduce the need for chronic administration of potentially harmful pain medications are needed.

About Vascarta

Vascarta Inc. is a clinical stage pharmaceutical company developing efficient transdermal and transmucosal delivery of pharmaceuticals to address inflammatory conditions with an initial focus on Sickle Cell Disease and osteoarthritis. To learn more, contact Vascarta Chairman, CEO & President, Dr. Richard Prince, at [email protected]. Media requests should be directed to David Hymson at [email protected].

SOURCE Vascarta Inc