Curcumin

SUMMIT, N.J., June 12, 2025 /PRNewswire/ -- Vascarta Inc., a biopharmaceutical company committed to advancing innovative therapies for underserved patient populations, announced today that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to its lead drug candidate, Vasceptor® (VAS-101), for the treatment of Sickle Cell Disease (SCD). Orphan Drug Designation is awarded to therapies intended to treat rare diseases that affect fewer than 200,000 people in the United States. This designation provides significant benefits, including seven years of market exclusivity upon approval, tax credits for qualified clinical trials, and exemption from certain FDA fees. "The attainment of Orphan Drug Designation represents an important regulatory milestone for Vascarta and underscores the therapeutic potential of Vasceptor® to provide safe and effective relief to patients living with this debilitating disease," said Dr. Richard Prince, CEO & President of Vascarta. "We plan to work closely with the United States Food & Drug Administration to bring Vasceptor® to market as rapidly as possible." 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 showing pain reduction and therapeutic efficacy resulting from our novel approach of targeting red blood cell instability, neuro-inflammation and vascular inflammation bodes well for the development of a widely accessible therapy that prevents and treats many if not most of the clinical consequences of SCD." Vasceptor® is exclusively licensed to Vascarta from the Albert Einstein College of Medicine (Bronx, New York, USA). 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). 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 use 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. WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

SAN DIEGO, Dec. 10, 2024 /PRNewswire/ -- Vascarta Inc. ("Vascarta") reported new research findings at the 66th Annual American Society of Hematology meeting in San Diego showing that their novel transdermal preparation of curcumin reduces nerve and organ damage while improving red blood cell health in humanized sickle cell mice. Continue Reading Vasceptor® is currently available from a 503a compounding pharmacy licensed in 22 states. Based on these and previous results in pre-clinical studies showing reduced pain, Vascarta's transdermal curcumin gel will be clinically tested next year in human sickle cell disease (SCD) sufferers by the Foundation for Sickle Cell Disease and Research (FSCDR (Hollywood, Florida, USA). In SCD, red blood cells (RBCs) are unstable. The unstable RBCs tend to release free hemoglobin and heme which sets in motion pro-inflammatory and oxidative processes that promote nerve and organ damage. There is a critical need for an FDA-approved treatment that is safe, prevents and treats SCD sequelae, and is easy to self-administer. As described in the American Society of Hematology meeting poster*, when humanized sickle cell mice were exposed to inflammation and oxidative stress associated with SCD, they experienced nerve, organ, and RBC damage, leading to pain. However, when scientists at the University of California – Irvine applied Vascarta's transdermal curcumin gel to humanized sickle cell mice, and to nerve tissue removed from these mice, they demonstrated reduced nervous tissue damage. Improvements were seen in the hippocampal neurons of the brain in the central nervous system and in dorsal root ganglia neurons in the peripheral nervous system. There was also clear evidence of reduced inflammation-induced activation of microglia in the brain - which is an essential step in protecting the central nervous system from the consequences of SCD - and reduced oxidative damage to the spinal cord. Experiments conducted by the FDA's Center for Biologics Evaluation and Research (CBER) showed the transdermal curcumin treatment of SCD mice i) improved the mitochondrial bioenergetic parameters in the heart, ii) reduced oxidative stress and iii) increased bioenergetic capacity in RBCs, all of which contribute to the complex constellation of symptoms observed in SCD patients. Principal investigator, Professor Kalpna Gupta (University of California – Irvine), stated that: "These breakthrough findings about the beneficial effects of the transdermal curcumin gel on nervous system, organs, and red blood cells were generated through collaboration among scientists from several institutions, including the University of California – Irvine, the US Food and Drug Administration (FDA), and Albert Einstein College of Medicine." Professor Joel Friedman (Albert Einstein College of Medicine and inventor of Vascarta's patented transdermal drug delivery technology) explained that: "The positive sickle cell results are consistent with multiple other preclinical studies that have shown, for example, therapeutic benefits of transdermal curcumin for inflammation, pain, cytokine storm, endothelial integrity, hypertension, aging." Dr. Richard Prince, Vascarta Chairman and CEO, commented that: "We are excited that these findings, along with other favorable pre-clinical results showing reduced pain, give us the confidence to progress into human clinical testing among sickle cell patients." *" Mechanism-based treatable targets for organ damage and pain in sickle cell disease" About Sickle Cell Disease Sickle cell disease is the most common inherited disease that primarily affects African Americans and Hispanics in the United States. Individuals with sickle cell disease may experience serious health complications, such as chronic pain, stroke, lung problems, eye problems, infections, and kidney disease, along with periodic acute pain crises. About Vascarta Inc. Vascarta is a clinical-stage pharmaceutical company developing transdermal delivery of pharmaceuticals to address several conditions including sickle cell disease, arthritis, hypertension, cancer, and aging. More information regarding Vascarta's existing research or future collaborations can be found at . Publication Authors & Affiliations Yugal Goel1, Sirsendu Jana2, Mya A. Arellano1, Reina A. Lomeli1, Graham J. Velasco3, Donovan A. Argueta1, Richard Prince4, Abdu I. Alayash2, Joel Friedman4,5, Kalpna Gupta1,6* 1Hematology/Oncology Division, Department of Medicine, University of California, Irvine, CA, USA; 2Laboratory of Biochemistry and Vascular Biology, Center for Biologics Evaluation and Research, Food and Drug Administration, Silver Spring, MD, USA; 3Pathology Department, VA Long Beach Healthcare System, Long Beach, CA; 4Vascarta Inc, Summit, NJ; 5Department of Microbiology and Immunology, Albert Einstein College of Medicine, Bronx, NY, USA; 6Division of Hematology, Oncology and Transplantation, Department of Medicine, University of Minnesota, Minneapolis, MN, USA. Corresponding Publication Author Email: [email protected] Vascarta contact: Richard Prince, PhD – Chairman & Chief Executive Officer Email: [email protected] SOURCE Vascarta Inc WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED