Latest news with #VAS-101
Malaysian Reserve
2 days ago
- Business
- Malaysian Reserve
Vascarta Receives FDA Orphan Drug Designation for Vasceptor® in the Treatment of Sickle Cell Disease
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 rprince@ Media requests should be directed to David Hymson at dhymson@
Associated Press
15-04-2025
- Health
- Associated Press
Vascarta commences Proof of Concept Phase 1 clinical study of VAS-101 in sickle cell disease in the United States
SUMMIT, N.J. , April 15, 2025 /PRNewswire/ -- A first in human proof of concept phase I clinical study of VAS-101 (Vasceptor®; topical curcumin gel) in sickle cell disease (SCD) is underway. The study is being conducted by the Foundation for Sickle Cell Disease Research (FSCDR) in Hollywood, Florida, under the supervision of the principal investigator, Dr. Gershwin Blyden. Ten (10) patients with sickle cell disease will be treated with VAS-101 twice per week over 4 weeks for a total of 8 treatments; their blood will be drawn weekly. Five (5) patients will be treated topically on their forearm and five (5) patients will be treated sublingually (under the tongue), the duration of treatment is 29 days. The primary study objectives are: (1) assess the safety and tolerability of VAS-101; and (2) assess its effects on impaired blood flow dynamics, including adhesion molecule expression and erythrocyte fragility parameters. The objective assessment of VAS-101 impact on red blood cell health will be performed by Functional Fluidics utilizing their proprietary assays at their specialty laboratory in Detroit, Michigan. Secondary study objectives include (1) an evaluation of the effect of VAS-101 on inflammatory markers associated with the activation of sterile inflammation over 28 days; (2) determination of the mean change in red blood cell sickling kinetics and oxygen dissociation curves will be assessed by The Biophysical Chemistry Section, Laboratory of Chemical Physics at the National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) in Bethesda, Maryland; and (3) assessment of the pain-relieving benefit and opioid-sparing effect of VAS-101. Dr. Lanetta Bronté-Hall, President of FSCDR, stated, 'I'm proud that we are taking part in Vascarta's pilot study, which explores a new transdermal approach to pain management for individuals living with Sickle Cell Disease (SCD). Pain remains one of the most challenging and life-disrupting aspects of this disease, and new approaches are urgently needed. While early in development, this research represents an important step toward finding new options that could one day help ease that burden. At FSCDR, we are committed to advancing care and bringing hope to the SCD community through innovation and collaboration.' About VAS101 VAS-101 (developed by Vascarta Inc., Summit, NJ) is a topical curcumin formulation that employs a patented transdermal delivery technology designed to increase bioavailability. A published study in the PNAS Nexus Journal indicated that VAS-101 'has the potential to ameliorate chronic pain, improve RBC stability, and reduce inflammatory consequences of SCD.' * While Curcumin possesses many beneficial attributes such as safety, anti-sickling, anti-inflammatory, and antioxidant properties, its clinical translational potential has been constrained due to limited bioavailability from oral administration. 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 >165,000 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 contribute 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 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. 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.' Dr. Friedman is the inventor of VAS-101 which is exclusively licensed to Vascarta from the Albert Einstein College of Medicine (Bronx, New York, USA). Dr. Richard Prince, Vascarta Chairman, CEO & President commented, 'We are excited at the prospect of bringing relief to SCD sufferers who have limited therapeutic options. VAS-101 has the potential to become the new standard of care in the management of SCD.' * 'Targeting sickle cell pathobiology and pain with novel transdermal curcumin' About Vascarta Vascarta is a clinical stage pharmaceutical company exploring efficient transdermal 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]. View original content to download multimedia: SOURCE Vascarta Inc
Yahoo
18-02-2025
- Health
- Yahoo
Patented transdermal drug candidate from Vascarta may offer a convenient & cost-effective way to treat sickle cell disease
SUMMIT, N.J., Feb. 18, 2025 /PRNewswire/ -- An article in the Proceedings of the National Academy of Sciences (PNAS) Nexus journal* by Goel et al. describes studies in which humanized sickle cell mice topically treated with Vascarta's transdermal curcumin gel formulation ("VAS-101") experienced reduced pain and inflammation along with improved red blood cell stability and functionality. Based in part on these favorable pre-clinical results, a clinical trial using VAS-101 in individuals with sickle cell disease will soon be initiated by the Foundation for Sickle Cell Disease and Research [FSCDR (Hollywood, Florida, USA)]. Patients will be treated with VAS-101 via the topical and sublingual routes of administration. In affected individuals, unstable red blood cells are prone to hemolysis and sickling causing inflammation, oxidative stress and blood flow stagnation which can produce acute and chronic pain as well as organ damage. There is a critical need for treatments which are cost effective address pain, easy-to-use, and without consequential side effects. Unfortunately, recently approved gene therapies cost up to $3.1 million/person and require a rigorous and toxic pretreatment regimen that still may not cure pain. Further, due to the complexity of SCD pathobiology many drugs are themselves toxic. The FDA approved Voxelotor was withdrawn from the market five years after its 2019 launch due to safety concerns (e.g., higher rate of vaso-occlusive crisis and more deaths in patients with sickle cell disease receiving this drug compared to placebo). VAS-101 reduced multiple indices of chronic pain in humanized sickle cell mice. In some instances, the reduction occurred within hours of application of the drug candidate. There was a substantial reduction in mast cell activation, a process that directly contributes to the onset and persistence of chronic pain. The topical treatment also resulted in a decrease in multiple markers of inflammation including interleukins 2, 4, and 6 (IL-2, IL-4, IL-6), interferon, and other inflammatory markers. Moreover, red blood cell stability and functionality were improved, with reduced levels of hemolysis, oxidative damage, increased hematocrit and increased levels of ATP (adenosine triphosphate). VAS-101 is highly bioavailable through a unique delivery route that bypasses drug metabolic roadblocks such as first pass modification by the liver that limit the intrinsic therapeutic efficacy for orally administered curcumin. VAS-101 results in rapid appearance of curcumin in both plasma and circulating blood cells. Uptake of curcumin in circulating red blood cells is postulated to be the likely basis for effective and sustained systemic delivery as well as the phenomena of improved health and stability of the human red blood cell. In the reported study, therapeutic efficacy was achieved by applying a single 0.1 ml drop of VAS-101 (0.23 M in curcumin **) to the abdomen once every two days for 21 days. These findings add to the list of therapeutic benefits of VAS-101 that are achievable through the transdermal delivery route and that bypass the limitations of oral delivery including the well-known gastrointestinal adverse effects from extended use of high doses of oral curcumin. Dr. Joel Friedman, Professor, Department of Microbiology & Immunology, Albert Einstein College of Medicine, and Scientific Founder & Chief Scientific Officer of Vascarta, Inc., stated, "These positive sickle cell results are consistent with multiple other preclinical studies that have shown therapeutic benefits of transdermal curcumin for pain, inflammation, cytokine storm, endothelial integrity, hypertension, aging and more." [Dr. Friedman is the inventor of VAS-101 which is licensed to Vascarta from the Albert Einstein College of Medicine (Bronx, New York, USA)]. Vascarta CEO & President Dr. Richard Prince commented that, "These breakthrough findings about the beneficial effects of our lead drug candidate (VAS-101) were generated through collaboration among scientists from many institutions. We will soon be initiating a pilot sickle cell clinical trial evaluating both topical and sublingual routes of administration. To help us speed the progression of VAS-101 into an eventual FDA-approved therapy, we welcome discussions with other pharmaceutical companies and/or strategic partners." * "Targeting sickle cell pathobiology and pain with novel transdermal curcumin" **Formulated with Curcugen® (Dolcas-Biotech, LLC) About Sickle Cell Disease Sickle cell disease is the most common inherited disease that primarily affects millions of individuals globally with the major burden shared by Sub-Saharan Africa and India. It is considered a rare disease in the United States, with majority of affected individuals suffering with debilitating chronic pain and unpredictable and recurrent episodes of acute pain requiring hospitalization and poor quality of life with reduced survival. About Vascarta Vascarta is a clinical stage pharmaceutical company exploring efficient 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 or by email to Vascarta's CEO, Dr. Richard Prince, at rprince@ Publication Authors & Affiliations Yugal Goel1, Mya A. Arellano1, Raghda T. Fouda1, Natalie R. Garcia1, Reina A. Lomeli1, Daniel Kerr2, Donovan A. Argueta1, Mihir Gupta3, Graham J. Velasco4, Richard Prince5, Probal Banerjee2, Sirsendu Jana6, Abdu I. Alayash6, Joel Friedman5,7, Kalpna Gupta1,8* 1Hematology/Oncology Division, Department of Medicine, University of California, Irvine, CA, USA. 2Department of Chemistry and Center for Developmental Neuroscience, The College of Staten Island (CUNY), NY, USA. 3Department of Neurosurgery, Yale School of Medicine, New Haven, CT, USA. 4Pathology Department, VA Long Beach Medical Center, Long Beach, CA, USA. 5Vascarta, Inc, Summit, NJ, USA. 6Laboratory of Biochemistry and Vascular Biology, Center for Biologics Evaluation and Research, Food and Drug Administration, Silver Spring, MD, USA. 7Department of Microbiology and Immunology, Albert Einstein College of Medicine, Bronx, NY, USA. 8Division of Hematology, Oncology and Transplantation, Department of Medicine, University of Minnesota, Minneapolis, MN, USA. *Corresponding Author, Kalpna Gupta, PhD Division of Hematology/Oncology, Department of Medicine UCI Health 200 S. Manchester Ave, Suite 400, Room 414, Zot 4061 Orange, CA 92868 Email: kalpnag@ View original content to download multimedia: SOURCE Vascarta Inc Sign in to access your portfolio
