SAN FRANCISCO, Oct. 26, 2024 /PRNewswire/ -- Kind Pharmaceutical ("Hangzhou Andao Pharmaceutical Ltd. and Kind Pharmaceuticals LLC"), a clinical-stage biopharmaceutical company focused on developing innovative medicines to treat hematological diseases and cancers, today announced that the U.S. Food and Drug Administration (FDA) Office of Orphan Products Development has granted Orphan Drug Designation (ODD) to AND017 in the treatment of Sickle Cell Disease (SCD). AND017 is being developed to treat various anemia indications associated with diseases including non-dialysis dependent chronic kidney disease (NDD-CKD) and dialysis-dependent chronic kidney disease (DD-CKD). The results for AND017's phase 1 in healthy subjects and phase 2 clinical trials in treatment of anemia in NDD-CKD and anemia in DD-CKD are currently being presented at the annual meeting of American Society of Nephrology (ASN) Kidney Week in San Diego. The preclinical work supporting AND017's ODD in SCD will be presented in future scientific meeting and subsequently published in a scientific journal.
Sickle Cell Disease (SCD) is a devasting genetic disease with US patient population around 120,000; SCD disproportionally affects Black and African Americans, comprising more than 98% of patients living with this disease. "The FDA's granting of ODD for AND017 underscores the urgent medical need for new therapies, particularly oral drugs to safely and effectively treat patients with SCD," said Dong Liu, Ph.D., Founder, Chairman, and CEO of Kind Pharmaceutical. "The granting of ODD also demonstrates the innovation capability and the vision of Kind Pharmaceutical."
"Hydroxyurea and L-glutamine are the limited FDA-approved oral treatments for SCD, AND017 from Kind Pharmaceutical might not only provide a novel oral treatment with unique mechanism of action, but also an obvious better safety and efficacy profile." said Prof. Gang Huang at UT Health San Antonio, Kathryn Mays Johnson Distinguished Chair in Oncology, who is an expert in studies of hematology and various blood diseases, and involved in the preclinical studies of AND017 in SCD. "I am eager to see how a compound with such unbelievable preclinical safety and efficacy data will translate to real world SCD patients."
FDA Orphan Drug Designation (ODD) is granted to a drug or biological product to prevent, diagnose or treat a rare disease or condition that affects fewer than 200,000 people in the United States. ODD qualifies KIND for incentives including tax credit for qualified clinical trials, exemption from user fees, and potential seven years of market exclusivity after AND017's approval for treatment of Sickle Cell Disease (SCD).
About Sickle Cell Disease (SCD)
Sickle Cell Disease (SCD) affects around 120,000 patients in US and more than 8,000,000 worldwide. As a genetic disease with a single amino acid mutation in the β-globin of mature adult hemoglobin, the hemoglobin molecules of in the red blood cells (RBCs) of SCD patients are prone to aggregate in the tissue after the dissociation of oxygen molecules, which would cause the RBCs sickling in the blood stream and eventually lead to hemolysis and occlusion of capillaries; therefore the hallmarks of SCD are hemolytic anemia and vaso-occlusive crises (VOCs); and SCD causes multiorgan failure and premature death.
About AND017
AND017 is a first-in-class hemoglobin elevating agent (HbEA) that targets multiple stages of the red blood cell (RBC) life cycle and is being developed to treat anemia associated with dialysis-dependent chronic kidney disease (DD-CKD), non-dialysis dependent chronic kidney disease (NDD-CKD), cancer-related anemia (CRA), myelodysplastic syndromes (MDS) anemia, sickle cell disease (SCD), and β-thalassemia.
About Kind Pharmaceutical ("Hangzhou Andao Pharmaceutical Ltd. and Kind Pharmaceuticals LLC")
KIND is a clinical-stage biopharmaceutical company focused on developing innovative medicines to treat hematological diseases and cancers. The company's mission, "kind to human, humble to science, good to patients", drives its commitment to advancing current science to meet unmet medical need. KIND's lead clinical candidate, AND017, first-in-class hemoglobin elevating agent (HbEA), is being developed for treating anemia of various disorders, including dialysis dependent chronic kidney disease (DD-CKD) associated anemia, non-dialysis dependent (NDD) CKD associated anemia, cancer related anemia (CRA), myelodysplastic syndromes (MDS) anemia, sickle cell disease (SCD), and β-thalassemia. KIND's second clinical candidate, AND019, an orally available brain penetrant selective estrogen receptor degrader (SERD), is being developed to treat ER+/Her2- breast cancer. KIND is also developing promising disruptive next generation ADC technologies. For details, please visit http://en.kindpharmaceutical.com.
Contact:
Dong Liu, PhD, Chairman and CEO of Kind Pharmaceutical
Email: [email protected]
Tel: 650-315 6151