KIND Presents Preclinical Results of AND017 in Animal Models of Sickle Cell Disease (SCD), Myelodysplastic Syndromes (MDS), and β-Thalassemia at the 66th American Society of Hematology Meeting and Exposition and Announces FDA Authorization of Investigatio
SAN FRANCISCO, Dec. 12, 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 preclinical results from its leading program AND017 in Townes Sickle Cell Disease (SCD) mouse model, inducible Mutant c-Myc Knock-in myelodysplastic syndromes (MDS) mouse model, and Hbbd3th β-thalassemia mouse model were presented at the 66th American Society of Hematology (ASH) Meeting and Exposition in San Diego from Dec. 7 to Dec. 10, 2024. Concurrently, the U.S. Food and Drug Administration (FDA) approved the investigational new drug application for AND017 for the clinical trial in treating SCD patients.
In the Townes SCD mouse model, AND017 was shown to elevate several important parameters related to anemia, including hemoglobin (Hb), red blood cells (RBC), and hematocrit (HCT), and significantly reduce mitochondrial retention in reticulocytes and mature RBCs in mice of the Townes SCD model. The details of the results are available from Kind's website, and the abstract can be found on ASH website.
In an inducible mutant c-Myc knock-in MDS mouse model, AND017 showed dramatic effects in improving hemoglobin level, red blood cell count, and hematocrit, while high-dose epoetin (EPO) had very little efficacy. The details of the results are available from Kind's website, and the abstract can be found on ASH website.
In the Hbbd3th β-thalassemia mouse model, AND017 (15 mg/kg, PO, QD, 42 days), when compared to vehicle, increased Hb level by 22%, increased RBC numbers by 14%, and increased HCT by 21%. AND017 also improved morphology of RBCs. The details of the results are available from Kind's website, and the abstract can be found on ASH website.
Sickle Cell Disease (SCD) is a devastating genetic disease, with a U.S. patient population of around 120,000. SCD disproportionately affects Black and African Americans, comprising more than 98% of patients living with this disease. The FDA's approval of the IND for AND017 to treat SCD patients follows the recent granting of Orphan Drug Designation (ODD) for AND017 in the treatment of SCD, as well as the successful completion of the Phase 2 clinical trials of AND017 in treating anemia in non-dialysis-dependent chronic kidney disease (NDD-CKD) and dialysis-dependent chronic kidney disease (DD-CKD) in the U.S. and China. The results were recently reported at the American Society of Nephrology (ASN) annual conference.
"I am very pleased to see a series of results from our leading product AND017," said Dong Liu, Ph.D., Founder, Chairman, and CEO of Kind Pharmaceutical. "We are geared toward the next stage of development for our clinical and basic research programs."
"In the Townes mouse model, beyond the erythropoiesis mechanism previously proposed for the treatment of CKD anemia with HIF-PH inhibitors, we found that AND017 could reduce red blood cell sickling and hemolysis by significantly improving their abnormal mitochondrial retention and reactive oxygen species (ROS) in SCD mice," said Prof. Gang Huang at UT Health San Antonio, Kathryn Mays Johnson Distinguished Chair in Oncology, an expert in hematology and various blood diseases, who was involved in the preclinical studies of AND017 in SCD and MDS models. "Mitochondrial retention and high ROS in red blood cells are hallmarks of sickle cell disease in patients, and the hemolysis and release of mitochondria from red blood cells into the bloodstream trigger cascades of inflammation in SCD patients, which could lead to other complications in SCD patients. I am eager to see the results of AND017 in future SCD clinical trials."
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 Anemia in Chronic Kidney Disease
Chronic kidney disease (CKD) is very common in general population; the prevalence for CKD in the United States is about 14% in adults and that in China is about 8%. Anemia is a common complication in chronic kidney disease. There are about half million dialysis-dependent CKD anemia patients and about five million non-dialysis-dependent CKD anemia patients in US; it is estimated there are about 700K DD CKD anemia patients and about 50 million NDD CKD anemia patients in China. Anemia in CKD can increase the risk of cardiovascular complications, such as heart failure and stroke, and increase morbidity and mortality.
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 https://en.kindpharmaceutical.com.
Contact:
Dong Liu, PhD, Chairman and CEO of Kind Pharmaceutical
Email: liudong@kindpharmaceutical.com
Tel: 650-315 6151
Source: KInd Pharmaceuticals