FDA Grants Orphan Drug Status to KIND's AND017 for Sickle Cell Disease

Kind Pharmaceutical, operating under the names Hangzhou Andao Pharmaceutical Ltd. and Kind Pharmaceuticals LLC, is a clinical-stage biopharmaceutical company based in San Francisco. The company primarily focuses on developing innovative treatments for hematological diseases and cancers. Recently, the U.S. Food and Drug Administration (FDA) Office of Orphan Products Development granted Orphan Drug Designation (ODD) to their drug candidate AND017 for the treatment of Sickle Cell Disease (SCD).

AND017 is being developed to tackle various types of anemia, including those associated with both non-dialysis dependent chronic kidney disease (NDD-CKD) and dialysis-dependent chronic kidney disease (DD-CKD). The promising results from phase 1 trials in healthy subjects and phase 2 trials for anemia in both NDD-CKD and DD-CKD are currently being presented at the American Society of Nephrology (ASN) Kidney Week in San Diego. Future scientific meetings will highlight the preclinical work supporting the ODD for AND017 in SCD, followed by publications in scientific journals.

Sickle Cell Disease (SCD) is a severe genetic disorder affecting approximately 120,000 people in the United States, with a disproportionate impact on Black and African Americans, who represent over 98% of the SCD patient population. The FDA's ODD recognition for AND017 highlights the critical need for new therapies, especially oral treatments that are safe and effective for SCD patients. Dong Liu, Ph.D., Founder, Chairman, and CEO of Kind Pharmaceutical, emphasized that the FDA's decision reflects Kind Pharmaceutical's innovative capabilities and vision.

Professor Gang Huang of UT Health San Antonio, an expert in hematology and blood diseases, has been involved in the preclinical studies of AND017. He highlighted the significance of AND017 as a potential new oral treatment with a unique mechanism of action and superior safety and efficacy profile compared to existing FDA-approved treatments like Hydroxyurea and L-glutamine. Prof. Huang expressed his eagerness to see how the compelling preclinical results will translate into real-world benefits for SCD patients.

The FDA grants Orphan Drug Designation to drugs or biological products intended to treat rare conditions affecting fewer than 200,000 people in the United States. This designation includes benefits such as tax credits for qualified clinical trials, exemption from user fees, and seven years of market exclusivity upon the drug's approval.

Sickle Cell Disease (SCD) impacts around 120,000 people in the U.S. and over 8 million globally. It is caused by a single amino acid mutation in the β-globin of mature adult hemoglobin, making the hemoglobin in red blood cells prone to aggregation after oxygen dissociation. This aggregation leads to the sickling of red blood cells, resulting in hemolysis and capillary occlusion, causing hemolytic anemia, vaso-occlusive crises (VOCs), multi-organ failure, and premature death.

AND017 is a pioneering hemoglobin elevating agent (HbEA) that targets multiple stages of the red blood cell (RBC) life cycle. It is being developed to treat various anemia indications, including those related to chronic kidney disease (both dialysis and non-dialysis dependent), cancer, myelodysplastic syndromes (MDS), Sickle Cell Disease (SCD), and β-thalassemia.

Kind Pharmaceutical is dedicated to advancing science to address unmet medical needs, driven by their mission to be kind to humans, humble to science, and good to patients. The company’s lead candidate, AND017, is aimed at treating diverse forms of anemia. Another clinical candidate, AND019, is being developed as an orally available brain-penetrant selective estrogen receptor degrader (SERD) for treating ER+/Her2- breast cancer. Kind Pharmaceutical is also working on next-generation ADC technologies.