Hydroxyurea

The FDA granted orphan drug designation to AND017 for sickle cell disease, with preclinical data to be presented. Phase 1 and 2 trials showed AND017's safety and efficacy in treating anemia in chronic kidney disease patients. AND017, a first-in-class hemoglobin elevating agent, is also being developed for cancer-related anemia, myelodysplastic syndes, and β-thalassemia.

Kind Pharmaceutical's AND017 receives FDA Orphan Drug Designation for Sickle Cell Disease, aiming to treat anemia in chronic kidney disease and other conditions. The designation highlights the urgent need for new therapies and Kind's innovation capability.

The FDA granted Orphan Drug Designation to AND017 for treating sickle cell disease (SCD), affecting 120,000 US patients. AND017, a first-in-class hemoglobin elevating agent, aims to offer a novel oral treatment with better safety and efficacy than current options. It targets multiple stages of red blood cell life cycle and is under development for various anemia indications. Clinical trial results are presented at the 2024 ASN Kidney Week, with preclinical SCD data planned for future presentation.

Jubilant Therapeutics Inc. has initiated global clinical trials for JBI-802 (LSD1/HDAC6 inhibitor) in heme-oncology and JBI-778 (PRMT5 inhibitor) in solid tumors. JBI-802 showed promising results in earlier trials, with potential in essential thrombocythemia and MDS/MPN. JBI-778, a brain-penetrant PRMT5 inhibitor, aims to address larger patient segments, including those with brain metastases. Both candidates were developed using Jubilant's TIBEO Discovery Engine, with initial clinical data expected in 2025.

Jubilant Therapeutics Inc. announced dosing of first patients in global clinical trials for JBI-802 in heme-oncology and JBI-778 in solid tumors. JBI-802 is a dual inhibitor targeting LSD1 and HDAC6, showing anti-tumor activity in NSCLC patients. JBI-778, an oral PRMT5 inhibitor, aims to treat mEGFR TKI-resistant NSCLC, IDH+ HGG, and ACC, with initial clinical data expected in 2025.

Jubilant Therapeutics Inc. announces first patient dosing in global clinical trials for JBI-802, a CoREST inhibitor, and JBI-778, a PRMT5 inhibitor, targeting oncology and autoimmune diseases.

Pitt researchers identified a common recessive genetic variant, CYB5R3 T117S, linked to poor fetal hemoglobin response in sickle cell disease patients, affecting 25% of African ancestry individuals. This variant diminishes hydroxyurea's therapeutic effects, highlighting the need for precision medicine to predict therapy response.

Pfizer's withdrawal of Oxbryta from global markets due to increased risk of deaths and complications has left the sickle cell disease community reeling. Despite recent setbacks, hope for SCD treatment lies in next-gen transplantation and gene therapy, with St. Jude developing its own gene therapy. Pfizer's inclacumab and osivelotor, Agios Pharmaceuticals' mitapivat, Novo Nordisk's etavopivat, and Fulcrum Therapeutics' pociredir are among investigational therapies in the SCD pipeline.

The review highlights inotuzumab ozogamicin's role in treating pediatric ALL, particularly R/R ALL, and evolving therapeutic options. It emphasizes the drug's FDA approval for pediatric patients, its efficacy in clinical trials, and ongoing research to improve ALL treatment strategies.

ICMR partners with Zydus Lifesciences for Phase 2 trials of Desidustat, a HIF-prolyl hydroxylase inhibitor, to treat sickle cell disease. The double-blind, randomized, placebo-controlled study aims to assess efficacy and safety, following DCGI approval.