Ajax Therapeutics announced the presentation of its ongoing first-in-human study of AJ1-11095, a next-generation Type II JAK2 inhibitor, at the 66th American Society of Hematology (ASH) Annual Meeting in San Diego on December 8, 2024. The Phase 1 study, led by principal investigator Dr. John Mascarenhas, focuses on AJ1-11095 as an oral monotherapy for patients with primary myelofibrosis (PMF), post-polycythemia vera myelofibrosis (PPV-MF), or post-essential thrombocythemia myelofibrosis (PET-MF) who have not responded to Type I JAK2 inhibitors. More information about the study is available on www.clinicaltrials.gov (NCT06343805). The poster presentation will take place during the “Myeloproliferative Syndromes and Chronic Myeloid Leukemia: Basic and Translational: Poster II” session from 6:00 – 8:00 p.m. PT in Halls G-H of the San Diego Convention Center.

Developed in collaboration with Schrödinger, AJ1-11095 utilizes advanced structure-based drug design and computational methods. It targets the Type II conformation of the JAK2 kinase, aiming for improved efficacy and disease modification compared to existing Type I JAK2 inhibitors like ruxolitinib. Preclinical studies indicate AJ1-11095’s potential to reverse bone marrow fibrosis, reduce mutant allele burden, and maintain efficacy against MPN cells resistant to Type I JAK2 inhibition.

Myelofibrosis (MF) is a rare blood cancer affecting approximately 20,000 individuals in the United States. Characterized by enlarged spleen, bone marrow fibrosis, progressive anemia, and debilitating symptoms like fatigue, night sweats, itching, and abdominal discomfort, MF significantly impacts patients’ quality of life. Current Type I JAK2 inhibitors primarily offer symptomatic relief and spleen size reduction but have limited impact on the underlying disease. Many patients eventually discontinue these treatments due to lack of benefit, adverse events, or disease progression, highlighting the urgent need for new therapeutic options.

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Jon Napitupulu is Director of Media Relations at The Clinical Trial Vanguard. Jon, a computer data scientist, focuses on the latest clinical trial industry news and trends.