Source – Jasper Therapeutics
Jasper Therapeutics has initiated a Phase I trial (NCT05903274) to evaluate the use of briquilimab as second-line therapy in patients with lower-risk myelodysplastic syndrome (LR-MDS). The trial has successfully dosed its first patient.
“LR-MDS patients often face limited treatment options that primarily focus on increasing blood cell production and survival rather than restoring normal blood homeostasis. By directly targeting and depleting the diseased stem cells, briquilimab has the potential to restore bone marrow to a healthier and more functional state, with the ultimate goal of improved long-term outcomes and quality of life. We are excited to test briquilimab in the LR-MDS setting.”
– Jeffery Lancet, MD, chair of the Department of Malignant Hematology at Moffitt Cancer Center
Briquilimab is an innovative antibody therapy that targets c-Kit and aims to address various diseases, including LR-MDS, chronic spontaneous urticaria, and novel stem cell transplant conditioning regimens. Previous studies have demonstrated the efficacy and safety of briquilimab in over 130 patients with conditions such as severe combined immunodeficiency, acute myeloid leukemia, MDS, sickle cell disease, and Fanconi anemia. Furthermore, briquilimab is being developed as a transformative non-genotoxic conditioning agent for gene therapy.
The ongoing Phase I trial is an open-label, single-arm study that aims to evaluate the safety and tolerability of briquilimab as a second-line therapy for LR-MDS patients. The trial will utilize a 3+3 dose-escalation design to determine the maximum tolerated dose or optimal biologic dose, as well as the recommended Phase II dose of briquilimab as a monotherapy for LR-MDS patients with documented cytopenia, such as red blood cell transfusion dependence, thrombocytopenia, or neutropenia.
Approximately 30 patients who are 18 years or older and have LR-MDS with very low-, low-, or intermediate-risk features and symptomatic cytopenias will be enrolled in the trial. These patients will receive treatment with briquilimab every 56 days for four consecutive cycles.
To be eligible for the study, women of childbearing potential must agree to use effective contraception methods throughout the study period and for three months after the last dose of briquilimab. Women who are not of childbearing potential must either be postmenopausal or surgically sterilized and provide a negative serum pregnancy test at the beginning of the study. Male patients must be surgically sterile or willing to use contraception during the study and for three months after the last dose of briquilimab.
The trial is being conducted at the Moffitt Cancer Center in Tampa, FL, and is expected to be completed by July 2025.
“The initiation of this trial in LR-MDS represents a significant milestone in our briquilimab development program. To date, the potential of briquilimab has been clinically assessed across five transplant indications and we are excited to explore its potential as a treatment option for a chronic disease such as LR-MDS. This trial underscores our commitment to improving outcomes and quality of life for patients with rare and chronic diseases driven by mast and stem cells.”
– Ronald Martell, president, and chief executive officer of Jasper
