Encouraging Results for Briquilimab in Fanconi Anemia Conditioning

Jasper Therapeutics, a biotech firm, has reported positive results from a Phase 1b/2a study on briquilimab, an innovative antibody therapy, in the context of treating Fanconi Anemia (FA). The study, which was presented at the 2024 Stanford Medicine Center for Definitive and Curative Medicine Symposium, California, indicates that briquilimab, when used as a conditioning agent, has a promising safety profile and is well tolerated by FA patients.

The ongoing clinical trial is assessing the efficacy of intravenous briquilimab as part of a conditioning regimen for FA patients experiencing bone marrow failure. The results are encouraging, as all six patients enrolled in the study have achieved full donor engraftment and a complete recovery of blood count. This suggests that briquilimab could potentially serve as a key component of non-toxic conditioning regimens for stem cell transplants.

Briquilimab is an aglycosylated monoclonal antibody that targets the c-Kit receptor (CD117), blocking the binding of stem cell factor and inhibiting the signaling that leads to mast cell depletion. This depletion is crucial for reducing the inflammatory response in mast cell-driven diseases, such as chronic urticaria. Jasper Therapeutics is currently conducting clinical trials to evaluate briquilimab's efficacy in treating chronic spontaneous urticaria (CSU), chronic inducible urticaria (CIndU), lower to intermediate risk myelodysplastic syndromes (MDS), and as a conditioning agent for cell and gene therapies for rare diseases.

Fanconi Anemia is a rare and severe blood disorder characterized by the bone marrow's inability to produce adequate red blood cells and the potential to produce abnormal blood cells. This disorder can lead to serious complications, including bone marrow failure and severe aplastic anemia, with the only current cure being a hematopoietic stem cell transplant (HSCT).

The Phase 1/2 clinical trial (NCT04784052), sponsored by Stanford and initiated by investigators, is an open-label study that evaluates briquilimab as a potential treatment for FA patients in bone marrow failure requiring an allogeneic transplant. The study eliminates the need for busulfan chemotherapy or total body irradiation, offering a safer alternative for patients.

Jasper Therapeutics is a clinical-stage company that is developing briquilimab for the treatment of chronic mast and stem cell diseases, such as chronic urticaria and lower to intermediate risk MDS, as well as for stem cell transplants for rare diseases like sickle cell disease, FA, and severe combined immunodeficiency. The company has demonstrated the efficacy and safety of briquilimab in over 145 dosed participants and healthy volunteers across various conditions.

The positive outcomes of the briquilimab study are a significant step forward in the treatment of FA and highlight the potential of this novel antibody therapy to improve patient outcomes through safer and more effective conditioning regimens for stem cell transplants.