Oral SYK Inhibitor for Autoimmune Hemolytic Anemia:
- Autoimmune hemolytic anemia (AIHA) is a rare, serious blood disorder where the immune system produces antibodies that result in the destruction of the body's own red blood cells.
- AIHA affects approximately 40,000 adult patients in the US and can be a severe, debilitating anemia. To date, there are no disease-targeted therapies for AIHA, despite the tremendous medical need that exists for these patients.
- Rigel's fostamatinib is a potent and relatively selective spleen tyrosine kinase (SYK) inhbitor. Pre-clinical research with fostamatinib, an orally bioavailable small molecule, has shown that inhibiting SYK may ameliorate red blood cell destruction in AIHA.
- There is potential commercial synergy in marketing fostamatinib in both the ITP and AIHA indications, since they are generally treated by the same hematology healthcare providers.
- Results for Stage 1 of the AIHA Phase 2 study met the pre-specified primary endpoint in October 2017. Stage 2 of the study, a further 20 patients, is currently enrolling.