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 approved therapies for AIHA, despite the tremendous medical need that exists for these patients.
- Rigel's fostamatinib is an oral spleen tyrosine kinase (SYK) inhibitor. Pre-clinical research with fostamatinib in AIHA has shown that inhibiting SYK may ameliorate red blood cell destruction.
- Results from Stage 1 of Rigel's fostamatinib Phase 2 trial in patients with AIHA met the pre-specified primary endpoint in October 2017.
- In February 2018, updated results from Stage 1 showed an increased clinical response rate after an additional patient met the primary endpoint, bringing the Stage 1 response rate to 53% (9/17) of evaluable patients receiving fostamatinib.
- Stage 2 of the study, a further 20 patients, is currently enrolling.
- On January 31, 2018, the FDA granted Orphan Drug designation to fostamatinib for the treatment of patients with AIHA.