FibroGen Announces Results for MATTERHORN, a Phase 3 Clinical Study of Roxadustat for the Treatment of Anemia in Patients with Myelodysplastic Syndromes (MDS)

Study Did Not Meet Primary Endpoint

SAN FRANCISCO, May 05, 2023 (GLOBE NEWSWIRE) -- FibroGen, Inc. (NASDAQ: FGEN) today announced that MATTERHORN, a Phase 3 clinical study of roxadustat for treatment of anemia in patients with transfusion-dependent lower risk myelodysplastic syndromes (MDS) did not meet its primary efficacy endpoint. The proportion of patients who achieved red blood cell transfusion independence in the first 28 weeks was 47.5% for the roxadustat arm compared to 33.3% for placebo (p=0.217). The adverse event profile of roxadustat that was observed in the preliminary safety analysis was generally consistent with previous findings. Safety will be further evaluated at study completion.

A total of one-hundred forty (140) patients were enrolled in MATTERHORN, a Phase 3, double-blind placebo-controlled study investigating the safety and efficacy of roxadustat for treatment of anemia in patients with lower risk transfusion-dependent myelodysplastic syndromes. The primary endpoint of the study is transfusion independence for ≥ 56 consecutive days during the first 28 weeks of treatment, and patients are followed for up to 52 weeks. The MATTERHORN study is sponsored and conducted by FibroGen and is part of FibroGen’s co-development collaborations with AstraZeneca and with Astellas Pharma Inc.

About Myelodysplastic Syndromes Anemia
Myelodysplastic syndromes (MDS) are a group of disorders characterized by poorly formed or dysfunctional blood cells, resulting in chronic anemia in most patients. Annual incidence rates of MDS are estimated to be 4.9/100,000 adults in the U.S¹. Approximately 80% of MDS patients have anemia at the time of diagnosis² and around 60% of MDS patients will experience severe anemia (hemoglobin <8 g/dL) at some point during the course of their disease³. Lower-risk MDS patients represent approximately 77% of the total diagnosed MDS population⁴. Anemia in MDS patients is associated with increased risk of cardiovascular complications and the need for blood transfusion⁵. Transfusion dependent MDS patients suffer higher rates of cardiac events, infections and transformation to acute leukemia, and a decreased overall survival rate when compared with non-transfused patients with MDS, and decreased survival compared to an age-matched elderly population⁶. In addition, anemia frequently leads to significant fatigue, cognitive dysfunction, and decreased quality of life. Currently, there are few options available for treating anemia in MDS. Patients with MDS typically rely on repeated blood transfusions and administration of ESAs.