FibroGen presents data from Phase 3 MATTERHORN trial of Roxadustat

FibroGen announced the presentation of efficacy and safety data from MATTERHORN, a Phase 3 clinical study of roxadustat for the treatment of anemia in patients with lower risk transfusion-dependent myelodysplastic syndromes at the 65th American Society of Hematology Annual Meeting and Exposition being held in San Diego, California. As previously disclosed, the initial analysis with all the patients who participated in the trial, showed that more patients receiving roxadustat achieved transfusion independence vs. placebo. While the primary endpoint of transfusion independence for greater than or equal to 56 consecutive days within the first 28 weeks of the study was not met, a post-hoc analysis, showed that roxadustat performed significantly better than placebo in the subset of patients having higher transfusion burden. The TI for greater than or equal to 56 days within the first 28 weeks of the study was 36.1% for the roxadustat group and 11.5% for the placebo group, and 44.4% vs 19.2% at the end of trial, respectively. Additionally, in TI responders, more patients in the roxadustat arm vs. placebo had hemoglobin concentration increases of greater than or equal to1.5 g/dL: 45.5% vs 17.4%. Roxadustat, an oral hypoxia-inducible factor prolyl hydroxylase inhibitor, was generally well tolerated, with treatment emergent adverse events of any grade similar across treatment groups. Overall, the adverse event profile that was observed during the double-blind portion of the study was generally consistent with previous findings in MDS patients.