Global Blood Therapeutics' Sickle Cell Med Results In Rapid, Durable Improvements In Hemoglobin Levels Throughout 72 Weeks Of Treatment

by Vandana Singh, Benzinga Editor
April 8, 2021 7:20 AM | 2 min read |
  • Global Blood Therapeutics Inc GBT has announced The Lancet Haematology has published the complete analysis of 72-week data from the Phase 3 HOPE Study evaluating Oxbryta (voxelotor) tablets in patients with sickle cell disease (SCD).
  • The results showed a significant and sustained improvement in hemoglobin levels, reduction in hemolysis, and improved overall health status, thus supporting the long-term use of Oxbryta.
  • Oxbryta directly inhibits hemoglobin polymerization, the root cause of the sickling and destruction of red blood cells in SCD. It is approved in the U.S. for the treatment of SCD in patients ages 12 years and older.
  • Approximately 90% of patients treated with Oxbryta achieved a hemoglobin improvement of >1 g/dL from baseline at one or more time points during the study compared to 25% on placebo.
  • Around 59% of patients treated with Oxbryta 1500 mg achieved a hemoglobin increase of over two g/dL, and 20% achieved more than three g/dL at one or more time points, compared to approximately 3% and no patients in the placebo group, respectively.
  • The analysis also showed Oxbryta was associated with fewer vaso-occlusive crises and was three times less likely to experience an acute anemic episode.
  • Additionally, some 74% of patients taking Oxbryta had their overall clinical status rated as "moderately improved" or "very much improved" by their clinician compared with approximately 47 % of the placebo group.
  • Treatment with Oxbryta remained generally well tolerated, and rates of adverse events were similar between treatment groups over 72 weeks.
  • Findings from a post hoc analysis of the HOPE study published in the American Journal of Hematology evaluated the incidence and outcomes of leg ulcers in SCD patients.
  • Results showed leg ulcers improved or resolved by week 72 in all patients (5 of 5) receiving Oxbryta 1500 mg compared with 63% of patients in the placebo group.
  • Price Action: GBT shares closed 0.4% lower at $42.28 on Wednesday.
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