GBT Presents Data on New Sickle Cell Disease Pipeline Therapies with Best-in-Class Potential – Inclacumab and GBT021601

Two Inclacumab Pivotal Phase 3 Clinical Trials Expected to Begin in First Half of 2021

GBT021601 – Potent Next-Generation Hemoglobin S Polymerization Inhibitor Shown to be Highly Effective in SCD Animal Models

SOUTH SAN FRANCISCO, Calif., Dec. 06, 2020 (GLOBE NEWSWIRE) -- Global Blood Therapeutics, Inc. (GBT) (NASDAQ: GBT) today announced new preclinical data on its sickle cell disease (SCD) pipeline therapies – inclacumab, a novel P-selectin inhibitor in development to reduce the frequency of vaso-occlusive crises (VOCs) in patients with SCD, and GBT021601, a next-generation hemoglobin S (HbS) polymerization inhibitor. These data are being presented at the all-virtual 62^nd American Society of Hematology (ASH) Annual Meeting and Exposition.

“In pursuit of our mission to transform the treatment of and care for people living with sickle cell disease, our research and development pipeline is targeting multiple pathologies, including vascular occlusion and hemoglobin polymerization,” said Ted W. Love, M.D., president and chief executive officer of GBT. “We’re very excited about the best-in-class potential of both inclacumab and GBT021601. In 2021, we plan to initiate two pivotal Phase 3 clinical trials evaluating inclacumab for its ability to reduce the frequency of VOCs and hospital readmissions caused by VOCs. In addition, this is the first time we are presenting data on GBT021601. These preclinical data are very promising, and we look forward to studying the safety and efficacy of this potentially innovative therapy in SCD patients once we enter the clinic as planned in the near future.”

Inclacumab: In Vitro Analysis and Phase 3 Clinical Study Program
In vitro study results (Abstract #1707) demonstrated that inclacumab has the potential to be a best-in-class P-selectin inhibitor for reducing the frequency of VOCs in patients with SCD. When characterized alongside crizanlizumab, an FDA-approved P-selectin inhibitor for treatment of VOCs, inclacumab:

• Binds P-selectin at the natural ligand binding site and has an affinity similar to crizanlizumab,
• Demonstrated rapid binding kinetics to P-selectin and remained bound for longer, and
• Inhibited platelet-leukocyte aggregation to a greater extent than crizanlizumab.
  

Additionally, prior clinical experience with inclacumab in more than 700 non-SCD participants demonstrated the potential for a substantially longer duration of exposure and near complete inhibition of platelet-leukocyte aggregation over a 12-week period. Taken together, we believe these characteristics will translate into quarterly dosing, improved patient adherence, and the potential to expand use to a broader patient population.