These data are being presented at the all-virtual 62nd
'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
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.
In 2021, GBT plans to initiate two global, randomized, placebo-controlled pivotal Phase 3 trials evaluating safety and efficacy of inclacumab. These trials are designed to enhance understanding of how P-selectin inhibitors could provide clinical benefit for patients with SCD and reduce overall healthcare utilization. One study is designed to reduce the frequency of VOCs over one year in patients with SCD when treated with inclacumab (30 mg/kg) or placebo every 12 weeks. The second study will evaluate inclacumab based on a primary endpoint of 90-day hospital readmission rates following a VOC hospitalization. Participants in that trial will receive either a single dose of inclacumab (30 mg/kg) or placebo, peri-discharge following a VOC hospitalization. Approximately 50 percent of
About Sickle Cell Disease
Sickle cell disease (SCD) affects an estimated 100,000 people in the United States,2 an estimated 52,000 people in Europe,3 and millions of people throughout the world, particularly among those whose ancestors are from sub-Saharan Africa.2 It also affects people of Hispanic, South Asian, Southern European and Middle Eastern ancestry.2 SCD is a lifelong inherited rare blood disorder that impacts hemoglobin, a protein carried by red blood cells that delivers oxygen to tissues and organs throughout the body.4 Due to a genetic mutation, individuals with SCD form abnormal hemoglobin known as sickle hemoglobin. Through a process called hemoglobin polymerization, red blood cells become sickled - deoxygenated, crescent-shaped, and rigid.4-6 The sickling process causes hemolytic anemia (low hemoglobin due to red blood cell destruction) and blockages in capillaries and small blood vessels, which impede the flow of blood and oxygen throughout the body. The diminished oxygen delivery to tissues and organs can lead to life-threatening complications, including stroke and irreversible organ damage.5-8
About Oxbryta (voxelotor) Tablets
Oxbryta (voxelotor) is an oral, once-daily therapy for patients with sickle cell disease (SCD). Oxbryta works by increasing hemoglobin's affinity for oxygen. Since oxygenated sickle hemoglobin does not polymerize, GBT believes Oxbryta blocks polymerization and the resultant sickling and destruction of red blood cells, which are primary pathologies faced by every single person living with SCD. Through addressing hemolytic anemia and improving oxygen delivery throughout the body, GBT believes that Oxbryta has the potential to modify the course of SCD. On
As a condition of accelerated approval, GBT will continue to study Oxbryta in the HOPE-KIDS 2 Study, a post-approval confirmatory study using transcranial Doppler (TCD) flow velocity to assess the ability of the therapy to decrease stroke risk in children 2 to 15 years of age.
In recognition of the critical need for new SCD treatments, the FDA granted Oxbryta Breakthrough Therapy, Fast Track, Orphan Drug and Rare Pediatric Disease designations for the treatment of patients with SCD. Additionally, Oxbryta has been granted Priority Medicines (PRIME) designation from the
GBT plans to seek regulatory approvals to expand the potential use of Oxbryta in
About
Forward-Looking Statements
Certain statements in this press release are forward-looking within the meaning of the Private Securities Litigation Reform Act of 1995, including statements containing the words 'will,' 'anticipates,' 'plans,' 'believes,' 'forecast,' 'estimates,' 'expects,' and 'intends,' or similar expressions. These forward-looking statements are based on GBT's current expectations and actual results could differ materially. Statements in this press release may include statements that are not historical facts and are considered forward-looking within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. GBT intends these forward-looking statements, including statements regarding GBT's priorities, dedication, focus, goals, mission and vision; safety, efficacy and mechanism of action of Oxbryta and other product characteristics; commercialization, delivery, availability, use, and commercial and medical potential of Oxbryta; inferences drawn from study results and related analyses, including with respect to the potential of inclacumab and GBT021601; ongoing and planned studies of Oxbryta and drug candidates and related protocols, activities, timing and other expectations; potential expansion of the approved use of Oxbryta for more patients in the
Contact:
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Email: simmergut@gbt.com
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