Driven by a deep and urgent desire to improve the care of people with sickle cell disease (SCD), we are collaborating with the SCD community to increase awareness of the disease and how it affects patients, changing the way people with SCD are perceived by society at large and helping patients gain access to innovative medicines. Building on the extensive knowledge gained from years of research, we are focused on changing the treatment paradigm for SCD.
| Program | Discovery | Preclinical | Early Clinical (Phase 1/2) | Late Clinical (Phase 3/4) | Approved |
|---|---|---|---|---|---|
VoxelotorFDA-approved SCD treatment for ages 12 and older | |||||
VoxelotorHOPE-KIDS 2: Post-approval confirmatory study measuring the mean change in transcranial doppler (TCD) in SCD patients 2-15 years of age | |||||
VoxelotorActIVe Study: Planned Phase 4 study evaluating daily physical activity in SCD patients 12 years of age and older | |||||
VoxelotorHOPE-KIDS 1: Ongoing study in SCD patients 9 months-17 years of age | |||||
VoxelotorDose escalation study evaluating the safety and tolerability of voxelotor at daily doses of 1500 mg to 3000 mg in SCD patients 18 years of age and older | |||||
Inclacumab*Potential treatment to reduce frequency of vaso-occlusive crises (VOCs) and reduce hospital re-admissions post-VOC | |||||
GBT021601Potential next-generation HbS polymerization inhibitor | |||||
HbF inducers†Potential treatments for SCD and beta thalassemia |
Unlike other biotech companies, which typically begin with a molecule and search for an indication, our founding vision involved developing therapies to specifically address SCD.
Voxelotor is an oral therapy taken once daily that directly inhibits sickle hemoglobin polymerization, which causes red blood cells (RBCs) to deform and become sickle shaped. Voxelotor works by binding to hemoglobin and stabilizing RBCs in an oxygenated state. This increases hemoglobin’s affinity for oxygen and inhibits polymerization and the resultant sickling and destruction of RBCs.
We are committed to studying the potential of voxelotor to slow the progression of SCD and its devasting downstream effects across a wide range of studies, including the HOPE-KIDS 2 post-approval confirmatory study using transcranial doppler (TCD) flow velocity to evaluate reduction in stroke risk in children 2 to 15 years of age.
In recognition of the critical need for new SCD treatments, voxelotor was granted Breakthrough Therapy, Fast Track, Orphan Drug and Rare Pediatric Disease designations by the U.S. Food and Drug Administration (FDA) for the treatment of patients with SCD, as well as Priority Medicines (PRIME) designation from the European Medicines Agency (EMA). The European Commission (EC) has designated voxelotor as an orphan medicinal product for the treatment of patients with SCD.
We are developing inclacumab – a novel, fully human monoclonal antibody P-selectin inhibitor – for SCD patients with frequent pain crises, also known as vaso-occlusive crises (VOCs). P-selectin inhibition is clinically validated to reduce VOCs,1 a complication of SCD.
We have an exclusive worldwide licensing agreement with Roche for inclacumab, which has established pharmacokinetic data, safety and tolerability in more than 500 patients. Manufacturing is currently underway, and we expect to initiate a pivotal study of inclacumab in 2021.
