Our development programs
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.
GBT is 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.
GBT is 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.
GBT has an exclusive worldwide licensing agreement with Roche for inclacumab, which has established pharmacokinetic data, safety and tolerability in more than 500 patients. We have initiated two pivotal Phase 3 studies evaluating the effect of inclacumab on reducing the frequency of VOCs and on hospital readmissions for people with SCD. The THRIVE (Therapy for Reduction with Inclacumab of VOC Episodes) Studies are currently recruiting.
Discovered and designed by GBT’s research and development team, GBT601 has the same mechanism of action as voxelotor, with the potential for greater efficacy by achieving higher hemoglobin levels and occupancy at lower doses, as demonstrated in preclinical studies.
GBT601 is being studied in a single and multiple ascending dose Phase 1 study assessing the safety, tolerability, pharmacokinetics and pharmacodynamics of GBT601 in up to six people with SCD ages 18 to 60 years. This study follows an ongoing first-in-human trial to determine safe and tolerable dosing.