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ATLANTA – Voxelotor, an investigational inhibitor of hemoglobin polymerization, demonstrated consistent and sustained improvements in hemoglobin levels in adolescents with sickle cell disease (SCD), according to first results of a phase 2b study.
A hemoglobin response of at least 1 g/dL was achieved in 55% of patients (6 of 11) at 16 weeks, in results of the ongoing open-label, 24-week study presented at the annual meeting of the American Society of Hematology.
The treatment was well tolerated, associated with mostly grade 1-2 adverse events, and also improved patient-reported symptoms, according to Carolyn C. Hoppe, MD, of University of California, San Francisco, Benioff Children’s Hospital.
at a press conference. “We need therapies to treat this disease and prevent the complications.”
Dr. Hoppe presented data on the first 12 patients in a study designed to assess the safety, pharmacokinetics, and efficacy of voxelotor in adolescent patients with homozygous hemoglobin SS or hemoglobin S beta-0 thalassemia.
All patients except one were already receiving standard care with hydroxyurea, suggesting that the two drugs may provide additive effects, according to investigators.
Treatment with voxelotor was associated with improvements in hemoglobin at 16 weeks, along with improvements in clinical measures of hemolysis – a 34% median reductions in reticulocytes and a 27% reduction in indirect bilirubin, Dr. Hoppe reported.
Patient-reported symptoms improved in 10 of 12 patients, with a median reduction of 94% in total symptom scores (TSS) at week 16, including 5 subjects with a mean TSS of 0.
Voxelotor is designed to address the cause of sickle cell disease, according to Dr. Hoppe, by modulating hemoglobin’s affinity for oxygen, which in turn inhibits hemoglobin polymerization.
“If we can interrupt that early on, we can hopefully disrupt or interrupt the biologic pathways that lead to the consequences of the disease,” she said.
The study is ongoing, as is a phase 3 clinical trial evaluating voxelotor in patients aged 12 years and older with SCD.
Voxelotor was previously known as GBT440
The study was sponsored by Global Blood Therapeutics. Dr. Hoppe reported research funding from and advisory relationships with Global Blood Therapeutics. Several coauthors are employees of the company.
SOURCE: Hoppe C et al. ASH 2017. Abstract 689
ATLANTA – Voxelotor, an investigational inhibitor of hemoglobin polymerization, demonstrated consistent and sustained improvements in hemoglobin levels in adolescents with sickle cell disease (SCD), according to first results of a phase 2b study.
A hemoglobin response of at least 1 g/dL was achieved in 55% of patients (6 of 11) at 16 weeks, in results of the ongoing open-label, 24-week study presented at the annual meeting of the American Society of Hematology.
The treatment was well tolerated, associated with mostly grade 1-2 adverse events, and also improved patient-reported symptoms, according to Carolyn C. Hoppe, MD, of University of California, San Francisco, Benioff Children’s Hospital.
at a press conference. “We need therapies to treat this disease and prevent the complications.”
Dr. Hoppe presented data on the first 12 patients in a study designed to assess the safety, pharmacokinetics, and efficacy of voxelotor in adolescent patients with homozygous hemoglobin SS or hemoglobin S beta-0 thalassemia.
All patients except one were already receiving standard care with hydroxyurea, suggesting that the two drugs may provide additive effects, according to investigators.
Treatment with voxelotor was associated with improvements in hemoglobin at 16 weeks, along with improvements in clinical measures of hemolysis – a 34% median reductions in reticulocytes and a 27% reduction in indirect bilirubin, Dr. Hoppe reported.
Patient-reported symptoms improved in 10 of 12 patients, with a median reduction of 94% in total symptom scores (TSS) at week 16, including 5 subjects with a mean TSS of 0.
Voxelotor is designed to address the cause of sickle cell disease, according to Dr. Hoppe, by modulating hemoglobin’s affinity for oxygen, which in turn inhibits hemoglobin polymerization.
“If we can interrupt that early on, we can hopefully disrupt or interrupt the biologic pathways that lead to the consequences of the disease,” she said.
The study is ongoing, as is a phase 3 clinical trial evaluating voxelotor in patients aged 12 years and older with SCD.
Voxelotor was previously known as GBT440
The study was sponsored by Global Blood Therapeutics. Dr. Hoppe reported research funding from and advisory relationships with Global Blood Therapeutics. Several coauthors are employees of the company.
SOURCE: Hoppe C et al. ASH 2017. Abstract 689
ATLANTA – Voxelotor, an investigational inhibitor of hemoglobin polymerization, demonstrated consistent and sustained improvements in hemoglobin levels in adolescents with sickle cell disease (SCD), according to first results of a phase 2b study.
A hemoglobin response of at least 1 g/dL was achieved in 55% of patients (6 of 11) at 16 weeks, in results of the ongoing open-label, 24-week study presented at the annual meeting of the American Society of Hematology.
The treatment was well tolerated, associated with mostly grade 1-2 adverse events, and also improved patient-reported symptoms, according to Carolyn C. Hoppe, MD, of University of California, San Francisco, Benioff Children’s Hospital.
at a press conference. “We need therapies to treat this disease and prevent the complications.”
Dr. Hoppe presented data on the first 12 patients in a study designed to assess the safety, pharmacokinetics, and efficacy of voxelotor in adolescent patients with homozygous hemoglobin SS or hemoglobin S beta-0 thalassemia.
All patients except one were already receiving standard care with hydroxyurea, suggesting that the two drugs may provide additive effects, according to investigators.
Treatment with voxelotor was associated with improvements in hemoglobin at 16 weeks, along with improvements in clinical measures of hemolysis – a 34% median reductions in reticulocytes and a 27% reduction in indirect bilirubin, Dr. Hoppe reported.
Patient-reported symptoms improved in 10 of 12 patients, with a median reduction of 94% in total symptom scores (TSS) at week 16, including 5 subjects with a mean TSS of 0.
Voxelotor is designed to address the cause of sickle cell disease, according to Dr. Hoppe, by modulating hemoglobin’s affinity for oxygen, which in turn inhibits hemoglobin polymerization.
“If we can interrupt that early on, we can hopefully disrupt or interrupt the biologic pathways that lead to the consequences of the disease,” she said.
The study is ongoing, as is a phase 3 clinical trial evaluating voxelotor in patients aged 12 years and older with SCD.
Voxelotor was previously known as GBT440
The study was sponsored by Global Blood Therapeutics. Dr. Hoppe reported research funding from and advisory relationships with Global Blood Therapeutics. Several coauthors are employees of the company.
SOURCE: Hoppe C et al. ASH 2017. Abstract 689
REPORTING FROM ASH 2017
Key clinical point: Voxelotor was well tolerated and demonstrated consistent and sustained improvements in hemoglobin levels in adolescents with sickle cell disease (SCD).
Major finding: A hemoglobin response of at least 1 g/dL was achieved in 55% of subjects at 16 weeks.
Data source: A phase 2a open-label, interventional study of 12 adolescent patients with sickle cell disease.
Disclosures: The study was sponsored by Global Blood Therapeutics. Dr. Hoppe reported research funding from and advisory relationships with Global Blood Therapeutics. Several coauthors are employees of the company.
Source: Hoppe C et al. ASH 2017. Abstract #689