Oxbryta directly inhibits polymerization to improve anemia and reduce hemolysis1,2

Studied in pediatric (12-17) and adult (>17) patients with sickle cell disease (SCD) in the HOPE trial1

Oxbryta tablets were studied in a multicenter, randomized, double-blind, placebo-controlled (Phase 3) trial of 274 patients aged 12-64 years.

Patients were randomized to either 1,500 mg/day of Oxbryta (n=90), 900 mg/day of Oxbryta tablets (n=92), or placebo (n=92). Randomization was stratified by patients already receiving xydroxyurea (HU), geographic region, and age.

Primary Endpoint1
  • Percentage of patients who had a hemoglobin (Hb) response (defined as an increase of >1 g/dL) from baseline to Week 24
Secondary Endpoints1,2
  • Change in Hb level from baseline at Week 24
  • Change in laboratory markers associated with hemolysis (indirect bilirubin level and percentage of reticulocytes) from baseline at 24 weeks

Patient Details

Select inclusion criteria1
  • Baseline Hb of 5.5-10.5 g/dL
  • 1-10 vaso-occlusive crisis (VOC) events within the last 12 months
  • Eligible patients on stable doses of HU for at least 90 days were allowed to continue HU therapy throughout the study
Baseline demographics for patients in the Phase 3 HOPE trial1

Characteristic

Patients

Receiving background
HU therapy

65%

Geographic region

North America, Europe, other

Median age

24 years (12 to 64 years)

HbSS or HbSβ0
thalassemia genotype

90%

Pediatric patients
12 to <17 years

17%

Median baseline Hb

8.5 g/dL (5.9 to 10.8 g/dL)

1 VOC event in 12 months prior to enrollment

42%

2–10 VOC events in
12 months prior to enrollment

58%

HbSS = homozygous hemoglobin S; HbSβ0 thalassemia = hemoglobin sickle beta thalassemia
 = stratified

Oxbryta significantly increased Hb levels in the Phase 3 HOPE trial1

51%
of patients (from the intent-to-treat* population) receiving Oxbryta achieved a >1 g/dL increase in Hb (compared to baseline) vs 7% in the placebo group (P<0.001)1

59%
of patients (from the per-protocol population) receiving Oxbryta achieved a >1 g/dL increase in Hb (compared to baseline) vs 10% in the placebo group (P<0.001)3
Change in Hb for individual patient at Week 241†
Chart of change in Hb for individual patient at Week 24. Chart of change in Hb for individual patient at Week 24.
Some patients receiving Oxbryta had increases in Hb beyond the primary endpoint3
Increased by >2 g/dL
27%
Oxbryta
vs
1%
Placebo
*Intent-to-treat: Included all randomized patients.3
Per-protocol: Included randomized patients who completed 24 weeks of study drug and adhered to defined trial protocols (approximately 83% of all randomized patients).1,3
Results from a multicenter, randomized, double-blind, placebo-controlled parallel group study of 274 patients, aged 12-64 years, with SCD (a majority with HbSS or HbSβ0 thalassemia), conducted in 3 groups of study participants over a 24-week period. All data in this section from the per-protocol analysis unless otherwise indicated.1,3

Increases in hemoglobin (Hb) maintained through Week 72 in the HOPE trial4

Mean change in Hb from baseline through Week 72 in the HOPE trial3,4

Chart of Placebo-adjusted LS mean (95% Cl) change from baseline in indirect bilirubin Chart of Placebo-adjusted LS mean (95% Cl) change from baseline in indirect bilirubin

The change from baseline to Week 24 represents a secondary endpoint and the change from baseline to Week 72 was a prespecified, exploratory endpoint2,4

Selected Safety Information
Warnings and precautions
Hypersensitivity Reactions

Serious hypersensitivity reactions after administration of OXBRYTA have occurred in <1% of patients treated. Clinical manifestations may include generalized rash, urticaria, mild shortness of breath, mild facial swelling, and eosinophilia.

If hypersensitivity reactions occur, discontinue OXBRYTA and administer appropriate medical therapy. Do not reinitiate OXBRYTA in patients who experience these symptoms with previous use.

Laboratory Test Interference

OXBRYTA administration may interfere with measurement of Hb subtypes (HbA, HbS, and HbF) by high-performance liquid chromatography (HPLC). If precise quantitation of Hb species is required, chromatography should be performed when the patient has not received OXBRYTA therapy in the immediately preceding 10 days.

Oxbryta provided a mean incremental increase in Hb from baseline regardless of baseline HU use3

Adjusted mean (95% CI) change in Hb from baseline stratified by baseline HU use
results-24-72-weeks results-24-72-weeks

Oxbryta reduced hemolysis in the Phase 3 HOPE trial1

Placebo-adjusted LS mean (95% CI) change from baseline in indirect bilirubin1-4
Chart of placebo-adjusted LS mean (95% Cl) change from baseline in indirect bilirubin. Chart of placebo-adjusted LS mean (95% Cl) change from baseline in indirect bilirubin.
Results from a multicenter, randomized, double-blind, placebo-controlled, parallel group study of 274 patients, aged 12-65 years, with SCD (a majority with HbSS or HbSβ0 thalassemia), conducted in 3 groups of study participants over a 24-week period (pivotal analysis) and a 72-week period (prespecified end-of-study endpoint).1,4
CI = confidence interval; LS = least squares
Responses are measurable and verifiable in patients with SCD1,3

Oxbryta reduced hemolysis in the Phase 3 HOPE trial1

Placebo-adjusted LS mean (95% CI) change from baseline in percent reticulocyte count1-4
Chart of placebo-adjusted LS mean (95% Cl) change from baseline in percent reticulocyte count. Chart of placebo-adjusted LS mean (95% Cl) change from baseline in percent reticulocyte count.
Results from a multicenter, randomized, double-blind, placebo-controlled, parallel group study of 274 patients, aged 12-64 years, with SCD (a majority with HbSS or HbSβ0 thalassemia), conducted in 3 groups of study participants over a 24-week period (pivotal analysis) and a 72-week period (prespecified end-of-study endpoint).1,4
CI = confidence interval; LS = least squares
Responses are measurable and verifiable in patients with SCD1,3

Studied in pediatric patients ages 4 to <12 years with sickle cell disease (SCD) in the Phase 2 HOPE-KIDS 1 trial1,5

Oxbryta was studied in an open-label, multicenter, Phase 2 study of 45 patients.

Patients aged 4 to <12 years received Oxbryta (voxelotor) tablets for oral suspension based on body weight at baseline.

  • Doses of 600 mg, 900 mg, or 1,500 mg once daily were administered to patients weighing 10 kg to <20 kg, 20 kg to <40 kg, or ≥40 kg, respectively
    • 35 patients received Oxbryta for 24 weeks
    • 26 patients received Oxbryta for 48 weeks
Primary Outcome1,5
  • Percentage of patients who achieved a response, defined as an Hb increase of >1 g/dL from baseline to Week 24
Secondary Outcome3,5
  • Percent change in clinical measures of hemolysis from baseline to Week 24

Patient Details

Select inclusion criteria1
  • Baseline Hb of ≤10.5 g/dL
  • Eligible patients on stable doses of hydroxyurea ≥90 days were allowed to continue hydroxyurea therapy throughout the study
Select baseline demographics1
  • Median age was 8 years (range: 4-15 years)
    • 45 (80%) of patients were 4 to <12 years of age
  • 80% received background hydroxyurea therapy
  • Patients had a mean baseline of 8.6 g/dL (range: 6.1-10.5 g/dL)
  • All patients had HbSS or HbSβ0 thalassemia genotype (100%)

Oxbryta significantly increased Hb levels in the HOPE-KIDS 1 trial1,3,5

36%
of patients (from the intent-to-treat* population) receiving Oxbryta achieved a response, defined as a Hb increase of >1 g/dL from baseline to Week 24 (95% CI: 21.6%, 49.5%)1,3,5

47%
of patients (from the per-protocol population) receiving Oxbryta achieved a response, defined as a Hb increase of >1 g/dL from baseline to Week 24 (95% CI: 29.8%, 64.9%)1,3,5
Change in Hb for individual patients 4 to <12 at Week 243
Change in Hb for individual patients 4 to <12 at Week 24 Change in Hb for individual patients 4 to <12 at Week 24
At Week 24 vs baseline
  • 35% of patients receiving Oxbryta achieved >1.5 g/dL increase in Hb from baseline3
  • 21% of patients receiving Oxbryta achieved >2.0 g/dL increase in Hb from baseline3
+1.0 g/dL mean increase in Hb levels above background HU benefits was achieved by those patients receiving Oxbryta who were already on stable HU therapy1,3
Results from an open-label, multicenter, Phase 2 study of 45 patients with SCD. Patients aged 4 to <12 years received Oxbryta tablets for oral suspension based on body weight at baseline. Doses of 600 mg, 900 mg, or 1,500 mg once daily were administered to patients weighing 10 kg to <20 kg, 20 kg to <40 kg, or ≥40 kg, respectively. All data presented in this section from the per-protocol analysis unless otherwise indicated.1,3,5
*Intent-to-treat: Included all patients who enrolled and received at least 1 dose of study drug regardless of adherence to defined trial protocols.3
Per-protocol: Included patients who completed 24 weeks of study drug and had a Hb measurement available at Week 24.3

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