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 was 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 (n=92), or placebo (n=92). Randomization was stratified by patients already receiving hydroxyurea (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

Significantly more patients had an increase in Hb levels compared to placebo during the HOPE trial1

51%
of patients receiving Oxbryta achieved a >1 g/dL increase in Hb (compared to baseline) vs 7% in the placebo group (P<0.001)1
Change in Hb for individual patient at Week 241*
efficacy-clinical-graph efficacy-clinical-graph
Some patients receiving Oxbryta had increases in Hb beyond the primary endpoint3
Increased by >2 g/dL
27%
Oxbryta
vs
1%
Placebo
Responses in Hb levels are measurable and verifiable in sickle cell disease patients1
*Patients who completed 24 weeks of treatment (approximately 82% of all randomized patients).
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.1
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 by (HbA, HbS, and HbF) HPLC. If precise quantitation of Hb species is required, chromatography should be performed when the patient is not receiving Oxbryta therapy.

For additional information and
results from the Phase 3 HOPE trial:

Oxbryta demonstrated an observed Hb response vs placebo regardless of1:

age
geographic region
HU use
efficacy-clinical-graph2 efficacy-clinical-graph2
The results shown are from an exploratory analysis using the prespecified subgroups. There was no hierarchical testing conducted for the subgroups, and sample size, data, and results should be considered and interpreted with caution.
*Patients who completed 24 weeks of treatment (approximately 82% of all randomized patients).
Results from a multicenter, randomized, double-blind, placebo-controlled, parallel group 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.

Oxbryta improved hemolysis in the Phase 3 HOPE trial1

Adjusted mean (SE) change in Hb and clinical measurements of hemolysis—baseline to Week 24
 

Oxbryta

1,500 mg
QD, n=90

Placebo

n=92

P Value

Hemoglobin

1.1
g/dL

(0.1)

-0.1
g/dL

(0.1)

<0.001

Indirect
bilirubin

-29.1%
(3.5)

-3.2%
(3.5)

<0.001

Percent reticulocyte count

-19.9%
(4.6)

4.5%
(4.6)

<0.001

SE = standard error; QD = once daily
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.

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