REDUCE OR
ELIMINATE TRANSFUSIONS

The efficacy of Reblozyl® in MDS is based on the double-blind, randomized, placebo-controlled, phase 3 MEDALIST trial (with 153 patients treated with Reblozyl® and 76 patients receiving placebo).1

Significantly more patients experienced RBC transfusion independence (TI) with Reblozyl® vs placebo. 1

  • 37.9% achieved TI for at least 8 weeks (vs placebo 13.2%, P<0.0001) during Weeks 1-24 1
  • 48% achieved TI for at least 8 weeks (vs placebo 16%, p=0.0001) over the entire period of treatment

Significantly more patients on Reblozyl®experienced a longer RBC-TI period vs placebo. 1

  • 28.1% achieved TI for at least 12 weeks (vs placebo 7.9%, P=0.0002) during Weeks 1-24 1
  • 33.3% achieved TI for at least 12 weeks (vs placebo 11.8%, P=0.0003) during Weeks 1-48 1

Patients receiving Reblozyl® had fewer RBC transfusion events vs placebo during Weeks 1-24 and Weeks 25-48 1

During Weeks 1-24:

  • With Reblozyl®, patients had 6.26 transfusion events vs 9.2 with placebo (0.68 relative risk vs placebo) 1

During Weeks 25-48:

  • With Reblozyl®, patients had 6.27 transfusion events vs 8.72 with placebo (0.72 relative risk vs placebo)1

Patients receiving Reblozyl® required fewer RBC transfusion units vs placebo during Weeks 1-24 and Weeks 25-48 1

During Weeks 1-24:

  • Of patients with a low baseline transfusion burden (<6 units/8 weeks), those receiving Reblozyl® received 7.2 units vs 12.8 units with placebo
  • Of patients with a high baseline transfusion burden (≥6 units/8 weeks), those receiving Reblozyl® received 18.9 units vs 23.7 units with placebo

During Weeks 25-48:

  • Of patients with a low baseline transfusion burden (<6 units/8 weeks), those receiving Reblozyl® received 7.5 units vs 11.8 units with placebo
  • Of patients with a high baseline transfusion burden (≥6 units/8 weeks), those receiving Reblozyl® received 19.6 units vs 22.9 units with placebo

Primary Endpoint: RBC transfusion independence (TI)
≥8 weeks • Weeks 1-241

Significantly more patients experienced RBC-TI with Reblozyl® vs placebo

Longer term Follow-up: RBC transfusion independence
≥8 weeks (over the entire period of treatment)2

Key Secondary Endpoint: RBC-TI ≥12 weeks • Weeks 1-481

Significantly more patients experienced a longer RBC-TI period with Reblozyl® vs placebo

Key Secondary Endpoint: RBC transfusion event frequency •
Weeks 1-241

Key Secondary Endpoint: RBC transfusion units • Weeks 1-241

INCREASE HEMATOLOGICAL IMPROVEMENT-ERYTHROID

More patients receiving Reblozyl® achieved a hematologic improvement through Week 48 vs placebo1

During Weeks 1-48:

  • 58.8% of patients achieved mHI-E vs 17.1% for placebo1.
    • 69.6% achieved a mean Hb increase of at least 1.5 g/dL for 8 weeks vs 5.0% for placebo1,c.
    • 54.2% achieved a RBC transfusion reduction of 4 units/8 weeks vs 21.4% for placebo1,c.

Exploratory Endpoint: Modified hematologic improvement-erythroid (mHI-E)a,b,c Weeks 1-481

More patients achieved mHI-E at Week 48 with Reblozyl® vs placebo

aResponse criteria were developed by the IWG.

bThe proportion of patients meeting the HI-E criteria as per IWG 2006 criteria sustained over a consecutive 56-day period during the indicated treatment period.

cIn patients with a baseline transfusion burden of ≥4 units/8 weeks, mHI-E was defined as a reduction in RBC transfusions of at least 4 units/8 weeks. For patients with a baseline RBC burden of <4 units/8 weeks, mHI-E was defined as a mean increase in hemoglobin of ≥1.5 g/dL for 8 weeks in the absence of transfusions.

PROVIDES CLINICAL BENEFIT

More patients receiving Reblozyl® achieved a clinical benefit over the entire treatment period.

  • Over the entire treatment period, 64% of patients treated with Reblozyl® achieved clinical benefit versus 26% for placebo
  • Clinical benefit is defined as transfusion independence for ≥ 8 weeks and/or modified hematological improvements on erythroid line as per IWG 20062

Clinical Benefit over the entire treatment period

RAPID AND
SUSTAINED RESPONSE

Response to Reblozyl® treatment was rapid and sustained.1

  • The median duration of the longest single episode of RBC-TI among responders in the Reblozyl® treatment arm was 30.6 weeks1
  • 69.9% of Reblozyl® responders had 2 or more episodes of response
  • The cumulative duration of response for patients achieving TI ≥ 8 weeks anytime during treatment, is 80 weeks (~1.5 years)
  • The cumulative duration of response for patients achieving a clinical benefit over the entire period of treatment is 92 weeks (~2 years)

Reblozyl® treatment response

69.9% of Reblozyl®responders had 2 or more episodes of RBC-TI2,7

IMPACT ON
SERUM FERRITIN LEVELS

Lower mean change in serum ferritin levels were observed from baseline with Reblozyl® compared with placebo.1

  • At Weeks 9-24, mean serum ferritin had increased 9.9 μg/L with Reblozyl® vs an increase of 190 μg/L with placebo
    • The least square mean difference (SE) was –180.1 μg/L

Exploratory Endpoint: Impact on serum ferritin levels with Reblozyl®1

Erythroid maturation for significant red blood cell (RBC) transfusion reduction

Primary Endpoint: RBC transfusion
independence (TI) ≥8 weeks •
Weeks 1-241

Significantly more patients experienced RBC-TI with Reblozyl® vs placebo

Longer term Follow-up: RBC transfusion independence ≥8 weeks (over the entire period of treatment)2

The efficacy of Reblozyl® in MDS is based on the double-blind, randomized, placebo-controlled, phase 3 MEDALIST trial (with 153 patients treated with Reblozyl® and 76 patients receiving placebo).1

Key Secondary Endpoint: RBC-TI ≥12 weeks • Weeks 1-481

Significantly more patients experienced a longer RBC-TI period with Reblozyl® vs placebo

  • 28.1% achieved TI for at least 12 weeks (vs placebo 7.9%, P=0.0002) during Weeks 1-24 1
  • 33.3% achieved TI for at least 12 weeks (vs placebo 11.8%, P=0.0003) during Weeks 1-48 1

Key Secondary Endpoint: RBC transfusion event frequency • Weeks 1-241

Patients receiving Reblozyl® had fewer RBC transfusion events vs placebo during Weeks 1-24 and Weeks 25-481

During Weeks 1-24:

  • With Reblozyl®, patients had 6.26 transfusion events vs 9.2 with placebo (0.68 relative risk vs placebo) 1

During Weeks 25-48:

  • With Reblozyl®, patients had 6.27 transfusion events vs 8.72 with placebo (0.72 relative risk vs placebo)1

Key Secondary Endpoint: RBC transfusion units • Weeks 1-241

Patients receiving Reblozyl® required fewer RBC transfusion units vs placebo during Weeks 1-24 and Weeks 25-481

Low baseline transfusion burden (<6 units/8 weeks)

High baseline transfusion burden (≥6 units/8 weeks)

During Weeks 1-24:

  • Of patients with a low baseline transfusion burden (<6 units/8 weeks), those receiving Reblozyl® received 7.2 units vs 12.8 units with placebo
  • Of patients with a high baseline transfusion burden (≥6 units/8 weeks), those receiving Reblozyl® received 18.9 units vs 23.7 units with placebo

During Weeks 25-48:

  • Of patients with a low baseline transfusion burden (<6 units/8 weeks), those receiving Reblozyl® received 7.5 units vs 11.8 units with placebo
  • Of patients with a high baseline transfusion burden (≥6 units/8 weeks), those receiving Reblozyl® received 19.6 units vs 22.9 units with placebo

Erythroid maturation for achieving hematologic improvement

More patients on Reblozyl® achieved a hematologic improvement, according to the International Working Group (IWG) 2006 response criteria, during Weeks 1-24.

Exploratory Endpoint: Modified hematologic improvement-erythroid (mHI-E)a,b,c Weeks 1-481

More patients achieved mHI-E at Week 48 with Reblozyl® vs placebo

aResponse criteria were developed by the IWG.

bThe proportion of patients meeting the HI-E criteria as per IWG 2006 criteria sustained over a consecutive 56-day period during the indicated treatment period.

cIn patients with a baseline transfusion burden of ≥4 units/8 weeks, mHI-E was defined as a reduction in RBC transfusions of at least 4 units/8 weeks. For patients with a baseline RBC burden of <4 units/8 weeks, mHI-E was defined as a mean increase in hemoglobin of ≥1.5 g/dL for 8 weeks in the absence of transfusions.

More patients receiving Reblozyl® achieved a clinical benefit over the entire treatment period.

Clinical Benefit over the entire treatment period

  • Over the entire treatment period, 64% of patients treated with Reblozyl® achieved clinical benefit versus 26% for placebo
  • Clinical benefit is defined as transfusion independence for ≥ 8 weeks and/or modified hematological improvements on erythroid line as per IWG 20062

Response to Reblozyl® treatment was rapid and sustained.

Reblozyl® treatment response

  • The median duration of the longest single episode of RBC-TI among responders in the Reblozyl treatment arm was 30.6 weeks1
  • With Reblozyl®, hemoglobin began to increase within 7 days from treatment initiation in patients who received <4 units of RBC transfusions within 8 weeks prior to the study 1

69.9% of Reblozyl®responders had 2 or more episodes of RBC-TI2,7

Lower mean change in serum ferritin levels were observed from baseline with Reblozyl® compared with placebo.1

Exploratory Endpoint: Impact on serum ferritin levels with Reblozyl®1

  • At Weeks 9-24, mean serum ferritin had increased 9.9 μg/L with Reblozyl® vs an increase of 190 μg/L with placebo
    • The least square mean difference (SE) was –180.1 μg/L