Efficacy and Safety of FG-4592 for Treatment of Anemia in Participants With Lower Risk Myelodysplastic Syndrome (MDS)

This is a Phase 2/3 trial with sequential Phase 2 and Phase 3 components. In each part, there is an up to 4 weeks screening period followed by a treatment period of 26 weeks and a 4 week follow-up period.

• FG-4592 (Drug), also known as Roxadustat, AZD9941: Oral
• Placebo (Drug): Oral

Primary outcomes

• Percentage of Participants With a Hemoglobin (Hb) Response to FG-4592 Without Transfusion [26 weeks]: Hemoglobin response to FG-4592 is defined as an increase in mean Hb of ≥1.0 grams (g)/deciliter (dL) from baseline within any 8-week period during the study without transfusion.

Secondary outcomes

• Number of Participants With Hb Increase ≥1.0 g/dL From Baseline [26 weeks]: Hb response to FG-4592 is defined as an increase in mean Hb of ≥1.0 g/dL from baseline within any 8-week period during the study without transfusion.
• Time to First Hb Response [26 weeks]: Hb response to FG-4592 is defined as an increase in mean Hb of ≥1.0 g/dL from baseline within any 8-week period during the study without transfusion.
• Change From Baseline in Mean Hb in Weeks 25 to 27 [Baseline, Weeks 25 to 27]
• Percentage of Participants With Mean Hb ≥10.0 g/dL Within Any 8-week Period During the Study Without Transfusion [26 weeks]
• Number of Participants that Require Transfusions or Hb <6 g/dL up to Week 27 [Up to Week 27]
• Mean Change From Baseline in Functional Assessment of Cancer Therapy - Anemia (FACT-An) and Functional Assessment of Chronic Illness Therapy Fatigue (FACIT-F) Scores at Week 27 [30 weeks]: Frame: Baseline, Week 27

Sex: All, ages: 18 Years to 80 Years

Key Inclusion Criteria:

* Diagnosis of primary MDS classified as very low, low or intermediate risk with <5% blasts (documented within 12 weeks prior to Day 1)
* Screening Hb <10 g/dL and ≥6g/dL
* Transfusion independent defined as no red blood cell transfusions within 12 weeks of Day 1
* Erythropoiesis-stimulating agent (ESA)-naïve (not within 30 days of Day 1)
* Eastern Cooperative Oncology Group (ECOG) performance status 0, 1, or 2

Key Exclusion Criteria:

* Diagnosis of secondary MDS
* Significant myelofibrosis (>2+fibrosis)
* Prior therapy with azacitidine, decitabine, antithymocyte globulin, cyclosporine, thalidomide, or lenalidomide within 12 weeks prior to Day 1
* Baseline erythropoietin level of >400 units (U)/liter (L)
* Clinically significant anemia due to non-MDS etiologies

Note: Other inclusion and exclusion criteria may apply.