Study Conclusion

1
Study Conclusion

• Alan List, MD

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Summary and Implications

• Well-characterized population
• Low/Int-1-Risk, del 5q patients
• Conventional cytogenetic testing
• Significant clinical benefit
• Durable resolution of refractory anemia
• Significant rise in hemoglobin
• Cytogenetic response and remissions
• Marrow normalization and improvements
• Well-characterized, manageable AEs

3
Sustained Transfusion Independence gt 3.5
YrUPN-105 (MDS-001)
Hemoglobin
Platelet
White blood cell
Sep-05
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Survival From MDS Diagnosis by Cytogenetics
Complexity (ITT)Del 5q (MDS-003) and Published
Data
MDS-003 N 148
Mayo Clinic N 50
100
100
80
80
5q- only (n 25)
60
60
Percent survival
50
50
P 0.012
40
40
5q- with otherabnormalities (n 25)
20
20
Isolated 5q-, N 111
Median 264 days
5q- plus 1 other, N 37
0
0
0
1
2
3
4
5
0
1
2
3
4
5
Time, yr
Time, yr
Dewald GW, et al. Blood. 198566189-197.
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• Graham Burton, MD

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RevAssistSMProposed RiskMAP

• Specifically tailored for lenalidomide
• Education
• Specialty pharmacy distribution
• Pregnancy testing for FCBP (interim requirement)

7
Lenalidomide

• Proposed label indication
• Lenalidomide is indicated for the treatment
  of patients with transfusion-dependent anemia due
  to Low- or Intermediate-1-Risk myelodysplastic
  syndromes associated with a deletion 5q
  cytogenetic abnormality with or without
  additional cytogenetic abnormalities