Karyopharm Therapeutics Inc. · NASDAQ:KPTI

Karyopharm Therapeutics — Technical Presentation (May 2015)

8 slides from Karyopharm Therapeutics's technical presentation, May 2015

  1. Slide 1: Preliminary Phase II Results of Ara-C and Idarubicin with Selinexor in Relapsed or Refractory AML

    Preliminary Phase II Results of Ara-­‐C and Idarubicin in Combina9on with Selec9ve Inhibitor of Nuclear Export (SINE) Compound Selinexor (KPT-­‐330) in Pa9ents with Relapsed or Refractory AML Walter Fiedler1, Michael Heuser2, Maxim Kebenko1, Felicitas Thol2, Arne Trummer2, Chris<an Brandts3, Carsten

  2. Slide 2: Background

    Background Acute myeloid leukemia (AML) is the most frequent acute leukemia in adults. Although 70-­‐80% of pa<ents achieve a complete remission (CR), pa<ents with AML who fail to achieve a CR a[er the first cycle of induc<on therapy and those with relapsed disease have a bleak prognosis (1). Current

  3. Slide 3: Study Design

    Study Design Study Objec9ves: • This Phase II trial explores the efficacy & tolerability of Ara-­‐C and idarubicin in combina<on with selinexor in pa<ents with relapsed or refractory AML. Study Methods: • Pa<ents with relapsed/refractory AML are treated with Ara-­‐C (100 mg/m2, con<nuous infusion, day

  4. Slide 4: Figure 1: Study Flow Chart

    Figure 1: Study Flow Chart Baseline(Transla+onal(Research( EOT( Recommend:( prior(to(first(induc+on(cycle( Allogeneic(stem(cell(transplanta+on((SCT)( (op+onal):( Bone(marrow(aspirates(pre;(and( CR( CR( 24(hr;post(single(Selinexor(dose( Maintenance( 1.(Induc+on(4(weeks( 2.(Induc+on(4(weeks( Selinexor(

  5. Slide 5: Patient Characteristics

    Pa9ent Characteris9cs A' B' C' 100' Percentage'of'pa#ents'with'specified' 100' unkown' unkown' Pa#ent'characteris#cs' 90' Percentage'of'pa#ents'(%)' 90' late'relapse' unfavorable' ' 80' 80' molecular'changes'(%)' Median'age''''''''''''''''''''57'(+/H'15)' early'relapse' intermediate' 70' 70' primary'

  6. Slide 6: Safety

    Safety Drug'related'adverse'events'(percentage'of'pa6ents)' Event& CTC&grade&3+4& CTC&grade&1+2& Anemia' 18%' 0%' Diarrhea' 45%' 36%' Cons6pa6on' 0%' 18%' Nausea' 18%' 45%' Vomi6ng' 9%' 55%' Fa6gue' 18%' 27%' Thrombocytopenia' 27%' 0%' Leukopenia' 27%' 0%' Hypokalemia' 9%' 9%' Anorexia' 45%' 18%' Ou

  7. Slide 7: Efficacy

    Efficacy A" B" Percentage(of(pa4ents(receiving( Percentage(and(number(of(pa4ents(with( 100( specified(response( 90( subsequent(SCT/DLi((%)( " 80( Overall"response"rate"""""""""10"(56%)"" 70( CR(((((((((((((((((((((((((((((((((((((((((((3((17%)( 60( CRi((((((((((((((((((((((((((((((((((((((((((6((33%)(

  8. Slide 8: Conclusions and References

    Conclusions • The prognosis of relapsed/refractory AML pa9ents is remarkably poor. • Our findings suggest that treatment with Ara-­‐C and idarubicin in combina9on with selinexor shows a promising complete response rate and may serve as a bridge to transplant for pa9ents with relapsed/refractory AML.

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