uniQure N.V. · NASDAQ:QURE

uniQure — ASH 2020

14 slides from uniQure's conference presentation, December 2020

  1. Slide 1: First Data from the Phase 3 HOPE-B Gene Therapy Trial

    First Data from the Phase 3 HOPE-B Gene Therapy Trial: Efficacy and Safety of Etranacogene Dezaparvovec (AAV5-Padua hFIX variant; AMT-061) in Adults with Severe or Moderate-Severe Hemophilia B Treated Irrespective of Pre-Existing Anti-Capsid Neutralizing Antibodies Steven W. Pipe, M.D.1, Michael Rec

  2. Slide 2: Goal of gene therapy in hemophilia B: Transformation of disease severity

    Goal of gene therapy in hemophilia B: Transformation of disease severity  Establish long-term benefit with sustained FIX activity from a one-time procedure  Control of bleeding with effective protection against bleeds  Elimination of the requirement for continuous prophylaxis  Improvement in qua

  3. Slide 3: Etranacogene dezaparvovec

    Etranacogene dezaparvovec  Etranacogene dezaparvovec (AAV5-Padua hFIX): Etranacogene dezaparvovec: Hyperactive FIX Padua variant  Developed by combining the AAV5 vector from AMT-060 (AAV5-WT hFIX) with the naturally occurring Padua FIX variant AAV5  Enhanced version of AMT-060, which demonstrated

  4. Slide 4: HOPE-B study design

    HOPE-B study design Screening visit Key inclusion criteria ~ 4 wks  Male adults ≥18 years Visits every 2 months,  FIX activity ≤2% of normal Lead-in phase phone calls on alternating  Continuous prophylaxis for ≥2 months (26 weeks) months ~ 4 wks Key exclusion criteria Single dose of AMT-061 Dosin

  5. Slide 5: HOPE-B study endpoints

    HOPE-B study endpoints  Primary endpoints  FIX activity (central one stage aPTT) at 26 weeks after dosing  FIX activity 52 weeks after dosing*  52-week ABR compared to lead-in*  Secondary endpoints  Rates of total, spontaneous, traumatic, and treated/untreated bleeds  FIX consumption  Correl

  6. Slide 6: Patient disposition

    Patient disposition  54 patients were dosed and completed 26-weeks of follow up 75 patients screened 8 screen failures 67 entered lead-in phase 13 patients discontinued 54 patients dosed prior to dosing (FAS population)b,c Ineligible fibroscana score, concomitant medication, co-morbidities, withdra

  7. Slide 7: Baseline demographics

    Baseline demographics Full analysis set (N = 54) Age, mean (SD, min-max), years 41.5 (15.8, 19-75) Severity of hemophilia B at time of diagnosis, n (%) Severe (FIX <1%) 44 (81.5) Moderately severe (FIX ≥1% and ≤2%) 10 (18.5) Positive HIV status, n (%) 3 (5.6) Prior hepatitis B infection, n (%) 3 (5.

  8. Slide 8: Overview of FIX activity: Up to 26 weeks (month 6)

    Overview of FIX activitya: Up to 26 weeks (month 6)  Mean (SD) FIX activity at Month 6: 37.2% (19.6); change from baseline +36.01% (19.693), p<0.0001 Q1-Q3 Median Mean Min/max 100 90 FIX activity, central one-stage (%) 80 70 60 50 40 30 20 10 0 1 2 3 4 5 6 7 8 9 10 11 12 Month 4 Month 5 Month 6 Wee

  9. Slide 9: Overview of FIX activity: Beyond 26 weeks

    Overview of FIX activitya: Beyond 26 weeks 120 Week 26 Q1-Q3 Median Mean Min/max 100 FIX activity, central one-stage (%) 80 60 Month 18 40 20 0 1 2 3 4 5 6 7 8 9 10 11 12 Month 4 Month 5 Month 6 Month 7 Month 8 Month 9 Month 10 Month 11 Month 12 Month 13 Month 14 Month 15 Month 16 Month 17 Month 18

  10. Slide 10: Pre-existing NAb titers vs week 26 FIX activity

    Pre-existing NAb titers vs week 26 FIX activitya  No correlation of pre-existing NAbs with FIX activity was identified up to a titer of 678.2  R2 = 0.078  One patient with a titer of 3212.3 did not respond (data not shown) 100 Pearson product-moment correlation (95% CI): -0.28 (-0.51, -0.00) Spea

  11. Slide 11: Bleeds in the first 26 weeks

    Bleeds in the first 26 weeks Lead-in Week 0-26 N = 54 N = 54 Total cumulative bleeds (treated), n 123 (107) 21 (10) Spontaneous (treated), n 42 (37) 7 (3) Traumatic (treated), n 66 (55) 11 (5) Other* (treated), n 15 (15) 3 (2) Patients with 0 bleeds, n (%) 16 (30%) 39 (72%) Relative to lead-in, in t

  12. Slide 12: FIX replacement use in the first 26 weeks

    FIX replacement use in the first 26 weeks Lead-in Week 0-26 N = 54 N = 54 Patient on prophylaxisa, n (%) 54 (100%) 2 (4%) 290,769 12,537a FIX usage (IU/yr/pt), mean (SD)b (170,148) (36,218)  In the Per-Protocol populationc, 98% of patients were able to discontinue prophylaxis and remain prophylaxis

  13. Slide 13: Treatment-related AEs with an incidence ≥5%

    Treatment-related AEs with an incidence ≥5% (safety population; post-treatment period)  53 patients had 324 AEs post-treatment, of AE, preferred term N = 54 n (%) which 37 patients had a total of 88 TRAE At least one related incident AEa 37 (68.5)  The majority (79.6%) of treatment-related AEs wer

  14. Slide 14: Conclusions

    Conclusions  First report of a phase 3 study in patients with Hemophilia B and the largest gene therapy trial cohort reported to date  Mean FIX activity significantly increased to near-normal levels at 26 weeks post-etranacogene dezaparvovec, meeting the first co-primary endpoint  No requirement

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