uniQure N.V. · NASDAQ:QURE
uniQure — EAHAD 2022
14 slides from uniQure's conference presentation, Feb 4, 2022
Slide 1: Final Analysis from the Pivotal Phase 3 HOPE-B Gene Therapy Trial
FINAL ANALYSIS FROM THE PIVOTAL PHASE 3 HOPE-B GENE THERAPY TRIAL: STABLE STEADY-STATE EFFICACY AND SAFETY OF ETRANACOGENE DEZAPARVOVEC IN ADULTS WITH SEVERE OR MODERATELY SEVERE HAEMOPHILIA B Wolfgang Miesbach University Hospital Frankfurt, Frankfurt, Germany EAHAD 2 0 2 2
Slide 2: Final Analysis from the Pivotal Phase 3 HOPE-B Gene Therapy Trial: Authors
FINAL ANALYSIS FROM THE PIVOTAL PHASE 3 HOPE-B GENE THERAPY TRIAL: STABLE STEADY-STATE EFFICACY AND SAFETY OF ETRANACOGENE DEZAPARVOVEC IN ADULTS WITH SEVERE OR MODERATELY SEVERE HAEMOPHILIA B Wolfgang Miesbach1, Frank W.G. Leebeek2, Michael Recht3, Nigel S. Key4, Susan Lattimore3, Giancarlo Castama
Slide 3: Disclosure for Wolfgang Miesbach
DISCLOSURE FOR WOLFGANG MIESBACH Shareholder No relevant conflicts of interest to declare Bayer, Chugai, Novo Nordisk, Octapharma, Pfizer, Grant/Research Support Takeda/Shire, UniQure Bayer, Biomarin, Biotest, CSL Behring, Chugai, Consultant Freeline, LFB, Novo Nordisk, Octapharma, Pfizer, Roche, Sa
Slide 4: Aim of Gene Therapy and Etranacogene Dezaparvovec
AIM OF GENE THERAPY AND ETRANACOGENE DEZAPARVOVEC Aim of etranacogene dezaparvovec: Provide long-term circulating FIX activity after a single injection; ameliorate the severe bleeding phenotype; eliminate the need for continuous prophylaxis AMT-060 Etranacogene dezaparvovec (AAV5-hFIX) (AAV5-Padua h
Slide 5: HOPE-B Study Design
HOPE-B STUDY DESIGN ≥6-month Etranacogene Post-treatment lead-in period dezaparvovec follow-up* Screening administration SOC continuous 2x10 13 gc/kg Weekly Monthly visits Twice-yearly visits FIX prophylaxis dose visits No prophylactic immunosuppression Primary efficacy endpoint (Months 7–18) 0 1 12
Slide 6: Trial Endpoints and Inclusion Criteria
TRIAL ENDPOINTS AND INCLUSION CRITERIA Primary endpoints Key inclusion criteria • ABR (all bleeds) comparison between AMT-061 and • Male adults aged ≥18 years prophylaxis for non-inferiority between the lead-in • FIX activity ≤2% of normal phase and the 52 weeks following stable FIX • Had received c
Slide 7: Participant Disposition and Baseline Characteristics
PARTICIPANT DISPOSITION AND BASELINE CHARACTERISTICS Full analysis set (N=54) Age, mean (SD, min-max), years 41.5 (15.8, 19–75) Severity of HB at 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,
Slide 8: Primary Endpoint Was Met with a Significant Improvement in ABR
PRIMARY ENDPOINT WAS MET WITH A SIGNIFICANT IMPROVEMENT IN ABR 5 4.5 4.19 64% 4 reduction All-bleeds ABR substantially reduced after single 3.65 3.5 in ABR treatment with etranacogene dezaparvovec p=0.0002 Mean ABR 3 2.5 2.35 2 1.51 1.52 1.5 1 0.84 0.44 0.51 0.5 0 ABR (all bleeds) Treated bleeds AsB
Slide 9: Sustained FIX Activity Levels up to 18 Months Post-Treatment
SUSTAINED FIX ACTIVITY LEVELS UP TO 18 MONTHS POST-TREATMENT 125 100 One-stage aPTT FIX activity (%) 75 50 25 0 Baseline W3 W4 W5 W6 W7 W8 W9 W10 W11 W12 M4 M5 M6 M7 M8 M9 M10 M11 M12 M18 [a] Time (Week) Number of Primary efficacy endpoint subjects 54 43 49 51 47 46 48 46 44 46 51 48 45 51 47 45 51
Slide 10: FIX Replacement Use Significantly Decreased from Baseline
FIX REPLACEMENT USE SIGNIFICANTLY DECREASED FROM BASELINE Significant reduction in FIX consumption from lead-in to 7–18 months post-treatment, mean (SD) difference in FIX consumption was –248,825 (21,102) IU/year/participant (P<0.0001) 450,000 257,339 FIX consumption for 54 participants 400,000 • 96
Slide 11: Safety Profile Is Consistent with Previously Presented HOPE-B Data
SAFETY PROFILE IS CONSISTENT WITH PREVIOUSLY PRESENTED HOPE-B DATA Most frequent TRAE Post-treatment events N, % ALT* 9 (16.7) • 92 TRAEs: 74 (80.4%) were mild, 16 (17.4%) moderate, and Headache 8 (14.8) two (2%) severe Influenza-like illness 7 (13.0) • Transaminase increase in nine participants • A
Slide 12: Participants Saw an Improvement in Quality-of-Life Score
PARTICIPANTS SAW AN IMPROVEMENT IN QUALITY-OF-LIFE SCORE Domain Mean in lead-in Mean in post- Mean difference Percentage change period (SE)* treatment period between treatment p-value** (SE)* periods (SE)* Total 25.56 (2.072) 20.06 (2.054) -5.50 (0.972) <0.0001 21.5% Feelings 20.61 (2.838) 11.19 (2.
Slide 13: Conclusions
CONCLUSIONS Safety profile is consistent with previously ABR of all bleed subtypes substantially reduced reported data from HOPE-B after achievement of stable FIX expression Mean FIX activity levels of 36.9 IU/dL have Improvement in quality of life (Haem-A-QoL), been sustained at 18 months post-trea
Slide 14: Thank You to the Study Participants, Investigative Site Teams and the Sponsor
THANK YOU TO THE STUDY PARTICIPANTS, INVESTIGATIVE SITE TEAMS AND THE SPONSOR STUDY TEAM MEMBERS HTTPS://WWW.CSLBEHRING.COM/-/MEDIA/SHARED/DOCUMENTS/HOPE-B-PH-III-DATA-18-MONTH-LAY-SUMMARY.PDF EAHAD 2 0 2 2