Uniqure N.v. · NASDAQ:QURE
Uniqure — Corporate Update (September 2025)
29 slides from Uniqure's corporate update, Sep 24, 2025
Slide 1: Pivotal Phase I/II AMT-130 Huntington's Disease Update
Pivotal Phase I/II AMT-130 Huntington’s Disease Update September 24, 2025
Slide 2: Disclaimer
Disclaimer This presentation contains forward-looking statements within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. All statements other than statements of historical fact are forward-looking statements, wh
Slide 3: Opening Remarks
Opening Remarks Matt Kapusta Chief Executive Officer
Slide 4: High-dose AMT-130 met primary and key secondary endpoints at 36-months
High-dose AMT-130 met primary and key secondary endpoints at 36-months Statistically-significant 75% slowing of disease based on cUHDRS (p=0.003) Statistically-significant slowing of disease based on TFC (p=0.033) CSF NfL levels below baseline at 36 months Continues to be generally well-tolerated Al
Slide 5: Slowing progression of Huntington's disease could extend patients' quality of life
Slowing progression of Huntington’s disease could extend patients' quality of life HD is a progressive neurodegenerative disease with no disease-modifying treatments available. AMT-130 aims… Untreated disease course 1 Disease worsening To slow the rate of disease progression 2 To provide HD patients
Slide 6: AMT-130: A potential first disease modifying treatment for HD
AMT-130: A potential first disease modifying treatment for HD The construct design and targeted administration of AMT-130 provide key advantages Key AMT-130 Attributes: • One-time administration with potentially long-term effects • Precision-delivery directly to diseased areas of the brain • Minimiz
Slide 7: Topline Data from Pivotal Phase I/II Study
Topline Data from Pivotal Phase I/II Study Walid Abi-Saab, M.D. Chief Medical Officer
Slide 8: The cUHDRS is a widely used efficacy outcome measure
The Composite Unified Huntington’s Disease Rating Scale is a widely used efficacy outcome measure Composite Unified Huntington’s Disease Rating Scale (cUHDRS) Total Functional Total Motor Symbol Digit Stroop Word Capacity Score Modalities Test Reading Test (TFC) (TMS) (SDMT) (SWRT) Impairments in oc
Slide 9: Pivotal Phase I/II Study of AMT-130
Pivotal Phase I/II Study of AMT-130 Prespecified statistical analysis plan Prespecified statistical analysis plan was aligned with and submitted to the FDA in advance of database lock 12 Months 24 Months 36 Months N=12 patients with 36-months of High Dose AMT-130 Arm (N=17) follow-up as of June 30,
Slide 10: Demographic and baseline characteristics of high-dose cohort
Demographic and baseline characteristics of high-dose cohort The propensity score- Demographics and Baseline AMT-130 PSM External Control Disease Characteristics High-Dose (Enroll-HD) matched external control Mean (SD) (N=17) (N=940) has well-matched Sex, Males (%) 47.1 55.6 baseline characteristics
Slide 11: cUHDRS Change from Baseline at 36 Months
cUHDRS Change from Baseline at 36 Months AMT-130 high-dose AMT-130 High-Dose Enroll-HD PSM External Control significantly reduced N=12 N = 568 0.0 HD progression by 75% -0.38 based on cUHDRS at 36 months -0.5 -1.52 Disease worsening -1.0 -1.5 p = 0.003 -2.0 Participants Baseline 36 months AMT-130 Hi
Slide 12: cUHDRS Change from Baseline Through Months
cUHDRS Change from Baseline Through 36 Months AMT-130 high-dose 1.0 significantly reduced HD 0.5 progression by 75% Disease worsening based on cUHDRS at 0.0 36 months -0.5 -1.0 -1.5 -2.0 1 2 3 4 Participants Baseline 12 months 24 months 36 months AMT-130 17 17 15 12 High-Dose PSM External 940 715 58
Slide 13: TFC Change from Baseline at 36 Months
TFC Change from Baseline at 36 Months AMT-130 high-dose AMT-130 High-Dose Enroll-HD PSM External Control significantly reduced N=12 N=583 0.0 HD progression by 60% based on TFC at 36 -0.36 months -0.2 Disease worsening -0.5 -0.88 -1.0 p = 0.033 Participants Baseline 36 months AMT-130 High-Dose 17 12
Slide 14: TFC Change from Baseline Through Months
TFC Change from Baseline Through 36 months AMT-130 high-dose 0.2 significantly reduced HD progression by 60% 0.0 Disease worsening based on TFC at 36 -0.2 months -0.4 -0.6 -0.8 -1.0 0 1 2 3 Participants Baseline 12 months 24 months 36 months AMT-130 17 17 15 12 High-Dose PSM External 940 725 597 583
Slide 15: High-dose showed trends supportive of disease slowing across cUHDRS subdomains
AMT-130 high-dose showed trends supportive of disease slowing across all other clinical subdomains of cUHDRS SDMT Change from Baseline at 36 Months SWRT Change from Baseline at 36 Months TMS Change from Baseline at 36 Months 5.0 5.0 5.0 4.88 3.0 3.0 3.0 Disease worsening Disease worsening Disease wo
Slide 16: Low-dose AMT-130 results at 36-months
Low-dose AMT-130 results at 36-months LSM Difference in Change Patients receiving AMT-130 Low- Enroll-HD PSM from Baseline, Slowing of Dose (N=12) External Control AMT-130 vs. Disease low-dose AMT-130 Low-Dose Enroll-HD Progression P-value showed variable LSM Change (SE) LSM Change (SE) PSM External
Slide 17: Independent study confirmed association between CSF NfL levels and HD severity
Relationship Between NfL and Age in HD An independent study has confirmed a strong association between CSF NfL levels and the clinical severity of HD The study demonstrated early-manifest HD patients will experience increases in CSF NfL of ~10% to 15% per year Recent data from HD-CSF study where CSF
Slide 18: CSF NfL change from baseline at 36 months
10 AMT-130 high dose CSF NfL Change from baseline at 36 months Change in Mean CSF NfL from Baseline (%) demonstrated a 5 reduction of CSF NfL at 36 months vs. baseline. 0 Disease worsening - 4.7 - 8.2 -5 -10 AMT-130 Low Dose N=12 -15 AMT-130 High Dose N=11* *1 of 12 patients declined to undergo a lu
Slide 19: AMT-130 remained generally well-tolerated
AMT-130 remained generally well-tolerated The majority of AMT-130 remained drug-related serious No new drug- generally well- adverse events related serious tolerated, with a occurred within the adverse events manageable safety first weeks post have been observed profile at both treatment and fully s
Slide 20: AMT-130 remained generally well-tolerated (adverse event table)
AMT-130 remained generally well-tolerated Low-dose AMT-130 High-dose AMT-130 Dose -Blinded All AMT-130 Sham Surgery (Cohort 1) (Cohort 2) (Cohort 3) (Cohorts 1, 2 and 3) (n=10) (n=13&) (n=20&) (n= 12) (n=45&) N (%) N (%) N (%) N (%) N (%) Any TEAEs 10 100.0 12 92.3 20 100.0 12 100 44 97.8 Any SAEs 1
Slide 21: Regulatory next steps
Regulatory next steps 4Q 25 - Hold pre-BLA meeting with the FDA 1Q 26 - Expected BLA submission for AMT-130 with a request for priority review Abbreviations: FDA, Food & Drug Administration; BLA, Biologics License Application. LEADERSHIP IN GENE THERAPY SEPTEMBER 24, 2025 | 21
Slide 22: Positive topline data from pivotal Phase I/II study
Positive topline data from pivotal Phase I/II study High-dose AMT-130 met its primary and key secondary endpoints at 36 months, with favorable trends observed across additional clinical and supportive endpoints 1 Statistically-significant 75% slowing of disease progression based on cUHDRS 2 Statisti
Slide 23: Key Opinion Leader Perspective
Key Opinion Leader Perspective Sarah Tabrizi, M.D., FRCP, FRS, FMedSci, Ph.D., University College London
Slide 24: Commercialization
Commercialization Kylie O’Keefe Chief Customer & Strategy Officer
Slide 25: Large anticipated total addressable US market at time of launch
Large anticipated total addressable US market at time of launch, with potential for further growth Potential Long-term Growth Levers ~100,000 patients Genetically Identifiable HD • Continued Disease Progression In Presymptomatic And Incident Patients ~40,000 patients Symptomatic HD • Increased Diagn
Slide 26: Established HDSA COEs and surgical infrastructure well positioned to serve HD patients
Established HDSA COEs and surgical infrastructure are well positioned to serve HD patients, with ongoing efforts to prioritize key treatment centers for launch Heat Map of Clearpoint Neuro Capable Facilities and HDSA COEs by Patient Volume Abbreviations: HDSA, Huntington’s disease Society of America
Slide 27: Research Analyst Questions
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Slide 28: Closing Remarks
Closing Remarks Matt Kapusta Chief Executive Officer
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