uniQure N.V. · NASDAQ:QURE
uniQure — Investor Presentation (October 2019)
24 slides from uniQure's investor presentation, October 2019
Slide 1: Corporate Presentation October 2019
Corporate Presentation October 2019 DELIVERING GENE THERAPY TO PATIENTS OCTOBER 2019 | 1
Slide 2: Forward-looking Statements
Forward-looking Statements This presentation contains forward-looking statements. All statements other than statements of historical fact are forward-looking statements, which are often indicated by terms such as “anticipate,” “believe,” “could,” “estimate,” “expect,” “goal,” “intend,” “look forward
Slide 3: Key corporate achievements and capabilities
Key corporate achievements and capabilities • Demonstrated clinical proof of concept in Hemophilia B; achievement of patient enrollment in HOPE-B pivotal study, with potential to be first- and best-in-class • Proprietary CNS platform with lead program AMT-130 in Huntington’s disease expected to init
Slide 4: Our proprietary pipeline
Our proprietary pipeline D E L I V E Rwith *Collaboration I N GBristol-Myers GENE TH ERAPY TO PATIENTS Squibb OCTOBER 2019 | 4
Slide 5: Leading the way in AAV manufacturing
Leading the way in AAV manufacturing Large-scale AAV Manufacturing • Based in Lexington, MA, expanded to 80,000 ft2 • Proprietary 3rd generation insect cell, baculovirus • Demonstrated 500L stirred-tank production • Scalable up to 2 x 2,000L • Strong intellectual property position Potential Benefits
Slide 6: Leveraging AAV5: a potentially best-in-class vector
Leveraging AAV5: a potentially best-in-class vector AAV5 – Clinically demonstrated tolerability and clinical effects AAV5 Vector observed to date • Long-term follow-up data demonstrating safety and tolerability • 25 patients have received AAV5 across 4 clinical studies1 • Observed clinical effects i
Slide 7: Hemophilia B
Hemophilia B Etranacogene dezaparvovec (AMT-061) DELIVERING GENE THERAPY TO PATIENTS OCTOBER 2019 | 7
Slide 8: FIX activity one-stage aPTT
Etranacogene dezaparvovec (EtranaDez): FIX activity at 36 weeks post-treatment in Phase 2b study Increases in FIX activity up to 54% of normal Main Efficacy Findings: Mean FIX activity at 36 weeks of 45% of normal • Sustained increases in FIX activity Participant 1 • No bleeding events post-treatmen
Slide 9: Etranacogene dezaparvovec (EtranaDez): HOPE-B Phase III pivotal study
Etranacogene dezaparvovec (EtranaDez): HOPE-B Phase III pivotal study • Achieved enrollment of 62 patients with severe and moderately-severe Hemophilia B • Open label, single-dose, multi-center, multi-national trial • Patients with AAV5 neutralizing antibodies not excluded • Patients serve as their
Slide 10: Huntington's Disease
Huntington’s Disease AMT-130 DELIVERING GENE THERAPY TO PATIENTS OCTOBER 2 0 1 9 | 10
Slide 11: Executing in Huntington's Disease
Executing in Huntington’s Disease Target product profile • One-time administration of disease-modifying therapy • Proprietary miQURETM silencing platform Huntington’s AMT-130 • Strong mutant HTT knockdown in deep structures and cortex • 3-7 per 100K people1 • Targets full length HTT protein aggregat
Slide 12: Huntington's disease: expected progression of brain pathology
Huntington’s disease: expected progression of brain pathology Somatomotor cortex • The striatum is the primary site of Frontal Somatosensory lobe pathology cortex 2 Parietal 3 3 lobe • Premanifest stage: atrophy spreads and cortical thinning occurs 1 Occipital lobe • Motor symptoms manifest as atrop
Slide 13: AMT-130: goal of clinical treatment to slow or halt disease progression
AMT-130: goal of clinical treatment to slow or halt disease progression from an early stage Penetration throughout Vector DNA distribution non-human primate brain 8 10 V e c t o r g e n o m e c o p ie s 7 10 p er µ g D N A 6 10 5 10 1 10 4 LLO D 3 10 P u ta m e n C a u d a t e T h a la m u s C o rte
Slide 14: AMT-130: strong reduction of mutant HTT
AMT-130: strong reduction of mutant HTT Libechov transgenic (tgHD) minipigs: Comparable mutant huntingtin protein • Life-span: 12-20 years knockdown at 6 and 12 months • Body weight: 50-140 kg • Brain weight: 90-100 g Cortex Striatum • Highly developed immune system 125 6 months MRI-guided 12 months
Slide 15: AMT-130: goal of clinical treatment
AMT-130: goal of clinical treatment Motor diagnosis Premanifest Manifest 100 AMT-130 ← Function (%) Slow or halt disease progression I II III IV V 0 Presymptomatic Prodromal Early Moderate Advanced Adapted from Ross CA, et al. Nat Rev Neurol 2014;10:204-16 DELIVERING GENE THERAPY TO PATIENTS OCTOBER
Slide 16: AMT-130: Phase I/II clinical trial
AMT-130: Phase I/II clinical trial Study Overview • Objectives: assess safety, tolerability and efficacy • Multicenter, randomized, double-blinded study • Controlled with imitation surgery • Two dose cohorts with a total of 26 patients • Early manifest patients with ≥ 44 CAG repeats • 18-month follo
Slide 17: AMT-130: Phase I/II clinical trial endpoints
AMT-130: Phase I/II clinical trial endpoints Efficacy Endpoints Clinical Parameters* Biomarkers • Total motor score • NF-L (neurofilament light) • Total functional capacity • mHTT in CSF • Composite score • Other exploratory markers Quantitative Motor Function Patient-reported outcomes • Finger, han
Slide 18: AMT-130: Phase I/II clinical trial design
AMT-130: Phase I/II clinical trial design Cohort 1 Cohort 2 Subject 1&2 Subject 3&4 Subject 5-10 Subject 11&12 Subject 13&14 Subject 15-26 1:1 1:1 2:1 1:1 1:1 2:1 randomization randomization randomization randomization randomization randomization 1 AMT-130 1 AMT-130 4 AMT-130 1 AMT-130 1 AMT-130 8 A
Slide 19: Research Pipeline
Research Pipeline DELIVERING GENE THERAPY TO PATIENTS OCTOBER 2 0 1 9 | 19
Slide 20: AMT-180: a novel approach to Hemophilia A
AMT-180: a novel approach to Hemophilia A Novel Approach • Product Construct – AAV5 including C7 Promoter and FIX-Super9™ • Super9™ is a proprietary modified FIX that, when activated through normal mechanisms, activates FX independently of FVIII Long-term and Effective in all HemA Compatible with Co
Slide 21: AMT-190: a new approach to Fabry disease
AMT-190: a new approach to Fabry disease Novel Approach • Product Construct – AAV5 including modified NAGA • Modified NAGA has therapeutic α-gal activity and gB3 reduction More effective than Patients with and without Non-immunogenic replacement therapy inhibitors • More stable in plasma • Many Fabr
Slide 22: AMT-150: a gene therapy for SCA3
AMT-150: a gene therapy for SCA3 Novel Approach • AAV5, SCA3 miRNA administered by intrathecal or cisterna magna injection • Leverages HD platform and experience, including miQURE™ gene silencing technology • Potential to be first to market Cause Damage Symptoms Unmet Need • CAG repeat expansion • B
Slide 23: Key corporate goals
Key corporate goals • Topline data from HOPE-B pivotal study in 2020 Hemophilia B • BLA submission in 2021 • Initiate dosing in Phase I/II study before end of 2019 Huntington’s • Preliminary biomarker data on initial patients in 2020 Hemophilia A • Submit IND for AMT-180 in 2020 • Initiate IND-enabl
Slide 24: uniQure
uniQure