Uniqure N.v. · NASDAQ:QURE
Uniqure — Investor Presentation (August 2022)
31 slides from Uniqure's investor presentation, August 2022
Slide 1: Leadership in Gene Therapy
Leadership in Gene Therapy August 2022
Slide 2: Our mission is to deliver curative, one-time genomic medicines
Our mission is to deliver curative, one-time administered genomic medicines that transform the lives of patients. inGENEuity through imagGENEation… RLEESA ED EC AR RHS H ANI PD IDNE G V EELNOEP M TEHNETR D AAPYY 2 0 2 1 A J UUNGEU2S2T, 22002212 | 2
Slide 3: inGENEuity: our history of innovation
inGENEuity: our history of innovation uniQure: A gene therapy pioneer with a >20-year history and deeply engrained culture of innovation across an increasingly validated platform 15 years 100+ patients World-class Leading LEADERSHIP IN GENE THERAPY AUGUST 2022 | 3
Slide 4: inGENEuity: our history of innovation
inGENEuity: our history of innovation uniQure: A gene therapy pioneer with a >20-year history and deeply engrained culture of innovation across an increasingly validated platform First First First First LEADERSHIP IN GENE THERAPY AUGUST 2022 | 4
Slide 5: inGENEuity: a case study in delivering value through innovation
inGENEuity: a case study in delivering value through innovation Leapfrogging hemophilia B with a first and best-in-class gene therapy 2017 2020 2022 Announced Completed dosing of Submitted BLA and transition to AMT- 54 patients in Ph 3 MAA 061 with AAV5/FIX- pivotal study Padua transgene 2015 2018 2
Slide 6: Re-imaGENEing our in-house manufacturing
Re-imaGENEing our in-house manufacturing Manufacturing for the future: Establishing larger scale and highly cost- effective capabilities to address more prevalent disorders LEADERSHIP IN GENE THERAPY AUGUST 2022 | 6
Slide 7: Re-imaGENEing our capabilities
Re-imaGENEing our capabilities Initiated construction of a second cGMP manufacturing facility in Amsterdam that will complement commercial manufacturing in Lexington, Massachusetts. Amsterdam, NL Lexington, MA 111,000 sq ft (~290 FTEs) Increasing to 100,000 sq ft (~200 FTEs) Research Process Develo
Slide 8: uniQure: our focus
uniQure: our focus AAV Engine: Leveraging our leading technology platform to develop and commercialize products targeting the CNS, liver and heart/muscle AAV Technology Engine Manufacturing & Enabling Tools LLEEAA DDEE RSRH SIHPI P I NI N G EGNEENTEH T EHRA EPRYA P Y AAUUGGUUSSTT 22002222 | 8
Slide 9: LEADERSHIP IN GENE THERAPY AUGUST 2022
LEADERSHIP IN GENE THERAPY AUGUST 2022 | 9
Slide 10: Re-imaGENEing the R&D pipeline
Re-imaGENEing the R&D pipeline Doubling the pipeline in 5 years 5-year Pipeline 3-4 Phase 3 commercial 5-8 Phase 1/2 programs 7-12 preclinical Goals: programs programs LEADERSHIP IN GENE THERAPY AUGUST 2022 | 10
Slide 11: Re-imaGENEing the R&D pipeline
Re-imaGENEing the R&D pipeline Larger market opportunities built on validated targets and technologies ● Best and/or first in class ● Human validation Key criteria: ● Leverage proven technologies ● Larger indications LLEEAA DDEE RSRH SIHPI P I NI N G EGNEENTEH T EHRA EPRYA P Y AAUUGGUUSSTT 22002222
Slide 12: Significant cash runway to transform our pipeline
Re-imaGENEing the R&D pipeline Significant cash runway to transform our pipeline ~$500M of cash on hand Runway to mid-2024 +$175M in near-term milestones Extends runway into 1H 2025 +$1.3B in potential other milestones + royalties Extends runway into 2026 and beyond LEADERSHIP IN GENE THERAPY AUGUST
Slide 13: Recent company progress & upcoming events
Recent company progress & upcoming events • Announced positive 78-week top-line data from HOPE-B pivotal trial Hemophilia B • MAA and BLA regulatory filings accepted • Received $55 million in additional milestone payments from CSL Behring • Announced 12-month data on lower-dose cohort in Phase I/II
Slide 14: Hemophilia B
Hemophilia B Hemophilia B Etranacogene dezaparvovec (AMT-061) Etranacogene dezaparvovec (AMT-161) LEADERSHIP IN GENE THERAPY AUGUST 2022 14
Slide 15: A global licensing deal with CSL Behring in hemophilia B
A global licensing deal with CSL Behring in hemophilia B Transaction is a historic collaboration in gene therapy and hemophilia, with uniQure eligible to receive more than $2B in total economics • $450M upfront payment received in 2Q 2021 • $1.6B in regulatory and commercial milestones • Double-digi
Slide 16: Overview of 78-week top-line data
Overview of 78-week top-line data • Achieved primary endpoint in HOPE B Pivotal trial • Also achieved secondary endpoint demonstrating statistical superiority in reduction of bleeding rate compared to FIX replacement therapy • Stable and durable FIX activity with mean of 36.9% of normal at month 18
Slide 17: Primary endpoint of annualized bleeding rate was statistically significant
Primary endpoint of annualized bleeding rate (ABR) was statistically significant Etranacogene dezaparvovec reduced ABR by 64% and demonstrated superiority to prophylaxis in the lead-in period One-sided p-value ≤0.025 for post-treatment/lead-in <1 is regarded as statistically significant. ABR, annual
Slide 18: Substantial reductions in FIX replacement and use of prophylaxis
Substantial reductions in FIX replacement and use of prophylaxis when compared with lead-in At 78 weeks follow-up, compared with the lead-in period • 96.3% (52/54) subjects discontinued prophylaxis and remain prophylaxis-free Analyses include two subjects who remain on prophylaxis (1 subject receive
Slide 19: Hemophilia B regulatory timeline
Hemophilia B regulatory timeline CSL Behring filled the BLA and MAA in the first half of 2022 • Granted Breakthrough Therapy Designation by FDA • BLA accepted in May 2022 under Priority Review • FDA does not expect an advisory panel • Approval expected as early as Q4 2022 • Granted access to Priorit
Slide 20: AMT-130
AMT-130 LEADERSHIP IN GENE THERAPY AUGUST 2022 | 20
Slide 21: Two ongoing studies in the U.S., EU and UK
Two ongoing studies in the U.S., EU and UK CT-AMT-130-01 (Phase Ia/II) CT-AMT-130-02 (Phase Ib/II) double-blind sham-controlled open-label 44 Patients Total 15 Patients Total 22 Treated, 16 Control 15 Patients Treated 26 Patients Enrolled 10 Patients Enrolled ✓ ✓ ✓ Low-dose 6e12 High-dose 6e13 Surgi
Slide 22: Encouraging data from lower dose cohort in U.S. Ph I/II study
Encouraging data from lower dose cohort in U.S. Ph I/II study • AMT-130 was generally well-tolerated at lower dose, with no reported adverse drug reactions or serious unexpected suspected adverse reactions • CSF NfL increased as expected following surgical administration of AMT-130 and neared baseli
Slide 23: NfL levels near baseline at 12-months in treated patients
NfL levels near baseline at 12-months in treated patients Neurofilament Light Chain (NfL) in Cerebral Spinal Fluid (CSF) Percent Change from Baseline 850% CSF NfL Percentage Change from Baseline 750% Low Dose 1 650% Low Dose 2 550% Low Dose 3 450% Low Dose 4 350% Low Dose 5 250% 150% Low Dose 6 50%
Slide 24: Declines in CSF mHTT support target engagement
Declines in CSF mHTT support target engagement 250 Percent Change in mHTT from Baseline 200 Percent Change mHTT 150 100 80.64 64.88 49.81 50 Low Dose 1.89 Control 0 -3.68 -13.17 -22.5 -16.81 -50 -26.42 -53.82 -100 Month 1 Month 3 Month 6 Month 9 Month 12 N= 4 3 3 3 4 3 4 3 4 3 LEADERSHIP IN GENE THE
Slide 25: August Clinical Update
August 8 Clinical Update • SUSARs reported in 3 of 14 patients treated at higher dose • Two were treated at a single clinical site in Europe • The third patient was treated back in March in the U.S. and subsequently reclassified as a SUSAR • All three patients have fully or substantially recovered •
Slide 26: Acquisition of Corlieve Therapeutics
Acquisition of HemophiliaTherapeutics Corlieve B Etranacogene dezaparvovec (AMT-061) LEADERSHIP IN GENE THERAPY AUGUST 2022 26
Slide 27: Acquisition of Corlieve Therapeutics
Acquisition of Corlieve Therapeutics • In June 2021, uniQure announced the acquisition of Corlieve Therapeutics and its gene therapy to treat refractory Temporal Lobe Epilepsy (rTLE) • Highly compelling and strategic transaction: • Expands uniQure’s pipeline of transformational gene therapies • Stre
Slide 28: Refractory temporal lobe epilepsy (TLE)
Refractory temporal lobe epilepsy (TLE) • TLE is the most common type of focal epilepsy • TLE is associated with damage to the temporal lobe and hyperexcitability of the hippocampus • It’s often caused by brain injury, tumors or a prolonged febrile seizure • TLE affects approximately 1.3 million peo
Slide 29: Near-term milestones
Near-term Hemophilia B milestones Etranacogene dezaparvovec (AMT-061) LEADERSHIP IN GENE THERAPY AUGUST 2022 29
Slide 30: Upcoming milestones
Upcoming milestones • U.S. approval by end of 2022/early 2023 Hemophilia B • European approval by early 2023 • CSL commercial launch in early 2023 ($175 million milestones) • Complete SUSAR investigation / resume higher-dose enrollment in Q4 2022 Huntington’s • Present 1-2 year data on 26 patients i
Slide 31: Slide 31