uniQure N.V. · NASDAQ:QURE
uniQure — Merger or Acquisition (June 2020)
20 slides from uniQure's merger presentation, Jun 24, 2020
Slide 1: Global License Agreement in Hemophilia B
Global License Agreement in Hemophilia B June 24, 2020
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: A Historic Collaboration in Gene Therapy and Hemophilia B
A Historic Collaboration in Gene Therapy and Hemophilia B 1 • One of the largest gene therapy deals announced to date 2 • CSL Behring is an ideal strategic partner with extensive commercial expertise in hemophilia that best positions uniQure to provide EtranaDez to the greatest number of global pati
Slide 4: A Historic Collaboration in Gene Therapy and Hemophilia B
A Historic Collaboration in Gene Therapy and Hemophilia B • The transaction is a historic collaboration in gene therapy and hemophilia, with uniQure eligible to receive more than $2B in total economics • $450M upfront payment • $1.6B in regulatory and commercial milestones • Double-digit royalty pay
Slide 5: A Historic Collaboration in Gene Therapy and Hemophilia B
A Historic Collaboration in Gene Therapy and Hemophilia B • Partnership enables uniQure to leverage CSL Behring’s world-class global hemophilia commercial infrastructure • Hemophilia is a well-established, specialized and highly competitive global market • CSL Behring has been a leader in bleeding d
Slide 6: Building Value Through Innovation
Building Value Through Innovation
Slide 7: A Rich History of Ingenuity
A Rich History of Ingenuity • uniQure is a gene therapy pioneer with 20+-year history of industry-leading innovation across products and platform • First approved gene therapy in the Western world • First AAV-delivered gene silencing therapy for Huntington’s to enter clinic • First AAV vector that c
Slide 8: Hemophilia B: A Case Study in Building Value Though Innovation
Hemophilia B: A Case Study in Building Value Though Innovation • Ingenuity in practice – leapfrogging hemophilia B with a potentially first- and best-in-class gene therapy • 2008: Licensed wtFIX gene cassette used by St. Jude in watershed first-in-human study • 2015: Initiated Ph I/II study of AMT-0
Slide 9: Generating Future Value Through Continued Ingenuity
Generating Future Value Through Continued Ingenuity Aggressively Advance & Invest in and Strengthen 1 2 Expand Gene Therapy Pipeline Gene Therapy Platform A GLOBAL LEADER IN GENE THERAPY JUNE 24, 2020 | 9
Slide 10: Innovative Gene Therapy Pipeline
Innovative Gene Therapy Pipeline A GLOBAL LEADER IN GENE THERAPY JUNE 24, 2020 | 10
Slide 11: Aggressively Advance & Expand Pipeline
Aggressively Advance & Expand Pipeline
Slide 12: Aggressively Advance & Expand the Product Pipeline
Aggressively Advance & Expand the Product Pipeline 1• Advance Huntington’s Disease program • Patient dosing initiated in June 2020 represents first-in-human AAV gene therapy for Huntington’s • Phase I/II safety data expected in 2020 with initial efficacy data expected in 2021 • Exploring next-genera
Slide 13: AAV5: An Engine for Potentially Best-in-Class Gene Therapies
AAV5: An Engine for Potentially Best-in-Class Gene Therapies • Rapid path to IND and high regulatory familiarity • Superior immunogenicity profile compared to other AAV vectors • Potential to treat all or nearly all of patients • Potential for no immunosuppression • Potential for re-administration •
Slide 14: Invest in and Strengthen Gene Therapy Platform
Invest in and Strengthen Gene Therapy Platform
Slide 15: Investing in and Expanding Our Gene Therapy Platform
Investing in and Expanding Our Gene Therapy Platform 1 Build out organization to support expansion of pipeline 2 Further invest in new tools and new technologies to enable potential improvements in efficacy, safety and applicability of our gene therapy candidates • Next-gen components (capsids, prom
Slide 16: Leveraging Our Platform
Leveraging Our Platform NEXT-GEN PLATFORM New Capsid New Production Technologies Technologies CORE PLATFORM Scalable, modular AAV5 vector design Liver and CNS delivery miQURE gene silencing technology Highly potent, tissue-specific promoters New Promoter 500L dual-bac production New Transgene Techno
Slide 17: Investing in and Expanding Our Gene Therapy Platform
Investing in and Expanding Our Gene Therapy Platform Develop Potentially Best-in-Class Gene Therapies Through Innovation More effective cell targeting, transduction and expression Improved Improved • Nanobody-designed AAVs Targeting Targeting • HDL-binding capsids • GenX: Highly-specific, potent pro
Slide 18: Our 5-Year Vision
Our 5-Year Vision
Slide 19: Our 5-Year Vision
Our 5-Year Vision 1 • Commercial-stage gene therapy company with best-in-class pipeline • 3 to 5 commercial/pivotal stage programs • 5 to 10 Phase I/II programs 2 • Industry-leading manufacturing and technology capabilities • Lower-cost, 2,000L scale process capable of expanding to 10,000L • Leading
Slide 20: uniQure
uniQure