Astellas Pharma Inc. · TSE:4503

Astellas Pharma — Merger or Acquisition (December 2019)

22 slides from Astellas Pharma's merger presentation, Dec 3, 2019

  1. Slide 1: Acquisition of Audentes

    ACQUISITION OF AUDENTES Establishing a leading position in gene therapy Naoki Okamura Representative Director, Corporate Executive Vice President, Chief Strategy Officer and Chief Financial Officer Astellas Pharma Inc. December 3, 2019

  2. Slide 2: Cautionary Notice Regarding Forward-Looking Statements

    Cautionary Notice Regarding Forward-Looking Statements This document contains “forward-looking statements” relating to the acquisition of Audentes by Astellas. Such forward-looking statements include, but are not limited to, the ability of Audentes and Astellas to complete the transactions contempla

  3. Slide 3: Agenda

    AGENDA 3 I Transaction Summary II Overview of Audentes III Strategic Rationale

  4. Slide 4: Transaction Summary

    TRANSACTION SUMMARY 4 Party • Audentes Therapeutics, Inc. (San Francisco); Listed on NASDAQ Purchase Price • $60.00 per share in cash • Premium of 110% to Audentes’ closing share price of $28.61 on December 2, 2019 Acquisition • Approximately $3 billion Amount* Acquisition Method • Asilomar Acquisit

  5. Slide 5: Agenda

    AGENDA 5 I Transaction Summary II Overview of Audentes III Strategic Rationale

  6. Slide 6: Company Overview

    COMPANY OVERVIEW 6 Audentes Therapeutics, Inc.  Headquartered in San Francisco, California  Founded in 2012; IPO on NASDAQ in July 2016  Clinical-stage AAV-based gene therapy company focusing on rare neuromuscular diseases  Experienced management  Of total 270 employees, 220 engage in R&D and m

  7. Slide 7: Key Gene Therapy Success Factors: Audentes' Outstanding Capability

    KEY GENE THERAPY SUCCESS FACTORS 7 Audentes’ Outstanding Capability Lead Program Establishes Clinical Proof-of-Concept for Audentes’ Know-how and Platform AT132 for XLMTM is in Phase 1/2 clinical stage Large-scale cGMP Manufacturing Capability AAV Technology Platform Fully integrated, in-house Propr

  8. Slide 8: AT132 Program Overview

    AT132 PROGRAM OVERVIEW 8  AAV8 capsid; Desmin promoter  One-time, systemic dose Product  Gene replacement of MTM1 gene encoding myotubularin in muscle cells  X-linked, loss of function mutations in MTM1 gene  Rare disease; Estimated 50% mortality by 18 months XLMTM  >80% require ventilator sup

  9. Slide 9: AT132 Phase 1/2 ASPIRO Study

    AT132 PHASE 1/2 ASPIRO STUDY 9  Male subjects (<5 yrs old) Inclusion Criteria  Genetically confirmed XLMTM  Requires ventilator support  Safety and tolerability Assessment  Neuromuscular function: CHOP INTEND, Developmental milestones  Respiratory function: MIP and Ventilator use  Cohort 1: 1

  10. Slide 10: AT132 Phase 1/2 ASPIRO Study

    AT132 PHASE 1/2 ASPIRO STUDY 10 Treated patients are achieving the ability to raise self to standing position, walk with support or walk alone Age (years) Only Patient 2 was able to sit unassisted for 30 seconds at baseline, Patient 3 not evaluable because of halo traction device Interim data as of

  11. Slide 11: AT132 Phase 1/2 ASPIRO Study: CHOP INTEND Score

    AT132 PHASE 1/2 ASPIRO STUDY 11 Treated patients showed rapid and sustained increase in CHOP INTEND Score Neuromuscular Functions (CHOP-INTEND Score) Treated (N=10) CHOP INTEND score (LS means) Control (N=2) Change from baseline in Weeks after ASPIRO baseline Interim data as of August 7, 2019 CHOP I

  12. Slide 12: AT132 Phase 1/2 ASPIRO Study: Ventilator Dependence

    AT132 PHASE 1/2 ASPIRO STUDY 12 Treated patients have achieved mean reductions in ventilator dependence nearing 20 hours per day Baseline: Ventilator use >12 hours ventilator dependence dependence over 24 hours (h) Treated (N=10) Reduction in ventilator Control (Patient 4) Control (Patient 11) (LS m

  13. Slide 13: AT132 Phase 1/2 ASPIRO Study: Respiratory Function (MIP)

    AT132 PHASE 1/2 ASPIRO STUDY 13 Increased respiratory muscle strength (MIP) observed as early as 4 weeks Respiratory Functions (MIP) Treated (N=10) Control (N=2) MIP (cm H2O) (LS means) Change from baseline in Weeks after ASPIRO baseline Interim data as of August 7, 2019 MIP: maximal inspiratory pre

  14. Slide 14: 10,000 Fold Higher Dose in Muscle Compared to Eye

    10,000 FOLD HIGHER DOSE IN MUSCLE COMPARED 14 TO EYE Established large-scale commercial manufacturing provides capacity required for higher-dose neuromuscular indications 1x1011 vg x10 dose of eye x1,000 dose of eye x10,000 dose of eye Adapted from https://www.nature.com/articles/d42473-018-00307-6

  15. Slide 15: Manufacturing Facility to Develop Multiple Pipeline Product Candidates

    MANUFACTURING FACILITY TO DEVELOP MULTIPLE 15 PIPELINE PRODUCT CANDIDATES Clinical Trial Materials Supply Commercial Production of AT132 Internal manufacturing capability to Large-scale production enabled by seamlessly supply drug product Audentes’ proprietary know-how Process Development Clinical T

  16. Slide 16: AAV-Based Gene Therapy

    AAV-BASED GENE THERAPY 16 AAV Surface Delivery of the transgene to a target cell Transgene Therapeutic gene that selectively functions in a target cell of patients Adeno Associated Virus AAV platform + Transgene  AAV8 is Audentes’ preferred capsid serotype for delivering transgenes to target tissue

  17. Slide 17: Audentes' Pipeline

    AUDENTES’ PIPELINE 17 AAV-based gene therapies for rare neuromuscular diseases Code Disease Transgene MOA Development Stage AT132 XLMTM MTM1 • Ph1/2 pivotal expansion ongoing AT845 Pompe Disease GAA • Preclinical AT702 DMD Exons 2, 1-5 • Pre-clinical AT751 DMD Exon 51 • Pre-clinical AT753 DMD Exon 5

  18. Slide 18: Agenda

    AGENDA 18 I Transaction Summary II Overview of Audentes III Strategic Rationale

  19. Slide 19: Genetic Regulation as Fifth Primary Focus

    GENETIC REGULATION AS FIFTH PRIMARY FOCUS 19 Genetic Regulation to be new Primary Focus Primary Focus + Genetic Immuno- oncology Mitochondria Biology Regulation  In-house R&D ASIM Regeneration  Asset acquisition through & Blindness Biology business development Harvard Medical School ASIM: Antigen-

  20. Slide 20: Combined Capabilities of Astellas and Audentes Allow Targeting of More Diseases

    THE COMBINED CAPABILITIES OF ASTELLAS AND 20 AUDENTES ALLOW THE TARGETING OF MORE DISEASES Modality (Audentes) License & Optimization to Process dev & Patients Biology Engineering Final products Manufacturing (Audentes + Astellas) Neuromuscular Gene Replacement diseases Audentes AAV Gene Regulation

  21. Slide 21: Acquisition of Audentes: Summary

    ACQUISITION OF AUDENTES: SUMMARY 21 Acquisition of Audentes is a major step to establishing a leading position in gene therapy +  Transforms Genetic Regulation Primary Focus into a new growth area for Astellas, building on a complementary technology platform and capabilities in gene therapy to swif

  22. Slide 22: On the Forefront of Healthcare Change

    ON THE FOREFRONT OF HEALTHCARE CHANGE

Astellas Pharma — Merger or Acquisition (December 2019) on 6ix