Kyowa Kirin Co., Ltd. · TSE:4151

Kyowa Kirin — Investor Day (December 2020)

42 slides from Kyowa Kirin's investor day presentation, Dec 10, 2020

  1. Slide 1: Kyowa Kirin R&D Day

    Kyowa Kirin R&D Day December 10, 2020 Yoshifumi Torii, Ph.D., Executive Officer, Vice President, Head of R&D Division

  2. Slide 2: Forward-looking statements disclaimer

    This document contains certain forward‐looking statements relating to such items as the company’s (including its domestic and overseas subsidiaries) forecasts, targets and plans. These forward‐looking statements are based upon information available to the company at the present time and upon reasona

  3. Slide 3: Agenda

    Agenda  Introduction  Topics ✱ Projects aiming to launch products within 10‐20 years Axcelead / InveniAI ✱ Projects aiming to launch products within 10 years Drug discovery using bispecific antibody technology developed in‐house / Collaboration‐leveraged drug discovery ✱ Projects aiming to launch

  4. Slide 4: Introduction: Challenging the creation of new value in R&D

    Introduction: Challenging the creation of new value in R&D Initiatives for expanding the opportunities to create value unique to Kyowa Kirin  Initiatives and innovation through internal activities + collaborations ‐ Axcelead, InveniAI, SBI Biotech ‐ Next generation antibody technology, Nucleic acid

  5. Slide 5: Projects aiming to launch products within 10-20 years

    Projects aiming to launch products within 10‐20 years © Kyowa Kirin Co., Ltd. 6

  6. Slide 6: Development of revolutionary small molecule drug discovery technology (Axcelead)

    Development of revolutionary small molecule drug discovery technology (collaboration with Axcelead)  Analysis of potential  Small molecule drug target drugs discovery infrastructure ・ Screening technology that imitates the ・ Leading capability in Japan pathology, and molecule design technology  V

  7. Slide 7: Promotion of data driven drug discovery (InveniAI)

    Promotion of data driven drug discovery (collaboration with InveniAI)  InveniAI: ‐ Possesses drug discovery technology that uses AI and machine learning and has experience working with multiple pharmaceutical companies ‐ Started collaborating with InveniAI in 2018 aimed at maximizing the value of K

  8. Slide 8: Projects aiming to launch products within 10 years

    Projects aiming to launch products within 10 years © Kyowa Kirin Co., Ltd. 9

  9. Slide 9: Drug discovery using in-house developed bispecific antibody technology

    Drug discovery using in‐house developed bispecific antibody technology © Kyowa Kirin Co., Ltd. 10

  10. Slide 10: In-House Development of Bispecific-Antibody Technology

    In‐House Development of Bispecific‐Antibody Technology Key points  Apply Kyowa Kirin’s unique bispecific‐antibody technology for new therapeutic antibodies  Pursue the development of “first in class” drugs by blending technology with biology Antigen A  Selection of linkers derived from binding si

  11. Slide 11: Flagship Project: Safety issues of conventional antibodies

    Flagship Project Unique ＝ Ideas Based on Bispecific‐ ‘First‐in‐Class’ Drugs That Research in Antibody Combine Efficacy and Safety Biology Technologies If targeted by conventional antibodies… Issues related to the limitations of conventional modalities Safety • Ideal efficacy, if the issue of systemi

  12. Slide 12: Realizing Ideal Efficacy with Safety Through Bispecific-Antibody Technologies

    Flagship Project Unique ＝ Ideas Based on Bispecific‐ ‘First‐in‐Class’ Drugs That Research in Antibody Combine Efficacy and Safety Biology Technology 2000 Vehicle 1500 Realizing Ideal Efficacy with Safety 1000 Through Bispecific‐ Chemotherapy Antibody 500 Technologies Bispecific‐Antibody 0 0 10 20 30

  13. Slide 13: Expansion of the Bispecific-Antibody Project

    Expansion of the Bispecific‐Antibody Project Combinations of the targets Type I （A,B… ) x (X, Y…） A B A B X X Y Y Selection of the types （Type I, II） Type II ＝ A B A B X X Y Y Challenges exceeding the limitations of conventional modalities © Kyowa Kirin Co., Ltd. 14

  14. Slide 14: Collaboration-leveraged drug discovery

    Collaboration‐leveraged drug discovery © Kyowa Kirin Co., Ltd. 15

  15. Slide 15: New antibody that targets pDCs (in-licensed from SBI Biotech)

    New antibody that targets pDCs (in‐licensed from SBI Biotech) In 2016 Kyowa Kirin concluded an exclusive licensing agreement with SBI Biotech Co., Ltd. covering development, manufacturing and sales worldwide for SBI‐9674, an antibody drug for autoimmune diseases * pDC : Plasmacytoid dendritic cell S

  16. Slide 16: New antibody that targets pDCs: type 1 interferon role

    New antibody that targets pDCs (in‐licensed from SBI Biotech)  pDCs are known to be the main source of type 1 interferons (Type 1 IFN), which are the cause of various autoimmune diseases  Type 1 IFN produced from pDCs coordinate immune functions through a variety of mechanisms (see the illustratio

  17. Slide 17: New antibody that targets pDCs: Phase 2 SLE/CLE results

    New antibody that targets pDCs (in‐licensed from SBI Biotech)  At this year’s ACR (American College of Rheumatology) convergence, the results of the Phase 2 study of the developed compound that targets pDCs (Anti‐BDCA2 mAb) in patients with SLE/CLE were reported Source: ACR convergence 2020 Abstrac

  18. Slide 18: Projects aiming to launch products within several years

    Projects aiming to launch products within several years © Kyowa Kirin Co., Ltd. 19

  19. Slide 19: ME-401

    ME‐401 © Kyowa Kirin Co., Ltd. 20

  20. Slide 20: ME-401: Results of a Phase 1b Study in B-cell Malignancies

    ME‐401: Results of a Phase 1b Study in B‐cell Malignancies ASCO 2020 ClinicalTrials.gov Identifier: NCT02914938  Treatment: ME‐401 monotherapy or in combination with rituximab administered in IS Daily No therapy Daily dosing x2 cycles x1 week x3 weeks Cycles 1 and 2 Intermittent Schedule on Cycles

  21. Slide 21: ME-401: Global Phase 2 TIDAL Study

    ME‐401: Global Phase 2 TIDAL* Study in r/r Follicular Lymphoma or Marginal Zone Lymphoma ClinicalTrials.gov Identifier: NCT03768505  Subjects: Patients with r/r FL after failure of at least two systemic therapies  Treatment: ME‐401 monotherapy administered on an intermittent schedule Daily No ther

  22. Slide 22: ME-401: Japanese Phase 2 K02 Study

    ME‐401: Japanese Phase 2 K02 Study in r/r Indolent B‐cell NHL ClinicalTrials.gov Identifier: NCT04533581  Subjects: Patients with r/r indolent B‐cell NHL* after failure of at least two systemic therapies  Treatment: ME‐401 monotherapy administered on an intermittent schedule Daily No therapy Daily

  23. Slide 23: KHK7791

    KHK7791 © Kyowa Kirin Co., Ltd. 24

  24. Slide 24: KHK7791 / tenapanor mechanism of action

    KHK7791 / tenapanor Phosphate Tight junction Mechanism of action  The primary uptake route of phosphorous is passive transport between cell membranes  Tenapanor inhibits NHE3 and thereby inhibits the uptake of sodium (Na+)  At the same time, the concentration of protons (H+) in the cells increase

  25. Slide 25: Summary of the Phase 3 studies (Dose-finding, Add-on, Switching)

    Summary of the Phase 2 studies (Dose‐finding, Add‐on, Switching) Serum KHK7791 or Placebo phosphorus level Objectives Dose‐finding  Explain that hyperphosphatemia can be 6.0 treated with a single agent, KHK7791  Explain the validity of using KHK7791 for Washout of patients who are starting treatme

  26. Slide 26: KHK7791: PB switching study design

    KHK7791: PB switching study design ClinicalTrials.gov Identifier: NCT03831607  Open‐label, single‐arm, phosphate binder (PB) switch study for hyperphosphatemia patients on hemodialysis Screening Pre‐enrollment Enrollment Phosphate binder (PB) 92 subjects 83 subjects 67 subjects + KHK7791 Enrollment

  27. Slide 27: KHK7791: PB switching study results

    KHK7791: PB switching study results  Primary endpoint Achievement ratio P value [95%CI] 30% decrease 71.6% (48/67) <0.001 [59.3, 82.0] CI confidence interval.  Mean change  Mean change in s‐P over time in total PB tablets over time Start of KHK7791 (mg/dL) Start of KHK7791 administration Mean cha

  28. Slide 28: KHK7791: Dose-finding study design

    KHK7791: Dose‐finding study design ClinicalTrials.gov Identifier: NCT03864458  A phase 2, randomized, double‐blind, placebo‐controlled, dose‐finding study of KHK7791 in hyperphosphatemia patients on hemodialysis 1st washout period Treatment period 2nd washout period Maximum 3 weeks 6 weeks 3 weeks

  29. Slide 29: KHK7791: Dose-finding study results

    KHK7791: Dose‐finding study results  Primary endpoint KHK7791 KHK7791 KHK7791 KHK7791 Placebo 30 mg dose‐ 5 mg 10 mg 30 mg titration N=41 N=42 N=41 N=42 N=41 Mean±SD 0.6±1.6 ‐0.9±1.7 ‐1.4±1.5 ‐1.9±1.2 ‐2.0±1.1 Week 6 Median 0.4 ‐1.0 ‐1.5 ‐2.1 ‐2.0 Serum phosphorus level Difference ‐ ‐1.6 ‐2.0 ‐2.6

  30. Slide 30: KHK7791: Add-on study design

    KHK7791: Add‐on study design ClinicalTrials.gov Identifier: NCT03864445  A phase 2, randomized, double‐blind, placebo‐controlled, phosphate binder‐ combination study of KHK7791 in hyperphosphatemia patients on hemodialysis Observation period Evaluation period Maximum 3 weeks 6 weeks KHK7791 30 mg B

  31. Slide 31: KHK7791: Add-on study results

    KHK7791: Add‐on study results  Primary endpoint and target achievement ratio of serum phosphorus level (3.5〜6.0 mg/dL) Placebo KHK7791 N=24 N=23 Mean±SD 0.1±1.5 ‐2.0±1.2 Median ‐0.2 ‐2.1 Week 6 Serum phosphorus level (mg/dL) Difference ‐ ‐2.1 95% Cl ‐ [‐2.9,‐1.3] p‐value ‐ <0.001 Week 6 % 37.5 87.0

  32. Slide 32: RTA 402

    RTA 402 © Kyowa Kirin Co., Ltd. 33

  33. Slide 33: RTA 402 / Bardoxolone methyl

    RTA 402 / Bardoxolone methyl Kanda H and Yamawaki K, Clinical and Experimental Nephrology 24:857–864, 2020 RTA 402 (Bardoxolone methyl) activates the Keap1‐Nrf2 pathway, which is an oxidative stress response system. © Kyowa Kirin Co., Ltd. 34

  34. Slide 34: RTA 402: CARDINAL Phase 3 for Alport Syndrome patients

    RTA 402: CARDINAL Phase 3 for Alport Syndrome patients What is Alport Syndrome? Alport Syndrome is a kidney disease caused by genetic mutations in either the type IV collagen α3, α4 or α5 chain. In Japan, Alport Syndrome is certified as designated intractable diseases and specific pediatric chronic

  35. Slide 35: RTA 402: CARDINAL Phase 3 for Alport Syndrome patients

    RTA 402: CARDINAL Phase 3 for Alport Syndrome patients ITT: Intent to treat mITT: modified‐ITT, analysis assesses the effect of receiving treatment by excluding values after patients discontinued treatment Both primary/key secondary endpoints were met. The results also show a favorable safety profil

  36. Slide 36: RTA 402: CARDINAL Phase 3 for Alport Syndrome patients

    RTA 402: CARDINAL Phase 3 for Alport Syndrome patients Effect for eGFR was observed over the study period in the RTA 402 group. Based on the current results, KKC also plans to submit a marketing approval application for the indication of Alport Syndrome in Japan. https://www.reatapharma.com/wp‐conte

  37. Slide 37: Summary

    Summary © Kyowa Kirin Co., Ltd. 38

  38. Slide 38: Summary (1): Challenging the creation of new value in R&D

    Summary (1): Challenging the creation of new value in R&D Initiatives for expanding the opportunities to create value unique to Kyowa Kirin  Initiatives and innovation through internal activities + collaborations ‐ Axcelead, InveniAI, SBI Biotech ‐ Next generation antibody technology, Nucleic acid

  39. Slide 39: Summary (2): R&D spirits

    Summary (2): Challenging the creation of new value in R&D R&D spirits 技術戦略 疾患戦略 © Kyowa Kirin Co., Ltd. 40

  40. Slide 40: Q&A Session

    Q&A Session © Kyowa Kirin Co., Ltd. 41

  41. Slide 41: Thank you.

    Thank you. Kyowa Kirin Co.,Ltd.

  42. Slide 42: Kyowa Kirin

Kyowa Kirin — Investor Day (December 2020) on 6ix