Kyowa Kirin Co., Ltd. · TSE:4151
Kyowa Kirin — Technical Presentation (December 2022)
34 slides from Kyowa Kirin's technical presentation, Dec 5, 2022
Slide 1: Kyowa Kirin R&D Meeting
Kyowa Kirin R&D Meeting December 5, 2022 Yoshifumi Torii, Ph.D., Executive Officer, Vice President, Head of R&D Division
Slide 2: Forward-looking statements
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
Slide 3: Vision: Our New Vision toward 2030
Vision Provide pharmaceuticals for unmet medical needs We are focused on developing medicines for diseases where there is a clear patient need for new options. We make full use of multiple Our New Vision toward 2030 therapeutic modalities, including biotechnology such as antibody technology, and bey
Slide 4: Kyowa Kirin's Strengths
Research & Development Concept Technology x Biology x OI The Kyowa Kirin Group is aiming to Technology Disease Biology build a competitive technology platform by enhancing and combining We will continue to evolve our Continue to provide “Only-one value its drug discovery technologies and its antibod
Slide 5: Today's Agenda
Today's Agenda ◼ Toward the Creation of Pharmaceuticals for High Unmet Medical Needs - From Our Investigational Products ⚫ KW-3357 (Preeclampsia) ⚫ KHK4951 (Tivozanib eye-drop) ◼ Toward the Successful Creation and Delivery of Life-changing Value - From Early-stage R&D Activities ⚫ Data-driven drug d
Slide 6: Toward the Creation of Pharmaceuticals for High Unmet Medical Needs
Toward the Creation of Pharmaceuticals for High Unmet Medical Needs - From Our Investigational Products ⚫ KW-3357 (Preeclampsia) ⚫ KHK4951 (Tivozanib eye-drop) 6 © Kyowa Kirin Co., Ltd.
Slide 7: Toward the Creation of Pharmaceuticals for High Unmet Medical Needs
Toward the Creation of Pharmaceuticals for High Unmet Medical Needs - From Our Investigational Products ⚫ KW-3357 (Preeclampsia) ⚫ KHK4951 (Tivozanib eye-drop) 7 © Kyowa Kirin Co., Ltd.
Slide 8: Preeclampsia (PE)
Preeclampsia (Preeclampsia, PE) ◼ One of the forms of Hypertensive Disorders of Pregnancy (HDP) *1: Shiozaki A,. Comparison of risk factors for gestational hypertension and preeclampsia in Japanese singleton pregnancies. J Obstet Gynaecol Res. 2013;39(2):492-9 ◼ Poor prognosis for mothers and fetuse
Slide 9: KW-3357 Overview
KW-3357 Overview ◼ Generic name: Antithrombin gamma ◼ Product name (in Japan) : ACOALAN® for Intravenous Injection 600, 1800 ◼ Indication: Congenital antithrombin (AT) deficiency-based thrombogenicity and disseminated intravascular coagulation syndrome ◼ Ph3 study (KOUNO-TORI) underway for preeclamp
Slide 10: The persistence of KW-3357 is comparable to human plasma-derived AT
The persistence of KW-3357 is comparable to human plasma-derived AT * From Interview Form of ACOALAN® for Intravenous Injection 600, 1800 Plasma AT activity https://www.info.pmda.go.jp/go/pack/6343444D1021_1_09/ Pharmacokinetics Parameter (plasma AT activity) KW-3357 (72IU/kg, n = 21) Human plasma d
Slide 11: Ph3 Test (KOUNO-TORI) Overview
Ph3 Test (KOUNO-TORI) Overview S Saito, K Takagi, J Moriya, et al, Contemp Clin Trials. 2021 ClinicalTrials.gov Identifier: NCT04182373 Main inclusion criteria: ✓ Pregnancy between 24 weeks 0 days and 31 weeks 6 days ✓ Severe preeclampsia nephropathy ✓ AT activity less than 100% Main Exclusion Crite
Slide 12: Toward the Creation of Pharmaceuticals for High Unmet Medical Needs
Toward the Creation of Pharmaceuticals for High Unmet Medical Needs - From Our Investigational Products ⚫ KW-3357 (Hypertensive nephropathy of pregnancy) ⚫ KHK4951 (Tivozanib eye-drop) 12 © Kyowa Kirin Co., Ltd.
Slide 13: Neovascular age-related macular degeneration (nAMD)
Neovascular age-related macular degeneration (nAMD) J Ophthalmol. 2015;2015:820605. ◼ A disease in which the macula is affected by abnormal choroidal neovascularization ◼ Rapidly progressive, causing significant vision loss ◼ Number of patients treated with drugs ⚫ Japan: approx. 200,000 ⚫ Global: a
Slide 14: KHK4951: Tivozanib Eye-drop
KHK4951: Tivozanib Eye-drop Tivozanib • The active ingredient, tivozanib, is a VEGFR inhibitor discovered by Kyowa Kirin. • Our Partner* markets tivozanib under the brand FOTIVDA® (oral formulation) for renal cell carcinoma in the U.S., Europe, etc. * Developed by AVEO oncology and EUSA Pharma, appr
Slide 15: Background to date
Background to date Kyowa Kirin Bought Back KHK4951 Tivozanib for its Non- Ph1 study started Ph1 study Oncology Rights from (Japan, nAMD) Last Patient Out AVEO December 2006 December 2015 August 2019 April 2020 March 2021 2022 and Beyond! Kirin Brewery Out- EUSA Pharma Acquires AVEO Receives Approval
Slide 16: Our Proprietary New Eye-drop Formulation
Our Proprietary New Eye-drop Formulation Drug concentration in posterior ocular tissue after topical instillation of tivozanib eye-drop to rats Concentration in posterior Anterior ocular tissue Posterior ocular tissue ocular tissue (ng/g) 500 Target API 400 300 200 100 0 Our new eye-drop Conventiona
Slide 17: Ph1 Study Synopsis (ID: 4951-001)
Ph1 Study Synopsis (ID: 4951-001) ClinicalTrials.gov Identifier: NCT04594681 Dosing Subjects No. of steps No. of subjects / step Cohort 1* Single administration Healthy volunteers 5 8 KHK4951:Placebo=6:2 Japanese/White Cohort 3W administration Healthy volunteers Cohort 2* Japanese/White 6 8 KHK4951:
Slide 18: Ph1 study results (ID: 4951-001)
Ph1 study results (ID: 4951-001) • Achieved Last Patient Out • Safety, pharmacokinetics, and pharmacodynamic data are under analysis • Received positive feedbacks from global KOLs in discussion regarding the Ph1 data Anatomical changes of retina: OCT (Optical Coherence Tomography) image Note: This i
Slide 19: Early-stage R&D Activities
Toward the successful creation and delivery of Life- changing value - Early-stage R&D Activities ⚫ Data-driven drug discovery - Collaboration with InveniAI ⚫ Deepening Kyowa Kirin’s Antibody Technology - Collaboration with Synaffix ⚫ The pursuit of innovation with a long-term perspective - VC fund i
Slide 20: Early-stage R&D Activities
Toward the successful creation and delivery of Life- changing value - Early-stage R&D Activities ⚫ Data-driven drug discovery - Collaboration with InveniAI ⚫ Deepening Kyowa Kirin’s Antibody Technology - Collaboration with Synaffix ⚫ The pursuit of innovation with a long-term perspective - VC fund i
Slide 21: Collaboration with InveniAI to date
Collaboration with InveniAI to date 1. 2018: Started collaboration Explore new indications for existing pipelines 2022: Obtaining new insights into target molecules and disease mechanisms of action suggesting efficacy by confirming preclinical POC 2. 3. 2020: Expanding collaboration 2021: Expanding
Slide 22: Exploration of new indications for existing pipelines
1. Exploration of new indications for existing pipelines • Found a target disease Kyowa Provides relevant data Kirin had not anticipated. Comprehensive AI-based tied to in-house pipeline inter-gene network analysis • Obtained non-clinical POC to search for diseases data Finding new drug discovery hy
Slide 23: Target Exploration for Next Generation Antibody Technology
2. Target Exploration for Next Generation Antibody Technology Bispecific antibody AI analysis and search for combinations of two target molecules technology and optimal diseases Extracts information/hypotheses that are difficult to be noticed Significantly streamlines the research for optimal target
Slide 24: Expanding collaboration for implementation of AI drug discovery capabilities
3. Expanding collaboration for implementation of AI drug discovery capabilities AlphaMeld® used directly by Kyowa Kirin researchers Accumulating experience in operating AI platforms to implement functions from both software and hardware Close interaction between AI experts from InveniAI and perspect
Slide 25: Early-stage R&D Activities
Toward the successful creation and delivery of Life- changing value - Early-stage R&D Activities ⚫ Data-driven drug discovery - Collaboration with InveniAI ⚫ Deepening Kyowa Kirin’s Antibody Technology - Collaboration with Synaffix ⚫ The pursuit of innovation with a long-term perspective - VC fund i
Slide 26: Kyowa Kirin's Antibody Drug Discovery Cycle
Kyowa Kirin's Antibody Drug Discovery Cycle Technology Successful creation and delivery of Know-how Production capacity innovative antibody drugs for antibody development New Open generation Innovation Antibody Technology etc… In-house Antibody Library 26 © Kyowa Kirin Co., Ltd.
Slide 27: Synaffix's ADC technology
Synaffix's ADC technology Nature, BioPharma-Dealmakers, March 2016 toxSYNTM Wide variety of payloads HydraSpaceTM Possible to select them depending on target diseases GlycoConnectTM The Payload conjugation technology by glycosylation enables our ADC research without modifying the amino acid sequence
Slide 28: License Agreement with Synaffix
License Agreement with Synaffix Proprietary Antibody Technology Proprietary ADC technology August, 2021 June, 2022 The pre-clinical stage studies are currently Licensing agreement Expansion of agreement underway for creation of Started ADC studies regarding - One more antibody added pipelines two an
Slide 29: Early-stage R&D Activities
Toward the successful creation and delivery of Life- changing value - Early-stage R&D Activities ⚫ Data-driven drug discovery - Collaboration with InveniAI ⚫ Deepening Kyowa Kirin’s Antibody Technology - Collaboration with Synaffix ⚫ The pursuit of innovation with a long-term perspective - VC fund i
Slide 30: Approach to generate more innovations for long-term growth
Approach to generate more innovations for long-term growth VC fund investment CVC activity Good balance between Exploration and Deepening Exploration for Knowledge Explore new technologies and new areas of business Conventional R&D activities innovation starvation Deepening Knowledge 30 © Kyowa Kiri
Slide 31: Outline of Activities
Outline of Activities VC Fund Investments ◼ To keep an eye out for the information related to drug discovery to search for innovative R&D seeds ◼ We have already invested in three companies to date and through this activity, a joint research agreement was signed with LUCA Science CVC Activities ◼ To
Slide 32: CVC and VC activities
Exploration for a wide range of drug discovery research seeds utilizing both activities Selecting company to invest Biotech ventures by Kyowa Kirin CVC CVC Activities B C CVC A Information and know-how E D f Partnering with companies G H if needed Information exchange VC Fund with VCs Investment J K
Slide 33: Summary
Summary ◼ Toward the Creation of pharmaceuticals for High-Unmet Medical Needs - From Our Investigational Products ⚫ KW-3357 (Preeclampsia) ⚫ KHK4951 (Tivozanib Eye-drop) ◼ Toward Continuous Creation of Life-changing Value - From Early-stage R&D Activities ⚫ Data-driven drug discovery - Collaboration
Slide 34: Gs yOwa KIRIN
Gs yOwa KIRIN