Kyowa Kirin Co., Ltd. · TSE:4151
Kyowa Kirin — Technical Presentation (March 2025)
25 slides from Kyowa Kirin's technical presentation, Mar 10, 2025
Slide 1: Rocatinlimab Update: Phase 3 ROCKET Program Summary of Results
Rocatinlimab Update Phase 3 Studies: ROCKET Program Summary of Results from 4 Trials in Adults with Moderate to Severe Atopic Dermatitis Director of the Board, Senior Managing Executive officer and Chief Medical Officer Takeyoshi Yamashita, Ph.D. Rocatinlimab is currently under clinical investigatio
Slide 2: Atopic Dermatitis is a chronic and heterogeneous inflammatory skin disease
Atopic Dermatitis (AD) is a chronic and heterogeneous inflammatory skin disease that imparts a significant burden on patients and caregivers ◼ AD causes excessively dry, itchy skin that can be painful ◼ Repeated scratching can cause the skin to thicken, Pain Pruritis harden or become vulnerable to i
Slide 3: Pathogenesis of Atopic Dermatitis and Existing Drug Targets
Pathogenesis of Atopic Dermatitis and Existing Drug Targets TH2 Cytokine Blockers1,2 Target TH2 responses via cytokines (e.g., IL- 13) or receptors (e.g., IL-4Ra) Inhibit downstream signal transduction Influence downstream effects JAK IL-4 IL-13 through the JAK- STAT pathway4 JAK Receptor Skin cell
Slide 4: T Cells Play a Central Role in inflammatory disease including AD
T Cells Play a Central Role in inflammatory disease including AD Effector T cells1 Memory T cells1,2 Key contributors to the release of cytokines that Persist after initial exposure to allergens, rapidly drive inflammation and contribute to the reactivating and releasing cytokines upon re- heterogen
Slide 5: AD cycles of exacerbation and remission driven by memory T cells
AD is a chronic condition characterized by repeated cycles of exacerbation and remission, where memory T cells in the skin are responsible for acute flare-ups (flares)1,2 T-cell driven lesions and Resolution of flares1,2; resident memory T cells Memory T cell reactivation immune memory are maintaine
Slide 6: Direct Role for T-Cells and the OX40 Receptor in the Pathogenesis of AD
Direct Role for T-Cells and the OX40 Receptor in the Pathogenesis of AD What is the OX40 Receptor? The OX40 receptor is a co-stimulatory molecule with increased expression on activated effector APC and memory T cells at sites of inflammation/disease, but is not expressed on naïve T cells2 OX40L OX40
Slide 7: T-cell Rebalance – Aiming for broad and sustained therapeutic effects
T-cell Rebalance – Aiming for broad and sustained therapeutic effects by addressing a root cause of inflammatory diseases T-cell balance1,2 T-cell imbalance1,5 Balance between naïve and activated T cells, Increased number and/or activity of T-cell Rebalance with appropriate effector and memory T-cel
Slide 8: Rocatinlimab – Phase 3 the ROCKET Program
Rocatinlimab – Phase 3 the ROCKET Program • Composed of eight global studies enrolling adult and adolescent moderate – severe AD patients • To date, over 3,300 patients have been enrolled with seven studies having completed enrollment • Studies were designed to examine long-term sustained efficacy a
Slide 9: Rocatinlimab – Today's agenda
Rocatinlimab – Today’s agenda Detailed data has been disclosed at the Late Breaking Abstract on the afternoon of March 8, 2025, US time, at the American Academy of Dermatology (AAD). Topline data disclosed on March 8, 2025 © Kyowa Kirin Co., Ltd. 10
Slide 10: ROCKET HORIZON AAD 2025 Presentation Summary
ROCKET HORIZON AAD 2025 Presentation Summary © Kyowa Kirin Co., Ltd. Rocatinlimab is currently under clinical investigation. Its efficacy and safety have not been evaluated by any health authority. 11
Slide 11: Phase 3 ROCKET-HORIZON Trial Evaluated Rocatinlimab Monotherapy vs PBO
Phase 3 ROCKET-HORIZON Trial Evaluated Rocatinlimab Monotherapy vs PBO in Adults With Moderate-to-Severe AD N = 543 Randomization Rocatinlimab 300 mg Q4W ROCKET-Ascend Screening + Wk 2 Loading Dose Long-Term Maintenance Trial (3:1) N = 726 N = 183 PBO Q4W + Wk 2 Loading Dose Safety Follow-up 16 Wks
Slide 12: Patient Demographics and Disease Characteristics at Baseline
Patient Demographics and Disease Characteristics at Baseline ROCA PBO Total Full Analysis Set N = 543 N = 183 N = 726 Age, yrs, mean (SD) 37.8 (14.6) 40.4 (15.6) 38.4 (14.9) Female, % 45.7 44.3 45.3 Hispanic/Latino, % 11.6 12.0 11.7 Race, % White 59.9 58.5 59.5 Asian 29.7 33.3 30.6 Black 3.5 5.5 4.0
Slide 13: Need for Rescue Therapy Was Reduced in the rocatinlimab Group vs PBO
Need for Rescue Therapy Was Reduced in the rocatinlimab Group vs PBO ROCA 300 mg Q4W PBO Full Analysis Set N = 543 N = 183 % % Any use of rescue therapy by Week 24* 33.5 41.5 Any use of topical rescue therapy onlya 28.7 29.5 Low/medium-potency topical therapy 15.5 13.1 High/super-high–potency topica
Slide 14: EASI-75 and vIGA-AD 0/1 Responses at Week 24
EASI-75 and vIGA-AD 0/1 Responses at Week 24 Primary analysis set (patients with any rescue use classified as nonresponders) Prespecified analysis (patients with any rescue use classified as observed) EASI-75 at Week 24 vIGA-AD 0/1 at Week 24 ROCA (N = 543) PBO (N = 183) ROCA (N = 543) PBO (N = 183)
Slide 15: EASI 75-89 and EASI 90-100 at Week 24
EASI 75-89 and EASI 90-100 at Week 24 Prespecified analysis (patients with any rescue use classified as observed) ROCA (N = 543) PBO (N = 183) EASI 90-100 EASI 75-89 EASI 90-100 EASI 75-89 50 47 50 44 Percent Responders (%) Percent Responders (%) 40 39 40 20 32 22 30 30 22 20 18 20 20 17 16 16 26 13
Slide 16: Longitudinal change of T cell number after rocatinlimab treatment
Longitudinal change of T cell number after rocatinlimab treatment CD4+ T Cells OX40+CD4+ T Cells 2 2 1 1 0 0 (cells/µL mean ± 95% CI) (cells/µL mean ± 95% CI) -2 Fold Change From Baseline Fold Change From Baseline -2 -4 -4 -6 -6 -8 -8 -10 -10 -12 -12 -14 ROCA -14 ROCA -16 PBO -16 PBO -18 -18 D1 W8 W
Slide 17: Safety Analysis
Safety Analysis ROCA 300 mg Q4W PBO Safety Analysis Set N = 544a N = 180b % % TEAEsc 68.4 63.3 Mild 28.1 16.1 Moderate 36.8 38.9 Severe 3.5 8.3 Serious adverse events 1.8 4.4 Fatal adverse events 0 0 Pyrexia and chills TEAEs leading to discontinuation of IP 2.6 2.8 were predominantly Serious 0.2 1.1
Slide 18: Topline Data of ROCKET IGNITE, SHUTTLE, and VOYAGER
Topline Data of ROCKET IGNITE, SHUTTLE, and VOYAGER © Kyowa Kirin Co., Ltd. Rocatinlimab is currently under clinical investigation. Its efficacy and safety have not been evaluated by any health authority. 19
Slide 19: ROCKET-IGNITE Topline Efficacy
ROCKET-IGNITE Topline Efficacy Treatment (24 Weeks) Rocatinlimab High Dose* Q4W + Loading dose at W2 ROCKET ASCEND 20210146 Long-term Randomization ASCEND Long-term Screening Maintenance Study Maintenance Trial N = 769 Rocatinlimab Low Dose Q4W + Loading dose at W2 Placebo Q4W + Loading dose at W2 1
Slide 20: ROCKET-SHUTTLE Topline Efficacy
ROCKET-SHUTTLE Topline Efficacy Treatment (24Weeks)* Rocatinlimab Dose 1 Q4W + TCS/TCI ROCKET ASCEND + loading dose at W2 Long-term Randomization Maintenance Trial Screening N = 746 Rocatinlimab Dose 1 Q4W + TCS/TCI + loading dose at W2 Safety Placebo Q4W + TCS/TCI Follow-Up + loading dose at W2 Wee
Slide 21: ROCKET-VOYAGER Topline Results
ROCKET-VOYAGER Topline Results ROCKET ASCEND Long-term Maintenance Trial • Tetanus and meningococcal vaccination occurred at week 20 prior to IP • IgG Abs were assessed at W20, Safety Follow-up pre vaccination, and at W24 The VOYAGER study successfully demonstrated that rocatinlimab does not interfe
Slide 22: ROCKET Program: Summary of four Phase 3 studies
ROCKET Program: Summary of four Phase 3 studies HORIZON Study Detailed Results @ 2025 AAD Late-breaking Abstract ◼ Patient background: Over 60% had previous experience with systemic therapy, and more than 20% had prior use of Biologics and JAK inhibitors ◼ Achieved co-primary endpoints and key secon
Slide 23: ROCKET Program – Future Plan
ROCKET Program – Future Plan Detailed data will be disclosed at the Late Breaking Session on the afternoon of March 8, 2025, local time, at the American Academy of Dermatology (AAD). (Follow-up at this meeting) Topline data disclosed on March 8 Detailed data to be disclosed in 2025/2026 Data readout
Slide 24: Future Looking: ROCKET ASCEND Design – Adult Cohorts
Presented at the 2025 American Academy of Dermatology Association Annual Meeting March 7–11, Orlando, Florida, US Future Looking: ROCKET ASCEND Design – Adult Cohorts1,* Study Subjects ROCKET-Ascend Cohort Key Questions (at 24 wks) (104 wks) • Long-term maintenance and/or additional efficacy on Adul
Slide 25: Anti-OX40 antibody – Potential new treatment option for multiple inflammatory diseases
Anti-OX40* antibody – Potential new treatment option for multiple inflammatory diseases Future expansion into indications where "T-cell rebalancing" is expected to lead to reduced disease activity Moderate to Severe Atopic Dermatitis Moderate to Severe P3 ROCKET Program in progress Asthma P2 study i