Kubota Pharmaceutical Holdings Co. Ltd. · TSE:4596
Kubota Pharmaceutical — Acucela — BioCentury NewsMakers in the Biotech Industry (September 2014)
36 slides from Kubota Pharmaceutical's conference presentation, Sep 26, 2014
Slide 1: Acucela at BioCentury NewsMakers in the Biotech Industry
Acucela is a clinical-stage biotechnology company that specializes in discovering and developing novel therapeutics to treat and slow the progression of sight-threatening ophthalmic diseases affecting millions of individuals worldwide. Acucela Inc. (TSE: 4589) BioCentury NewsMakers in the Biotech In
Slide 2: Disclaimer
Disclaimer This presentation contains forward-looking statements concerning our product development, our technology, our competitors, our intellectual property, our financial condition and our plans for research and development programs that involve risks, uncertainties and assumptions. These statem
Slide 3: Company Overview/Introduction
Company Overview/Introduction
Slide 4: Company Snapshot
Company Snapshot BioCentury NewsMakers in the Biotech Industry - September 26, 2014 4 An ophthalmology-focused, science-driven biotechnology company People and Infrastructure • Broad-skilled employee base in research, development and operations • Three Locations: Seattle, WA Global Headquarters; Bot
Slide 5: Corporate Strategy
Corporate Strategy BioCentury NewsMakers in the Biotech Industry - September 26, 2014 5 Three core strengths drive opportunities to expand research and business development Business development Expanded research Leader in VCM research Large potential opportunity with emixustat Unique and robust part
Slide 6: Ryo Kubota, MD, PhD Profile
Ryo Kubota, MD, PhD Profile • Scientific Research and Ophthalmology − Performed over 1,000 ocular surgeries to treat cataract, retinal detachment, diabetic retinopathy glaucoma, strabismus, and other conditions − Discovered a glaucoma gene, myocilin; earned the Suda Award − Established Acucela in 20
Slide 7: Senior Leadership
Senior Leadership BioCentury NewsMakers in the Biotech Industry - September 26, 2014 7 Hien DeYoung, CHIC, SPHR Vice President Human Resources Keiko Mitsunobu Executive Director Alliance Management & Business Development Karin Ludwig, MD Vice President Clinical Development Hans-Peter Pfleger, PhD Vi
Slide 8: Board of Directors
Board of Directors BioCentury NewsMakers in the Biotech Industry - September 26, 2014 8 Ryo Kubota, MD, PhD Founder, Chairman and CEO Acucela Inc. Peter Kresel Sr. Vice President, Global Regulatory Affairs, Allergan (retired) Glen Sato, JD Partner, Cooley LLP Michael Schutzler CEO, The Washington Te
Slide 9: Unique and Robust Partnership with Otsuka Pharmaceutical
Unique and Robust Partnership with Otsuka Pharmaceutical BioCentury NewsMakers in the Biotech Industry - September 26, 2014 9 Investigational Product Candidate Potential Indication License Territory Financial Terms Emixustat hydrochloride (developed by Acucela) Dry AMD and other ophthalmic indicatio
Slide 10: Visual Cycle Modulation (VCM) Technology
Visual Cycle Modulation (VCM) Technology
Slide 11: The Visual Cycle
The Visual Cycle BioCentury NewsMakers in the Biotech Industry - September 26, 2014 11 The visual cycle is a biological process in which dietary vitamin A is converted to a light -sensitive compound. This process takes place in the photoreceptors and in a specialized tissue called the retinal pigmen
Slide 12: Visual Cycle Modulators
Visual Cycle Modulators BioCentury NewsMakers in the Biotech Industry - September 26, 2014 12 Visual cycle modulators are small molecule compounds which target specific proteins of the visual cycle. The intended effect of VCM compounds is to reduce retinal toxins and preserve the integrity of retina
Slide 13: Age-related Macular Degeneration (AMD)
Age-related Macular Degeneration (AMD)
Slide 14: About AMD
About AMD • The Disease − AMD occurs when the small central portion of the retina, known as the macula, deteriorates − AMD exhibits in two forms – dry and wet – Dry (non-neovascular) AMD occurs in progressive stages – Early Dry AMD can cause blurry vision – Geographic Atrophy (GA) is the advanced fo
Slide 15: AMD Disease Progression
AMD Disease Progression BioCentury NewsMakers in the Biotech Industry - September 26, 2014 15 Normal Retina Dry AMD (Drusen) Wet AMD (Neo-vascularization) Late-Stage Dry AMD (Geographic Atrophy or GA)
Slide 16: AMD: Proposed Disease Progression and Potential Targets for Intervention
AMD: Proposed Disease Progression and Potential Targets for Intervention BioCentury NewsMakers in the Biotech Industry - September 26, 2014 16 » Visual Cycle Modulators » Anti-amyloid ß » Complement Inhibitors » Anti-VEGF Medications (CNV) Early Pathology Intermediate Pathology Late Pathology Aging,
Slide 17: Visual Impact of GA Associated with Dry AMD
Visual Impact of GA Associated with Dry AMD BioCentury NewsMakers in the Biotech Industry - September 26, 2014 17
Slide 18: Autofluorescence Associated with the Progression of Atrophy
Autofluorescence is Suggested to be Associated with the Progression of Atrophy* BioCentury NewsMakers in the Biotech Industry - September 26, 2014 18 Photo courtesy of Professor Dr. Frank G. Holz, Director of University Eye Hospital, Bonn, Germany. *Frank G. Holz, MD, Erich C. Strauss, MD, Steffen S
Slide 19: Emixustat in Clinical Studies for Geographic Atrophy Associated with Dry AMD
Emixustat in Clinical Studies for Geographic Atrophy (GA) Associated with Dry Age- Related Macular Degeneration (AMD)
Slide 20: Emixustat Hydrochloride Overview: Lead Investigational Product Candidate
Emixustat Hydrochloride Overview: Lead Investigational Product Candidate • Non-retinoid small molecule • Oral administration • Designed to modulate visual cycle activity and reduce the accumulation of toxins in the retina • Targets a key rate-limiting enzyme of the visual cycle • Measureable pharmac
Slide 21: Drug Development Path: Emixustat for GA Associated with Dry AMD
Drug Development Path: Emixustat for GA Associated with Dry AMD BioCentury NewsMakers in the Biotech Industry - September 26, 2014 21 2005 2007 2009 2012 2013 » Phase 2b/3 clinical trial commences » Two-year treatment » Toxicology » Proof-of-concept in pre- clinical model » Medicinal chemistry » Fiv
Slide 22: Reduction of Rod Photoreceptor Activity and Protection from Light Damage in Mice
Reduction of Rod Photoreceptor Activity and Protection from Light Damage in Mice Treated with Single Dose of Emixustat BioCentury NewsMakers in the Biotech Industry - September 26, 2014 22 wild-type outer nuclear layer Emixustat (1 mg/kg), single dose pre-treatment Protection from light damage +10K
Slide 23: Attenuation of A2E Accumulation in Mice Treated with Emixustat
25 20 15 10 5 Attenuation of A2E Accumulation in abca4-/- Mice Treated Three Months with Emixustat BioCentury NewsMakers in the Biotech Industry - September 26, 2014 23 Reduction of A2E/lipofuscin Emixustat (3 mg/kg), 3-month treatment abcr-/- abcr-/- wild-type A 2E ( pmol /e ye ) (% No rma liz ed t
Slide 24: Emixustat Reduces Retinal Neovascularization in Mouse Model
Emixustat Treatment Reduces Retinal Neovascularization in Mouse Model of Oxygen- Induced Retinopathy BioCentury NewsMakers in the Biotech Industry - September 26, 2014 24 75% O2 + vehicle 75% O2 + Emixustat ED50 = 0.46mg/kg 40 30 20 10 % N eo va sc ul ari zat io n Treatment ED50 = 0.46 mg/kg 75% O2
Slide 25: Emixustat Phase 1a Clinical Trial Results: Mean Oral PK Profiles
Emixustat Phase 1a Clinical Trial Results: Mean Oral PK Profiles BioCentury NewsMakers in the Biotech Industry - September 26, 2014 25 20 15 10 5 Time (h) 4 12 24 20 16 8 0 Mea n Plasma Co nc ent rat io n (n g/ m l) 2mg/subject 7mg/subject 10mg/subject 20mg/subject 20mg/subject (cohort 4) 40mg/subje
Slide 26: Phase 2a Dose Escalation Study
Phase 2a Dose Escalation Study • Design − Multi-center, randomized, double-masked, placebo-controlled, dose-escalation study of the safety, tolerability, pharmacokinetics and pharmacodynamics of emixustat – A total of 72 patients with GA associated with dry AMD were enrolled – Treatment period for u
Slide 27: Rod Visual Cycle Suppression with Emixustat in Patients with GA
Minutes After Bleach 200 150 100 50 20 30 10 Mea n b- W av e Ampl itu de (µV ) Rod Visual Cycle Suppression with Emixustat in Patients with GA (at Day 14) BioCentury NewsMakers in the Biotech Industry - September 26, 2014 27 Placebo (N=14) 2mg (N=12) 5mg (N=11) 7mg (N=11) 10mg (N=5) *Dose *Suppressi
Slide 28: Clinical Development Progress to Date
Clinical Development Progress to Date • Completed Clinical Trials to Date − Five phase 1 clinical trials (normal healthy adults) − One phase 2a clinical trial (GA subjects) − Total of 179 human subjects exposed to emixustat • On-going Clinical Trial – Phase 2b/3 “SEATTLE” Study − Design – Two year,
Slide 29: OPA-6566 - Investigational Compound for Open-angle Glaucoma and/or Ocular Hypertension
OPA-6566 - Investigational Compound for Open-angle Glaucoma and/or Ocular Hypertension
Slide 30: OPA-6566 Overview and Status
OPA-6566 Overview and Status • Approach − Adenosine A2a receptor agonist • Development Status − IND filed in June 2011 − US phase 1/2 clinical trial conducted in patients with open-angle glaucoma and/or ocular hypertension • Current Status − After a phase 1/2 clinical trial in the United States, it
Slide 31: Financials
a ACUC ELA Financials
Slide 32: Condensed Statements of Operations
Condensed Statements of Operations (in thousands, except per share data) BioCentury NewsMakers in the Biotech Industry - September 26, 2014 32 2014 2013 2014 2013 Revenue from collaborations 9,086$ 11,023$ 19,632$ 27,003$ Expenses: Research and development 6,501 7,845 14,471 15,973 General and admin
Slide 33: Condensed Balance Sheets
Condensed Balance Sheets (in thousands) BioCentury NewsMakers in the Biotech Industry - September 26, 2014 33 June 30, December 31, 2014 2013 (unaudited) Assets Total current assets 128,843 42,281 Property and equipment, net 861 1,112 Long-term investments 63,229 3,478 Long-term deferred tax asset 1
Slide 34: Financial Overview
Note: FY09 and FY10 are unaudited figures. Financial Overview 27,019 36,457 34,226 46,424 52,947 - 10,000 20,000 30,000 40,000 50,000 60,000 FY09 FY10 FY11 FY12 FY13 Revenues from Collaboration 18,274 25,297 24,183 31,604 36,405 - 10,000 20,000 30,000 40,000 FY09 FY10 FY11 FY12 FY13 R&D 23,638 34,80
Slide 35: Investor Rationale
Investor Rationale • Significant unmet medical need (GA associated with dry AMD) − No approved therapy currently on the market • Differentiated product candidate (emixustat) − Oral administration − Unique mechanism of action − Ongoing phase 2b/3 clinical trial (ClinicalTrials.gov identifier: NCT0180
Slide 36: Acucela at BioCentury NewsMakers in the Biotech Industry
Acucela is a clinical-stage biotechnology company that specializes in discovering and developing novel therapeutics to treat and slow the progression of sight-threatening ophthalmic diseases affecting millions of individuals worldwide. Acucela Inc. (TSE: 4589) BioCentury NewsMakers in the Biotech In
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