Kyntra Bio, Inc. · NASDAQ:KYNB
Kyntra Bio — Fibrogen — Investor Presentation (February 2017)
36 slides from Kyntra Bio's investor presentation, Feb 16, 2017
Slide 1: Corporate Overview February 2017
Corporate Overview February 2017 Exhibit 99.1
Slide 2: Forward-looking statements
This presentation, the accompanying modules, and in each case the oral commentary contain “forward-looking” statements that involve substantial risks and uncertainties. All statements other than statements of historical facts contained in this presentation, the accompanying modules, and in each case
Slide 3: Investment Highlights: Late-Stage Pipeline and Roxadustat
Investment Highlights: Late-Stage Pipeline and Roxadustat Late-stage first-in-class therapeutics targeting multiple indications and multi-billion dollar markets Roxadustat: Oral HIF-PH Inhibitor in WW Phase 3 development Potential safety advantages over ESAs Anemia in CKD patients and in cancer pati
Slide 4: Investment Highlights: Pamrevlumab and Corporate
Investment Highlights: Pamrevlumab and Corporate Pamrevlumab: Disease-modifying anti-CTGF antibody Fibrotic disease therapeutic with multiple potential indications Idiopathic pulmonary fibrosis (IPF) Pancreatic cancer Duchenne Muscular Dystrophy (DMD) Monotherapy and/or combination cancer therapy St
Slide 5: Our Platforms and Product Portfolio
HIF PLATFORM PRECLINICAL PHASE 1 PHASE 2 PHASE 3 Roxadustat (HIF-PH Inhibitor for CKD anemia) United States / Europe China Japan Our Platforms and Product Portfolio FIBROTIC DISEASE PLATFORM PRECLINICAL PHASE 1 PHASE 2 PHASE 3 FG-3019 (Anti-CTGF Antibody) Idiopathic Pulmonary Fibrosis Pancreatic Can
Slide 6: Roxadustat (FG-4592)
FIBROGEN Roxadustat (FG-4592)
Slide 7: Roxadustat mechanism: HIF stabilization
LOW OXYGEN (e.g., High Altitude) HIFα ROXADUSTAT HIFβ HIFα HIF-PH1 Enzymes HIF-PH Enzymes NORMAL OXYGEN ✕ Degradation HIFα Roxadustat Transiently Stabilizes HIFα Gene Transcription HIFα Degrades Rapidly EPO Within or Near Physiological Range RBC Production Hepcidin Levels Iron Transport to Bone Marr
Slide 8: Anemia Treatment and ESA Limitations
Anemia Treatment and ESA Limitations ESAs¹: Current SOC Post-FDA Actions: ESA Declining but Substantial Mean EPO/ Week (IUs) as of Dec 31 ESA Revised Label July 2011 CHRONIC KIDNEY DISEASE “In controlled trials, patients experienced greater risks for death, serious adverse cardiovascular reactions,
Slide 9: Roxadustat EPO levels within physiological range
1 Cmax data for roxadustat estimated for a subset of 243 patients who achieved Hb response and were dosed at expected therapeutic doses. 2 Milledge & Cotes (1985) J Appl Physiol 59:360. 3 Goldberg et al. (1993), Clin Biochem 26:183, Maeda et al. (1992) Int J Hematol 55:111. 4 Kato et al. (1994) Ren
Slide 10: Phase 2 Program Clinical Results
Phase 2 Program Clinical Results Hb correction Regardless of inflammation No IV iron supplementation required Potential to avoid high-dose ESA risk Favorable safety profile vs. ESAs No hypertensive effect No thrombocytosis Other effects observed Reduced hepcidin Reduced cholesterol Optimal dosing re
Slide 11: Global Phase 3 Program Regulatory Pathways
Global Phase 3 Program 4 Regulatory Pathways U.S./EU Global Phase 3 with target 2018 NDA submission Astellas Japan Phase 3 (4 DD-CKD; 2 NDD-CKD): all studies underway FibroGen China Phase 3 (1 DD-CKD; 1 NDD-CKD): topline data reported in early 2017 *AST’s Dolomite CL-610 NDD-CKD, darbepoetin compara
Slide 12: China Phase 3 Studies – Primary Endpoints Met
Primary Assessment period Subjects Arm 204 Roxadustat 26 weeks 100 ESA 52 weeks 101 Roxadustat 8 weeks 50 Placebo Total 2 Studies: 305 Roxadustat 150 455 Non-dialysis (Naïves) Dialysis (Conversion & Maintenance) 100 Roxadustat Roxadustat China Phase 3 Studies – Primary Endpoints Met 808 Study 806 St
Slide 13: Roxadustat vs Kirin EPO in Dialysis Patients (Study 806)
Roxadustat – More Efficacious Than Kirin EPO (Active Control) in Maintaining Hb levels in Stable CKD-Dialysis Patients (Study 806) PPS Mean (SD) Hb Values (g/dL) Roxadustat (n=196) Kirin EPO (n=98) Baseline 10.4 (0.7) 10.4 (0.7) Week 23-27 11.2 (0.9) 10.9 (0.8) Change from baseline 0.75 (1.1) 0.46 (
Slide 14: Roxadustat Corrects Anemia in CKD-NDD Patients (Study 808)
Roxadustat Corrects Anemia In CKD-NDD Patients (Study 808) Mean (SD) Hb Values (g/dL) Roxadustat (n=101) Placebo (n=50) Baseline Hb mean (SD) 8.9 (0.8) 8.9 (0.7) Week 7-9 Hb, mean (SD) 10.8 (1.4) 8.5 (0.98) Change in Hb from baseline 1.9 (1.2) -0.4 (0.8) Difference (SE), P-value 2.25 (0.19); p<0.000
Slide 15: Global Phase 3 Development Program
Global Phase 3 Development Program STUDY # COMPARATOR TARGET # LOCATIONS CHINA EUROPE U.S SPONSOR Phase 3 Trials in Process Stable Dialysis CL-0613 Epoetin α/Darbe 750 EU, MEA3 36+ wks 52+ wks Astellas FG-064 Epoetin α Up to 750 US+/-Global 36+ wks 52+ wks FibroGen FG-806 Epoetin α 300 China 26-52 w
Slide 16: Roxadustat Potential to Address Unmet Need in Multiple Markets
Disease Dialysis Pre-Dialysis Limitations on ESA Treatment Clinician Nephrologist Where ESA Is Avail Now 36% Anemic¹ 20% Anemic¹ Endocrinologist/ Diabetologist Cardiologist/ Internist Myelodysplasia Chemotherapy Induced Anemia Oncologist IBD, Lupus, RA, etc. GI/Rheumatologist/ Internist Roxadustat H
Slide 17: Roxadustat Market Opportunity
Disease Dialysis Pre-Dialysis Roxadustat Market Opportunity Clinician Nephrologist Where ESA Is Avail Now 36% Anemic¹ 20% Anemic¹ Endocrinologist/ Diabetologist Cardiologist/ Internist Myelodysplasia Chemotherapy- Induced Anemia Oncologist IBD, Lupus, RA, etc. GI/Rheumatologist/ Internist Potential
Slide 18: Expanding into Oncology-Related Anemias
Expanding into Oncology-Related Anemias China Significant unmet need in oncology-related anemias Anemia associated with MDS and chemotherapy-induced anemia Severe blood shortage in China CFDA reviewing CTA for MDS anemia placebo-controlled Phase 2/3 study U.S. FDA accepted Phase 3 study IND for anem
Slide 19: Roxadustat Global Anemia Partnerships
Roxadustat Global Anemia Partnerships $ Millions JAPAN, EU, ETC. U.S., CHINA, ROW Payments Received to Date Equity Investment in FibroGen $81 $20 $101 Upfront, Non-Contingent $360 $402 $762 Development & Reg. Milestones $543 $571 $128 Commercial Milestones $15 $653 $0 POTENTIAL TOTAL $918M $1,626M $
Slide 20: Pamrevlumab (FG-3019)
FIBROGEN Pamrevlumab (FG-3019)
Slide 21: Central Role of CTGF in Fibrosis
Central Role of Connective Tissue Growth Factor (CTGF) in Fibrosis LEADERSHIP IN FIBROSIS CELLULAR RESPONSES FIBROSIS CANCER IPF DMD Cirrhosis Cardiovascular disease Target Diseases Pancreatic cancer Squamous cell lung cancer Melanoma LPA TGFβ IL-13 PDGF FGF VEGF CTGF FG-3019 Myofibroblast Activatio
Slide 22: IPF, Pancreatic Cancer, and DMD
IPF, Pancreatic Cancer, and DMD Completed Phase 2 in advanced disease Positive data on median survival and one-year survival Ongoing Phase 2 locally advanced inoperable disease; data in Q1 2017 Randomized (pamrevlumab + gemcitabine + nab-paclitaxel) vs. (gemcitabine + nab-paclitaxel) Treatment durat
Slide 23: Improved Fibrosis Shown in Preclinical and Phase 2 Data
Improved Fibrosis Shown in Preclinical Data and Open-Label Phase 2 Study Improved / Stable Fibrosis Correlates with Improved Lung Function Improved/ Stable Fibrosis Worse Fibrosis Reversal of Lung Fibrosis by HRCT at Week 48 Improved/Stable 35% Subject NORMAL Hounsfield Units Increasing Fibrosis Rev
Slide 24: IPF Opportunity
IPF Opportunity Orphan disease: U.S. prevalence 106,400 (2014); ~120,800 (2019) 1 Progressive disease can result in irreversible loss of lung function with high morbidity and mortality rates Median survival of 3-5 years following diagnosis Pirfenidone and nintedanib approved by FDA in 2014 and EMA i
Slide 25: IPF Development Progress
IPF Development Progress Ongoing Phase 2 trial Completed enrollment Placebo-controlled portion of study with 103 patients Sub-study combining pamrevlumab with pirfenidone or nintedanib Expect topline data for both study arms in third quarter Presented data from open-label extension study at ICLAF 20
Slide 26: Relationship of One-Year Survival to Dose
Relationship of One-Year Survival to Dose Percent One-Year Survival Rate Dose-related increase in survival Day 15 minimum FG-3019 plasma level ≥ 150 µg/mL 2x Median survival (9.4 vs. 4.8 months) (p=0.025) 3x One-year survival (37% vs. 11%) (p=0.01) Phase 2 Dose-Dependent Survival in Advanced Pancrea
Slide 27: Critical Need in Locally Advanced Pancreatic Cancer
Critical Need in Locally Advanced Pancreatic Cancer Population 53,000 new cases per year in U.S.1 ~26,500 (50%) patients have no detectable metastases at presentation ~8,000 (15-20%) classified as resectable1 ~18,500 (30-35%) with pancreatic cancer that precludes resection1 Differential outcomes and
Slide 28: Randomized Open-Label Phase 1/2 Trial in Pancreatic Cancer
Randomized Open-Label Phase 1/2 Trial in Pancreatic Cancer Subjects with locally advanced inoperable pancreatic cancer Randomized 2:1 (N=42): chemotherapy vs. chemotherapy+pamrevlumab Treatment duration six months Re-assess for surgery eligibility and surgical outcome Results reported January 2017 a
Slide 29: Biosynthetic Cornea Program
Biosynthetic Cornea Program
Slide 30: Major Unmet Need in China and Emerging Markets
U.S. (Canada and EU) China (and Other Emerging Markets) Major Unmet Need In China and Emerging Markets 42,000 transplants/year in U.S. Established donor banking system Requires long-term corticosteroid use 5-year rejection rate of 35% Cadaver transplants limited Lack institutionalized tissue banking
Slide 31: Published Proof-of-Concept Study with Four-Year Follow Up
Published Proof-of-Concept Study with Four-Year Follow Up Recombinant Human Collagen III Corneas Implanted by Deep Anterior Lamellar Keratoplasty (DALK) Subjects Single center, 10 subjects Inclusion criteria: patients on wait list for cornea transplant Diagnosis: keratoconus - 9; corneal scar - 1 Re
Slide 32: China Launch First with Global Markets Following
China Launch First with Global Markets Following Regulatory Strategy Designated Chinese Domestic Class 3 medical device Seek marketing approval in China first Seek CE mark and export from China Program Status Clinical study-enabling evaluation in 2017 with GMP facility-produced cornea Physical chemi
Slide 33: Upcoming Milestones
FIBROGEN Upcoming Milestones
Slide 34: 2017 Milestones
2017 Milestones Roxadustat in China Anemia associated with CKD in both DD-CKD and NDD-CKD Phase 3 topline data (January 2017) NDA submission Q3 2017 Anemia associated with MDS Phase 2/3 clinical trial application under review by CFDA Roxadustat U.S./ROW Large ongoing global CKD anemia program (NDA s
Slide 35: 2017 Milestones (continued)
2017 Milestones (continued) Pamrevlumab Phase 2 placebo-controlled IPF clinical data (Q3 2017) Phase 2 pancreatic cancer study results (YE 2017/Q1 2018) FG-5200 biosynthetic cornea Complete preclinical testing of cornea from GMP facility (2017) Initiate clinical evaluation of novel medical device (2
Slide 36: Thank you
Thank you