Full Press Release Details
Corporate Presentation January 2018
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update these forward-looking statements publicly, or to update the reasons why actual results could differ materially from those anticipated in the forward-looking statements, even if new information becomes available in the future. Forward-Looking
2017: A Great Year Pamrevlumab
Idiopathic Pulmonary Fibrosis Positive topline Phase 2b data reported, and presented at ERS Pancreatic Cancer Promising interim topline Phase 2 data Roxadustat Anemia in CKD (dialysis-dependent and non-dialysis dependent) Positive Phase 3 results
reported from two Phase 3 trials in China NDA accepted by China FDA U.S. NDA filing on track for 2018
2017: A Great Year Corporate and
Financial Strong and well-managed cash position Reported $762.7M cash balance as of September 30, 2017 Two successful financings $115.1M in net proceeds in April, supporting expansion of roxadustat beyond CKD in China $356.2M in net proceeds in
August, supporting pamrevlumab Phase 3 studies Received $15M milestone payment upon completion of roxadustat NDA submission to CFDA (China)
FibroGen in 2018 Science, Pipeline,
Partners Two late-stage, first-in-class programs with multiple indications Multi-billion dollar anemia, cancer, and fibrotic disease markets Roxadustat is the leader in HIF-PHI therapeutics Significant clinical and regulatory catalysts anticipated
for 2018 Partnerships with AstraZeneca and Astellas for global anemia markets Pamrevlumab is the leader in anti-CTGF therapeutics Significant clinical and regulatory catalysts anticipated for 2018 Wholly owned commercial rights
HIF-PHI PRECLINICAL PHASE 1 PHASE 2
PHASE 3 Roxadustat for CKD Anemia United States, Europe China [NDA Submitted 10/17] Japan Roxadustat for MDS Anemia United States China1 Product Portfolio ANTI-CTGF PRECLINICAL PHASE 1 PHASE 2 PHASE 3 Pamrevlumab Idiopathic Pulmonary Fibrosis
Pancreatic Cancer Duchenne Muscular Dystrophy BIOMATERIALS PILOT PIVOTAL FG-5200 (Biosynthetic Cornea)2 Corneal Blindness 2Five-year POC study in 10 patients completed; filed as device in China Wholly Owned Partnered Wholly Owned Medical Device
CTA Approved for Phase 2/3 Study
Pamrevlumab Fibrosis and Cancer
Pamrevlumab Innovative
Treatment for Fibroproliferative Diseases in Fibrosis and Cancer Strong Phase 2 Clinical Data: PoC for both disease categories Poised for Pivotal Trials in IPF and Pancreatic Cancer CELLULAR RESPONSES FIBROSIS CANCER IPF DMD Cirrhosis Cardiovascular
disease Target Diseases Pancreatic cancer Squamous cell lung cancer Melanoma LPA TGF IL-13 PDGF FGF VEGF CTGF PAMREVLUMAB Myofibroblast Activation ECM Deposition and Remodeling De-differentiation Proliferation Survival Motility and Invasion
Pamrevlumab: Pursuing Three Indications
With Unmet Medical Needs IPF & Pancreatic Cancer: Preparing for Phase 3 Completed Phase 2 in advanced disease (n=75) Positive exposure response relationship Blood level Cmin 150 g/mL: 2X median and 3X one-year survival Ongoing Phase
2 locally advanced inoperable disease- completing Randomized (pamrevlumab + gemcitabine + nab-paclitaxel) vs.(gemcitabine + nab-paclitaxel) Treatment duration 6 months, enrolled 37 Endpoints: conversion rate to resectable, successful resection, and
survival Results encouraging: Pivotal study design in progress PANCREATIC CANCER Ongoing open-label study in non-ambulatory patients Treatment duration 52 weeks, n=22 Endpoints: change in FVC (% predicted), and other measures of pulmonary and upper
body muscle function, and cardiac MRI DMD Completed open-label Phase 2 (n=89) Reversal of fibrosis, stable or improved fibrosis by HRCT, correlating to FVC data; observed in extension study beyond 48 weeks Well tolerated Completed double-blind Phase
2, topline data reported Aug, 2017 Randomized placebo-controlled trial (48 weeks), n=103 Met primary endpoints of change in FVC % predicted from baseline to week 48 Subgroup Analysis (N=90) Significant attenuation at 24 weeks of % change in fibrosis
by HRCT compared to placebo, p=.048; treatment effect relative to placebo > 3X reported in nintedanib Phase 3b study at 24 weeks Significant improvement in SGRQ relative to placebo, p<0.02, larger treatment effect than reported in nintedanib
Phase 3 studies. Phase 3 Study design in progress IPF
IPF Phase 2b Placebo-Controlled
Study (067) Promising Results Study Design Double-blind, placebo-controlled 103 patients randomized (1:1) to receive pamrevlumab or placebo for 48 weeks Phase 2 Positive Results Phase 3 Enabling Magnitudes of treatment effects larger than
published results on approved agents (pirfenidone or nintedanib) on 4 efficacy measures Difference in FVC change from baseline versus placebo Proportion of patients with FVC %-declined > 10% or death % Change in fibrosis (by HRCT) from baseline
at 24 weeks (New Analysis) St. George's Respiratory Questionnaire score (SGRQ) (New Analysis) Consistency of efficacy & safety results PRAISE (067) consistent with earlier single-arm Phase 2 study 049
PRAISE 049 Study Placebo PRAISE
(067) IPF Phase 2b Met Primary Endpoint; Significantly Less Decline in FVC% Predicted Change From BL at Week 48 Pamrevlumab Pamrevlumab *Both studies analyzed using the Linear Slope method Treatment effect with pamrevlumab consistent with results
from prior 049 Phase 2 Open-Label study*
PRAISE (067) IPF Phase 2b
Pamrevlumab Attenuated FVC Decline Pamrevlumab significantly attenuated FVC decline from Baseline to Week 48. p = 0.0249 In separate studies, pamrevlumab has larger treatment difference from placebo than nintedanib and pirfenidone in preservation of
FVC. Pamrevlumab Ph 2 PRAISE Study (067) Nintedanib INPULSIS Ph 3- 52 Weeks Pirfenidone ASCEND Ph 3- 52 wks 48- week Result Projected 52 wks Study 32 52 wks Study 34 52 wks (Linear Slope) 52 wks Pamrevlumab Placebo Pamrevlumab Placebo Nintedanib
Placebo Nintedanib Placebo Pirfenidone Placebo N 50 51 50 51 309 204 329 219 278 277 FVC (mL) -129 -308 -138 -332 -115 -240 -113.6 -207.3 -164 -280 LS mean diff 178 195 125 93.7 116 p-value 2.4899999999999999E-2 2.4E-2 <0.001 <0.001
<0.001 Relative difference 0.57899999999999996 0.58499999999999996 0.52100000000000002 0.45200000000000001 0.41399999999999998 Richeldi, NEJM 2014, 370: 22; 2071-2082 King, NEJM 2014; 370: 2083-92 Suppl 48 Weeks Subgroup Analysis (N=90)
Pamrevlumab Placebo FVC (mL) -143 -341 LS mean diff 198 P-value 2.12E-2
Visit (Weeks) P-Value* 0.1235 0.0527
0.0172 0.0103 Pamrevlumab, n (%) 3 (6.0%) 3 (6.0%) 5 (10.0%) 5 (10.0%) Placebo, n (%) 7 (13.7%) 9 (17.6%) 15 (29.4%) 16 (31.4%) Difference (%) -11.6% - 21.4% Relative Difference -56% -66% -66% -68% PRAISE (067) IPF Phase 2b Pamrevlumab Reduces The
Proportion of Subjects with FVC % Predicted Decline 10% or Death Relative Difference 68% ITT Analysis Both ITT and subgroup analyses of PRAISE show larger reduction in IPF progression (FVC % predicted decline >=10%) or death than
pirfenidone in ASCEND (ph 3 study). Summary of Efficacy Endpoints for Study FG-3019-067 Randomized Treatment Period ITT Population ALL (Total n = 103) ITT Population Excluding India (Total n = 90) Nintedanib Ph 3B Phase 3- Impulsis 1 Phase 3-
Impulsis 2 Efficacy Endpoint LS Mean of Change from Baseline LS Mean of Change from Baseline nintedanib Placebo Treatment nintedanib Placebo Treatment p-value nintedanib Placebo Treatment p-value FG-3019 Placebo Treatment
p-value FG-3019 Placebo Treatment p-value Change from BL to Week 48 in FVCpp and FVC (L) [1] n n 43 47 56 57 309 204 329 219 FVCpp48 FVCpp48 -3.05 -8.2799999999999994 5.23 2.0899999999999998E-2 FVC48 (mL) -120
-279 159 4.6899999999999997E-2 FVC48 (mL) -143 -341 198 2.12E-2 FVCpp decline >=10% or death at Week 48 Rate Rate 9.2999999999999999E-2 0.34 -0.247 7.6E-3 Hazard Ratio Hazard Ratio 0.27 1.8499999999999999E-2 Percent Change from BL in
Fibrosis Score by Visit [2] n n 41 45 42 45 % FIB 24 % FIB 24 5.5 17.100000000000001 -11.7 4.7899999999999998E-2 11.4 14.6 3.2 % FIB 48 % FIB 48 24.4 29.2 -4.8 0.63149999999999995 Absolute Change from BL in Fibrosis Score by
Visit [2] n n 41 45 FIB 24 FIB 24 0.8 2.1 -1.4 7.6200000000000004E-2 FIB 48 FIB 48 2.2000000000000002 4.4000000000000004 -2.2999999999999998 0.127 St. George's Respiratory Questionnaire (SGRQ) [3] n n 40 42
Symptom48 Symptom48 -5.34 2.92 -8.26 2.6599999999999999E-2 Activity48 Activity48 -4.66 3.44 -8.1 2.9100000000000001E-2 Impact48 Impact48 -0.15 6.27 -6.42 5.1700000000000003E-2 Total48 Total48
-2.29 5.07 -7.36 1.8499999999999999E-2 Total52 4.3499999999999996 4.3899999999999997 -0.05 0.97 2.8 5.48 -2.69 0.02 Efficacy Endpoint LS Mean of Change from Baseline LS Mean of Change from Baseline Pirfenidone Ph 3 ASCEND FG-3019 Placebo
Treatment p-value FG-3019 Placebo Treatment p-value Pirfenidone placebo Treatment Change from BL to Week 48 in FVCpp and FVC (L) [1] n 43 47 n 43 47 278 277 FVCpp48 -3.05 -8.2799999999999994 5.23 2.0899999999999998E-2
FVCpp48 -3.05 -8.2799999999999994 5.23 2.0899999999999998E-2 FVC48 (L) -0.14299999999999999 -0.34100000000000003 0.19800000000000001 2.12E-2 FVC48 (L) -0.14299999999999999 -0.34100000000000003 0.19800000000000001 2.12E-2 FVCpp
decline >=10% or death at Week 48 Rate 9.2999999999999999E-2 0.34 -0.247 7.6E-3 Rate 9.2999999999999999E-2 0.34 -0.247 7.6E-3 6.5% 0.17699999999999999 -0.11199999999999999 Hazard Ratio 0.27 1.8499999999999999E-2 Hazard Ratio 0.27
1.8499999999999999E-2 Pirfenidone Phase 3 ASCEND (52 Wks) P-Value <0.001 pirfenidone 6.5% Placebo 0.17699999999999999 Difference -0.112 Relative Difference -0.43 Summary of Efficacy Endpoints for Study FG-3019-067 Randomized Treatment Period ITT
Population Excluding India (Total n = 90) Nintedanib Ph 3B Phase 3- Impulsis 1 Phase 3- Impulsis 2 Efficacy Endpoint LS Mean of Change from Baseline nintedanib Placebo Treatment nintedanib Placebo Treatment p-value nintedanib Placebo
Treatment p-value FG-3019 Placebo Treatment p-value Change from BL to Week 48 in FVCpp and FVC (L) [1] n 43 47 56 57 309 204 329 219 FVCpp48 -3.05 -8.2799999999999994 5.23 2.0899999999999998E-2 FVC48 (L)
-0.14299999999999999 -0.34100000000000003 0.19800000000000001 2.12E-2 FVCpp decline >=10% or death at Week 48 Rate 9.2999999999999999E-2 0.34 -0.247 7.6E-3 Hazard Ratio 0.27 1.8499999999999999E-2 Percent Change from BL in Fibrosis Score by Visit
[2] n 41 45 42 45 % FIB 24 5.5 17.100000000000001 -11.7 4.7899999999999998E-2 11.4 14.6 3.2 % FIB 48 24.4 29.2 -4.8 0.63149999999999995 Absolute Change from BL in Fibrosis Score by Visit [2] n 41 45 FIB 24 0.8 2.1 -1.4
7.6200000000000004E-2 FIB 48 2.2000000000000002 4.4000000000000004 -2.2999999999999998 0.127 St. George's Respiratory Questionnaire (SGRQ) [3] n 40 42 Symptom48 -5.34 2.92 -8.26 2.6599999999999999E-2 Activity48 -4.66 3.44 -8.1
2.9100000000000001E-2 Impact48 -0.15 6.27 -6.42 5.1700000000000003E-2 Total48 -2.29 5.07 -7.36 1.8499999999999999E-2 Total52 4.3499999999999996 4.3899999999999997 -0.05 0.97 2.8 5.48 -2.69 0.02 Efficacy Endpoint LS Mean of
Change from Baseline Pirfenidone Ph 2 (study 016) FG-3019 Placebo Treatment p-value Pirfenidone placebo Treatment Change from BL to Week 48 in FVCpp and FVC (L) [1] n 43 47 278 277 FVCpp48 -3.05 -8.2799999999999994 5.23
2.0899999999999998E-2 FVC48 (L) -0.14299999999999999 -0.34100000000000003 0.19800000000000001 2.12E-2 FVCpp decline >=10% or death at Week 48 Rate 9.2999999999999999E-2 0.34 -0.247 7.6E-3 6.5% 0.17699999999999999 -0.11199999999999999
Hazard Ratio 0.27 1.8499999999999999E-2 PRAISE Subgroup Analysis of N=90 Treatment 48 wk -0.247 p- value 7.6E-3
PRAISE (067) IPF Phase 2b
Pamrevlumab Attenuates Fibrosis Progression (HRCT) Preliminary ITT and subgroup analyses of HRCT data show evidence for pamrevlumab to attenuate lung fibrosis. Full analysis coming soon. Pamrevlumab subgroup analysis of relative change in HRCT
compared to nintedanib Phase 3b 24-week result announced by Boehringer Ingelheim in 11/2017* Pamrevlumab *Nintedanib Pirfenidone No published data on HRCT change Difference between active and placebo in % change of lung fibrosis (HRCT) from baseline
to 24-weeks , Pamrevlumab significantly attenuated fibrosis in comparison to placebo in % change in fibrosis (HRCT) from baseline to 24 weeks, p=0.048. Preliminary HRCT analysis suggests placebo arm has greater increase in fibrosis than pamrevlumab
from baseline to 48 weeks Comparison to Boehringer Ingelheim - 24 week analysis: % change HRCT score v baseline Pamrevlumab favorably compares in treatment difference vs placebo control in relative changes in lung fibrosis (HRCT) from baseline
to 24-weeks across the two separate studies Relative change in fibrosis (HRCT) from baseline to 24 weeks Treatment difference of - 11.7%, p=0.048 *Ref: Nintedanib Ph 3b study: Lancaster, at Pulmonary Fibrosis Foundation (PFF) conference Nov.
PRAISE (067) IPF Phase 2b
Pamrevlumab Improves HRQoL (Lowers SGRQ Score) St. Georges Respiratory Questionnaire (SGRQ) Respiratory-specific questionnaire developed to assess HRQoL in patients with lung disease (originally in obstructive lung disease), used in a number of IPF
trials, including nintedanib Phase 3 studies & PRAISE 50 items grouped into 3 domains: symptoms, activity, and impact ITT Subgroup Pamrevlumab achieved improvement (reduction) in total SGRQ Score from baseline to 48 weeks, whereas placebo
resulted in decline (increase), significant difference with p= 0.019 in subgroup analysis. Pamrevlumab achieved substantially larger magnitude in difference than placebo in SGRQ change than nintedanib. Change in SGRQ Total Score From Baseline to
48-Weeks Treatment difference = -7.36 P= 0.019 pamrevlumab *Impulsis-1 *Impulsis-2 Pamrevlumab Ph 2 (48wk) *Nintedanib Ph 3 (52wk) Pirfenidone No published data on SGRQ Treatment difference of study drug from placebo l: Change In Total SGRQ From
Baseline to End of Treatment * Ref : Richeldi, NEJM 2014, 370: 22; 2071-2082
PRAISE (067) IPF Phase 2b
Pamrevlumab Safety Profile Pamrevlumab was well-tolerated, no safety signals; favorable relative to placebo in key safety variables in PRAISE study 067 Deaths: 3 (pamrevlumab) vs. 6 (placebo) TESAE (treatment-emergent serious adverse events) leading
to discontinuation: 3 (pamrevlumab) vs. 7 (placebo) Pamrevlumab has potential for a better GI safety profile than current drugs % patient on drug with TEAE of diarrhea/nausea Comparison of Proportion of treated Patients Reported TEAE of diarrhea or
nausea Pamrevlumab Nintedanib Pirfenidone *Ref: Ragu, ERJ 2016; Lancaster, poster at PFF 2017; Richeldi, NEJM 2014, 370: 22; 2071-2082; King, NEJM 2014
Market Opportunity for New IPF
Therapy Orphan disease U.S. prevalence ~44,000 to 135,0001 U.S. incidence ~21,000 to 52,0001 cases per year Progressive disease can result in irreversible loss of lung function with high morbidity and mortality rates Median survival of 3-5 years
following diagnosis Current treatments, pirfenidone and nintedanib Annualized sales rate of $1.4B Slow pulmonary function loss Modest effect on slowing disease progression No demonstration of reversal Require management of side effects 1Raghu 2006
and United Nations Population Division
Relationship of One-Year Survival to
Dose Percent One-Year Survival Rate Dose-related increase in survival Day 15 minimum pamrevlumab plasma level 150 g/mL 2x median survival (9.4 vs. 4.8 months) (p=0.025) 3x one-year survival (37% vs.11%) (p=0.01) Completed Ph 2 for
Treatment of Advanced Pancreatic CA (included patients with metastasis): Dose Response and Exposure/Survival Response Relationship of Survival to Pamrevlumab Day 15 Plasma Levels Percent Overall Survival Overall Survival Time (Months) Pamrevlumab in
Combination with Gemcitabine and Erlotinib (N=75) 22.5 35 45 25 15 10 3 17.5 KEY FINDINGS Weekly mg/kg Biweekly mg/kg FG-3019 >150 g/mL; n=38 FG-3019 <150 g/mL; n=37 p=0.025 Percent Overall Survival 0 5 10 15 20 25 30 35 40 45 0 20