New Ideas. Better Medicines. Presentation to Investors. January 2019
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1 New Ideas. Better Medicines. Presentation to Investors January 2019
2 Forward Looking Statements 2 This presentation contains forward looking statements that involve substantial risks and uncertainties. In some cases, you can identify forward looking statements by the following words: may, will, could, would, should, expect, intend, plan, anticipate, contemplate, believe, estimate, predict, project, seek, potential, continue, ongoing or the negative of these terms or other comparable terminology, although not all forward looking statements contain these words. These statements relate to future events or our future financial performance or condition and involve known and unknown risks, uncertainties and other factors that could cause our actual results, levels of activity, performance or achievement to differ materially from those expressedorimpliedbytheseforward looking statements. These risks, uncertainties and other factors are described more fully in our periodic reports filed with the Securities and Exchange Commission (SEC), including our Annual Report on Form 10 K for theyear ended December 31, 2017 filed with the SEC on March 12, 2018, particularly in the sections titled Risk Factors and Management s Discussion and Analysis of Financial Condition and Results of Operations and our Quarterly Reports on Form 10 Qforthequarters ended March 31, 2018, June 30, 2018 and September 30, 2018 filed with the SEC on May 9, 2018, August 9, 2018 and November 8, 2018, respectively. In light of the significant uncertainties in our forward looking statements, you should not placeunduereliance on these statements or regard these statements as a representation orwarrantybyusoranyotherpersonthatwewillachieveour objectives and plans in any specified timeframe, or at all. The forward looking statements contained in this presentation represent our estimates and assumptions only as of the date of this presentation and, except as required by law, we undertake no obligation to update or revise publicly any forward looking statements, whether as a result of new information, future events or otherwise after the date of this presentation. This presentation also contains estimates, projections and other information concerning our industry, our business, and the markets for our drug candidates, as well as data regarding market research, estimates and forecasts prepared by our management. Information that is based on estimates, forecasts, projections, market research or similar methodologies is inherently subject to uncertainties and actual events or circumstances may differ materially from events and circumstances reflected in this information.
3 CCXI Value Creation: From Basic Science to Commercialization Precision Approach Leads to Better Medicines Broad Pipeline of Unique, Orally Administered, Small Molecule Therapeutics Advanced Programs Focused on Orphan Diseases Establishing Strong Global Commercial Capabilities CCXI drug candidates selectively target a specific chemoattractant or chemokine receptor Targeting indications with clear regulatory pathways and multibillion dollar market potential Enrollment complete in landmark Phase III ADVOCATE trial in ANCA vasculitis CCXI owns commercial rights in the U.S. Building marketing, medical, quality capabilities Leaves rest of the immune system intact Robust clinical data generated with avacopan and CCX140 Extending the reach of avacopan initiated clinical development in hidradenitis suppurativa (HS) Ex U.S. strategic commercial alliance with Vifor Pharma 3
4 Differentiated Approach: Inhibiting Diseases Driven by Dysregulated Cell Migration Chemoattractation Exclusive Focus ChemoCentryx is a leader in chemoattractant system science Small Molecules Orally Administered, convenient, greater patient compliance Highly Selective Leaves rest of immune system intact; Not immunosuppressive Broad Applicability Approach applicable to Inflammatory, autoimmune diseases and cancer 4
5 Broad Pipeline from Novel Discovery Platform THERAPEUTIC AREA DRUG/ INDICATION PRECLINICAL PHASE 1 PHASE 2 PHASE 3 TARGET Complement Inhibition in Orphan Diseases Avacopan (formerly CCX168)/ C5aR ANCA ASSOCIATED VASCULITIS C3 GLOMERULOPATHY HIDRADENITIS SUPPURATIVA Chronic and Orphan Kidney Diseases CCX140/CCR2 ORPHAN KIDNEY DISEASE FOCAL SEGMENTAL GLOMERULOSCLEROSIS DIABETIC KIDNEY DISEASE, SUCCESSFULLY COMPLETED PHASE II Other Inflammatory and Autoimmune Diseases CCX507/CCR9 CCR6 IBD: ULCERATIVE COLITIS TH17 DRIVEN DISEASE, e.g. PUSTUALAR PSORIASIS Immuno Oncology CCX872/ CCR2 ADVANCED PANCREATIC CANCER OTHER ONCOLOGY INDICATIONS 5
6 Goals We Established at JP Morgan 2018 Complete ADVOCATE phase 3 pivotal trial enrollment mid year Launch clinical trials in 3 other orphan indications Update results of other pipeline programs CCX872 in pancreatic cancer Continue progress with new pipeline candidates Conclusion 2018: Very Strong Year of Execution 6
7 What You Will Hear Today 1 Spotlight on Anticipated ADVOCATE Pivotal Phase III Data 2 Compelling Opportunity in Hidradenitis Suppurativa 3 Exciting News Flow Anticipated Over the Next 18 Months 7
8 Avacopan Targeted Approach to Complement Diseases: Pipeline in a Drug 8
9 Avacopan: Unique Orally Administered C5aR Inhibitor in Late Stage Clinical Development Targeting the Complement Pathway Downstream (Left on Chart) is Best Avacopan avoids long term biological consequences of upstream complement inhibition C5a Antibodies Avacopan C5aR Avacopan does not block production of C5b 9; leaving host defense mechanism (MAC) in place 3. Reason for black box warning for eculizumab 3. Avacopan preserves beneficial functions of C5L2 pathway 9
10 Avacopan Targets Only C5aR (C5aR1 / CD88); Why Preserving C5L2 (C5aR2) is Important An Anti inflammatory Function for the Complement Anaphylatoxin C5a binding Protein, C5L2. Gerard et al, J. Biol Chem, 2005 Disruption of the Complement Anaphylatoxin Receptor C5L2 Exacerbates Inflammation in Allergic Contact Dermatitis. Wang et al, Jour Immunol, 2013 C5L2, the Second C5a Anaphylatoxin Receptor, Suppresses LPS Induced Acute Lung Injury. Wang et al, Am Jour Resp Cell and Mol Bio, 2016 C5a Receptor (CD88) Blockade Protects Against MPO ANCA Glomerulonephritis (GN). Xiao et al, Jour Am Soc Nephrology, 2014 (Paper contains data that C5L2 deficiency exacerbates GN) 10
11 Avacopan Advantage Beneficial Properties of the Medicinal Compound Avacopan Makes the C5aR Pharmacologically Inert Exquisitely potent on target (inhibits at concentrations of 10 s 100 s picomolar ) Exquisitely specific for C5aR No off target effects observed at multiples of therapeutic doses Clean in vivo safety profile (NOEL is highest dose tested) In vivo avacopan inhibits entirely C5a activation of neutrophils Avacopan ablates C5a induced neutrophil migration Presence Avacopan of anti neutrophil ablates C5a induced cytoplasmic neutrophil auto antibodies (ANCA) Leads degranulation to complement system activation and C5a generation C5a via C5aR is main activator of neutrophils Avacopan ablates C5a induced reactive oxygen Neutrophils inflame and destroy blood vessels = vasculitis intermediate (ROI) production Avacopan ablates C5a induced CD11b upregulation (adhesion molecule induction) Neutrophils still respond to other physiologic signals Things That Avacopan Does Not Do It does not cause a build up of C5a in the body It does not cause to C5a to accumulate in the cell It does not lead to large change of the number of C5aR sites/cell It does not impede the ability of the cell to respond to other signals (mechanism is antiinflammatory, not immunosuppressive) It does not impede the beneficial effects of C5a binding to its second receptor C5L2 11
12 Broad Pipeline from Novel Discovery Platform THERAPEUTIC AREA DRUG/ INDICATION PRECLINICAL PHASE 1 PHASE 2 PHASE 3 TARGET Complement Inhibition in Orphan Diseases Avacopan (formerly CCX168)/ C5aR ANCA ASSOCIATED VASCULITIS C3 GLOMERULOPATHY HIDRADENITIS SUPPURATIVA Chronic and Orphan Kidney Diseases CCX140/CCR2 ORPHAN KIDNEY DISEASE FOCAL SEGMENTAL GLOMERULOSCLEROSIS DIABETIC KIDNEY DISEASE, SUCCESSFULLY COMPLETED PHASE II Other Inflammatory and Autoimmune Diseases CCX507/CCR9 CCR6 IBD: ULCERATIVE COLITIS TH17 DRIVEN DISEASE, e.g. PUSTUALAR PSORIASIS Immuno Oncology CCX872/ CCR2 ADVANCED PANCREATIC CANCER OTHER ONCOLOGY INDICATIONS 12
13 Anti Neutrophil Cytoplasmic Auto antibody (ANCA) Vasculitis: A Complement C5a Activated Neutrophil Driven Disease Overview Highly inflammatory and progressive autoimmune disease caused by the over activation of the complement system; generation of C5a Kidney is a major target organ Characterized by recurring flares, accruing into irreversible organ system damage (end stage renal disease) and death; relapse is common Significantly impacts multiple aspects of physical function, emotional wellbeing, and overall productivity Rationale for Avacopan Auto Antibodies Activation of Complement Cascade Generation of C5a C5a Binds C5a Receptor (C5aR) on Neutrophils C5aR Activated Neutrophils Destroy Blood Vessels Prevalence 40 75K ~4,000 new cases per year in U.S K ~5,000 new cases per year in EU 13
14 Anti Neutrophil Cytoplasmic Auto antibody (ANCA) Vasculitis: A Complement C5a Activated Neutrophil Driven Disease Overview Highly inflammatory and progressive autoimmune disease caused by the over activation of the complement system; generation of C5a Kidney is a major target organ Characterized by recurring flares, accruing into irreversible organ system damage (end stage renal disease) and death; relapse is common Significantly impacts multiple aspects of physical function, emotional wellbeing, and overall productivity Rationale for Avacopan Prevalence 40 75K ~4,000 new cases per year in U.S. Current Treatments K ~5,000 new cases per year in EU Auto Antibodies Activation of Complement Cascade Generation of C5a C5a Binds C5a Receptor (C5aR) on Neutrophils C5aR Activated Neutrophils Destroy Blood Vessels High dose steroids for 6 months Prednisone Methylprednisone Immuno suppressants Cyclophosphamide OR Rituximab Status Phase III ADVOCATE trial fully enrolled; Top line data expected in Q
15 Urgent Need to Modernize Standard of Care (SOC) in ANCA Vasculitis Current ANCA Vasculitis Chronic High Dose Steroid SOC Major Unmet Needs: Steroids for 6 Months Prednisone Methylprednisone or Cyclophosphamide Rituximab With Current SOC: 11 16% die within 1st year of diagnosis* Greatest risk to patients in first year comes from steroid induced infections Current treatments contribute ~60% of the mortality rate* Need to taper steroid = high relapse rate Irreversible organ damage with relapse (especially in the kidney) Rapid induction of remission Prevent damage and preserve renal function Durable remission Majority of patients at risk for relapse for many years Reduced side effects Prevent glucocorticoid related toxicity and infection * Little et al, 2010 Ann Rheum Dis 69: , Flossmann et al 2011 Ann Rheum Dis 70:
16 Need: Reduce Total Burden of Disease in ANCA Vasculitis Stop active vasculitis (as soon as possible) Eliminate current therapyinduced illness Stop accumulated organ damage Improve patient quality of life Reduction in vasculitis activity score (BVAS 1, a signs and symptoms index) From chronic steroids, From cyclophosphamide; rituximab From both vasculitis and from steroid therapy toxicity Validated quality of life (QOL) instruments Patient reported outcomes (PROs) 1 Birmingham Vasculitis Activity Score 16
17 Clinical Evidence: Avacopan Markedly Inhibits Complement Driven Activated Neutrophils Avacopan Efficacy Endpoints Reached in Phase II CLEAR Trial Primary endpoint achieved Reduction in vasculitis activity score (BVAS 1, a signs and symptoms index) at week 12 with avacopan Rapid onset of action with avacopan treatment Disease remission by BVAS week 4 Phase II CLEAR Trial Multi center, randomized (1:1:1), doubleblind Phase II trial in patients with newly diagnosed or relapsed ANCA Vasculitis A. SOC Control Group = Placebo + High Dose Steroid (+ CYC/RTX) B. Avacopan + Low Dose Chronic Steroid (1/3 of SOC) (+ CYC/RTX) C. Avacopan + No Chronic Steroid (+CYC/RTX) Marked improvements in renal parameters Significant lowering of proteinuria and shedding of kidney inflammation markers; decrease in egfr 2 Improved Patient Quality of Life Statistically significant enhancement in quality of life scores and patient reported outcomes; favorable safety profile ANCA Vasculitis Regulatory Designations for Avacopan: o FDA Grant and orphan drug designation o EMA PRIME designation and orphan medicinal product designation o MHLW designated ANCA an intractable disease o SwissMedic orphan drug designation 1 Birmingham Vasculitis Activity Score 2 Estimated Glomerular Filtration Rate 17
18 Avacopan: Landmark Phase III ADVOCATE Pivotal Trial Fully enrolled, top line data expected in Q Goals: Two primary endpoints: (both analyzed after 52 weeks) Remission (based on BVAS) at 26 Weeks Sustained remission (based on BVAS) at 52 Weeks Rationale Behind ADVOCATE Design 1. Demonstrate effective, rapid and sustained remission of ANCA vasculitis by BVAS 2. Eliminate need for chronic corticosteroids in standard of care and associated toxic side effects Test Group (N = 158) Control SOC Group (N = 158) 52 week treatment period Avacopan, 30 mg twice daily RTX, 4 weeks or CYC, 12 weeks followed by AZA Placebo avacopan twice daily Placebo to Prednisone Prednisone, 60 mg/day with standard tapering to 0 over 21 weeks + BVAS Remission Score Avacopan Treatment (Test Group) Control SOC Group 26 Weeks: Numerical Superiority / Non Inferiority High rates of relapse over time associated with SOC 1 52 Weeks: Sustained Remission Difference in Relapsed Patients 3. Beneficial effects across the total burden of disease RTX, 4 weeks or CYC, 12 weeks followed by AZA 4 Weeks 26 Weeks 52 Weeks Time 18 1 Potentially leading to greater separation of BVAS between SOC and Avacopan groups
19 Path to Approval in ANCA Vasculitis BVAS Remission = BVAS score of zero and off steroids 4 weeks prior Comparing avacopan with no chronic high dose steroid vs. steroid containing standard of care (SOC) Statistical null hypotheses will be tested in a hierarchical manner o Non inferiority (previous regulatory standard), then superiority 19 Two Primary Endpoints (not Co Primaries) Evaluated at Weeks 26 and 52 Key Secondary Endpoints Reduce the Total Burden of Disease Change in health related Quality of Life Biological response measures: o Glucocorticoid induced toxicity o Estimated glomerular filtration rate (egfr) o Urinary albumin:creatinine ratio (UACR) o Urinary MCP 1:creatinine ratio o Change in Vasculitis Damage Index (VDI) Incidence of withdrawals due to adverse event Primary BVAS Endpoint Achieved at Week 12 in Avacopan Phase II CLEAR Trial Treatment Groups N BVAS 1 Stat Sig 2 Total Avacopan Patients 36/43 84% P = Avacopan + low dose steroid 19/22 86% P = Avacopan + no steroid 17/21 81% P = 0.01 High dose steroid SOC 14/20 70% N/A 1 BVAS (Birmingham Vasculitis Activity Score) response defined 50% from baseline, and no worsening in any body system; 2 P values refer to comparison of avacopan to SOC for non inferiority General health perception Vitality Mental health Patient Reported Outcomes Statistically Significant at Week 12 in Avacopan Phase II CLEAR Trial Physical Functioning Bodily pain Role Physical Role emotional Social functioning High dose steroid 1 group 1 Prednisone / Methylprednisone General health perception Vitality Physical Functioning * * Mental * Social health Bodily pain functioning * All avacopan Role Physical * Role emotional * *Significant improvement over 12 week dosing course General Physical health Functioning perception Vitality Mental health Role Physical Role emotional Social functioning Bodily pain Avacopan + no steroid vs. general population controls 2 2 Basu et controls al, 2014 Ann 2 Rheum Dis 73:
20 Avacopan: Rapid Improvement in Lab, Renal Parameters in CLEAR Phase II Provide Confidence in Totality of Data **** P < , *** P < 0.001, ** P < 0.01, * P < 0.05 for superiority of avacopan vs. steroid control group; LSM change from baseline Neutrophil counts normalize within days *** P < 0.001, ** P < 0.01, * P < 0.05 for superiority of avacopan vs. steroid control group; LSM change from baseline Proteinuria improves faster and at greater magnitude in avacopan groups vs. control ** P < 0.01, * P < 0.05 for avacopan vs. control No requirement for chronic high dose steroids to stabilize kidney function Jayne et al, 2017 JASN doi: 20
21 Avacopan for C3 Glomerulopathy (C3G) Overview Uncontrolled activation of the complement system leading to complement protein deposition in the kidney (glomeruli), disrupting kidney function Can be life threatening; half of all persons with C3G have kidney failure Kidney transplant does not cure the disease; relapsing disease is common Primarily affects the young; huge economic burden on health care systems Rationale Prevalence 4K new cases per year in U.S. Current Treatments 4K ~ new cases per year in EU Characterized by C3 but also C5 / C5a deposition in glomeruli C5a contributes to the inflammatory hypercellularity in the glomeruli, leading to kidney damage, a main feature of C3G Avacopan targets C5aR, which blocks the effects of C5a No Approved Therapies Non specific treatment approaches include blood pressure control and broad immunosuppression FDA and EMA Orphan Drug Designations Status Potentially registration supporting clinical trial underway 21
22 Avacopan: Clinical Development in C3G Screening (Biopsyproven C3G) Baseline and Randomize Assess patient C5b 9 levels Stratum 1 (1:1): C5b 9 level >244 ng/ml Stratum 2 (1:1): C5b 9 level 244 ng/ml Placebo BID (n=22) Placebo BID (n=22) Avacopan, 30 mg BID (n=22) Avacopan, 30 mg BID Avacopan, 30 mg BID (n=22) Avacopan, 30 mg BID 0 6* 12 Renal Biopsy Assessments (months) Study Design Randomized (1:1), double blind, placebo controlled clinical trial in patients with a confirmed diagnosis of C3G Two treatment stratums: (C5b 9 level >244 ng/ml and C5b 9 level 244 ng/ml) Study well underway approximately 40% enrolled Primary Endpoint: *Percent change in C3G Histologic Index (CHI) at 6 months Secondary Endpoints: Urinary protein:creatinine, MCP 1:creatinine, QOL, and serum creatinine measurements (for egfr) over the course of the study 22
23 Avacopan for Hidradenitis Suppurativa (HS) Overview Chronic disabling skin autoimmune disease that relentlessly progresses, frequently causing keloids, contractures, and immobility Extremely painful inflammatory nodules, boils or abscesses; Most common in the armpit, groin, and genital regions More common in females, patients years old, and African American and biracial patients Prevalence (Moderate to Severe HS) 200K 200K Rationale Neutrophil driven disease where C5a involvement is validated C5a blockade with avacopan via C5aR offers a strong potential to control neutrophil activation Oral application offers advantages over injections or infusions Current Treatments Adalimumab is the only approved drug for HS; widely regarded as only having moderate efficacy Still, sales in adalimumab in HS last year >$1B Precedent for FDA Orphan Drug Designation for moderate or severe HS Status Potentially registration enabling Phase IIb trial initiated in Q
24 Avacopan for HS: AURORA Trial Underway Screening (HS Hurley Stage II III) Baseline and Randomize Placebo (n~130) Avacopan, 30 mg BID (n~130) Avacopan, 10 mg BID (n~130) Avacopan, 30 mg BID HiSCR (weeks) Study Design Randomized (1:1:1), double blind, placebo controlled clinical trial in patients with moderate to severe HS Placebo crosses over to active at 12 weeks, follow all groups for additional 24 weeks >145 clinical sites Primary Endpoint: Proportion on patients with clinical response (HiSCR) at 12 weeks Secondary Endpoints: Percent improvement baseline to week 12 between groups Validated secondary measurements in HS HS Quality of Life Instrument (HiSQOL) and SF36 24
25 Three Successive Major Opportunities Planned for Avacopan Starting Q ANCA Vasculitis Potentially Revolutionizing Treatment Paradigm ADVOCATE Phase III Pivotal Trial Top Line Data Expected Q C3G No Approved Current Treatment Options Large Controlled Phase IIb Trial Potentially to Support Registration 3 Hidradenitis Suppurativa Mechanism Based Effective New Therapy Large Controlled Phase IIb AURORA Trial Potentially to Support Registration 25
26 The Avacopan Advantage Over Other Approaches Advantage in ANCA Vasculitis and HS Avacopan significantly ahead in clinical development Avacopan completely inhibits C5aR Convenient orally administered capsule vs weekly antibody infusion For antibody therapy impossible to assess how much antibody required for full C5a inhibition Chimeric (mouse) antibody could be immunogenic (leading to loss of efficacy) Avacopan leaves beneficial C5L2 unimpeded, unlike approaches that take C5a away from C5L2 Advantage in C3G Avacopan more precise mode of action for C3G Avacopan C3G trial controlled, blinded, large (for a rare disease); histology primary endpoint 26
27 CCR2 Inhibitor CCX140 Targeted Approach to Chronic and Orphan Kidney Diseases 27
28 CCX140 for Focal Segmental Glomerulosclerosis (FSGS) Overview Orphan disease of the kidney s filtering units (glomeruli), and is characterized by serious scarring that leads to permanent kidney damage Presents with proteinuria, in which protein is found in the urine due to a breakdown of the normal filtration mechanism in the kidney One of the causes of a serious condition known as Nephrotic Syndrome and often leads to End Stage Renal Disease (ESRD) Rationale Histologic lesion from glomerular injury affecting specialized kidney filtering cells, especially podocytes CCR2 in FSGS kidney role for CCR2 in renal cell (podocyte) protection CCX140 has demonstrated significant reduction in proteinuria in Phase II clinical trial in diabetic nephropathy patients Prevalence Number of FSGS cases are rising more than any other cause of Nephrotic Syndrome 60K ~2300 new cases per year in U.S. Current Treatments No Approved Therapies 60K Non specific treatment approaches include steroids to control proteinuria or immuno suppressants Two clinical trials underway; nephrotic syndrome potentially registration supporting 28
29 CCR2 Inhibitor CCX140: Clinical Development in FSGS LUMINA 2 Screening LUMINA 2: Nephrotic syndrome primary FSGS ( 3 gram/day baseline proteinuria) CCX140, Dose range from 5 mg QD to 15 mg BID (n=6 to 13) Rescue if no partial response; otherwise continue Expand to patients for pivotal part 2 1 LUMINA 1 Screening LUMINA 1: Sub nephrotic primary FSGS (at least 1 gram/day baseline proteinuria) Stratify by level of proteinuria and immunosuppressant treatment (yes/no) (Biopsy proven primary FSGS & assess proteinuria levels) 0 CCX140, 5 mg QD (n=10) CCX140, 10 mg BID (n=10) CCX140, 15 mg BID (n=10) Placebo (n=10) Highest safe dose ( 12 weeks open label) Study Assessments Reduction in Proteinuria from Baseline (weeks) LUMINA 2: Nephrotic Syndrome Primary FSGS 3 gram/day baseline proteinuria First presentation or new flare following prior effective treatment *Significant decrease in proteinuria from baseline approvable endpoint LUMINA 1: Sub Nephrotic Primary FSGS At least 1 gram/day baseline proteinuria Decrease in proteinuria from baseline anticipated accelerated approval endpoint Reduced in decline in egfr anticipated full approval endpoint 1 Pending favorable results 29
30 The CCX140 Advantage in FSGS CCX140 highlights Small molecule CCX140 selectively inhibits CCR2 Study found presence of CCR2 on renal progenitor cells that are destined to become podocytes CCX140 shown to increase podocyte density in two FSGS models CCX140 well tolerated in one year Phase II diabetic kidney disease trial; durably lowered proteinuria No evidence of edema, fluid retention in humans or pharmacologic models Direct comparison with sparsentan in in vivo models show substantial and rapid proteinuria reduction with CCX140 and ET1/ARB CCX140 groups did not show such side hemodilution side effects 30
31 Summary: CCXI Orphan Disease Assets Avacopan (C5aR Inhibitor): New mode of action potential paradigm shift for treatment in complement driven autoimmune destruction Clinical benefit observed in ANCA vasculitis Phase II trials Pivotal Phase III ADVOCATE trial in ANCA fully enrolled top line data expected Q Trials for additional indications C3G and HS; readouts expected after ADVOCATE CCR2 Inhibitor CCX140: Extensive clinical experience, including a successful 1yr Phase II trial New science supports role of CCR2 in FSGS and in podocyte biology Initiated FSGS clinical studies (LUMINA trials) 31
32 Global Commercial Plan CCXI retains all rights to avacopan and CCX140 in the US The Vifor agreements ROW commercialization CCXI ROW Vifor Pharma Territory
33 Potential Multi Billion Commercial Opportunity (US) CCXI owns 100% of U.S. commercial rights; Non US Royalties teens to mid 20s on avacopan and CCX140 net sales 6,000 Potential Addressable Patients U.S. ~200,000 HS ~40,000 ANCA Vasculitis (top line data Q4, 2019) ~60,000 FSGS ~4,000 C3G Gross Sales ($MM) 5,000 4,000 3,000 2,000 1,000 $200K/Pt/Year $50K/Pt/Year 0 5,000 10,000 15,000 20,000 25,000 Patients >300K Potential Addressable Patients in the U.S. 1 Illustrative Example of Potential Sales at Orphan Drug Pricing 1 Based on estimated prevalence numbers 33
34 History of Capital Efficiency and Current Strong Financial Position Financial Highlights End of Quarter Cash and Investments $186M cash and investments 1 o Not including $35M potential capital under credit facility History of non dilutive funding from successful partnering strategy / funding (while retaining 100% US rights) $85M in May 2016 avacopan Presence of anti neutrophil cytoplasmic auto antibodies (ANCA) Leads to complement system activation and C5a generation $50M C5a via in C5aR December is main activator 2016 CCX140 of neutrophils Neutrophils inflame and destroy blood vessels = vasculitis $20M in February 2017 avacopan Asia (excl.china) $50M in January 2018 avacopan CMA validation $21.5M in June 2018 China rights $ (MM) * * *Proforma gives effect to cash commitments and milestones earned but not received until subsequent periods. * * * 1 As of September 30,
35 Taking CCXI to the Next Level Significant Multiple Growth Drivers Recent Achievements Phase III ADVOCATE trial in ANCA vasculitis (AAV) fully enrolled Initiated avacopan AURORA trial in Hidradenitis Suppurativa (HS) Building US commercial capability Strong cash reserves $186M Q cash and investments; >$90MM cash receipts in 2018 Anticipated 2019 Deliverables o ADVOCATE top line results Q4 o Avacopan trial in C3G fully enrolled o Avacopan AURORA trial in HS fully enrolled o Following readout on other sponsor s Phase II study in HS o CCX140 LUMINA trials in FSGS fully enrolled Anticipated into 2020 and 2021 o File avacopan NDA and MAA for AAV o Commercial launch of avacopan in US, EU and Japan o Key data readouts o Avacopan C3G and HS o CCX140 FSGS o Additional milestone payments from Vifor Pharma o Further expansion of pipeline into additional indications 35
36 January 2019 Thank You
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