New Ideas. Better Medicines. Third Quarter Financial Results Conference Call

Transcription

1 New Ideas. Better Medicines. Third Quarter 2018 Financial Results Conference Call

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 expressed or implied by these forward-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 the year 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-Q for the quarters 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 place undue reliance on these statements or regard these statements as a representation or warranty by us or any other person that we will achieve our 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 Key Takeaways: Springboard for the Future Rapid cadence of top-line avacopan data: o ADVOCATE in Q4, 2019 o C3G and Hidradenitis Suppurativa in 2020 Received FDA Orphan Drug Designation for CCX140 in FSGS o New mechanism of action data presented at ASN Strong financial position enables multiple clinical trials and planning for commercialization in U.S. 3

4 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 CYC; rituximab From both vasculitis and from steroid therapy toxicity Validated quality of life (QOL) instruments Patient reported outcomes (PROs) 1 Birmingham Vasculitis Activity Score 4

5 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 Potential registration-supporting clinical trial underway 5

6 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 Plan to initiate comprehensive placebo-controlled trial in Q

7 Three Successive Major Opportunities Planned for Avacopan Starting Q ANCA Vasculitis Potentially Revolutionizing Treatment Paradigm ADVOCATE Phase 3 Pivotal Trial Top Line Q C3G No Approved Current Treatment Options Large Controlled Phase 2b Trial Potentially to Support Registration 3 Hidradenitis Suppurativa Mechanism Based Effective New Therapy Large Controlled Phase 2b Trial Potentially to Support Registration 7

8 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 DN 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 8

9 Q Financial Results September 30, 2018 Cash, cash equivalents and investments - $186 Million Total shares outstanding 50.4 million Consolidated statement of operations (in thousands) - Revenue: Collaboration and license revenue Total revenue Three Months Ended September 30, (unaudited) $ 8,975 $ 9,029 8,975 9,029 9 Operating expenses: Research and development General and administrative Total operating expenses Loss from operations 15,135 12,315 5,373 3,624 20,508 15,939 (11,533) (6,910) Total other income, net Net loss $ (10,890) $ (6,560)

10 Key Takeaways: Springboard for the Future Rapid cadence of top-line avacopan data: o ADVOCATE in Q4, 2019 o C3G and Hidradenitis Suppurativa in 2020 Received FDA Orphan Drug Designation for CCX140 in FSGS o New mechanism of action data presented at ASN Strong financial position enables multiple clinical trials and planning for commercialization in U.S. 10