Ardelyx, Inc. · NASDAQ:ARDX
Ardelyx — JPMorgan 2017 Annual Healthcare Conference
40 slides from Ardelyx's conference presentation, Jan 9, 2017
Slide 1: Ardelyx JPMorgan 2017 Annual Healthcare Conference
THE INTERSECTION OF BREAKTHROUGH SCIENCE AND BETTER HEALTH ARDELYX JPMorgan 2017 Annual Healthcare Conference NASDAQ: ARDX
Slide 2: Forward-Looking Statements
FORWARD-LOOKING STATEMENTS To the extent that statements contained in this presentation are not descriptions of historical facts regarding Ardelyx, they are forward-looking statements reflecting the current beliefs and expectations of management made pursuant to the safe harbor of the Private Securi
Slide 3: Dedicated to a Bold Mission and Vision
DEDICATED TO A BOLD MISSION AND VISION OUR MISSION We are committed to bringing effective medicines with distinct safety and dosing advantages to underserved patients by using the gut as the gateway to better health. OUR VISION Our vision is to dramatically enhance the way patients with gastrointest
Slide 4: Using the Gut as the Gateway to Better Health
USING THE GUT AS THE GATEWAY TO BETTER HEALTH The gut is a critical system that plays a central role in many diseases by signaling to other organs how to respond to a variety of factors such as a meal, the microbiome or even pathogens. By developing therapies that work via the gut, we can target a v
Slide 5: Breakthrough Science for Better Health
BREAKTHROUGH SCIENCE FOR BETTER HEALTH PROPRIETARY platform for rapid discovery and design of new drugs Focus on GUT-RESTRICTED, small molecule medicines GROWING PIPELINE of targeted treatments 3 Phase 3 programs underway 100+ YEARS cardiorenal and GI experience among management team $ Financially s
Slide 6: 2017: Game-Changing Year
2017: GAME-CHANGING YEAR SETTING UP FOR COMMERCIALIZATION 5 DATA READOUTS DRIVING LONG-TERM STRATEGIC PLANS & VALUE CARDIORENAL Phase 3 data for tenapanor for hyperphosphatemia Start 2nd Phase 3 study for hyperphosphatemia Results from onset-of-action study with RDX7675 in hyperkalemia GASTROINTESTI
Slide 7: Enhancing Care of Patients with Cardiorenal Diseases
ENHANCING CARE OF PATIENTS WITH CARDIORENAL DISEASES ARDELYX JPMorgan 2017 Annual Healthcare Conference NASDAQ: ARDX
Slide 8: Advancing a Diverse Cardiorenal Portfolio
ADVANCING A DIVERSE CARDIORENAL PORTFOLIO PROGRAM INDICATION RESEARCH PHASE 1 PHASE 2 STAT PHASE 3 TENAPANOR ESRD (NHE3 Inhibitor) Hyperphosphatemia RDX7675 Hyperkalemia (Potassium Binder) RDX011 Cardiorenal (NHE3 Inhibitor) Indications RDX013 Hyperkalemia (Potassium Secretagogue) More than 2,000 in
Slide 9: Tenapanor: Highly Differentiated Mechanism of Action
TENAPANOR: HIGHLY DIFFERENTIATED MECHANISM OF ACTION Without Tenapanor GUT Sodium NHE3 Phosphate Protons Macromolecules Blood stream With Tenapanor GUT Sodium Tenapanor NHE3 Phosphate Protons Macromolecules Blood stream NHE3 imports sodium (Na+) in exchange for protons (H+) Tenapanor blocks NHE3, in
Slide 10: Hyperphosphatemia: Life-Threatening Condition for ESRD Patients on Dialysis
HYPERPHOSPHATEMIA: LIFE-THREATENING CONDITION FOR END-STAGE RENAL DISEASE PATIENTS ON DIALYSIS 735,000+ ESRD patients with hyperphosphatemia (HP) in major developed countries1 ~70% of U.S. dialysis patients taking phosphate binders to manage HP2 Up to15 pills per day with phosphate binders3 45% of p
Slide 11: Tenapanor Reduces Serum Phosphorus in Phase 2b
TENAPANOR REDUCES SERUM PHOSPHORUS IN PHASE 2B Statistically significant and clinically meaningful dose-related decrease in serum phosphorus levels in patients with hyperphosphatemia (fixed doses in Phase 3 trial) 0 -0.5 -1 -1.5 -2 -2.5 3MG BID N=21 -1.18 10MG BID N=23 -1.70 PLACEBO N=26 -0.54 Notab
Slide 12: Hyperphosphatemia Phase 3 Data Expected in 1Q17
HYPERPHOSPHATEMIA PHASE 3 DATA EXPECTED IN 1Q17 n = ~200 (1:1:1) RANDOMIZATION TENAPANOR 3 MG BID TENAPANOR TITRATION 30, 20, 15, 10, 3 MG BID TENAPANOR 10MG BID END OF TREATMENT 8 WEEK TREATMENT PERIOD TENAPANOR (SAME DOSE) PLACEBO END OF RW PERIOD 4 WEEK RW PERIOD Double-blind, placebo-controlled,
Slide 13: Tenapanor Targeted Benefits for HP Patients
TENAPANOR TARGETED BENEFITS FOR HP PATIENTS COMMERCIAL OPPORTUNITIES First-in-class, unique mechanism of action for dialysis patients Significantly reduced pill burden and mass allows for increased patient compliance Improvements in bowel frequency may provide important patient benefit Deep and succ
Slide 14: Hyperkalemia: Greatly Underserved Patient Population
HYPERKALEMIA: GREATLY UNDERSERVED PATIENT POPULATION People with heart failure, chronic kidney disease (CKD) and diabetes at greatest risk of developing hyperkalemia (HK) due to kidney’s weakened ability to excrete potassium resulting from these conditions and drugs prescribed as treatments ~2M peop
Slide 15: RDX7675 Designed to Provide Unique Product Advantages
RDX7675 DESIGNED TO PROVIDE UNIQUE PRODUCT ADVANTAGES Available Treatments Gritty texture Sodium counter-ion; leads to fluid overload and edema Spherical beads Calcium-sorbitol counter-ion RDX7675 Bead design enhances mouth feel and palatability Lack of added sodium or sorbitol aligned with best cli
Slide 16: Phase 2 Study of RDX7675 in Hyperkalemia Underway
PHASE 3 STUDY OF RDX7675 IN HYPERKALEMIA UNDERWAY PART A PART B Patients with Potassium level of ³ 3.8 - < 5.1 are randomized SCREENING POTASSIUM 5.5 TO < 6.5 RDX7675 TITRATION N = ~250 POTASSIUM 5.1 TO < 5.5 PRIMARY ENDPOINT 4 WEEK TREATMENT PERIOD RDX7675 (FIXED DOSE) PLACEBO OPEN LABEL EXTENSION
Slide 17: High-Value Improvements Over Today's Treatments
HIGH-VALUE IMPROVEMENTS OVER TODAY’S TREATMENTS COMMERCIAL OPPORTUNITIES Eliminates limitations of current and familiar treatment regimens Enhanced tolerability and palatability support patient adherence Low cost of goods relative to other treatment options Manageable distribution model with no need
Slide 18: Enhancing Care of Patients with Gastrointestinal Diseases
ENHANCING CARE OF PATIENTS WITH GASTROINTESTINAL DISEASES ARDELYX JPMorgan 2017 Annual Healthcare Conference N A S D A Q : A R D X
Slide 19: Accelerating Development of Novel GI Portfolio
ACCCELERATING DEVELOPMENT OF NOVEL GI PORTFOLIO PROGRAM INDICATION RESEARCH PHASE 1 PHASE 2 PHASE 3 TENAPANOR (NHE3 Inhibitor) IBS-C RDX8940 (TGR5 Agonist) GI Indications RDX011 (NHE3 Inhibitor) GI Indications RDX023 (FXR Agonist) GI Indications ARDELYX 19
Slide 20: IBS-C: Underserved and Highly Burdensome GI Disorder
IBS-C: UNDERSERVED AND HIGHLY BURDENSOME GI DISORDER ~11M people in the U.S. with IBS-C1 MAJOR REDUCTION IN HEALTH-RELATED QUALITY OF LIFE and work productivity2 ECONOMIC BURDEN to society, managed care and employers3 ADDED PATIENT COSTS due to physician office visits and outpatient services3 ~5 day
Slide 21: Statistically Significant Responder Rates in Tenapanor Phase 2b IBS-C Study
STATISTICALLY SIGNIFICANT RESPONDER RATES IN TENAPANOR PHASE 2B IBS-C STUDY Statistically and Clinically Meaningful CSBM Responder Rates & Combined Responder Rates PERCENT RESPONSE 70 60 50 40 30 20 10 0 P < 0.001 50.0% 26.4% 23.6% COMBINED RESPONDER(3) P < 0.001 60.7% 27.0% 33.7% CSBM RESPONDER(2)
Slide 22: Tenapanor Strong Safety Profile in IBS-C in Phase 2b
TENAPANOR STRONG SAFETY PROFILE IN IBS-C IN PHASE 2B 50mg twice daily (BID): mild to moderate AEs Tenapanor has little to no systemic availability >3000 plasma samples analysed (>99% BLQ) AEs observed due to exaggerated pharmacology AE Summary n (%) C Tenapanor 50 mg BID (n = 89) Placebo (n = 90) An
Slide 23: Tenapanor T3MPO Pivotal Trials Underway for IBS-C
TENAPANOR T3MPO PIVOTAL TRIALS UNDERWAY FOR IBS-C T3MPO-1: 12-Week Tenapanor IBS-C Phase 3 RANDOMIZATION TENAPANOR 50MG BID N = ~300/GROUP PLACEBO BID PRIMARY ENDPOINT 12 WEEK TREATMENT PERIOD TENAPANOR 50 MG BID PLACEBO BID TENAPANOR 50 MG BID END OF TREATMENT 4 WEEK RANDOMIZED WITHDRAWAL TREATMENT
Slide 24: Focused Commercial Effort Will Drive Tenapanor Value
FOCUSED COMMERCIAL EFFORT WILL DRIVE TENAPANOR VALUE Large patient population that takes time to diagnose and start treatment 60-70% of patients try self-treatment before seeing a physician1 Patients and physicians unsatisfied with current treatment options1 Patients PCPs OB/GYNs Ges 1 = not at all
Slide 25: Well-Positioned for Short- & Long-Term Growth
WELL-POSITIONED FOR SHORT- & LONG-TERM GROWTH ARDELYX JPMorgan 2017 Annual Healthcare Conference NASDAQ: ARDX
Slide 26: Unique Approach to Drug Discovery
UNIQUE APPROACH TO DRUG DISCOVERY Technology designed to emulate human gut; offers predictive ability for drug leads and mechanisms Integrated know-how and proprietary gut-restriction chemistry Development of small molecules with optimized pharmaceutical properties Broad application to evaluate new
Slide 27: Platform Supports Ardelyx Evolution and Growth
PLATFORM SUPPORTS ARDELYX EVOLUTION AND GROWTH Provides early insights into clinical viability Generates broad pipeline of clinical assets Broadly applies to diseases beyond GI and cardiorenal, enables partnership potential 2008-2009 Established leading discovery and design platform for gut-restrict
Slide 28: Financially Strong to Drive Toward Vision
FINANCIALLY STRONG TO DRIVE TOWARD VISION $ CASH AND SECURITIES OF ~$201M 12/31/16 $ PIPELINE INVESTMENT $ NO DEBT OPERATING RUNWAY THROUGH 2Q18 ARDELYX 28
Slide 29: Ardelyx Delivers for 2021
ARDELYX DELIVERS FOR 2021 DELIVER 2021 ARDELYX Independent, fully integrated, revenue-generating biotech company Profitable cardiorenal business unit; among top 2 cardiorenal companies in biotech Double-digit growth in GI business unit and on a path to blockbuster status; among top 3 GI companies in
Slide 30: Passionately Committed to Enhancing Care for People with GI and Cardiorenal Diseases
PASSIONATELY COMMITTED TO ENHANCING CARE FOR PEOPLE WITH GI AND CARDIORENAL DISEASES “We do it all for the patients. We’re committed to the vision of the company and delivering on the promise of bringing better treatments to the patients who need them.” – Chiamaka, Ardelyx quality assurance team “Wh
Slide 31: Back-Up Slides
BACK-UP SLIDES ARDELYX JPMorgan 2017 Annual Healthcare Conference NASDAQ: ARDX
Slide 32: Tenapanor IBS-C Phase 2b Clinical Trial: CSBM Response
TENAPANOR IBS-C PHASE 2B CLINICAL TRIAL Dose-Dependent CSBM Response Maintained for 12 Weeks MEAN AVERAGE WEEKLY CSBMs Placebo Tenapanor 5 mg BID Tenapanor 20 mg BID Tenapanor 50 mg BID 7 6 5 4 3 2 1 0 -2 -1 0 1 2 3 4 5 6 7 8 9 10 11 12 1 2 3 4 WEEK SCREENING TREATMENT FOLLOW UP ARDELYX CSBM: Comple
Slide 33: Tenapanor IBS-C Phase 2b: Responder Rates (6 of 12 weeks)
TENAPANOR IBS-C PHASE 2B CLINICAL TRIAL Responder Rates ( ³ 6 of 12 treatment weeks) PERCENT RESPONSE 70 60 50 40 30 20 10 0 P<0.001 50.0% 26.4% 23.6% COMBINED RESPONDER(3) P<0.001 60.7% 27.0% 33.7% CSBM RESPONDER(2) P<0.026 65.5% 17.2% 48.3% ABDOMINAL PAIN RESPONDER(1) Tenapanor 50 mg Placebo CSBM
Slide 34: Tenapanor IBS-C Phase 2b: Responder Rates (9 of 12 weeks)
TENAPANOR IBS-C PHASE 2B CLINICAL TRIAL Responder Rates ( ³ 9 of 12 treatment weeks) PERCENT RESPONSE 60 50 40 30 20 10 0 P = 0.01 20.2% 13.5% 6.7% COMBINED RESPONDER(3) P = 0.004 23.8% 15.9% 7.9% CSBM RESPONDER(2) P = 0.022 48.8% 17.3% 31.5% ABDOMINAL PAIN RESPONDER(1) (1) Decrease of 30% mean abdo
Slide 35: Tenapanor ESRD Phase 2b: Effects on FGF-23
TENAPANOR ESRD 1ST PHASE 2B CLINICAL TRIAL Effects of Tenapanor on FGF-23 (Exploratory Analysis) Versus Placebo Treatment Percent Decrease p-value 1 mg BID 28% 0.0366 3 mg BID 40% 0.0011 10 mg BID 38% 0.0017 30 mg BID 32% 0.0099 3 mg QD 27% 0.0426 30 mg QD 42% 0.0013 Placebo NA NA Note: Change from
Slide 36: Tenapanor ESRD Phase 2b: Phosphorus Effect Does Not Correlate with Diarrhea
TENAPANOR ESRD 1ST PHASE 2B CLINICAL TRIAL Phosphorus Effect Does Not Correlate with Diarrhea Serum-phosphorus (from baseline) 8 6 4 2 0 y = -0.005x - 1.6196 -2 -4 -6 -8 0 1 2 3 4 5 6 Diarrhea score 0 = No episodes of diarrhea 6 = Discontinued due to diarrhea 36
Slide 37: RDX7675 Preclinical Study
RDX7675 PRECLINICAL STUDY Preclinical Efficacy Demonstrated in Male CD1 Mice Fecal Potassium Excretion in Male CD1 Mice FECAL K (MG /24 H) 25 20 15 10 5 0 P < 0.0001 P < 0.0001 P = 0.045 P < 0.0001 P = 0.0009 ONE-WAY ANOVA FOLLOWED BY TUKEY TEST (COMPARES ALL POSSIBLE PAIRS OF MEANS) VEHICLE SPS CAL
Slide 38: RDX7675 Pharmacodynamic Clinical Trial
RDX7675 PHARMACODYNAMIC CLINICAL TRIAL Clinically Meaningful Potassium Binding RDX7675 demonstrated comparable results to those observed with SPS RDX7675 was generally well-tolerated at all doses evaluated up to 27.6 g/day No unexpected changes observed in other fecal electrolytes monitored, includi
Slide 39: Minimally-Systemic TGR5 Agonist RDX8940
MINIMALLY-SYSTEMIC TGR5 AGONIST RDX8940 Efficacious in a Model of Inflammatory Bowel Disease Minimally-systemic RDX8940 candidate demonstrates no gallbladder effects RDX8940 in combo with a DPPIVi significantly improves colon length and colon cytokine levels (IL-6 levels shown) in an Inflammatory Bo
Slide 40: Oral Potassium Secretagogue RDX013
ORAL POTASSIUM SECRETAGOGUE RDX013 PROOF OF CONCEPT IN PRECLINICAL MODELS FECAL K+ (MG) 25 20 15 10 5 0 P = 0.0003 ONE-WAY ANOVA FOLLOWED BY TUKEY TEST VEHICLE RDX013 (3 MG/KG) RDX013 (10 MG/KG) RDX013 (30 MG/KG) Goal: An oral potassium secretagogue that increases secretion by 10 to 20 mmol, shows e