Ardelyx, Inc. · NASDAQ:ARDX
Ardelyx — Investor Presentation (August 2014)
37 slides from Ardelyx's investor presentation, Aug 6, 2014
Slide 1: Investor Presentation, August 2014
Exhibit 99.1 Exhibit Investor Mike AUGUSTRaab 99.1 Presentation 2014 CEO
Slide 2: Special Note Regarding Forward-Looking Statements
Forward Special To made from substantial statements. upon any 2Ardelyx’s Commission. the forward-looking our Sanofi pursuant extent Note Looking collaboration business risks Such for Regarding that tothe and risks the statements Statements in discovery uncertainties statements. safe general, and par
Slide 3: Clinical-Stage Biopharmaceutical Company with Drug Discovery Platform
The Power Clinical-Stage Oral, Rapid, Large, Populations META- CARDIO BOLIC Phase Multiple Figure CKD IBS-C 3ESRD-Pi Small 2Global 1Efficient GI Programs RENAL from of Molecule, and Ardelyx Patient Sato Biopharmaceutical Drug Markets for T and Tenapanor Discovery Non-Systemic, Cleversand H., Company
Slide 4: The Result: Robust Product Pipeline
The Result: PHASE PROGRAM Tenapanor 1H:2015 ESRD-Pi (NHE3 IBS-C Phase Validated RDX002 2H:2015 CKD (NaP2b RDX009 (TGR5 RDX013 (K+ Hyper- RDX020 (Cl- 4Modulator) Channel 2b 2a Agonist) Inhibitor) Inhibitor) 2Data Wholly-Owned ESRD-Pi IBD Fluid Phase kalemia Overload Robust INDICATION Research Researc
Slide 5: A History of Efficient Development
A History Idea TENAPANOR DEVELOPMENT NHE3 Company Formed 2007 $26M FINANCINGS NEA Amgen LICENSES Upfront Milestone 5Up NaP2b toto& 2008 $870M $198M Series Lead P2a Ventures CMEA Inhibitor Development Sodium of Milestone 2009 Data $35M IND Rapid, Afor $30M NEA 2010 in Tenapanor Filed Proposed Transpo
Slide 6: Proven Management Team
Proven MIKE DOMINIQUE MARK DAVID ELIZABETH JEFF GEORGE ROB 6ANDYBLANKS JACOBS, RAAB Management SPENCER, KAUFMANN ROSENBAUM, JUEGRAMMER Chief CHARMOT, VP Sr. PhD Finance Dir. PhD Executive Team VP Chief Regulatory Sr. Chemistry PhD VP and Dir. PhD Financial VP Officer and Operations R&D Chief Drug Af
Slide 7: Investment Highlights
Investment 2PROPRIETARY WHOLLY-OWNED CAPITAL PROVEN Discovery Small Tenapanor: Phase NaP2b Multiple Pipeline Strong 7Deep VALIDATED Domain 2Molecule History Inhibition: in Provides Near-Term MANAGEMENT Three EFFICIENCY and AstraZeneca Highlights Expertise Design PROGRAMS Therapeutics PLATFORM Indica
Slide 8: The Intestine: Many Potential Drug Targets
Intestinal THE Brush Microvilli PROTEIN Membrane MANY 3,800 Translation 8Transporter INTESTINE Proteins Border POTENTIAL Epithelium ANALYSIS RegulatorIsTranscription DRUG Rich with TARGETS Potential Factor Enzyme Targets Regulator Catalytic Enzyme Receptor Antioxidant Structural Molecule Cellular Bi
Slide 9: Enhancing Ardelyx Drug Discovery
Enhancing Ardelyx Standard drug Fig Harvest from THE Discovery: Translation Cell Powerful Phenotypic 9Figure 1Physiology Gut BENEFITS 1Stem Primary from or Tool: Format Drug Rapid Screening to Transwell Cells Humans: Sato Mechanisms, Discovery Enterocyte Screening TGrow andSimulate Clevers inwith of
Slide 10: Tenapanor
210 Tenapanor PROPRIETARY Small Tenapanor: NaP2bMolecule Multiple VALIDATED and WHOLLY-OWNED CAPITAL PROVEN AstraZeneca Our Therapeutics PLATFORM Inhibition: Near-Term MANAGEMENT EFFICIENCYCollaboration PROGRAMS SanofiCollaboration; Catalysts PIPELINE Discovery with Phase Collaboration Strong TEAM A
Slide 11: The Impact of Sodium Overload in Declining Kidney Function
The Declining Sodium in Tissue Body Organ Progression Cardiovascular 11 CKD Impact Sodium Damage Stores Overload &Kidney ESRD of of“Fill Overload Sodium CKD Risk Organ in Hyperphosphatemia Function Up” CKD and Damage and Vascular Coronary Increased &Phosphorus ESRDCalcification ArteryOverload Diseas
Slide 12: Tenapanor Reduces Sodium and Phosphorus Absorption in the Gut
WITHOUT Dietary Into Local Phosphorus Sodium Tenapanor 12 Circulation Activity Sodium/Phosphorus Reduces TENAPANOR infrom theSodium Gut Circulation WITH and Passes Phosphorus TENAPANOR Diverts Sodium/Phosphorus Absorption
Slide 13: Studies Support Sodium and Phosphorus Impact
Studies SODIUM1 ACE 2.5 N Proteinuria 12(g/Day) 0R 2.20 0.5 1.80 140 1.60 Systolic 135 Blood Pressure 1.00 (mmHg) 115 110 Regular 1. *P<0.05 13 .40 egular *= Slagman, ACE Fresenius <3 Serum 1.40 40,538 Inhibition 130 3-4 Support *†‡ Sodium Blood *† v125 120 Inhibition-ARB ACE *† Phosphorus 4-5 Relat
Slide 14: Beneficial Effects of Tenapanor in CKD Models
214 Beneficial SODIUM Urinary 5/6th †2.5 200 150 SBP†ACE* Albumin/ (mmHG) 100 0N 50 + *ACE ** (mg/g Creatinine (mg/mg) 10.5 Normal Systolic 55/6th 0ormal ** Nephrectomized p<0.01; †*** *** Albumin/Creatinine †Enalapril PHOSPHORUS tissue) Effects Blood 0** 1.5 1*** †=†Vehicle 5/6th of Tenapanor 15Pre
Slide 15: Significant Global Market Opportunity for Tenapanor
Significant #U.S. 2011 ESRD: Hyperphosphatemia Sevelamer: Late (with IBS-C Sources: European Hepatol. 15of Patients Stage EUROPE 2015 Type 4.4M $1.5B 2012, Technavio ERA-EDTA CKD 2Market $1B 6.6M #Diabetes) $2.3B of Aliment JAPAN in (3b-4) Patients 3.4M 2013 Opportunity Insights: 270K Registry (1.8M
Slide 16: Extensive Clinical Experience with Tenapanor
Extensive 12 ESRD Phase 16 Clinical 1CKD 2 7 Phase 5 Clinical Clinical Trials IBS-C 2ainTrials Experience &1,200+ 2b in Clinical Healthy Subjects withTrials Tenapanor Adults; in Patients; n=347 in Multiple n=935 Indications
Slide 17: Tenapanor: Single Non-Systemic Drug, Generally Well-Tolerated
Tenapanor 830+ 291 ~415 ~125 3Single Non-Systemic: Most No 17Months Drug-Related Healthy AEs IBS-C CKD Subjects Dose Up Due Generally and Subjects Up Volunteers toExposed to >99.3% ESRD-HD 100 toSerious Exaggerated 900 mg/Day Well-Tolerated mg of toAdverse All Drug Subjects Tested Pharmacology Event
Slide 18: Limitations of Phosphate Binders in ESRD
LIMITATIONS Pill ESRD Prescribed Non-Compliance Safety Long-Term Gastrointestinal PILL BREAKFAST Calcium-Based Ca 1-2.5 Renvela Sevelamer/Renagel/ BINDERS Common Lanthanum/Fosrenol Meal 0.5-1.5 Elemental Each Tenapanor Daily Targeted £45mg TENAPANOR (30mg for to Not 18 Right) Acetate/PhosLo Illustra
Slide 19: Comparative Human Response: Sevelamer vs. Tenapanor
119010 Comparative SEVELAMER1 Fecal (mmol/Day Mean 40 30 20 010 Baseline (5g Dose Similar But40 30 20 Group) 1/500th 15,000 Phosphorus ± Results, SE) Burke 1g (mmol/Day TID the Mean mg/Day Human etDose TENAPANOR2 2.5g al. Fecal ± 1997; SE) Response: 30 TID Phosphorus mg/Day 5g 2 Study TID Sevelamer
Slide 20: Phase 1 and 2a Data: Tenapanor Diverts Sodium to Feces
1N=8 Tenapanor PHASE Mean VOLUNTEERS1 -P 10 20 30 40 50 (mmol) Sodium Fecal N=21 lacebo ESRD 24hr N=12 N=830mg 1 RDX5791-102; IN Diverts N=12 HEALTHY QD 15mg PATIENTS2 BID 2a Dietary ESRD-FLUID Dosing 2BID ADULT Study Sodium IN D5611C00001 to the SUBSET OFFeces in Healthy Adults and ESRD Patients
Slide 21: ESRD Fluid and Hyperphosphatemia Clinical Trials
ESRD FLUID PHASE HYPERPHOSPHATEMIA ESRD- Design: Objective: Status: 21 Clinical Completed Enrolling; Double-Blind; Double 2a Efficacy 2b Safety, Trials Blind; Data Tolerability, and4Expected Safety Weeksand ofOf of1H Tenapanor Treatment, Pharmacodynamics Treatment, 2015 for n=88 n=150 the Treatment
Slide 22: Limitations of Current Treatments to Delay CKD Progression
Tenapanor Sodium LIMITATIONS Poor Diuretics ACE 22 Compliance Inhibitors and Lose for Fluid Treating Efficacy Reduce OF with Overload CURRENT Low CKD Blood and Sodium Cause Pressure TREATMENTS Electrolyte Diets but Hyperkalemia Disorders TO DELAYLimits CKD Widespread PROGRESSION Use in CKD Patients
Slide 23: Phase 2a CKD Clinical Trial
PHASE CKD Design: Objective: Status: 23 Clinical Enrolling, Double-Blind, 2a Safety, Trials Data Tolerability, 12 Expected Weeks and 2H ofPharmacodynamics Treatment, 2015 n=140 (70 of Tenapanor Active/70 Placebo) in CKD Patients with Type 2 Diabetes Mellitus and Albuminuria
Slide 24: Limitations of Current IBS-C Therapies
Tenapanor GI LIMITATIONS OTC –Amitiza® Medical 24 Achieve Side Disorder: Drugs Effects Need and Endpoint for Inexpensive Constipation (e.g., IBS-C for Linzess® OFImproved CURRENT Diarrhea) in Only Fall but and Therapies 7% Not Short: Abdominal TREATMENTS to Very 20%Effective with of Pain Patients Be
Slide 25: Phase 2a IBS-C Data
*25 Phase DUAL End 40% 0% 10% Weekof Placebo 12a Although QOL These Treatment ENDPOINT: 100mg Responders Percent 30% Demonstrates (n=47) Measurements Data Week p<0.05 theWere 20% QD the(n=46) Primary 2 Week >30% versus Consistent Basis DECREASE 3Endpoint, placebo Week for 4Our Effects Follow-Up Chan
Slide 26: IBS-C Clinical Trials
PHASEClinical IBS-C Design: Objective: Status: 26 Completed Enrollment Double-Blind, 2a Efficacy 2b Safety, Trials Tolerability, Completed, and12 4Safety Weeks Weeks and of Data ofof Tenapanor Treatment, Pharmacodynamics Treatment, Expectedfor n=186 4Q n=371 the 2014 Treatment (139 (3:1 of Tenapanor
Slide 27: AstraZeneca/Tenapanor Collaboration Terms
Theto Upfront Additional PAYMENTS Up Milestones $50M Total $20M $25M $15M Co-Funding Royalties OTHER Responsibilities: AstraZeneca Ardelyx *Exercisable 27 AstraZeneca/Tenapanor 2HDevelopment 1H Received Rights: TERMS Incremental, with 2015 $35M Option Within $20M, May December Right All $237.5M at60
Slide 28: RDX002: NaP2b Inhibition
RDX002: 2PROPRIETARY WHOLLY-OWNED CAPITAL PROVEN Discovery Tenapanor: RDX002 Multiple Pipeline 28VALIDATED Provides Near-Term NaP2b NaP2b MANAGEMENT EFFICIENCY and AstraZeneca Design PROGRAMS Inhibition: PLATFORM and Additional Catalysts PIPELINE Our ofCollaboration; Non-Systemic, Collaboration Sano
Slide 29: NaP2b Phosphate Transport Inhibitor vs. Binders
Binder Phosphate Phosphate Increased GI-Upset/Pill-Burden Capillary Enterocyte Transporter NaP2b Inhibitor Transport Concerns Potential 29 for of Calcium Binder Metal Dramatically Use inTransport vs. Phosphate Accumulation Load Combination Phosphate with Reduced Inhibitor Phosphate Calcium Transport
Slide 30: RDX002: Preclinical Data on Urine Phosphorus
RDX002: 0R -30 -20 (%) Vehicle 500 Combination Sevelamer NTX1942 Data 30eduction ** mg/kg Shown *** Sevelamer NTX1942 Is Response -10 and 50 *** is Additive ofmg/kg the Mean NTX1942 Is Reduces Superior ± SEM; Combo Urine to**Sevelamer Phosphorus = p<0.01;at*** 1/10 Levels =p<0.001, of in theNormal D
Slide 31: Sanofi/NaP2b Collaboration Terms
Theto Licensed Royalties Milestones Other Upfront PAYMENTS Up Incremental, $1.25M Total $198M NaP2b Complete Option Commercialize Sanofi and Commercialization Ardelyx 31 if Sanofi/NaP2b Terms ItResponsible Patent toExercises Potential Has Technology Obtain Activities Tiered the Portfolio NaP2b Right
Slide 32: Ardelyx-Owned Pipeline
Ardelyx-Owned 2PROPRIETARY WHOLLY-OWNED CAPITAL PROVEN Discovery Tenapanor: NaP2b Multiple Pipeline 32VALIDATED Inhibition: Provides Near-Term MANAGEMENT EFFICIENCY and AstraZeneca Design PROGRAMS Pipeline PLATFORM Additional Sanofi Catalysts PIPELINE ofCollaboration; Non-Systemic, Collaboration TEA
Slide 33: RDX009: TGR5 Agonist
RDX009: TGR5 Enhances Anti-Inflammation Gattex® –Intercept ARDELYX No Long Significantly 33Daily Gallbladder Short Gallbladder Lasting STIMULATION Lasting Injections =and GLP1 TGR5 GLP2 TGR5 Improves Exelixis/BMS Incretin Emptying Incretin Issues and Agonists approved AGONISTS and GLP2 Secretion Muc
Slide 34: RDX013: Potassium Secretagogue for Hyperkalemia
RDX013: THE All Therapeutic The ARDELYX To Used Augment Maintain 34 Move Potassium Limiting CHALLENGE as aNormal Potassium Patient Stand Hyperkalemia POTASSIUM Dose Factor Binders Alone Compliance Serum forinto inKayexelate, or (e.g. Efficacy Potassium Colon (Elevated in“SECRETAGOGUE” Patiromer) Com
Slide 35: Near-Term Data Catalysts to Drive Value
Near-Term IBS-C Tenapanor DATA Data Q4 2014 35 1HData 2015 Phase ESRD-Pi 2H Data Catalysts 2b Phase CKDto2b Drive Phase Value 2a
Slide 36: Financial Overview
$Financial $33.2M Cash $61M Operating Debt Capital Series Ventures Nasdaq: 2008 *Excludes 36MILLIONS $0 2011 and A received Raised (as $26M ARDX Cash Overview Expenses* amounts of 3/31/14) Equivalents Series fromreimbursed IPO $12.5M B $30M in $25M July (12 IPO by 2014 Months Additional AZN $69MorEn
Slide 37: Summary
PROPRIETARY 2Summary WHOLLY-OWNED CAPITAL PROVEN Discovery Tenapanor: NaP2b Multiple Pipeline 37VALIDATED Inhibition: Provides Near-Term MANAGEMENT EFFICIENCY and AstraZeneca Design PROGRAMS PLATFORM Additional Sanofi Catalysts PIPELINE ofCollaboration; Non-Systemic, Collaboration TEAM Opportunity P