Full Press Release Details
ARDELYX JPMorgan 2017
Annual Healthcare Conference
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 Securities Reform Act of 1995, including the potential for Ardelyx s product candidates in treating the diseases and conditions for
which they are being developed; Ardelyx s future development plans for its product candidates and the expected timing thereof; Ardelyx s expected timing for the receipt of results from its clinical trials evaluating its product candidates;
Ardelyx s 2021 goals; and the potential of Ardelyx s drug discovery and design platform. Such forward-looking statements involve substantial risks and uncertainties that could cause the development of Ardelyx s product candidates or
Ardelyx s future results, performance or achievements to differ significantly from those expressed or implied by the forward-looking statements.
and uncertainties include, among others, the uncertainties inherent in research and the clinical development process and the uncertainties in the manufacture of clinical trial material, including process development, and scale up of manufacturing
processes. Ardelyx undertakes no obligation to update or revise any forward-looking statements. For a further description of the risks and uncertainties that could cause actual results to differ from those expressed in these forward-looking
statements, as well as risks relating to Ardelyx s business in general, please refer to Ardelyx s Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission on November 7,
2016, and its subsequent current and periodic reports filed and to be filed with the Securities and Exchange Commission.
DEDICATED TO A BOLD MISSION AND VISION
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 is to dramatically enhance the way patients with gastrointestinal and cardiorenal diseases are treated by delivering medicines that matter.
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
By developing therapies that work via the gut, we can target a vast range of diseases, beginning with gastrointestinal (GI) and cardiorenal diseases.
BREAKTHROUGH SCIENCE FOR BETTER HEALTH
platform for rapid discovery and design of new drugs
small molecule medicines
of targeted treatments
cardiorenal and GI experience among management team
2017: GAME-CHANGING YEAR SETTING UP FOR COMMERCIALIZATION
5 DATA READOUTS DRIVING LONG-TERM STRATEGIC PLANS & VALUE
Phase 3 data for tenapanor for hyperphosphatemia
Start 2nd Phase 3 study for hyperphosphatemia
Results from onset-of-action study with RDX7675 in hyperkalemia
T3MPO-1 Phase 3 data for tenapanor in
T3MPO-2 Phase 3 data for tenapanor in
Complete T3MPO-3 open-label safety study in
ARDELYX JPMorgan 2017
Annual Healthcare Conference
ADVANCING A DIVERSE CARDIORENAL PORTFOLIO
PROGRAM INDICATION RESEARCH PHASE 1 PHASE 2 STAT PHASE 3
(NHE3 Inhibitor) Hyperphosphatemia
RDX7675 Hyperkalemia
(NHE3 Inhibitor) Indications
(Potassium Secretagogue)
More than 2,000 individuals treated with tenapanor across 18 clinical
TENAPANOR: HIGHLY DIFFERENTIATED MECHANISM OF ACTION
NHE3 imports sodium (Na+) in exchange for protons (H+)
Tenapanor blocks NHE3, increasing protons inside cells
Increased protons selectively tighten
junctions between cells, reducing phosphate absorption
Clinical and preclinical data suggest no effect on macromolecule absorption pathway
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 patients are non-compliant with current treatment4
NO TREATMENTS available that are not phosphate binders4
1. USRDS 2014; European ERA-EDTA Registry Annual report 2012; Nakai S, et al, 2008 2. Decision Resources 2016 3. Ficociello 2016 4. Fissell
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
Notable safety with low rate of discontinuations due to AEs (fixed doses in Phase 3
TOTAL DISCONTINUATIONS DUE TO AE/GROUP (2) 3 3
SERUM CALCIUM DECREASE 0 0
HYPERPHOSPHATEMIA 0 0
(1) Diarrhea is used for all similar terminology
(2) Multiple AEs possible per discontinuation
HYPERPHOSPHATEMIA PHASE 3 DATA EXPECTED IN 1Q17
TENAPANOR TITRATION 30, 20, 15, 10, 3 MG BID
8 WEEK TREATMENT PERIOD
TENAPANOR (SAME DOSE)
Double-blind, placebo-controlled, randomized withdrawal (RW)
Primary Endpoint: Difference in change between tenapanor and placebo arm from treatment end to RW end in responder population
Secondary Endpoints: Change in serum phosphorus levels from baseline to end of 8 week treatment and effect of different dosing regimens for patients reaching serum phosphorus goal
levels of < 5.5 mg/dl during 8 weeks of treatment
Down titration scheme used to optimize phosphate lowering and GI tolerability, based on experience in previous
SECOND PHASE 3 STUDY EXPECTED TO BEGIN 1H 2017
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
and successful internal cardiorenal commercial expertise
Addressable market with specialty sales force; synergistic opportunities within cardiorenal franchise
Ex-U.S. access through strategic partnerships
CALCIUM-BASED BINDERS: CA ACETATE/PHOSLO (1)
SEVELAMER / RENAGEL / RENVELA (1)
LANTHANUM / FOSRENOL (1)
FERRIC CITRATE / AURYXIA (1)
SUCROFERRIC OXYHYDROXIDE / VELPHORO (1)
TENAPANOR TARGETED DAILY DOSE (1)(2)(3)
(1) Not actual size; however, relative sizes are to scale
(2) Tenapanor pill color may change
(3) Tenapanor (10 mg twice daily used for illustration purposes)
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 people in U.S. with CKD and/or heart failure have HK1
DOSE REDUCTION with RAAS inhibitors remains standard and problematic part of treatment management among physicians2
HK SIDE EFFECT common with high blood pressure and some anti-diabetic treatments3
PALATABILITY and sodium-ion leads to poor patient compliance4
CHRONIC MARKET emerging, but unaddressed by today s