Novel Mechanisms to Solve Significant Medical Challenges

For decades, the gut had been under-recognized as a viable site for new therapeutic points of intervention for treating disease. Using our discovery model, which recreates environments within certain areas of the gastrointestinal tract and kidney, Ardelyx® scientists have been able to elucidate new and previously unexploited ion transport mechanisms. Focusing on these mechanisms, we are creating targeted, small molecule drugs that are orally active but with limited systemic absorption, aiming to develop potent and efficacious therapies that minimize the side effects and drug-drug interactions frequently encountered with traditional, systemically absorbed medicines.

Our Pipeline

Based on our successes to date in translating scientific insights into targeted therapies, we are advancing a pipeline of promising, novel mechanism, first-in-class drug candidates that target specific pharmacologic pathways and have the potential to meet significant unmet medical needs.

Tenapanor for Hyperphosphatemia 

Tenapanor is a first-in-class, targeted, small molecule therapy in development for the treatment of hyperphosphatemia, a condition of elevated levels of phosphate in the blood, common among patients with chronic kidney disease on dialysis. Tenapanor has a unique mechanism of action and acts locally in the gut to inhibit the sodium/hydrogen exchanger 3 (NHE3). This results in a conformational change of the epithelial cell junctions, reducing permeability specific to phosphate, resulting in decreased phosphate absorption through the paracellular pathway, the primary pathway of dietary phosphate absorption. As a first-in-class, phosphate absorption inhibitor, tenapanor has the potential to completely change the hyperphosphatemia treatment paradigm.

RDX013 for Hyperkalemia

Our discovery platform has enabled the systematic study of the biological mechanisms of potassium secretion and has led us to the discovery of RDX013, an investigational potassium secretagogue for the treatment of hyperkalemia. Hyperkalemia is a common problem in patients with kidney and heart disease and can cause a significantly increased risk of death because of the potential for heart conductance issues. RDX013 pharmacologically targets potassium secretion through the lumen of the gut, thus lowering levels of serum potassium.

Unlike current treatments that bind potassium, our lead candidate RDX013 represents a novel mechanism, targeted therapy that could create a completely new treatment paradigm for patients with hyperkalemia if successfully approved.

RDX020 for Metabolic Acidosis

Our research efforts have identified a set of potent, selective, and proprietary bicarbonate secretion inhibitors. The resulting RDX020 program has the potential to provide a novel approach to treating metabolic acidosis, by targeting the intestinal bicarbonate exchange mechanism. Metabolic acidosis is a highly prevalent comorbidity in CKD patients and is strongly correlated with disease progression and adverse outcomes. There are currently no approved treatments for chronic metabolic acidosis.