AAKP Innovator Series | Ardelyx

Tell us about your first experience with a kidney patient, personally or professionally, and what impressions that left on you as a fellow human being?

My first experience with patients with chronic kidney disease (CKD) was during my residency at University of Michigan, evaluating and caring for them across multiple stages of CKD. I worked with patients in earlier stages of the disease to manage co-morbidities like diabetes and hypertension to hopefully slow the progression of CKD. For those whose kidneys had eventually failed, such that they required renal replacement therapy (RRT), mostly in the form of maintenance dialysis, we worked together to address complications and issues. This sparked my initial interest in the field of nephrology and led me to do research work with Professor Friedrich (Fritz) Port, comparing mortality rates for RRT (Transplant vs. Maintenance Dialysis) in Blacks with end stage kidney disease (ESKD).1 Those experiences also led me to develop drugs in the renal space for patients with CKD on maintenance dialysis in my early years of pharmaceutical drug development and continues to fuel my interests today.

My most personal experience with kidney disease is as a patient myself. I had a cardiovascular event that led to an initial acute injury to my kidneys, and I was not sure if I would recover. I worried about disease progression and that fear was palpable. The difficulties navigating the health care system, even as a physician, were shocking. It made me want to advocate even more for patients, particularly those suffering from CKD on maintenance dialysis who are often unable to advocate for themselves.

Can you tell our AAKP national and global audiences why/how Ardelyx first came into kidney disease space and about the company’s commitment to kidney patients and their families?

In the early development of tenapanor, an NHE3 inhibitor, we initially identified for gastrointestinal issues, our lead clinical scientist noticed that the molecule also blocked some absorption of dietary phosphorus. Due to his prior experience developing medications to lower phosphorus in patients receiving dialysis, he realized the potential benefit that tenapanor might provide to patients with CKD on maintenance dialysis. Ardelyx worked diligently to make XPHOZAH® (tenapanor) available to patients with high blood phosphorous because of our ongoing commitment to patients with kidney disease and their families. Ardelyx continues to work to ensure that all dialysis patients have continued access to XPHOZAH.

As we know, many dialysis patients experience hyperphosphatemia or high phosphate levels in their blood, even with prescribed dialysis treatments. Can you share how and why Ardelyx developed XPHOZAH tablets to help patients with this condition?

Ardelyx was aware of the challenges dialysis patients faced when trying to control their blood phosphate. One challenge being that there was only a single class of therapy approved by the FDA to lower phosphorus, phosphate binders. While there were several binders approved, they all worked by the same mechanism of action, and although phosphate binders can be effective at lowering and managing phosphorus, it was often challenging for patients to adhere to the administration requirements and achieve sustained goal-recommended phosphorus levels.

Through our research, our scientists identified that tenapanor was able to block the absorption of phosphate at the paracellular pathway, the primary pathway of phosphate absorption. The discovery of this novel mechanism created a new option for nephrologists to manage elevated phosphorus levels and a distinct dosing regimen of only two small pills daily. Ardelyx conducted multiple clinical studies demonstrating the efficacy and safety of tenapanor, which ultimately lead to the approval of tenapanor, branded as XPHOZAH, in October of 2023. We are already hearing and seeing evidence that it is helping patients to lower and maintain their phosphorus levels.

We understand that XPHOZAH is an important novel medication, in fact the only therapy approved for patients who have an inadequate response to phosphate binder therapy. How can patients learn more about this option, how they may be able to access it, and current efforts to keep it accessible to patients who need it?

In addition to speaking directly with their healthcare provider, patients can also go to XPHOZAH.com to learn more about this treatment option and the best ways to access it.

Innovation in the kidney space is important to patients, they want to be involved and have their voice heard. Can you express the importance of why patients, in consultation with the healthcare professionals they choose to care for them, should have access to and choice of what treatment, whether established or new innovation, is right for them?

Ardelyx firmly believes that no two individuals are alike. This is true in general and especially true for patients with kidney disease. It is, therefore, critically important for healthcare professionals to take a patient-oriented approach to care. This includes taking time not only to understand an individual patients’ medical problems, but also try to understand their personal goals, aspirations and lifestyle. All medications have potential side effects, and some medications work better in one patient vs. another. It is therefore critical that healthcare professionals are able to provide choices to patients and to work with patients to find the best medication for their specific needs. As healthcare providers, it is important to have a variety of options in our “toolbox” to achieve key treatment goals.

As a leader in healthcare, you know well that it is not easy to develop a novel approach to treat or enhance care. What do you draw upon internally to keep your drive, optimism, and focus on patients going at full speed during the tough days?

My mantra has always been, “the patients are waiting…” When you focus on areas where there is a clear unmet medical need and innovative approaches are potential options to address those needs, it is the patients who are facing those challenges that drive us. They become our North Star; we are in lockstep with the task ahead and we are driven by the promise that we can make a difference in the lives of patients.

Final question – AAKP patients think this last question reveals a lot about a person – who is one of your heroes and why?

Derek Forfang, a true warrior for patients with kidney disease, who lost his fight to CKD in October 2023. Derek was an incredible person and advocate who meant so much to so many people, especially in the kidney community. For Ardelyx, he was the patient voice on our Scientific Advisory Board and was the driving force behind the creation of the Patient Advisory Council. Derek was a warrior who despite suffering many health setbacks, always persevered, put others first and continued his mission with a smile. To honor his memory, we created an award in his honor: the Derek Forfang Patient Advocate Award. We are currently asking the kidney community to nominate champions who like Derek, advocate for patients with CKD. We also changed the name of the Patient Advisory Council where he was a founding member to the “Derek Forfang Patient Advisory Council”.

Source: https://ardelyx.com/
1. Ojo AO, Port FK, Wolfe RA, Mauger EA, Williams L, Berling DP. Comparative Mortality Risks of Chronic Dialysis and Cadaveric Transplantation in Black End-Stage Renal Disease Patients. AJKD. 1994 Jul; 24 (1):59-64.

This article was originally published in the Jan./Feb. 2025 aakpRENALIFE magazine.