Who we do it for
Our patients are at the forefront of everything we do. Our team is passionately committed to bringing medicines that matter to underserved patients with gastrointestinal (GI) and cardiorenal diseases.
OUR CLINICAL TRIALS
GI CLINICAL TRIALS
IBS-C CLINICAL TRIALS
Two ongoing, pivotal Phase 3 clinical trials, T3MPO-1 and T3MPO-2, are being conducted in the U.S. with tenapanor in patients with irritable bowel syndrome with constipation (IBS-C). T3MPO-1 is a 12-week double-blind, placebo-controlled, multi-center, randomized trial with a four-week, placebo-controlled randomized withdrawal period. T3MPO-2 is a six-month, double-blind, placebo-controlled, multi-center, randomized trial. Subjects completing T3MPO-1 and T3MPO-2 are eligible to enroll into T3MPO-3, an open-label, long-term safety study where subjects can continue to receive tenapanor for up to one year. T3MPO-1 and T3MPO-2 are both fully enrolled, with over 600 patients enrolled in each trial, and the primary endpoint for both trials is the six of 12-week combined overall responder rate compared to placebo. A combined overall responder is defined as a weekly responder to tenapanor for six of 12 weeks where both an abdominal pain response and a complete spontaneous bowel movement (CSBM) response criteria are met during the same week. An abdominal pain responder is defined as a patient with a 30 percent or greater reduction in average weekly worst abdominal pain compared to baseline during the week, and a CSBM responder is defined as a patient who has an increase of one or more on average weekly CSBMs compared to baseline during the week. We expect to announce results from T3MPO-1 and T3MPO-2 in the second quarter and second half of 2017, respectively. T3MPO-3 is also fully enrolled with over 300 patients and is expected to be complete by late 2017.
CARDIORENAL CLINICAL TRIALS
Tenapanor for the treatment of hyperphosphatemia, or excess levels of phosphorus, in ESRD patients on dialysis is in Phase 3 development. We reported positive efficacy and safety data from the first Phase 3 trial in this program in February 2017. The study was an eight-week, double-blind, randomized trial, with a four-week placebo-controlled randomized withdrawal (RW) period. The study met its primary endpoint, demonstrating a statistically significant difference in change of serum phosphorus between tenapanor and placebo arms from the end of treatment to the end of the RW period in the “responder” patient population and demonstrated a favorable GI tolerability profile. We plan to initiate a second Phase 3 trial of tenapanor in this patient population in mid-2017.
We are evaluating RDX7675 for the treatment of hyperkalemia, or high levels of potassium in the blood, a common condition in patients with chronic kidney disease (CKD), heart failure, and those who are also taking renin-angiotensin-aldosterone system inhibitor (RAASi) medications.
We initiated a Phase 3 clinical trial with RDX7675 for the treatment of hyperkalemia in December 2016. The study will enroll patients with hyperkalemia who are taking RAASi medications. The trial will include three parts: Part A will be a single-blind study in which all subjects receive RDX7675 for four weeks; Part B will be an eight-week, double-blind, placebo-controlled, randomized withdrawal study; and Part C is an open-label, long-term safety study for subjects from Parts A and B. The primary endpoint for Part A is serum potassium change from Part A baseline, and the primary endpoint for Part B is serum potassium change from Part B baseline (RDX7675 versus placebo).
In addition to the Phase 3 trial, we initiated an onset-of-action study in December 2016, designed to evaluate the effect of RDX7675 on the rate of blood potassium lowering, along with safety and efficacy, in approximately 60 patients with hyperkalemia. Data from this trial are expected in the third quarter of 2017.
Irritable Bowel Syndrome with Constipation (IBS-C): a gastrointestinal disorder characterized by significant abdominal pain and constipation (when a bowel movement is difficult due to insufficient/decreased amount of fluid in the GI, or happens less often than normal). IBS-C significantly impacts the health and quality of life of affected patients. The cause of IBS-C is unknown, and there are currently no specific diagnostic tests or biomarkers for detection. Therefore, IBS-C is diagnosed by symptoms and by eliminating other disorders. IBS-C is similar to chronic constipation but is clinically distinct as a result of the significant abdominal pain component.
people in the U.S. with IBS-C1
in health-related quality of life and work productivity2
1. Lovell 20122. Shin 20153. Doshi 20144. Heidelbaugh, et al 2015
RENAL DIALYSIS PATIENTS
End-Stage Renal Disease (ESRD): the final stage of chronic kidney disease, occurring when a person’s kidneys can no longer support the body’s needs to remove waste and excess fluid from the body. The most common causes of ESRD in the U.S. are diabetes and high blood pressure, and it is often treated with dialysis.
Hyperphosphatemia: the medical term for an abnormally elevated level of phosphorus in the blood. Phosphorus is one of the most abundant and essential elements in the body, and plays an important role in multiple biological processes. The kidney is the major organ involved in regulating phosphorus levels in the body. When kidney function is impaired, phosphorus is not excreted adequately from the body. Therefore, hyperphosphatemia is a common condition associated with ESRD in people receiving dialysis.
ESRD patients with hyperphosphatemia (HP) in major developed countries1
of U.S. dialysis patients taking phosphate binders to manage HP2
1. USRDS 2014; European ERA-EDTA Registry Annual Report 2012; Nakai S, et al, 2008. includes U.S. EU and Japan.2. Decision Resources 2015 3. Chiu 20094. Lederer 2016
CKD/HEART FAILURE PATIENTS
Chronic Kidney Disease (CKD): a condition characterized by gradual loss of kidney function over time. CKD can be caused by diabetes, high blood pressure and other disorders. CKD also increases a person’s risk of having cardiovascular disease. As CKD progresses, it can lead to kidney failure, which ultimately requires dialysis or a kidney transplant.
Hyperkalemia (HK): the medical term that describes a potassium level in a person’s blood that is higher than normal. Potassium is a nutrient that is critical to the function of nerve and muscle cells, including those in the heart. HK does not affect all patients in the same manner and there is no single threshold which is considered dangerous; however, having a blood potassium level higher than 5.0mEq/L can be life threatening and requires immediate treatment. People with heart failure, CKD and diabetes are at the greatest risk of developing HK due to side effects of the drugs these patients take to manage their underlying disease and the kidney’s weakened ability to excrete excess potassium as a result of these conditions.
people in the U.S. with CKD and/or heart failure have HK1
with RAAS inhibitors remains standard and problematic part of treatment management among physicians2
1. Einhom et al, 2009, HF; M. RDX-022 Market Opportunity – Spherix – 2015-07-08.pptx. Independent Market Research, Spherix Global Insights2. Maggioni 20133. Kovesday 2015, Jain 2012, Chaing 20164. Tomino 2007 5. Perleberg 2016 and ARDX research