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
In 2017, we successfully completed our Phase 3 T3MPO program, which included two registration studies for tenapanor in IBS-C – T3MPO-1 and T3MPO-2 – as well as a long-term safety study, T3MPO-3.
T3MPO-1 was a 12-week double-blind, placebo-controlled, multi-center, randomized trial with a four-week, placebo-controlled randomized withdrawal period. T3MPO-2 was a six-month, double-blind, placebo-controlled, multi-center, randomized trial. Both trials achieved their primary endpoints and tenapanor demonstrated the ability to meaningfully reduce abdominal pain and relieve constipation, with an increase in complete spontaneous bowel movements among patients treated with tenapanor versus those given placebo. Tenapanor was well-tolerated across studies.
With the completion of our Phase 3 program, we now have all data necessary to submit our planned New Drug Application to the U.S. Food and Drug Administration early in the fourth quarter of 2018.
CARDIORENAL CLINICAL TRIALS
Tenapanor for the treatment of hyperphosphatemia, or excess levels of phosphorus, in ESRD patients on dialysis is in Phase 3 development. In February 2018, we initiated the second Phase 3 clinical trial of tenapanor for hyperphosphatemia in patients with ESRD on dialysis. This clinical trial includes a 26-week open-label treatment period, with a 12-week placebo-controlled randomized withdrawal period followed by an additional 14-week open-label safety extension period for a total of up to 52 weeks. An active control group, for safety analysis only and consistent with other Phase 3 registration studies for hyperphosphatemia, will receive sevelamer, open-label, for the entire 52-week study period. We currently expect to report topline data from this clinical trial in 2019.
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.
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