Keryx Biopharmaceuticals Announces Positive Top-line Results from Pivotal  Phase 3 Study of Ferric Citrate for the Treatment of Iron Deficiency Anemia  in Adults with Non-Dialysis Dependent Chronic Kidney Disease

Registration trial demonstrated statistically significant differences versus placebo for the primary and all pre-specified secondary endpoints --- Majority of patients in the ferric citrate group (52 percent) achieved a 1 g/dL increase in hemoglobin vs. 19 percent in the placebo group --- Safety profile consistent with previously reported clinical studies --- Data support Keryx’s plan to submit a supplemental new drug application (sNDA) in the third quarter of 2016 seeking to expand ferric citrate’s indication --- Conference call to be held today at 8:00 a.m. ET

 

BOSTON, March 29, 2016 -- Keryx Biopharmaceuticals, Inc. (KERX), a biopharmaceutical company focused on bringing innovative medicines to market for people with renal disease, today announced positive top-line results for its pivotal 24-week Phase 3 study of ferric citrate, an oral, iron-based medicine in development for the treatment of iron deficiency anemia (IDA) in adults with stage 3-5 non-dialysis dependent chronic kidney disease (NDD CKD). The study met its primary endpoint and all pre-specified secondary endpoints with statistical significance.

Iron deficiency anemia is a common complication of CKD, and the prevalence and severity of IDA increases as kidney disease progresses. It is estimated that there are approximately 1.6 million people living in the U.S. with stage 3-5 non-dialysis dependent chronic kidney disease and iron deficiency anemia. If ferric citrate is approved for the treatment of iron deficiency anemia in stage 3 – 5 NDD CKD patients, it would be the only FDA-approved iron treatment in a tablet form available to these patients.

 

The Phase 3 study compared treatment with ferric citrate to placebo in 234 patients who previously had not adequately responded to or tolerated current oral iron therapies. The study achieved the primary endpoint with 52 percent (61/117) of patients who received ferric citrate achieving a 1g/dL or greater rise in hemoglobin at any time point during the 16-week randomized efficacy period, compared to 19 percent (22/115) in the placebo group (p<0.001), a clinically meaningful and statistically significant improvement. Two patients in the placebo group discontinued the study and were not included in the efficacy analysis – one discontinued after randomization prior to receiving placebo, one discontinued after taking a dose of placebo but before having laboratory values drawn. Statistically significant differences in all pre-specified secondary efficacy endpoints were also observed. During the full 24 weeks of the study, ferric citrate was generally well tolerated and adverse events were consistent with its known safety profile, with diarrhea reported as the most common adverse event.

 

“These Phase 3 results demonstrate that ferric citrate can effectively raise hemoglobin levels in stage 3 – 5 non-dialysis dependent chronic kidney disease patients, with effects observed as early as two weeks post-treatment initiation,” said John Neylan, M.D., chief medical officer of Keryx Biopharmaceuticals. “Based on these results, we plan to submit an sNDA to the FDA in the third quarter, seeking to expand the label for ferric citrate to include the treatment of iron deficiency anemia in people with stage 3 – 5 NDD CKD. We believe that the ability to treat iron deficiency anemia, managing hemoglobin and iron levels, could have an important effect on the way kidney specialists treat these patients.”

 

About the Phase 3 Registration Study

The pivotal Phase 3 study enrolled 234 patients who previously had not adequately responded to or tolerated oral iron therapies at 32 clinical sites in the United States. Patients were randomized 1:1 (ferric citrate versus placebo). Patients enrolled in this study were not allowed to receive any intravenous (IV) or oral iron, or erythropoiesis-stimulating agents (ESAs) during this study. The study had a 16-week, randomized, double-blind, placebo-controlled, efficacy period followed by an 8-week open-label safety extension period in which all patients remaining in the study, including the placebo group, received ferric citrate. During the 16-week efficacy period, ferric citrate was administered at a starting dose of three tablets per day with food and could be titrated every four weeks by an additional three tablets for up to a maximum of 12 tablets per day; the mean dose received in ferric citrate treated patients was 5 tablets per day. The primary endpoint was the proportion of patients achieving a 1 g/dL or greater increase in hemoglobin at any point during the 16-week efficacy period. Baseline laboratory values were similar between the treatment arms.