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Keryx Biopharmaceuticals Initiates Pivotal Phase 3 Study of Ferric Citrate for the Treatment of Iron Deficiency Anemia in Patients With Non-Dialysis Dependent Chronic Kidney Disease

NEW YORK, Sept. 29, 2014 (GLOBE NEWSWIRE) -- Keryx Biopharmaceuticals, Inc. (Nasdaq:KERX) announced today the initiation of its pivotal Phase 3 study of Ferric Citrate for the treatment of iron deficiency anemia in patients with non-dialysis dependent (Stage 3-5) chronic kidney disease ("NDD-CKD"). On September 5, 2014, the U. S. Food and Drug Administration (FDA) approved Ferric Citrate (formerly known as Zerenex™) for the control of serum phosphorus levels in patients with chronic kidney disease (CKD) on dialysis.

"We are thrilled to be initiating this pivotal study to evaluate Ferric Citrate as a treatment for iron deficiency anemia in patients with stages 3 - 5 of CKD," commented Ron Bentsur, Keryx's Chief Executive Officer. "We are encouraged by the positive results observed in our Phase 2 study in this patient population and we believe that the study commencing today, if successful, will support indication expansion for Ferric Citrate."

The Phase 3 study initiated today, entitled "A Phase 3 Study of KRX-0502 (Ferric Citrate) for the Treatment of Iron Deficiency Anemia in Adult Subjects with Non-Dialysis Dependent Chronic Kidney Disease," is a multi-center clinical trial, comprised of a 16-week, randomized, double-blind, placebo-controlled period ("Randomized Period"), followed by an 8-week open-label safety extension period in which all subjects receive Ferric Citrate ("Extension Period"), for a total of 24 weeks. Approximately 230 subjects, who have previously had an inadequate response to oral iron supplements, will be randomized into the Randomized Period in a 1:1 ratio to receive either Ferric Citrate or matching placebo. Intravenous (IV) iron and erythropoiesis-stimulating agent (ESA) use is prohibited during and leading up to the study. There are currently no oral iron formulations that are FDA approved for the treatment of iron deficiency anemia in CKD patients.

This study's primary endpoint is the between group comparison of the proportion of patients achieving a 1 g/dL or greater increase in hemoglobin at any point during the 16-week Randomized Period. In the 12-week Phase 2 study in NDD-CKD previously conducted by Keryx, a post-hoc analysis of this endpoint demonstrated that the proportion of patients achieving a 1g/dL or greater increase in hemoglobin at any time point during the study was 40% in the Ferric Citrate arm vs. 15% in the placebo arm (p-value <0.001). Secondary endpoints in the Phase 3 study include change from baseline to end of Randomized Period for hemoglobin, ferritin, TSAT and serum phosphorous.

The study's Co-Chairmen are Geoffrey Block, MD, Director of Clinical Research at Denver Nephrology; Glenn Chertow, MD, Professor of Medicine and Chief, Division of Nephrology at Stanford University School of Medicine; and Steven Fishbane, MD, Division Chief, Kidney Disease and Hypertension at North Shore University Hospital/Long Island Jewish Medical Center. The study will be conducted at approximately 40 sites in the U.S.

Dr. Block, commented, "We believe that Ferric Citrate could represent an advancement in the treatment of iron-deficiency anemia for this underserved patient population. The ability to potentially treat CKD patients with iron deficiency anemia with an oral agent could be compelling for physicians and patients."

Dr. Chertow, added, "Current treatment strategies for iron deficiency anemia in CKD are clearly not optimal. There are safety concerns regarding the use of intravenous iron. Moreover, IV iron administration always requires supervision in a healthcare setting, and frequently obligates cannulation of the forearm veins we aim to preserve for hemodialysis vascular access. Commercially available oral iron preparations are generally ineffective due to limited absorption and are poorly tolerated. As such, the population of patients with CKD and iron deficiency anemia remains underserved."

Dr. Fishbane, stated, "As CKD progresses, the prevalence of iron deficiency anemia increases. It is estimated that there are well over 1.5 million CKD stages 3-5 patients with iron deficiency anemia. If approved in this indication, Ferric Citrate could represent a viable treatment option for these patients."

Keryx Biopharmaceuticals retains a worldwide exclusive license (except for the Asian Pacific Region) to Ferric Citrate from Panion & BF Biotech, Inc. The Company has sublicensed the development of ferric citrate in Japan to Japan Tobacco Inc. and Torii Pharmaceutical Co., Ltd.

About Non-Dialysis Dependent Chronic Kidney Disease and Iron Deficiency Anemia

It is estimated that approximately 10% to 15% of the U.S. adult population is affected by chronic kidney disease (CKD), a condition generally characterized by greater than 50% reduction of normal kidney function. Iron deficiency anemia, which develops early in the course of CKD and worsens with disease progression, is extremely prevalent in the NDD-CKD population and is associated with fatigue, lethargy, decreased quality of life and is also believed to be associated with cardiovascular complications, hospitalizations, and increased mortality. Based on data contained in a 2009 publication in the Journal of the American Society of Nephrology, it is estimated that over 1.5 million adults with NDD-CKD in the U.S. alone are also afflicted with iron deficiency anemia. To combat this anemia, iron replacement therapy is essential to increase iron stores, such as ferritin and TSAT levels, and raise hemoglobin levels.

About Ferric Citrate

Ferric Citrate was approved by the U.S. Food and Drug Administration on September 5, 2014 and is indicated in the U.S. for the control of serum phosphorus levels in patients with chronic kidney disease (CKD) on dialysis. Keryx plans to make Ferric Citrate available to U.S. dialysis patients in the fourth quarter 2014.

In January 2014, ferric citrate was approved for the treatment of patients with all stages of CKD in Japan, where it is being marketed as Riona® by Keryx's Japanese partner, Japan Tobacco Inc. and Torii Pharmaceutical Co. Ltd.

In March 2014, Keryx has filed a Marketing Authorization Application (MAA) with the European Medicines Agency (EMA), seeking the approval of Ferric Citrate as a treatment of hyperphosphatemia in patients with CKD, including dialysis and non-dialysis dependent patients, and that application is currently under review.

About Keryx Biopharmaceuticals, Inc.

Keryx Biopharmaceuticals, headquartered in New York, is focused on bringing innovative therapies to market for patients with renal disease. The Company's lead product, Ferric Citrate, is approved in the United States for the control of serum phosphorus levels in patients with chronic kidney disease on dialysis. Keryx plans to commercially launch Ferric Citrate in the U.S. in the fourth quarter of 2014. In January 2014, ferric citrate was approved for the treatment of patients with all stages of CKD in Japan, where it is being marketed as Riona® by Keryx's Japanese partner, Japan Tobacco Inc. and Torii Pharmaceutical Co. Ltd.

Important Safety Information about Ferric Citrate

Contraindication: Patients with an accumulation of iron in their body, e.g. hemochromatosis, should not take Ferric Citrate.

Iron Overload: Iron absorption from Ferric Citrate may lead to increased iron in storage sites. Iron parameters should be monitored prior to and while on Ferric Citrate. Patients receiving IV iron may require a reduction in dose or discontinuation of IV iron therapy.

Accidental Overdose of Iron: Keep Ferric Citrate away from children as it contains iron. Accidental overdose of iron containing products is a leading cause of fatal poisoning in children under 6 years of age. Call a poison control center or your physician in case of an accidental overdose in a child.

Patients with Gastrointestinal Bleeding or Inflammation: Safety has not been established for these patients.

Adverse Events: The most common adverse events with Ferric Citrate were diarrhea (21%), nausea (11%), constipation (8%), vomiting (7%) and cough (6%). Gastrointestinal adverse reactions were the most common reason for discontinuing Ferric Citrate (14%).

Ferric Citrate contains iron and may cause dark stools, which is considered normal with oral medications containing iron.

Drug Interactions: Doxycycline should be taken at least 1 hour before Ferric Citrate.

Cautionary Statement

Some of the statements included in this press release, particularly those regarding the commercialization and subsequent clinical development of Ferric Citrate, may be forward-looking statements that involve a number of risks and uncertainties. For those statements, we claim the protection of the safe harbor for forward-looking statements contained in the Private Securities Litigation Reform Act of 1995. Among the factors that could cause our actual results to differ materially are the following: whether Ferric Citrate will be successfully launched and marketed in the U.S.; whether the FDA will approve a brand name for Ferric Citrate prior to the projected launch date; whether Riona® will be successfully marketed by our Japanese partner, Japan Tobacco, Inc. and Torii Pharmaceutical Co., Ltd; the risk that the EMA may not concur with our interpretation of our Phase 3 study results, supportive data, conduct of the studies, or any other part of our MAA submission and could ultimately deny approval of the MAA; the risk that we may not be successful in the development of ferric citrate for the treatment of iron deficiency anemia in non-dialysis chronic kidney disease patients; and other risk factors identified from time to time in our reports filed with the Securities and Exchange Commission. Any forward-looking statements set forth in this press release speak only as of the date of this press release. We do not undertake to update any of these forward-looking statements to reflect events or circumstances that occur after the date hereof. This press release and prior releases are available at http://www.keryx.com. The information found on our website is not incorporated by reference into this press release and is included for reference purposes only.

CONTACT: KERYX CONTACT: Amy Sullivan Vice President - Corporate Development and Public Affairs Keryx Biopharmaceuticals, Inc. Tel: 617.466.3447 E-mail: amy.sullivan@keryx.com

Source:Keryx Biopharmaceuticals, Inc.