KERX Keryx Biopharmaceuticals, Inc. (delisted)

Keryx Biopharmaceuticals, Inc. (Nasdaq:KERX), a biopharmaceutical company focused on bringing innovative medicines to people with renal disease, today announced that the U.S. Food and Drug Administration (FDA) has accepted for review the supplemental New Drug Application (sNDA) for Auryxia® (ferric citrate) tablets. The sNDA acceptance by the FDA indicates that the application is sufficiently complete to permit a substantive review. The Prescription Drug User Fee Act (PDUFA) target action date for completion of the FDA’s review is November 6, 2017.

Auryxia is currently approved for use in the U.S. for the control of serum phosphorus levels in patients with end-stage renal disease requiring dialysis. Keryx, with this sNDA submission, is seeking to expand the indication for Auryxia to include the treatment of iron deficiency anemia (IDA) in patients with non-dialysis dependent chronic kidney disease (NDD-CKD).

“Iron deficiency anemia is a common complication in people with non-dialysis dependent chronic kidney disease, yet currently there are no FDA-approved oral medicines to treat this debilitating disease,” said John Neylan, M.D., senior vice president and chief medical officer of Keryx Biopharmaceuticals. “Acceptance of the sNDA filing not only brings us one step closer to providing this medicine to patients in need; it is an important milestone for the company and our efforts to leverage ferric citrate’s mechanism of action.”  

The sNDA was based on data from a 24-week placebo controlled Phase 3 trial in 234 adults with stage 3-5 NDD-CKD. Patients enrolled in the trial had hemoglobin levels between 9.0 g/dL and 11.5 g/dL and were intolerant to or had an inadequate response to prior treatment with oral iron supplements. The starting dose in the study was three tablets per day taken with meals. Importantly, during the study, patients were not allowed to receive any IV or oral iron, or ESAs. In the study, treatment with Auryxia demonstrated significant increases in hemoglobin levels of >1 g/dL at any point during the 16-week efficacy period for the majority of patients (52.1 percent; n=61/117), a clinically meaningful result. In the trial, 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.

About Iron Deficiency Anemia, NDD-CKDKeryx estimates that of the 1.7 million Americans with NDD-CKD under the care of a nephrologist, approximately 650,000 are treated for IDA and another 250,000 – 400,000 could have IDA but are not treated today. The prevalence and severity of IDA increases as kidney disease progresses. According to market research, nephrologists report that the majority of their patients are sub-optimally treated with currently available oral iron supplements and that a need exists for a new treatment option for their patients with iron deficiency anemia and NDD-CKD.

About Auryxia Auryxia (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 CKD on dialysis. Each Auryxia tablet contains 210 mg of ferric iron, equivalent to 1 g of ferric citrate. The U.S. approval of Auryxia was based on data from the company's Phase 3 registration program in dialysis patients. In the Phase 3 clinical trials, Auryxia effectively reduced serum phosphorus levels to within the KDOQI guidelines range of 3.5 to 5.5 mg/dL.

Auryxia binds with dietary phosphate in the GI tract and precipitates as ferric phosphate. The unbound portion of Auryxia has been shown to increase serum iron parameters including ferritin and transferrin saturation (TSAT). Iron absorption from Auryxia may lead to excessive elevations in iron stores. Accordingly, physicians should assess and monitor iron parameters before starting and while on Auryxia, and may need to decrease or discontinue IV iron for these patients. The most common adverse events for Auryxia treated patients were gastrointestinal related, including diarrhea, nausea, vomiting and constipation. For more information about Auryxia and the U.S. full prescribing information, visit www.Auryxia.com.

Use of ferric citrate in patients with NDD-CKD and IDA, as highlighted above, is investigational and has not been determined to be safe or efficacious.

IMPORTANT U.S. SAFETY INFORMATION FOR AURYXIA® (ferric citrate)

Contraindication: Patients with iron overload syndrome, e.g. hemochromatosis, should not take Auryxia®.

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

Accidental Overdose of Iron: Accidental overdose of iron containing products is a leading cause of fatal poisoning in children under 6 years of age. Keep Auryxia away from children as it contains iron. 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 Auryxia were diarrhea (21%), nausea (11%), constipation (8%), vomiting (7%) and cough (6%). Gastrointestinal adverse reactions were the most common reason for discontinuing Auryxia (14%). Auryxia 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 Auryxia. Ciprofloxacin should be taken at least 2 hours before or after Auryxia.

For Full Prescribing Information for Auryxia, please visit http://auryxia.com/important-safety-information/

Forward Looking Statements Some of the statements included in this press release, particularly those regarding the commercialization and ongoing clinical development of Auryxia and the FDA’s acceptance of the sNDA to expand the label of ferric citrate to include the treatment of IDA in adults with stage 3-5 NDD-CKD and the potential approval in this indication and the impact thereof on Keryx, 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 we can increase adoption of Auryxia in patients with CKD on dialysis; the risk that the FDA may not concur with our interpretation of our Phase 3 study results in NDD-CKD, supportive data, conduct of the studies, or any other part of our regulatory submission and could ultimately deny approval of ferric citrate for the treatment of IDA in adults with stage 3-5 NDD-CKD; the risk that if approved for use in NDD-CKD that we may not be able to successfully market Auryxia for use in this indication; our ability to continue to supply Auryxia following the recent resupply to the market; 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.

About Keryx Biopharmaceuticals, Inc.Keryx Biopharmaceuticals, Inc., with headquarters in Boston, Massachusetts, is a commercial stage company focused on bringing innovative medicines to people with renal disease. Keryx developed and commercializes Auryxia® (ferric citrate), an iron-based phosphate binder, in the U.S. Ferric citrate is marketed as Riona® by Keryx’s Japanese partner, Japan Tobacco Inc. and Torii Pharmaceutical Co. Ltd. In September 2015, the European Commission granted European market authorization for Fexeric® (ferric citrate coordination complex). Keryx has programs underway to leverage its development and commercial infrastructure, including evaluation of iron deficiency anemia in adults with non-dialysis dependent chronic kidney disease and in-licensing medicines for renal disease. For more information about Keryx, please visit www.keryx.com.

KERYX BIOPHARMACEUTICALS CONTACTS:
Amy Sullivan
Senior Vice President, Corporate Affairs
T: 617.466.3519 
amy.sullivan@keryx.com

Lora Pike
Senior Director, Investor Relations
T: 617.466.3511
lora.pike@keryx.com