SOURCE: Rockwell Medical Technologies, Inc.

Rockwell Medical Technologies, Inc.

May 20, 2013 08:00 ET

Rockwell Medical Presents PRIME Study Results at the 50th ERA-EDTA Meeting in Istanbul, Turkey

Unique Iron Drug SFP Met Primary Endpoint Demonstrating Statistically Significant 35% Reduction in ESA Dose; Safety Profile Similar to Placebo

WIXOM, MI--(Marketwired - May 20, 2013) - Rockwell Medical (NASDAQ: RMTI), a fully-integrated biopharmaceutical company targeting end-stage renal disease (ESRD) and chronic kidney disease (CKD) with innovative products and services for the treatment of iron deficiency, secondary hyperparathyroidism and hemodialysis, announced today PRIME clinical study results for Soluble Ferric Pyrophosphate (SFP) at the 50th ERA-EDTA congress in Istanbul, Turkey. SFP is the Company's investigational iron-delivery drug for the treatment of iron deficiency in hemodialysis patients, nearing completion of its Phase 3 clinical studies. Dr. Ajay Gupta, MD, Chief Scientific Officer of Rockwell presented the PRIME study results at the Late Breaking Clinical Trials session. This is fully-analyzed data, including atypical data subsequently found in a few study subjects, superseding initial top-line data announced Feb 4, 2013.

"SFP is a very promising parenteral iron supplement for the treatment of anemia in CKD-HD patients and could address the unmet need of correcting their functional iron deficiency," stated Professor Francesco Locatelli, MD, Scientific Director of the Department of Nephrology, Dialysis and Renal Transplantation at Alessandro Manzoni Hospital Lecco, Italy. "The results of this study are important as it is the first demonstration that administration of soluble iron salts can be achieved safely without the risk of high serum iron concentrations, which can potentially lead to oxidative stress. This study further demonstrates the potential for reducing IV iron and/or ESA doses while maintaining hemoglobin levels in CKD-HD patients."

"We are very pleased to present the exciting results of this well-run study," stated Dr. Raymond Pratt, Chief Medical Officer of Rockwell Medical. "The SFP dose of 11 µg iron/dL dialysate delivered sufficient iron without increasing iron stores while greatly reducing ESA and IV iron requirements. The safety profile of SFP was excellent. The fact that the incidence of intra-dialytic hypotension was numerically less in SFP compared to placebo group supports the safety of SFP-iron delivery via dialysate. The 35% higher ESA dose required in the placebo arm to maintain hemoglobin compared to the SFP arm, supports our belief that SFP will demonstrate efficacy in the Phase 3 CRUISE clinical studies, where ESA titration and IV iron is not permitted." 

The PRIME study was a nine-month, prospective, randomized, placebo-controlled, double-blinded, multi-center study in United States that randomized 108 patients equally to dialysate containing SFP-iron versus conventional dialysate. A total of 103 patients received blinded study drug (52 SFP, 51 Placebo; modified ITT population). In all study patients, ESA doses were titrated to maintain hemoglobin in a target range of 9.5 to 11.5 g/dL according to an algorithm managed by an independent centralized anemia management group. At the end of treatment, the hemoglobin value in the SFP group was 10.5 g/dL and 10.4 g/dL in the placebo group. Intravenous (IV) iron was administered according to a protocol to treat iron deficiency. 

The primary objective of the study was to determine whether regular administration of SFP via dialysate reduced the requirement for ESA dose by maintaining iron balance and optimizing iron delivery. The primary endpoint was the percent change in ESA dose from baseline to end of treatment (final two weeks of treatment period). Baseline ESA dose was similar between SFP (9483 U/wk) and placebo (9205 U/wk). In the modified ITT population, at the end of the study, ESA dose in the SFP arm was 9871 U/wk and placebo was 12628 U/wk. After adjusting for differences in baseline hemoglobin, the SFP arm required 35% less prescribed ESA compared to placebo. The difference between the two groups was statistically significant (p=0.045). The ESA sparing effect from SFP was observed without any increase in serum ferritin or pre-dialysis transferrin saturation. The PRIME study demonstrated that regular administration of SFP-iron via dialysate reduced the usage of erythropoietin stimulating agents (ESAs) during hemodialysis by 35% while maintaining iron balance and maximizing iron delivery.

The adverse event profile of the SFP group was similar to placebo with respect to event, frequency and severity. The numbers and types of serious adverse events were also similar in both groups. Importantly the incidence, frequency and severity of intradialytic hypotension in the SFP group were similar to placebo. There were no hypersensitivity or anaphylactoid reactions reported in the SFP group over the 9 months of the study.

About SFP
SFP is a unique iron compound that is delivered to the hemodialysis patient via dialysate, replacing the 5-7 mg of iron lost during a dialysis treatment. SFP is introduced into the sodium bicarbonate concentrate that subsequently is mixed into dialysate. Once in the dialysate, SFP crosses the dialyzer membrane and enters the bloodstream where it immediately binds to apo-transferrin and is taken to the bone marrow, mimicking the way dietary iron is processed in the human body. In completed clinical trials to date, SFP has demonstrated that it can safely deliver iron and maintain hemoglobin levels, while decreasing ESA use without an increase in iron stores.

About Rockwell Medical
Rockwell Medical is a fully-integrated biopharmaceutical company targeting end-stage renal disease (ESRD) and chronic kidney disease (CKD) with innovative products and services for the treatment of iron deficiency, secondary hyperparathyroidism and hemodialysis. 

Rockwell's lead drug candidate in late-stage clinical development is for the treatment of iron deficiency in dialysis patients and is called Soluble Ferric Pyrophosphate (SFP). SFP delivers iron to the bone marrow of dialysis patients in a non-invasive, physiologic manner via dialysate during their regular dialysis treatment. In completed clinical trials to date, SFP has demonstrated that it can safely deliver sufficient iron to the bone marrow. SFP is nearing completion of its Phase 3 clinical studies (CRUISE-1 and CRUISE-2) and is expected to address an estimated $600M U.S. market. 

Rockwell is preparing to launch its FDA approved generic drug called Calcitriol to treat secondary hyperparathyroidism in dialysis patients. Calcitriol active vitamin D injection is indicated in the management of hypocalcemia in patients undergoing chronic renal dialysis. It has been shown to significantly reduce elevated parathyroid hormone levels. Reduction of PTH has been shown to result in an improvement in renal osteodystrophy. Rockwell intends to launch Calcitriol as soon as it receives FDA manufacturing approval, addressing an estimated $350M U.S. market. 

Rockwell is also an established manufacturer and leader in delivering high-quality hemodialysis concentrates/dialysates to dialysis providers and distributors in the U.S. and abroad. As one of the two major suppliers in the U.S., Rockwell's products are used to maintain human life by removing toxins and replacing critical nutrients in the dialysis patient's bloodstream. Rockwell has three manufacturing and distribution facilities located in the U.S. and its operating infrastructure is a ready-made sales and distribution channel that is able to provide seamless integration into the commercial market for its drug products, Calcitriol and SFP upon FDA market approval. 

Rockwell's exclusive renal drug therapies support disease management initiatives to improve the quality of life and care of dialysis patients and are intended to deliver safe and effective therapy, while decreasing drug administration costs and improving patient convenience. Rockwell Medical is developing a pipeline of drug therapies, including extensions of SFP for indications outside of hemodialysis. Please visit www.rockwellmed.com for more information. For a demonstration of SFP's unique mechanism of action in delivering iron via dialysate, please view the animation video at http://www.rockwellmed.com/collateral/documents/english-us/mode-of-action.html.

Certain statements in this press release constitute "forward-looking statements" within the meaning of the federal securities laws, including, but not limited to, Rockwell's intention to launch Calcitriol and SFP following FDA approval. Words such as "may," "might," "will," "should," "believe," "expect," "anticipate," "estimate," "continue," "predict," "forecast," "project," "plan," "intend" or similar expressions, or statements regarding intent, belief, or current expectations, are forward-looking statements. While Rockwell Medical believes these forward-looking statements are reasonable, undue reliance should not be placed on any such forward-looking statements, which are based on information available to us on the date of this release. These forward looking statements are based upon current estimates and assumptions and are subject to various risks and uncertainties, including without limitation those set forth in Rockwell Medical's SEC filings. Thus, actual results could be materially different. Rockwell Medical expressly disclaims any obligation to update or alter statements whether as a result of new information, future events or otherwise, except as required by law.

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