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Rockwell Medical Announces SFP Phase 3 Efficacy Study CRUISE-2 Completes Patient Dosing

WIXOM, MI--(Marketwired - Jul 22, 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 that its second Phase 3 efficacy study for SFP, called CRUISE-2, has completed patient dosing. The CRUISE-2 clinical study is the final Phase 3 study for Soluble Ferric Pyrophosphate (SFP), Rockwell's investigational iron-delivery drug for the treatment of iron deficiency in chronic kidney disease patients receiving hemodialysis.

"The completion of the CRUISE-2 Phase 3 study marks the closing of our Phase 3 efficacy trials and is truly a significant milestone achievement for Rockwell," stated Rob Chioini, Founder, Chairman and CEO of Rockwell Medical. "We anticipate releasing CRUISE-2 top-line results in September, and expect to submit our new drug application approximately 4 to 6 months after that. We expect CRUISE-2 top-line results to confirm the positive CRUISE-1 results that were announced a couple weeks ago. We are very focused in moving SFP through the regulatory process. We believe SFP has the potential to become the new standard of care in iron therapy for dialysis patients."

On July 11, 2013, Rockwell announced positive Phase 3 efficacy results from the CRUISE-1 study, demonstrating that SFP met the primary and key secondary endpoints. SFP met the primary endpoint by achieving a statistically significant mean change in hemoglobin from baseline to End-of-Treatment (p=0.011). Key secondary endpoints were also achieved, including maintenance of hemoglobin, maintenance of reticulocyte hemoglobin, and increase in serum iron pre-to-post treatment without an increase in ferritin.

"We are very pleased to complete this final Phase 3 study," stated Dr. Ray Pratt, Chief Medical Officer of Rockwell Medical. "SFP demonstrated a statistically significant difference in hemoglobin levels between patients receiving SFP and placebo in CRUISE-1, while showing a very clean safety profile, and we expect CRUISE-2 to do the same. The study clearly showed that SFP maintains hemoglobin in patients not receiving any supplemental oral or intravenous iron, and effectively delivers iron to the bone marrow without increasing iron stores. All clinical data to date tells us that SFP is a very promising iron drug that targets the unmet need for correcting functional iron deficiency in CKD-HD patients."

The CRUISE Studies
Rockwell has conducted two pivotal Phase 3 efficacy studies called CRUISE-1 and CRUISE-2. Each study is a prospective, randomized, placebo-controlled, multicenter study to demonstrate efficacy and safety of SFP-iron, delivered via dialysate, in adult CKD patients requiring hemodialysis. Each study comprises approximately 300 patients, randomized equally between SFP and placebo groups with a treatment period of up to 12 months. The primary efficacy end-point for both studies is the mean change in hemoglobin from baseline.

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, similar to how 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 increasing 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 has completed its Phase 3 clinical study program (CRUISE-1 and CRUISE-2). SFP is expected to address an estimated $600M U.S. market. 

Rockwell is preparing to launch its FDA approved generic drug 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 once 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/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.