WIXOM, MI--(Marketwired - February 04, 2014) - Rockwell Medical, Inc. (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 replacement, secondary hyperparathyroidism and hemodialysis, today announced its large-scale, long-term safety study is complete. This final Phase 3 study and the two CRUISE clinical efficacy and safety studies were conducted in support of the New Drug Application (NDA) for marketing approval of Triferic. Triferic is the Company's late-stage investigational iron-replacement drug for treating iron deficiency in chronic kidney disease patients receiving hemodialysis.
"We are thrilled to have achieved this significant milestone event," stated Mr. Robert L. Chioini, Founder, Chairman and CEO of Rockwell. "The Phase 3 clinical program is now complete and we are focused on filing our NDA with the FDA in the near-term. This drug's unique mode of action, its delivery via dialysate and efficient iron delivery directly to the bone marrow, enables it to be used immediately, like dietary iron, and maintain hemoglobin while significantly reducing the need for ESA. These clinical benefits highlight its potential to be the market-leading iron therapy treatment for CKD-HD patients."
"We continue to be impressed with Triferic and its safety profile," stated Dr. Raymond Pratt, CMO of Rockwell. "Our clinical program is easily the largest and longest cohort of any parenteral iron drug. We have dosed Triferic in over 100,000 separate administrations and we have not identified an acute safety signal or anaphylactic reaction. This additional safety data, coupled with the successful Phase 3 CRUISE studies and ongoing open-label study, gives us great confidence that Triferic can provide dialysis patients a safe, effective maintenance iron therapy."
These safety studies were the continuation of the CRUISE efficacy trials and Study SFP-6, a prospective, randomized, double-blinded, placebo-controlled, crossover, multicenter, multinational, Phase 3 study with an enrollment of 718 CKD-HD patients in the US and Canada. Both studies allowed patients to continue to receive Triferic in the open-label extension study for up to one additional year. Consistent with expectations, a preliminary review of the safety data revealed:
- Triferic reliably delivers iron via dialysate.
- There were no specific adverse events directly attributable to Triferic™.
- The adverse events during Triferic administration were those common in CKD-HD patients.
- No difference in type, frequency, severity or resolution of adverse events between Triferic and placebo.
- No anaphylaxis or hypersensitivity attributable to Triferic™.
- No evidence of changes in hepatic enzymes between Triferic and placebo.
- No evidence of first use events or an increase in intradialytic hypotension.
Triferic is a unique iron compound that is delivered to the hemodialysis patient via dialysate, replacing the 5-7 mg of iron that is lost during every dialysis treatment. Triferic is introduced into the sodium bicarbonate concentrate on-site at the dialysis clinic, which is subsequently mixed into dialysate. Once in the dialysate, Triferic crosses the dialyzer membrane and enters the blood 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, Triferic has demonstrated that it can safely and effectively deliver sufficient iron to the bone marrow, maintain hemoglobin and not increase iron stores (ferritin), while significantly reducing ESA dose.
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 replacement, secondary hyperparathyroidism and hemodialysis.
Rockwell's lead drug candidate Triferic is in late-stage clinical development for the treatment of iron replacement in dialysis patients. Triferic delivers iron to the bone marrow of dialysis patients in a non-invasive, physiologic manner during their regular dialysis treatment, using dialysate as the delivery mechanism. In completed clinical trials to date, Triferic has demonstrated that it can safely and effectively deliver sufficient iron to the bone marrow, maintain hemoglobin and not increase iron stores (ferritin), while significantly reducing ESA dose. Triferic has successfully completed the efficacy trials of its Phase 3 clinical study program (CRUISE-1 and CRUISE-2). Triferic 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 Triferic 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 Triferic for indications outside of hemodialysis. Please visit www.rockwellmed.com for more information. For a demonstration of the Triferic 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 Triferic 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.