SFP Clinical Development Progress

Complete:

Comprehensive pharmacology/toxicology program comprising of multiple studies
Phase 2a dose range study
SFP formulation GMP grade (patent pending)

Ongoing:

Phase 2b dose range study (data expected Q1 2010)
SFP vs. IV iron (data expected late 2010)

Anticipated:

Phase 3 clinical program

Clinical Studies

File NDA with FDA
Commercial launch U.S. (upon market approval)

A successful Phase 2a study evaluated the short-term safety and efficacy of delivering SFP to dialysis patients by way of dialysate. In the results, the authors concluded that a slow, frequent delivery of iron was a safe and effective alternative to IV iron dextran delivery. In the Phase 2a study, hemodialysis patients were stabilized on regular IV iron dextran and split into two groups: 10 who received SFP via dialysate and a control group of 11 who continued on IV iron dextran. At the end of the study, IV iron was required by all 11 patients in the control group while 8 of the 10 patients in the SFP group did not need any IV iron (Note: the 2 patients who received IV iron experienced excessive blood losses caused by amputation and bypass surgery which required hospitalization and removal from the study). The study demonstrated that patients receiving SFP regularly via dialysate did not require IV iron. (Source: Gupta, A. et al. Dialysate iron therapy: Infusion of soluble ferric pyrophosphate via the dialysate hemodialysis. Kidney International 1999; 55: 1891-1898)

Rockwell is currently conducting a Phase 2b, six-month, prospective, randomized, placebo-controlled, double-blind, dose-ranging study consisting of 131 patients at more than 30 dialysis centers in the United States and Canada. Patients receive dialysate containing SFP or standard dialysate during their normal, three times per week dialysis treatment. The primary objective of this study is to evaluate the safety of SFP at varying dose levels. Data from this study is expected to contribute to determining the optimal concentration of SFP needed to maintain iron balance within target hemoglobin range. The targeted enrollment in this study has been met and dosing completion is expected to occur in October 2009.

A NIH-funded Study is being conducted. This is a multi-center, prospective, randomized, placebo-controlled, 9-month study being conducted to examine oxidative stress markers in dialysis patients receiving SFP vs. IV iron sucrose (Venofer® is manufactured under license from Vifor (International) Inc., Switzerland). Clinical study design includes a target of 30 patients to examine maintenance of hemoglobin, iron parameters, the need for intravenous (IV) iron, and oxidative stress markers in patients receiving SFP via dialysate vs. patients receiving conventional (iron-free) dialysate. Patients in both groups are eligible to receive intermittent IV iron sucrose per protocol if iron deficiency develops. Note: Rockwell has assumed sponsorship of the study.

Future Phase 3 SFP Clinical Program

Rockwell intends to initiate a Phase 3 pivotal clinical program.