SFP Clinical Development Progress

Complete:

Comprehensive pharmacology/toxicology program comprising of multiple studies
Phase IIa dose escalating study
Phase IIb dose range study (Data released Feb 2010)
Proprietary GMP grade SFP formulation; patent protected through 2029

Ongoing:

Phase III efficacy study, CRUISE - Continuous Replacement Using Iron Soluble Equivalents (March 2011):

Two efficacy trials running simultaneously
300 patients per trial; 150 patients per arm with 1:1 randomization between SFP and placebo
Primary efficacy endpoint: mean change from baseline in Hgb compared to placebo

PRIME ESA-sparing study (March 2011):

Compartive study; 9-month duration
100 patients receiving SFP or leading IV iron
Designed to capture ESA-sparing data; bundled reimbursement in effect Jan 2011

SFP brand extensions targeting new markets
Joint venture development and licensing opportunities

Anticipated:

Clinical development initiated for delivering iron in TPN solution (2011)
SFP commercial launch U.S. (upon FDA market approval est. 2013)

A successful Phase IIa 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 IIa 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 in the SFP group 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 has completed a Phase IIb, 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 evaluated the safety of SFP at varying dose levels. Data from this study determined the optimal concentration of SFP needed to maintain iron balance within target hemoglobin range. This study was completed in October 2009 and top line clincial trial results were published February 2010. The Phase IIb study demonstrated that SFP maintains iron balance and hemoglobin while eliminating need for IV iron for treatment related blood losses. SFP iron safely crosses the dialyzer membrane during dialysis and enters the bloodstream, immediately binding to apo-transferrin and then traveling to the bone marrow. SFP has an excellent safety profile and has not caused a single anaphylactic episode.

Phase III SFP Clinical Program

Phase III CRUISE efficacy studies; began Q1 2011.

Rockwell is conducting two pivotal Phase III clinical trials for SFP 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 will comprise 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.

How to participate in the Phase III Study: If you are a clinical investigator and interested in participating in SFP clinical studies or learning more about a clinical study in your area, please fill out the Clinical Trial Request form. If you are a dialysis patient interested in learning more about a clinical study in your area, please contact the physician overseeing your dialysis care.

PRIME Study

PRIME Study ESA sparing; began Q1 2011

The PRIME study is a multi-center, 100-patient, randomized controlled 9-month study with 100 patients receiving either SFP dialysate or standard (iron free) dialysate. IV iron sucrose (Venofer ®) can be dosed in either group per protocol in cases of iron deficiency. Clinical study design is aimed at demonstrating ESA sparing as well as to examine maintenance of hemoglobin, iron parameters, the need for intravenous (IV) iron, and oxidative stress markers in patients receiving SFP. Note: Rockwell has assumed sponsorship of the study, previously funded by the NIH.