Rockwell Medical announced successful top-line results from the long-term CRUISE-2 Phase 3 efficacy study of Soluble Ferric Pyrophosphate (SFP), the company's late-stage investigational iron-delivery drug for the treatment of iron deficiency in chronic kidney disease patients receiving hemodialysis. SFP is an iron compound that is delivered to the hemodialysis patient via dialysate, replacing the 5-7 mg of iron that is lost during a dialysis treatment.
"As we were with CRUISE-1, we are thrilled with the successful outcome of this CRUISE-2 efficacy study," said Rob Chioini, chairman and CEO of Rockwell Medical. "This second Phase 3 study produced identical results to the first in demonstrating statistical significance and meeting the primary efficacy endpoint."
In this study, SFP met the primary endpoint, demonstrating a statistically significant mean change in hemoglobin from baseline to end-of-treatment, according to Rockwell. Additionally, SFP met key secondary endpoints, including maintenance of hemoglobin, maintenance of reticulocyte hemoglobin, and increase in serum iron pre-to-post treatment without an increase in ferritin. This long-term study is the second and final study of two identical Phase 3 efficacy studies to provide clinical data required for the company to file a new drug application with the U.S. Food and Drug Administration.
"We believe that upon FDA approval, SFP will become the new standard of care in iron therapy," said Rob Chioini.
Primary efficacy endpoint
The CRUISE-2 study successfully met its pre-defined primary efficacy endpoint, which was a change in hemoglobin from baseline to end-of-treatment between the SFP and placebo groups. The mean difference between SFP and placebo was 3.6 g/L (95% CI 0.8, 6.3) in favor of SFP, and was statistically significant (p=0.011).
At baseline the two groups had similar hemoglobin levels (109.6 g/L SFP and 109.3 g/L placebo). The mean adjusted change from baseline hemoglobin to the end of the randomized treatment period in the SFP group was -0.5 g/L (95% CI -2.6, 1.7). In placebo there was a statistically significant decline of -4.0 g/L (95% CI -6.2, -1.9).
Key secondary endpoints
The key secondary endpoints at end-of-treatment showed statistically significant differences between the SFP and placebo groups, including the pre-dialysis reticulocyte hemoglobin (CHr) and serum ferritin. CHr, an early index and the best marker of iron-delivery to the bone marrow, was maintained at baseline levels in the SFP group during the randomized treatment, compared to a significant decrease in the placebo group. At End-of-Treatment, the difference between groups was a statistically significant 0.97% difference in favor of SFP (p=0.017). Serum ferritin, a marker of tissue iron stores, declined by 11.6% from baseline in the SFP arm while the placebo group ferritin level declined by 21.7%. The difference between groups was statistically significant (p < 0.001). These results indicate that hemodialysate containing 2 µM SFP iron delivers