"The Phase 3 study, conducted in Japan, was an open-label, randomized study evaluating the efficacy and safety of ferric citrate against an active control, sevelamer hydrochloride, over 12 weeks in hemodialysis patients with hyperphosphatemia. In the top-line results, which evaluated the change of serum phosphorus from baseline, the primary endpoint of efficacy met non-inferiority to sevelamer hydrochloride. Furthermore, there were no clinically significant findings on safety and tolerability of ferric citrate within the treatment period."
Very important paragraph. Essentially what we have read is that the study was conducted and designed to evaluate efficacy and safety. The study successfully demonstrated efficacy of the drug, but the study failed to demonstrate anything about safety. No significant findings regarding safety could be made despite having designed the study specifically to demonstrate something about safety, good or bad, instead it could demonstrate nothing. This is not a good thing. It is actually worrisome for the researchers because it adds concern to whether or not their study design is even capable of demonstrating safety.
Our concern regarding efficacy and safety studies is two-fold in nature: 1) First - The study must be capable of producing significant results (good or bad), in other words the study itself must be significant in that it must have a scientific and logically sound basis and structure, which will effect the significance of the results and thus the total significance study itself. Essentially bad assumptions or weak assumptions can render the study ineffective at PROVING anything, in other words the study-design intended to address safety could be inherently flawed.
2) Second - The study hopefully produces good results, or so-called "top-line results." Note, in this Japanese partner's study, part of the study regarding safety must have been flawed by design or there may unknown mechanics regarding the drug. More information may be needed before a successful safety study can be designed, nevermind conducted.
So that said, if anyone remembers the run-up to Perifosine, this run-up for Zerenex is JUST as suspect...For the reason I just mentioned. Lots of "hype" being produced by the company when they are simply misleading you with their tone of voice and/or verbiage.