Word of Caution About KERX
While checking out Keryx Pharmaceuticals' 10K form, I noticed something that caught my attention. They make the following note under a "Risks" section:
We may also opt to change the delivery method, formulation or dosage which could affect efficacy results for the drug candidate. For example, we have limited clinical experience with our new one gram caplet formulation for Zerenex, and therefore, there is no assurance that this new formulation will be safe and efficacious when assessed in a large and/or long-term clinical trial setting. We used this one gram caplet formulation in our completed Phase 3 short-term study for Zerenex and are currently using it in our ongoing Phase 3 long-term study for Zerenex.
I'm certainly no expert, but I have seen several companies have drug applications turned down by the FDA due to insufficient safety and efficacy data. The company has been one of my best performers in my portfolio, so I do not want to see it all come crashing down because the FDA rules there isn't enough data. To be fair, KERX also states:
In November 2010 we reported positive top-line results from the Phase 3 short-term efficacy study component of our Phase 3 registration program of Zerenex for the treatment of elevated serum phosphorus levels, or hyperphosphatemia, in patients with ESRD on dialysis. In this study, conducted pursuant to a SPA with the FDA, Zerenex met the study’s primary endpoint, described below, demonstrating a highly statistically significant dose response. In addition, key secondary endpoints were also met.
You can read the 10k here. Let me know your thoughts. Does anyone know what dosage capsule they were using prior to the switch to 1g? I have read most of the 10k, but I cannot find it.