RE: RE: ApoA1 Study Posted by BFW CSL-112 is the 2nd re-tooling of this formulation. There are a few generally accepted PC's for complexing that have similar half lifes. They were using human apoa-1 mined through plasma delipidation. I am not sure if they are recombinantly expressing now or not. This could also impact the ability to compare to RVX.
However, its very difficult to compare pre-beta particles formulated with PC to endogenous apoa-1. As seen from the Torcetrapib data, its not about increasing HDL but may be as much about the quality of HDL. The phospholpid fraction, number of proteins per particle, apoa-1 subparticles, size heterogeneity etc.. There are so many variables that its really not comparable to say that positive or negative rHDL will have any bearing on RVX. The question for RVX is what type of particles the apoa-1 forms and whether a slower, but more continuous release will have similar functions to larger concentration infusions. HDL has so many functions. RCT gets all the attention, but there are many other functions. I still see this drug more as an atherogenics play where the real benefit is capitalizing on the potential anti-inflammatory properties of apoa-1 versus its ability to promote cholesterol efflux. Keep in mind, RCT is great for presentations and press releases, but FDA approval will require an outcomes based trial. Meaning they will have to show an improvement in events. They need to show plaque regression to get the funding for an outcomes trial. So yes, it is the next step, but plaque regression is irrelevant if it has no impact on outcomes.
After Pfizer and Roche pulling out of the HDL market, I think most news like this is generally good though for RVX as the general public equates them. The risk is that this approach is different enough that it will stand or fall on its own merits.