drbio45,
are they getting better because they are on the drug or because they know they are on the drug
Yes Photosensitivity and GI issues likely make those who are on drug know they are. "getting better" is defined as slower decline in Forced Vital Capacity in InterMune's CAPACITY trials. My understanding is this is a measure of expiration from the lung that measures both volume and velocity. With several studies now measuring this (including Placebo controlled studies), I believe it has been validated as a clinically meaningful end-point for IPF. Pirfenidone will have been tested in close to 1000 patients (in IPF alone) with the completion of the CAPACITY study. In addition to the Shionogi study showing positive results, there were 2 prior Placebo phase 2 studies that were stopped for efficacy in the Pirfenidone arm. I think it unlikely that all 3 of these Placebo controlled studies favored pirfenidone because the patients new they were taking the drug (the Phase 2 IPF secondary to HPS were multi-year studies too).
You've posted a couple times directly/indirectly on Pipex. I was wondering if you could answer something for me on Coprexa. Why do you see it is a better drug for IPF? It seems to me it is VERY premature to suggest that?
My thinking on it goes like this...
I looked at Pipex (back when they were Sheffield Pharma) and I had some thoughts on why I didn't see Coprexa as viable in the near term for IPF. My understanding is The Phase 1/2 trial they will announce results for shortly is 20 patients and was open label and only measuring safety. I would think that is unlikely to give them enough direction to go into a Phase 3 trial. Further more marketing the drug for Wilsons doesn't seem viable, so I wouldn't count on off label use (plus with just 20 patients I can't see it getting much use even if it were available). The existing therapies are relatively cheap and the very rare nature of the disease does not seem to lend itself to justifying building a mfg/sales infrastructure. Furthermore Pipex doesn't have the financial resources to do. To me the best bet for Pipex is to get a sponsor for a larger trial (in IPF) to really see if this has some therapeutic benefit there. I think it puts them behind several other therapies being investigatetd.
The preclincal work with the drug showing anti-inflamatory effects if I recall were with pretreatments before doxorubicin. I think this is a stretch to go from mice to humans and preventing something to reversing/stopping progression in a disease that is not well understood and likely has many different sub-types.
Don't get me wrong, I haven't ruled out at some point looking to invest in Pipex (under the right circumstances). I think IPF will be treated in combination and perhaps some of its anti-inflamatory effects may be of benefit to patients with IPF which would be synergistic with Pirfenidone.
AI
