I've researched this stock but decided to pass playing on it. I thoroughly agree success could quickly lead to a $50m-$100m enterprise value but here's the problem ...
This trial has 2 co-primary end-points : signs & symptoms of dry eye versus placebo. ISTA Pharmaceutical failed a Phase III dry eye trial on 28 July 2011. It too had 2 co-primary end-points : signs (measured by dye staining - like Regenerx) & symptoms (measured by patient questionnaire - like Regenerx) of dry eye. ISTA showed <5% for the primary end-points versus baseline but only signs had p<5% when compared to placebo (a saline eye-drop solution). The failure on symptoms versus placebo cost ISTA $73m in market capitalisation on result's day.
The ISTA failure to beat placebo on symptoms I think had 2 causes :
* Patient symptoms of dry eye are all pain-related and placebos are notorious for affecting pain related end-points * Several commentators on the result felt the saline solution did actually relieve discomfort in the eye and so has a real (but non-disease modifying) therapeutic impact.
I would note that RGN-259 did show decent sign (i.e. eye staining) efficacy in animal pre-clinical tests but animals aren't great at filling out discomfort questionnaires so there's no evidence for efficacy on that score.
Putting all this together, I think they have a fighting chance of passing on the signs end-point but will fail on the symptoms end-point (versus placebo). Given they are co-endpoints, that's a trial failure. Bear in mind ISTA had an SPA for their Phase III so the FDA want both signs & symptoms efficacy.
It is possible getting a result on the sign end-point and/or spinning a yarn about getting p<5% versus baseline rather than placebo could lead to a temporary spike on results day - maybe a 2-3 hour blip.
In my view, the only real thing going for this trial is management have been very specific on the catalyst timeline. This may lead to a share price run-up & I have retained the stock on my watchlist for this reason - and this reason alone.
I'll review the ISTA trial design. I believe they used a different staining system measure, and I think they used a different discomfort measure.
I do not see in RGRX's design that the placebo is "saline solution". So why do you com[are it tp ISAT.
As to the discomfprt index? It is a secondary endpoingt. However all you do is talk about the animals tests and they cannot tell discomfort. but if you REALLY have followed RGRX as you say you have...then ypu totally ignore that RGRX has already done a few human tests already including one FDA bles Compasionate Use IND. In the small tests, TB 4 got a 70% response rate and a 50% cure rate. And if you read closer, it was repotrted that all the pts who did not get a good response rate.....all reported that theoir discomfort rate declined. So you are wrong on that. I suggest to go back and read all the human results mpre carefully.
Also the ISTA trial used a gren dye and not the industry standard orange flourscein (sp)....Then ISTA used a 12 point measure for ocular discomfort . RGRX trial is using a 6 point discomfort index.
I do not know what the placebo is. Saline or not. But I do see that ISTA did teh Phase 3, and ORA Inc did not. Ora is the indsutry standard. They are using standar staining and a 6 point discomfort index, not 12.