Osiris Therapeutics last week announced positive data that came out of their Phase I/II double blinded Chondrogen trial. They demonstrated statistically significant improvements in joint pain in patients with osteoarthritis, however this seems to be an about face compared to when they announced their “disappointing” (the CEO’s words) interim 6 month data from this trial. Osiris is getting beat up for this in the media, notably at my favorite stock website TheStreet.com. The articles author, Adam Feuerstein, didn’t like the fact that Osiris cherry picked the data and only announced an analysis from just over half of the patients (and apparently they wouldn’t comment on the other half). Osiris got a similar left hook from an analyst when they announced their cardiac trial results in March (I am still trying to find this reference), for similar reasons.
I, personally, am encouraged by the results, but this demonstrates how early the cell therapy field really is. The trial kind of reminds me of an experiment that I ran in grad school that didn’t turn out as expected (failed hypothesis), but generated some cool data that led to a very interesting paper -in my humble opinion- completely unrelated to the original hypothesis. This Phase I/II Chondrogen trial’s main endpoint was safety, and the II aspect was a ‘let’s look and see if there is efficacy?’ experiment. Osiris and their clinical investigators guessed incorrectly on where they thought they would see a difference (meniscus regeneration – which they state in the 1 year results as being too noisy), but other data points showed some very promising results and will point them in (hopefully) exciting directions. Osiris is now set up to run a full Phase II efficacy trial, or attempt to shorten the regulatory time lines with a less traditional (a.k.a. more risky) Phase II/III trial.
It is important for journalists like Adam Feuerstein to realize that this is not traditional drug development (this was a combined Phase I/II, not a traditional Phase II). You cannot run a traditional Phase I safety study using cells, as you don’t want to test cellular therapy in healthy subjects. This has advantages as you can obtain some (not perfect) efficacy data from these trials – which is what was reported by Osiris. Osiris may have made a mistake by not telling “the truth, the whole truth, and nothing but the truth…” There are obviously people out there with a close eye on this space, and by omission, they will assume there is something to hide. With that said; I don’t think Osiris is ‘hyping’ their story, but full disclosure would be nice so that we can do our own analysis.