Ann: Mesoblast Phase 3 Chronic Heart Failure Results, page-373

Absolutely amazing results - the ball wasn't just hit over the fence for 6, but ended up on the roof of the member's stand. You couldn't wish for a better result. The only endpoint that really matters is a reduction in deaths, all the other endpoints are just poor proxies for survival improvement.

The bad news is thanks to some less than stellar trial design the trial missed its primary endpoint. The FDA is going to have to make a very difficult decision on what should have been an easy straightforward approval. If we are very lucky the FDA will approve subject to MSB running a post-approval trial, although I have a bad feeling they will want another Phase III trial first. It is going to be hard for the FDA to approve a completely novel treatment with an unknown mechanism of action off a trial that didn't meet its primary endpoint - when the unknowns start to add up the FDA tends to get risk averse. On the other hand a 60% reduction in deaths is a very, very compelling result that is hard to ignore.

The big issue is while this trial shows that MSCs work, they don't work in the way MSB's management thinks (or at least thought). This is similar to the LVAD trial where the cells worked by preventing gastric bleeding, not directly on the heart. Why do I feel MSB is the Steven Bradbury of the ASX (this is a little unfair on Steve).