During the webcast, SI said a couple of key things that stand out for regulatory approval: - The therapy provides an extra 3-4 years of reduction in mortality (at least) and this is independent of other treatments - He thinks that the mortality result as a secondary endpoint is enough to justify an accelerated approval pathway
How I read this is that CHF goes into the same bucket as GVHD. We have a therapy that works, and has been proven to work in a trial, but hasn't satisfied the regulatory requirement for normal FDA approval. So we go into this bureaucratic process of trying to seek approval for two of our therapies, and if we're knocked back then we're looking at another trial.
Beware the tide of downrampers, desperados and wolves that will post all sorts of stupid stuff today.
What I don't like about this: 1. MSB management in designing the trial hasn't selected the appropriate primary endpoint to get regulatory approval. Some people can blame the FDA and say they are killing poor heart patients, but everyone knows they have a stupid bureaucratic process that has to be followed to gain approval and that's part of doing business as a biotech company. Was this able to be anticipated? I'm not sure it could have been. The phase 2 data certainly showed that MACE events were a good primary endpoint to choose, but there were other indications that were also strong, so it's definitely a very tricky one to assess... but still the people designing the trial have to take responsibility for that if they can't get regulatory approval, even if it was difficult or impossible to anticipate, and they have certainly done that to some degree by having mortality as a secondary endpoint. Read more here: https://www.mesoblast.com/clinical-trial-results/mpc-150-im-phase-2-trial#:~:text=Trial%20Results%3A%20MPC%2D150%2D,III%20(NYHA)%20Chronic%20Heart%20Failure&text=The%20150%20million%20cell%20dose,HF%2DMACE%20long%2Dterm. I wonder if newer treatments on the market between the trial design and conclusion had an impact in the primary result... who knows?
2. The way this news was delivered is somewhat devoid of "so what" information. I recognise that they can't speak for the FDA but they should be clearer on what the news means for approval. There was always a chance (even with a significant result) that CHF would require another phase 3 trial, so expectations could have been more clearly managed in this area both in the release and ahead of the announcement. Mesoblast, as you would expect form a company full of scientists and geniuses, is terrific at developing products and doing the scientific work of running a world leading MSC company, and below average at the communications and investor relations
What I do like about this: 1. The result of 60% reduction in mortality, if it can be repeated in an confirmatory trial is a long-term shareholder dream. The significance of this result can't be overstated, it's exactly what MSB was hoping for in terms of efficacy, or maybe even more. This is a valuable treatment that will make a lot of money when it is commercialised
2. The CHF trial was not certain to gain approval without a second phase 3 trial anyway, so while there is some disappointment in the result, it's probably no different or even somewhat better than a mild result in the primary end point.
Overall, I understand why the share price has dropped today, but it's hovering between a 10-15% drop which is not indicative of a total failure on approval - just look at the GVHD CRL which sent the SP down 30%+
If these kind of SP fluctuations make you feel like you have CHF then I can understand why you might reduce your exposure, but I think today is a significant day on the results front, despite being a disappointing day for prospective approval.
MSB Price at posting:
$3.97 Sentiment: Hold Disclosure: Held
(20min delay)