The results do look reasonably good in a first pass comparison comparison to historicals (my eyeball estimate is a median event free survival of at least 12 to 15 months - vs historical of 10-ish). But the caveats are:
a) there aren't many historical compares using genetic definitions of SMA type.
b) SMA endpoint is particularly sensitive to protocol - e.g. there has been a movement to do heroic efforts to keep kids off ventilation (ISIS trial is off permanent ventilation and alive) and it does seem to work. Although it may not prolong survival?
Within those caveats there are oddities that require explanation (or balancing if comparing to historicals):
A) All of the events were in those who were first treated at less than or equal to 5 months old. 7 out of 9 of those patients evented. None of the patients first treated after 5 months of age (11 patients) have yet had an event. My suspicion is that the age of first treatment is a proxy for age at diagnosis (i.e. more severe disease) or how long they survived without treatment. In either case even a slight imbalance in this between the historical compare and the ISIS trial would completely invalidate the comparison.
So - in conclusion. Interesting results - but within the context of the large noise that the confounders seem to be able to bring it is nowhere near conclusive. The CEO is completely correct to be waiting for the RCTs.
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