I think the probability that at least a subset of patients will be helped is quite high. Why has Stephen Hawking survived 48 years after an ALS variant diagnosis? The FDA has approved quite a few drugs treating rare conditions with only modest efficacy.
I must agree with your comments and knowledge of this matter, unfortunately for us longs the company is very vulnerable at this stage in time where big pharmas are desperate to enter new fields in science given the strong approach some of their produts are of being substituded by similars due to expiring patents. Big pharmas are swimming in cash and they are on the hunt for small to medium size bios where they can recover future lost revenues from their old meds. We won´t live to see the results CYTK has to offer from their breaktrough revolutionary muscle science in OM or tirasemtiv because the company could be sold out to either Amgen or Biogen anytime now IMO. I could welcome a $4-5 deal instead of a partership for tirasemtiv.
The Home Run would be if Tirasemtiv outperformed placebo enough in the current trial for ALL participants to be transferred to Tirasemtiv, because of ethical considerations. It doesn't happen often in trials, but if it did CYTK stock would become a multibagger.