CLDN:
Share price obviously depends on your view of the likely outcome. Perhaps the only reasonable point I can make is that some caution is warranted if the outcome is "positive." The joint frailty model is not the same as an all cause mortality endpoint, so I think risk exists even with a positive outcome. In Cupid-1, the joint frailty model gave statistical significance for the high dose group, but after 3 years of follow-up, mortality did not. The FDA may require additional data.
Regarding AAV persistence and the next step: I doubt they'd return to animal models. The company does have a higher dose trial ongoing at 2.5e13 viral genomes, which suggests that they have their own internal curiosities about dosing and benefit. Only they can answer whether that curiosity is based on a nagging concern or confidence in seeing an increased benefit. My bet would be on the former.
As for Cupid-1's high dose arm being a false positive, I can't make a meaningful quantitative argument. It's more a gut feeling based on the preclinical data, and the fact that I've sat through many presentations describing trials with large effects in tiny CV cohorts. Acquired heart failure is a relatively heterogenous population, and many of the classifications are still based on symptomatic findings. For that reason, I would be cautious of depending on the outcome of a 23 patient study.
