Clinical Trial ChallengesThe cystic fibrosis (CF) program is not considered worth progressing towards later stage clinical trials due to lack of efficacy signals.
Efficacy ConcernsThe data failed to demonstrate a meaningful improvement in FEV1/ppFEV1, had a high level of variability between patients, and had a lack of concordance between FEV1 and mucus plug/volume reductions.
Stock ValuationThe value from the CF and OTC deficiency programs has been removed from the valuation, reducing the price target to $12 and downgrading the stock to Neutral.