Clinical Trial ConcernsThere is a debate on the primary endpoint for the approval of the gene therapy, which could affect the regulatory outcome and stock volatility.
Regulatory ChallengesFollowing FDA/EMA discussions, Phase 3 is expected to be placebo-controlled, evaluating changes in FEV1, quality of life and frequency of pulmonary exacerbations across 60-80 modulator-ineligible/intolerant CF patients - expected to initiate 2H25.
Safety And Efficacy IssuesNo new safety events were identified, and previously reported cases of atypical hemolytic uremic syndrome (aHUS) remain a concern.