Krystal Biotech (NasdaqGS:KRYS) Earnings Call Presentation

CORAL-1 Clinical Data Update - Krystal Biotech's KB407 is a gene therapy for cystic fibrosis (CF) and has the potential to become a leadership opportunity in the treatment of CF[9] - The company plans to initiate the CORAL-3 study in the first half of 2026 to evaluate the safety and efficacy of repeat KB407 administration, which could support registration of KB407[4, 93] - The company estimates a $2 billion+ market opportunity in modulator-ineligible or refractory CF patients[12] KB407 Phase 1 CORAL-1 Highest Dose Cohort Results - In patient 03-01 with CFTR genotype 2184delA/W1282X, 42.1% of conducting airway cells expressed CFTR[55] - In patient 03-02 with CFTR genotype R553X/M1V, 29.4% of conducting airway cells expressed CFTR[68] - In patient 03-03 with CFTR genotype C1210-12T/1408A>G, 36.5% of conducting airway cells were viral marker positive[76] - In patient 03-04 with CFTR genotype R334W/R1162X, 33.8% of conducting airway cells were viral marker positive[79] - In patient 03-05 with CFTR genotype F508del/F508del, 36.8% of conducting airway cells were viral marker positive[81] - In patient 03-06 with CFTR genotype F508del/F508del, 31.4% of conducting airway cells were viral marker positive[83]