My dears, I will remain invested here as well.
PITTSBURGH - Krystal Biotech, Inc (NASDAQ:KRYS) announced Thursday that its experimental gene therapy KB407 successfully delivered and induced expression of the wild-type CFTR protein in the lungs of cystic fibrosis patients in the highest dose cohort of the Phase 1 CORAL-1 trial. The company, which is currently valued at USD 7.2 billion and whose shares are trading near a 52-week high of USD 265.92, is in impressive financial shape with an overall rating of "EXCELLENT" according to data from InvestingPro.
The interim results showed KB407 transduction in all six patients with successful bronchoscopy. The percentage of transduced cells in the conducting airways ranged from 29.4% to 42.1%, regardless of the modulator therapy status or genetic background of the patients.
The highest dose cohort of the study comprised seven patients who received KB407 by inhalation on four consecutive days. Four of these patients were not eligible for modulator therapy due to their specific genetic mutations.
According to the company, KB407 was generally well tolerated, with most adverse events being mild to moderate and transient. One serious adverse event was reported in the form of an asthma exacerbation, but this was attributed to the bronchoscopy procedure and not the treatment.
"The molecular confirmation of the incorporation and expression of the unmodified wild-type CFTR protein in clinically relevant ciliated and secretory cells of the lungs of patients with cystic fibrosis is a tremendous breakthrough," Jorge Lascano, Professor of Medicine at the University of Florida, is quoted as saying in the press release.
The company submitted the design for its pivotal repeat-dose study, CORAL-3, to the FDA in late December. Krystal Biotech expects to coordinate with the FDA on the study design in the first quarter and to begin patient enrollment in the second quarter of 2026.
