Two further adult patients for Krystal Biotech DEB trial

Dystrophic epidermolysis bullosa is a rare, incurable blistering condition, caused by mutations in the COL7A gene encoding type VII collagen, and can be fatal. Krystal Biotech has engineered KB103, a viral vector to deliver functional human COL7A1 genes directly to the patients’ dividing and non-dividing skin cells. The interim KB103 data results on two patients met all primary efficacy and safety endpoints in topically administered KB103 wounds. The Phase I/II trial is recruiting two more adult patients in addition to the original four patients, with the final results expected in the first half of 2019, with a view to a pivotal phase III study starting in the second half of 2019.
Two adult patients aged 28 and 35, and two patients five years old and above will take part in the open-label Phase II part of the study, following conversations with the FDA on enrolling paediatric patients. Once the full data set for the current Phase I/II is released, Krystal Biotech is also contemplating human trials on dystrophic epidermolysis bullosa affecting the eyes, using KB103 eye drops, and a study on patients with fused hands.