Krystal Biotech's KB105 Shows Detectable, Functionally Active TGM-1 Enzyme In Genetic Skin Condition

Krystal Biotech Inc KRYS announced updated results from the Phase 1/2 trial evaluating topical administration of KB105 in patients with autosomal recessive congenital ichthyosis (ARCI) associated with mutations in the TGM1 gene.
Data showed that repeat doses of KB105 were well-tolerated with no adverse events and no evidence of immune response, systemically or at the application sites.
No vector shedding was detected in swabs, blood, or urine in all three patients.
KB105 treatment restored functional TGM1 protein expression and activity in all treated sites.
KB105-expressed TGM1 was correctly localized in the epidermis, colocalizing with Loricrin, and was functionally active.
TGM-1 turnover was observed to be variable and rapid, and pharmacokinetic data suggested that the optimal dosing frequency may be more frequent.
Small areas limited phenotypic evaluation, but KB105 treated areas showed reduced reversion to ichthyotic scaling phenotype.
No drug-related adverse effects were reported.
The company plans to include pediatric patients in 2022.
See the data slide presentation here.
Price Action: KRYS shares closed at $71.77 on Thursday.