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

• Krystal Biotech Inc (NASDAQ: 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.

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