Business
Krystal Biotech Announces Positive Interim Results from Phase 1/2 Clinical Trial of KB105 in Patients with TGM1-related Autosomal Recessive Congenital Ichthyosis (ARCI)
KB105 was well tolerated with no adverse events or immune response following redosing Clearly detectable TGM-1 expression in all treated areas following
About this update from Krystal Biotech, Inc.
[{"type":"text","content":"KB105 was well tolerated with no adverse events or immune response following redosing\n Clearly detectable TGM-1 expression in all treated areas following initial and repeat administration. KB105-expressed TGM1 was correctly localized in the epidermis, co-localizing with loricrin, and was functionally active Phenotypic evaluation of KB105 treated areas showed reduced reversion to ichthyotic scaling phenotype Amy Paller, M.D., Professor and Chair of Dermatology at Northwestern University shares detailed results at the Society for Investigative Dermatology (SID) annual meeting today Phase 2 study in 4-6 pediatric patients anticipated to initiate in the second half of 2020 PITTSBURGH, May 14, 2020 (GLOBE NEWSWIRE) -- Krystal Biotech, Inc. (Nasdaq:KRYS), a gene therapy company dedicated to developing transformative medicines to treat diseases caused by protein or gene dysfunction, today announced interim results for a first-in-human Phase 1/2 placebo controlled clinical trial evaluating topical administration of KB105 in patients with autosomal recessive congenital ichthyosis (ARCI) being presented at the Society for Investigative Dermatology (SID) annual meeting. Following today’s results, Krystal plans to enroll pediatric patients in the Phase 2 portion of the clinical trial. “Topical administration of KB105 affords a simple painless way to treat the causative defect in patients suffering from TGM1-deficient ARCI, a debilitating skin disease in which patients have disfiguring generalized scaling and poor skin barrier function, putting them at increased risk for superficial infections caused by bacteria and fungi,” said Amy Paller, M.D., Walter J. Hamlin Professor and Chair of Dermatology and Professor of Pediatrics at Northwestern University, Feinberg School of Medicine and principal investigator of the KB105 Phase 1/2 clinical trial. “These early data support and warrant continued evaluation of KB105, including extension to pediatric patients and optimization of dose and dosing regimen.” Pooja Agarwal, Ph.D., Senior Vice President of product development at Krystal Biotech added, “These results are an early positive signal that KB105 has the potential to provide a meaningful benefit to ARCI patients and supports the planned expansion of our other pipeline programs based on our platform.” KB105 Phase 1/2 Interim Clinical Trial Resu...