Krystal Biotech (KRYS) Study Result summary

Program Overview and Technology

• KB301 is a gene therapy candidate delivering COL3A1 via a non-integrating HSV-1 vector to restore type III collagen, targeting skin rejuvenation and reversing age-related collagen decline.

• The platform enables delivery of multiple genes, aiming to rebuild skin structure and improve appearance, and supports future pipeline expansion targeting other extracellular matrix proteins.

• Jeune Aesthetics, a subsidiary of Krystal Biotech, is focused on clinical development, leveraging Krystal's manufacturing and regulatory expertise.

Study Design and Objectives

• KB301 was evaluated in PEARL-I, a multicenter, open-label, split-body study for dynamic wrinkles of the décolleté and lateral canthal lines at rest in adults up to 75 years, with no recent skin treatments in target areas.

• Key objectives included safety and preliminary efficacy, assessed by both investigators and subjects using GAIS and SSQ.

• Cohorts III and IV focused on lateral canthal lines at rest and dynamic wrinkles of the décolleté, both areas lacking approved injectables.

• Dosing regimens included both low and high dose KB301, with various administration techniques explored.

Efficacy and Durability Results

• KB301 showed clinically meaningful improvements: in the décolleté, 94% of subjects had at least a one-point improvement at two months, with 28% achieving a two-point gain; in lateral canthal lines, over 90% improved by at least one GAIS point at one month, and 75% at two months.

• 100% of subjects in both cohorts were responders by GAIS at two months in some studies, with improvements increasing over time.

• Improvements extended to skin texture, hydration, radiance, elasticity, and crepiness, with investigator-reported improvements in up to 94% of décolleté subjects.

• Subject satisfaction scores remained elevated for at least 7-9 months after last dose in earlier cohorts, with durability studies showing sustained efficacy up to nine months.

• Before-and-after images demonstrated visible reduction in wrinkles and improved skin appearance.