Krystal Biotech (KRYS) Study Result summary

Program and Study Design

• KB301 is a gene therapy delivering COL3A1 to restore type III collagen and rejuvenate skin, evaluated in the multicenter, open-label PEARL-I study for dynamic wrinkles of the décolleté and lateral canthal lines in adults up to 75 years.

• Both microneedling and standard syringe injection techniques were assessed, with three weekly treatments and follow-up at one and two months.

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

• Key objectives included safety and preliminary efficacy, assessed by investigators and subjects using GAIS and Subject Satisfaction Questionnaire.

• Subjects had no recent skin treatments in target areas.

Efficacy and Durability Results

• KB301 showed clinically meaningful improvements in both décolleté and lateral canthal lines, with 94% of décolleté subjects and 75% of lateral canthal subjects achieving at least a one-point GAIS improvement at two months.

• 28% of décolleté subjects and 50% of lateral canthal subjects achieved a two-point GAIS improvement at two months.

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

• Subject satisfaction was high, with 94% of décolleté subjects reporting increased satisfaction at two months.

• Durability data showed sustained efficacy and satisfaction for at least 7-9 months post-treatment in earlier cohorts.

Safety and Tolerability

• KB301 was generally well tolerated, with most adverse events being mild or moderate, including swelling, redness, itching, pain, and transient injection site reactions.

• No drug-related serious adverse events were reported, and no increase in adverse events with repeat dosing.

• Few systemic adverse events were reported, such as headache, chills, and muscle aches.

• Safety profile was consistent with other injectable aesthetic products.