Krystal Biotech (KRYS) Guggenheim Securities Inaugural Healthcare Innovation Conference summary

Pipeline and clinical development updates

• Data from cohorts one and two of the Alpha-1 antitrypsin program expected by year-end, focusing on safety, delivery, and efficacy in the lung.

• Bronchoscopy was added to cohort two due to site and patient willingness, providing deeper insights for decision-making.

• Decision to proceed to cohort three or redosing will depend on comprehensive biomarker and safety data, with 0.5–1 micromolar lung levels as a key threshold.

• No FDA discussions yet; redosing and biomarker data collection are prerequisites for accelerated approval talks, targeted for late 2025.

• Success in AAT program would de-risk the CF and immuno-oncology lung programs, supporting broader pulmonary franchise ambitions.

Oncology and other pipeline programs

• Biomarker data for the oncology program in lung and skin expected by year-end; focus remains on safety and immune profiling.

• Expansion cohort underway, with narrowed tumor types based on preliminary safety and efficacy signals.

• Dose and tumor type selection driven by early signals, though sample sizes remain small for definitive conclusions.

• Ongoing discussions with KOLs will determine the extent of efficacy data to be released.

Commercial performance and patient access

• Approximately 460 patients currently on reimbursed therapy, with a goal of 720 by mid-2025, representing 60% market share.

• Patient identification efforts include phenotypic matching and expanded claims data analysis to reach untapped populations.

• Compliance rates are in the mid-80% range, well above initial projections, with strong patient and physician satisfaction.

• Home dosing has been a key differentiator, with plans to expand this model to other formulations and indications.