Krystal Biotech (KRYS) investor relations material

Commercial performance and growth drivers

• Q4 saw a return to growth for VYJUVEK, supported by U.S. sales force expansion and a label update enabling at-home self-dosing, with the latter expected to drive medium- to long-term compliance improvements.

• Reimbursement approvals have surpassed 660, growing by over 45 per quarter, with a near-term goal of reaching 720.

• Rest of world (ROW) sales, especially in Germany, France, and Japan, are anticipated as the primary growth driver for 2026, with launches exceeding expectations and pricing negotiations ongoing.

• U.S. growth is influenced by the start-stop regimen as patients heal, with stabilization expected in 2026 and a shift toward moderate and mild patient uptake.

• Self-launches are planned for the UK and Japan, while distribution agreements are pursued elsewhere; pricing negotiations in Germany are expected to conclude in Q3, with France, UK, and Spain following in 2025.

Pipeline and clinical development updates

• Ocular program studies (KB803 in ocular DEB and KB801 in NK) increased dosing frequency to support at-home self-dosing, requiring re-enrollment of pivotal trials.

• Human factors and safety studies were conducted to ensure safe home administration, leading to protocol adjustments and endpoint agreements with the FDA.

• Phase III re-enrollment for ocular DEB is expected to complete in the first half of the year, with top-line data targeted by year-end.

• KB801 in NK also shifted to daily dosing for operational simplicity and compliance, with over 30 mostly academic sites now active for the pivotal trial.

• Both pivotal studies are placebo-controlled, with statistical endpoints fixed; detailed study design will be disclosed after enrollment completion.

Cystic fibrosis and other pipeline highlights

• Molecular correction data for the CF program has generated strong enthusiasm from scientific partners and the FDA, with ongoing positive regulatory discussions.

• The FDA acknowledged molecular correction and functional CFTR expression in vitro, but clinical efficacy endpoints remain under discussion.

• The CF pivotal trial is expected to require six months of chronic administration to meet regulatory standards.

• Among other programs, Hailey-Hailey is seen as the fastest path to approval, while KB707 is considered the most compelling pipeline asset.