Krystal Biotech reports Q1 2026 progress on KB803, KB801 and VYJUVEK expansion

Krystal Biotech said enrollment is complete in the registrational IOLITE study of KB803 for the treatment and prevention of corneal abrasions in dystrophic epidermolysis bullosa patients, and said it remains on track for KB803 and KB801 registrational data readouts in 2026. The company also said the FDA granted platform technology designation for KB407 and KB111, while VYJUVEK continued global expansion.

The company said a total of 16 patients have been enrolled in IOLITE, an intra-patient, double-blind, decentralized, placebo-controlled study with crossover design evaluating KB803. The primary efficacy endpoint will be the change in the average number of days per month with corneal abrasion symptoms while receiving KB803 versus placebo, and the company expects to report top-line results in 4Q 2026.

For KB801 for the treatment of neurotrophic keratitis, the company said it continues to enroll in EMERALD-1, a registrational, 1:1 randomized, double-masked, multicenter, placebo-controlled study. The company expects to complete full enrollment of 60 patients in EMERALD-1 and announce data before year end.

For KB407 for the treatment of cystic fibrosis, the company said the FDA granted platform technology designation to the genetically modified, non-replicating herpes simplex virus type 1 viral vector used in the program. Based on interactions with the FDA, the company is initiating an open-label, single-arm study to evaluate safety of repeat dose KB407 for 24 weeks in five patients with cystic fibrosis who are ineligible for, do not tolerate, or do not benefit from modulator therapy, with dosing expected to start later this month, enrollment expected to be complete in 2Q 2026, and results expected before year end.

In January 2026, the company announced a positive clinical update from the highest dose cohort of CORAL-1, confirming lung delivery and expression of wild-type cystic fibrosis transmembrane conductance regulator protein following inhaled administration of KB407. Airway cell transduction by KB407 was observed in all patients with successful bronchoscopies, with broad airway distribution and transduction as assessed by CFTR or viral marker immunofluorescence ranging from 29.4% to 42.1% of conducting airway cells, and inhaled KB407 continued to be well tolerated at the highest dose.

For KB408 for alpha-1 antitrypsin deficiency lung disease, the company said it continues to enroll patients in repeat-dose Cohort 2B of SERPENTINE-1 and expects to report interim data for this cohort in 2026. Cohort 2B is designed to evaluate the safety and tolerability of repeat KB408 dosing at the same dose level that was previously shown to safely deliver SERPINA1 to the lungs of patients after a single dose.

For KB111 for the treatment of Hailey-Hailey disease, the company said the FDA also granted platform technology designation to the viral vector used in the program, providing the same regulatory efficiencies available for KB801 and KB407. The company said it expects to complete development and validation of an HHD-specific severity scale in 1H 2026, initiate HALITE-1 later this month in approximately seven patients, report HALITE-1 results in 2H 2026, and submit the results along with the registrational study design for discussions with the FDA in 2H 2026 to enable a potential registrational study start in 2027.

In oncology, the company said the FDA granted RMAT to KB707 for the treatment of advanced non-small cell lung cancer in 2025. The company also said it is enrolling patients with advanced non-small cell lung cancer in a dose expansion cohort of KYANITE-1 evaluating inhaled KB707 in combination with chemotherapy.

For VYJUVEK, the company recorded $107.1 million in net product revenue for the fourth quarter of 2025 and $389.1 million for the full year of 2025, with gross margin of 94% for both periods. The company said it has secured over 660 reimbursement approvals in the United States, estimated that over 90 patients had been prescribed VYJUVEK therapy across Germany, France and Japan, and later estimated that over 140 patients overseas were in active stages of access or treatment. The company said it remains on track to expand its specialty distributor network to cover over 40 countries by the end of 2026.