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Krystal Biotech Announces First Patient Dosed in Phase 3 Clinical Trial of KB803 for the Treatment and Prevention of Corneal Abrasions in Patients with Dystrophic Epidermolysis Bullosa
Intra-patient, double-blind, multicenter, placebo-controlled study with crossover design Repeat administration under compassionate use was previously shown to
About this update from Krystal Biotech, Inc.
[{"type":"text","content":"Intra-patient, double-blind, multicenter, placebo-controlled study with crossover design Repeat administration under compassionate use was previously shown to be well tolerated and associated with full corneal healing by 3 months as well as significant visual acuity improvement from hand motion to 20/25 at 8 months PITTSBURGH, June 24, 2025 (GLOBE NEWSWIRE) -- Krystal Biotech, Inc. (the “Company”) (NASDAQ: KRYS) announced today that the first patient has been dosed in its Phase 3 clinical trial (“IOLITE”), an intra-patient, double-blind, multicenter, placebo-controlled study with crossover design evaluating KB803 for the treatment and prevention of corneal abrasions in dystrophic epidermolysis bullosa (DEB) patients. KB803 is designed to deliver two copies of the COL7A1 transgene to the corneal epithelium and enable local type VII collagen production in the front of the eye. “The initiation of IOLITE is another important step for Krystal as we work tirelessly to treat DEB as comprehensively as possible,” said Suma Krishnan, President, Research & Development, Krystal Biotech, Inc. “With dramatic and durable improvements already reported for the patient treated under compassionate use, we are excited by the potential of KB803 to restore full eye function and reduce or eliminate the otherwise persistent threat of vision loss imposed by these recurring corneal abrasions.” Over 25% of DEB patients are thought to suffer from ocular complications of the disease, including over 50% of patients with the recessive form of DEB (RDEB). The estimated number of DEB patients affected exceeds 750 in the United States and 2,000 worldwide. There are no corrective therapies available to the treatment or prevention of ocular complications associated with DEB. Current disease management is limited to supportive wound care and in some cases surgical interventions to remove scar tissue. IOLITE Phase 3 Study IOLITE is an intra-patient, double-blind, placebo-controlled, multicenter Phase 3 study with a crossover design to evaluate KB803, administered as an eye drop, for the treatment and prevention of corneal abrasions in DEB patients, 6 months of age or older. Patients seeking to participate in IOLITE must first enroll in the ongoing natural history study and complete a 12-week run-in period, during which they report the number of days that they experi...