Claris Biotherapeutics Reports Positive CSB-001 Data in LSCD Clinical Trial
News related to:Claris Biotherapeutics · 2 min read
SAN FRANCISCO, Oct. 11, 2026 /CourierPR/ -- Claris Biotherapeutics has announced positive results from a clinical proof-of-concept study evaluating its CSB-001 (oremepermin alfa ophthalmic solution) in patients with limbal stem cell deficiency (LSCD), a rare, sight-threatening disease. The study, presented at the American Academy of Ophthalmology (AAO) 2026 Annual Meeting, showed that 41.4% of CSB-001-treated eyes gained 15 or more letters in visual acuity, compared to none in the non-treatment arm. Additionally, 55.2% of CSB-001-treated eyes gained 10 or more letters, compared to just 5% in the non-treatment group.
The data, from an open-label proof-of-concept study (NCT06452316) involving 49 LSCD eyes from 39 subjects, demonstrated durable responses. At Week 20, 50% of treated eyes maintained a 15-letter improvement, and 81% maintained a 10-letter improvement for 16 weeks post-treatment. These findings suggest that CSB-001 could provide meaningful vision improvements for patients with LSCD.
Limbal stem cell deficiency is a disease where specialized epithelial stem cells that continuously replenish and repair the corneal surface are dysfunctional or deficient, leading to severe vision loss or blindness. There are no approved pharmacological treatments for LSCD. CSB-001, containing recombinant human deleted hepatocyte growth factor (dHGF) as its active ingredient, aims to promote corneal epithelial regeneration while modulating inflammation, inhibiting fibrosis, and promoting cell survival.
Claris Biotherapeutics plans to initiate two pivotal studies in the first half of 2027, evaluating the safety and efficacy of CSB-001 compared to a vehicle in a total of approximately 400 LSCD subjects. The primary efficacy endpoint will be visual acuity, supported by anatomical endpoints. The company believes that if approved, CSB-001 could become the first pharmacologic treatment for LSCD, providing an efficacious, safe, accessible, and scalable non-surgical treatment option.
According to estimates, at least 30,000 LSCD patients in the U.S. are actively being managed by an eye care professional, with at least 40% of the patients having bilateral disease. This underscores the potential impact of CSB-001 on the lives of many patients who currently have limited or no treatment options.