ESCRS - PP25.10 - Topical Deleted Hepatocyte Growth Factor Improves Visual Acuity And Corneal Conjunctivalization In Limbal Stem Cell Deficiency

Topical Deleted Hepatocyte Growth Factor Improves Visual Acuity And Corneal Conjunctivalization In Limbal Stem Cell Deficiency

Published 2026 - 44th Congress of the ESCRS

Reference: PP25.10 | Type: Free paper | DOI: 10.82333/5kds-ar62

Authors: Dr John Affeldt* 1 , Dr Francis Price 2 , Dr Oday Alsheikh 3 , Dr Jennifer Nottage 4 , Dr David Hardten 5 , Dr Michael Straiko 6

1Ophthalmology,Loma Linda University,Loma Linda,United States, 2Price Vision Group,Indianapolis,United States, 3Stuart Terry Eye Associates,San Antonio,United States, 4Midwest Cornea Associates, LLC,Indianapolis,United States, 5Minnesota Eye Consultants, P.A.,Minnetonka,United States, 61040 NW22nd Ave, Suite 200,Portland,United States

Purpose

To report interim efficacy outcomes from CSB-C23-001, an open-label study evaluating topical CSB-001 (deleted hepatocyte growth factor (dHGF)) compared with control eyes in subjects with limbal stem cell deficiency (LSCD) and Baseline visual acuity (VA) ≤65 EDTRS letters.

Setting

Prospective multi-center open-label controlled study ongoing at six private and academic clinics in the United States in eyes diagnosed with chronic LSCD and corneal conjunctivalization (CJTV) within 2 mm of the corneal apex. 

Methods

Forty-six LSCD eyes of 37 subjects were enrolled and followed for 20 weeks. Twenty nine eyes of 23 subjects received topical CSB-001 four times daily for two 8-week periods separated by a 4-week Dosing Holiday. Seventeen eyes of 14 subjects served as untreated controls and were observed for 20 weeks. VA was measured in ETDRS letter scores. The area of conjunctivalization (CJTV) was quantified from slit lamp images with fluorescein staining using MATLAB based image analysis.

Results

At Week 20, mean VA change from baseline was +10.9 letters (n=29) in CSB-001 eyes and -4.7 letters in control eyes (n=17). In the CSB-001 group, 41.4% of CSB-001 eyes gained ≥15 letters and 55.2% gained ≥10 letters. No control eye gained ≥15 letters and one eye gained ≥10 letters (5.9%). Mean CJTV change within the central corneal area (7.1 mm2) was -46.7% in CSB-001 eyes gaining ≥ 15 letters and -22.8% in eyes gaining ≥10 letters, and -1.4% in control  eyes. During the 4-week Dosing Holiday, mean VA change in CSB-001 eyes was -1.7 letters with VA improvement resuming upon CSB-001 dosing restart.

Conclusions

Topical CSB-001 demonstrated improvements in visual acuity and reductions in corneal onjunctivalization in eyes with LSCD compared with untreated controls. These findings suggest that dHGF may represent a novel pharmacologic approach for LSCD and support further clinical evaluation.