Objective Acuity announces successful pilot study in children with CLN2 Batten disease

Objective Acuity today announced the completion of a pilot study using OAL's threshold visual acuity test for young children diagnosed with late-infantile neuronal ceroid lipofuscinosis Type 2 (CLN2 disease), a form of Batten disease.

A prominent feature of CLN2 disease is vision loss, which typically develops when cognitive, motor and language impairments are already present. Measurement of visual acuity of those with the disease using standard methods is difficult and unreliable due to the age of those affected and their cognitive, language, and motor impairments.

Pursuant to an agreement between OAL and REGENXBIO Inc., a leading clinical-stage biotechnology company seeking to improve lives through the curative potential of gene therapy, a pilot study was conducted at Universitätsklinikum Hamburg-Eppendorf (UKE) in Hamburg, Germany, using OAL's objective optokinetic nystagmus (OKN) detection technology to determine its usefulness in monitoring children with CLN2 disease.

OAL's technology uses a camera to measure the movement of each eye while the child looks at a screen displaying a "drifting stimulus" pattern. Data from the camera is used by OAL's proprietary algorithms to detect OKN, an involuntary motion of the eye that typically occurs as a reflex response to the visual stimuli.

Visual acuity read-outs using OAL's technology were compared to measurements of central retinal thickness (CRT) obtained under anaesthesia. The results of the pilot study demonstrate a high correlation between visual acuity results determined using OAL's technology and the CRT measurements. Therefore, determining visual acuity results using OAL's technology could potentially be used as a significantly less subjective method for measuring visual acuity of those with CLN2 disease. Further details were presented by REGENXBIO at the ARVO 2021 Annual Meeting in a presentation titled "Pilot Study of Novel Optokinetic Nystagmus-Based Visual Acuity Test in Children with CLN2 Disease."

As a result of the successful pilot study, REGENXBIO has signed an agreement with OAL to use OAL's technology in future studies for the development of RGX-381, REGENXBIO's gene therapy candidate to treat ocular manifestations of CLN2 disease.

Christina Ohnsman, M.D., Senior Clinical Development Lead of REGENXBIO, who presented the work at ARVO, said: "We are pleased with the results of this study.  The objective visual acuity measurement obtained using OAL's technology may allow REGENXBIO to obtain further data on the range, severity and impact of visual impairment in patients with CLN2 disease as we advance our gene therapy candidate to treat ocular manifestations of the disease."

Adam Podmore, Chief Executive Officer of OAL, said: "It is exciting to be working with REGENXBIO and to have OAL's technology potentially be deployed in REGENXBIO's studies."

About Objective Acuity ("OAL")

OAL aims to develop objective vision tests for children and adults. The tests do not require children to provide a response, unlike existing picture or letter chart tests. We are developing two tests, a vision screening test for young children displayed on an iPad Pro and a threshold visual acuity test.

OAL's patented technology involves a "drifting stimulus". The camera measures movement of the eyes and OAL's proprietary algorithms use data from the camera to detect optokinetic nystagmus (OKN). OKN is an involuntary sawtooth motion of the eye that typically occurs as a reflex response to the visual stimuli. The test is 100% objective and the system indicates the presence or absence of an OKN response. Intuitively, the presence of OKN indicates that the child can see the drifting stimulus, while the absence of OKN indicates the opposite.