Life Biosciences reports first human data for ER-100: three participants with glaucoma received the low dose, and two showed improvements on visual field testing
Life Biosciences reports first human data for ER-100: three participants with glaucoma received the low dose, and two showed improvements on visual field testing
On October 8, the company released interim phase one data: through day 56, three participants with open-angle glaucoma tolerated the low dose without serious complications or dose-limiting reactions. The data monitoring committee recommended proceeding to the next dose level.
In its October 8 press release, Life Biosciences also reported visual field test results. In two of the three participants, at least 28% of the points tested on the Humphrey visual field test improved by more than 4 decibels on day 56. During this test, a person keeps their gaze fixed on a single point and indicates when they see flashes of light in different parts of their visual field.
ER-100 is a gene therapy administered to one eye. A viral vector delivers instructions for three proteins, OCT4, SOX2 and KLF4, collectively called OSK. After the injection into the eye, participants take doxycycline for eight weeks to activate production of these proteins. The treatment targets retinal ganglion cells, which transmit visual signals from the eye to the brain. Glaucoma damages both these cells and their axons.
This approach was chosen following a 2020 study in mice. In a glaucoma model, the researchers first used microparticles to raise pressure inside the eye and waited until the numbers of retinal ganglion cells and their axons had declined. They then administered a viral vector carrying OSK. In mice with OSK activated, axon density returned to the level seen in eyes without glaucoma, visual acuity increased, and the retina’s electrical response improved. This experiment tested whether controlled activation of OSK could change the condition of cells after damage had occurred.
The trial registry describes the human study as open-label, with sequential dosing: participants with glaucoma receive the low dose first, followed by a higher dose in a subsequent group. The primary aim of phase one is to assess safety and tolerability. The committee’s recommendation to proceed to the next dose therefore followed directly from the initial 56-day observation period.
In June, Life Biosciences administered ER-100 to the first human participant. The current report adds 56 days of follow-up and company-reported visual field test results for the first three participants.