Life Biosciences has announced interim data from its phase 1 clinical trial of ER-100 in 3 participants with Open Angle Glaucoma (OAG). The data demonstrated that a single intravitreal administration along with daily oral doxycycline was well-tolerated through day 56 in all 3 participants and showed preliminary signals of improvement in visual field testing in 2 participants.

Sharon Rosenzweig-Lipson, PhD, Chief Scientific Officer, Life Biosciences, said, “These initial results provide early information regarding the tolerability of ER-100 and epigenetic restoration in people with Open Angle Glaucoma. We are encouraged by the Data Safety Monitoring Board’s recommendation to escalate to dose level 2, a decision that allows us to keep moving this trial forward on behalf of people living with optic neuropathies like glaucoma, a disease where vision loss is often considered irreversible. While this phase 1 study is designed to evaluate safety and tolerability, the preliminary observations of visual field improvements in 2 participants are encouraging as we continue to advance the ER-100 program. This milestone marks an important step in Life Bio’s progress translating epigenetic restoration into a new class of medicines for age-related diseases.”

A single intravitreal administration of dose level 1 ER-100 (2×1011 vg) along with daily oral doxycycline to induce OSK expression was well-tolerated in 3 participants with OAG through day 56. There were no reported dose-limiting toxicities, Adverse Events (AEs) of special interest or serious AEs. All reported AEs were related to procedure, steroid, or pre-existing conditions and were not directly associated with ER-100.

Data Safety Monitoring Board (DSMB) recommendation was to escalate to dose level 2 (6×1011 vg) in OAG participants, with the trial currently ongoing.

Visual field observations included improvements in Humphrey Visual Fields (HVFs) measurements, a standard eye exam that measures a participant’s ability to detect light across many points in their field of vision, scored in decibels (dB), were observed in 2 of 3 OAG participants at day 56.

In each of these 2 participants, ≥ 28 percent of points in the visual fields showed a > 4 dB improvement.

This first-in-human, phase 1, open-label clinical trial (NCT07290244) is evaluating the safety and tolerability of ER-100 in adults with optic neuropathies.

ER-100 uses controlled expression of 3 transcription factors, OSK (OCT4, SOX2, KLF4), delivered via a single intravitreal injection, with OSK expression induced by systemic doxycycline administered for 8 weeks (56 days).

An oral prednisolone taper is administered to minimise risk of inflammation due to AAV-2 immune response. Multiple study visits assessing tolerability and visual endpoints were included in this first 56 day evaluation.