Key Takeaways

  • ER-100 was well tolerated through Day 56 in the first 3 patients with open-angle glaucoma, with no dose-limiting toxicities, serious adverse events, or adverse events of special interest reported

  • Two participants demonstrated greater than 4 dB improvements at at least 28% of tested visual field locations, although efficacy cannot be established from these early findings.

Life Biosciences announced interim data from its phase 1 clinical trial of ER-100 in three 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 three participants and showed preliminary signals of improvement in visual field testing in two participants.

“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,” said Sharon Rosenzweig-Lipson, Ph.D., Chief Scientific Officer of Life Bio. “While this Phase 1 study is designed to evaluate safety and tolerability, the preliminary observations of visual field improvements in two 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.”

Presentation highlights:

Safety and Tolerability Observations

  • 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 open angle glaucoma (OAG) through Day 56
  • There were no reported dose-limiting toxicities, adverse events of special interest or serious adverse events
  • All reported adverse events were related to procedure, steroid, or pre-existing conditions and were not directly associated with ER-100
  • Data Safety Monitoring Board recommendation was to escalate to Dose Level 2 (6×1011 vg) in OAG participants, with the trial currently ongoing

Visual Field Observations

  • Improvements in Humphrey Visual Fields 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% 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 three 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 minimize 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.