Clinical Trials
ONL is leveraging its innovative neuroprotection platform technology to develop new treatments for multiple, vision-threatening retinal diseases
ONL’s lead therapeutic candidate, ONL1204, is a first-in-class small peptide that effectively inhibits the Fas receptor, thus blocking the activation of the Fas pathway and the related immune signaling which results in the death of key retinal cells and loss of vision.
ONL1204 is designated an investigational drug and is not approved for commercial use. As such, the information on this page was created for scientific exchange with Healthcare Professionals only.
Across all completed clinical studies to date, ONL1204 has been found to be generally safe and well tolerated, under the planned oversight and guidance of safety review committees.
Learn more about our clinical trials below:
Dry AMD/GA
A Phase 2 Multicenter, Randomized, Double-masked, Sham-controlled, Reference-arm Study to Evaluate Efficacy and Safety of ONL1204 in Patients With Geographic Atrophy (GA) Associated With Age-related Macular Degeneration (AMD)
The purpose of this study is to collect efficacy and safety information across 3 experimental arms, including 2 dose levels of ONL1204 and 2 treatment frequencies (once every 12 weeks or 24 weeks) in patients with GA associated with AMD.
ACTIVE
NCT06659445
Phase 2
Intravitreal (IVT) Injection ONL1204:
- Dosing: Injection every 12 weeks or 24 weeks for 72 weeks
- Doses: 100 µg (50 µl), 200 µg (100 µl)
Dry AMD/GA
A Phase 1b Multicenter, Randomized, Controlled, Multi-dose Study of the Safety and Tolerability of ONL1204 Ophthalmic Solution in Patients with Geographic Atrophy (GA) Associated with Age-related Macular Degeneration (AMD)
The purpose of this study was to evaluate the safety and tolerability of ONL1204 Ophthalmic Solution in patients with geographic atrophy associated with AMD.
ONL1204 was generally safe and well tolerated in this study. Data assessments showed efficacy signals observed after 6 months, with ONL1204-treated eyes slowing GA lesion growth compared to untreated eyes.
COMPLETED
NCT04744662
Phase 1b
Intravitreal (IVT) Injection ONL1204:
- Dosing:
- Component 1: Single injection with patients followed for 6 months
- Component 2: 6-month natural history period followed by 6-month treatment period with two injections delivered 12 weeks apart
- Doses: 50 µg (100 µl), 200 µg (100 µl)
Progressive Open Angle Glaucoma
A Phase 1b Multicenter, Randomized, Single-Masked, Sham-Controlled Study of the Safety and Tolerability of ONL1204 Ophthalmic Solution in Patients with Progressing Open Angle Glaucoma
The purpose of this study was to demonstrate the safety and tolerability of ONL1204 Ophthalmic Solution in patients with progressing open angle glaucoma.
ONL1204 was generally safe and well tolerated in this study. Data assessments demonstrated that ONL1204-treated eyes showed sustained increases in retinal nerve fiber layer thickness and improved or stabilized visual fields.
These initial data points demonstrate the potential for ONL1204 as a neuroprotection treatment for progressing open-angle glaucoma patients.
COMPLETED
NCT05160805
Phase 1b
Intravitreal (IVT) Injection ONL1204:
- Dosing: Two injections, 90 days apart followed by a 180-day wash-out
- Doses: 50 µg (100 µl), 100 µg (50 µl)
Rhegmatogenous Retinal Detachment (Macula-Off)
A Phase 2, Randomized, Sham-Controlled, Single-Masked, Dose-Ranging, Multi-Center Study to Assess the Safety and Efficacy of Intravitreal ONL1204 Ophthalmic Solution in Subjects with Macula-off Rhegmatogenous Retinal Detachment
The goal of this Phase 2 clinical trial was to learn about ONL1204 Ophthalmic Solution in terms of safety and how well the drug works in patients that have a macula-off (central point of vision) rhegmatogenous retinal detachment (RRD).
While the study overall did not meet the primary endpoint, ONL1204 did show benefit in patients at highest risk for vision loss, consistent with a potential neuroprotective effect seen across all clinical studies to date.
COMPLETED
NCT05730218
Phase 2
Intravitreal (IVT) Injection ONL1204:
- Dosing: Once at randomization
- Doses: 50 µg (100 µl), 200 µg (100 µl)
Rhegmatogenous Retinal Detachment (Macula-Off)
A Phase 1 Open-Label, Dose Escalation Study to Assess the Safety and Tolerability of Intravitreal ONL1204 in Patients with Macula-off, Rhegmatogenous Retinal Detachment
The purpose of this study was to evaluate the safety and tolerability of ONL1204 in participants with Macula-off, Rhegmatogenous Retinal Detachment (RRD).
In this study, ONL1204 was generally safe and well tolerated at all evaluated doses when administered as a single IVT injection in patients with macula-off RRD. Several cytokines that were predicted to be Fas-dependent exhibited a trend toward reduction following the administration of ONL1204 Ophthalmic Solution, including MCP-1.
COMPLETED
NCT03780972
Phase 1
Intravitreal (IVT) Injection ONL1204:
- Dosing: Once at randomization
- Doses: 25 µg (50 µl), 50 µg (100 µl), 100 µg (50 µl), 200 µg (100 µl)