ONL is leveraging its breakthrough vision protection technology to develop novel treatments for a range of serious, vision-threatening retinal diseases and conditions.
The company’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.
ONL Therapeutics is currently conducting clinical studies of ONL1204 Ophthalmic Solution across the following indications:
- A Phase 2 clinical study for the treatment of patients with macula-off, rhegmatogenous retinal detachment. This study is ongoing and is being conducted at sites in the United States.
- A Phase 1b clinical study for the treatment of patients with geographic atrophy (GA) associated with age-related macular degeneration (AMD). This study is ongoing and is being conducted at sites in Australia and New Zealand.
- A Phase 1b clinical study for the treatment of patients with open angle glaucoma. This study is ongoing and is being conducted at sites in Australia and New Zealand.
Learn more about our ongoing clinical trials below.
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
Rhegmatogenous retinal detachment (RRD) is an acute and serious vision-threatening condition in which a tear in the retina, typically resulting from a posterior vitreous separation, allows liquefied vitreous to enter the subretinal space, thereby detaching the photoreceptor (PR) layer of the neurosensory retina from the retinal pigment epithelium (RPE). Since the RPE is the principal source of metabolic support for the PR layer, once detached, PRs undergo a cascade of inflammation and cell death. PR cell death is the primary mechanism of vision loss after RRD. The separation of PRs from the RPE leads to the disruption of normal PR homeostasis and activates cell death in the PRs.
Treatment with ONL1204 Ophthalmic Solution has shown promise in an open-label, dose escalation (25 μg to 200 μg) Phase 1 study (ONL1204-RRD-001) with 14 subjects with a macula-off RRD. ONL1204-RRD-001 evaluated the safety and tolerability of IVT ONL1204 and found no cases of dose-limiting toxicity and no serious adverse events (SAE) related to ONL1204. Therefore, the primary objective of this Phase 2 study is to evaluate the safety and efficacy of two doses (50 μg and 200 μg) of a single IVT injection of ONL1204 Ophthalmic Solution as an adjunct to standard-of-care (SOC) surgical repair in subjects with a macula-off RRD.
A Phase 1b Study to Assess the Safety and Tolerability of Intravitreal ONL1204 Ophthalmic Solution in Patients with Geographic Atrophy (GA) Associated with Age-related Macular Degeneration (AMD)
Age-related macular degeneration (AMD) has become a major cause of visual disability and legal blindness globally. Although generally affecting only the central retina (macula), this region of photoreceptors provides the visual acuity necessary for reading, driving, and the performance of fine vision-related tasks. Associated with aging, cigarette smoking, obesity, diets low in certain nutrients, a lifestyle related to cardiac risk, and a growing list of genetic factors, AMD is becoming an increasingly prevalent public health concern, especially as the global population ages. Geographic atrophy (GA), also called atrophic AMD, is an advanced form of AMD.
Open Angle Glaucoma
A Phase 1b Study to Assess the Safety and Tolerability of Intravitreal ONL1204 Ophthalmic Solution in Patients with Progressing Open Angle Glaucoma
Open angle glaucoma is a leading cause of blindness in people aged 60 years and older and affects approximately 44.7 million people worldwide with an estimated prevalence in the United States of 2.7 million in 2011, which is expected to increase to 7.3 million by 2050. Open angle glaucoma is characterized by damage to retinal ganglion cell axons at the optic nerve head, resulting in progressive visual field defects. Elevated intraocular pressure (IOP) is the primary modifiable risk factor and reducing IOP is the only clinical approach shown to slow/prevent vision loss. Despite the availability of effective IOP lowering drugs, many patients require multiple agents to control IOP that together often fail to achieve a “target” IOP. An alternative approach is to directly protect retinal ganglion cells, independent of IOP. There are currently no approved pharmacological therapies for neuroprotection in glaucoma. The development of ONL1204 Ophthalmic Solution addresses a clear unmet medical need: the prevention of vision loss in patients suffering from deterioration of visual function because of progressing open angle glaucoma.