In short
OCUL101 is an investigational medicine being studied for neovascular age-related macular degeneration (nAMD), a common cause of vision loss in older adults. It is a bi-specific, humanized antibody-like “trap” designed to block two pathways involved in the disease: vascular endothelial growth factor (VEGF) and complement C5. One registered clinical trial is currently authorised, a Phase II/III study planning to enroll 255 participants to compare two doses of OCUL101 against aflibercept (Eylea).
Key points
- OCUL101 is an investigational bi-specific antibody-like trap targeting VEGF and complement C5.
- It is being studied in neovascular age-related macular degeneration (nAMD), with or without geographic atrophy.
- The authorised trial is a Phase II/III randomised, masked, active comparator-controlled study.
- The study plans to enroll 255 participants and compare two doses of OCUL101 with aflibercept 2 mg.
- The primary measure in Part B is the change in best corrected visual acuity (BCVA) at Week 36.
- OCUL101 is given by intravitreal injection directly into the eye.
What is OCUL101?
OCUL101 is an investigational medicine, which means it has not yet received marketing authorisation and is still being studied in clinical trials. It is described in the trial record as an anti-VEGF and anti-complement C5, bi-specific humanized antibody-like “trap”. The term “bi-specific” means the molecule is designed to attach to two different targets, while “antibody-like trap” refers to a protein engineered to bind and neutralise its targets in a way similar to an antibody.
How does OCUL101 work?
OCUL101 is designed to block two biological pathways that contribute to neovascular age-related macular degeneration. The first target is vascular endothelial growth factor (VEGF), a protein that promotes the growth of abnormal, leaky blood vessels under the retina. The second target is complement C5, a component of the immune system's complement pathway that can drive inflammation and tissue damage in the back of the eye. By blocking both VEGF and complement C5, OCUL101 aims to reduce both the abnormal vessel growth and the inflammatory damage seen in nAMD.
Which conditions is OCUL101 studied for?
- Neovascular age-related macular degeneration (nAMD) — also called wet AMD, a condition in which abnormal blood vessels grow under the macula, the central part of the retina, causing fluid leakage and vision loss.
- nAMD with geographic atrophy — the trial also includes participants who have geographic atrophy, areas of retinal cell loss that can occur alongside the wet form of the disease.
How is OCUL101 given?
OCUL101 is given by intravitreal injection, which means it is injected directly into the clear gel that fills the inside of the eye. The trial record lists a dose of 10.4 mg for OCUL101 administered by intravitreal use. The comparator, aflibercept (Eylea), is given as a 2 mg intravitreal injection from a 40 mg/mL solution in a pre-filled syringe.
What do the clinical trials show?
One clinical trial of OCUL101 is registered in the EU Clinical Trials Information System. It is a Phase II/III multicentre, randomised, masked, active comparator-controlled study called the STAT Study. “Phase II/III” means the trial combines elements of early efficacy testing (Phase II) and larger confirmatory testing (Phase III). The study plans to enroll 255 participants with neovascular age-related macular degeneration, with or without geographic atrophy.
The trial has two parts. Part A focuses on safety, measuring the incidence and rate of adverse events, serious adverse events, and treatment-emergent adverse events. Part B focuses on efficacy, with the primary measure being the change from baseline in best corrected visual acuity (BCVA) letters using the ETDRS eye chart at Week 36, compared with the active control aflibercept 2 mg. BCVA is a standard measure of how well a person can see with the best possible corrective lenses.
The study is currently authorised, meaning it has received regulatory approval to begin. It is randomised, so participants are assigned by chance to one of the treatment groups, and masked, meaning participants and researchers do not know which treatment is being given. The active comparator is aflibercept (Eylea), an already authorised anti-VEGF medicine used for nAMD.
What side effects are known?
The trial record does not list specific side effects for OCUL101. Part A of the study is designed to measure the incidence and rate of adverse events, serious adverse events, and treatment-emergent adverse events, which means safety information will be collected during the trial.