Ocugen announce Phase 3 enrolment completion for OCU400, the largest gene agnostic therapy trial for RP
Biotechnology company Ocugen has announced the enrolment completion of their phase 3 clinical trial for OCU400.
In a nutshell: Retinitis pigmentosa (RP) is a common inherited retinal condition that causes gradual vision loss due to damage of the retina’s light-sensing cells. OCU400 is an experimental gene therapy designed to support overall retinal health that could potentially work across many different genetic forms of RP. A large Phase 3 trial involving 140 participants in the US and Canada has now finished enrolling and is testing whether the treatment can improve people’s ability to navigate in low light over 12 months. Early trial results have been promising, and if successful, the therapy could move toward approval as early as 2027, although it may still take time before becoming widely available
Biotechnology company Ocugen has announced the enrolment completion of their phase 3 clinical trial for OCU400. OCU400 is a gene-agnostic therapy for retinitis pigmentosa (RP), meaning that the treatment has the potential to help a large proportion of our community with RP regardless of their genetic diagnosis.
“With enrolment complete for OCU400, we enter into a very significant time as a Company. This milestone brings us even closer to potentially delivering our first novel modifier gene therapy candidate to market and providing a one-time treatment for life to hundreds of thousands of RP patients across the globe with unmet medical need.” – Dr. Shankar Musunuri, Chairman, CEO, and Co-founder of Ocugen.
What happens in RP?
RP is one of the most common types of inherited retinal disease (IRD), associated with faults in over 100 genes, affecting around 1 in 4,000 people globally. RP causes progressive vision loss, starting peripherally, due to the loss of light-sensing rod and cone photoreceptors in the retina. To learn more about RP, please visit Retinitis pigmentosa – Retina UK.
What is OCU400?
OCU400 is based on a gene called NR2E3, which regulates various physiological functions within the retina like photoreceptor development and maintenance, metabolism, inflammation and cell survival networks. It works by resetting the affected cellular networks in the retina to establish a state of healthy balance (homeostasis). The treatment has the potential to improve retina health and function in people with RP.
OCU400 is the first gene-agnostic gene therapy product to enter the phase 3 study stage and is the largest known Phase 3 orphan gene therapy trial. This marks a significant milestone in non-gene specific treatment development!
The trial:
This phase 3 trial, known as liMeliGhT, has now completed the enrolment of 140 participants of various ages including children, spanning early-to-late disease progression and involving a number of different genetic mutations. This is a multicentre trial taking place in 17 sites across the US and Canada.
The 140 participants have been split into 2 groups in the trial. The first group includes participants with RP caused by mutations in the RHO gene specifically. The second group includes participants with various different genetic diagnoses or that possibly do not know the gene causing their RP, making up the ‘gene-agnostic’ arm of the study. Participants were randomised in the ratio 2:1 into the treatment group and untreated control group, meaning that within each arm of the study, one third of the participants will not receive treatment and act as controls.
Participants randomised to the treatment group will receive a sequential, bilateral sub-retinal injection of OCU400 if both eyes meet inclusion criteria. This means that participants will receive the treatment in both eyes over time (one at a time) via a subretinal injection. Those in the untreated control group will receive OCU400 subretinal injections after completion of the trial’s 12-month follow-up period.
The primary endpoint of liMeliGhT is a 12-month change in visual function assessed by LDNA (luminance dependent navigation assessment) with improvement in Lux Level from baseline to 12 months. This means that researchers are looking for improvement in vision by testing if people can move through a space at dimmer light levels than when they began the trial.
What happens next?
This trial is hoping to build on the positive long-term, 3-year phase 1/2 data for OCU400. As a one-year clinical trial, topline data is expected to be available in the first quarter of 2027. This data, combined with previous trial data, is then anticipated to support the Biologics License Application (BLA) filing for OCU400 and potential approval in 2027.
Please note that different regulatory bodies operate in the US, UK, Europe and elsewhere, and obtaining regulatory approval can take a long time, even if Ocugen seeks to work with all of them. Reaching agreement for NHS availability is a further, separate process. As a result, there may be a significant wait before the treatment becomes widely available. To read more about Ocugen please visit their website here. To read more about the trial itself, please visit the following link Study Details | NCT06388200 | A Phase 3 Study Of OCU400 Gene Therapy for the Treatment Of Retinitis Pigmentosa | ClinicalTrials.gov
Ocugen has also recently announced promising early results for OCU410ST, their modifier gene therapy approach in development for Stargardt disease. They have also recently announced the completion of dosing in this phase 2/3 trial. To read more about OCU410ST, please visit our article here.