Press Releases
OpRegen® (RG6501) Phase 1/2a Clinical Study 24-Month Visual Acuity Results Featured at 2024 Retinal Cell & Gene Therapy Innovation Summit
- BCVA gains of +5.5 letters at 24 months in Cohort 4 patients (n=10)
- BCVA gains of +7.4 letters among Cohort 4 patients with extensive OpRegen bleb coverage of the GA lesion (n=5)
- Maintenance or increases in external limiting membrane (ELM) and retinal pigment epithelium (RPE) layers observed by optical coherence tomography (OCT) in patients with extensive bleb coverage of the GA lesion
- Data suggests OpRegen may counteract RPE cell dysfunction and cell loss in GA by providing support to remaining retinal cells, with effects observed through at least 2 years following a single administration
RG6501 (OpRegen) is a suspension of human allogeneic retinal pigment epithelial (RPE) cells currently in development for the treatment of GA secondary to AMD. OpRegen subretinal delivery has the potential to counteract RPE cell loss in areas of GA lesions by supporting retinal cell health and improving retinal structure and function. It is being developed under an exclusive worldwide collaboration between Lineage, Roche, and
“The products approved recently for the treatment of GA secondary to AMD have not demonstrated a compelling functional benefit, so there remains a need for treatments that can positively affect patient vision,” stated
2024 Retinal Cell & Gene Therapy Innovation Summit Highlights
- Improvement in visual acuity and outer retinal structure in patients with extensive OpRegen bleb coverage of their GA area was present at 12 months (primary endpoint) and persisted through 24 months
- BCVA gains in Cohort 4 patients (less advanced GA than in other cohorts) measured at 12 months were sustained at 24 months following a single subretinal administration of OpRegen
- Mean change in BCVA among treated eyes for patients (n=10) completing 2-year follow up was +5.5 letters (Early Treatment Diabetic Retinopathy Study [ETDRS] assessment)
- Effects were greater on average in the five (5) patients with extensive OpRegen coverage of atrophic areas at the time of surgical delivery
- In these patients’ treated eyes, the mean change in BCVA was +7.4 ETDRS letters for those completing 2-year follow-up (n=5)
- Maintenance or improvements in external limiting membrane (ELM) and RPE drusen complex (RPEDC) structure on OCT were observed in five patients in Cohort 4 with extensive OpRegen coverage of atrophic areas at the time of surgical delivery
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Mean improvement of RPEDC area compared with baseline was maintained in treated eyes from 12 months (+2.6 mm2; n=5) to 24 months (+2.6 mm2; n=4)
- In comparison, mean change in RPEDC area decreased in untreated fellow eyes from 12 months (-1.1 mm2; n=5) to 24 months (-2.8 mm2; n=4)
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Mean change in ELM area increased in treated eyes from 12 months (+0.4 mm2; n=5) to 24 months (+0.8 mm2; n=4)
- In comparison, mean change in ELM area decreased in untreated fellow eyes from 12 months (-1.3 mm2; n=5) to 24 months (-1.9 mm2; n=4)
- Overall, these data suggest that OpRegen RPE cells may counteract RPE cell dysfunction and loss in GA by providing support to the remaining retinal cells within atrophic areas and such effects are durable through at least 24 months after a single administration
Dr. Telander’s presentation is now available on the Events and Presentations section of Lineage’s website.
About the Innovation Summit
The annual Retinal Cell and Gene Therapy Innovation Summit 2024 is jointly organized by the
About the OpRegen Phase 1/2a Study
The Phase 1/2a study is an open-label, single-arm, multi-center, dose-escalation trial evaluating a single administration of OpRegen delivered subretinally in patients with bilateral GA secondary to AMD. Twenty-four patients were enrolled into 4 cohorts. The first 3 cohorts enrolled only legally blind patients with a best corrected visual acuity (BCVA) of 20/200 or worse. The fourth cohort enrolled 12 patients with impaired vision (BCVA from 20/65 to 20/250 with smaller mean areas of GA). Cohort 4 also included patients treated with a new “thaw-and-inject” formulation of OpRegen, which can be shipped directly to sites and used immediately upon thawing, removing the complications and logistics of having to use a dose preparation facility. The primary objective of the study was to evaluate the safety and tolerability of OpRegen as assessed by the incidence and frequency of treatment-emergent adverse events. Secondary objectives include evaluating the preliminary activity of OpRegen treatment by assessing the changes in ophthalmological parameters measured by various methods of primary clinical relevance.
About Geographic Atrophy
GA is an advanced form of AMD characterized by severe loss of visual function. GA is a leading cause of adult blindness in the developed world, affecting at least 5 million people globally. There are two forms of advanced AMD: neovascular AMD and GA. GA and neovascular AMD can occur simultaneously in the same eye, and patients treated for neovascular AMD may still go on to develop GA. GA typically affects both eyes.
About
Forward-Looking Statements
Lineage cautions you that all statements, other than statements of historical facts, contained in this press release, are forward-looking statements. Forward-looking statements, in some cases, can be identified by terms such as “believe,” “aim,” “may,” “will,” “estimate,” “continue,” “anticipate,” “design,” “intend,” “expect,” “could,” “can,” “plan,” “potential,” “predict,” “seek,” “should,” “would,” “contemplate,” “project,” “target,” “tend to,” or the negative version of these words and similar expressions. Such statements include, but are not limited to, statements relating to: the potential therapeutic benefits of OpRegen in patients with GA secondary to AMD; the significance of clinical data reported to date from the Phase 1/2a study of OpRegen, including the findings of retinal tissue restoration, durability and visual function improvements; and the timing and availability of clinical data updates on the OpRegen program. Forward-looking statements involve known and unknown risks, uncertainties and other factors that may cause Lineage’s actual results, performance or achievements to be materially different from future results, performance or achievements expressed or implied by the forward-looking statements in this press release, including, but not limited to, the following risks: that positive findings in early clinical studies of a product candidate may not be predictive of success in subsequent clinical studies of that candidate; that Roche and
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