OpRegen®
for Dry AMD
On December 20, 2021, Lineage entered into an exclusive worldwide collaboration and license agreement with Roche and Genentech, a member of the Roche Group, for the development and commercialization of OpRegen for the treatment of ocular disorders, including advanced dry age-related macular degeneration (dry AMD) with geographic atrophy (GA) in a transaction worth up to $670 million in addition to double digit royalties. In January 2022, Lineage received a $50 million upfront payment from Genentech.
In November 2022, Genentech launched a Phase 2a, multicenter, open-label, single arm clinical study of RG6501 (OpRegen) in patients with geographic atrophy (GA) secondary to age-related macular degeneration (AMD).
Image adapted from scienceofamd.org
OpRegen is currently being evaluated in a Phase 2a study intended to optimize subretinal surgical delivery and evaluate safety and activity in approximately 30, and up to 60, patients. The primary objectives of the study are to evaluate (i) the proportion of patients with subretinal surgical delivery of OpRegen to target regions under the retina, and (ii) to evaluate the safety of subretinal surgical delivery of OpRegen as measured by the incidence and severity of procedure-related adverse events at 3 months following surgery. A key secondary objective is to evaluate the proportion of patients with qualitative improvement in retinal structure, as determined by Optical Coherence Tomography (SD-OCT) imaging, within 3 months following surgery.
The FDA has granted OpRegen with Fast Track Designations and, as such, is on an expedited regulatory path that includes the ability for increased interfacing with the FDA during clinical development and enhanced favorability for marketing approval.
Clinical Data
In May 2022, data was presented at the 2022 Association for Research in Vision and Ophthalmology Annual Meeting. OpRegen Phase 1/2a clinical results support the potential for OpRegen to slow, stop or reverse disease progression in geographic atrophy secondary to age-related macular degeneration.
- 12-month primary endpoint data suggest OpRegen is well tolerated with an acceptable safety profile
- Preliminary evidence of visual function and outer retinal structure improvements observed in five Cohort 4 patients with GA and impaired vision
Market Opportunity
AMD affects more than 30 million people worldwide and approximately 1.6 million people are newly diagnosed annually in the U.S.1 It is a leading cause of vision loss in people over the age of 60 in the developed world. There are two forms of AMD: “wet AMD,” which affects only 10-15% of patients and “dry AMD,” which affects 85-90% of patients.2 Currently, there are only two FDA-approved therapies for the less common wet AMD, yet they constitute an estimated market of more than $10 billion.3
There are no FDA approved medical therapies for the 85 to 90 percent of AMD patients who suffer from the dry form. We believe one of the most promising future therapies for dry AMD is the replacement of the layer of damaged RPE cells that support and nourish the retina.
Sources: (1) Pennington and DeAngelis, Eye and Vision, 2016 3:34; (2) JM Seddon, Epidemiology of age-related macular degeneration. (AP Schachat, S Ryan eds.) Retina, 3rd ed. St. Louis, MO: Mosby; 2001;1039-50 and (3) 2018 product sales summary based on publicly reported revenue figures for Lucentis and Eylea.
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