OpRegen® is currently being evaluated in a Phase 1/2a open-label, dose escalation safety and efficacy study of a single injection of human retinal pigment epithelium cells (RPE cells) derived from an established pluripotent cell line and transplanted subretinally in patients with advanced dry age-related macular degeneration (AMD) with geographic atrophy (GA). In November 2020, Lineage announced the completion of patient enrollment in this study.

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 September 2021, Lineage reported updated interim results from its ongoing, 24-patient Phase 1/2a clinical study of OpRegen. These updated results include a minimum of 9 months of follow-up in all 12 patients treated in Cohort 4, which as a group had better baseline vision and smaller areas of GA at baseline than earlier cohorts. Overall, in the study (N=24), OpRegen has been well tolerated to date and there have been no new, unexpected ocular or systemic adverse events or serious adverse events not previously reported.

  • First reported case of retinal tissue restoration showed zero growth of atrophy at 33 months
  • Second case of retinal tissue restoration exhibited a 10% reduction in atrophy size at 8 months
  • Third case of retinal restoration is 18 letters above bseline at last available time point
  • Average difference in BCVA between treated and untreated eyes was more than two ETDRS lines (10.8 letters read) in Cohort 4 patients at 9-12 months post-treatment
  • Overall, 8/12 (67%) of the Cohort 4 patients’ treated eyes were at or above baseline visual acuity at their last assessment, based on per protocol scheduled visits ranging from 9 months to over 3 years post-transplant. Conversely, 9/12 (75%) of the patients’ untreated eyes were below baseline visual acuity at that assessment.

Dry AMD RPE degeneration and drusen accumulation

Image adapted from scienceofamd.org

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% of patients and “dry AMD,” which affects 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 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.

AMD Market

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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