Lineage Cell Therapeutics Announces OpRegen 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

Lineage Cell Therapeutics, Inc. (NYSE American and TASE: LCTX), a clinical-stage biotechnology company developing allogeneic cell therapies for unmet medical needs, today announced that 24 month visual acuity results from patients enrolled in a Phase 1/2a clinical study (ClinicalTrials.gov Identifier: NCT02286089) of RG6501 (OpRegen) in patients with geographic atrophy (GA) secondary to age-related macular degeneration (AMD), were presented at the 2024 Retinal Cell & Gene Therapy Innovation Summit. The meeting was jointly organized by the Foundation Fighting Blindness and the Oregon Health & Science University Casey Eye Institute. The presentation, "OpRegen® Retinal Pigment Epithelium (RPE) Cell Therapy for Patients with Geographic Atrophy (GA): Month 24 Results from the Phase 1/2a Trial," was presented by David Telander, MD, PhD, Retinal Consultants Medical Group, on behalf of Roche and Genentech, a member of the Roche Group.

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 Genentech, a member of the Roche Group, and is currently being evaluated in a Phase 2a clinical study in patients with GA secondary to AMD (ClinicalTrials.gov Identifier: NCT05626114).

"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 Brian M. Culley, Lineage CEO. "We are encouraged by the apparent durability of visual performance being achieved by patients in this study, notably in the 5 patients who received a thorough coverage of OpRegen cells across the majority of their atrophic areas and who had experienced a cessation or reversal of their areas of atrophy, evidenced by outer retinal structural improvements on optical coherence tomography (OCT). Dry AMD has traditionally been considered to be an irreversible and progressively degenerative disease leading to vision loss. However, these data suggest that OpRegen RPE cells may provide a one-time treatment which could slow or even reverse this damage by way of direct support to the patients' remaining retinal cells, including those near or within atrophic areas. We look forward to additional, future clinical data updates on the OpRegen program from our partners, Roche and Genentech."

2024 Retinal Cell & Gene Therapy Innovation SummitHighlights

- 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

• 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)
• 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.