OpRegen®

OpRegen® is currently being tested in a Phase 1/2a clinical trial for the treatment of dry AMD with GA. OpRegen® is a cell-based product composed of retinal pigment epithelial (RPE) cells, derived from human embryonic stem cells (hESC) and administered as a cell suspension in ophthalmic Balanced Salt Solution Plus (BSS Plus).

OpRegen Data Update & Highlights from the 2020 American Academy of Ophthalmology Presentation Announced November 16, 2020

(data presented on 20 patients, dosed through October 5, 2020)

• Continued progressive functional improvement.
  • In Cohort 4, 6 out of 7 (86%) of patients’ treated eyes measured above their baseline vision (Best Corrected Visual Acuity, or BCVA) at 12 months, a clinically relevant timeframe, or as of the longest available timepoint less than 12 months (data collection continues for more recently-treated patients).
  • Data to date demonstrate a localized slowing of GA progression in the treated areas with a trend towards slower GA growth in treated versus fellow eyes in pooled analyses.
• Long-term engraftment is supported with imaging observations up to more than 4 years, even with a short immunosuppression regimen.
  • In all Cohort 4 patients receiving OpRegen TAI formulation, per protocol, immunosuppressants have been discontinued as scheduled, typically within 90 days post-operatively, and no cases of acute or delayed rejection or inflammation have been reported.
  • One Cohort 4 patient was treated only with mycophenolate mofetil and received no tacrolimus for immunosuppression.
• Anatomical restoration of retinal tissue.
  • A Cohort 4 patient with evidence of retinal restoration and confirmed history of GA growth, which was first reported at 9 months, continues at month 23 to have an area of GA smaller than at baseline.
  • This patient also experienced additional improvement in BCVA from 9 to 23 months post-treatment, while the untreated eye has experienced further reduction in visual acuity.
  • Long-term monitoring on this patient is expected to continue.
• Treatment overview.
  • As of October 5, 2020, 16 patients were treated via pars planar vitrectomy (PPV), while 4 were treated with the Gyroscope SDS.
  • As of November 10, 2020, 17 patients were treated via PPV, while 7 were treated with the Gyroscope SDS.
  • Enrollment in the phase 1/2a study is complete; follow-up continues for safety and efficacy.
• Safety and tolerability.
  • The primary objective of the study is to evaluate the safety and tolerability of OpRegen at 12 months, and in patients which have reached this time point OpRegen appears well tolerated.
  • There have been no unexpected adverse events (AEs) or treatment-related systemic serious AEs reported in enrolled patients.
  • The most common and expected ocular AEs were the formation or exacerbation of mild to moderate epiretinal membranes (ERMs) and a single report of a retinal detachment, with cause unknown (all occurring in patients receiving OpRegen via the PPV route of administration).
  • The Gyroscope SDS is an alternative to the PPV route and is designed to avoid ERM formation.
    • Through October 2020, 16 patients were treated via PPV while 4 were treated with the Gyroscope SDS. ERMs were observed in 13 PPV patients.
    • One patient treated with the Gyroscope SDS developed a mild choroidal neovascularization (CNV) at the site of needle penetration 6 months post-treatment which was successfully treated with a single dose of an approved anti-VEGF agent. The cause was unknown.
    • One patient treated via PPV developed a mild CNV at > 24 months post-treatment.
  • Other changes observed following OpRegen treatment persisted through the last time point examined (> 4 years in some patients), including subretinal pigmentation and hyper-reflective areas seen on optical coherence tomography (OCT).
• Visit our Media page for a replay of our therapeutic expert call to discuss the interim results, hosted on on November 17, 2020.

About the Phase 1/2a Clinical Study

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 derived from an established pluripotent cell line and transplanted subretinally in patients with advanced dry AMD with GA. The study enrolled 24 patients into 4 cohorts. The first 3 cohorts enrolled only legally blind patients with best corrected visual acuity (BCVA) of 20/200 or worse. The fourth cohort enrolled 12 better vision patients (vision from 20/65 to 20/250 with smaller 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. In total, 17 patients were treated via PPV, while 7 were treated with the Gyroscope SDS.  The primary objective of the study is to evaluate the safety and tolerability of OpRegen as assessed by the incidence and frequency of treatment emergent adverse events. Secondary objectives are to evaluate the preliminary efficacy of OpRegen treatment by assessing the changes in ophthalmological parameters measured by various methods of primary clinical relevance. Additionally, for the patients in Cohort 4 that receive subretinal delivery of OpRegen utilizing the Gyroscope SDS, objectives will include the evaluation of the safety of delivery of OpRegen using the Gyroscope SDS.

OpRegen is a registered trademark of Cell Cure Neurosciences Ltd., a majority-owned subsidiary of Lineage Cell Therapeutics, Inc.