Vitelliform Maculopathy Patient Treated in Israel with OpRegen Under Named Patient Compassionate Use
29 March 2021 - - A patient suffering from adult-onset vitelliform macular dystrophy had recently been treated with US biotechnology company Lineage Cell Therapeutics, Inc's (NYSE American: LCTX) (TASE: LCTX) lead product candidate, OpRegen, at Hadassah-Hebrew University Medical Center in Jerusalem, using a named patient compassionate use approval granted by the Israeli Ministry of Health, the company said.

OpRegen is an investigational cell therapy consisting of allogeneic retinal pigment epithelium cells administered to the subretinal space and is currently being investigated in a 24-patient phase 1/2a clinical trial for the treatment of dry age-related macular degeneration with geographic atrophy.

This patient presented to the Department of Ophthalmology at Hadassah-Hebrew University Medical Center in late December 2020 with sudden and severe visual acuity decreases in one eye.

BCVA in the worse vision eye was measured at 20/200, compared to 20/40 in the patient's contralateral eye.

After an onset of blurred vision in 2018, evaluation and imaging diagnosed the patient as suffering from AVMD.

Because AVMD is a disease of impaired RPE function leading to atrophy and shares similar characteristics to dry AMD, the team at Hadassah approached Lineage about the potential to treat this patient on a compassionate use basis.

Lineage submitted a request on behalf of Hadassah-Hebrew University Medical Center which was approved by the Israeli Ministry of Health.

Following approval from the University's Ethics Committee, the patient was treated in February 2021.

The delivery of OpRegen RPE cells via pars plana vitrectomy was successful, with no complications arising during the procedure and the patient remains in follow-up.

Adult-onset Vitelliform Maculopathy AVMD is an eye disorder that can cause progressive vision loss and usually begins after age 40. AVMD affects an area of the retina called the macula, which is responsible for sharp central vision.

The condition causes a fatty yellow pigment to accumulate in cells underlying the macula, eventually damaging the cells.

Some people remain without symptoms throughout their life while others may slowly develop blurred and/or distorted vision, that can progress to central vision loss over time.

There is currently no effective treatment for vitelliform macular dystrophy.

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 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.

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.

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

Lineage Cell Therapeutics is a clinical-stage biotechnology company developing novel cell therapies for unmet medical needs.