Brian Culley, chief executive officer, Lineage Cell Therapeutics, discussed updated data from the phase 1/2 study of OpRegen.
Lineage Cell Therapeutics’ OpRegen has continued to show efficacy in dry age-related macular degeneration (AMD), with demonstrated anatomical and functional improvements in geographic atrophy (GA).
Updated interim results from an ongoing phase 1/2a clinical study (NCT02286089) showed the investigative therapy’s efficacy, including an average difference in Best Corrected Visual Acuity (BCVA) of over 2 ETDRS lines (10.8 letters read) between treated and untreated eyes in a cohort of patients 9 to 12 months after treatment.
The trial has so far enrolled 24 patients in 4 cohorts, the first 3 of which enrolled participants with BCVA of 20/200 or worse. The fourth cohort enrolled 12 participants with BCVA from 20/65 to 20/250 with smaller mean areas of GA and also investigated a new “thaw-and-inject” formulation of OpRegen.
The trial is assessing the safety and efficacy of a single injection of the therapy to the subretinal space, with a primary outcome of incidence and frequency of treatment-emergent adverse events (TEAEs). Secondary outcomes are assessing changes in ophthalmological parameters. Safety results are in line with previous data, with no new, unexpected ocular or systemic AEs or serious AEs. The updated results now include a minimum of 9 months follow-up in all 12 patients of Cohort 4
GeneTherapyLive spoke with Brian Culley, chief executive officer, Lineage Cell Therapeutics, to learn more about the new data and OpRegen’s potential in treating AMD.
Brian Culley: Treatment with OpRegen has been generally well tolerated. Among patients which reflect the intended patient population, notable effects have included functional improvements such as improved vision in the treated eye while the untreated eyes have reduced vision. Anatomical changes include three patients with confirmed growth in areas of geographic atrophy which then measured smaller than or unchanged from baseline, observed as early as two months and lasting as long as 33 months. None of the 24 treated patients have shown evidence of rejection of the OpRegen cells and each have received a single injection of OpRegen.
OpRegen is the ONLY investigational therapy which has shown evidence of retinal restoration. All other approaches in clinical development are directed toward slowing the expansion of the area of atrophy and cannot reverse it. Other than cell therapy, all treatments currently in development also are directed toward a single pathway or group of pathways. Cell therapy (i.e OpRegen) replaces the entire cell, which means every area of dysfunction is being replaced with a healthy, functional cell. This may permit whole cell approaches to provide the maximal benefit to the patient, while targeted therapies may only deliver a partial benefit. However, cell therapy might equally provide one of the large benefits of gene therapy, which is a once in a lifetime dose, but without the risks which have been observed with gene editing or gene manipulation.
There is nothing approved in dry AMD. Notably, the leading approaches have not shown any improvement to visual acuity. OpRegen may be able to address not only the underlying anatomical problem, but also provide improved visual acuity, all with one-time dosing.
Additional follow-up and data updates, meeting with FDA to discuss various aspects of the program, and further clinical testing in late-stage trials.
Transcript edited for clarity.