Lineage Cell Therapeutics announced promising data from interim results of the phase 1/2a clinical trial assessing the cell therapy.
OpRegen, a cell therapy from Lineage Cell Therapeutics, continues to show efficacy, with demonstrated anatomical and functional improvements in geographic atrophy (GA) in dry age-related macular degeneration (AMD), according to updated interim results from its ongoing phase 1/2a clinical study (NCT02286089).
Lineage announced the data in support of 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.
“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,” Brian Culley, chief executive officer, Lineage Cell Therapeutics, told GeneTherapyLive.
OpRegen, an allogeneic retinal pigment epithelium (RPE) cell therapy, is currently being evaluated in the phase 1/2 open-label, dose escalation study which 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.
Twenty-four patients have been enrolled 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.
“Even in patients with an incomplete coverage of OpRegen over the primary area of atrophy, we have observed resolution of not only lesions of iRORA (incomplete retinal pigment epithelial and outer retinal atrophy), but also resolution of areas with features of cRORA, which is a state of complete loss of the RPE and outer retinal tissue. Additionally, the structural benefits may help explain the improvement in visual acuity. I eagerly look forward to new data as they are collected,” Jordi Monés, MD, PhD, director, Institut de la Màcula and Barcelona Macula Foundation, said in a statement.
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. Eight of 12 treated eyes in this cohort improved in BCVA, compared with 9 of 12 (75%) untreated eyes which worsened in BCVA. There was an average difference of 10.8 letters between treated and untreated eyes at last assessment.
Previous improvements in retinal structure and drusen density, as well as a trend toward slower GA progression in treated eyes compared to untreated eyes continue to remain, and evidence of durable engraftment the RPE cells has extended to more than 5 years in the earliest treated patients.
“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,” Culley told GeneTherapyLive.