Lineage Cell Therapeutics announced additional patient data from its ongoing phase 1/2a clinical study of OpRegen, the company’s retinal pigment epithelium (RPE) transplant therapy, for the treatment of dry age-related macular degeneration (AMD).
The first Cohort 4 patient treated using both a new subretinal delivery system and the company’s new thaw-and-inject (TAI) formulation of OpRegen has continued to demonstrate notable improvements in vision, having gained 25 readable letters (or 5 lines) 6 months following administration of OpRegen RPE cells, as assessed by the Early Treatment Diabetic Retinopathy Scale (ETDRS). This represents an improvement in visual acuity from a baseline of 20/250 to 20/100 in the treated eye. A second Cohort 4 patient has been similarly dosed, and though early, the patient has shown a small improvement in visual acuity in the treated eye at just 14 days following treatment. To date, improvements have become most evident approximately 3 to 6 months after treatment. Both patients had rapid healing at the surgical site with no unexpected complications or any serious adverse events.
“We are increasingly optimistic about the data we are collecting in dry AMD,” Brian M. Culley, CEO of Lineage, said in a company news release. “We have treated five patients in Cohort 4, those with less advanced disease, which more closely match our intended patient population. At the longest-available assessment point for each patient, all five have shown an increase in the number of letters they can read on an ETDRS eye chart. Importantly, these gains have been maintained for as long as 15 months, which is the longest time point for which we have collected data in the better vision cohort. Notably, the first two patients dosed with the new sub-retinal delivery system by Gyroscope Therapeutics and our innovative TAI formulation of OpRegen had no unexpected complications, so we intend to request the removal of the enrollment treatment stagger from the protocol, which should permit us to significantly accelerate our rate of enrollment. Our objective is to combine the best cells, the best production process and the best delivery system, which we believe will position us as the front-runner in the race to address the unmet opportunity in the potential billion-dollar dry AMD market.”
“We expect 2020 will be a year of major milestones for Lineage. Based on our existing cash and the current value of our marketable securities, we believe we will be able to achieve these milestones under our reduced 2020 spending plan,” Mr. Culley said.
“Having dosed a patient with the combination of Gyroscope’s recently 510(k)-cleared Orbit Subretinal Delivery System alongside Lineage’s new thaw-and-inject formulation of OpRegen RPE cells, I found the procedure to be relatively straightforward, leading to the successful delivery of RPE cells to the subretinal space,” said Judy Ju-Yi Chen, MD, a retinal surgeon at West Coast Retina, San Francisco. “I am hopeful that additional procedures will show that this combination provides superior dose control, safety, and efficacy compared to conventional procedures.”
The company also is collecting data on rate of geographic atrophy (GA) growth, best corrected visual acuity (BCVA), low-light visual acuity, reading speed, quality of life questionnaires, microperimetry, and assessing structural changes using optical coherence tomography (OCT), fundus autofluorescence (FAF), and color fundus photography.