Pharma Update
OpRegen in GA: Replenishing the retinal pigment epithelium
Encouraging early clinical data presented at ARVO 2022 and 2023
Potential to counteract RPE cell loss in
Before OpRegen
subretinal delivery
Photoreceptor
degradation
GA
Roche
OARVO.
Ph I/lla data: Outer retinal structure and visual function improvements
in patients with impaired vision 1,2
Before OpRegen
After OpRegen
After single
OpRegen
subretinal
Cohort 4 (n=12):
Patients with bilateral GA secondary to AMD with BCVA 220/250 and ≤20/64
and GA area ≥4 and ≤11mm2
delivery
RPE degeneration
and loss
OpRegen RPE Cells
OpRegen has the potential to counteract RPE cell
loss in areas of GA by supporting retinal structure
and function
.
BCVA, ETDRS letters, mean
(±SE)
SD-
60-
> +1.3
letters
OCT
n=
n 12
n12
n 12
n 12
n 11
n 12
50-
n=12
n=12
n=11
n 12
n12n=120 12
}
+7.6
letters
n=12
40-
CFP
30-
≥15 Letter Gain at Month 12, n/N (%)
20-
Fellow Eye
Study Eye
0/12 (0%)
3/12 (25%)
0.0 1.0 2.0 3.0 4.5 6.0
9.0
12.0
Time, months
FAF
Preliminary evidence of outer retinal structure and visual function improvements with OpRegen was
observed in patients with GA and impaired vision (Cohort 4 [n=12])
•
Average 7.6 letter gain and 25% of patients with ≥15 Letter gain in Cohort 4
Ph lla trial initiated Q1 2023, continuing to optimize
subretinal surgical delivery
.
OpRegen well tolerated in Ph I/lla GA study with an acceptable safety profile and mostly mild AEs
In collaboration with Lineage Cell Therapeutics, Inc. (LCTX); 1. Ho et al. ARVO 2022; 2. Banin et al. ARVO 2023; RPE=Retinal pigment epithelium; GA-Geographic atrophy; AMD-Age-related macular degeneration; BCVA-Best-
corrected visual acuity; ETDRS=Early treatment of diabetic retinopathy study; SD-OCT-Spectral-domain optical coherence tomography; CFP-Color fundus photography; FAF=Fundus autofluorescence; AEs=Adverse events;
SE=Standard error
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