The results of the 20-patient first-in-human Phase 1 dose-escalation study were reported in April 2016 and presented at the European Society of Retina Specialists (EURETINA) Congress on in Copenhagen, Denmark in September 2016.
The combined results of the 51-patient Phase 1/2a study were reported in April 2017 and subsequently published in a peer-reviewed ophthalmology journal (Dugel et. al., Ophthalmol. Retina, (2020), Volume 4 (Issue 3), 250-263: Phase 1 Study of OPT-302 Inhibition of Vascular Endothelial Growth Factors C and D for Neovascular Age-Related Macular Degeneration).
The primary outcome measurement of the study was assessment of safety and tolerability of OPT-302 administered via ocular (intravitreal) injection as a monotherapy and in combination with Lucentis®. The secondary outcome measurements of the trial included preliminary determinations of clinical activity, including evaluation of visual acuity using eye charts, and evaluation of anatomical changes in the wet AMD lesions.
Of the 51 patients enrolled, 25 were newly diagnosed treatment-naïve patients, and 26 had received prior intravitreal anti-VEGF-A therapy. The majority of lesion types (72.5%) were occult, 23.5% were minimally classic and 4.0% were predominantly classic.
OPT-302 demonstrated a favourable safety profile in this study as a monotherapy and in combination with Lucentis® treatment.
In patients who received the combination OPT-302 (any dose) with Lucentis®, the mean change in best corrected visual acuity (BCVA) from baseline at 12 weeks was +10.8 letters in the treatment-naïve patients (n=18) and +4.9 letters in the prior-treated patients (n=19). The mean central subfield thickness (CST) also decreased in both patient groups, the mean change from baseline at 12 weeks for the treatment-naïve and prior-treated patients being -119 μM and -54 μM, respectively.