Opthea has completed a Phase 1/2A clinical trial in 51 patients with wet AMD, and is currently enrolling patients in two clinical trials:
A randomised, controlled Phase 2b clinical trial of OPT-302 in treatment-naive wet AMD patients, and
A randomised, controlled Phase 1b/2a clinical trial investigating OPT-302 in patients with diabetic macular edema (DME).
Opthea's Phase 1/2A Clinical Trial in wet AMD
Opthea's Phase 1/2A clinical trial of OPT-302 in 51 patients with wet AMD was conducted under an IND at 14 clinical sites in the USA. The first-in-human multicentre dose-escalation and dose-expansion trial investigated OPT-302 administered alone or in combination with Lucentis™ on a monthly basis for 3 months by ocular (intravitreal) injection. The trial enrolled patients with wet AMD who had either not been treated previously (treatment naïve patients) or who had demonstrated a sub-optimal response to prior anti-VEGF-A therapy.
The primary endpoint of the Phase 1/2A study was the assessment of the safety of OPT-302 administered via intravitreal injection as a monotherapy and in combination with Lucentis™. Secondary endpoints of the study included preliminary measures of clinical activity, including evaluation of visual acuity using eye charts as well as changes in wet AMD lesions, including fluid and thickness of the tissue at the back of the eye, using sophisticated imaging techniques such as fluorescein angiography and spectral-domain optical cohorence tomography (SD-OCT).
Results from the Phase 1/2A clinical trial in wet AMD patients can be found here (Opthea_Phase 1 2A wAMD Trial Update Pres_17 04 03_Final.pdf).
Of the 51 patients enrolled, 25 were treatment naive 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% were predominantly classic.
No dose-limiting toxicities (DLTs) were observed and the maximum tolerated dose (MTD) was not reached.
In participants who received combination OPT-302 (any dose) with Lucentis™, the change from baseline in mean best corrected visual acuity (BCVA) at 12 weeks was +10.8 letters in treatment naive patients (n = 18) and +4.9 letters in prior treated participants (n = 19). The mean change from baseline in central subfield thickness (CST) at 12 weeks decreased in both patient groups by -119 uM and -54 uM respectively. In patients receiving OPT-302 monotherapy (2.0 mg), 7/13 (54%) did not require anti-VEGF-A rescue therapy through the dosing period up to week 12, and the mean change from baseline at 12 weeks in mean visual acuity was +5.6 letters.