Wet AMD trial meets primary safety objective

Opthea (ASX:OPT), a developer of novel biologic therapies for the treatment of eye diseases, has met the primary objective of safety in the dose escalation of its ongoing first-in-human clinical trial of OPT-302, a therapy for wet age-related macular degeneration (wet AMD) — the leading cause of blindness in the Western world.

The dose escalation trial is investigating OPT-302 administered either alone or in combination with ranibizumab (Lucentis) on a monthly basis for three months by ocular injection. The 28-day safety assessment period has now been completed for all 20 patients in the trial, after full enrolment in the trial was achieved late last month.

The company has revealed that OPT-302 administered by intravitreal injection as a monotherapy (2 mg) or at three escalating doses (0.3, 1 or 2 mg) in combination with Lucentis (0.5 mg) was safe and well tolerated at all dose levels in 20 patients with wet AMD who were either treatment naive or previously treated with standard of care. There were no dose-limiting toxicities and a maximum tolerated dose (MTD) was not reached.

Adverse events were primarily related to the intravitreal injection procedure and were mild and manageable, according to Opthea. Additionally, there were no signs of infection inside the eye, or endophthalmitis, and no clinically significant changes in intraocular pressure, electrocardiograms, blood pressure or other vital signs during the safety assessment period at all dose levels.

“The investigators involved in the study are encouraged by the clinical data showing the feasibility and favourable safety profile of OPT-302 as a monotherapy and in combination with Lucentis, particularly as a number of patients thus far have received at least two or three repeat doses on a monthly basis,” said Dr Pravin Dugel, a member of Opthea’s clinical advisory board. Opthea CEO Dr Megan Baldwin concurred, stating that the study’s success in meeting the primary objective marks “a major milestone”.

“We look forward to reporting longer term patient outcomes and secondary measures of clinical activity from the Phase 1 dose escalation study in the third quarter of 2016, when all 20 patients have completed dosing on a monthly basis for three months,” Dr Baldwin said.

With the safety assessment period now complete, patient recruitment for the randomised dose expansion Phase 2A study may now commence. The Phase 2A will enrol an additional ~30 subjects with wet AMD, randomised in a 1:1 ratio to two treatment groups of OPT-302 given as monotherapy or in combination with Lucentis administered by intravitreal injection on a monthly basis for three months. Primary analysis data from the Phase 2A study is anticipated by the end of 2016.

Opthea (ASX:OPT) shares ended 1.16% lower at $0.425 on Thursday 21 April, the day of the announcement. They have since risen to $0.51 as of 12.30 pm on Tuesday.