Melbourne-based biotech company Opthea has demonstrated that combination therapy involving its lead drug candidate OPT-302 is superior to standalone Lucentis treatment among neovascular age-related macular degeneration (AMD) patients.
The ocular therapeutics company last week revealed the findings of its Phase 2b clinical trial involving 366 treatment-naïve wet AMD patients. The results take the drug a step closer to commercialisation and sent its Australian Stock Exchange share price soaring from $0.87 to a peak of $2.91.
The trial investigated the effectiveness of OPT-302 – a VEGF-C/D inhibitor – which was administered monthly in either 0.5 or 2.0mg doses, in combination with Novartis’ blockbuster drug Lucentis 0.5mg (ranibizumab). This was compared alongside a control group that received Lucentis 0.5mg monotherapy, which is considered the current standard of care.
Patients who received the OPT-302 2.0mg formulation gained a mean of 14.2 letters of vision from baseline on the Early Treatment of Diabetic Retinopathy Study (ETDRS) standardised eye chart at 24 weeks, compared with 10.8 letters in the control group.
The 0.5 mg OPT-302 low dose group had a similar outcome to the control group, a gain of 9.4 letters. Compared with standalone Lucentis therapy, the OPT-302 2.0 mg therapy also donstrated improvents across multiple secondary endpoints.
“In testing for superiority against very intensive anti-VEGF-A therapy [Lucentis], the bar was set high,” the study’s chief investigator Professor Tim Jackson, a Consultant ophthalmic surgeon at King’s College London, said.
“Despite this, OPT-302 2.0 mg combination therapy showed statistical superiority for the most accepted and sensitive primary efficacy outcome – mean visual acuity. Key secondary endpoints were very supportive of the primary outcome, and safety was favourable.
“Taken together, these results indicate that combined suppression of VEGF A, C and D has considerable potential as a novel treatment for wet AMD. OPT- 302 may emerge as a combination treatment that can offer better vision gains than standard of care. Further registrational trials are clearly justified.”
Opthea CEO Dr Megan Baldwin previously told Insight the trial was one of largest Phase 2b neovascular AMD trials being conducted in the world. The study also recruited the required number of patients ahead of schedule, and was completed six months early. Enthusiasm for the drug also pointed to a significant need in a patient group that only has access to two approved products – Lucentis and Eylea – as well as off-label cancer drug Avastin, she said.
“[These results] further support our conviction that OPT-302, the first ‘Trap’ inhibitor of VEGF C and D designed specifically for the eye, can improve patient outcomes in wet AMD and other retinal vascular diseases,” Baldwin said.
“We are delighted that the clinical results from this study support advancing OPT-302 into pivotal, registrational Phase 3 development, and firmly believe that OPT-302 will have a significant commercial role in the treatment landscape for wet AMD patients.”
Opthea also claims to be in a strong cash position, with approximately $20 milion cash and an additional $14 million from an anticipated research and development tax rebate later this year. Furthermore, early completion of the trial led to substantial cost savings.
Opthea is also fully funded through the remainder of the Phase 2b trial. The company has reports to have sufficient capital to prepare for registrational Phase 3 trial activities.
