Roche has begun phase III trials of lampalizumb, moving it a step closer to becoming the first approved treatment for the eye condition geographic atrophy (GA).
A late stage form of age-related macular degeneration (AMD), GA involves the progressive atrophy of the retinal pigment epithelium, choriocapillaris and photoreceptors, and can result in blindness.
Sandra Horning, head of global product development and chief medical officer at Roche, said: “Lampalizumab has the potential to represent a significant breakthrough for this disease and could provide real hope for GA patient.
“It is the first complement targeted therapy for GA to enter phase III and the only ophthalmic drug in clinical development that specifically targets complement factor D.”
To date lampalizumab showed a 20% reduction in GA lesion progression in patients treated monthly with the drug compared with placebo after 18 months in the phase II MAHALO phase II trial.
Roche was keen to point out that a sub-population of GA patients receiving monthly lampalizumab, and who tested positive for the complement factor I (CFI) biomarker, demonstrated a 44% decrease in the rate of disease progression.
The pharma company said analysis of the biomarker would be further explored during phase III, when it will look at whether people with the CFI biomarker could benefit more than those without it from lampalizumab treatment.
Roche’s phase III study programme will evaluate lampalizumab’s safety and efficacy, and its potential to slow the progression of GA, which affects more than 4.5 million people worldwide.
The phase III trials will involve 936 patients in each of the two randomised studies – 188 biomarker-positive patients and 124 biomarker-negative patients each for the placebo, both lampalizumab q4w and q6w arms of the trial.
The primary objective of the studies is to demonstrate a reduction in the rate of GA disease progression. This will be determined by the mean change in the GA lesion area of the chosen study eye from baseline in one year – week 48 – as measured by fundus autofluorescence (FAF), an imaging technique used to provide information about the size and type of GA lesions in the macula