Advances in the GA pipeline bring the first wave of therapies closer to patients

A series of recent advances in late-stage therapies for geographic atrophy (GA) give rise to hopes of introducing, potentially, the first wave of therapies for this age-related macular degeneration (AMD) subtype.

The company’s latest announcements have been a major reason for raising the positive outlook for this AMD subtype. At the annual meeting of the Association for Vision and Ophthalmology Research (ARVO), held from 1 to 4 May 2022, Apellis Pharmaceuticals announced 18-month results of a phase III DERBY and OAKS study of pegcetacoplan for GA applied monthly and every other month. The results show a reduction in the growth of lesions in the foveal and extrafoveal area, which is clinically significant. Mechanically, pegcetacoplan inhibits the C3 component of the complement system.

Following a similar mechanism of action is Zimura of IVERIC BIO (avacincaptad pegol), which is understood to inhibit the C5 component of the complement system. During the World Retina Congress held on 13 May 2022, the company provided a post hoc analysis of its Phase III GATHER 1 clinical trial; for this study, a statistically significant 27% reduction in GA growth over 12 months was reported. Another late-stage therapy currently being evaluated for GA is ALK-001 by Alkeus Pharmaceuticals, which targets the accumulation of residues in the retinal pigment epithelium (RPE) by inhibiting the formation of toxic vitamin A aggregates. expecting results from phase III, the company aims to assess the impact of this therapy on the growth rate of GA lesions.

Companies are also looking for other innovative therapies to deal with GA. An important approach in this regard is gene therapies and stem cell therapies. According to the GlobalData Pharma Intelligence Center, there are three cell therapies and two gene therapies that are in the early stages of GA development. The need to develop therapies that involve longer treatment intervals is an area of ​​constant interest to AMD drug developers. In this regard, the aforementioned newer classes of drugs can help address this gap.

Gene therapies are usually positioned as one-time treatment options, so if any of the gene therapies currently under development are approved, this will be seen as a major event that creates a paradigm shift in the way AMD and in particular GA, can be treated more effectively. In addition, the proposal to develop therapies that aim to replace the damaged RPE cell layer with new, healthy and functional RPE cells is one that has huge potential to transform the AMD treatment landscape, with the ultimate advantage of long-term treatment . lasting benefit of a single application of therapy. Although positive points can be made for these innovative products under development, characteristics such as side effect profiles, pricing, efficacy and frequency of use remain factors that will ultimately determine the success of medicines entering this market. .