Emerging therapies in AMD

Published on 9 September 2020

Emerging therapies in AMD:

A second look at next-generation treatments

Table of Contents

Age-related macular degeneration: Wide opportunities

Current NVAMD treatments focus on VEGF-A inhibition

Commercialisation considerations for AMD products

A closer look at the NVAMD pipeline

Next-generation NVAMD therapies

Dry-AMD treatment is the next frontier

Visual rehabilitation for late-stage GA patients

On the cusp of breakthroughs in AMD treatment

Age-related macular degeneration (AMD) remains the leading cause of blindness in older adults in western countries. While neovascular AMD (NVAMD, or wet-AMD) can be controlled in most cases with recurring antivascular endothelial growth factor A (VEGF-A) intravitreal injections (IVT), there is a substantial unmet need for those with the dry form of the disease (whose prevalence is about six- to nine-fold higher), particularly those who develop geographic atrophy (GA). There also remains a need to improve NVAMD treatments, as recurring IVT injections are burdensome, and about a third of patients will become refractory to anti-VEGF-A over time. This report provides an updated overview of many of the leading candidates and technologies that will shape the AMD market in the coming decade. We expect that while not all of these products will be successful, those that are could potentially generate significant returns for investors.

A huge market opportunity

The neovascular age-related macular degeneration (NVAMD) market size is already substantial, at over $5.75bn worldwide revenue, as ranibizumab and aflibercept combined had c 9% sales growth in 2019. All NVAMD cases are preceded by dry-AMD, and NVAMD reflects roughly only half of late-stage AMD patients. We estimate late-stage AMD (defined as those patients with geographic atrophy (GA) or NVAMD) affects roughly 5.5 million people across the US and Europe, and most of these patients will become legally blind without treatment. Another c 18–22 million people in these regions have early-to-intermediate forms of dryAMD and could be at risk of developing late-AMD, but there is no widely accepted and approved treatment for this stage of the condition. Altogether, effective GA or dry-AMD treatments could add billions of dollars to an already huge AMD market.

Pixium Vision investment summary

Pixium Vision is developing the Prima wireless photovoltaic sub-retinal implant, which transforms images into electrical signals to elicit a form of central visual perception in patients with severe retinal disease. In Q120, positive 18-month data was reported from its EU feasibility study in patients with geographic atrophy associated with dry age-related macular degeneration (GA-AMD), showing continued safety and improvements of between three and seven lines on the Landolt C visual acuity scale versus baseline. Pixium started a US feasibility study in Q120 and we expect it to file for the PRIMAvera pivotal study in H220 and start implantation in this trial in H121.

Oxford Biomedica investment summary

Oxford Biomedica (OXB) is a global leader in lentiviral development and manufacturing. OXB is expanding its manufacturing facilities through OxBox, a 84,000 sq ft state-of-the-art bioprocessing facility, significantly increasing its production capacity to match increasing demand and to continue growing its platform revenues. In the near term, revenues will continue to be driven by the Novartis partnership for CAR-T Kymriah as the commercial roll-out continues. OXB has several established development and manufacturing partnerships including Novartis, Juno Therapeutics (BMS), Bioverativ (Sanofi), Orchard Therapeutics, Axovant, Boehringer Ingelheim, Santen and Beam Therapeutics.