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Promising pipeline of opportunities for investors in the coming year with pharmaceutical giant Roche among a number of pharma companies with promising late – stage candidates


Next five to ten years could be potentially transformational in a segment that until now has had little to offer to patients

London, 14 October- Companies operating in the age-related macular degeneration (AMD) market are an untapped equity opportunity set to generate billions in new potential sales. There is a wealth of investment opportunities on offer in the form of businesses racing to find the first effective dry-AMD treatments, and improve the effectiveness of existing treatments for wet-AMD. As a result, capital is likely to flow strongly into the sector over the next few years.

This is the key finding of Edison Group’s latest report ‘BLINDNESS: THE UNDER-RATED INVESTMENT CASE’, which gives an in-depth analysis of the investment case in upcoming AMD treatments and provides an updated overview of the leading candidates and technologies that will shape the AMD market in the coming decade.

Age-related macular degeneration (AMD) is the leading cause of blindness in older adults in western countries. The treatment market for the “wet” form of AMD (also called neovascular AMD) is already valued at over US$5.7bn and is targeted at around 3 million people across the US and Europe.

However, there exists a need to improve wet-AMD treatments, as they involve recurring injections into the eyeball which are burdensome and which about a third of patients become resistant to over time. There is a promising pipeline of opportunities for wet AMD that investors could capitalise on in the coming years. For instance, companies are looking at ways to extend  treatment durability to reduce the frequency of injections into the eyeball and so improve compliance. Roche has two late-stage candidates (faricimab, PDS-ranibizumab) seeking to do this. Meanwhile, Kodiak’s pivotal-stage KSI-301 may reduce dosing frequency to every six months. The overall wet AMD pipeline is robust with many candidates on the horizon such as OPT-302, ICON-4 or AKST4290, which may in the next half-decade or so provide new treatment options for wet AMD patients resistant to current treatment.

Around 18–22 million people in the US and Europe have early-to-intermediate forms of dry-AMD, but there is no widely accepted and approved treatment for this condition. Nearly 3 million additional people have late-stage dry-AMD, also referred to as geographic atrophy (GA), which often causes significant visual impairment. Given the prevalence of dry-AMD, it is foreseeable that this market could easily eclipse the wet-AMD market.

Several mid-to late-stage drugs are targeting inflammation and/or oxidative stress for dry-AMD or GA, and promising data has been released in the past 18 months for pegcetacoplan, Zimura and risuteganib. Zimura has already shown positive 18-month GA data in a Phase II/III study and a second Phase III is underway. There are also oral drugs (Oracea and ALK-001) in Phase III trials. Proprietary light-based therapy (2RT and Valeda) may also potentially decelerate dry-AMD progression of early-to-intermediate stage patients, although further clinical studies will be needed to confirm earlier data and drive adoption.

There is currently no effective treatment to restore vision in severely affected patients who have already lost a majority of their central visual photoreceptors to the disease.  However, Pixium Vision is advancing a photovoltaic retinal implant-based platform that aims to provide a new form of vision to restore functioning to those with profound vision loss attributable to retinal diseases such as GA. A registration-enabling study is expected to start in H121, which could lead to EU commercialisation in H223.

The Edison report concludes that the next five to 10 years are expected to be potentially revolutionary in a segment that until now has had little to offer to patients beyond dietary supplements and lifestyle modifications.   

Edison Group´s Pooya Hemami, author of the report, will be participating in the Dry AMD Therapeutics Summit, the only platform discussing issues present in the development of Dry AMD therapeutics on October 28th and 29th. The event will unite industry specialists from pharma, biotech, academia and solution providers to examine the most pressing issues important to bringing the next generation of Dry AMD drugs to market.

Pooya Hemami, Healthcare Analyst, Edison Group said: “Backing healthcare companies, which are developing new treatments and technologies to address the leading causes of blindness, give investors an opportunity to contribute positively to society as well as generate robust returns. The market for wet-AMD is already sizable while the need to develop better treatments for dry-AMD could well see this market grow considerably too. The coming decade could be potentially pivotal in the development of new approaches, drugs and products to address blindness, which would have significant societal as well as economic benefits.”

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Last Updated: 14-Oct-2020