Wet AMD represents the advanced stage of age-related macular degeneration, where abnormal blood vessels grow beneath the macula, often leading to rapid and severe vision loss. This condition is a leading cause of irreversible blindness and visual impairment, particularly among individuals over 60. Wet AMD currently impacts around 3.5 million patients worldwide, many of whom have been diagnosed and are receiving treatment. For those affected, managing this progressive disease is crucial to maintaining their quality of life, highlighting the importance of early detection and effective therapies.
Wet AMD brings on sudden, rapidly progressing symptoms, including visual distortions where straight lines appear bent or wavy. People often experience a sharp decrease in central vision in one or both eyes. This makes everyday tasks like reading or driving difficult. Many need brighter light for reading or performing tasks that require close-up focus. They also struggle to adapt to low light environments, such as dimly lit restaurants or theaters. As the condition progresses, printed text becomes blurry, making reading harder. Recognizing familiar faces also becomes challenging due to blurred or distorted vision.
Overview of Established Treatments for Wet AMD
The cornerstone of wet AMD therapy is the inhibition of Vascular Endothelial Growth Factor (VEGF). VEGF is a key driver of the pathological angiogenesis that defines this condition. The American Academy of Ophthalmology (AAO) reports that anti-VEGF treatments help up to 90% of wet AMD patients preserve their vision. This benefit also extends to patients with other retinal diseases. In fact, approximately one-third of these patients may experience improvements in visual acuity with these therapies.
Ranibizumab and Aflibercept have long dominated the treatment landscape, backed by robust clinical data and widespread adoption. However, the introduction of biosimilars will significantly lower therapy costs. This will make these critical treatments more accessible to a broader patient population.
The therapeutic landscape is also evolving with the advent of newer agents like Faricimab and high-dose Aflibercept. These treatments aim to reduce the frequency of intraocular injections. This addresses one of the key challenges faced by patients—treatment burden. Reducing the need for frequent injections could enhance patient compliance. These innovations also have the potential to improve overall outcomes in managing wet AMD.
Understanding some upcoming new modalities of therapies
Gene Therapy: Gene therapy is emerging as a promising approach in the treatment of wet AMD by offering a potential long-term solution that addresses the disease at a genetic level. RGX-314, for example, utilizes a viral vector to deliver genes directly into the eye. Scientists design these genes to produce anti-VEGF proteins continuously, targeting the suppression of abnormal blood vessel growth in wet AMD. Gene therapy provides the potential to reduce or eliminate the frequent intraocular injections that are currently necessary to manage the disease. Early clinical trials have shown that a single administration of RGX-314 can maintain visual stability over time with minimal adverse effects.
Tyrosine Kinase Inhibitors (TKIs): TKIs target VEGF receptors at the intracellular tyrosine kinase domain. This domain plays a key role in regulating signaling pathways that promote abnormal blood vessel growth in the retina. By inhibiting these receptors, TKIs block internal cell signaling triggered by VEGF. This provides a more comprehensive method of preventing pathological angiogenesis. Unlike current anti-VEGF treatments that primarily work outside the cell, TKIs intervene within the cell’s signaling pathways. This new class of therapies is still in the clinical trial phase. However, it may reduce the frequency of injections. These therapies could also improve long-term management of the disease.
The Cost of Therapy
The annual cost of treatments is significant for the payers and patients.
There is a need for longer-acting therapies to reduce the treatment burden, frequency of injections, and allow patients to see their physicians predictably.
This would allow to generate savings for the healthcare system and avoid unnecessary visits to doctors. This would help to build the case to payers for reimbursement of future drugs/
Industry Dynamics & Opportunity in Wet AMD
Analysts project the market will grow quickly, reaching more than $15B by 2028.
Pharmaceutical and biotech companies remain highly active in the ophthalmology field, as evidenced by significant licensing agreements and mergers. For instance, Abbvie, in the licensing agreement for RGX-314 involved an upfront payment of $370 million, highlighting the substantial investments and the commercial potential of newer treatments. Similarly, the recent acquisition of Iveric Bio, by Astellas, for $5.9 billion reflects the high stakes involved in securing promising therapies.
Overcoming the challenges: from clinical to commercial stage
Despite the progress made, several challenges remain to effectively manage wet AMD:
- Treatment burden: Current therapies often require frequent hospital visits for injections, placing a logistical and financial burden on patients.
- Effectiveness and safety: Variability in patient response to treatment requires a personalized approach to drug development and administration.
- Access to healthcare: Limited access to specialist care, particularly in less urbanized areas, prevents timely diagnosis and treatment, worsening outcomes for patients.
Genchrome’s Recommendations:
There are still significant unmet needs in the field of Wet AMD
While current anti-VEGF has significantly changed the outcome of patients, there is a need for improving treatment burden with potent treatments.
Launching new treatments requires sufficient commercial and market access skills to ensure commercial success to ensure proper differentiation, unique positioning, and market access success.
Strategic pharma partnerships, particularly in ophthalmology, are crucial for enhancing patient outcomes and ultimately building commercial success.
Genchrome is here to help you define the right strategy and find the right pharma partner!
To get the full wet AMD presentation send us an email at: contact@genchrome.com
By Caroline BOULLIAT, PharmD and CEO at Genchrome
