The pharmaceutical industry is intensely focused on developing innovative treatments for dry age-related macular degeneration (AMD), a leading cause of irreversible blindness in developed countries. A recent report highlights that over 70 companies are actively involved in advancing more than 80 therapeutic candidates through the clinical pipeline.
Current Landscape of Dry AMD Therapies
Currently, there are no approved treatments for dry AMD, and management primarily involves monitoring and managing symptoms. This unmet need has spurred significant research into various therapeutic strategies, including visual cycle modulators, anti-inflammatory agents, complement inhibitors, gene therapy, and cell-based therapies.
Key Players and Pipeline Therapies
Several companies are at the forefront of developing novel therapies. Alkeus Pharmaceuticals is investigating ALK-001, while Belite Bio is studying Tinlarebant (LBS-008). Other notable companies include Cognition Therapeutics (CT1812), Aviceda Therapeutics (AVD-104), and Stealth BioTherapeutics (Elamipretide).
Recent Clinical Trial Developments
- Ocugen, Inc.: In November 2024, Ocugen reported positive preliminary efficacy and safety data from its Phase 1/2 OCU410 ArMaDa clinical trial. OCU410 (AAV5-hRORA), a novel modifier gene therapy, showed reduced lesion growth and preservation of retinal tissue, with a positive effect on low luminance visual acuity (LLVA).
- Eyestem Research: In October 2024, Eyestem Research announced positive outcomes from the Drug Safety Monitoring Board (DSMB) review of its Phase 1 trial for Geographic Atrophy secondary to Dry AMD, marking a significant achievement for Indian medical research. Eyecyte RPE is a potential treatment for geographic atrophy arising from dry age-related macular degeneration (AMD).
- Galimedix Therapeutics: Initiated its Phase II eDREAM study in December 2024 to assess the safety and efficacy of GAL-101 eye drops in patients with dry AMD.
- Stealth BioTherapeutics: Announced the enrollment of the first patient in the ReNEW trial, part of its Phase III clinical program for elamipretide, in June 2024. The ReNEW and ReGAIN trials will evaluate the efficacy, safety, and pharmacokinetics of daily subcutaneous injections of elamipretide in participants with dry AMD.
Novel Approaches in Development
The pipeline includes a variety of therapeutic modalities, such as:
- Gene Therapies: OCU410 (Ocugen) represents a novel approach using gene therapy to modify disease progression.
- Cell-Based Therapies: Eyecyte RPE (Eyestem Research) is a cell therapy aimed at treating geographic atrophy.
- Complement Inhibitors: Several companies are exploring complement inhibitors to target the inflammatory pathways involved in AMD.
The Need for Combination Therapies
While pathway-based therapies like ALK-001 and LBS-008 show promise, some experts suggest that a combination approach may be necessary to fully inhibit disease progression. The focus is on developing treatments that can prevent AMD, slow its progression, or restore vision.
Impact of Advanced Imaging
Advanced imaging techniques like optical coherence tomography (OCT) have significantly improved the early detection, monitoring, and treatment assessment of dry AMD. Emerging technologies like polarization-sensitive OCT (PS-OCT) may further revolutionize AMD research and clinical practices.
