About Age-related Macular Degeneration
Age-Related Macular Degeneration (AMD) affects the central part of the retina, known as the macula, which is responsible for central vision and its sharpness. AMD is one of the leading causes of irreversible vision loss and blindness in people over the age of 50 worldwide. There are two types of AMD:
- Dry AMD is characterized by the presence of drüsen in the macula. Early-stage dry AMD is characterized by small drüsen accumulation, which may not cause changes in vision, but as drüsen grow in size and increase in number, they may lead to a dimming or distortion of vision that people find most noticeable when they read. Intermediate stage dry AMD is defined by more abundant and larger drüsen and the appearance of early atrophies. Patients at this stage are at high-risk of advancing into geographic atrophy, or GA, a late stage form of AMD. Patients in the late stage of AMD may have blind spots in the center of their vision and may lose central vision.
- Wet AMD is a late stage form of AMD characterized by abnormal blood vessels from the choroid underneath the macula. This is called choroidal neovascularization. These blood vessels leak blood and fluid into the retina, causing distortion of vision that makes straight lines look wavy, as well as blind spots and loss of central vision. These abnormal blood vessels and their bleeding eventually form a scar, leading to permanent loss of central vision.
Approximately 85% to 90% of AMD patients suffer from dry AMD. Dry AMD impairs many functions affecting quality of life and independent living such as reading, driving, and facial recognition. The prevalence of dry AMD increases significantly with advancing age.
We believe that photo-oxidative and inflammatory stresses induced by the accumulation of A2E in RPE cells are the main factors responsible for the degenerative process of the retina in diseases such as AMD. We believe the biggest opportunity in treating dry AMD is preventing advancement into the later stages, GA or wet AMD, where vision loss is severe and can lead to visual disability.
MAKA-PK PHASE 1 CLINICAL TRIAL
We have completed chronic and acute animal toxicology studies to support our IND and clinical trial applications. We plan to hold scientific advice meetings with the applicable regulatory agencies in Europe in the second half of 2019 regarding our clinical development plan in dry AMD, commencing with a Phase 1 clinical trial (MACA-PK) in healthy volunteers. We expect the MACA-PK Phase 1 clinical trial will assess the safety, pharmacokinetics and pharmacodynamics of Macuneos (BIO201).