First Oral Therapy for Macular Degeneration

First Oral Therapy for Macular Degeneration

Macular degeneration is currently incurable, but there are several treatment options capable of slowing down the progression of this sight stealing disease. These treatments often consist of medicine injected in or around the eyeball, retinal replacement or repair, stem cell applications and, in some cases, prosthesis tools to enhance viable vision. 

Now, the first oral treatment for macular degeneration and Stargardt’s disease (the inherited juvenile form of macular degeneration, which affects 1 in 8,000-10,000 children) may soon be available. This treatment offers some promising results and may possibly reduce or even eliminate the need for many invasive macular degeneration therapies currently being used. 


The first oral therapy for macular degeneration is labeled LBS-008. It was funded by a program developed by the National Institutes of Health (NIH) known as the highly selective Blueprint Neurotherapeutics (BPN) Network. 

The NIH describes BPN, 

“The Blueprint Neurotherapeutics Network provides non-dilutive support for small molecule drug discovery and development, from hit-to-lead chemistry through phase I clinical testing.” 

LBS-008 is the first-in-class oral therapy graduate of the BPN program which supported research by contributing $10M and the resources of 15 NIH centers to develop this revolutionary drug. Belite Bio is the company that was granted BPN support and completed a phase 1 clinical trial in 2018. 

Belite Bio is described as a subsidiary of Lin BioScience, a San Diego based clinical stage biopharmaceutical company targeting untreatable diseases, including age-related macular degeneration.

After the phase 1 trial, it was passed by the FDA (Food and Drug Administration) for the development of LBS-008 to advance to the next stage which are human trials. 

The first phase of human trials began in October of 2018 at a research facility in Australia and are expected to soon complete the second phase in the United States which began April of 2019.

Belite Bio reports on the details of the mechanism of LBS-008 on macular degeneration, 

“LBS-008 is an RBP4 [retinol binding protein] inhibitor that prevents the buildup of toxins in the eye that cause Dry AMD [age-related macular degeneration] and Stargardt. The toxins are a byproduct of the eye’s visual cycle, which is dependent on the presence of vitamin A (retinol). LBS-008 works by reducing the amount of circulating vitamin A, and consequently reducing vitamin A uptake into the eye, thus preventing the formation of the toxins associated with macular degeneration.”

With these trials of LBS-008 moving at such a rapid pace, an oral therapy for macular degeneration may only be a year or two away. This is exceptional due to the fact that most trials move much slower and can take a decade or more to make it to market, if they don’t fail before then. This oral therapy has already surpassed rigorous lab testing and one full human trial all because of its incredible ability to reduce a toxin called lipofuscin. 

Taking On Lipofuscin

The macula is the center of your retina which consists of many intricate layers. When light hits this area, photoreceptors (rods and cones) instantaneously react with a millisecond speed response directly to your brain. These signals are immediately returned with instructions on how to rapidly develop the image you are seeing. This reaction relies on the retinal pigment epithelium (RPE) cells. RPE cells provide structural support for the retina as well as the transfer of nutrients and removal of waste. 

However, according to researchers, age-related macular degeneration (AMD) may be caused by a buildup of a yellow pigmented plaque underneath the RPE cells compromising proper functioning of the photoreceptor rods and cones. 

RPE cells transport retinol (vitamin A) but when too much makes it to the retina, this yellow plaque toxin known as lipofuscin causes the photoreceptors to die. This can eventually cause macular degeneration and inevitably, blindness. 

LBS-008 reduces the formation of the toxin lipofuscin by regulating the amount of retinol that enters visual function permitting the retina to operate normally. 

Hope Into Reality

The development of LBS-008 is receiving rave reviews in the industry with descriptions such as “remarkable” bringing hope into the realm of reality for macular degeneration treatment

As reported by Ophthalmology Times, 

“Another important finding was the “remarkable” pharmacodynamic effect of the drug on the biomarker. One dose of LBS-008 produced a sustained reduction of serum RBP4 for several days, which, he pointed out, is consistent with a slow elimination rate.”

Dr. Konstantin Petrukhin, Associate Professor of Ophthalmic Science at Columbia University Medical Center, and inventor of the technology commented, 

“The drug was very well tolerated systemically and the ocular adverse events were mild, transient, and reversible…We were able to demonstrate the desired effect on the serum biomarker, and the reduction in RBP4 [retinol binding protein] was below the level that may be considered associated with clinical efficacy in AMD studies…LBS-008 is expected to bring hope to millions of patients suffering from untreatable forms of macular degeneration.”

Just the fact that LBS-008 is the first graduate of the BPN program shows promise of it becoming a game changer in the field of ocular disease. 

Dr. Tom Lin, Chairman of Belite Bio recognized all the support toward an AMD cure,  

“We’re encouraged by the ongoing favorable regulatory view of our technology, including a systematic review published by the UK National Institute for Health Research identifying reduction of RBP4 as the most promising treatments for dry AMD and Stargardt disease. We plan to continue to work closely with our collaborators to the next clinical and regulatory milestone.”

Nobody really wants to take a pill for anything but when it comes to defending yourself from developing macular degeneration, an oral therapy may be your best bet. LBS-008 may soon turn into a fancy named pharmaceutical you watch advertised in a long television commercial. Keep your eyes open (while you can) for this treatment that could mean the difference between a bright future or a dark one.