Vision is one of the most important elements of our life, if not the most important one. A 2010 survey by Surge Research found that 60% of Americans are more afraid of blindness than of heart disease, the primary killer of both men and women in the United States.

For a massive 79% of the surveyed, other than their own death or the death of a loved one, losing their eyesight is "the worst thing that could happen to me."

Unfortunately, this is a scenario that is all too real for a growing number of people, due to macular degeneration, a progressive eye condition that affects as many as 16 million people in the US and millions more around the world.

The fastest-growing form of the disease is age-related macular degeneration, or AMD. It is the number-one cause of severe vision loss and legal blindness in adults over 60 in the United States; and as baby boomers advance into their sixties and seventies, we will see a virtual epidemic of the disease.

Current estimates show that AMD affects between 14% and 24% of the US population aged 65 to 74 years and as much as 35% of people over 75.

AMD itself can be separated into two types: the wet form and the dry form. Wet AMD is the more severe type of the disease; and while only 10% of AMD patients suffer from it, it accounts for 90% of the severe vision loss caused by macular degeneration.

According to the NIH's National Eye Institute, more than 1.6 million Americans suffer from wet AMD-a figure that's expected to climb to just under 3 million by 2020.

Wet AMD involves the growth of an abnormal bundle of blood vessels (called a "lesion") behind the macula, the central part of the retina at the back of the eye that allows us to see fine details clearly. When the macula doesn't function correctly, we experience blurriness or darkness in the center of our vision. In wet AMD, the abnormal blood vessels that have grown behind the macula tend to be very fragile and often leak blood and fluid into the region, raising the macula from its normal place and distorting vision.

There's No Cure, and Current Therapies All Suffer from the Same Drawbacks

Today, the condition is treated primarily with one of three macromolecular drugs: Lucentis, Eylea, and off-label Avastin. All of them work by directly inhibiting vascular endothelial growth factor (VEGF), and all of them have been shown to be essentially clinically equivalent (meaning each of them works as well as the others).

Annual worldwide revenue for Lucentis and Eylea is about $5.3 billion, though off-label use of the much cheaper Avastin (estimated at 60% by volume) has severely eaten into their market share. This global market is expected to grow to $8.2 billion by 2016.

The current treatments are somewhat effective, but they only address part of the problem. According to Dr. Peter Kaiser, the editor-in-chief of the Retinal Physician journal and one of the thought leaders in the industry:

"For the past 7 years, we focused our attention on blocking VEGF in order to reduce angiogenesis in wet-AMD patients. But as more and more data appeared, it seemed that the VEGF inhibitors largely worked by reducing the leakage, not by eliminating choroidal neovascularization. It is this neovascularization, otherwise known as the lesion, that causes the leakiness. Therefore, we have been patching leaks all these years and not fixing the problem."

What's more, a substantial number of patients with wet AMD do not respond well to anti-VEGF treatment, and more than 10% of patients don't respond at all. Plus, the therapy, which involves frequent injections in the eye, holds associated risks such as retinal tear or detachment.

Thus, there's a clear need for a wet-AMD drug that gets to the actual source of the leakiness (i.e., the lesion itself)-and does so with fewer injections at a comparable cost.

Lpath Inc. (LPTN)-Savior from Blindness?

That's where San Diego, California-based Lpath Inc. (LPTN) and its lead drug candidate iSONEP come in. iSONEP is a genetically engineered protein that acts as an antibody against the bioactive lipid called sphingosine-1-phosphate (S1P).

Bioactive lipids in general make for interesting drug targets. S1P in particular is a well-validated target that plays a key role as a signaling mechanism in many pathologic conditions. A growing body of evidence suggests that inhibiting the action of S1P could be an effective therapeutic treatment for wet AMD that may offer distinct advantages over the anti-VEGF drugs currently on the market.

The data so far seems to bear this out. In 2009, Lpath completed a Phase I trial of iSONEP in patients with wet AMD. The results showed that patients tolerated the drug well in various doses, with no serious side effects. It also showed lesion regression in a number of patients.

Of the five patients in the study who had occult-type disease- an earlier-stage wet AMD that affects about 80% of the newly diagnosed patients worldwide-four of them showed a 30%-plus reduction in lesion size from a single injection of iSONEP.

Over the course of the treatment, scientists observed an average 76% reduction in lesion size, with two of the patients showing complete (100%) regression of their lesion. What's more, neither of those patients required any more injections for the entire 12-month follow-up period. (In contrast, Lucentis and Eylea are typically administered once a month and once every other month, respectively.)

Is there risk? Of course. Trying to assess efficacy from a phase I trial always has its dangers-but the limited data we have on iSONEP so far are intriguing. More data are coming soon, too: the results from the Phase II Nexus trial are expected in the second half of 2014.

Positive results from the trial will no doubt send the company's stock price higher-maybe much higher. If the trial is unsuccessful, however, LPTN could lose much of its value.

This is not a play for the faint of heart, but if you are looking to add to the speculative portion of your portfolio, LPTN might be a great place to start.

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The article Healing the Blind. and Boosting Your Portfolio was originally published at caseyresearch.com