Lpath, Inc. the industry leader in lipidomics-based therapeutics, has initiated enrolment in its PEDigree clinical trial of iSONEP, the company's ocular drug candidate with which it has partnered with Pfizer.

In this phase I b/II a trial, Lpath plans to dose 32 subjects that have retinal Pigment Epithelium Detachment (PED) that is secondary to wet Age-related Macular Degeneration (wet AMD) or Polypoidal Choroidal Vasculopathy (PCV).

PED is a potentially serious condition that is often part of the pathology of wet AMD and PCV, yet no drug has yet been approved to treat PED. Even though patients with PED were excluded from the Lucentis pivotal trials, this drug is often used to treat such patients, but with incomplete success. In addition, PCV frequently does not respond well to Lucentis and is often misdiagnosed as wet AMD.

Subjects in the PEDigree trial will receive up to three monthly intravitreal injections of iSONEP, and two dose levels will be tested. The primary safety endpoint is the tolerability of consecutive monthly injections, and the primary efficacy endpoint is the percentage of subjects that experience flattening of their PED.

In Lpath's phase I trial, where subjects with wet AMD received only one injection, iSONEP met its primary endpoint of being well tolerated in all 15 patients. It also succeeded in meeting a key secondary endpoint in that a positive biological effect -- with a single dose -- was observed in most patients, almost all of whom had failed to respond to Lucentis and/or Avastin treatment.

One of the distinctive benefits in phase I was the resolution (the complete flattening) of PEDs in two out of the two subjects that presented with PED. One of these patients received no further treatment (Lucentis or Avastin) during the entire 12-month monitoring period following the iSONEP injection. The other was not re-treated until day 105.

It is hypothesized that such distinctive benefits are due to iSONEP's powerful anti-inflammatory and anti-fibrotic mechanisms of action. In various animal models of disease, iSONEP was shown to substantially reduce inflammation in the eye and significantly mitigate ocular fibrosis.

The potential market for a drug that fully resolves PED is significant. It is estimated that more than 500,000 people currently have PED, and with reasonable assumptions, the attainable market size is estimated to be more than $1.0 billion. These market-size figures are expected to grow significantly as the baby-boomers fill up the ranks of the 65+ age group.

Lpath plans to begin another iSONEP trial in June of this year, a phase II human-proof-of-concept trial to study the efficacy and safety of iSONEP in wet AMD patients that do not have PED.

Glenn Stoller, MD, head of Lpath's ocular division, commented: “iSONEP is a first-in-class drug that has distinguished itself with promising animal data and, more recently, human data. A growing body of literature suggests that iSONEP's distinctive anti-inflammatory, anti-angiogenic and anti-fibrotic mechanisms could prevent both the early and the late stages of retinal damage, including the damage that often results when a PED fails to flatten over time.”

Scott Pancoast, Lpath's president and chief executive officer, added: “Because the anti-VEGF agents have demonstrated lackluster results in the treatment of PED patients, there is a significant unmet medical need. Given that both subjects that presented with PED in our phase I trial experienced a complete flattening of their PED, we like the odds of success in this larger group of patients.”