), an ophthalmology research and development company, today announced positive top-line interim results for its double-masked, placebo-controlled Phase II clinical trial of Squalamine eye drops in patients with wet age-related macular degeneration (wet AMD). The data demonstrated a positive benefit in visual function across multiple clinically relevant endpoints, including a mean change in visual acuity at the end of study visit for the interim analysis group of +10.4 letters with Squalamine eye drops plus Lucentis^® PRN versus +6.3 letters in the placebo eye drops plus Lucentis PRN arm, a 65 percent additional relative benefit (p=0.18). The visual acuity improvements were seen as early as four weeks and the relative difference in visual acuity between the two treatment arms continued to increase throughout the study. All patients in the study received an initial Lucentis injection followed by Lucentis as needed (PRN) based on clinical response. The two treatment arms were Squalamine eye drops administered twice daily plus Lucentis PRN ("Squalamine" arm or group) versus standard-of-care treatment: placebo eye drops administered twice daily plus Lucentis PRN ("placebo" arm or group). This planned interim analysis was conducted on the first 62 patients (29 treated in the Squalamine arm, 33 treated in the placebo arm), who completed the entire nine months of the treatment protocol (representing approximately 50 percent of the targeted study population). The Squalamine-treated group demonstrated improved best-corrected visual acuity (BCVA) gains relative to the placebo group at all timepoints evaluated from four to 38 weeks. In the interim analysis group, 48.3 percent of Squalamine-treated patients showed BCVA gains of ≥ 15 letters (≥ 3 lines) on a standard ETDRS eye-chart, compared with 21.2 percent in the placebo arm at the end of the study (p=0.025). In addition, patients receiving Squalamine drops were more than twice as likely to gain ≥ 4 and ≥ 5 lines of vision compared with patients in the placebo eye drop arm (≥ 4 lines p=0.022, ≥ 5 lines p=0.059). Importantly, the visual acuity gains for the placebo eye drop arm were consistent with those observed in previous clinical studies using Lucentis monotherapy treatment. Squalamine eye drops were well tolerated and had a comparable safety profile to placebo eye drops. "The beneficial effects of Squalamine on visual acuity that we've seen thus far, through its inhibition of multiple angiogenic growth factors and pathways, and in particular, the improvement in gains of three or more lines in vision compared with the placebo group, are truly remarkable," said Dr. Jason Slakter, Chief Medical Officer of Ohr and retina specialist at Vitreous-Retina-Macula Consultants of NY. "Visual acuity is the most clinically relevant endpoint for back-of-the-eye disorders. For wet-AMD patients, such enhanced gains of visual acuity over standard-of-care anti-VEGF treatments, and the restoration of vision lost to this devastating disease of the elderly using a convenient eye drop therapy is a very important clinical outcome." In the interim analysis, there were no significant differences in the frequency of Lucentis PRN injections, which was the primary endpoint of the study. The mean number of Lucentis injections was 6.2 for the Squalamine arm and 6.4 for the placebo arm, which included the baseline injection and any injections required up to and including the final study visit for the interim analysis group. "The interim results seen in this trial are encouraging," said Dr. Jeffrey S. Heier, Director of Vitreoretinal Service at Ophthalmic Consultants of Boston, member of Ohr's scientific advisory board, and study investigator. "The potential to treat patients with a non-invasive therapeutic option to provide additional visual acuity benefit over the current standard-of-care, and do it with a less than monthly injection frequency, would be a significant advance in the treatment of retinal neovascular disease and beneficial for our patients. We look forward to the results of the full data set and Phase III trials." "We are very excited by these promising interim results from this wet-AMD trial," said Dr. Irach B. Taraporewala, President and Chief Executive Officer of Ohr. "The data further validate not only the clinical utility of non-invasive topical eye drop therapies for macular and retinal disorders, but also the soundness of our company's drug development science, and our proprietary formulation technologies that enable topical dosing to achieve positive therapeutic effects in back-of-the-eye disorders. These data give us a clear path for future registration studies, and we plan to discuss Phase III registration study design and the path forward with the regulatory authorities in the coming months." The company plans to present the full data from this interim analysis at an ophthalmology conference in the second half of this year, with final clinical trial data expected in the first calendar quarter of 2015. Study Design The ongoing clinical trial (Study OHR-002) is a randomized, double-masked, placebo-controlled Phase II study to evaluate the efficacy and safety of Squalamine eye drops used in combination with Lucentis PRN for the treatment of the advanced, exudative form of age-related macular degeneration (AMD), also known as "wet AMD." The trial has enrolled 142 newly diagnosed, treatment naïve patients, of which the first 62 had completed the treatment period at the time of the interim analysis. The inclusion criteria allowed for patients with visual acuity levels similar to previous Lucentis trials, varying lesion sizes up to 12 disc areas in size, and any lesion composition, including classic and occult only types of wet AMD. The trial also included diabetics with no concomitant diabetic retinopathy. Patients received an initial intravitreal injection of Lucentis at study entry, and then underwent a 1:1 randomization to receive a daily dose of either Squalamine eye drops or placebo eye drops administered twice daily for nine months. Patients had monthly follow-up clinic visits, where they were evaluated and retreated as needed with Lucentis if pre-specified clinical criteria were met. The primary endpoint was the mean number of Lucentis injections and secondary endpoints included visual acuity as well as diagnostic imaging outcomes. The planned interim analysis was performed when more than 50 percent of the study population finished their final study visit.