On June 24, 2014, Ohr Pharmaceutical, Inc. announced positive top-line interim results for its Phase 2 clinical trial of Squalamine eye drops in patients with wet age-related macular degeneration (wet AMD). The data demonstrated a positive benefit in multiple visual function including a gain in visual acuity of +10.4 letters with Squalamine eye drops plus Lucentis® compared to +6.3 letters in the placebo group. This is a 65% additional relative benefit. 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.
“The beneficial effects of Squalamine on visual acuity . . . 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 . . . using a convenient eye drop therapy is a very important clinical outcome.”
The company presented the full data from the Phase 2 interim analysis at the 2015 annual meeting of the American Academy of Ophthalmology. Of particular note is that the addition of 2% Squalamine (OHR-102) to Lucentis increased visual acuity, with a mean gain, and in the proportion of patients with gains, of three, four, and five lines. There was, however, no reduction in the number of Lucentis injections, which was the primary endpoint of the study. For the 128 patients who completed the study, Squalamine in combination with Lucentis was shown to be more effective than Lucentis alone.
On April 17, 2016, Ohr Pharmaceutical announced that the first patient had been enrolled in a Phase III clinical trial of Squalamine.
“The Phase III clinical program will examine the potential of Squalamine, when administered as part of a combination therapy, to significantly improve visual acuity in patients with wet AMD,” said Dr. Jason Slakter.
The first of two randomized, double-masked, placebo-controlled trials was to include approximately 165 centers in the United States and Canada with a target enrollment of 650 treatment subjects newly diagnosed with wet AMD.
The double-masked, placebo-controlled, multicenter, international study would be administered twice in combination with Lucentis® injections. The primary endpoint was stated as a measurement of visual acuity gains at nine months, with subjects followed to two years for safety. The eligibility criteria was to include subjects with choroidal neovascularization (CNV) secondary to AMD.
More information about the trial from clinical trials.gov. (NCT02727881).