Ohr Pharmaceutical Inc. (OHRP) has enrolled half of the 120 patients needed for a mid-stage study for an age-related eye disorder, as it seeks to compete with Regeneron Pharmaceuticals Inc. (REGN) and Roche Holding AG.
Ohr’s lead drug Squalamine, an eye drop formulation for wet age-related macular degeneration, is undergoing Phase II trials and interim results are expected by the second quarter of 2014, Chief Financial Officer Sam Backenroth said. Squalamine is a novel small molecule that inhibits growth factors, and eye drops would offer an advantage compared with available injections developed by Tarrytown, New York-based Regeneron and Basel, Switzerland-based Roche, Backenroth said.
“What’s important to note about the 50 percent enrollment is that the clock starts ticking for the interim analysis,” Backenroth said in a telephone interview. When the first enrollees complete a nine-month treatment period, followed by analysis, the data will give “the first initial proof-of-concept as to the efficacy of the eye drop,” he said.
Ohr, meaning light in Hebrew, is trying to grab part of what it estimates to be a $5 billion market, dominated by Regeneron’s Eylea, and Lucentis, sold in North America by Roche unit Genentech Inc. New York-based Ohr wouldn’t be the first drugmaker seeking to improve on intravenous treatments for the leading cause of blindness in the elderly. London-based GlaxoSmithKline Plc (GSK) did a mid-stage study for wet-AMD using eye drops of its Votrient medicine.
Glaxo’s Votrient “was a less potent drug that acts further downstream in the biochemical pathway,” Ohr Chief Executive Officer Irach Taraporewala said in an interview. “They haven’t officially terminated their program but both their Phase II study results have now read out and they’re clearly not moving forward.”
Wet AMD is caused by abnormal blood-vessel formation at the back of the eye, triggering fluid to leak into the macula and creating scarring that obscures vision. Regeneron’s drug is designed so it can be administered less frequently than Lucentis. (ROG) About 200,000 people in North America are diagnosed annually with wet AMD, according to Genentech.
In June, Ohr announced a one-for-three reverse stock split required for a Nasdaq listing. The shares advanced 38 percent this year through the July 5 close, giving the company a market value of $133 million. Started in 2008 by investor Orin Hirschman to look for mid-stage drugs that showed efficacy in a large therapeutic area, Ohr now has $6.7 million in cash and doesn’t plan on raising money through 2014, Backenroth said.
The company plans to focus on ophthalmology and will seek to “create value” with its other assets such as a drug for cancer cachexia, according to Backenroth. While the drug failed to meet a key clinical trial goal, the original study design was flawed and the drug showed “clinical utility” such as significant increase in patient appetite and stability in body mass, Backenroth said.
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