OHR Pharmaceutical Inc.

Squalamine

Wet Age-related Macular Degeneration (wet AMD) was estimated to affect 1.75 million Americans in 2004, and the number is expected to increase to 3 million by 2020. The disease manifests via aberrant vascularization in the back of the eye that damages the retina and causes vision loss. Wet AMD is associated with age, and other factors such as smoking are also correlated with the disease. Treatments for wet AMD can also be useful for diabetic macular edema (DME) and retinal vein occlusion (RVO), expanding the therapeutic potential of therapies developed for this indication.

Squalamine is a first-in-class, small molecule, anti-angiogenic drug with a novel intracellular mechanism of action. The drug acts against the development of aberrant neovascularization through potent inhibition of multiple protein growth factors of angiogenesis, including vascular endothelial growth factor (VEGF), platelet-derived growth factor (PDGF) and basic fibroblast growth factor (bFGF).

Ohr Pharmaceutical has developed an eye drop formulation of squalamine for the treatment of wet AMD. The current FDA approved pharmaceuticals  available to treat  wet AMD are administered chronically, and delivered via injection directly into the eye. Ohr’s innovative formulation is designed for self-administration and may provide several potential advantages over currently available therapies, including:

  • Eye drops may reduce or eliminate painful and inconvenient intravitreal injections that patients must undergo on a monthly or bi-monthly basis.
  • Potential for additional visual acuity gains through the inhibition of multiple growth factors.
  • Cost advantage of a small molecule when compared to the current standards of care which are biological drugs that cost $1850-$2000 per injection.

Preclinical studies have demonstrated that squalamine eyes drops are safe and that squalamine can reach the back of the eye at concentrations that are known to inhibit neovascularization in tissue. The studies also support a twice-daily dosing structure based on the retention characteristics of squalamine in posterior ocular tissues. Twice daily eye drop administration is a convenient and patient-friendly dosing structure that can allow Ohr to achieve the goal of improving patient care and patient outcomes.

Ohr is conducting a randomized, double-blind, placebo-controlled Phase 2 clinical trial using squalamine eye drops in patients with newly diagnosed wet AMD. The trial reached 50% enrollment in July 2013, and interim data are expected in the second quarter of 2014.