Research and Development

SQUALAMINE/EVIZON(TM)

Squalamine is a first-in-class systemic intracellular anti-angiogenic drug with a novel mechanism of action. Its ophthalmic formulation, Evizon (TM), has been evaluated against the wet form of age-related macular degeneration, a leading cause of blindness in the elderly, which affects over 200,000 new patients a year in the US alone.

In Phase II trials, in which no drug-related ocular or systemic effects were observed, stabilization or improvement in visual activity was observed in the vast majority of patients, with both early and advanced lesions responding. In patients in whom the worst AMD-affected eye was not a candidate for therapy with the currently approved wet-AMD drug therapy, the administration of squalamine produced beneficial effects in the worse otherwise non-treatable "fellow" eye as well. As opposed to the current approved standard of therapy, squalamine does not require direct injection into the eyeball.

Because of its potent anti-angiogenic effects, squalamine also shows considerable promise in the treatment of solid tumors such as ovarian cancer. Based on its mechanism of action, Squalamine essentially acts as a "trojan horse", creating entry points in the cell membranes of resistant Cancer cells to allow for standard chemotherapeutic agents to enter and attack. In a concluded Phase IIa study, patients with stage III & IV Refractory and Resistant Ovarian Cancer received Squalamine in conjunction with Carboplatin. Among 31 Evaluable patients, 5 had a complete response, 5 had a partial response, and 10 had stable disease for this highly difficult-to-treat cancer type.