Sirolimus studied in posterior uveitis

Napa, CA-Santen Inc., the U.S. subsidiary of Santen Pharmaceutical Co. Ltd. (Osaka, Japan), has initiated a global phase III clinical study evaluating its investigational drug sirolimus (DE-109) for the treatment of noninfectious posterior uveitis.

Sirolimus, originally known as rapamycin, is a highly potent, broad-acting compound that has been demonstrated to combat disease through multiple mechanisms of action. It is known to be an immunosuppressive and anti-proliferative agent in humans and an anti-angiogenic, anti-migratory, anti-fibrotic, and anti-permeability agent in animal models.

Enrollment for the phase III SAKURA (Study Assessing double-masKed Uveitis tReAtment) study opened in May. This is a randomized, double-masked study assessing the safety and efficacy of three different doses of sirolimus. The doses will be administered every 2 months in subjects with active, noninfectious uveitis of the posterior segment of the eye. About 500 subjects with active, noninfectious posterior, intermediate, or panuveitis will be enrolled at about 150 sites.

The primary endpoint, the proportion of subjects with a vitreous haze score of zero (Standardized Uveitis Nomenclature Photographic scale) will be assessed at month 5 and subjects will be followed for an additional 7 months for safety evaluation.

“We are thrilled about the initiation of the SAKURA study, as the biggest thing that the program addresses is a major unmet need in the posterior uveitis [field],” said Naveed Shams, MD, PhD, head of global clinical development and medical affairs, Santen Global Research & Development division. “If proven to be clinically effective, it should significantly [affect] the quality of life of patients with non-infectious, posterior uveitis. Intravitreal sirolimus is expected to reduce significantly the use of systemic and local corticosteroids to treat non-infectious posterior uveitis and hence reduce the incidence of corticosteroid-induced co-morbidities.”