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Intravitreal Sirolimus Meets Phase III Goal as Uveitis Therapy
SAKURA (Study Assessing Double-MasKed Uveitis TReAtment) Study 1, the first of two global phase III trials evaluating intravitreal injections of sirolimus (Santen Inc.) in patients with non-infectious posterior-segment uveitis (NI-PSU), has met its primary endpoint.
NI-PSU is a debilitating eye disease that affects working-aged adults worldwide. There are no FDA-approved nonsteroidal treatments for this sight-threatening disorder.
SAKURA Study 1 is an ongoing multinational, randomized, double-blind trial assessing the safety and efficacy of sirolimus in 347 patients with non-infectious posterior uveitis, intermediate uveitis, or panuveitis. The patients were randomly assigned to three treatment arms, each receiving different doses of sirolimus by intravitreal injection. The study’s primary endpoint was the proportion of patients achieving a vitreous haze score of zero at month 5 (Standardized Uveitis Nomenclature [SUN] Photographic scale).
SAKURA Study 2 continues to enroll patients under the same protocol.
Sirolimus is a mammalian target of rapamycin (mTOR) inhibitor, an immunomodulator, which is the same active pharmaceutical ingredient in two FDA-approved products: Rapamune (Pfizer), an immunosuppressive agent used in renal transplant patients, and Cypher (sirolimus-eluting coronary stent, Cordis Corp.), approved for improving the coronary luminal diameter in patients with symptomatic ischemic disease. Intravitreal sirolimus is a proprietary ocular formulation of sirolimus.
Uveitis is an intraocular inflammatory condition that may or may not have an infectious component. It is often classified by its anatomic location in the eye. NI-PSU includes intermediate uveitis, which can affect the ciliary body and vitreous; posterior uveitis, which can involve the vitreous, choroid, retina, and/or optic nerve; and panuveitis, which encompasses anterior, intermediate, and posterior segments. Currently, steroids and systemic immunosuppressants are often used to reduce the inflammation in the eye, but these treatments can be associated with significant morbidity.
Source: PipelineReview; April 4, 2014.