Spheramine®

Overview

Spheramine is an investigational, targeted cell-based therapy for the treatment of moderate to advanced Parkinson’s disease. In July 2008, Titan announced the results of its Phase IIb placebo controlled study. Spheramine did not meet the trial’s primary or secondary endpoints. Spheramine is an investigational therapy that uses normal human retinal pigmented epithelial (hRPE) cells to provide a targeted and continuous source of L-DOPA in the brain, which may alleviate symptoms and may modify disease progression. RPE cells are known to produce levodopa (L-DOPA), the chemical precursor of the transmitter substance dopamine, which is depleted in the brain of Parkinson’s disease patients. Spheramine consists of hRPE cells attached to a gelatin Microcarrier Support Matrix (MSM) and is administered in the brain by steriotactic injection to patients in a one-time procedure that does not require extensive follow up, potentially providing a local source of L-DOPA in the brain. Unlike other experimental cell-based therapies, Spheramine does not require suppression of the immune system to prevent rejection.

Market Opportunity for the Potential Treatment of Parkinson’s Disease

Parkinson’s disease is a progressive disorder affecting the central nervous system that affects more than 1.5 million people in the U.S. and 6 million worldwide. Long-term treatment of Parkinson’s disease is a largely unmet need with few treatment options for patients in advanced stages of the disease. Current treatments involve daily administration of oral agents containing dopamine precursors or dopamine agonists that raise levels of dopamine activity in the brain – this can lead to troubling side effects and, over time, most patients develop a “wearing off” effect.

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