MPS Program
The Mucopolysaccharidoses (MPS) are a family of lysosomal storage diseases characterized by specific deficiencies in the lysosomal enzymes which degrade glycosaminoglycans (GAGs). As a result, chronic accumulation of the undigested GAGs leads to dramatic morbidity and premature death, often in childhood.
Currently there are no treatments capable of reaching the CNS, the site of most disease burden for many patients with MPS. In addition to neurological decline, patients continue to experience other debilitating and sometimes fatal symptoms despite existing treatments. The combined incidence of MPS classes associated with neurological decline (MPS I, II, and III) is approximately 1 in 35,000.
To address these needs, Zacharon is developing a novel small molecule therapy which selectively modulates the biosynthesis of the GAGs which accumulate in patients with MPS I, II, and III. Due to this selective modification, the deficient enzyme is no longer required for GAG degradation thus reducing lysosomal accumulation.
Zacharon has completed important preclinical development activities including the demonstration of proof of concept using MPS animal models including reduction in GAG accumulation in the brain. In March of 2011, Zacharon formed a partnership with Pfizer Inc.'s Orphan and Genetic Disease Unit to complete preclinical development and successfully advance this program through clinical trials and subsequent commercialization. The successful completion of these activities is designed to enable the first small molecule therapy capable of addressing neurological decline and other needs of patients with MPS I, II, and III. Zacharon has also partnered with the National MPS Society, Team Sanfilippo, and the National Institute of Neurological Disorders and Stroke for financial support for this important program. For more information, please visit www.zacharon.com or contact [email protected].