Transition Therapeutics is a biopharmaceutical company developing novel therapeutics for diseases with large end markets. The company’s lead drug candidate is ELND005 for the treatment of Alzheimer’s disease and bipolar disorder. It is also developing TT-401 for the treatment of type 2 diabetes and accompanying obesity.

The company announced today the dosing of the first patient in a Phase 2a study of ELND005 for Down syndrome. Down syndrome is the most common genetic form of intellectual form of disability, occurring in approximately 1 in 700 births in the United States. It is caused by an extra copy of chromosome 21. There are, at the moment, no drugs approved for the treatment of cognitive dysfunction in Down syndrome, which is thought to be caused by excessive activity of genes on chromosome 21.

The ELND005-DS201 study will evaluate the safety and pharmacokinetics of two doses of ELND005 and a placebo in young adults with Down syndrome without dementia. The study also includes select cognitive and behavioral measures. The first dosing occurred at the site of Dr. Ira Lott, Professor of Neurology and Pediatrics and the University of California, Irvine. Dr. Lott is a renowned Down syndrome expert.

Transition’s licensing partner, Elan Corporation, is responsible for all development and commercialization activities and costs for ELND005. This small molecule drug has a dual mechanism action including ß-amyloid anti-aggregation and regulation of brain myo-inositol levels. For additional information about Transition Therapeutics, ELND005, and the ongoing study, please visit www.transitiontherapeutics.com.

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