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The Disease

Down syndrome (DS, Trisomy 21), caused by an extra copy of chromosome 21, is the most common genetic form of intellectual disability with a prevalence of approximately 1 in 700 live births in the US. Children with DS exhibit developmental delay and various degrees of intellectual disability, while adults are at increased risk of Alzheimer’s dementia. There are currently no drugs approved for the treatment of cognitive dysfunction in DS.

The Drug Candidate

ELND005, scyllo-inositol, is an orally bioavailable small molecule drug candidate that crosses the blood-brain-barrier in humans. An extensive clinical program of Phase 1 and Phase 2 studies have been completed with ELND005 to support clinical development.

Mechanism of Action

Excess activity of genes on chromosome 21, such as amyloid precursor protein (APP) and sodium-myo-inositol active transporter (SMIT), are thought to play a role in the cognitive dysfunction of DS. Life-long exposure to increased amyloid and myo-inositol levels in the brain are thought to lead to synaptic dysfunction and cognitive disability.

ELND005 has been shown to significantly reduce both beta-amyloid and myo-inositol levels in the brain in a clinical trial with Alzheimer’s patients. Acting through this dual mechanism of action to DS, ELND005 may have the potential to improve cognition in persons with DS by decreasing amyloid levels and regulating myo-inositol-dependent neuronal signalling.

Current Clinical Studies

Phase 2A Study

Study ELND005-DS201 will evaluate the safety and pharmacokinetics of two doses of ELND005 and placebo in young adults with Down Syndrome without dementia, and will also include select cognitive and behavioural measures.

© 2013 Transition Therapeutics Inc.