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SCIENCE & technologies
The Disease

Alzheimer’s disease (AD) is a progressive brain disorder that gradually destroys a person’s memory and ability to learn, make judgments, communicate and carry out daily activities. As AD progresses, individuals may also experience changes in personality and behaviour, such as anxiety, suspiciousness or agitation, as well as delusions or hallucinations. Neuropsychiatric symptoms associated with AD are a significant problem for AD patients and their caregivers. Approximately 90% of AD patients develop neuropsychiatric symptoms, and up to 60% develop agitation/aggression over the course of their disease. Agitation/aggression are among the most disruptive neuropsychiatric symptoms in AD and are associated with increased morbidity and caregiver burden. With an aging population, there is a great need for new therapies that can effectively reduce or delay the neuropsychiatric symptoms associated with AD.

The Drug Candidate

ELND005, scyllo-inositol, is an orally bioavailable small molecule drug candidate that crosses the blood-brain-barrier in humans. In clinical studies, ELND005 has demonstrated an acceptable safety and tolerability profile. Currently ELND005 is being investigated for multiple neuropsychiatric indications on the basis of its proposed dual mechanism of action. A completed Phase 2 study (ELND005-AD201) which evaluated ELND005 in more than 350 mild to moderate AD patients was published in the peer-reviewed journal, Neurology. In this completed study, ELND005 appeared to decrease the emergence and severity of specific neuropsychiatric symptoms, an effect which seemed to correlate with drug exposure for some symptoms.

Mechanism of Action

ELND005’s dual approach to AD also aligns with its effect on two distinct mechanisms of action. The completed Phase 2 AD study demonstrated ELND005 treatment led to reduction in myo-inositol levels in the brain, an effect that is shared by other approved neuropsychiatric drugs such as lithium and valproic acid. In addition, ELND005 was associated with a reduction in the levels of beta amyloid and tau proteins in the cerebrospinal fluid, illustrating target engagement (beta amyloid pathology) as well as neuronal protection (tau).

Current Clinical Studies

Phase 2 Study in Agitation/Aggression in AD

The objectives of Study AG201 are to evaluate the efficacy, safety and tolerability of ELND005 over 12 weeks of treatment in patients with moderate to severe AD, who are experiencing at least moderate levels of agitation/aggression. The study is expected to enroll approximately 400 patients at multiple sites in the US, Canada and other selected regions. Dosing of patients commenced in November 2012.

Completed Clinical Studies

Phase 2 Study in 350 Mild to Moderate AD Patients

Link to press release

Fast-Track Designation

The FDA has granted Fast Track Designation to the development program for ELND005 which was submitted for the treatment of Neuropsychiatric Symptoms in Alzheimer's disease. The FDA concluded that the development program for ELND005 for the treatment of NPS in AD meets their criteria for Fast Track Designation.

© 2013 Transition Therapeutics Inc.