Lumateperone (ITI-007)

Lumateperone (ITI-007) is an investigational therapy that had been developed by Intra-Cellular Therapies to treat agitation in patients with dementia.

The oral therapy was being investigated for its potential benefits for people with Alzheimer’s disease. However, the company terminated clinical trials with this group of patients based on interim results from a Phase 3 study.

The therapy is still being assessed as a possible treatment for schizophrenia and bipolar disorder.

How lumateperone works

Alzheimer’s disease, the most common cause of dementia, results from damage to nerve cells in the brain that are involved in memory, and in the ability to think and learn. Anxiety and agitation are prevalent behavioral symptoms of the disease, and a source of considerable distress for patients and caregivers.

Lumateperone is a first-in-class molecule designed to selectively and simultaneously act on certain neuronal pathways. It regulates the release, uptake, and transmission of neurotransmitter or cell signaling molecules such as serotonindopamine, and glutamate. The misregulation of neurological pathways involving these neurotransmitters is linked to severe mental illness.

Lumateperone in clinical trials

The treatment’s safety, tolerability, and pharmacokinetics — how a therapy is processed in the body — were first assessed in a dose-escalation Phase 1/2 trial (NCT02078310). That trial tested lumateperone in healthy volunteers and patients with dementia, including those with Alzheimer’s disease. Results, announced by Intra-Cellular in 2014, indicated that the therapy was well-tolerated and safe across a range of low doses, and showed “clinical signals for improved cognition.”

Intra-Cellular initiated a multicenter, double-blind, placebo-controlled Phase 3 clinical trial (NCT02817906), also known as Study 201, in 2016. That trial was designed to evaluate the efficacy and safety of lumateperone taken by mouth at a dose of 9 mg once daily for four weeks in about 360 people diagnosed with probable Alzheimer’s disease, and with clinically significant symptoms of agitation.

The trial’s primary outcome was to evaluate treatment efficacy as measured by the Cohen-Mansfield agitation inventory, community version (CMAI-C), a tool that assesses the frequency of agitated behaviors in patients. Secondary outcomes included efficacy measurements using the clinical global impression scale for severity (CGI-S) of illness and other behavioral disturbances associated with dementia.

The trial was terminated in 2018 after enrolling 177 participants. The data monitoring committee for the study concluded that the trial would not meet its primary endpoints after reviewing interim data. Based on these recommendations, Intra-cellular decided to terminate the study.


Last updated: July 23, 2019


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