Randomised controlled trials

Promising Alzheimer's drug disappoints in phase III trials

Idalopirdine is the latest failure in the elusive quest for a treatment for the degenerative neurological disease.

A drug that appeared a promising candidate for treatment for Alzheimer’s has failed to show benefits in phase III clinical trials.

Phase II trials had suggested that Lundbeck’s idalopirdine might improve cognition in patients with Alzheimer’s disease (AD) taking cholinesterase inhibitors, but this was not borne out in three phase III clinical trials, the result of which have been in the Journal of the American Medical Association. The researchers conclude: “The findings do not support the use of idalopirdine for the treatment of AD”.

The trials included 2525 patients aged 50 years or older with mild to moderate AD randomised to receive a daily dose of idalopirdine 10mg, 30mg, or 60mg/day, or placebo, in addition to their cholinesterase inhibitor for 24 weeks.

Idalopirdine is a selective 5-hydroxytryptamine-6 receptor antagonist, and therefore has cholinergic, glutamatergic, dopaminergic, and noradrenergic properties, so was considered to have the potential to augment multiple neurotransmitter systems to improve cognition.

The disappointing results come in the same week that pharma giant Pfizer announced that it was pulling out of early stage research into Alzheimer’s and Parkinson’s diseases to focus on other areas of its pipeline in which expertise is strongest.

In a statement Pfizer said that the company recognised that “neuroscience is an area of tremendous unmet need” and that it “will continue to explore opportunities outside of Pfizer to out-license our existing programs”, although, “the late-stage Lyrica programs and the tanezumab development program will continue as planned”.

The news is the latest in a long history of failure by drugs companies to make progress in a field desperate for new therapies. In an accompanying editorial in JAMA, David A. Bennett of the Rush Alzheimer’s Disease Center, Rush University Medical Center, Chicago, Illinois, points out that over the past 15 years, more than 400 clinical trials of therapeutics for Alzheimer’s disease have been registered, and the failure rate of those that reported has been nearly 100%.

Citation: The Salvadore DOI: 10.1211/PJ.2018.20204224

Have your say

For commenting, please login or register as a user and agree to our Community Guidelines. You will be re-directed back to this page where you will have the ability to comment.

Recommended from Pharmaceutical Press

  • Paediatric Drug Handling

    Written for new pharmaceutical scientists, this book provides a background in paediatric pharmacy and a comprehensive introduction to children's medication.

  • Physicochemical Principles of Pharmacy

    This established textbook covers every aspect of drug properties from the design of dosage forms to their delivery by all routes to sites of action in the body.

  • Prescribing Medicines for Children

    Prescribing Medicines for Children is designed to improve understanding on all aspects of paediatric prescribing, from the development of suitable drugs through to their practical administration.

  • FASTtrack: Pharmacology

    FASTtrack: Pharmacology is a study guide providing an account of drug action, as well as dealing with molecular pharmacology at a more advanced level.

  • Drugs in Use

    Optimise drug therapy for your patients. These case studies help you bridge the gap between theoretical medicines knowledge and practical applications.


Search an extensive range of the world’s most trusted resources

Powered by MedicinesComplete

Supplementary images

  • brain scan

Newsletter Sign-up

Want to keep up with the latest news, comment and CPD articles in pharmacy and science? Subscribe to our free alerts.