Idalopirdine

Drug Overview

Idalopirdine (Lundbeck/Otsuka) is an active, potent, and selective oral 5-hydroxytryptamine-6 receptor antagonist. Antagonism of this receptor is associated with the potentiation of cholinergic, glutamatergic, norepinephrinergic, and dopaminergic signaling pathways, which corresponds to improvements in cognition.

Lundbeck, which specializes in developing and marketing drugs for the central nervous system, and its developmental and commercial partner Otsuka are investigating idalopirdine in a global Phase III program for the symptomatic treatment of mild to moderate Alzheimer’s disease. Idalopirdine is being tested as an adjunct to cholinesterase inhibitor treatment. Lundbeck had also attempted to develop idalopirdine as an adjunctive treatment to address the negative symptoms of schizophrenia, although this indication was terminated after a failed proof-of-concept study. In July 2016, Lundbeck and Otsuka announced that the US Food and Drug Administration has granted idalopirdine fast track designation for the treatment of mild to moderate Alzheimer’s disease.

Analyst Outlook

Following positive Phase II data in moderate Alzheimer’s disease, Lundbeck joined forces with Otsuka and quickly transitioned idalopirdine into Phase III trials. While a Phase III study of idalopirdine’s competitor RVT-101 (Axovant Sciences) was recently launched, idalopirdine is well ahead in its development, ensuring that it would enjoy first-to-market status should its pivotal clinical trials prove successful. As a 5-hydroxytryptamine-6 (5-HT6) receptor antagonist, idalopirdine is unlikely to have any effect on the underlying pathology of Alzheimer’s disease. However, it would represent an entirely novel class of drugs, and any symptomatic benefit above and beyond existing treatments would be very well received in the market. With Lundbeck’s EU presence and Otsuka’s geographic reach, the companies are well placed to capitalize on idalopirdine’s novel mechanism.