Intepirdine from Axovant was an experimental oral, once-daily treatment for people with mild to moderate Alzheimer’s disease who were also taking Aricept (donepezil). After a failed Phase 3 clinical trial, Axovant is no longer developing intepirdine. 

How intepirdine works

Intepirdine blocks the 5HT6 receptor that promotes the release of acetylcholine within the brain. 5HT6 is mainly produced in the brain and spinal cord and is involved in learning and memory processes. Acetylcholine is an important neurotransmitter or cell signaling molecule responsible for alertness, memory, thought, and judgment which are key components of thinking ability.

When used together with Aricept, a molecule that increases acetylcholine signaling by preventing its breakdown, intepirdine increases the concentration of acetylcholine in the brain. It was hoped that this would reduce the symptoms of Alzheimer’s disease.

Intepirdine in clinical trials

Results from a Phase 2b study were presented at the 2017 Annual Meeting of the American Association for Geriatric Psychiatry (AAGP). During the study, participants with mild to moderate Alzheimer’s disease who were receiving stable Aricept treatment were randomized to either receive 35 mg of intepirdine, 15 mg of intepirdine, or a placebo. Results were assessed at week 24 and showed that participants receiving 35 mg of intepirdine had a statistically significant difference in the Katz index of independence in activities of daily living, a scale to measure disease severity, suggesting that the treatment had the potential to provide benefits for Alzheimer’s disease patients.

A Phase 3 study (NCT02585934), called MINDSET, to confirm the efficacy of intepirdine as adjunctive therapy to Aricept for the treatment of Alzheimer’s disease was completed in September 2017. Primary objectives included changes in the Alzheimer’s disease assessment scale to test functionality and Alzheimer’s disease cooperative study to measure behavioral changes at 25 weeks. Secondary objectives included disease progression and adverse events.

Axovant announced negative results from this study in September 2017, stating that the primary objectives were not met. Participants treated with 35 mg of intepirdine did not improve in any of the disease assessment scales compared with participants treated with a placebo. Following these results, Axovant terminated its development of intepirdine for Alzheimer’s disease.


Last updated: September 9, 2019


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