Two More Phase 3 Trials of Alzheimer's Drug Idalopirdine Fail

Deborah Brauser

February 10, 2017

Two phase 3 trials of the experimental drug idalopirdine (Lundbeck) for the treatment of Alzheimer's disease (AD) symptoms have failed, the company reports.

The news was published in the manufacturer's 2016 annual report released to investors earlier this week.

In September, the company announced that STARSHINE, the first of its phase 3 studies assessing id alopirdine, was not positive in patients with mild to moderate AD. Although it showed that the drug was safe and well tolerated, it did not meet its primary efficacy endpoint vs placebo.

Two additional studies, STARBEAM and STARBRIGHT, have now followed the same pattern.

"The efficacy profile in all three clinical studies do…not demonstrate efficacy as observed in the positive clinical phase 2 study and hence do not suffice to support a regulatory submission," the company notes.

Idalopirdine is a 5-HT6 antagonist — as is the similarly named intepirdine, which is still being developed by Axovant Sciences. However, Pfizer announced last year that a phase 2 trial of its own 5-HT6 antagonist, known as PF-05212377, was not effective.

STAR Trials Go Dim

Last July, Lundbeck announced that the FDA had granted Fast Track Designation to the drug, based on its promising phase 2 data. However, the phase 3 trials did not live up to that promise.

STARSHINE evaluated two dosages of the study drug. But when added to donepezil, neither dose of idalopirdine was significantly associated with a reduction in total score on the AD Assessment Scale-cognitive subscale (ADAS-cog) vs placebo — the primary endpoint.

Secondary endpoints "also did not show separation from placebo," the company reported at the time.

"The phase 2 data were very encouraging but unfortunately, these data failed to replicate those findings," Anders Gersel Pedersen, executive vice president and chief scientific officer at Lundbeck, said in a statement.

In the newly reported trials, STARBEAM aimed for enrollment of 858 patients, according to It evaluated placebo or two doses of the drug formerly known as Lu AE58054 as an adjunct to donepezil.

STARBRIGHT aimed for 758 patients and evaluated placebo or two dosage groups as an adjunct to base treatment with an acetylcholinesterase inhibitor.

The primary endpoint for both, which was not reached, was significant change in ADAS-cog total score between baseline and week 24.

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