Targacept's Alzheimer's hopeful misses the mark in Phase II

Stephen Hill

Targacept ($TRGT) kept the faith in its late-stage Alzheimer's disease drug after ex-partner AstraZeneca ($AZN) walked away from the treatment, but a new mid-stage failure has forced the biotech to pull the plug on TC-1734.

In a Phase IIb study on 293 patients, the drug went head to head against Eisai and Pfizer's ($PFE) now-generic Aricept (donepezil), with a primary endpoint of proving superiority over the most commonly prescribed Alzheimer's treatment. In top-line results disclosed Monday, TC-1734 missed its main goal, and Targacept didn't say whether it met co-primary endpoints of cognitive function and global function.

AstraZeneca washed its hands of the treatment in March, restructuring its deal with Targacept to leave the alpha4beta2 blocker out of a dwindling partnership between the two. Now Targacept is accepting defeat on the program, CEO Stephen Hill said.

"We designed a rigorous study to provide a definitive answer on whether TC-1734 could be a better treatment option than the current standard of care in what has been a very difficult disease area for the development of novel therapeutics," Hill said in a statement. "Based on these results, we do not intend to invest in further development of TC-1734."

The latest setback sent Targacept's shares south by about 16% on Monday, and its roughly $3.70 share price is down almost 50% from the company's December heights.

TC-1734's failure puts further pressure on the North Carolina company's thinning pipeline. In February, AstraZeneca again distanced itself from Targacept by handing back some early-stage candidates and terminating an Alzheimer's program for AZD1446, keeping that drug around for now to see if it could be useful in another indication.

In December, the company bailed out on TC-5619 after it missed its primary and secondary endpoints in a Phase IIb study for schizophrenia after failing a trial in ADHD. And the previously AstraZeneca-partnered TC-5214 endured four Phase III setbacks that forced Targacept to cut its payroll and refocus its R&D efforts. That therapy is now in a Phase IIb study to see if it can treat overactive bladder patients.

- read the statement