Epix Pharmaceuticals is having a wild year with its Alzheimer’s drug development program. Shares in the Lexington, MA-based company (NASDAQ: EPIX) shot up 35 percent in one day in December after it said its drug produced one of the best improvements ever in memory and thinking skills for Alzheimer’s patients in a clinical trial. One month later, the gains were wiped out when a major goof-up was revealed. Epix said its contract research organization made mistakes crunching the numbers, so the improvement in patients’ cognition score was 3.6 points on a standard scale, not the 5.7 point gain previously reported.
“Obviously, we are not using those folks anymore,” says Epix CEO Michael Kauffman.
Now Epix is trying to set the record straight for good. The company plans to present full results from the mid-stage trial at the International Conference on Alzheimer’s Disease in Chicago that runs July 26-31. About 5,000 researchers will gather there to learn about the latest research against this incurable disease, which robs people of their memory and thinking skills and affects more than 5 million Americans, according to the Alzheimer’s Association.
The key finding for Epix is still the same as what the company reported January—that 10 patients who took a daily, 150 milligram dose of the drug by itself for two weeks had a 3.6 point improvement in their ADAS-cog score, a standard measurement of memory and thinking skills, compared with a 0.9 point worsening for 10 patients on placebo. Some more data is available from a computer test that patients took, which verifies the benefit, and suggests Epix’s drug may also work in tandem with Aricept, the Alzheimer’s treatment from New York-based Pfizer (NYSE: PFE) and Tokyo-based Eisai, Kauffman says. No serious side effects of Epix’s drug were seen in the study.
One of the strengths of the study is that it showed a benefit so quickly, although critics will say Epix still needs to demonstrate that the drug has a sustained effect over six months, Kauffman says. The company doesn’t have that proof yet, Kauffman says, but it plans to present some interesting results from a couple of trial volunteers who asked to continue taking the drug after the two-week study ended.
In response to the patients’ request for more drug, the company made a special petition to the FDA for a six-month extension of the study for those two individuals. The request was granted, Kauffman says, because Alzheimer’s patients have so few treatment options. The two patients in the extended trial had periods where they went off treatment, and showed “profound” improvement when they went back on, Kauffman says.
“You don’t have to be a physician to see progress when a patient can suddenly play cards when they couldn’t before,” Kauffman says.
Epix’s drug, called PRX-03140, is designed to work unlike any marketed Alzheimer’s drug, by stimulating production of a neurotransmitter in the brain called acetylcholine. In the study, it didn’t produce the key side effect of Aricept, nausea. The market is obviously huge for Alzheimer’s—Aricept, the most-prescribed Alzheimer’s medicine worldwide, generated $2.16 billion in worldwide sales in the fiscal year ended in March 2007. That’s for a drug that doesn’t really improve cognition, but can slow down the patients’ decline.
Epix and partner GlaxoSmithKline (NYSE: GSK) are recruiting patients into larger trials that could go a long way toward demonstrating if the experimental drug really has legs. One trial of 420 patients will compare two different doses of the Epix drug in combination with Aricept against placebo, measuring the ADAS-cog score after six months. A second study of 240 patients will compare the Epix drug to Aricept and to placebo. That trial will run for three months, with another three-month extension.
The latter study should produce its first results in mid-2009, Kauffman says, while the larger trial in combination with Aricept will take longer, delivering findings in the first half of 2010, he says. By that time, more data will be available on competitors like drug giant Wyeth (NYSE: WYE) and Elan’s bapineuzumab, and Medivation’s (NASDAQ: MDVN) Dimebon. Some of the buzz at the meeting will also be about the failure last month of a clinical trial of Flurizan, an experimental drug from Salt Lake City-based Myriad Genetics. There may be something to learn from it, Kauffman says.
The failure of Flurizan has gotten some researchers casting around again for promising new leads. It certainly hasn’t hurt a company like Epix, with a new method of treatment, at an earlier stage of development. “People in the field are starting to feel like we’re asking the right questions,” Kauffman says. “We’re getting some more calls and more interest about our program.”