Eli Lilly & Co. (LLY)’s experimental Alzheimer’s treatment slowed memory loss and cognitive decline in early-stage patients by about 30 percent, offering the first evidence that a medication may hamper the course of the ailment, researchers said.
The benefit, though small, supports further research targeting a protein called beta amyloid, said Rachelle Doody, chairwoman of Alzheimer’s disease research at Baylor College of Medicine who presented the findings today at the American Neurological Association meeting in Boston.
The independent analysis by Doody and colleagues at the University of California, San Diego, supported Lilly’s report in August. At that time, Lilly said the drug, solanezumab, slowed mental decline in those with mild Alzheimer’s while providing no benefit to more advanced patients. To market the drug, Lilly may need to do another study to confirm the findings, said Maria Carrillo, a vice president of medical and scientific relations at the Alzheimer’s Association.
“This isn’t the home run that is going to get you a medication by the end of the year, but it is very encouraging because with subsequent studies this could be a viable therapeutic option,” Carrillo said in an interview. “We need more options for our patients and families. We are very encouraged by what this could mean.”
Lilly hasn’t met with the U.S. Food and Drug Administration to discuss the next steps for gaining approval of the drug, said Eric Siemers, a senior medical director at Indianapolis-based Lilly.
The improvement may not be enough for regulatory clearance, according to Alex Arfaei, an analyst with BMO Capital Markets in New York.
“We continue to believe the absolute difference is only nominally statistically significant and indicative of a ’hint of clinical activity,’ but not clinically meaningful, and most likely not enough to warrant approval,” Arfaei wrote in a note today.
Lilly rose 5.3 percent to $50.78 at the close of trading in New York, the company’s biggest single-day gain since March 2009, and highest level since April 2008. Shares of Lilly have increased 22 percent this year.
About 5.4 million Americans have Alzheimer’s, the most- common form of dementia, and the number is expected to surge to as many as 16 million by 2050 as the population ages, according to the Alzheimer’s Association. Drugs on the market now address only the symptoms, not the underlying cause, and none has been shown to slow progression of the disease.
Solanezumab attaches to a protein called beta amyloid that builds up into clumps in the brains of Alzheimer’s patients. Lilly’s treatment is designed to prevent those clumps from forming.
Whether preventing the buildup of beta amyloid will have an effect on patients has been debated for years by Alzheimer’s researchers. Pfizer Inc. (PFE) and Johnson & Johnson (JNJ) stopped late- stage testing of a drug that also targeted beta amyloid after it failed to show a benefit to patients. The medicine appeared to lessen a measure of nerve cell damage in the brain and the buildup of amyloid plaque, signaling it could show promise for early use.
Today’s findings add evidence to the argument that targeting beta amyloid provides a benefit to patients and should be tested further, Doody said.
The only other therapy in final-stage testing is Baxter International Inc. (BAX)’s Gammagard. The product is an expensive, scarce treatment derived from donated blood plasma that replaces antibodies in people whose immune systems can’t protect them from infection. Results on whether it could slow or stop Alzheimer’s may be available next year.
Lilly tested its drug in two studies involving a total of 2,000 patients, about two-thirds of whom had the mild form of the disease. Most patients in the studies were also taking one of the drugs currently on the market to treat the Alzheimer’s symptoms, such as Forest Laboratories Inc. (FRX)’s Namenda.
In the first study, called EXPEDITION 1, patients with mild Alzheimer’s had a 42 percent reduction in cognitive decline at 18 months, Lilly said today in a statement. In the second study, dubbed EXPEDITION 2, there was just a 20 percent reduction. When the mild patients from both studies were pooled together, Lilly said they found a 34 percent reduction in cognitive decline. Doody said researchers typically look for a 25 percent reduction when designing studies.
In one study, the treatment showed an improvement in daily activity functioning in patients with mild Alzheimer’s, though that benefit wasn’t statistically significant in the second study, Doody said.
None of the studies showed any benefit in the patients with the moderate stage of the disease.
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