Genentech readies for groundbreaking $100M trial of Alzheimer's therapy

Cross-section of a normal brain (right) and one damaged by advanced AD (left)--image courtesy of Merck & Co. (AP Photo)

Within months, Roche's ($RHHBY) Genentech and its collaborators will embark on the biggest test yet for an experimental therapy that they hope could slow the onset of Alzheimer's in at-risk patients. And the Los Angeles Times took a closer look at the $100 million effort this week, homing in on the rural town in Colombia where many of the pioneering study's patients will come from.

The mystery around the cause of Alzheimer's has befuddled Big Pharma for years. Amyloid proteins in the brain have drawn considerable interest as a culprit in the memory-stealing disease with mostly negative results. An amyloid-targeting drug from Eli Lilly ($LLY) and another from Johnson & Johnson ($JNJ) and Pfizer ($PFE) both failed in large late-stage studies this year. Yet both those efforts involved patients whose disease had already advanced.

Genentech hasn't given up on the amyloid case, hoping to show that its amyloid-inhibiting antibody crenezumab could slow down progression of Alzheimer's in at-risk patients who don't yet have dementia. Next spring, as the L.A. Times reported, about 100 people from Colombia who have a gene that has been linked to early-onset Alzheimer's will begin treatment on Genentech's antibody as part of a wider 5-year trial funded by biotech giant, the NIH and Banner Alzheimer's Institute. The groups announced plans for the trial earlier this year.

The design of the $100 million study calls for 100 additional patients with the same Alzheimer's-related gene to take a placebo and an equal number of other at-risk patients without the gene to take crenezumab, as the newspaper reported. A branch of the trial will include U.S. patients as well. The results should provide some solid evidence on the merits of targeting amyloid at an early stage or even before patients show signs of dementia.

"Anti-amyloid drugs have been used before, but they have failed because they have been taken by patients already suffering from dementia--in other words, too late," Dr. Francisco Lopera, a co-manager of the trial based in Colombia, said, as quoted by the newspaper. "We are optimistic. We aren't sure it will prevent the disease, but it could delay it for many years, and that's important."

Roche/Genentech, which licensed crenezumab from Swiss biotech AC Immune (a 2012 Fierce 15 company), has a bevy of candidates against Alzheimer's disease in the pipeline as the company pursues one of the biggest opportunities in pharma. U.S. cases of the disease exceed 5 million and are expected to grow as the population ages. Yet there are no therapies that can even delay progression of the disease on the market.

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