With clinical data showing that the Alzheimer's disease drug Alzhemed might slow or even stop progression of the disease, Neurochem Inc. launched a Phase III program in North America.
The randomized, double-blind and placebo-controlled trial will enroll 950 mild to moderate patients at 50 U.S. and 20 Canadian clinical centers. Patients will receive either placebo or one of two dose levels of Alzhemed for an 18-month period. Neurochem expects to start a similar Phase III trial of the drug in Europe early in 2005.
"This product is strategic to the company, and it's also strategic to the disease itself, probably more to the disease than the company in the sense that Alzhemed is a drug candidate that appears to have the potential of modifying or stopping the progression of the disease," said Claude Michaud, the Montreal-based company's chief financial officer.
Phase II data of Alzhemed show the drug addresses not only the symptoms of disease, but also has potential to affect its progression, particularly in mild Alzheimer's disease (AD) patients. Current therapies treat only the symptoms of the disease.
"What's available at the moment is really memory boosters," Michaud said, "but they don't last more than 12 months because the disease continues to progress."
Orally administered, Alzhemed is a small organic molecule that has been designed to modify the course of AD by binding to amyloid-beta protein and keeping it in a non-fibrillar form. It is expected to act by preventing and stopping the formation and deposit of amyloid fibrils in the brain, and by inhibiting the inflammatory response associated with amyloid build-up in AD. Neurochem licensed the drug's original scientific platform from Queens University in 1993.
The Phase II trial was conducted in 58 patients and was not large enough to demonstrate statistical significance, Michaud said.
"However, results are what I would say are good enough," Michaud told BioWorld Today. "Indications are strong enough to help us make the decision to go ahead and do the Phase III."
Neurochem made the decision to move into Phase III last year, but is just now recruiting patients. Michaud said the trial should be completed in late 2006 or early 2007, and a new drug application filing in the U.S. would follow positive results.
Alzhemed has been shown in preclinical studies to target the amyloid peptide, which researchers have called the most promising target in AD therapeutics. A Phase II study showed the product is well tolerated in AD patients. Patients receiving the therapy, particularly those with the mild form of the disease, for up to 16 months have demonstrated stability in cognitive function tests.
Phase II data also displayed no apparent safety concerns. And Alzhemed was detected in the cerebrospinal fluid of patients, suggesting its ability to cross the blood-brain barrier and to act on amyloid.
Estimated to cost the U.S. about $100 billion per year, AD is the most common cause of dementia among people aged 65 and older. Scientists estimate that up to 4.5 million people in the U.S. suffer with the disease. About 360,000 new cases will occur each year in the U.S.
"That's only the U.S. market," Michaud said. "So around the world, as the population is aging AD becomes even a more significant problem than we're trying to tackle here."
Aside from Alzhemed, Neurochem is studying Fibrillex in a Phase II/III trial to treat amyloid A amyloidosis, and Cerebril in a Phase II trial to prevent hemorrhagic stroke caused by cerebral amyloid angiopathy.
As of March 31, the company reported cash, cash equivalents and marketable securities of C$68.4 million (US$50.1 million). Last September, Neurochem priced its initial public offering in the U.S., raising $62.5 million. (See BioWorld Today, Sept. 19, 2003.)
Its stock (NASDAQ:NRMX) rose 28 cents in the U.S., to close at $21.78.