Novartis plans to test Alzheimer's drugs in patients without symptoms

Learning the lessons of failed trials in mild, moderate and severe Alzheimer's disease, Novartis ($NVS) is planning to study two new treatments on patients who are yet to show any symptoms, hoping to succeed where many others have slipped and delay the onset of the memory-destroying ailment.

In collaboration with the Banner Alzheimer's Institute, Novartis plans to study whether its two treatments--an immunotherapy and a BACE inhibitor--can prevent the buildup of amyloid proteins, considered by many to be a cause of Alzheimer's development. The first treatment, an injectable called CAD106, is designed spur the immune system's natural development of amyloid-blocking antibodies and is in Phase II on its own. The second drug, yet to enter clinical trials, is an oral inhibitor of an enzyme tied to amyloid production.

The plan is to recruit more than 1,300 cognitively healthy adults who have two copies of the APoE4 gene, which increases their risk of developing the disease. Participants will get either CAD106, the BACE inhibitor or placebo, and Novartis hopes to get started next year, pending regulatory approval.

"This trial reinforces Novartis' focus to following the science of the disease and outlines our continued commitment to the study of Alzheimer's disease," Novartis pharma chief David Epstein said in a statement. "There is a huge unmet need for treatments that prevent or delay the development of the disease, and we are excited about taking research in Alzheimer's to the next level."

Drug developers have been looking to patients at earlier and earlier stages of Alzheimer's after witnessing costly Phase III failures for drugs from Eli Lilly ($LLY), Johnson & Johnson ($JNJ) and Pfizer ($PFE) that target patients already damaged by the disease. Roche's ($RHHBY) Genentech is already taking a similar tack with the amyloid-fighting crenezumab, in 2012 teaming with the National Institutes of Health to study its effects on healthy patients genetically disposed to the disease.

Despite years of work and billions in R&D costs, Big Pharma has repeatedly failed to work up a disease-modifying treatment for Alzheimer's, with all approved therapies treating only its symptoms. From 2002 to 2012, a galling 99.6% of clinical trials in Alzheimer's ended in failure, according to the Cleveland Clinic.

But the prospect of becoming the first on the market with an approved therapy that treats the root cause of Alzheimer's has been enough to keep Big Pharma interested, spurring major investments from Merck ($MRK), GlaxoSmithKline ($GSK), Biogen Idec ($BIIB) and others.

- read the statement