Luca Santarelli, Roche's head of neuroscience

The drug: Gantenerumab
The disease: Alzheimer's
The developers: Roche, MorphoSys AG
Peak sales potential: The sky's the limit.

No list of top late-stage blockbusters would be complete without at least one Alzheimer's hopeful in the mix. After "bapi" and solanezumab flunked their primary endpoints, Roche ($RHHBY) decided to go back and double the size of the study for gantenerumab after regulators indicated that they could potentially use it as a pivotal study.

As Luca Santarelli, Roche's head of neuroscience, explained to us recently, investigators will be relying on biomarkers found in spinal fluid to identify presymptomatic patients. Now that the beta amyloid theory--the notion that reducing the level of the toxic protein in patients can delay or ameliorate the disease--has been found wanting among patients with an established case, scientists are now clamoring to see if early intervention with a safe compound could be the answer.

In a small early-stage study that came out in late 2011, the antibody successfully lowered levels of beta amyloid. Now researchers will see if preventing accumulations could delay or prevent the disease. Data from the pivotal study is now expected in 2015.

Any Alzheimer's drug that makes it through the pipeline will be worth anywhere from $8 billion or $9 billion-plus per year. The drugs on the market now offer little hope to the millions of patients with this disease. But the odds remain nothing less than terrible--with only three approvals and more than 100 defeats.

For more:
Pharma counts just 3 Alzheimer's drug wins in 13 years (101 losses!)
Roche stakes its claim as the leader in high-risk Alzheimer's R&D
Big Pharma's high-risk pursuit of CNS gold faces major market doubts
Roche spotlights PhI efficacy of Alzheimer's antibody


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