Lundbeck stakes out a leading role for itself in late-stage Alzheimer's R&D

While Alzheimer's has long reigned as one of the most challenging fields in R&D, it has also inspired some of the most confident projections in the industry. And now Lundbeck is joining the ranks of the optimists.

Lundbeck and its partner Otsuka are running a slate of late-stage studies for Lu AE58054, and they are on track to file for an approval in 2017--if their drug works at improving cognition and daily functions of Alzheimer's patients.

"If the studies that we are currently running end well, then we will probably be the first company to launch a new Alzheimer's drug in 10 to 15 years," Lundbeck's Chief Scientific Officer Anders Gersel Pedersen confidently told Reuters.

What he evidently didn't mention are the odds that he faces. Alzheimer's has defied a string of attempts by some of the world's biggest pharma companies--Johnson & Johnson ($JNJ), Pfizer ($PFE) and Eli Lilly ($LLY) among them. In fact, more than 100 studies have gone down in flames, and so far there is no drug that can be used to really get at the root cause of the disease. But Lundbeck and Otsuka may improve their odds considerably with an approach that steers clear of two of the primary targets that have vexed researchers: amyloid beta and tau.

Their drug plays on a G protein-coupled receptor found almost exclusively in the brain which plays a role in cognition, among other things. Blocking it can stimulate the release of dopamine and norepinephrine in the frontal cortex, offering a new approach to treating the memory-destroying disease.

A few months ago investigators said they tracked a clear, statistically significant improvement in the combo arm of the Phase II study, which compared results for Lu AE58054/donepezil with a control arm that was provided donepezil and a placebo. The primary endpoint was ADAS-Cog, which measures the severity of the most important symptoms, at week 24. The drug failed to distinguish itself for secondary endpoints on two other measures, though the companies say the trial was not designed to demonstrate statistically significant improvements on either of those measures.

Pedersen also notes that the drug would be worth more than $1 billion a year in sales, which is something of an understatement. Given the millions of patients who are desperate to see some new treatment come along, most analysts expect that any new drug that can work for patients would easily earn more than $5 billion a year. -- John Carroll, Editor-in-Chief. Follow me on Twitter and LinkedIn.