A major collapse in the Alzheimer's drug world generated buzz around the pharma industry about what comes next. Eli Lilly ($LLY), of course, stands alone with a late-stage drug for slowing progression of the memory-thieving illness, now that Pfizer ($PFE) and Johnson & Johnson ($JNJ) threw in the towel Monday on a Phase III program for their blockbuster contender bapineuzumab or "bapi."
Bapineuzumab's failure in two late-stage studies has left many industry watchers scratching their heads over the utility of attacking beta amyloid--the Alzheimer's-associated protein targeted by bapi and Lilly's late-stage candidate solanezumab--after toxic deposits have already damaged the brain. And with analysis giving Lilly's drug a one in 10 shot at success, the spotlight has shifted toward other targets and treatment strategies such as giving at-risk patients beta-amyloid drugs as a preventive measure.
Pfizer and J&J have revealed that bapi failed to beat placebo in helping patients with mild or moderate disease with their memories in two late-stage studies, but the drug giants haven't shed light on how well the drug wiped out deposits of beta amyloid. Researchers are expected to scrutinize such findings next month when more details from the trials are presented at a medical meeting in Europe, with beta amyloid on trial as a worthy target for combatting the poorly understood illness.
"Even though the scientific rationale might still be valid and strong and not adequately tested in the phase of the disease where you might expect the therapy to work, that may be lost to investors," Dr. Ronald Petersen, director of the Mayo Clinic Alzheimer's Disease Research Center in Rochester, MN, told Reuters. "I hope that is not the case."
Lilly certainly has a lot riding on the beta-amyloid target with late-stage data on solanezumab expected next month, as the antibody drug could become a mega-blockbuster if successful and begin to replace revenues lost as generics competition plagues the Indianapolis-based drugmaker's two top drugs Zyprexa and Cymbalta. CEO John Lechleiter looks like a genius for swearing off huge mergers if the drug becomes a hit and an unwise victim of the patent cliff if the compound fails. Good or bad, the results from Lilly's late-stage trials for the drug are likely to be a turning point for the drugmaker.
A win "would be euphoria, crazy party-time" for Lilly, Mark Schoenebaum, an analyst at ISI Group, told Bloomberg. "It could be the biggest upside surprise in the history of the pharmaceutical and biotech industry." Yet he and many of his colleagues don't expect to see Lechleiter and Lilly celebrating.
Even after the bapi disaster, drugmakers have every reason to keep digging for gold in the Alzheimer's field; More than 5 million Americans suffer from the disease and there are no approved therapies that slow the progression of the illness. After solanezumab, there are no such drugs in late-stage development. And the elusive winner that patients and the industry hope to find could come from treatments that home in on other suspected disease drivers such as tau or focus on at-risk patients.