Alzheimer’s is a debilitating neurodegenerative disorder affecting approximately 5 million Americans, yet its causes are remarkably enigmatic. Nonetheless, pharma and biotech companies continually throw money at potential blockbusters that lack innovative scientific forethought and inevitably fail to meet primary endpoints in clinical trials. Their gamble is over a piece of the $203 billion pie that the Alzheimer’s Association estimates as a 2013 cost of care (rising to an astounding $1.2 trillion in 2050). In the spirit of President Obama’s Brain Initiative, the private sector should instead invest in the development of novel diagnostic tools to better define potential drug targets and disease progression.
Just this month Baxter International Inc. (NYSE:BAX) announced results of its Phase 3 trial examining the effects of Gammagard, an immunoglobulin bolus from healthy donors aimed at clearing amyloid-beta from the brain before it aggregates into neuron-killing plaques. Gammagard failed to meet its primary endpoint of an improvement in cognitive or functional deficiencies in patients diagnosed with Alzheimer’s. It did appear to at least slow the progression of cognitive decay over the course of the trial, but those results were not statistically significant. Baxter International Inc. (NYSE:BAX) is still contemplating the fate of Gammagard for Alzheimer’s treatment.
Such was the fate of Eli Lilly & Co. (NYSE:LLY)‘s solanezumab, which failed to meet primary endpoints but showed promise for a pre-specified secondary outcome, a slowing of cognitive decay with time, as well as a reduction in some, but not all, biochemical markers of disease. This result was as positive as the field has seen for some time, and the stock rallied on the announcement that the EXPEDITION trial would be extended as a longitudinal study of disease progression and drug safety over extended periods.
Comparable results have been seen by other groups like Pfizer Inc. (NYSE:PFE) and Johnson & Johnson (NYSE:JNJ), who discontinued trials of their intravenous drug bapineuzumab after it failed to reach similar endpoints last year. Those trials looked specifically at patients both with and without a gene that enhances the likelihood of disease. Further trials using alternative methods of drug application are still underway.
In summary, large investments in novel drugs have produced incremental advances in Alzheimer’s treatment, but have not yielded the blockbuster investors are hoping for. Perhaps it is time to step back and reevaluate short-term goals in the context of long-term investments in drug discovery.
Can diagnostics generate revenue?