So which take-away message is the most likely scenario for the Eli Lilly experimental treatment for Alzheimer's? Is this a battle between hype and hope? The drugmaker yesterday released full study results and emphasized that its solanezumab compound slowed cognitive decline in patients with mild disease. While there was no statistically significant reduction in functional decline, some Wall Street analysts are upbeat, if cautious, about the prospects.
"The odds that solanezumab may become the first 'disease modifying' drug have risen, yet ascribing exact odds of success remains difficult," write Sanford Bernstein analyst Tim Anderson in an investor note. "...In our prior reports we put the odds of technical success at 20 percent. It would not be a stretch to say that this figure has doubled to 40 percent, yet solanezumab still remains a Phase III asset where the risk is likely higher than normal."
Just two months ago, Lilly (LLY) released top-line results indicating solanezumab failed to meet the primary endpoints, both cognitive and functional, in a pair of Phase III double-blind, placebo-controlled trials in patients with mild-to-moderate Alzheimer’s disease (back story). The complete results indicated a pre-specified secondary analysis of pooled data yielded a 34 percent reduction in cognitive decline in patients with mild Alzheimer’s, which was statistically significant.
The findings did not generate the sort of excitement that is being reserved for a cure, but were sufficient for Maria Carillo, the chief science officer at the Alzheimer's Association to express some optimism "It’s certainly not the home run we all wanted, but we’re very encouraged by these results,” she told the Associated Press.
For its part, Lilly was, not surprisingly, doing its best to put an optimistic face on the results. The drugmaker, after all, emphasized the analysis of pooled data, which was undertaken because statistically significant cognitive decline was only found in one of the two clinical trials (look here). This may be kosher, but only suggests that further trials will be needed if the drugmaker has a reasonable chance of winning FDA approval.
"Despite an 'excellent' safety profile, clinicians (we spoke with believe a disease-modifying effect must be confirmed in a second and, perhaps, longer Phase III study to argue for reimbursement of a potentially costly, once-monthly IV-administered product that delivers a limited functional benefit after 18 months of treatment," writes Leerink Swann analyst Seamus Fernandez in an investor note. He looks for a trial that will run up to three years and involve as many as 2,000 patients.
[UPDATE: A survey of 111 buy-side fund managers by ISI Group analyst Mark Schoenebaum finds that 54 percent expected these results, 11 percent say they were worse than expected and 35 percent believe they were better than expected. And on a scale of 1 to 10, with 1 being horrible and 10 being phenonemal, the results notched a 4.4. And only 21 percent believe Lilly could win FDA approval based on the results.]
It is not surprising that Lilly plans to speak with the FDA about how to proceed, though. Like its rivals, the drugmaker has been facing the so-called patent cliff, in which big-selling medicines are gradually losing patent protection and facing lower-cost generic competition. Moreover, Lilly has placed a big bet on its Alzheimer's program and to scrap its research - as Pfizer and Johnson & Johnson recently did (see here) - would weaken its strategy.
As Anderson notes, the Aricept pill, which is made by Eisai and sold by Pfizer, managed to generate about $4 billion in annual sales, even though the drug only improves symptoms and does not not have an effect on the progression of the disease. "A disease modifying therapy like solanezumab could well have higher pricing and therefore higher sales, even if the efficacy benefits only start out modestly but assuming they increase with continued usage," he writes. In other words, there is plenty of upside for Lilly to push ahead. Meanwhile, by emphasizing the possibility that mild Alzheimer's patients may benefit from early-stage treatment is a way to generate public support, of sorts, for its program. There is a tremendous unmet medical need for Alzheimer's treatments and FDA actions will be closely watched by millions of people who not only hope the Lilly compound can yield some benefit, but that the agency will work to ensure an effort is made - and not kill their hopes. What do you think?
Are The Solanezumab Results More Hype Than Hope?
- Yes (73%, 108 Votes)
- No (27%, 41 Votes)
Total Voters: 148
question mark pic thx to purpeslog on flickr