CAMBRIDGE, Mass. (TheStreet) -- Researchers on behalf of Biogen Idec (BIIB) - Get Report on Friday will present detailed results for the first time from an early-stage study of the company's experimental drug BIIB037 in patients with the mildest forms of Alzheimer's disease.
Friday's presentation is important because it will fill in key details about BIIB037 that were left unanswered last December, when Biogen first told investors the drug demonstrated a statistically significant reduction in amyloid levels and improved cognition compared to a placebo.
The successful phase I study was small, and Biogen still must conduct larger phase III studies to confirm the positive effect of BIIB037 on Alzheimer's patients. (The company announced in December that it was "fast tracking" the drug, and skipping the phase II study.) But Biogen has added $25 billion in market value since the December announcement -- a sign investors are willing to believe BIIB037 might succeed where several other similar Alzheimer's drugs have failed.
The rising incidence of Alzheimer's in an aging population would turn any drug which can significantly slow or reverse the loss of memory and function seen with the devastating disease into a multibillion dollar commercial blockbuster.
BIIB037 is a monoclonal antibody that targets beta amyloid, a sticky protein that clumps together to form nerve-deadening plaques in the brain. Scientists have long assumed that sopping up beta amyloid in the bloodstream and removing the amyloid plaques from the brain should have a beneficial effect on Alzheimer's patients. The problem is that the "beta-amyloid hypothesis" has never been verified.
Pfizer (PFE) - Get Report and Johnson & Johnson's (JNJ) - Get Report bapineuzumab, Eli Lilly's (LLY) - Get Report solanezumab, and most recently Roche's (RHHBY) gantenerumab -- all beta amyloid targeting monoclonal antibodies -- all failed in phase III studies as Alzheimer's treatments, despite promising earlier data.
Biogen designed BIIB037 to better target and eliminate the "insoluble" amyloid beta which forms sticky plaques. The company also believes testing the drug in patients with the mildest forms of Alzheimer instead of more advanced disease could have a greater benefit.
The phase I study to be presented Friday enrolled just under 200 patients diagnosed with mild and prodromal Alzheimer's. The patients were then randomized to treatment with BIIB037 injected once per month or a placebo injection. Treatment lasted for 52 weeks.
In December, Biogen announced that patients treated with BIIB037 achieved a statistically significant, time and dose-dependent reduction in amyloid levels in the brain at 54 weeks, relative to patients taking a placebo. BIIB037-treated patients also had statistically significant improvements in two measures of cognition at 54 weeks: The Clinical Dementia Rating Scale Sum of Boxes Scores (CDR-SB) and the Mini Mental State Examination (MMSE.)
On safety, however, BIIB037 was associated with an increase in amyloid related imaging abnormalities, a form of brain swelling.
Researchers on Friday will pull back the curtain on the BIIB037 study to show the specific magnitude of the drug's effect on cognition.
Buyside investors surveyed by J.P. Morgan analyst Cory Kasimov are looking for a 1 to 3 point difference in the CDR-SB score between BIIB037 and placebo. Alzheimer's doctors surveyed similarly by Kasimov focus more on the MMSE cognition scale and want to see a 2 to 3 point difference between BIIB037 and placebo.
"Given that we already know [BIIB037] results are statistically significant, we anticipate the actual magnitude of separation from placebo (i.e treatment effect) will be the key factor in how the results are ultimately interpreted," Kasimov writes.
Evercore ISI analyst Mark Schoenebaum, in his own preview note, cautioned that investor expectations for the details of the BIIB037 study were "very high." He believes the BIIB037 results will be received warmly by the Street if the drug shows a minimum 30% improvement versus placebo on the MMSE and a 20% improvement from baseline on the CDR-SB.
Those cognitive improvement benchmarks are important because they would match or exceed results shown by Lilly's solanezumab in mild Alzheimer's patients. Lilly conducted two large phase III studies of solanezumab which failed to show any improvement in cognition over placebo, although subsequent analyses showed the drug had a more profound and positive effect on patients with mild Alzheimer's.
"Bottom line, [BIIB037 data] needs to be clean data and optically better than solanezumab," writes Schoenebaum.
The presentation of the BIIB037 study results is scheduled for Friday at an Alzheimer's conference in France, starting at 5:35 AM EDT.
Adam Feuerstein writes regularly for TheStreet. In keeping with company editorial policy, he doesn't own or short individual stocks, although he owns stock in TheStreet. He also doesn't invest in hedge funds or other private investment partnerships. Feuerstein appreciates your feedback; click here to send him an email.