Success in Phase 1 Alzheimer's trial adds $40 bn

Biogen (BIIB) yesterday announced successful results from an early-stage trial of its drug, aducanumab, in patients with minimal to mild Alzheimer’s disease symptoms.

The Phase 1 trial of 166 patients, who were tracked over 54 weeks, demonstrated a reverse in build-up of beta amyloid in the brain. Beta amyloid is a protein fragment that creates plaque in the brain and is a marker of Alzheimer’s. After a year at the highest dose of treatment, PET scans showed that plaque was reduced to a level that was “very close to the upper limit of normal”. However, at the lowest dose, the reduction wasn’t found to be statistically significant. The trial also demonstrated a reduction in cognitive decline. Based on the Clinical Dementia Rating, patients in the placebo group worsened by an average 2.04 points after one year, compared with 1.7 points among patients who took the lowest dose of aducanumab and 0.59 points in the highest dose.

The drug had side effects, notably brain swelling known as amyloid-related imaging abnormalities, or ARIA, that can cause headaches. Among patients carrying the ApoE4 gene, which is strongly linked with Alzheimer’s, 55 percent of patients taking the trial’s highest dose of 10 mg reported ARIA, compared with 5 percent among those in the 1 mg dose group. That resulted in 35% of the ApoE4 carriers on the highest dose discontinuing treatment.

It is thought that Biogen's trial design may have avoided the pitfalls that have seen other therapies fail in clinical trials (Roche has been the latest casualty. It abandoned its Phase 3 trial in Alzheimer’s last December on the grounds of futility). Biogen’s trial focused on patients in the early stage of Alzheimer’s disease, and first made sure trial participants  had beta amyloid in the brain (by screening with PET scans) to verify they had Alzheimer’s.

Although the company first announced in December that aducanumab had produced a statistically significant effect on cognition, it hadn’t released detailed results until yesterday, when data from the study was presented at a medical meeting in France. So pleased is Biogen with its data that it says it will skip midstage testing and move directly into a final-stage trial of more than 1,000 patients, which will begin later this year.

The market has reacted enthusiastically to the news that there may finally be a drug that is able to address the underlying cause of Alzheimer’s.  Since the preliminary findings from the Alzheimer’s trial were released last December, Biogen’s market capitalization has risen by US$40 billion.



http://www.bloomberg.com/news/artic...-s-drug-slows-progression-of-disease-in-trial

http://www.wsj.com/articles/biogen-details-data-from-early-stage-alzheimers-drug-study-1426844101


http://fortune.com/2015/03/20/how-biogen-scored-the-most-successful-alzheimers-drug-so-far/

http://www.forbes.com/sites/matthew...d-inspire-new-hope-for-alzheimers-treatments/