American multinational biotechnology company Biogen in a reversal of its earlier decision to halt the study of an Alzheimer's drug that it previously had said failed, will ask the Food and Drug Administration to approve it. It said a new analysis of a "larger dataset" on multiple measures of Aducanumab drug's effectiveness shows the drug reduces cognitive decline in early Alzheimer's patients.
The new finding contradicts the company's March decision to halt studies of the therapy based on the recommendations of an independent monitoring board charged with protecting patients in the study. Aducanumab, the last of a series of drugs, all failed, from many different companies, targets a protein in the brain called beta-amyloid that is the main component of the amyloid plaques found in the brains of people with Alzheimer's disease.
The disease affecting about 5.8 million Americans causes people's memory and mind to deteriorate to the point where they cannot function on their own and eventually die. It's the sixth leading cause of death in the US. Most researchers and biotechnology executives after the March decision of the company saw little hope for a drug helpful for patients with Alzheimer's disease that affects tens of millions of people worldwide, according to StatNews.
The company in a new analysis in consultation with the Food and Drug Administration (FDA) of a larger data set from the discontinued studies included additional data available after the previous analysis showed the studies were "futile." The company said the new data showed Aducanumab was "pharmacologically and clinically active" and that it reduced patients' clinical decline based on the results of a survey called Clinical Dementia Rating-Sum of Boxes (CDR-SB).
Biogen chief executive Michel Vounatsos in a statement said the company was "hopeful about the prospect of offering patients the first therapy to reduce the clinical decline of Alzheimer's disease and the potential implication of these results for similar approaches targeting amyloid beta".
Al Sandrock, Biogen's head of research and development and chief medical officer, said it felt "amazing to have this change". Sandrock said as researchers analyzed the full dataset by June, they started to realize a different picture of Aducanumab was emerging. The company said it initially was worried about a potential side effect — brain swelling — and limited the dosage of the drug but later allowed patients to have a higher dose of the medicine.
Sandrock said the futility analysis had happened too soon, and the company after March halt ran two studies – one was positive in its own right for the high dose, and a second still failed but showed signs of benefit in those patients who received the higher dose of Aducanumab. Patients, in a study called Emerge, had a 23 percent reduction in their rate of decline when on the high-dose Aducanumab as compared to those on placebo.
The statistically insignificant results of those on the low dose suggested the reduction was 11 percent lower than the placebo. But in the second trial called Engage, the decrease was only two percent in patients on the high dose. According to Sandrock, it will be up to the FDA to decide whether to approve the drug based on a single positive trial.
Umer Raffat, an analyst at the investment bank Evercore/ISI, asked whether, in a way, one trial was successful and another had failed. Raffat, however, noted the patients who did not complete seemed to show benefit as well.
