Biogen ‘continues to work’ with FDA on Alzheimer's drug candidate despite regulatory setback

By Jane Byrne contact

- Last updated on GMT

© GettyImages/designer491
© GettyImages/designer491

Related tags: Alzheimer's disease, Monoclonal antibody, anti-amyloid beta

Biogen Inc’s shares tumbled around 30% yesterday with the company hitting a roadblock late last week as it chases regulatory approval for its investigational Alzheimer's treatment.

On Friday last [November 6], an independent advisory committee to the US Food and Drug Administration (FDA), in a review of the clinical data arising from two Phase 3 trials on Biogen's drug, aducanumab, announced the outcome - the clinical data doesn’t show the drug to be effective for the treatment of Alzheimer’s disease.

In August this year, the FDA accepted the aducanumab biologics license application (BLA), placing it in priority review. The advisory committee's analysis of the clinical data on the anti-amyloid beta drug forms part of the registration process.

Biogen says FDA advisory panels provide non-binding recommendations for consideration by the US regulator.

However, Wall Street analysts considered that the near-unanimous votes against the drug from those experts make it difficult for the FDA to go against the panel, according to an article on Reuters.

That piece did note, though that, bowing to pressure from patient advocates, the FDA approved a treatment for Duchenne muscular dystrophy from Sarepta Therapeutics Inc in 2016, even though an outside panel of experts and the agency's own reviewers questioned the drug's efficacy.

“The FDA will continue the review [the] process with a decision on whether to approve aducanumab by March 21, 2021,”​ added Biogen.

The biotech said it will continue to work with the pharma watchdog as it completes its review.

Development to date

Aducanumab (BIIB037) is an investigational human monoclonal antibody studied for the treatment of Alzheimer’s disease. Biogen says it is designed to treat patients with mild cognitive impairment due to Alzheimer’s disease and mild Alzheimer’s disease, and that aducanumab has the potential to impact underlying disease pathophysiology, slow cognitive and functional decline.

The biotech licensed aducanumab from Neurimmune under a collaborative development and license agreement. Since October 2017, it has collaborated with Eisai on the development and commercialization of the investigational drug globally.

In March 2019, Biogen announced that two international clinical trials of the experimental drug in patients with early stage disease, referred to as studies 301 (Engage) and 302 (Emerge) were being abandoned, after an independent data monitoring committee suggested that the trials were unlikely to meet their primary endpoint upon completion.

“This disappointing news confirms the complexity of treating Alzheimer’s disease and the need to further advance knowledge in neuroscience,”​ said Michel Vounatsos, CEO of Biogen, at the time.

However, in a rapid turnaround, in October 2019, the biotech reported that, in fact, it would be pursuing FDA approval, with it confident it had the data to show the antibody works.

It said then that patient data, not included in the earlier analysis, indicated that after 78 weeks, the cohort getting the higher of two aducanumab doses in the Emerge trial had 22% less cognitive decline on a standard dementia assessment than those in a placebo group.

If approved, aducanumab would become the first new treatment for Alzheimer’s disease (AD) for many years; it would likely bring in tens of billions of dollars in sales for Biogen

In February, we reported that the developer was readying manufacturing operations to meet anticipated “large demand”​ for the drug.

Roche and AC Immune hit AD drug trial stumbling block  

Biogen is not the first company to struggle to make headway in the search for an Alzheimer’s treatment.

In September, we heard a drug targeting the neurodegenerative disorder from partners AC Immune and Roche failed a mid-stage clinical trial.

The drug, semorinemab, was tested in patients with early Alzheimer’s disease. It did not achieve the Phase 2 study’s main goal of coming out better than a placebo in slowing decline according to a dementia scale, reported AC Immune.  

Meanwhile, Amylyx Pharmaceuticals, Inc., a pharmaceutical company focused on developing new treatments for amyotrophic lateral sclerosis (ALS), Alzheimer’s disease and other neurodegenerative diseases, yesterday announced that the last participant has completed the planned 24 weeks in the Phase II PEGASUS trial.

That study is assessing the safety and biological activity of AMX0035 administration in people with Alzheimer's disease.

A spokesperson for Amylyx told us that, unlike previous efforts, AMX0035 targets the classic pathologies of the disorder. “The PEGASUS trial is one of the first co-formulation trials to target nerve cell death and degeneration,”​ he said.

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