BIIB037

Aducanumab

Neurology/psychiatric - Alzheimer’s disease

All eyes are on aducanumab, which could become the first disease-modifying therapy (DMT) for Alzheimer’s disease (AD), a landmark achievement following decades of failure in this perennially underserved market.

About aducanumab

  1. Developed by Biogen Inc and Eisai Co Ltd
  2. Recombinant chimeric human IgG1 mAb targeting beta-amyloid
  3. Priority review by FDA; November 2020, negative review by FDA Advisory Committee that available clinical trial evidence does not prove effectiveness
  4. After submission of additional data and analysis, PDUFA date extended by three months to June 7, 2021 (as of Jan 29, 2021)
  5. Undergoing standard MAA/NDA review by the EMA and PMDA
$3.74B
expected sales in 2025

About the Alzheimer’s disease market

  1. Currently only symptomatic therapy: AChEIs and memantine, now generic, are the standard of care across mild, moderate and severe disease
  2. No novel therapies approved in >15 years
  3. Other key anti-beta-amyloid DMTs in late-phase development: lecanemab (BAN2401; Eisai/Biogen) and gantenerumab (Roche)
  4. Many more drugs from a range of MOAs (e.g., tau-based therapies, sigma-1 receptor inhibitors and SIGLEC3 and Trem2 antibodies) are in mid- and late-phase trials
~50 million
people with AD globally

Unmet patient needs

The most critical need for patients has been a safe, effective DMT that slows cognitive decline. Tools that enable earliest possible diagnosis are also critical.

Why is it a drug to watch?

Aducanumab is the first putative DMT to demonstrate a clinical effect in early AD patients in a Phase III study and the first to undergo regulatory review.

Results from the terminated ENGAGE/EMERGE and completed PRIME trials indicate that aducanumab is biologically active and suggest the potential for efficacy with sufficient exposure to the 10 mg/kg dose.

If approved, it would mark a major clinical, commercial and regulatory milestone.

However, the overall data package is complex and contentious given the limitations associated with the data and its analysis.

>30%
expected increase in total prevalent cases of early Alzheimer’s disease by 2029 due to aging population

What you need to know

Aducanumab could unlock a monumental opportunity to radically change AD patient care and transform the market. If approved, demand for treatment will be enormous, potentially even decreasing willingness to forgo this treatment for an investigational drug in future clinical trials.

It could have a similar effect on companies’ decisions about which drugs to develop, bypassing other sorely needed MOAs to develop next-gen anti-amyloid drugs.

Nevertheless, many questions remain about the drug’s prospects, including, first and foremost, the probability of approval given the questions about its true efficacy, followed by future competition from other DMTs and overall health system preparedness—availability, cost and reimbursement of the drug and requisite diagnostic testing; patient/physician awareness to drive early presentation, specialist referral and diagnosis; and infusion infrastructure.

“I have to say that the data are convincing…there is clear clinical effect. I don’t think it’s a false positive. …aducanumab is, biomarker-wise, the strongest thing possible… from the point of MOA, biomarkers and the effect on the brain amyloid and tau… for how long would you treat patients and what their dosage would be to achieve the clinical effects? If you look closely, the limited data they have sort of parallels the observations they made during Phase I.”

Neurologist,
United States

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