Category: Clinical Trials

BREAKING NEWS: Alexion, AstraZeneca Rare Disease, has announced that the Phase 3 CARES clinical trial did not meet its main goal. Anselamimab did not show a statistically significant improvement in survival during the study period.

The goal of the CARES study was to find out if the investigational drug anselamimab could help newly diagnosed AL amyloidosis patients live longer. Sadly, the drug did not show a significant benefit during the study period. Though this main goal of the trial was not met, anselamimab was considered generally safe and well-tolerated.

While the study found no significant improvement in overall survival, researchers did see signs that the drug may help some patients live longer and reduce their frequency of hospitalizations. These findings were seen in a specific subgroup of participants, but not across the full study population. Alexion, AstraZeneca Rare Disease, the company behind the trial, says more detailed analysis of these findings is still underway and they plan to share results with global health agencies and present their findings in the near future. We will keep you updated as we learn more.

Ashutosh Wechalekar, MD, Consultant Haematologist at University College London Hospitals NHS Foundation Trust (UCLH), and lead principal investigator for the CARES trial said, “While the study did not meet the primary endpoint in the overall patient population, results from a pre-defined subgroup suggest that anselamimab, by targeting and clearing amyloid deposits, may address a leading cause of organ damage and functional impairment in these patients.”

We know this news may be hard. If you or a loved one is affected and need support, please reach out to us at 617-467-5170 or support@arci.org. ARC remains deeply committed to driving research forward and building a path to more and better treatments for people living with AL amyloidosis.

What is AL Amyloidosis?

AL amyloidosis is one of the most common types of amyloidosis and involves proteins called light chains. Light chains make up part of the structure of immunoglobulins (antibodies) which play an important part in the immune system. They are produced by plasma cells in the bone marrow. In AL amyloidosis, abnormal plasma cells make excessive amounts of abnormal light chain proteins. Instead of forming immunoglobulin, they become misfolded and deposit as amyloid in various organs of the body.

What was the CARES Study?

The CARES study was a phase 3 clinical trial evaluating anselamimab, an investigational “anti-fibril” drug designed to improve organ function by reducing or eliminating amyloid deposits in the tissues and organs of patients living with AL amyloidosis. The trial enrolled 406 patients across 19 countries around the world.

ARC and the amyloidosis community thanks those who participated in this and all clinical trials, which play a vital role in advancing treatment. Each trial, regardless of outcome, provides critical information that helps the entire field move forward. Negative results still teach us valuable lessons about the disease and what’s needed in future drug development efforts.

Read the press release from Alexion, AstraZeneca Rare Disease, here.

Learn about AL amyloidosis here.