ACZ885 (canakinumab) is an investigational therapy for sickle cell anemia being developed by Novartis.

How ACZ885 works

Sickle cell anemia is an inherited disorder of red blood cells. These disc-shaped cells contain a protein called hemoglobin to which oxygen binds. Red blood cells travel through blood vessels to deliver oxygen to organs and tissues throughout the body.

Mutations in the HBB gene, which provides the instructions to make hemoglobin, causes an abnormal hemoglobin to be produced. This alters the shape of the affected red blood cells, making them adopt a sickle-like shape instead of a disc shape. These abnormal cells struggle to move through blood vessels and can become trapped, restricting blood flow, reducing oxygen supply, and triggering inflammation. Inflammation can cause heat, swelling, and pain at the affected site. In patients with sickle cell anemia, inflammation can be one cause of chronic pain and sudden debilitating pain crises.

ACZ885 acts as an anti-inflammatory. It is an antibody, or a protein specially designed to interact with a specific target. ACZ885 binds to and inhibits the action of a protein called interleukin-1 beta (IL-1β), which normally works to trigger inflammation. Through this mechanism, ACZ885 should reduce inflammation in sickle cell anemia and potentially reduce the pain experienced by patients.

ACZ885 in clinical trials

A Phase 2 clinical trial (NCT02961218) is currently recruiting up to 60 children and young adults with sickle cell anemia in the U.S, Germany, Israel, South Africa, Turkey, and the U.K. The placebo-controlled, double-blind study aims to assess the safety and tolerability of ACZ885, and its effect on daily pain compared to a placebo.

Patients, ages 12 to 20, will receive six doses of either a placebo or ACZ885 (300 mg) over a 24-week period, delivered as an injection under the skin. They will then be given the option to continue for a further six doses in an open-label phase of the trial.

The primary outcome will be to assess any reduction in the average daily pain after 8–12 weeks, compared to pain levels at baseline or study start. Researchers will also evaluation reported reductions in pain after 24 weeks of treatment, and whether this translates into fewer missed days from work or school due to pain.

The trial is expected to be finish in June 2020.

Other information

Canakinumab is marketed under the name Ilaris to treat juvenile arthritis and periodic fever syndromes. It is also being investigated for several other conditions, including lung cancer and cardiovascular disease.

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