Proof-of-concept efgartigimod trial for AAV expected this year

Argenx therapy approved as Vyvgart for another autoimmune disease

Margarida Maia, PhD avatar

by Margarida Maia, PhD |

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Argenx is planning to launch a proof-of-concept clinical trial later this year to test its therapy efgartigimod, approved to treat another autoimmune disease, in people with ANCA-associated vasculitis (AAV).

Data from proof-of-concept studies can help companies and their researchers decide whether to develop a medicine for a specific indication.

Efgartigimod was approved, under the brand name Vyvgart, in late 2021 for certain patients with generalized myasthenia gravis (gMG).

The announcement of the new study, in AAV, was part of an Argenx business update. The company said it plans to launch the trial between October and December of this year. However, no further details on the upcoming trial were disclosed.

“By the end of 2023, efgartigimod is expected to be approved, in regulatory review or in development in 13 severe autoimmune diseases,” Argenx said in the update.

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Argenx is testing efgartigimod for range of autoimmune diseases

Antibodies, or immunoglobulins, are proteins produced by the immune system to help protect against any substance that comes from outside the body and that can potentially be damaging. These immunoglobulins fall into five classes: IgG, IgA, IgM, IgD, and IgE.

In autoimmune diseases such as AAV, the body’s immune system makes antibodies that wrongly recognize and attach to its own cells or tissues. These are called self-reactive antibodies or autoantibodies, and they usually belong to the IgG class.

AAV is characterized by the production of anti-neutrophilic cytoplasmic autoantibodies, or ANCAs. These self-reactive antibodies mistakenly attack neutrophils, a type of immune cells, making them go haywire and causing inflammation and damage to small blood vessels.

Argenx’s efgartigimod works by blocking the action of the neonatal Fc receptor (FcRn), a protein that usually helps stabilize and prevent the destruction of IgG antibodies circulating in the blood. By suppressing FcRn, the therapy is expected to lower the number of self-reactive IgG antibodies that cause autoimmune diseases such as AAV.

Efgartigimod, sold as Vyvgart, is approved for adults with gMG, a disease in which self-reactive antibodies against a protein critical for nerve-muscle communication lead to muscle weakness and tiredness.

In that patient population, the medication has been shown to improve the ability of muscles to contract and to reduce disease symptoms and their impact on daily activities.

We have an exciting year ahead as we grow Vyvgart as a new standard of care in gMG [and] leverage its potential in other indications.

Now, the company is looking to “expanding the scope of IgG-mediated autoimmune diseases in development and further demonstrating the potential of FcRn blockade in ongoing clinical trials,” the business update stated.

Besides AAV, Argenx is running clinical trials of efgartigimod in a range of other autoimmune diseases, such as primary immune thrombocytopenia, chronic inflammatory demyelinating polyneuropathy, and primary Sjögren’s syndrome.

“We have an exciting year ahead as we grow Vyvgart as a new standard of care in gMG, leverage its potential in other indications and progress our broader immunology pipeline, all of which brings us one step closer to achieving our goals and innovating on behalf of patients,” Tim Van Hauwermeiren, CEO of argenx, said in the business update.