The immune system is the body’s defense against infection by disease-causing pathogens such as bacteria and viruses. Immune system modulation, also called immunomodulation, is the process that involves the use of therapy to modify the immune response, often to prevent tissue damage resulting from an excessive response.
Why is immune system modulation important?
The activity of the immune system needs to be finely balanced. A hyperactive immune system can lead to inflammation and autoimmune disorders.
Muscular dystrophy is a group of inherited diseases that lead to progressive muscle weakening and loss of muscular control. In some cases, the immune system can attack diseased muscle and damage tissue.
Using small molecules, the activity of the immune system can be regulated to minimize tissue damage while still allowing it to confer necessary protection.
Immune system modulators in development
Resolaris (ATYR1940) is an intravenous protein therapy designed to act on the resokine pathway to modulate the immune system and reduce muscle tissue damage in facioscapulohumeral muscular dystrophy (FSHD) and limb-girdle muscular dystrophy 2B (LGMD2B). It is being developed by aTyr Pharma.
Resolaris aims to slow T-cell activation and prevent these immune system cells from attacking muscle tissue. Phase 1b/2 clinical trials have shown that Resolaris has good efficacy and tolerability in patients with FSHD.
MNK-1411 is the synthetic equivalent of the adrenocorticotropic hormone. It is a synthetic melanocortin agonist that acts on melanocortin receptors. (An agonist is a substance that acts like another to stimulate a certain action). Active melanocortin receptors are known to modulate immune system activity.
MNK-1411, being developed by Mallinckrodt Pharmaceuticals, may delay the progression of Duchenne muscular dystrophy (DMD) by modulating immune system activity and reducing muscle inflammation. Its effectiveness in reducing muscle inflammation has been shown in animal models, and MNK-1411 was evaluated in Phase 1 clinical trial in healthy volunteers to determine optimal dosing.
An ongoing placebo-controlled, and double-blind Phase 2 trial (NCT03400852) is evaluating MNK-1411’s safety and effectiveness in about 132 boys with DMD, ages 4 to 8, at sites across the U.S., Europe, and elsewhere. Patients are being given either MNK-1411 at one of two doses twice weekly as an under-the-skin injection for up to 48 weeks, or a placebo injection for up to 24 weeks. Improvements in muscle health will be evaluated by performance tests at 24 weeks, including the 10-meter walk/run, the NorthStar Ambulatory Assessment, and the four-stair climb test. A number of trial sites may be enrolling eligible patients.
Last updated: July 26, 2019
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