Although the treatment has not been approved yet for any indication in the U.S., it is used elsewhere to treat certain inflammatory and autoimmune conditions. However, it has received orphan drug designation from the U.S. Food and Drug Administration (FDA) for the treatment of Duchenne.
How MNK-1411 works
MNK-1411 is an injectable medication composed of a formulation of Tetracosactide (cosyntropin), a synthetic melanocortin receptor agonist, or molecule that can stimulate the activity of the melanocortin receptor. It is a man-made equivalent of the adrenocorticotropic hormone (ACTH), which binds to melanocortin receptors.
Activated melanocortin receptors are known to modulate the activity of cells of the immune system. It is thought that MNK-1411 may delay the progression of DMD by stimulating cells through their melanocortin receptors to reduce inflammation and diminish muscle damage.
MNK-1411 in clinical trials
MNK-1411 is being evaluated for its potential effect on motor function and muscle strength and has been shown to have potential therapeutic effects on mouse models of DMD.
A Phase 1 clinical trial testing MNK-1411 in healthy volunteers was completed in 2017. A Phase 2 clinical trial (NCT03400852) testing the safety and effectiveness of the treatment in DMD patients is now being conducted in Atlanta, Georgia, and Las Vegas, Nevada and is still recruiting participants. The trial is expected to enroll 132 boys with DMD, ages 4 to 8. The patients will be divided into four groups. The first two groups will receive different doses of MNK-1411 twice weekly for up to 48 weeks and the other two groups will receive different placebo suspensions twice weekly for up to 24 weeks.
The primary outcome is a motor performance test involving a 10-meter walk or run. Secondary outcomes will be other measures of motor performance and muscle strength such as the NorthStar ambulatory assessment, four-stair climb test, rise from supine test, and quantitative muscle testing using a dynamometer. The study is expected to be completed in April 2021.
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