How MNK-1411 works
DMD is an inherited disease caused by mutations in the gene that encodes for the dystrophin protein, which is essential for muscle structure and function. Patients experience progressive muscle weakness and degeneration. It is hypothesized that MNK-1411 can improve or preserve motor function and muscle strength by reducing inflammation and muscle damage.
MNK-1411 is available as an injectable medication containing a formulation of Tetracosactide (cosyntropin), a synthetic melanocortin receptor agonist or molecule that stimulates the activity of the melanocortin receptors. The treatment is a man-made equivalent of the adrenocorticotropic hormone, which binds to melanocortin receptors.
Activated melanocortin receptors are known to modulate the activity of immune system cells. MNK-1411 is thought to delay the progression of DMD by stimulating muscle cells through their melanocortin receptors to reduce inflammation and muscle damage.
MNK-1411 in clinical trials
MNK-1411 is being evaluated for its potential effect on motor function and muscle strength.
In a mouse model of DMD, the treatment prevented muscle inflammation, as assessed by magnetic resonance imaging, and gait abnormalities, according to a study published in the journal Neurology in April 2018.
A Phase 1 clinical trial testing MNK-1411 in healthy volunteers was completed in 2017 to select the optimal dosing for subsequent clinical trials.
A Phase 2 clinical trial (NCT03400852) testing the safety and effectiveness of the treatment is currently recruiting participants at several sites around the world. The company reported the enrollment of the first participant in this trial, called BRAVE, which aims 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 doses of a placebo twice weekly for up to 24 weeks.
The primary outcome measure is a motor performance test involving a 10-meter walk or run. Secondary outcomes include motor performance and muscle strength using other measures such as the NorthStar ambulatory assessment, the four-stair climb test, the rise-from-supine test, and quantitative muscle testing using a dynamometer. The study is expected to be completed in August 2020.
MNK-1411 has not been approved yet for any indication in the U.S or Europe but has been used outside the U.S. to treat certain inflammatory and autoimmune conditions.
The medication has received orphan drug and fast track designation from the U.S. Food and Drug Administration, and orphan medicinal product designation from the European Medicines Agency for the potential treatment of DMD.
Last updated: July 12, 2019
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