MIN-102, developed by Minoryx Therapeutics, is an oral experimental medication for the treatment of adrenomyeloneuropathy (ALD).

MIN-2 is being investigated as a potential treatment for adrenomyeloneuropathy (AMN) and cerebral ALD (CALD), the two main clinical subtypes of ALD. Existing ALD therapies are only for the treatment of CALD.

The European Medicines Agency (EMA) granted MIN-102 orphan drug designation in 2016, and the U. S. Food and Drug Administration (FDA) in 2017.

How MIN-102 works

MIN-102 is a PPAR-gamma agonist, or a molecule that activates PPAR-gamma. PPAR-gamma is a protein that regulates genes involved in glucose and lipid metabolism and inflammation. MIN-102 is a synthetic compound that binds to and activates PPAR-gamma.

ALD is caused by a mutation in the ABCD1 gene that leads to the accumulation of a compound called saturated very long chain fatty acid (VLCFA) in the blood and central nervous system. VLCFA accumulation triggers an inflammatory response, which causes the destruction of myelin, the protective sheath that insulates nerve cells in the brain. MIN-102 is thought to protect nerve cells by stimulating anti-inflammatory and inhibiting pro-inflammatory genes, resulting in less inflammation and oxidative stress, and overall improved cellular function.

MIN-102 is a modified version of Actos (pioglitazone), a medication for the treatment of type 2 diabetes. MIN-102 has a superior brain penetration and safety profile compared to Actos.

MIN-102 in clinical trials

A Phase 1 clinical trial in healthy male volunteers showed that MIN-102 is well-tolerated and safe. No serious adverse events occurred, even at higher doses than needed. The trial confirmed that MIN-102 penetrates the brain and is active.

Following the successful completion of the Phase 1 trial, Minoryx started a randomized, placebo-controlled Phase 2/3 clinical trial (NCT03231878) to evaluate the efficacy of MIN-102 on the progression of AMN. It is a multi-center trial, with patients being treated in Spain, France, Hungary, Germany, Italy, the U.K., the Netherlands, and the U.S.

The efficacy of the treatment will be evaluated by measuring the ability of the patients to stand and walk as indicators of motor function. Efficacy and safety will be assessed every 24 weeks, in addition to two safety assessments after four and 12 weeks. After 96 weeks, participants will have the possibility to enter an open-label extension phase.

The randomization of 116 participants was completed in December 2018, and results are expected at the end of 2020.


Last updated: Dec. 27, 2018


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