The company’s leading program is a differentiated PPAR gamma agonist (MIN-102) being developed for X-linked Adrenoleukodystrophy (X-ALD) that has successfully completed a phase 1 clinical trial. It has the potential to treat both cerebral ALD (cALD) and adrenomyeloneuropathy (AMN), the two main forms of X-ALD.
Minoryx is also working on a new class of compounds, non-competitive pharmacological chaperones, identified through its innovative proprietary platform, SEE-Tx.
Pharmacological chaperones (PCT) are an innovative class of small molecule drugs that stabilize unstable proteins. This approach has a tremendous potential in the treatment of genetic diseases, where usually the cause of the disease is a protein malfunction due to a mutation on the wild-type sequence.