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Marc Martinell, co-founder and CEO of Minoryx Therapeutics (Photo: ©Minoryx).

Minoryx Therapeutics Initiates Phase 1 clinical trial of MIN-102

Minoryx Therapeutics, a drug development company specialized in the discovery and development of new drugs for orphan diseases, today announces the initiation of its first-in-man Phase 1clinical trial for its lead compound MIN-102. The company –located at TecnoCampus Mataró-Maresme, with laboratories at Barcelona Science Park (PCB)– will harness its unique mechanism of action for potential use in X-linked Adrenoleukodystrophy (X-ALD), a genetic disease characterized by progressive neurologic deterioration with no available pharmacological treatment. 


MIN-102 is a differentiated PPAR gamma agonist with a superior profile for central nervous system related diseases. It has shown robust preclinical proof of concept. The aim of the Phase 1 study is to assess pharmacokinetics, safety, tolerability and brain penetration of MIN-102 after administration of single ascending doses and multiple ascending doses in healthy volunteers. Results are expected by the end of the year.

X-linked Adrenoleukodystrophy (X-ALD) is rare and chronically debilitating life threatening neurodegenerative disease, caused by mutations on the ABCD1 gene. It is characterized by the accumulation of very long chain fatty acids (VLCFA) leading to a neurodegenerative disorder where the most affected tissues are the spinal cord, the brain and the adrenal cortex. Its estimated incidence is 1:17,000 newborns worldwide, primarily males Currently, there are no therapeutic drugs for X-ALD. MIN-102 is the only product in development for potential use across all the main phenotypes.

“The dosing of the first subjects in the Phase 1 trial is an important milestone for Minoryx. It brings us a step closer to offering a potentially meaningful therapeutic option for X-ALD,” said Marc Martinell, CEO of Minoryx. “We eagerly await the clinical readout from this study and plan to initiate clinical trials in X-ALD patients by early 2017.”

Minoryx is a drug development company specializing in the discovery and development of new drugs for orphan diseases. The company –located at TecnoCampus Mataró-Maresme, with laboratories at Barcelona Science Park (PCB)– targets Inborn Errors of Metabolism, a group of rare diseases of genetic origin with a high unmet medical need. The Minoryx team is made up of a group of drug discovery and development experts with several decades of experience in biotech and pharma. The company is backed by a syndicate of experienced investors and has support from a network of other organizations. Minoryx was founded in 2011 and has raised a total of €24.4M ($27.6M).