Spanish drug development company Minoryx Therapeutics has begun its first-in-man Phase I clinical trial for its lead compound MIN-102 to treat X-linked Adrenoleukodystrophy (X-ALD).
MIN-102 is a differentiated, peroxisome proliferator-activated receptor (PPAR) gamma agonist indicated to be administered for central nervous system related diseases.
The trial is designed to evaluate the pharmacokinetics profile, safety, tolerability and brain penetration of MIN-102 after being administered in single ascending doses and multiple ascending doses in healthy volunteers.
The results of the Phase I trial are expected by the end of the year.
Minoryx CEO Marc Martinell said: "The dosing of the first subjects in the Phase I trial is an important milestone for Minoryx.
"It brings us a step closer to offering a potentially meaningful therapeutic option for X-ALD.
"We eagerly await the clinical readout from this study and plan to initiate clinical trials in X-ALD patients by early 2017."
X-ALD is a widely occurring peroxisomal disease triggered by genetic mutations of the ABCD1 gene.
The disease affects almost 1:17,000 newborns across the world. It primarily affects males, as well as heterozygous women.
X-ALD causes accumulation of very long chain fatty acids (VLCFA) leading to a neurodegenerative disorder where the most affected tissues are the spinal cord, brain and adrenal cortex.
Minoryx Therapeutics' MIN-102 targets X-ALD and is the only existing therapeutic drugs for X-ALD.
Image: White matter of brain displaying X-linked adrenoleukodystrophy. Photo: courtesy of Frank Gaillard via Wikipedia.