Minoryx Therapeutics, a drug development company specialized in the discovery and development of new drugs for orphan diseases, announced the initiation of its first-in-man Phase 1 clinical trial for its lead compound MIN-102.
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.
Minoryx Therapeutics’ MIN-102 targets X-ALD, a rare and chronically debilitating life-threatening neurodegenerative disease. 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.”
X-ALD is the most prevalent peroxisomal disease. It is caused by mutations on the ABCD1 gene. Its estimated incidence is 1:17,000 newborns worldwide. Although it primarily affects males, heterozygous women also develop the disease later in life. X-ALD 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. The CNS related effects lead to two main phenotypes: adrenomyeloneuropathy (AMN), characterized by progressive motor dysfunction, and cerebral ALD (cALD), characterized by severe neuroinflammation leading to early death. There is currently no pharmacological treatment available on the market. The only available alternative for cALD patients is hematopoietic stem cell transplantation (HSCT). This approach does not prevent the development of the AMN phenotype, for which there are no therapies available.
About Minoryx Therapeutics
Minoryx is a drug development company specializing in the discovery and development of new drugs for orphan diseases. The company targets Inborn Errors of Metabolism, a group of rare diseases of genetic origin with a high unmet medical need. The company’s leading program, now in Phase 1 clinical trials, is a differentiated PPAR gamma agonist (MIN-102) that has multiple CNS indications. Minoryx harnesses its unique mechanism of action for potential use in X-ALD, a genetic disease characterized by progressive neurologic deterioration with no available pharmacological treatment. Minoryx is also working on a new class of compounds, non-competitive pharmacological chaperones, identified through its innovative proprietary platform – SEE-Tx. 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).