Minoryx Therapeutics, a drug development company specialized in the discovery and development of new drugs for orphan diseases, today announces that its lead compound MIN-102 has been granted Orphan Drug Designation by the US Food and Drug Administration body (FDA). The drug candidate targets X-linked Adrenoleukodystrophy (X-ALD), a life threatening orphan CNS disease with high unmet medical need. MIN-102 now has Orphan Drug Designation from both the FDA and the European Medicines Agency (EMA).

To receive the FDA Orphan Drug status, a drug must be aimed at a rare disease or at a condition that affects less than 200,000 people in the United States.  Orphan Drug Designation by the FDA grants seven years of market exclusivity in the US and has other benefits such as tax credits, protocol assistance and research grants.

At the end of 2016, MIN-102 received Orphan Drug Designation from the European Medicines Agency (EMA), guaranteeing ten years of market exclusivity in the European markets, among other benefits.

“We are delighted that our lead candidate, MIN-102, now has Orphan Drug Designation from both the FDA and the EMA,” said Marc Martinell, CEO of Minoryx. “These acknowledgements prove that our drug candidate addresses an unmet need in orphan diseases. We are committed to progressing it rapidly through the next phases of drug development in order to offer a pharmacological treatment for X-ALD.”

A rare inherited neurodegenerative disorder and life threatening

MIN-102 targets X-linked adrenoleukodystrophy (X-ALD), a rare and chronically debilitating life threatening neurodegenerative disease. There are currently no pharmacological treatments for X-ALD. MIN-102 is the only product in development for potential use across two main clinical phenotypes of  X-ALD: adrenomyeloneuropathy (AMN), characterized by progressive motor dysfunction, and inflammatory cerebral ALD (cALD), characterized by severe neuroinflammation leading to early death. A phase 2/3 trial in adult AMN patients will be launched during the first half of 2017.

X-ALD is the most prevalent peroxisomal disease. It is caused by mutations in the ABCD1 gene. Its estimated incidence is 1:17,000 newborns. Although it primarily affects males, heterozygous women also develop the disease later in life. 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.

MIN-102 has the potential to treat both adrenomyeloneuropathy (AMN) and cerebral ALD (cALD). Phase 1 results are expected by Q1 2017. A phase 2/3 trial in adult AMN patients is planned by the first half of 2017.

Innovative treatments for life threatening rare diseases

Minoryx is a clinical stage biotech company leading the development of new therapies for X-ALD and other 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.

The biotechnology company, based in the TecnoCampus Mataró-Maresme Park, opened in February 2016 a research and development laboratory at the Barcelona Science Park.