Oryzon Genomics, S.A. announced that it will present positive preclinical efficacy data of ORY-4001 in a CMT model at the 2023 Peripheral Nerve Society (PNS) Annual Meeting, which will be held June 17-20 at the Bella Center in Copenhagen, Denmark. The scientific committee of the 2023 PNS Annual Meeting has selected ORY-4001, a Novel Potent and Selective Oxadiazole-Based HDAC6 Inhibitor Shows Pre-Clinical Therapeutic Efficacy in CMT1A" for an oral presentation, which will take place on June 20, at 08:40 am CEST. In 2022, Oryzon and the CMT Research Foundation (CMTRF), a U.S.-based patient-led, non-profit organization focused on delivering treatments and cures for CMT, entered into an agreement to explore the therapeutic potential of Oryzon's histone deacetylase 6 (HDAC-6) inhibitors.

Following positive preclinical results obtained under this collaboration, Oryzon selected a candidate for clinical development, ORY-4001. This compound is a HDAC6 inhibitor with excellent pharmacology and very high selectivity against other HDAC classes, resulting in a remarkable safety profile avoiding hematotoxicity. The compound exhibits strong anti-inflammatory properties and positive in-vivo data in inflammatory models.

In the study to be presented at the 2023 PNS annual Meeting, ORY-4001 showed multiple positive responses in a validated CMT1A peripheral neuropathy in vivo model which effectively mimics many of the symptoms of this condition in humans. It affects 150,000 Americans and more than 3 million people around the world. It is one of the most prevalent conditions among rare diseases and currently lacks effective treatments or cures.

CMT is caused by a variety of genetic mutations. CMT1A is the most prevalent form, accounting for approximately half of all people with CMT.