Oryzon Genomics, S.A. presented positive preclinical efficacy data of ORY-4001, a selective histone deacetylase 6 (HDAC-6) inhibitor, in an oral communication at the 2023 Peripheral Nerve Society annual meeting (PNS-2023) held in Copenhagen from 17-20 June 2023. ORY-4001 treatment was able to reverse disease progression symptoms in a Charcot-Marie-Tooth (CMT) mice model. The presented results are the fruit of a collaboration agreement between Oryzon and the CMT Research Foundation (CMTRF), a U.S.-based patient-led, non-profit organization focused on delivering treatments and cures for CMT, which was entered in 2022 to explore the therapeutic potential of Oryzon's HDAC-6 inhibitors.

ORY-4001 was able to reverse in a dose-dependent manner the CMT1A phenotypes. Notably, ORY-4001 was ability to improve myelination and restore axon integrity in the sciatic nerve, and improved compound muscle action potential and nerve conduction in comparison with untreated animals. Remarkably, ORY-4001-treated animals displayed a significant body weight recovery, suggesting an overall physiological improvement.

The doses used in this study were safe and below the maximal tolerated dose, which allows room for dose increases in additional studies to further deepen the therapeutic benefit. ORY-4001 is an HDAC6 inhibitor with excellent pharmacology and very high selectivity against other HDAC classes, resulting in a remarkable safety profile avoiding hematotoxicity. CMT is a progressive, degenerative disease involving the peripheral nerves.

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.