Spanish biopharma company Oryzon Genomics, S.A. is starting a preclinical collaboration with the CMT Research Foundation (CMTRF), a U.S.-based patient-led, non-profit organization focused on delivering treatments and cures for Charcot-Marie-Tooth (CMT) disease, to explore Oryzon’s histone deacetylase 6 (HDAC6) inhibitors.
HDAC6 inhibitors have been previously described as potentially effective treatments for CMT. Oryzon has recently completed an HDAC6 discovery program leading to the selection of two potential preclinical candidates with promising efficacy, selectivity and safety. Under this collaboration, CMTRF will be financing a series of in vivo tests with Oryzon’s HDAC6 candidates in a murine model of CMT1A, which reliably recapitulates many of the symptoms of this condition in humans.
CMT is a progressive, degenerative disease involving the peripheral nerves. It affects one in 2,500 people (about the same prevalence as multiple sclerosis), including 150,000 Americans and more than 3 million people globally.
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 of all, accounting for approximately half of all people with CMT.
Hoping for clinical development
Jordi Xaus, Oryzon’s CSO, said: “CMT is the most promising indication for our HDAC6 precandidates. CMTRF is the perfect ally to explore our compounds in this indication, as it has a strong connection with the CMT patient community, a very experienced scientific advisory board and a strong commitment to finding treatments for CMT. We are delighted to start this collaboration. If the results are positive, our HDAC6 program would be closer to clinical development and to offering hope to CMT patients. This program would become our second epigenetic program in nervous diseases.”
“We are excited to collaborate with Oryzon on this important project,” said Cleary Simpson, CEO of the CMT Research Foundation.
“HDAC6 inhibitors are promising as potential therapeutics for several forms of CMT, including the most common form—CMT1A. With funding from the CMT Research Fountation, Oryzon will be able to rapidly test their newly discovered HDAC6 inhibitors in CMT1A model mice, paving the pathway for further development. People with CMT currently have no effective treatments or cures, and we believe this project has the potential to change that for millions of families.”
The CMT Research Foundation (CMTRF) is focused solely on delivering treatments and cures for CMT. Founded by two patients who are driven to expedite drug delivery to people who live with CMT globally, the organization funds research for drug development.