Oryzon Genomics - New clinical candidate expands CNS portfolio

Oryzon Genomics has nominated a new central nervous system (CNS) clinical development candidate, ORY-4001, for the treatment of rare CNS disorders including Charcot-Marie-Tooth (CMT) disease and amyotrophic lateral sclerosis (ALS). The decision follows positive data in which ORY 4001 demonstrated both an encouraging selectivity and safety profile as well as efficacy signals, triggering a strong anti-inflammatory response in preclinical in vivo models; the compound also showed positive responses in a validated CMT1A peripheral neuropathy in vivo model, which reliably recapitulates many of the symptoms of this condition in humans. In July 2022, Oryzon collaborated with the CMT Research Foundation, which helped fund the preclinical studies for ORY-4001 in CMT and, in our view, highlights the positive impact Oryzon’s partnerships can have on expediting development. The company now intends to start investigational new drug enabling studies as it progresses ORY-4001 towards clinical trials, the initiation of which would represent a future catalyst for investor attention.