On January 7, ReviR Therapeutics announced that it has recently received the "Drug Clinical Trial Approval Notice" from the National Medical Products Administration (NMPA) for its small molecule pipeline RTX-117, indicated for the treatment of Charcot-Marie-Tooth disease (CMT). The company plans to initiate Phase I clinical trials in the first quarter of 2026.

Reportedly, RTX-117 is China's first Class 1 innovative drug pipeline targeting CMT. It is also the first project to enter clinical development from a series of rare disease drug pipelines co-developed by ReviR and XtalPi through AI + robotic-assisted research. Prior to this, the pipeline had already obtained IND approval and Orphan Drug Designation from the U.S. FDA.

CMT is listed as the 17th disease in the "First List of Rare Diseases" announced by the National Health Commission in 2018. It is a highly progressive and disabling rare disease with a global prevalence of 17.7~40 per 100,000 people, affecting a total of over 2.6 million patients worldwide, making it one of the more common rare diseases.
According to the latest surveys, the median age of onset for CMT patients is 7.3 years old. Disease symptoms often include leg muscle weakness and atrophy, accompanied by skeletal deformities, pain, sensory loss, and motor developmental delay. Many patients become disabled due to the disease and can only alleviate symptoms through surgery and rehabilitation training, indicating a huge unmet medical need.

RTX-117 precisely targets the key physiological mechanism causing CMT. It exerts its therapeutic effect by inhibiting the abnormally activated Integrated Stress Response (ISR) pathway to restore normal mRNA translation (activating eIF2B to restore normal mRNA translation, rebuilding protein expression homeostasis, and improving neuromuscular function).

Preclinical data shows that RTX-117 not only significantly inhibited the ISR at the molecular level but also effectively restored motor function in model animals at the functional level. The development of this targeted therapeutic drug is expected to enable intervention in the early stages of the disease, reducing or even preventing the occurrence and progression of disability to a greater extent, thereby effectively filling the therapeutic gap. It has the potential to become a first-in-class / best-in-class treatment option.

Founded in 2021, ReviR is committed to combining AI technology with RNA biology to precisely regulate RNA expression through small molecules, intervening in and blocking pathological processes at the molecular source, thereby achieving a leap from "symptomatic treatment" to "root cause modification." Leveraging its proprietary VoyageR platform, the company has established multiple innovative drug pipelines in neurological diseases and autoimmune disorders, and has several preclinical pipeline collaborations with XtalPi.