Today, XTALPI (02228), an innovative platform company empowering drug and new material R&D with AI and robotics, announced that its incubated company, ReviR, has achieved a major milestone with its innovative drug pipeline RTX-117 successfully obtaining IND approvals in both China and the US. In light of this and other successful collaborative progress, XTALPI will receive a milestone payment totaling tens of millions of Hong Kong dollars from ReviR. ReviR will continue to complete the subsequent clinical development of RTX-117, while XTALPI is entitled to participate in the sales sharing and future licensing revenue sharing for this pipeline.

CMT, caused by genetic mutations, is characterized by its progressive and highly disabling nature. It is both a rare disease and the most common type of inherited peripheral neuropathy, affecting over 2.6 million patients globally. RTX-117 was co-developed by XTALPI and ReviR based on XTALPI's AI and robotic drug R&D platform. It is the first Class 1 innovative drug pipeline in China targeting CMT and is currently the only CMT drug in China that addresses the disease process at the molecular source by targeting the relevant pathological pathway. This breakthrough has the potential to end the long-standing lack of effective CMT treatments and tap into a global potential therapeutic market exceeding $10 billion.

Notably, beyond its applicability to the existing CMT patient population, RTX-117 possesses significant potential for indication expansion. The potential coverage is estimated to exceed 50 million people, nearly 20 times the number of current CMT patients. The mechanism of action of RTX-117 involves precisely targeting the key pathway responsible for CMT pathogenesis. It works by inhibiting the abnormally activated Integrated Stress Response (ISR), thereby restoring normal mRNA translation to exert its therapeutic effect.

According to ReviR, preclinical data indicate that RTX-117 demonstrates high potency and a strong ability to bind to its target. It shows a significant inhibitory effect on the ISR and effectively restores motor function in animal models. RTX-117 represents the first major milestone achievement from a series of in-depth collaborative R&D pipelines between XTALPI and ReviR. Both the preclinical data and the speed of clinical approval have exceeded the expectations of both parties. The upcoming clinical studies will evaluate the safety and efficacy of this breakthrough therapy, offering potential treatment hope for millions of patients worldwide affected by CMT and related diseases.