Recently, XtalPi (2228.HK) incubator company ReviR Therapeutics (“ReviR”) received the Clinical Trial Notification (CTN) from China’s National Medical Products Administration (NMPA) for its small molecule pipeline, RTX-117, targeting Charcot-Marie-Tooth (CMT) disease. Phase I clinical trials are scheduled to commence in the first quarter of 2026.
RTX-117 is the first Class 1 innovative drug pipeline in China targeting CMT. It also marks the first project to enter the clinical stage among a series of rare disease pipelines developed through deep collaboration between ReviR and XtalPi, leveraging AI and robotics-assisted R&D. XtalPi will receive corresponding milestone payments and is entitled to share in future sales royalties and sublicensing revenues. Previously, the pipeline received Investigational New Drug (IND) approval and Orphan Drug Designation (ODD) from the U.S. FDA, qualifying it for prioritized review and extended market exclusivity in overseas markets.
Addressing Significant Unmet Clinical Needs in CMT
CMT is a progressive and highly disabling rare disease. Ranked 17th in China’s First National List of Rare Diseases, it has a global prevalence of 17.7–40 per 100,000, affecting over 2.6 million people. According to IMARC Group, the CMT treatment market in the seven major markets (US, EU4, UK, and Japan) reached $1.013 billion in 2024 and is projected to surge to $11.394 billion by 2035, with a CAGR of 24.62%.
Despite the significant market potential, there are currently no approved therapies worldwide. RTX-117, as the first Class 1 innovative drug for CMT to enter clinical trials in China, represents a breakthrough in China’s contribution to global CMT therapeutic R&D.
A Paradigm Shift in AI-Driven Drug Discovery
Developed through the synergy of XtalPi’s AI + Robotics platform and ReviR’s proprietary VoyageR AI, RTX-117 achieved a rapid transition from algorithm design to clinical translation. The drug precisely targets the Integrated Stress Response (ISR) pathway, inhibiting abnormal activation to restore normal mRNA translation. Preclinical data shows that RTX-117 significantly inhibits ISR at the molecular level and effectively restores motor function in animal models, positioning it as a potential First-in-Class/Best-in-Class therapy.
Platform-Based Success and Long-Term Value
As the first “AI4Science” and “AI + Robotics” stock on the HKEX, XtalPi utilizes its “Quantum Physics + AI + Large-scale Robotics” closed-loop technology to provide standardized, automated R&D infrastructure. Unlike traditional biotech companies, XtalPi’s platform-based model allows it to:
- Provide “0-to-1” innovation breakthroughs for partners and secure milestone payments.
- Share in the long-term value of pipelines through equity and royalty interest.
Dr. Shuhao Wen, Chairman of XtalPi, commented: “The clinical progress of RTX-117 validates XtalPi’s ability for rapid ‘algorithm-to-clinical’ translation. AI-driven precision drug discovery is breaking the economic bottlenecks of traditional R&D, filling clinical voids and creating sustainable value for patients and investors alike.”
Dr. Yang Li, Co-founder and Chairman of ReviR, added: “The clinical approval of RTX-117 underscores the innovative power generated by the collaboration between ReviR and XtalPi in the ‘AI + RNA’ space. We are accelerating the delivery of more effective and accessible treatment options for CMT and beyond.”
Dr. Paul August, Chief Scientific Officer (CSO) of ReviR, stated: “RTX-117 is a small molecule drug we independently developed based on the underlying disease mechanism of CMT. It represents a significant milestone in applying AI to drug discovery and has met all necessary criteria to advance into clinical trials. This clinical approval validates the scientific integrity of ReviR’s translational research approach. We will continue to advance our pipeline as planned, driving clinical breakthroughs in therapies for rare neurological diseases.”
About ReviR Therapeutics
ReviR Therapeutics is a clinical-stage global biotechnology company dedicated to combining AI technology with RNA biology to develop highly specific, effective, and safe disease-modifying therapies. Leveraging the profound technical foundations of its proprietary VoyageR platform, ReviR has established a diverse pipeline of innovative drug candidates across the fields of neurological disorders and autoimmune diseases. Its lead small molecule candidate, RTX-117, is currently advancing through clinical trials for Charcot-Marie-Tooth (CMT) disease, with ongoing efforts to expand its indications to other conditions.
About Charcot-Marie-Tooth (CMT)
CMT is a group of inherited peripheral neuropathies that cause progressive muscle weakness, atrophy, and sensory loss. Currently, there is no known cure or effective treatment for CMT.
About RTX-117
RTX-117 is a self-developed small molecule drug by ReviR. It is the world’s first AI-developed small molecule therapy for CMT and the first Class 1 innovative drug for CMT to be approved for clinical trials in China. It works by inhibiting the ISR and activating eIF2B to restore protein homeostasis.
