XtalPi (2228.HK), a leading AI and robotics-driven R&D platform, and its incubated portfolio company ReviR Therapeutics (“ReviR”), announced that the innovative pipeline RTX-117 has received clinical trial approval from China’s National Medical Products Administration (NMPA). This latest approval targets Vanishing White Matter (VWM) disease, a devastating neurodegenerative condition.
RTX-117 has already achieved dual IND approvals in both China and the U.S. as a first-in-class treatment for Charcot-Marie-Tooth (CMT) disease. Securing approvals for two rare disease indications in rapid succession underscores XtalPi’s ability to discover high-value assets and highlights the immense potential of the “AI+RNA” drug discovery pathway.
Targeting the Root Cause of Neurodegeneration
VWM is a progressive, life-threatening genetic disorder primarily affecting children and adolescents, characterized by ataxia and motor decline. There are currently no approved therapies worldwide for VWM, representing a massive unmet medical need.
RTX-117 is a highly selective eIF2B small molecule activator co-developed by ReviR and XtalPi using XtalPi’s AI + Robotics platform. Unlike traditional symptomatic treatments, RTX-117 intervenes at the molecular source by targeting the Integrated Stress Response (ISR) pathway. By inhibiting an overactive ISR, the drug restores normal mRNA translation. Preclinical data for VWM demonstrated effective ISR regulation and significant improvement in motor function.
“One Drug, Multiple Indications”: Unlocking a Multi-Billion Dollar Market
The expansion from CMT (peripheral nervous system) to VWM (central nervous system) demonstrates the exceptional pharmacological profile of RTX-117. Achieving clinical entry for a CNS disorder validates the molecule’s superior Blood-Brain Barrier (BBB) penetration, systemic distribution, and targeting precision.
Furthermore, ISR pathway dysregulation is linked to major neurodegenerative diseases, including ALS, Parkinson’s (PD), and Alzheimer’s (AD). By validating RTX-117 in CMT and VWM, ReviR and XtalPi are positioning the molecule to potentially address a broader spectrum of CNS disorders, tapping into a multi-billion dollar therapeutic market.
Scaling Innovation through AI and Robotics
“The rapid clinical entry of RTX-117 for VWM is a testament to our team’s insight into underlying pathology and the power of our VoyageR AI platform,” said Dr. Yang Liu, Vice President of ReviR. “The professional support from XtalPi’s AI + Robotics platform provided a solid foundation for accelerating our clinical milestones.”
Under the agreement, XtalPi will receive tens of millions of HKD in milestone payments from ReviR following these clinical approvals. XtalPi also holds rights to global sales royalties and future out-licensing revenue shares, ensuring sustainable value creation for its shareholders and partners.
With over 7,000 known rare diseases affecting 400 million people—over 90% of whom lack treatment—XtalPi and ReviR are demonstrating that AI could solve the ROI challenges of orphan drug development. This solution offers both high social impact and significant economic returns.
About ReviR Therapeutics
ReviR Therapeutics is a clinical-stage global biotechnology company focused on combining AI with RNA biology to develop disease-modifying therapies. Its proprietary VoyageR platform identifies small molecules that target RNA to treat CNS and autoimmune disorders. Its lead candidate, RTX-117, is currently in clinical trials for CMT and VWM.
