ReviR Therapeutics, Powered by XtalPi’s AI+RNA Platform, Achieves China’s First Clinical Approval for a Novel Small Molecule Drug Targeting Charcot-Marie-Tooth Disease

Recently, XtalPi (2228.HK) Incubator company ReviR (hereinafter referred to as "ReviR") has received the Clinical Trial Approval Notice issued by the National Medical Products Administration (NMPA) for the small molecule pipeline RTX-117, intended for the treatment of Charcot-Marie-Tooth disease (CMT). The Phase I clinical trial is scheduled to commence in the first quarter of 2026.RTX-117 is China's first Class 1 innovative drug pipeline targeting CMT., which is also the first project in a series of rare disease drug pipelines developed through the deep collaboration between ReviR and XtalPi, utilizing AI and robotic assistance to enter clinical trials.XtalPi will receive corresponding milestone payments and is entitled to participate in the sales sharing and subsequent licensing revenue sharing of this pipeline.Previously, the pipeline had received the FDA's IND approval and Orphan Drug Designation (ODD) in the United States, qualifying for FDA priority review and a longer period of market exclusivity overseas.

CMT ranks 17th in the "First List of Rare Diseases" published by China's National Health Commission in 2018. It is a rare disease with strong progression and high disability rates, with a global prevalence of 17.7~40 per 100,000.The total number of patients exceeds 2.6 million people, belonging to one of the more common diseases within the category of rare diseases.According to statistics from IMARC Group,The CMT treatment market size in the seven major global markets (the United States, the four EU countries, the United Kingdom, and Japan) reached $1.013 billion in 2024 and is expected to surge to $11.394 billion by 2035 at a compound annual growth rate (CAGR) of 24.62%, demonstrating significant clinical demand and market growth potential.According to the latest survey by the School of Public Health, Sun Yat-sen University (Shenzhen) in 2025,The median age of onset for CMT patients is 7.3 years, with early disease onset, long diagnostic cycles, and a lack of effective treatment options.SymptomsIt is often characterized by leg muscle weakness and atrophy, accompanied by skeletal deformities, pain, sensory loss, and delayed motor development. Many patients become disabled due to the disease, and can only alleviate symptoms through surgery and rehabilitation training, representing a significant unmet treatment need.

Globally, the pipeline for Charcot-Marie-Tooth disease (CMT) is currently scarce, with no approved drugs on the market. RTX-117 is the first domestically developed Class 1 innovative drug approved to enter clinical trials for Charcot-Marie-Tooth disease (CMT) in China, representing a breakthrough in China's contribution to global CMT therapy research and development.This drug is the result of a deep collaboration between ReviR and XtalPi., based on XtalPi's AI + robotics drug discovery platform, combined with ReviR’s self-developed VoyageR AI and its long-term research and experience accumulation in the CMT field, has rapidly achieved breakthroughs from algorithm design to clinical translation. This once again validates the crucial role of AI + RNA modulation pathways in accelerating innovative therapy breakthroughs for important diseases such as rare diseases. The company has multiple ongoing pipeline project collaborations with XtalPi, which are expected to systematically address the global treatment challenges of rare diseases, bringing new hope to more patients who currently have no available treatments.

RTX-117 precisely targets the key physiological mechanism causing CMT, exerting its therapeutic effect by inhibiting the abnormally activated Integrated Stress Response (ISR) pathway and restoring normal mRNA translation.Preclinical data show that RTX-117 not only significantly inhibits ISR at the molecular level but also effectively restores motor function in model animals.。The development of this targeted therapeutic drug,Intervening in the early stages of CMT patients has the potential to significantly reduce or even prevent the occurrence and progression of disability., effectively filling the therapeutic gap,Has the potential to become the first-in-class/best-in-class treatment option, accelerating market entry and unlocking a massive pharmaceutical market.

Public data shows that among the more than 7,000 rare diseases discovered globally, which involve 400 million patients, only 5% of these diseases have clear treatment options. Moreover, very few of these options are truly disease-modifying solutions.Looking globally, the rare disease drug market represented by Europe and the United States exhibits a vast commercial blue ocean, with its well-established diagnostic systems and diverse payment mechanisms driving an orphan drug market worth trillions.By contrast, rare disease patients in China face severe reality challenges, with high costs of imported drugs and low accessibility becoming urgent social pain points to be addressed.Empowering Rare Disease Targeted Drug R&D from the Source Biological Mechanism with AI + Robotics Technology: A Promising Approach to Design Innovative Therapies with Both Clinical Value and Market Potential at a Faster Speed and Lower Cost, Bringing New Opportunities and Technological Revolution to the Vast Patient Populations in China and Abroad and the Expansive Blue Ocean of Rare Disease TherapiesRTX-117, as an advanced practice in the field of AI-driven rare disease drug development, advances clinical trials in China, which is conducive to reshaping the equity of rare disease treatment and demonstrating China's technological strength in competing for the global high-net-worth market.

Dr. Shuhaotem Wen, Chairman of the Board of XtalPi, said： “The economic bottleneck of traditional drug research and development has long constrained breakthroughs in the treatment of rare diseases. AI-driven precision drug discovery is changing this paradigm——The clinical progress of RTX-117 once again validates XtalPi's rapid transformation capability 'from algorithm to clinic,' as well as the significant potential of the AI+RNA research and development pathway in the field of rare disease drug discovery.。With the accumulation of data and experience, the prediction accuracy of AI models and the output rate of pipelines will continue to improve, which is expected to fill more clinical gaps and create sustainable value for patients, partners, and investors.。”

Dr. Yang Li, co-founder and chairman of ReviR, said:"ReviR focuses on patient populations with unmet clinical needs and no available treatments, dedicated to leveraging AI and RNA functionality to pioneer innovative therapeutic paradigms."The clinical approval of RTX-117 demonstrates the innovative power generated by ReviR and XtalPi in the "AI + RNA" field.This is not only a breakthrough in a single pipeline, but also an elevation in the R&D paradigm. We are accelerating to bring more effective and accessible treatment options to patients with CMT and a broader population.。”

ReviR Chief Scientific Officer (CSO) Dr. Paul August stated： “RTX-117 is a small molecule drug independently developed by us targeting the disease mechanism of CMT, and it represents a significant practical achievement of applying AI to drug discovery. It now meets all the necessary conditions to enter clinical trials. This clinical approval verifies the scientific validity of ReviR’s translational research strategy, and we will proceed as planned to further develop our pipeline and advance clinical breakthroughs in therapies for rare neurological diseases.

● CloseInReviR Therapeutics (ReviR Therapeutics)●

ReviR Therapeutics is a global biotechnology company at the clinical stage, dedicated to combining AI technology with RNA biology to develop highly specific, effective, and safe disease-modifying therapies. With the deep technical accumulation of its self-developed platform VoyageR, ReviR has established multiple innovative drug pipelines in the fields of neurological diseases and autoimmune disorders. Its leading small-molecule drug candidate, RTX-117, is currently advancing in clinical trials for Charcot-Marie-Tooth disease (CMT) and continues to expand into new indications.

● CloseInAbout Charcot-Marie-Tooth Disease (CMT) ●

Charcot-Marie-Tooth disease (CMT) is a group of hereditary peripheral neuropathies that cause nerve degeneration, listed in the "First Batch of Rare Diseases Catalogue" by China's National Health Commission in 2018. Due to genetic mutations, the peripheral nerves responsible for motor and/or sensory functions are affected, leading to progressive muscle weakness and atrophy in the distal limbs, as well as sensory loss. Symptoms include skeletal deformities such as pes cavus and scoliosis, along with gait abnormalities and difficulties in grasping. Currently, there are no available treatments or cures for CMT.

● CloseInAbout RTX-117 ●

RTX-117 is a small-molecule drug independently developed by ReviR Therapeutics, marking the world's first small-molecule therapy for Charcot-Marie-Tooth disease (CMT) developed using AI, and also China's first approved Class 1 innovative CMT drug. Its core mechanism involves inhibiting the Integrated Stress Response (ISR) by activating eIF2B to restore normal mRNA translation, rebuild protein expression homeostasis, and improve neuromuscular function. Clinical trials for CMT are currently underway, with new indications being explored simultaneously.