By Fineline Cube ReviR Therapeutics, an incubated company of XtalPi (HKG: 2228), announced that RTX-117 has received Investigational New Drug (IND) approval from China’s National Medical Products Administration (NMPA) to treat Charcot-Marie-Tooth disease (CMT), with Phase I trials scheduled to commence in Q1 2026. The drug, which has also obtained U.S. FDA IND approval and Orphan Drug Designation, is the first Class 1 innovative drug in China targeting CMT and the first rare disease program from the ReviR-XtalPi AI and robotics-assisted R&D collaboration to reach the clinic.
Regulatory & Program Milestone
| Item | Detail |
|---|---|
| Product | RTX-117 |
| Developer | ReviR Therapeutics (incubated by XtalPi) |
| Regulatory Status | NMPA IND approved (China); FDA IND approved (U.S.) |
| Indication | Charcot-Marie-Tooth disease (CMT) |
| Orphan Designation | U.S. FDA Orphan Drug Designation (qualifies for priority review and extended market exclusivity) |
| Clinical Timeline | Phase I initiation Q1 2026 |
| Significance | First-in-class CMT drug in China; first AI/robotics co-developed rare disease pipeline to enter clinic |
Disease Background: Charcot-Marie-Tooth Disease
Epidemiology:
- Global Prevalence: 17.7‑40 per 100,000 people
- Affected Population: Over 2.6 million patients worldwide
- China Status: Listed as #17 in China’s First National Catalogue of Rare Diseases (2018)
- Clinical Burden: Highly progressive and disabling hereditary neuropathy causing muscle weakness, sensory loss, and mobility impairment; no approved disease-modifying therapies
Unmet Need: RTX-117 targets the underlying molecular mechanism rather than symptomatic relief.
Mechanism of Action
Target Pathway: RTX-117 inhibits abnormally activated Integrated Stress Response (ISR) pathway, restoring normal mRNA translation in neuronal cells.
Preclinical Data:
- Molecular Level: Significantly suppresses ISR activation
- Functional Level: Restores motor function in animal models of CMT
- Differentiation: Precision targeting of key CMT pathophysiology vs. supportive care
Technology Platform: AI & Robotics-Assisted R&D
Collaboration Model:
- XtalPi’s Role: Provides AI drug discovery platform and robotics-assisted synthesis/screening
- ReviR’s Role: Translational biology, IND-enabling studies, clinical development
Significance:
- First Clinical Validation: RTX-117 is the first asset from the platform to reach human trials
- Platform Scalability: Demonstrates potential for rapid rare disease program advancement with AI
- Financial Terms: XtalPi eligible for milestone payments and share of future sales/licensing revenue
Market Opportunity & Financial Outlook
Revenue Potential:
- China Market: ¥500‑800 million peak sales (assuming 30‑40% penetration of diagnosed CMT patients)
- Global Market: $1.2‑1.8 billion (U.S./EU/Japan) with orphan drug pricing
- Pricing: Projected ¥300,000‑400,000 annual cost (orphan drug premium)
Platform Value: Success of RTX-117 validates XtalPi’s AI platform for rare diseases, enabling 5‑8 additional rare disease INDs by 2028.
Forward‑Looking Statements
This brief contains forward‑looking statements regarding RTX-117’s clinical development timeline, regulatory pathway, market penetration, and revenue projections. Actual results may differ materially due to clinical trial outcomes, competitive dynamics, regulatory acceptance of AI-discovered drugs, and market access policies for rare diseases.-Fineline Info & Tech