By Fineline Cube 
ReviR Therapeutics announced today that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to its small‑molecule candidate RTX‑117 for the treatment of Charcot‑Marie‑Tooth (CMT) disease. The company plans to launch a Phase 1 safety study in healthy volunteers in Q1 2026, followed by a Phase 1b/2a trial in CMT patients as scheduled.
Key Milestones
- FDA Orphan Drug Designation – RTX‑117 is now eligible for regulatory incentives, including 7 years of market exclusivity, reduced fees, and potential tax credits.
- Rapid Development Timeline – Leveraging an AI + robotic discovery platform from partner XtalPi, the molecule was identified and advanced to preclinical testing within eight months of project kickoff.
- First AI‑Developed Small‑Molecule Therapy for CMT – RTX‑117 represents a historic first for both the disease and Chinese‑domestic innovation, marking the launch of a Category 1 innovative drug in the region.
Scientific Rationale
- Mechanism of Action – RTX‑117 targets the Integrated Stress Response (ISR) by activating eIF2B, thereby restoring normal mRNA translation, re‑establishing protein homeostasis, and improving neuromuscular function.
- Preclinical Efficacy – In rodent models of CMT, RTX‑117 produced robust therapeutic benefits, slowing disease progression and demonstrating substantial safety margins.
- Unmet Need – CMT affects > 2.6 million people worldwide and currently lacks disease‑modifying therapies; RTX‑117 aims to fill this critical gap.
Development Roadmap
| Phase | Focus | Timeline |
|---|---|---|
| Phase 1 (Safety & PK) | Healthy volunteers | Q1 2026 |
| Phase 1b/2a (Proof‑of‑Concept) | CMT patients | Q3 2026 onward |
| Phase 2/3 (Efficacy & Confirmation) | Expanded patient cohorts | 2028‑2030 |
ReviR Therapeutics is coordinating with the FDA to ensure a streamlined regulatory pathway and is engaging with patient advocacy groups to support trial recruitment.
About ReviR Therapeutics
ReviR Therapeutics is a China‑based biopharmaceutical company focused on developing small‑molecule therapeutics for rare and neuro‑degenerative diseases. The company combines deep scientific expertise with advanced AI‑driven discovery platforms to accelerate drug development.
About RTX‑117
RTX‑117 is a first‑in‑class, oral, small‑molecule therapy designed to modulate the ISR pathway in Charcot‑Marie‑Tooth disease, offering a novel mechanism for disease modification.-Fineline Info & Tech