ReviR Therapeutics Announces First Participant Dosed in Phase 1 Study and Secures CDE Clearance for Two Orphan Indications in China, Supporting Future U.S. Expansion Strategy

Milestone marks the first clinical program for Charcot-Marie-Tooth disease and Vanishing White Matter disease.

SAN FRANCISCO and SHANGHAI, March 2, 2026 /PRNewswire/ -- ReviR Therapeutics, a clinical-stage biotechnology company pioneering AI-driven RNA-modulating small molecules to treat rare and genetic diseases, today announced a pivotal clinical and dual regulatory milestone: the first participant has been dosed in the First-in-Human (FIH) Phase 1 clinical trial of its lead candidate, RTX-117. This clinical progress follows the recent clearance by the Center for Drug Evaluation (CDE) of China's National Medical Products Administration (NMPA) of Investigational New Drug (IND) applications for two rare neurological indications: Charcot-Marie-Tooth disease (CMT) and Vanishing White Matter disease (VWM). The pipeline has also received Investigational New Drug (IND) clearance from the U.S. Food and Drug Administration (FDA), enabling the expansion of its clinical program into the United States.

The Phase 1 study is a randomized, double-blind, placebo-controlled, dose-escalation trial designed to evaluate the safety, tolerability, pharmacokinetics (PK), and pharmacodynamics (PD) of RTX-117 in healthy participants, supporting the transition into subsequent clinical trials for patients with CMT and VWM.

Advancing Treatment for Rare Neurological Disorders

The severity of these diseases targeted in ReviR's clinical trials underscores the urgency of ReviR's mission, while the clearance further validates the unmet need for patients in China and worldwide:

• Charcot-Marie-Tooth disease (CMT): A spectrum of progressive and debilitating inherited disorders that cause nerve damage, resulting in muscle atrophy and loss of sensation in the extremities, with some subtypes leading to high mortality rates.
• Vanishing White Matter disease (VWM): A progressive and catastrophic leukodystrophy that primarily affects children, leading to irreversible motor decline and cognitive deterioration, often with fatal outcomes.

"The milestone is a testament to the rigor of our preclinical data and the efficiency of our global team," said Paul August, PhD, Chief Scientific Officer of ReviR. "We are committed to working closely with clinical investigators and patient organizations in China to bring these innovative therapies forward as safely and rapidly as possible."

"The initiation of dosing in our first-in-human study, coupled with the dual IND clearance in China, marks a transformative leap for ReviR Therapeutics," added Peng Yue, PhD, CEO of ReviR. "By leveraging our VoyageR AI platform and our integrated cross-border operations in the U.S. and China, we are accelerating the development of our small molecule pipelines designed to address critical gaps where no approved disease-modifying treatments exist."

VoyageR AI is Fundamental to Disease Strategy

RTX-117 has previously received IND approval and Orphan Drug Designation from FDA, and it is also currently the only pipeline in China granted IND clearance for both CMT and VWM. It was discovered and developed using ReviR's proprietary VoyageR AI platform in collaboration with XtalPi's AI+robotics drug discovery engine. By leveraging deep insights into RNA biology and protein translation homeostasis, RTX-117 acts on the Integrated Stress Response (ISR) pathway to restore normal protein expression at the molecular source. Beyond the lead program, the VoyageR platform serves as a powerful engine for precision medicine, identifying chemical starting points based on their specific impact on RNA splicing. This enables the modulation of protein expression at the genetic source, empowering ReviR to target complex neurological conditions, immune & inflammatory pathways, and beyond - that have long been considered "undruggable."

About ReviR Therapeutics

ReviR Therapeutics is a global biotechnology company committed to discovering and developing innovative therapies for patients with serious genetic diseases. ReviR was founded in 2021 by leaders in computational biology, RNA biology, genomics, and drug discovery, with the aim to create disease-modifying therapies that are highly specific, efficacious, and safe. ReviR's lead candidate, RTX-117, is an investigational small molecule therapy for Charcot-Marie-Tooth disease (CMT)/Vanishing White Matter disease (VWM). In addition, ReviR's pipeline includes other orally administered small molecules designed to modulate RNA function by splicing. For more information about ReviR Therapeutics, visit www.revirtx.com.

About RTX-117

RTX-117 is a potential first-in-class, orally administered small molecule therapy developed by ReviR Therapeutics, with discovery and molecular design powered by XtalPi's AI and robotics drug discovery platform. The therapy targets the Integrated Stress Response (ISR) pathway by activating eIF2B, a key protein translation initiation factor. By restoring protein synthesis homeostasis and improving neuromuscular function, RTX-117 aims to address the underlying molecular pathology of related genetic disorders. The candidate is currently undergoing a Phase 1 clinical trial, with Investigational New Drug (IND) clearance already secured for both Charcot-Marie-Tooth disease (CMT) and Vanishing White Matter disease (VWM).

Forward-Looking Statements

This press release contains forward-looking statements regarding ReviR Therapeutics' clinical trials and regulatory pathways. Actual results may differ materially based on clinical outcomes and regulatory decisions.

SOURCE ReviR Therapeutics