ReviR Therapeutics Doses First Patient in RTX-117 Phase 1 Trial Following Dual U.S. and China IND Clearances

Image Courtesy: Public Domain

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."