RiboX Therapeutics (“RiboX”), a clinical-stage biotechnology company pioneering the discovery and development of fully engineered circular RNA (circRNA) therapeutics, today announced that the U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to RXRG001 for the treatment of radiation-induced xerostomia (RIX) and hyposalivation. The designation was granted based on preliminary clinical evidence from the ongoing Phase 1/2a SPRINX-1 Study.
RXRG001 is being evaluated in a first-in-human, multi-center Phase 1/2a clinical trial in adults with radiation-induced xerostomia and hyposalivation ongoing in the US. The dose-escalation study is evaluating the safety, tolerability and preliminary efficacy of intraductal administration of RXRG001 to the parotid glands.
RMAT designation provides opportunities for early and enhanced interactions with FDA to support efficient drug development and may provide access to expedited development and review pathways. RXRG001 previously received FDA Fast Track designation.
“The FDA’s RMAT designation for RXRG001 represents an important milestone for RiboX and recognizes the promising preliminary clinical evidence generated with our circular RNA platform,” said Weiyi Zhang, Ph.D., Co-Founder and Chief Executive Officer of RiboX. “Radiation-induced xerostomia and hyposalivation can have a significant impact on patients following treatment for head and neck cancer, yet current treatment options are primarily focused on symptom management. We look forward to working closely with FDA as we advance RXRG001 and evaluate its potential to restore salivary gland function for these patients.”
The RMAT designation was supported by preliminary clinical data from SPRINX-1 (NCT06714253), an ongoing first-in-human, multicenter Phase 1/2a study evaluating the safety, tolerability and preliminary efficacy of RXRG001 administered directly into the parotid gland ducts in adults with RIX and hyposalivation.
RXRG001 is a proprietary circular RNA therapeutic encoding human aquaporin-1 (hAQP1), a water-channel protein involved in fluid transport, encapsulated in a lipid nanoparticle (LNP). Administered locally to the salivary glands, RXRG001 is designed to enable expression of functional hAQP1 with the goal of restoring saliva production in patients whose salivary glands have been damaged by radiation therapy.
RMAT designation is an FDA program designed to expedite the development and review of regenerative medicine therapies intended to treat, modify, reverse or cure serious or life-threatening diseases or conditions. To qualify, a therapy must have preliminary clinical evidence indicating the potential to address unmet medical needs. RMAT designation provides opportunities for early and enhanced interactions with FDA and intensive guidance on efficient drug development.
About Radiation-Induced Xerostomia (RIX) and Hyposalivation
RIX and hyposalivation are common and potentially debilitating complications of radiation therapy for head and neck cancers. Radiation can cause lasting damage to the salivary glands, resulting in reduced saliva production and persistent dry mouth. These conditions can significantly affect patients’ ability to eat, chew, swallow and speak, contribute to oral and dental complications, and negatively impact quality of life. Current treatment approaches are primarily focused on symptom management, highlighting the need for therapies that can restore salivary gland function.
About RXRG001
RXRG001 is an investigational circular RNA therapeutic designed to restore salivary gland function in patients with radiation-induced xerostomia and hyposalivation. RXRG001 consists of circular RNA encoding human aquaporin-1 encapsulated in lipid nanoparticles and is administered directly into the salivary glands. By enabling local expression of hAQP1, RXRG001 is designed to restore fluid transport and saliva secretion in radiation-damaged salivary glands. RXRG001 is currently being evaluated in SPRINX-1 (NCT06714253), a Phase 1/2a clinical study evaluating its safety, tolerability and preliminary efficacy in adults with RIX and hyposalivation.
About RiboX Therapeutics
RiboX is a clinical-stage biotechnology company pioneering the discovery and development of fully engineered circular RNA therapeutics to address unmet medical needs across a broad range of diseases. The company’s proprietary circular RNA technology and delivery platforms are designed to enable durable protein expression and unlock the therapeutic potential of RNA medicines. For more information, visit www.riboxtx.com.
View source version on businesswire.com: https://www.businesswire.com/news/home/20260908357531/en/
Media gallery
