RiboX Therapeutics has announced that its circular RNA therapeutic candidate, RXRG001, has been granted the Regenerative Medicine Advanced Therapy (RMAT) designation by the U.S. Food and Drug Administration (FDA) for the treatment of radiation-induced xerostomia (dry mouth) and hypo-salivation (reduced saliva production). This designation is based on preliminary clinical data from the ongoing Phase 1/2a SPRINX-1 trial conducted in the United States. Notably, RXRG001 had previously received Fast Track designation from the FDA, underscoring its potential to address unmet medical needs.

RXRG001 is engineered to encode human aquaporin-1, a protein that facilitates water transport. It is delivered via lipid nanoparticles and administered directly through the parotid duct, with the aim of restoring the function of salivary glands damaged by radiation therapy. The RMAT designation enables RiboX Therapeutics to engage in earlier and more frequent interactions with the FDA, facilitating a streamlined development and review process for RXRG001.

Radiation-induced xerostomia is a prevalent and debilitating side effect among patients undergoing radiotherapy for head and neck cancers. Current treatment options primarily focus on alleviating symptoms rather than addressing the underlying cause, highlighting the urgent need for innovative therapies that can restore salivary gland function.

RiboX Therapeutics is a clinical-stage biotechnology company dedicated to advancing fully engineered circular RNA therapeutics. With this RMAT designation, RXRG001 moves one step closer to potentially transforming the treatment landscape for patients suffering from radiation-induced salivary gland dysfunction.