PRINCETON, N.J., Oct. 25, 2024 — RiboX Therapeutics Ltd. (RiboX), a pioneering biopharmaceutical company specializing in the discovery and development of fully engineered circular RNA therapeutics, announced that the US Food and Drug Administration (FDA) has cleared its IND application for the Phase I/IIa Study of RXRG001 (SPRINX-1 Study) on October 25th, 2024. RXRG001 is the first circular RNA therapy to receive FDA IND clearance. This achievement marks a significant milestone as circular RNA drugs enter the clinical development phase.
The first-in-human SPRINX-1 study is designed to assess the safety and efficacy of RXRG001 in patients experiencing radiation-induced xerostomia (RIX) (dry mouth) and hyposalivation (low saliva secretion).
Xerostomia and hyposalivation are common side effects of radiation therapy used to treat head and neck cancer (HNC) patients. The incidence of HNC is on the rise, making it the sixth most common malignant tumor globally. Radiation therapy, a primary treatment for HNC, frequently causes damage to salivary glands, leading to a substantial decrease in saliva secretion. This often results in dry mouth, difficulties with chewing and swallowing, impaired oral health, and a diminished quality of life. The last xerostomia treatment approved by the FDA was two decades ago. Patients have reported that current treatments are ineffective, temporary, and costly. A significant unmet medical need exists for a new treatment for xerostomia and hyposalivation.
RXRG001 is a proprietary product developed using innovative circular RNA technology. In recent years, circular RNA has emerged as the next generation mRNA therapy due to its high protein expression efficiency, low immunogenicity, and manufacture stability. RXRG001 consists of circular RNA encoding human aquaporin 1 (hAQP1, a water channel protein found in cell membranes), encapsulated in lipid nanoparticles (LNPs). RXRG001 enhances saliva production by restoring water permeability through overexpression of hAQP1, thus alleviating dry mouth symptoms. Non-clinical studies have demonstrated a favorable risk and benefit profile of RXRG001 in animal models. For example, a single administration of RXRG001 resulted in a significant increase in salivary flow, which persisted for approximately four weeks.
“RIX is a debilitating, lifelong health issue for HNC patients. While radiation therapy improves patient survival, its damage to salivary glands significantly impacts the quality of life of many cancer survivors,” stated Dr. Yizhen Xu, Chief Medical Officer of RiboX. “RXRG001 may offer a potentially effective and sustained therapeutic option for RIX patients. We are eager to conduct clinical trials to further evaluate its efficacy and safety in patients.”
“The FDA’s clearance of the IND application for RXRG001 acknowledges our innovative capabilities, robust technology, and competitiveness in the field of circular RNA therapeutics.” Dr. Weiyi Zhang, Chief Executive Officer of RiboX, stated “RiboX will advance the clinical development of RXRG001, continue leveraging the unique advantages of circular RNA technology in therapeutic applications, and introduce more pioneering treatments to patients worldwide.”
About RiboX Therapeutics
RiboX is a globally operating biotech company, dedicated to discovering and developing fully engineered circular RNA therapeutics, cofounded by Drs. Ling-Ling Chen, Dan Peer and Weiyi Zhang in 2021. The company has operations in Princeton, NJ, USA, Rehovot, Israel and Shanghai, China. Circular RNA therapy is a novel modality, considered the next generation RNA therapy. Compared to mRNA, circular RNA has a covalently closed single-stranded RNA structure that provides superior stability, lowers immunogenicity, and allows for tunable protein expression. This overcomes the current limitations of mRNA therapeutics and unlocks the full potential of RNA medicines.
RiboX has developed proprietary plug-and-play circular RNA drug design and engineering, combined with novel LNPs platforms and robust manufacturing capabilities. These innovative technologies provide support for the clinical development of circular RNA therapeutics. RiboX’s pipeline spans various therapeutic areas, including radiation-induced xerostomia, monogenic diseases, autoimmune diseases, and vaccines. RiboX is committed to pioneering innovative research in next-generation RNA therapies, with a vision to improve the quality of life for patients worldwide.
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