RiboX Therapeutics has received Regenerative Medicine Advanced Therapy (RMAT) designation from the US Food and Drug Administration (FDA) for RXRG001, an investigational treatment for radiation-induced xerostomia and hyposalivation.

The designation is based on preliminary clinical evidence from the ongoing Phase 1/2a SPRINX-1 study in adults whose salivary glands have been damaged by radiation treatment. The first-in-human, multi-centre trial is evaluating the safety, tolerability and preliminary efficacy of RXRG001 administered directly into the parotid gland ducts.

RXRG001 is a circular RNA therapy that encodes human aquaporin-1 (hAQP1), a protein involved in fluid transport. The therapy is delivered using a lipid nanoparticle and is designed to enable the local production of functional hAQP1 in the salivary glands, with the aim of restoring saliva production.

The RMAT designation provides RiboX with opportunities for earlier and more frequent interaction with the FDA, as well as regulatory guidance to support the development and review of the therapy. RXRG001 had previously received FDA Fast Track designation.

Radiation-induced xerostomia and hyposalivation can occur following radiation treatment for head and neck cancer and can significantly affect patients' quality of life. Current treatment options mainly focus on managing symptoms rather than restoring salivary gland function.

RiboX is continuing the SPRINX-1 study to assess RXRG001 and generate further clinical evidence on its potential to restore salivary function in affected patients.

 

Source: businesswire.com/

Report Abuse