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Trial suggests RRx-001 effective for oral mucositis following head and neck chemoradiotherapy

RRx-001 reduced the duration of severe oral mucositis symptoms in patients with squamous cell carcinoma undergoing chemoradiation

A first-in-class agent, RRx-001, has been found to reduce the duration of severe oral mucositis in patients undergoing chemoradiotherapy, in a Phase II placebo-controlled trial undertaken by US researchers.

Oral mucositis (OM) represents a common and highly symptomatic complication of cancer therapy which negatively impacts upon patient’s quality of life. OM affects virtually all patients undergoing radiation treatment for head and neck cancers and given the inadequacy of pain relief offered by opiates, preventative rather than palliative measures are needed. OM represents a significant unmet need for patients undergoing chemoradiation (CRT) for head and neck cancers, affecting individual’s ability to eat, drink and swallow, yet there are currently there are no approved treatments for the condition. While the underlying cause of OM remains to be determined, it is thought to arise largely the consequence of cumulative CRT-induced biological changes overwhelming physiologic self-protective mechanisms.

RRx-001 is a white, crystalline, nitrogen containing small molecule with demonstrated anti-cancer activity affecting a number of pathways including nitric oxide. Moreover, data suggests that the compound is able to selectively protect normal cells, while inducing apoptosis within tumour cells.

In the current study, researchers randomised patients with squamous cell carcinoma and who received either definitive or postoperative cisplatin-based CRT, to three different dosing schedules of RRx-001 (arms 1 to 3) and a placebo arm. The severity of OM was assessed using the WHO grading scale and the incidence of severe OM was bases on WHO scores > 3.

RRx-001 and duration of severe OM

Although 53 patients were enrolled, only 46 contributed to the efficacy data. Overall, there were no severe adverse reactions attributed to the drug. Patients in arm 1 had the lowest median duration of severe OM which was 8.5 days compared to 17 days and 10 days in arms 2 and 3 respectively. Among those in the placebo arm, the median duration of severe OM was 24 days.

The authors concluded that these data supported both the safety and efficacy of RRx-001 while also highlighting the need for further studies to confirm these findings.

Bonomi R et al. PREVLAR: Phase 2a randomized trial to assess the safety and efficacy of RRx-001 in the attenuation of oral mucositis in patients receiving head and neck chemoradiotherapy Int J Radiat Oncol Biol Phys 2023

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