Oral N2O Therapy in Treating Acute Vaso-Occlusive Pain in Sickle Cell Disease - The goal of the proposed project is to evaluate the potential of and develop HBI-201, a novel oral formulation of nitrous oxide (N2O), that enables the home use of N2O to treat pain associated with Vaso-Occlusive Crises (VOCs) in Sickle Cell Disease (SCD). The analgesic properties of N2O are well known, and N2O is an FDA- designated medical gas that is used for its analgesic, among other, properties. Importantly, current analgesic uses of N2O using inhaled gas include pain from SCD VOC in the emergency department (ED) in France, as well as in obstetric labor, acutely painful procedures such as fracture reduction, and in the management of the pain of ureteric colic. As a widely used, approved drug in the US, EU, and other geographies, the safety, tolerability, and efficacy of inhaled N2O (iN2O) has been demonstrated. However, barriers to home dosing of iN2O have prevented the development of an N2O therapeutic for home treatment of SCD VOC pain. Inhaled N2O gas is the approved modality for N2O. However, inhaled N2O is not a viable therapeutic option for home use due to poor dosing control, potential inhalation equipment issues and variability in patient ventilation and pulmonary absorption, the risk of inadvertent exposure due to leaking or incorrectly operated compressed gas cylinders, and patient resistance to gas therapies due to issues with mask and cannula use. HBI-201, a novel oral N2O drug product, enables home use of N2O in SCD and is being developed for the treatment of pain associated with VOCs in SCD. The administration of a defined dose of N2O delivered by oral administration of HBI-201 enables the further development of N2O as a therapeutic for home use while obviating the problems associated with iN2O. In this multi-PI application, we combine the expertise of the Simone and Belcher laboratories with Hillhurst’s experienced team and innovative technology and propose to build on compelling data to develop HBI-201 as a treatment for SCD VOC pain.
