§ Trappsol Cyclo may be Differentiated from Competition by a Safer Route of Administration. HPßCD is primarily administered via either intravenous (IV) and intrathecal (IT) routes. CTD Holdings is currently developing Trappsol Cyclo, a proprietary IV HPßCD formulation, while Vtesse (private) is developing VTS-270, a mixture of HPßCD that is delivered via IT injection. IV administration of HPßCD has been assessed in preclinical and clinical studies, which have found improvements in several physical and neurological disease symptoms, critical plasma NPC biomarkers, hepatosplenomegaly, reductions in neurodegeneration and cholesterol storage, and even a possible survival benefit. IV HPßCD has been generally safe and well tolerated in numerous preclinical and clinical studies for NPC, with a mild adverse event profile that mainly consisted of infusion site reactions and infections.
Similarly, IT administration of HPßCD has been tested in preclinical and clinical studies to assess the impact of the drug on the CNS, which have found similar results that include: normalizations in certain biomarkers, prevention of neurodegeneration, and temporary stabilization of disease.
HOWEVER , investigators have reported high frequency HEARING LOSS in both preclinical and clinical studies with IT administration of HPßCD, a deleterious AE that could HAMPER future clinical development, enrollment of ongoing clinical trials, or drug uptake in the event of approval.
