PaxMedica, Inc. announced that it has entered into an agreement with PoloMar Health to investigate the use of emodin in a clinical program as a potential treatment for patients with autism spectrum disorder. Emodin is an orally bioavailable selective P2X7 inhibitor, and is currently available as a component in a number of over-the-counter nutritional supplements. Emodin is a naturally occurring substance that is found in several plants, including rhubarb and Japanese knotweed.

PoloMar Health is also working in partnership with The BRAIN Foundation on this initiative. Under the agreement, PoloMar Health will retain rights to develop and commercialize any non-prescription supplement form of the product, while PaxMedica will retain exclusive rights to develop and commercialize a highly purified form of emodin as a prescription pharmaceutical product following FDA and other regulatory authorities' guidance. In 2019, PaxMedica completed several in vivo animal studies in a mouse model of autism and demonstrated positive results for emodin in several measures of cognition, memory, and behavior.

These findings were the basis of a patent filing that was initiated that same year by PaxMedica. A Phase II proof-of-concept trial, using a proprietary, highly bioavailable form of emodin in patients with ASD, is anticipated to begin in second quarter of 2023. PoloMar Health will be the sponsor of the U.S.-based study, and will, along with The BRAIN Foundation, provide the funding for the study.

Depending on the outcome of this study, PaxMedica may pursue a formal development program for a potential prescription-grade product to treat patients with ASD.