Industry Spotlight: Paxmedica

About Paxmedica

PaxMedica is a clinical-stage biopharmaceutical company developing innovative treatments for unmet needs in neurodevelopmental disorders, including Autism Spectrum Disorder (ASD) and Fragile X-associated tremor/ataxia (FXTAS).

Their focus is on anti-purinergic therapies (APT), i.e. therapies that target the excess production of purines in cells. An overexpression of purines can offset homeostasis and result in an overproduction of cellular adenosine triphosphate (ATP), the main energy molecule in all living cells.

Paxmedica’s lead programs PAX-101 and PAX-102 utilize their proprietary source of suramin sodium, a broadly acting APT that has been around for over 100 years. Given suramin’s historical use as a treatment for Human African Trypanosomiasis (HAT) or African Sleeping Sickness, Paxmedica is also developing PAX-101 as a treatment for HAT. They expect that an FDA approval in HAT would grant PaxMedica with a Priority Review Voucher (PRV).

PAX-101 (IV Suramin) for ASD – drug development pipeline

PAX-101 completed a Phase 2B study for ASD in 2021. Suramin is a broadly acting anti-purinergic therapy and has demonstrated safety and efficacy in a small pilot study. The results of Paxmedica’s Phase 2B study, which targeted 52 subjects across six sites in South Africa, were presented to AACAP in October 2021.

PAX-102 (Intranasal Suramin)

PaxMedica has developed a proprietary intranasal formulation of suramin that is currently being evaluated in ASD and other neurodevelopmental conditions.

Visit Paxmedica website to learn more

Anti-Purinergic Therapeutics for Autism – BRAIN Synchrony Symposia Talks