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 Syndrome (FXTAS).
Our 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.
Our lead programs PAX-101 and PAX-102 utilize our 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, we are also developing PAX-101 as a treatment for HAT. We expect that FDA approval in HAT could confer, upon PaxMedica, a potential Priority Review Voucher (PRV).
PAX-101 completed a Phase 2B study for ASD in 2021. Suramin is a broadly acting anti-purinergic therapy and has reported positive results from a dose range study. The results of our Phase 2B study, which targeted 52 subjects across six sites in South Africa, were presented to AACAP in October 2021.
PAX-101 has been evaluated in several preclinical studies of Fragile X related animal models. PaxMedica intends to submit an Investigational New Drug (IND) application and start a Phase 2 study in 2021. PAX-101 for FXTAS is expected to qualify for Orphan Drug exclusivity.
PaxMedica has developed a proprietary intranasal formulation of suramin that is currently being evaluated in ASD and other neurodevelopmental conditions.
PaxMedica has conducted several preclinical studies to evaluate other anti-purinergic therapies (APTs) that are more selective to specific purinergic receptors and may offer additional benefits over suramin.
The FD&C Act section 524, authorizes FDA to award priority review vouchers (PRVs) to sponsors of approved tropical disease product applications that meet certain criteria. Given suramin’s historical use as a treatment for Human African Trypanosomiasis (HAT), or African Sleeping Sickness, we are also developing PAX-101 as a treatment for HAT. Should we receive a PRV, we intend to monetize it to raise funds to support the later stage development and commercialization of PAX-101 and PAX-102 in the treatment of ASD and FXTAS.
PaxMedica’s most advanced program is the pursuit of PAX-101 for early stage East African HAT. Suramin has been in continuous use as the standard of care for the treatment of early stage East African sleeping sickness for more than 100 years. We seek to leverage this history of use, as well as patient data that we have exclusively licensed, in order to obtain an FDA approval for PAX-101. We believe that an approval of PAX-101 in HAT would qualify for New Chemical Entity exclusivity, Orphan Drug exclusivity, and could confer upon us the receipt of a Priority Review Voucher (PRV) by the FDA.