Our Development Pipeline

PaxMedica is a clinical-stage biopharmaceutical company focused on developing innovative treatments for unmet needs in neurodevelopmental disorders. Our lead programs are focused on two of the world’s most challenging conditions – Autism Spectrum Disorder (ASD) and Fragile X-associated Tremor / Ataxia (FXTAS).

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PAX-101 (IV Suramin) for ASD

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 our Phase 2B study, which targeted 52 subjects across six sites in South Africa, were presented to AACAP in October 2021.

Brain Scan

PAX-101 (IV Suramin) for FXTAS

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.

PAX-102 (Intranasal Suramin)

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

intranasal spray bottles

Selective APTs

PaxMedica has conducted several preclinical studies to evaluate other APTs that are more selective to specific purinergic receptors and may offer additional benefits over suramin.


Our PRV Pipeline​

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 would 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.

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PAX-101 (IV Suramin) for HAT

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.