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 is currently in a Phase 2B study for ASD. Suramin is a broadly acting anti-purinergic therapy and has demonstrated safety and efficacy in a small pilot study. Our Phase 2B study is targeting 48 subjects across six sites in South Africa. We expect to announce data from this study in 1H 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.


PAX-101 (IV Suramin) for HAT