Friday, PaxMedica Inc ( PXMD ) completed the execution of its three pivotal registration/validation batches of PAX-101, an IV formulation of suramin. 

This achievement is an essential milestone in enabling a New Drug Application submission to the FDA, which is currently planned for Q4 2024, and the potential commercial availability in the U.S. of the first and only form of suramin for Stage 1 Human African Trypanosomiasis (HAT), caused by Trypanosoma brucei rhodesiense, a fatal, neglected tropical disease if approved by the FDA.

According to the CDC, reaching this milestone is critical to PaxMedica’s journey toward potential FDA approval for PAX-101, currently the accepted standard of treatment outside the United States for the deadliest cause of HAT. 

A successful review and approval of the NDA could further qualify the company for a Tropical Disease Priority Review Voucher (PRV). 

PaxMedica ( PXMD ) plans to fund a sustainable global supply chain for PAX-101 and further advance research and clinical trials to address Autism Spectrum Disorder.

“This is a very significant milestone for PaxMedica ( PXMD ) as it endeavors to move PAX-101 towards an NDA submission,” said Howard Weisman, chairman and CEO of PaxMedica ( PXMD ). “The long-term vision for PaxMedica ( PXMD ) is to further clinical investigation of PAX-101 as a treatment for individuals who struggle with Autism Spectrum Disorder.” 

In July 2023, PaxMedica ( PXMD ) released topline data from PAX-HAT-301 Retrospective Analysis of Suramin Treatment for Stage 1 Trypanosoma Brucei Rhodesiense Human African Trypanosomiasis (S1 TBR HAT).

The study findings affirm that suramin, administered in the treatment of Stage 1 TBR HAT, resulted in improved health outcomes compared to a natural history control group of patients assessed and treated between 1900-1910 before suramin became available in Africa. 

Among the suramin-treated patients, 94% survived and completed the treatment, while in the natural history cohort, only 14% were recorded as cured, improved, or discharged. 

Price Action: PXMD shares are up 106.40% at $0.85 on the last check Friday.