PaxMedica Inc., a Tarrytown-based clinical stage biopharmaceutical company focusing on the development of anti-purinergic drug therapies for the treatment of disorders with intractable neurologic symptoms, has initiated a Phase 3 trial, HAT-301, as part of its strategic plan to obtain U.S. market approval of PAX-101 (intravenous suramin).
According to the company, the Phase 3 HAT-301 trial is a pivotal, retrospective, controlled analysis of suramin for the treatment of the rare and fatal tropical disease, Stage 1 Trypanosoma Brucei Rhodesiense Human African Trypanosomiasis (Stage 1 TBR HAT). The study is underway at multiple primary HAT treatment sites in Uganda and Malawi.
The primary objective of the study is to demonstrate that the standard of care treatment using suramin, as currently practiced in Uganda and Malawi, leads to better clinical outcomes in patients with Stage 1 TBR HAT than observed in an untreated natural history cohort, that had documented illness prior to availability of suramin. The World Health Organization lists suramin, discovered in 1916, and found to be effective against Stage 1 HAT since at least 1920, as a standard of care for Trypanosoma b. Rhodesiense.
“We”™re excited to have launched this critical study that, if successful, is an important milestone for the submission of a New Drug Application (NDA) for PAX-101 to treat the rare tropical disease, HAT,” said CEO Howard Weisman. “This is an important part of PaxMedica”™s strategy that we believe is the fastest route to funding widespread clinical testing of suramin in autism spectrum disorder and other neurological conditions that lack effective treatments. We look forward to reviewing the final results of the study, which are expected in the first half of 2023.”
