Suramin for Autism Update: 2023 and Beyond
Brought to you by the BRAIN Foundation
In partnership with PaxMedica
About the event
An exclusive interview with Howard Weisman, CEO of PaxMedica, on the latest developments and plans for FDA approval for suramin, a potential breakthrough treatment for autism.
Hosted by the BRAIN Foundation on June 17, 2023, this online interview provided an update on the development of suramin as an evidence-based therapeutic for autism, clinical trial results, go-forward plans to seek FDA approval, international availability, and the possibility of expanded access for compassionate use for autism.
Watch the recording of the event
Suramin is a 100 year old drug that has been traditionally used to treat African sleeping sickness and river blindness. In addition to its antiparasitic action, it is also a non-selective inhibitor of purinergic signaling, or in other words, an antipurinergic drug.
The Naviaux Lab at University of California, San Diego led by Robert K. Naviaux, MD, PhD, discovered that the unique properties of suramin made it the first in a new class of drugs that could be used to test the cell danger response (CDR) hypothesis for the origin and treatment of Autism Spectrum Disorder (ASD). Suramin successfully improved all the core symptoms of ASD and the metabolic and GI abnormalities in both animal models and a small human clinical trial.
About suramin clinical trials
This human clinical trial, published in 2017, was a small, randomized Phase 1 study of low-dose suramin in 10 children with ASD. The results demonstrated that a single intravenous dose of suramin improved core symptoms of autism in the five children who received the drug, compared to placebo. The improvements were noted in the domains of language, social interaction, and repetitive behaviors.
This study was followed by PaxMedica’s Phase 2 trial of Pax-101 (IV suramin) for ASD, the results of which were presented to AACAP in October 2021. This study was a dose-ranging, randomized, double-blind, placebo-controlled, multi-dose trial evaluating the safety and efficacy of suramin in 52 children with moderate to severe autism across 6 sites in South Africa.
PaxMedica is a clinical-stage biopharmaceutical company developing innovative treatments for unmet needs in neurodevelopmental disorders, including ASD. Read more about PaxMedica here.
The Naviaux Lab: Suramin research summary and mechanisms of action
UCSD press release (2017): Researchers Studying Century-Old Drug in Potential New Approach to Autism
Paxmedica press release (2021): PaxMedica Announces Positive Results from Phase 2 Trial of PAX-101 (IV suramin) in Children with Autism Spectrum Disorder
Synchrony Symposium Presentations: Watch Dr Naviaux Synchrony conference presentations on suramin for autism
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