An exclusive interview on the latest developments and plans for FDA approval for suramin, a potential breakthrough treatment for autism.
Howard Weisman CEO, PaxMedica shares with us a detailed update on the development of suramin as an evidencebased therapeutic for autism, clinical trial results, goforward plans to seek FDA approval, international availability, and the possibility of expanded access for compassionate use for autism.

00:04:40 Introduction about The BRAIN Foundation
00:06:10 Howard Weisman’s journey and becoming PaxMedica CEO
00:09:01 About purinergic signaling, Dr Naviaux’s Cell Danger Response theory and its relevance to autism
00:11:50 First study in of suramin in children with autism carried shows significant reduction in core autism symptoms, inspires creation of PaxMedica
00:13:20 Repurposing a hundred year old antiparasitic drug overcoming regulatory challenges
00:18:40 The second part of the big challenge: massproducing suramin to ensure a steady supply
00:21:00 About the second suramin in autism study that took place in 2020 in South Africa, and what was learnt from is
00:27:30 Tracking changes in autism clinical trials the differences in measuring symptoms in the South African trial vs Dr Naviaux original suramin study
00:32:30 2021 onwards advancing PaxMedica’s program, raising funds, planning mass production of suramin and the next clinical trial for autism
00:35:47 Timeline for obtaining FDA approval for suramin drug approval
00:42:25 Exploring drug safety and potential risks
00:46:00 Approving suramin outside the US and the possibility for extended/compassionate use
00:53:35 The importance of community input for PaxMedica’s mission
00:55:40 Announcement: new placebocontrolled clinical trial to take place in South Africa
01:03:36 Raising funds to support treatment trials and how PaxMedica plans to reach its goals
01:09:09 Live audience Questions & Answers
01:20:22 Alternative compounds with antipurinergic effects and the upcoming Neuromodin™ clinical trial
01:27:55 What else is happening in autism research and science and why attend The BRAIN Foundation annual Synchrony Symposium (October 2023 2023). Info & registrations: https://brainfoundation.org/synchrony...


ABOUT SURAMIN:
a 100 year old drug that has been traditionally used to treat African sleeping sickness and river blindness. In addition to its antiparasitic action, suramin is also a nonselective 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 lowdose 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 Pax101 (IV suramin) for ASD, the results of which were presented to AACAP in October 2021. This study was a doseranging, randomized, doubleblind, placebocontrolled, multidose trial evaluating the safety and efficacy of suramin in 52 children with moderate to severe autism across 6 sites in South Africa.

ABOUT PAXMEDICA
PaxMedica is a clinicalstage biopharmaceutical company developing innovative treatments for unmet needs in neurodevelopmental disorders, including ASD.


This talk was part of was part of BRAINTalk Webinar Series by the BRAIN Foundation
For more BRAINTalk Webinars see:    • BRAIN Community Webinars  



The BRAIN Foundation https://brainfoundation.org/ is a 501c(3) nonprofit. The founders of BRAIN envision a world where every child and adult on the autism spectrum is healthy, participates fully in education and employment, and has a better quality of life. It aims to catalyze research that results in evidencebased interventions for the disabilities associated with autism, and also results in better medical standard of care.

To accomplish this, it funds impactful research through #philanthropy and our network of partners in the venture, corporate, and grassroots community.