PaxMedica Inc. (NASDAQ: PXMD) is a clinical stage biopharmaceutical company focusing on the development of novel anti-purinergic therapies (APTs) for the treatment of Autism Spectrum Disorder (ASD) and other serious conditions with intractable neurologic symptoms.
The company’s lead programs are focused on ASD, for which there are currently no approved pharmacologic treatments that target its cause and symptoms. Currently used treatments only address the symptoms of the condition, rather than targeting the pathophysiology itself.
PaxMedica is on a promising path to address these unmet medical needs, bringing hope to millions. Anti-purinergic therapies target the excess production of purines in cells. An overexpression of purines can offset homeostasis and result in an overproduction of cellular adenosine triphosphate, the main energy molecule in all living cells.
The company is headquartered in Tarrytown, New York.
PaxMedica is building a robust pipeline of products targeting ASD and related neurodevelopmental conditions. The company’s lead product in development may help eliminate, reduce or modulate some of the more troublesome aspects of ASD. That would open the potential for people with autism to integrate their behavior with others more successfully and improve their lives.
PaxMedica’s lead programs, PAX-101 and PAX-102, utilize the company’s proprietary source of suramin sodium, a broadly acting anti-purinergic therapy that has been known for over 100 years. Its current pipeline includes:
- PAX-101 (IV Suramin) for ASD – PAX-101 completed a Phase 2B study for ASD in 2021. Suramin is a broadly acting APT and has reported positive results from a dose range study. The results of PaxMedica’s Phase 2B study, which targeted 52 subjects across six sites in South Africa, were presented to AACAP in October 2021.
- PAX-102 (Intranasal Suramin) – PaxMedica has developed a proprietary intranasal formulation of suramin that is currently being evaluated in ASD and other neurodevelopmental conditions.
- PAX-101 for HAT – Given suramin’s historical use as a treatment for Human African Trypanosomiasis (HAT), or African Sleeping Sickness, the company is also developing PAX-101 as a treatment for HAT. PaxMedica’s most advanced program is the pursuit of PAX-101 for early-stage East African HAT.
- 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.
According to a report by Fortune Business Insights, a leading global market research company, the global ASD therapeutics market was estimated at $1.93 billion in 2022 and is projected to grow from $2.01 billion in 2023 to $3.42 billion by 2030, a CAGR of 7.9% over the forecast period. As there is no current treatment for the core symptoms of autism, PaxMedica believes the addressable market for PAX-101, if approved, could greatly exceed these forecasts.
Autistic disorder, Asperger’s Syndrome and Pervasive Development Disorder are the three main types of ASD, affecting millions of people globally. A 2020 report by the U.S. Centers for Disease Control & Prevention estimated that one in 36 children in the U.S. have been diagnosed with autism disorder.
Several factors are expected to contribute to market growth prospects. A growing prevalence of the condition globally and rising awareness coupled with available treatment options are key factors expected to drive ASD therapeutics market growth during the forecast period. Growing investment in R&D to find effective treatments is also expected to fuel global market growth.
Howard Weisman is Chairman and CEO of PaxMedica. He has been a founder and CEO of several specialty pharma and medical device companies. Most recently, he was executive chairman and co-founder of Sofregen, a biotech company. He also served as CEO and president of Seventh Sense Biosystems, a medical device development company. He also was founder, chairman and CEO of EKR Therapeutics, a specialty pharmaceutical company, and founder and COO of ESP Pharma, a company focused on cardio and neurovascular products. He has a bachelor’s degree in chemistry from Rutgers University.
David Hough, M.D., is Chief Medical Officer at PaxMedica. He is a neuroscience clinical development consultant who previously served as vice president at Janssen Research and Development and in various leadership roles over 17 years. Most recently, he was the compound development team leader for SPRAVATO® for treatment-resistant depression. Prior to that, he was the schizophrenia disease area leader. He played a pivotal role in the development programs for oral INVEGA®, INVEGA SUSTENNA® and XEPLION® for schizophrenia. He is a graduate of West Point and is board certified in psychiatry.
Stephen Sheldon is COO and CFO at PaxMedica. He has served as CEO of Thailand-based specialty healthcare company Indochina Healthcare Co. Ltd. since 2015. Previously, he was a consultant for PricewaterhouseCoopers Healthcare Advisory in the Chicago office. He was responsible for developing specialty pharmacy patient programs, strategy development for specialty products and compliance programs. He has an MBA from Thunderbird School of Global Management and a bachelor’s degree in computer science and visual arts from Bowdoin College.
- PaxMedica recently announced positive top line results from its PAX-101 Phase 3 African Sleeping Sickness Study, paving the way for an expected New Drug Application filing in 2024.
- The company granted exclusive pharmacy distribution for PAX-101 to Vox Nova LLC, the first purpose-built limited distribution specialty pharmacy benefit manager for people with ASD.
- PaxMedica submitted PAX-101 Clinical Trial Request to the South African Health Products Regulatory Authority, requesting approval for a double-blind, placebo-controlled study in ASD.
- The company entered a Research Collaboration Agreement with PoloMar Health and The BRAIN Foundation for a Phase II proof-of-concept trial in ASD.