- One in 36 children are diagnosed with autism spectrum disorder (“ASD”) – with ASD four times more common among boys than girls
- The SenseToKnow app accurately predicted eventual autism diagnoses in a group of 475 children – according to the study, performance was similar across sex, race, and ethnicity
- PaxMedica is leveraging historical data on the drug suramin to fund trials aimed at treating Autism Spectrum Disorder and Fragile X-Associated Tremor/Ataxia Syndrome
According to the CDC, about 1 in 36 children are diagnosed with autism spectrum disorder (“ASD”) – with ASD four times more common among boys than girls (https://ibn.fm/06kly). Currently, there is no medical test to diagnose ASD, and families often face a delay in receiving a diagnosis. Doctors must rely on developmental history and the behavior of the child to make an ASD diagnosis. A diagnosis in girls and children with minority backgrounds often has a longer wait time due to the challenges in finding appropriate experts and the variable symptoms that accompany the disorder.
In a study published in Nature Magazine, researchers introduced a digital screening device that uses machine learning to analyze different behavioral aspects to assess the likelihood of childhood ASD. The SenseToKnow app accurately predicted eventual autism diagnoses in a group of 475 children – according to the study, performance was similar across sex, race, and ethnicity (https://ibn.fm/d4xhR).
PaxMedica (NASDAQ: PXMD), a clinical-stage biopharmaceutical company focusing on the development of novel anti-purinergic therapies (“APTs”) for the treatment of ASD and other serious conditions with intractable neurologic symptoms, is focusing its lead program on ASD, which there is no approved pharmacologic treatment targeting cause and symptoms. The current ASD treatments only address the symptoms of the condition but do not target the pathophysiology itself. PaxMedica is on a promising path to address the unmet medical needs of families with ASD – bringing hope to millions.
PaxMedica is using existing research on a drug called suramin, initially created to treat a disease called Human African Trypanosomiasis (“HAT”), to support new studies for treating Autism Spectrum Disorder and a related condition known as Fragile X-Associated Tremor/Ataxia Syndrome (“FXTAS”). The original developer of suramin, the German company Bayer, had used it to treat HAT. Now, the U.S. Food and Drug Administration (“FDA”) is allowing PaxMedica to apply this old research to see if suramin can also help treat rare instances of HAT in Americans, typically caught during visits to Africa. If these trials prove that suramin is effective, PaxMedica could not only get the drug approved for use, but also receive a special coupon from the FDA called a Priority Review Voucher (“PRV”). This voucher, which can accelerate the review of a new drug, is quite valuable and could be sold to another pharmaceutical company for a significant amount of money. The company intends to channel these funds into conducting extensive trials for ASD and FXTAS treatment through its lead program, PAX-101(IV Suramin). These programs utilize a proprietary source of suramin, a century-old drug with broad therapeutic potential. Although initial FDA approval for suramin in treating sleeping sickness is anticipated within months, funding from the sale of the PRV will expedite PaxMedica’s autism application trials. The final approval for the drug’s use in treating autism may take a couple of years to materialize.
In 2024, PaxMedica is positioned to continue its momentum, sustained by achievements in 2023. The company is working to expand its product pipeline, centered on its proprietary source of suramin. PaxMedica technology is designed to help people with ASD integrate more successfully into society, enhancing their overall quality of life. PAX-101 and PAX-102’s promise extends beyond ASD, with potential applications in treating human African trypanosomiasis and other conditions characterized by intractable neurologic symptoms.
For more information, visit the company’s website at www.PaxMedica.com.
NOTE TO INVESTORS: The latest news and updates relating to PXMD are available in the company’s newsroom at https://ibn.fm/PXMD
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