Suramin Sodium

TARRYTOWN, New York, May 07, 2024 (GLOBE NEWSWIRE) -- via IBN -- PaxMedica, Inc. (OTC:PXMD), a biopharmaceutical company focused on advancing treatments for neurological disorders, provided a corporate update in light of its transition off the NASDAQ stock market, emphasizing this change will not affect the company’s commitment to advancing treatments for neurological disorders. Howard Weisman, CEO of PaxMedica, affirmed, "Our determination to progress with the NDA submission for PAX-101 remains unaffected, as does our commitment to our clinical program to further explore the potential for anti-purinergic therapies to address the core symptoms of autism spectrum disorder (ASD)." The company is preparing to file a New Drug Application (NDA) with the FDA for the use of suramin in treating Human African Trypanosomiasis (HAT) as it has previously reported. Suramin has been recognized for nearly a century as a standard treatment for HAT in East Africa. In April 2024, PaxMedica responded to an urgent request from Malawi for an emergency release of suramin, underscoring the company’s commitment to addressing global health crises. "We are not just continuing; we are intensifying our efforts to achieve significant milestones for these programs by the end of this year," Weisman added. "We are on track to submit an NDA for PAX-101, and potentially secure an FDA Priority Review Voucher if approved." This proactive approach illustrates PaxMedica's dedication to advancing healthcare solutions globally. About PaxMedica PaxMedica, Inc. is a forward-looking clinical-stage biopharmaceutical firm specializing in cutting-edge anti-purinergic drug therapies (APT) aimed at addressing a range of challenging neurologic disorders. Our comprehensive portfolio encompasses a spectrum of conditions, including neurodevelopmental disorders such as Autism Spectrum Disorder (ASD), as well as other critical areas within the neurology field. Additionally, we intend to provide the rest of the world with an additional, reliable source of suramin, the accepted standard of care for Stage 1, Trypanosoma Brucei Rhodesiense. We are dedicated to the continuous development and evaluation of our pioneering program, PAX-101, an intravenous suramin formulation that lies at the heart of our efforts, particularly focused on innovative ASD treatment solutions. Our ongoing research initiatives not only prioritize the needs of ASD patients, but also extend to exploring potential therapeutic applications for related conditions. To learn more about our transformative work, please visit www.paxmedica.com. Interested investors and shareholders are encouraged to sign up for press releases and industry updates by registering for Email Alerts at https://www.paxmedica.com/email-alerts and by following PaxMedica on Twitter and LinkedIn. Forward-Looking Statements This press release contains “forward-looking statements.” Forward-looking statements reflect our current view about future events. Investors can identify these forward-looking statements by words or phrases such as “may,” “will,” “could,” “expect,” “anticipate,” “aim,” “estimate,” “intend,” “plan,” “believe,” “is/are likely to,” “propose,” “potential,” “continue” or similar expressions. These forward-looking statements include our anticipated clinical program, the timing and success of our anticipated data announcements, pre-clinical and clinical trials and regulatory filings, and distribution and demand for our product candidates. These forward-looking statements involve known and unknown risks and uncertainties and are based on the Company’s current expectations and projections about future events that the Company believes may affect its financial condition, results of operations, business strategy and financial needs. Such risks and uncertainties include, but are not limited to, risks associated with the Company’s development work, including any delays or changes to the timing, cost and success of the Company’s product development and clinical trials, risk of insufficient capital resources, cash funding and cash burn, regulatory approvals and risks associated with intellectual property and infringement claims. The Company undertakes no obligation to update or revise publicly any forward-looking statements to reflect subsequent occurring events or circumstances, or changes in its expectations, except as may be required by law. Although the Company believes that the expectations expressed in these forward-looking statements are reasonable, it cannot assure you that such expectations will turn out to be correct, and the Company cautions investors that actual results may differ materially from the anticipated results and encourages investors to review other factors that may affect its future results described in the Company’s “Risk Factors” section and other sections in its most recent Annual Report on Form 10-K, and subsequent quarterly and other filings with the U.S. Securities and Exchange Commission. Contacts PaxMedica, Inc. 303 S Broadway, Suite 125. Tarrytown, NY 10591 www.paxmedica.com Media Contact media@paxmedica.com Investor Contact Scott McGowan InvestorBrandNetwork (IBN) Phone: 310.299.1717 ir@paxmedica.com www.paxmedica.com/investors SOURCE: PaxMedica, Inc.

Pictured: A child covering his ears over a neuron background/Taylor Tieden for BioSpace With the prevalence of autism on the rise in the U.S., the biopharma industry is making a concerted effort to develop new treatments to alleviate the challenges associated with the neurological condition. Diagnoses of autism spectrum disorder (ASD) rose from 1 in 54 in 2016 to 1 in 36 in 2020, according to the Centers for Disease Control and Prevention. However, efforts by the biopharma industry to find treatments have also accelerated; more than 100 drugs are in trial or have completed studies, according to the autism news site Spectrum. But despite this significant push, challenges remain when it comes to developing a successful treatment for ASD. R&D Hurdles Remain One of the biggest hurdles involves measuring the condition precisely. Paul Wang, the director of clinical research associates and a senior clinical research scientist at the Simons Foundation, said that unlike in cancer, where specific metrics such as survival can be measured, or diabetes, where blood sugar can be precisely tracked, measuring improvements in autism is more of an “open question.” “Up to this point, the best things that we have are surveys and questionnaire rating scales, and . . . for kids and for those who have a more severe impairment, they can’t respond to those rating scales themselves,” Wang told BioSpace. “It depends on a parent’s impression or clinicians’ impression, and there’s always going to be a lot of imprecision and noisiness around that.” Additionally, Michael Tranfaglia, medical director and chief scientific officer at the FRAXA Research Foundation—an organization dedicated to finding treatments for Fragile X syndrome, the most common inherited cause of autism—told BioSpace that, despite ongoing research, the specific genetic mutations or factors causing ASD have yet to be determined. The heterogeneity of the condition poses another challenge, Tranfaglia said. Selective serotonin reuptake inhibitors (SSRIs) have been tested for ASD, but the results were “very disappointing” for this reason. “It was really hard to show that [SSRIs] worked in autism because we got a whole mixed bag of results.” He noted that there were subgroups of patients who appeared to have a “dramatically positive response,” while others got worse, and still others had no response at all. Wang agreed that one drug or treatment will not help 100% of individuals with ASD. Because of this, he said there has been a reluctance by most of the larger pharmaceutical companies to go into the ASD treatment space. Autism Candidates in the Clinic One large pharma bucking this trend is Roche. The company is investigating the small molecule alogabat, a highly selective positive allosteric modulator of the GABAA α5 receptor, which is expressed in brain regions that are key to ASD. Roche is investigating the molecule’s efficacy, safety and tolerability in an ongoing Phase II study for patients aged 15–45, a company spokesperson told BioSpace in an email, adding that alogabat has been safe and well tolerated so far. “No approved pharmacological treatment exists for the core social communication and social interaction deficits or restricted and repetitive behaviors of ASD, and this disorder continues to be an area of high-unmet medical need,” the spokesperson said. In Westchester County, New York, Curemark is on a mission to develop new therapies for neurodegenerative and neuro-developmental disorders, including ASD. Founder and CEO Joan Fallon told BioSpace that when autism was first emerging in the 1980s and 1990s, she noticed that affected patients were not eating very much protein. Investigating further, she found that children with ASD could not digest protein due to low enzyme activity. This spurred Fallon to launch Curemark in 2007. The company’s lead asset, an enzyme called CM-AT, aims to enhance protein digestion, restoring the essential amino acids in children to boost neurological performance. In November 2023, Curemark published long-term data from a clinical study of CM-AT in JAMA. Curemark reported that the trial, which investigated the irritability/agitation subscale of the Aberrant Behavior Checklist (ABC-I), showed a statistically significant difference for individuals taking CM-AT after 12 and 36 weeks. “What we saw in our trials gives me a lot of hope that the children can change, and there can be a sustained change for them over time,” Fallon said. Curemark is in the process of submitting a Biologics License Application for CM-AT and beginning more clinical trials. Also based in New York, Yamo Pharmaceuticals is developing L1-79, a tyrosine hydroxylase inhibitor that aims to improve communication and socialization in individuals with ASD by modulating the catecholaminergic pathways associated with the disorder. Yamo kicked off a Phase II trial in 2022 and is expecting results by early August. CEO Chuck Bramlage told BioSpace that two previous trials have shown “positive results.” Bramlage, the former CEO of Pearl Therapeutics, said he joined Yamo to get back to working in a therapeutic area with a massive unmet need, especially for children. “I feel bad for the parents of children with autism because they’ve been told a lot of things that would work, and nothing has,” he said. Still others are investigating existing medicines as potential treatments for ASD. Tarrytown, New York–based PaxMedica is studying PAX-101 (suramin), a therapy for human African trypanosomiasis, or African sleeping sickness, for autism. An over abundance of purines in cells can offset hemostasis and cause an overproduction of cellular adenosine triphosphate. Suramin targets extra purines with the aim of reducing the symptoms of ASD, PaxMedica CEO Howard Weisman told BioSpace. The company posted results from a 14-week proof-of-concept study of 44 ASD patients who completed the study in the Annals of General Psychiatry last year. Weisman said the results were “encouraging” and noted that PAX-101 was well-tolerated. Preclinical assets for ASD are also showing promise. Calgary-based Marvel Biosciences posted interim results of a study of MB-204, a derivative of an adenosine A2A receptor antagonist used to treat Parkinson’s disease, in early March. The study found that a single oral dose of MB-204 restored social deficits and enhanced certain social interaction behaviors to normal levels in an autism mouse model. Mark Williams, president and chief science officer at Marvel, told BioSpace that based on this result, the company is planning for Phase I study, which it expects to initiate in 2025. “I’m very hopeful that if we’re right, it’s possible that after a short course of therapy, you might see dramatic behavioral changes with people on a drug like [MB]-204,” he said. While these clinical and preclinical data are encouraging, Wang said it will take time to get autism treatments across the line. “The brain is the most complex organ in the body, and we just don’t understand it completely,” Wang said. Despite this, he expressed optimism that progress is being made toward a greater understanding of ASD, and said he is hopeful that “we will eventually get to the point where we have helpful supports.” Tyler Patchen is a staff writer at BioSpace. You can reach him at tyler.patchen@biospace.com. Follow him on LinkedIn.