Trypanosomiasis, African

The US gave the WHO $1.28bn in funding in 2022-2023, far more than China’s $157m. Image credit: Shutterstock / Viacheslav Lopatin. The World Health Organization (WHO) has called for country commitment to combat neglected tropical diseases, as the agency braces itself for losing its biggest funder. The WHO released a short video of director-general Dr Tedros Adhanom Ghebreyesus on World Neglected Tropical Diseases Day (30 January), outlaying the challenges facing programmes that address these types of diseases across the globe. “These programmes face challenges including funding gaps, service disruptions, and climate change,” Dr Ghebreyesus said. In a post on X , the WHO outlined the 21 tropical diseases adversely affecting human health. These diseases are called “neglected” because they have historically ranked very low on the global health agenda, leading to little funding and attention. Mosquito-borne viruses such as dengue and chikungunya, sleeping sickness, and leprosy, amongst others, feature on the list. The agency estimates that 1.5 billion people each year require treatment due to a neglected tropical disease. There are 21 neglected tropical diseases. Find out what they are and what causes them: https://t.co/kozvJDK7Ps #NeglectedNoMore How well do you really know your competitors? Access the most comprehensive Company Profiles on the market, powered by GlobalData. Save hours of research. Gain competitive edge. View profiles in store Company Profile – free sample Thank you! Your download email will arrive shortly Not ready to buy yet? Download a free sample We are confident about the unique quality of our Company Profiles. 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Submit and download Visit our Privacy Policy for more information about our services, how we may use, process and share your personal data, including information of your rights in respect of your personal data and how you can unsubscribe from future marketing communications. Our services are intended for corporate subscribers and you warrant that the email address submitted is your corporate email address. — World Health Organization (WHO) (@WHO) January 30, 2025 The WHO has a roadmap to ensure neglected tropical diseases are controlled, eliminated, or eradicated by 2030. However, as a result of reduced investment and other challenges, the agency states that this goal is at risk of not being achieved. The organisation receives funding from two main sources, notably member states and voluntary contributions. The US was the largest contributor to the WHO in 2022-2023 under former President Joe Biden, giving the agency $1.28bn. This amounted to 12% of the WHO’s total budget – in comparison, China donated $157m. President Donald Trump has recently signed an executive order detailing the country’s intent to withdraw from the WHO, citing the health agency’s political sways, mishandling of Covid-19, and unfair payment demands. Trump’s administration has already frozen the supply of HIV, malaria and tuberculosis drugs to countries supported by USAID. Dr Atul Gawande who served as assistant administrator for global health at USAID under the Biden Administration, told PBS News that waivers do not seem to include programmes for eliminating neglected tropical diseases. The WHO did announce a global health win on the eve of Neglected Tropical Diseases Day, reporting that Guinea has eliminated the gambiense form of sleeping sickness, also known as human African trypanosomiasis. Programmes in the country used insecticides to interrupt contact between infected tsetse flies and humans, along with increasing accessibility to treatment. Then on 30 January, the agency congratulated Niger on becoming the first country in Africa to eliminate onchocerciasis, commonly known as river blindness. Dr Ghebreyesus said: “To address them, programmes to fight neglected tropical diseases must be integrated into primary healthcare. We also need a ‘One Health’ approach to address the links between the health of humans, animals, and the environment. “On World Neglected Tropical Diseases Day, we’re calling on all partners to unite, act, eliminate.” Other tropical diseases such as dengue and chikungunya are more likely to require vaccinations . The WHO has previously called on member states to increase vaccine coverage in countries across the world and has placed importance on children missing out on key vaccinations. The WHO expanded its partnership with the Global Health Innovative Technology (GHIT) Fund to promote access to safe, effective and affordable drugs, vaccines and diagnostics in the area of neglected tropical diseases. GHIT Fund is a public-private partnership between the Japanese Government, 16 pharmaceutical companies, and non-profits such as the Bill & Melinda Gates Foundation. WHO’s stance on the critical need for vaccines is set against a changing US public health landscape under the new administration in the US. Neglected Tropical Diseases Day comes a day after Robert F Kennedy (RFK) Jr – Trump’s nominee for secretary for Health and Human Services (HHS) – was questioned over his position on vaccines at a Senate Confirmation Meeting. During the hearing, RFK Jr denied being anti-vaccine, despite senators bringing up previous comments that suggested otherwise.

TARRYTOWN, New York, May 15, 2024 (GLOBE NEWSWIRE) -- via IBN -- PaxMedica, Inc. (the “Company” or “PaxMedica”) (OTC:PXMD), a biopharmaceutical company dedicated to advancing treatments for neurological disorders, today announced it has timely filed a formal notice of appeal with the Nasdaq Office of Appeals and Review to reverse the decision of a Nasdaq Hearings Panel to delist the Company from Nasdaq. Following receipt of notification from Nasdaq on April 30th, PaxMedica determined to appeal the decision and intends to maintain that the Hearings Panel did not consider sufficiently or appropriately crucial aspects of PaxMedica’s recent achievements and ongoing actions to regain compliance with all applicable requirements for continued listing. PaxMedica has retained Jacob Frenkel, Dickinson Wright, PLLC as its lead counsel in the appeal, working with the consulting firm that interacted previously with the Hearings Panel. Howard Weisman, CEO of PaxMedica, commented, "We are seeking a reevaluation of Nasdaq’s recent action based on new developments that underscore our ongoing progress and commitment to addressing global health challenges. Notably, following our announcements on the completion of registration lots of PAX-101 in mid-April, the Company just submitted a briefing book for an important FDA Type C meeting scheduled for June 27th, during which we will present key updates on our plans for NDA submission later this year. We believe this, as well as our financing plans, which we made known to the Hearings Panel, support persuasively our arguments to appeal and restore trading on Nasdaq." Recent milestones for PaxMedica include: April 11th: Completion of three pivotal registration/validation batches of PAX-101, an IV formulation of suramin, crucial for ongoing regulatory submissions. April 16th: Receipt of an urgent request from Malawi’s Ministry of Health for emergency access to suramin to address a looming humanitarian crisis due to the scarcity of treatment for TBr HAT, a fatal form of African Sleeping Sickness. April 23rd: Agreement to supply Malawi with PAX-101 from our stock, reinforcing our role in combating global health emergencies. May 10th: PaxMedica submits FDA Briefing Book for June 27, 2024, Type C meeting. Weisman stated "these actions not only demonstrate our operational effectiveness, but also our vital role in the global healthcare landscape. We are encouraged that the upcoming FDA meeting, and our continuous efforts to supply life-saving treatments, will result in favorable consideration by Nasdaq in our appeal." Nevertheless, PaxMedica acknowledges that it cannot predict the outcome of the appeal. Weisman added that “we are committed to advancing expeditiously our efforts to maximize shareholder opportunities and value and pursue approval of our solutions for neurological disorders.” PaxMedica remains resolute in its mission to develop and provide innovative treatments for neurological disorders, ensuring significant contributions to public health worldwide. For more information on PaxMedica’s ongoing initiatives and developments, please visit . 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 Interested investors and shareholders are encouraged to sign up for press releases and industry updates by registering for Email Alerts at 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 Media Contact media@paxmedica.com Investor Contact Scott McGowan InvestorBrandNetwork (IBN) Phone: 310.299.1717 ir@paxmedica.com SOURCE: PaxMedica, Inc.

Researchers have shed new light on the changes in metabolism that occur between birth and the presentation of autism spectrum disorder (ASD) later in childhood. The researchers discovered that a small number of biochemical pathways are responsible for the majority of these changes, which could help inform new early detection and prevention strategies for autism. Researchers at the University of California San Diego School of Medicine have shed new light on the changes in metabolism that occur between birth and the presentation of autism spectrum disorder (ASD) later in childhood. The researchers discovered that a small number of biochemical pathways are responsible for the majority of these changes, which could help inform new early detection and prevention strategies for autism. "At birth, the physical appearance and behavior of a child who will develop autism over the next few years are indistinguishable from that of a neurotypical child. Indeed, in most cases the fate of the child with regard to autism is not set at birth," said Robert Naviaux, M.D., Ph.D., professor in the Departments of Medicine, Pediatrics and Pathology at UC San Diego School of Medicine. "We're starting to learn about the governing dynamics that regulate the transition from risk to the actual appearance of the first symptoms of ASD. Early diagnosis opens the possibility of early intervention and optimal outcomes." ASD is a developmental disorder characterized by difficulties in socializing and communication, as well as repetitive and/or restrictive behaviors. For the majority of people with ASD, the condition is a significant disability, with only 10-20 percent of children diagnosed before 5 years of age able to live independently as adults. While autism is known to have strong genetic risk factors, there are also environmental risk factors that play a role in the development and severity of ASD. Naviaux and other researchers are discovering that the development of autism is governed by the real-time interaction of these varied factors. By studying the developmental biology of metabolism and how it differs in autism, new insights are emerging in ASD and other complex developmental disorders. "Behavior and metabolism are linked -- you cannot separate them," added Naviaux. To learn more about the early metabolic changes that occur in children with autism, researchers studied two cohorts of children. One cohort consisted of newborn children, in whom autism can't be detected. The second cohort consisted of 5-year-old children, some of whom had been diagnosed with autism. When comparing the metabolic profiles of children in the cohort who were eventually diagnosed with autism to those who developed neurotypically, they found striking differences. Of the 50 different biochemical pathways the researchers investigated, just 14 were responsible for 80 percent of the metabolic impact of autism. The pathways that were most changed are related to the cell danger response, a natural and universal cellular reaction to injury or metabolic stress. The body has biochemical safeguards in place that can shut down the cell danger response once the threat has passed, and Naviaux hypothesizes that autism occurs when these safeguards fail to develop normally. The result is heightened sensitivity to environmental stimuli, and this effect contributes to sensory sensitivities and other symptoms associated with autism. "Metabolism is the language that the brain, gut and immune system use to communicate, and autism occurs when the communication between these systems is changed," added Naviaux. The cell danger response is primarily regulated by adenosine triphosphate (ATP) the body's chemical energy currency. While these ATP-signaling pathways do not develop normally in autism, they may be partially restorable with existing pharmaceutical drugs. In 2017, Naviaux and his team completed early clinical testing for suramin, the only drug approved in humans that can target ATP signaling and which is normally used to treat African sleeping sickness. Now, the researchers hope that by revealing the specific ATP-related pathways that are altered in autism, their work will help scientists develop more drugs that target these pathways to manage the symptoms of ASD. "Suramin is just one drug that targets the cell danger response," he said. "Now that we're closely interrogating how metabolism changes in ASD, we could be at the beginning of a drug renaissance that will create new options for treatment that never existed before." Co-authors on the study include: Sai Sachin Lingampelly, Jane C. Naviaux, Jonathan M. Monk, Kefeng Li and Lin Wang at UC San Diego School of Medicine and Luke S. Heuer, Lori Haapanen, Chelsea A. Kelland and Judy Van de Water at University of California Davis This work was funded, in part, by Autism Speaks (grant 7274), the National Center for Research Resources (grant UL1TR001442), and through various philanthropic gifts.