Trypanosomiasis, African

EMA gives positive opinion to Fexinidazole Winthrop as first oral treatment of acute form of sleeping sickness (rhodesiense) found in East and Southern Africa Paris, Geneva and Nairobi. December 15, 2023. Sanofi, DNDi and the HAT-r-ACC consortium announce the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) has adopted a positive scientific opinion of Fexinidazole Winthrop as first oral treatment of acute form of sleeping sickness (rhodesiense). This positive opinion is for the treatment in adults and children six years of age or older and weighing at least 20 kg, of both first-stage (haemo-lymphatic) and second-stage (meningo-encephalitic) Trypanosoma brucei (T.b.) rhodesiense sleeping sickness, an acute and lethal form of this parasitic disease found in Eastern and Southern Africa. This CHMP opinion follows an application by Sanofi under Article 58 and clinical trials in Malawi and Uganda led by the non-profit medical research organization Drugs for Neglected Diseases initiative (DNDi). The CHMP first adopted in 2018, a positive opinion of Fexinidazole Winthrop as the first all-oral treatment , in adults and children six years of age or older and weighing at least 20 kg, of both first-stage (haemo-lymphatic) and second-stage (meningo-encephalitic) of the more common T.b. gambiense form of sleeping sickness found in West and Central Africa. Sleeping sickness, or human African trypanosomiasis (HAT), is usually fatal without treatment. Both forms of sleeping sickness are transmitted by the bite of infected tsetse flies, which are found in 36 African countries. It causes neuropsychiatric symptoms, including aggressiveness, psychosis, a debilitating disruption of sleep patterns that have given this neglected disease its name, and ultimately, death. Dietmar Berger, MD, PhDHead of Development and Chief Medical Officer, Sanofi“The CHMP’s positive opinion is another step forward in Sanofi’s commitment to help deliver innovative treatments to vulnerable patient communities impacted by sleeping sickness, a deadly neglected tropical disease. By working with WHO and DNDi, we have made tremendous progress in improving treatment outcomes and simplifying treatment delivery. This partnership and our donation of Fexinidazole Winthrop through Foundation S, reflect our mission to provide innovative treatments to patients, no matter where they live.” For the T.b. rhodesiense variant, Fexinidazole Winthrop is indicated as a 10-day, once-daily oral treatment. Data from DNDi’s Phase 2/3 clinical trial were recently presented at the European Congress of Tropical Medicine and International Health and showed that Fexinidazole Winthrop was highly effective in treating the T.b. rhodesiense form of sleeping sickness and is a safe alternative to the existing drugs. In periodic follow-up evaluations that continued for 12 months after treatment, only one patient (2.94%) with the advanced form of the disease had relapsed and required treatment with the arsenic derivative that is the standard of care for patients with the most severe stage of the disease. Dr Westain NyirendaPrincipal Investigator and physician at Rumphi Hospital in Malawi“T.b. rhodesiense sleeping sickness is a terrifying disease that progresses more rapidly than T.b. gambiense, killing quickly if untreated. Until now, due to the lack of innovation for this strain of sleeping sickness, old and toxic treatment options have to be administered in a hospital under strict surveillance. Having a simple and safer oral pill to treat this frightening disease will allow doctors to rapidly save lives. It will also help patients to trust the new treatment.” While humans are the main host of T.b. gambiense, T.b. rhodesiense is a zoonotic disease, meaning that the infection can spread from animals to humans. Cattle and wild animals such as bushbucks and zebras are the most common reservoirs for this disease. Movements of these animals – potentially sparked by droughts or changes in climate – could put new populations at risk of T.b. rhodesiense sleeping sickness. In some cases, tourists visiting game reserves have been infected with T.b. rhodesiense. Dr Olaf Valverde MordtClinical Project Leader for sleeping sickness, DNDi“We are already seeing how an all-oral treatment for T.b. gambiense sleeping sickness has simplified the treatment of this variant in countries like the Democratic Republic of Congo. Although there are comparatively few cases of T.b. rhodesiense, last year Ethiopia recorded its first cases since the 1970s. The drought at the time of the infections brought humans and cattle in closer proximity to tsetse flies’ habitat. Environmental changes could be one of the reasons behind this resurgence.” The CHMP opinion today paves the way for the update of WHO guidelines on treatment for sleeping sickness, and distribution by WHO of Fexinidazole Winthrop in African countries where T.b. rhodesiense is prevalent. Fexinidazole Winthrop will be donated to WHO by Foundation S, Sanofi’s philanthropic organization. Dr Ibrahima Socé FallDirector of Neglected Tropical Diseases, World Health Organization “The impact of climate change extends to the shifting geographical spread of vector-borne diseases such as sleeping sickness, heightening the likelihood of spillover events where diseases transfer from animals to humans. These shifts disproportionately affect the most vulnerable communities, underscoring the urgency of sustained investment in programs addressing NTDs. This includes the development of innovative tools and improved treatment methods. We extend our gratitude to our partners DNDi for their research for the most neglected and Sanofi for their ongoing support and contribution to these vital efforts.” Fexinidazole Winthrop has already been registered in the Democratic Republic of the Congo and Uganda as a treatment for T.b. gambiense and is recommended for use in a further 10 African countries: (Angola, Burkina Faso, Central African Republic, Chad, Congo, Côte d'Ivoire, Equatorial Guinea, Gabon, Guinea, and South Sudan). Dr Michelle HelinskiSenior Project Officer for Neglected Infectious Diseases at the European and Developing Countries Clinical Trials Partnership Association (EDCTP) “We congratulate the HAT-r-ACC consortium on developing a better treatment option for this truly neglected disease and are thrilled with the positive opinion by CHMP.” The DNDi clinical trial for T.b. rhodesiense was conducted by the HAT-r-ACC Consortium, with funding from the European and Developing Countries Clinical Trials Partnership Association (EDCTP2) programme supported by the European Union; Fundação para a Ciência e a Tecnologia from Portugal; Swiss Agency for Development and Cooperation (SDC), from Switzerland; Médecins Sans Frontières International; and UK International Development, United Kingdom; and other private foundations and individuals. About SanofiWe are an innovative global healthcare company, driven by one purpose: we chase the miracles of science to improve people’s lives. Our team, across some 100 countries, is dedicated to transforming the practice of medicine by working to turn the impossible into the possible. We provide potentially life-changing treatment options and life-saving vaccine protection to millions of people globally, while putting sustainability and social responsibility at the center of our ambitions. Sanofi is listed on EURONEXT: SAN and NASDAQ: SNY About DNDiThe Drugs for Neglected Diseases initiative (DNDi) is a not-for-profit medical research organization that discovers, develops, and delivers safe, effective, and affordable treatments for neglected people. DNDi is developing medicines for sleeping sickness, leishmaniasis, Chagas disease, river blindness, mycetoma, dengue, paediatric HIV, advanced HIV disease, cryptococcal meningitis, and hepatitis C. Its research priorities include children’s health, gender equity and gender-responsive R&D, and diseases impacted by climate change. Since its creation in 2003, DNDi has joined with public and private partners across the globe to deliver twelve new treatments, saving millions of lives. dndi.org About HAT-r-ACCThe HAT-r-ACC consortium brings together a broad range of partners with expertise in sleeping sickness and capacity building in remote health settings. This research, training, and community engagement experience is essential to run the clinical trial in remote settings with a very small target population. The consortium partners include the Malawi Ministry of Health (MMoH), the Uganda National Health Research Organisation (UNHRO), the Makerere University in Uganda, Epicentre (MSF) in France, the Lisbon Institute of Hygiene and Tropical medicine (IHMT) in Portugal, the Institut de Recherche pour le Développement (IRD) in France, the WHO, and the Swiss Tropical and Public Health Institute (Swiss TPH). Sanofi Media RelationsSandrine Guendoul | + 33 6 25 09 14 25 | sandrine.guendoul@sanofi.comEvan Berland | +1 215 432 0234 | evan.berland@sanofi.comNicolas Obrist | + 33 6 77 21 27 55 | nicolas.obrist@sanofi.comVictor Rouault | + 33 6 70 93 71 40 | victor.rouault@sanofi.com Sanofi Investor RelationsEva Schaefer-Jansen | + 33 7 86 80 56 39 | eva.schaefer-jansen@sanofi.comArnaud Delépine | + 33 6 73 69 36 93 | arnaud.delepine@sanofi.comCorentine Driancourt | + 33 6 40 56 92 21 | corentine.driancourt@sanofi.comFelix Lauscher | + 1 908 612 7239 | felix.lauscher@sanofi.comTarik Elgoutni| + 1 617 710 3587 | tarik.elgoutni@sanofi.comNathalie Pham | + 33 7 85 93 30 17 | nathalie.pham@sanofi.com DNDi Media ContactFrédéric Ojardias in Geneva | +41 79 431 62 16 | fojardias@dndi.org Sanofi Forward-Looking StatementsThis press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, as amended. Forward-looking statements are statements that are not historical facts. These statements include projections and estimates and their underlying assumptions, statements regarding plans, objectives, intentions and expectations with respect to future financial results, events, operations, services, product development and potential, and statements regarding future performance. Forward-looking statements are generally identified by the words “expects”, “anticipates”, “believes”, “intends”, “estimates”, “plans” and similar expressions. Although Sanofi’s management believes that the expectations reflected in such forward-looking statements are reasonable, investors are cautioned that forward-looking information and statements are subject to various risks and uncertainties, many of which are difficult to predict and generally beyond the control of Sanofi, that could cause actual results and developments to differ materially from those expressed in, or implied or projected by, the forward-looking information and statements. These risks and uncertainties include among other things, the uncertainties inherent in research and development, future clinical data and analysis, including post marketing, decisions by regulatory authorities, such as the FDA or the EMA, regarding whether and when to approve any drug, device or biological application that may be filed for any such product candidates as well as their decisions regarding labelling and other matters that could affect the availability or commercial potential of such product candidates, the fact that product candidates if approved may not be commercially successful, the future approval and commercial success of therapeutic alternatives, Sanofi’s ability to benefit from external growth opportunities, to complete related transactions and/or obtain regulatory clearances, risks associated with intellectual property and any related pending or future litigation and the ultimate outcome of such litigation,  trends in exchange rates and prevailing interest rates, volatile economic and market conditions, cost containment initiatives and subsequent changes thereto, and the impact that pandemics or other global crises may have on us, our customers, suppliers, vendors, and other business partners, and the financial condition of any one of them, as well as on our employees and on the global economy as a whole. The risks and uncertainties also include the uncertainties discussed or identified in the public filings with the SEC and the AMF made by Sanofi, including those listed under “Risk Factors” and “Cautionary Statement Regarding Forward-Looking Statements” in Sanofi’s annual report on Form 20-F for the year ended December 31, 2022. Other than as required by applicable law, Sanofi does not undertake any obligation to update or revise any forward-looking information or statements. Attachment Press_Release

DUBAI, United Arab Emirates--( BUSINESS WIRE )--Global donors at the 2023 Reaching the Last Mile Forum today pledged a collective US$777.2 million to help control, eliminate, and eradicate neglected tropical diseases (NTDs), in a landmark push to accelerate progress towards achieving the goals outlined in the World Health Organization’s 2030 roadmap on NTDs. Uniting efforts with NTD-endemic countries, donors answered the urgent call to step up the fight against NTDs in the face of climate change, and to work together to improve the lives of the 1.6 billion people worldwide affected by these devastating yet preventable diseases. The pledging event was hosted by Reaching the Last Mile (RLM), the global health initiative driven by the philanthropy of His Highness Sheikh Mohamed Bin Zayed Al Nahyan, President of the UAE, in partnership with the Bill & Melinda Gates Foundation. The forum took place on the first ever Health Day during the United Nations Climate Change Conference (COP28). His Highness Sheikh Abdullah bin Zayed Al Nahyan, Minister of Foreign Affairs, UAE, was joined by world leaders including H.E. Samia Suluhu Hassan, President of Tanzania; and H.E. Dr Austin Demby, Minister of Health and Sanitation, Sierra Leone, in a demonstration of endemic country leadership against NTDs. Bill Gates, Co-Chair of the Bill & Melinda Gates Foundation, and His Highness Sheikh Theyab bin Mohamed Al Nahyan, Chairman of the Office of Development Affairs at the UAE’s Presidential Court, which oversees Reaching the Last Mile, were also in attendance, alongside ministers and global health leaders. His Highness Sheikh Abdullah bin Zayed Al Nahyan stated: “ The UAE is proud to host this pivotal conference and expresses gratitude to all those who have seized the opportunity to renew their commitment to end neglected tropical diseases. Today’s demonstration of global solidarity reflects a shared determination to deliver a better and more dignified future for all. Incredible progress over the last two decades has proved that a world free of NTDs is an achievable goal. We warmly thank governments, donors, and partners for standing with us as we strive to achieve it.” Reaching the Last Mile joined with the Gates Foundation and global partners to announce a milestone expansion of the Reaching the Last Mile Fund (RLMF) from $100m to $500m. The expansion will increase the reach of the fund from seven countries to 39 across Africa and Yemen, with the audacious goal of eliminating two NTDs, lymphatic filariasis and onchocerciasis (river blindness), from the continent of Africa. The funding builds on the pioneering success of the RLMF, which launched in 2017 as a 10-year, multi-donor fund, to establish a model for eliminating the two diseases in sub-Saharan Africa. His Highness Sheikh Theyab bin Mohamed Al Nahyan said: “ Today we reaffirm our pledge to eliminate these diseases of poverty, and save and improve lives worldwide. Climate change is already affecting the patterns of infectious diseases and risking the tremendous gains countries have made to date. “ With endemic countries leading the way, the Reaching the Last Mile Fund represents a historic opportunity to free communities across Africa from the threat of two NTDs, and in partnership unlock the potential for a healthier world.” “ Climate change is inextricably linked to poverty and diseases affecting millions,” said Bill Gates, Co-chair of the Bill & Melinda Gates Foundation. “ That is why funding for lifesaving healthcare is critical and why we’re joining global partners to build on the tremendous progress of the Reaching the Last Mile Fund to date, and accelerate the elimination of LF and river blindness across Africa and beyond.” Reaching the Last Mile’s new $100m commitment, matching the Gates Foundation’s $100m commitment, represents a five-fold increase over the contributions made, respectively, to the fund at its launch. Further contributions from country and donor partners including Sierra Leone, the Carter Center, Sightsavers, the Children’s Investment Foundation Fund (CIFF), The Helmsley Charitable Trust, the END Fund, and Abu Dhabi National Insurance Company will see RLMF significantly broaden its scope, with the aim of ensuring over 350 million people no longer require treatment for these diseases by 2030. H.E. Julius Maada Bio, President of Sierra Leone, said: “ There is clarity of vision as well as growing demand and energy in Africa to see the continent free of NTDs. Political commitment has been sustained and energised through the Kigali Declaration, and through the Continental Framework and Common Africa Position on NTDs, which Sierra Leone is proud to have endorsed. That is why Sierra Leone is proud to pledge towards an Africa free of river blindness and lymphatic filariasis. Neglected tropical diseases cause suffering to millions, and we simply cannot afford to neglect them any longer.” H.E. Samia Suluhu Hassan, President of Tanzania, said: " I am immensely proud to have been at the centre of the launch of this ambitious vision of an Africa free of river blindness and lymphatic filariasis. Tanzania commits to increase domestic contribution for combating NTDs but also counts on continued support from development partners in this endeavor. " While gaps in global funding for NTDs remain, this initiative signifies a crucial step forward in enhancing the health and quality of life not just for Tanzanians, but for all who live on the African continent. Together, we can end these terrible diseases, forging a path to a healthier and brighter future for us all." Over five years the RLMF has provided over 100 million treatments, trained 1.3 million health workers, and established nine laboratories to support NTD surveillance and testing. The fund also played a critical role in interrupting the transmission of river blindness in Niger, an achievement once thought to be scientifically impossible in Africa, and has supported Senegal in nearing this milestone. The wider pledging moment saw new, multimillion-dollar commitments made by organizations including the Anesvad Foundation, CIFF, USAID, Global Health EDCTP3, UBS Optimus Foundation, NALA Foundation, Evidence Action, Helen Keller Intl, and the Fred Hollows Foundation, in addition to the governments of Germany and Belgium. These pledges will help close the funding gap needed to expedite progress towards the WHO roadmap targets which call for at least 100 countries to have eliminated at least one NTD by 2030, and to reduce by 90% the number of people requiring treatment for NTDs. To date, 50 countries have eliminated at least one NTD and 600 million people no longer require treatment. Cases of some diseases that have plagued communities for centuries, such as Guinea worm disease and sleeping sickness, are at an all-time low. However, climate change is affecting the reach and prevalence of infectious diseases like NTDs, threatening to erase elimination gains and stall future progress. The commitments will help finance essential programs and treatments, support new research and innovations, and strengthen frontline health systems and workforces, among other investments. Dr Tedros Adhanom Ghebreyesus, Director General of the World Health Organization, said: " In light of the pledges made today at the 2023 Reaching the Last Mile Forum, I would like to express my profound gratitude to the global community, especially the pivotal leadership role of the United Arab Emirates, led by His Highness Sheikh Mohamed bin Zayed Al Nahyan. “ This commitment is a significant stride towards eradicating, eliminating, and controlling NTDs, aligning seamlessly with the 2021- 2030 NTD road map. The collaborative efforts of countries and global donors exemplify an unwavering dedication to improving the lives of the 1.6 billion people affected by these diseases. This partnership, especially in the face of challenges posed by climate change, represents a beacon of hope and a testament to what can be achieved through united global action" The 2023 Reaching the Last Mile Forum convened more than 450 government ministers, global health and development experts, philanthropists, and civil society leaders to galvanize concrete commitments towards mitigating the effects of the climate crisis on human health. About Reaching the Last Mile Reaching the Last Mile (RLM) is a portfolio of global health programs, investments, and initiatives working towards disease elimination that is driven by the philanthropy of His Highness Sheikh Mohamed bin Zayed Al Nahyan, President of the UAE. Through its partnerships and programs, RLM provides treatment and preventative care in communities that lack access to quality health services, with a focus on last-mile disease elimination. RLM’s mission represents His Highness’s dedication to ending preventable diseases that affect the world’s most vulnerable communities, and to helping millions of children and adults live healthy, dignified lives. https://www.reachingthelastmile.com About the Bill & Melinda Gates Foundation Guided by the belief that every life has equal value, the Bill & Melinda Gates Foundation works to help all people lead healthy, productive lives. In developing countries, it focuses on improving people's health and giving them the chance to lift themselves out of hunger and extreme poverty. In the United States, it seeks to ensure that all people—especially those with the fewest resources—have access to the opportunities they need to succeed in school and life. Based in Seattle, Washington, the foundation is led by CEO Mark Suzman, under the direction of co-chairs Bill Gates and Melinda French Gates and the board of trustees.

TARRYTOWN, NY / ACCESSWIRE / November 22, 2023 / PaxMedica, Inc. (NASDAQ:PXMD) ("PaxMedica" or the "Company"), a biopharmaceutical company focused on advancing treatments for neurological disorders, today announced the closing of its previously announced public offering of an aggregate of 5,384,615 shares of its common stock (or common stock equivalents in lieu thereof) and warrants to purchase up to 5,384,615 shares of common stock, at a public offering price of $1.30 per share (or common stock equivalent in lieu thereof) and accompanying warrant. The warrants have an exercise price of $1.30 per share, are exercisable immediately upon issuance, and will expire five years after the initial exercise date. H.C. Wainwright & Co. acted as the exclusive placement agent for the offering. Total gross proceeds to the Company from the offering, before deducting the placement agent's fees and other offering expenses, were approximately $7.0 million. The Company intends to use the net proceeds from this offering to repay in full the approximately $0.2 million outstanding under the convertible promissory note held by Lind Global Fund II LP, to advance the Company's development programs and for general corporate purposes. The securities described above were offered pursuant to a registration statement on Form S-1 (File No. 333-275416), which was declared effective by the Securities and Exchange Commission (the "SEC") on November 20, 2023. The offering was made only by means of a prospectus forming part of the effective registration statement relating to the offering. A preliminary prospectus relating to the offering has been filed with the SEC and is available on the SEC's website at Electronic copies of the final prospectus may be obtained on the SEC's website at and may also be obtained by contacting H.C. Wainwright & Co., LLC at 430 Park Avenue, 3rd Floor, New York, NY 10022, by phone at (212) 856-5711 or e-mail at placements@hcwco.com. This press release shall not constitute an offer to sell or a solicitation of an offer to buy any of the securities described herein, nor shall there be any sale of these securities in any state or other jurisdiction in which such offer, solicitation, or sale would be unlawful prior to the registration or qualification under the securities laws of any such state or other jurisdiction. About PaxMedica, Inc. 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, including Autism Spectrum Disorder (ASD) and Human African Trypanosomiasis (HAT). Our portfolio encompasses critical areas within the neurology field, with a focus on pioneering advancements in both ASD and HAT treatments. 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 and HAT treatment solutions. Our ongoing research initiatives not only prioritize the needs of ASD and HAT patients but also extend to exploring potential therapeutic applications for related conditions. Forward-Looking Statements: This release contains forward-looking statements within the meaning of Section 27A of the Securities Act of 1933, as amended and Section 21E of the Securities Exchange Act of 1934, as amended. The use of words such as "will," "expect," or "intend" and other similar expressions are intended to identify such forward-looking statements. All forward-looking statements are inherently uncertain as they are based on current expectations and assumptions concerning future events or future performance of the company. Such forward-looking statements include, but are not limited to, the intended use of proceeds from the offering. Readers are cautioned not to place undue reliance on these forward-looking statements, which are only predictions and speak only as of the date hereof. In evaluating such statements, prospective investors should review carefully various risks and uncertainties identified in this release and set forth in the company's SEC filings, including the risks found in the section entitled "Risk Factors" in PaxMedica's most recent Annual Report on Form 10-K and subsequent Quarterly Reports on Form 10-Q, as well as discussions of potential risks, uncertainties, and other important factors in any subsequent reports filed with the SEC. These risks and uncertainties could cause the company's actual results to differ materially from those indicated in the forward-looking statements. 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. View source version on accesswire.com: