Dimauro Disease

Acoziborole: Investigational single-dose oral treatment raises hope for elimination of sleeping sickness in Africa Positive Phase II/III study results support acoziborole’s potential in treatment for deadly disease Geneva, Kinshasa, Paris – November 30, 2022. The Drugs for Neglected Diseases initiative (DNDi) and Sanofi announce treatment success rates of up to 95% from a Phase II/III study investigating the safety and efficacy of single-dose acoziborole, a potentially transformative investigational treatment for sleeping sickness, published today in The Lancet Infectious Diseases medical journal. The clinical trial was led by DNDi and its partners in the Democratic Republic of the Congo (DRC) and Guinea. Dr Victor KandeFormer Neglected Tropical Diseases Expert Advisor at the Ministry of Health of the DRC, principal investigator of the trial, and lead author of the Lancet article “Sleeping sickness is a nightmare disease that affects patients in some of the most remote settings in West and Central Africa, where distance from hospital can be measured in days. We are now on the cusp of a potential treatment that can be given in one day, in a single dose of three pills – this would be a revolution for doctors and communities.” Transmitted by the bite of an infected tsetse fly, sleeping sickness is fatal without treatment. In the early stage of the disease, people suffer from headaches or fever. In the late stage, the parasite crosses the blood-brain barrier and invades the central nervous system, causing neuropsychiatric symptoms such as sleep disruption, confusion, lethargy, and convulsions – and ultimately, death. Never been so close to elimination in decades Treatment for sleeping sickness has radically improved in the last decade, thanks to the successes of a partnership bringing together DNDi, Sanofi, the national sleeping sickness control programmes of the DRC and Guinea, the World Health Organization (WHO), Médecins Sans Frontières (MSF), and other partners. The number of reported cases of sleeping sickness has fallen sharply in the last 20 years, from almost 40,000 reported cases in 1998 (with estimates of over 300,000 undiagnosed cases) to less than 1,000 in 2020. Though an encouraging trend, it should not be a reason for complacency, as the disease is known to resurge in the form of devastating outbreaks. Dr Antoine TarralHead of the sleeping sickness programme at DNDi and co-author of the paper“By simplifying the treatment paradigm, acoziborole would be an innovation that enables a sustainable response to sleeping sickness for health systems. With these new data, we have hope that we may be able to finally eliminate the disease, once and for all, by opening the door to a ‘screen-and-treat' approach at the village level.” Dr Wilfried Mutombo KalonjiDNDi Clinical Project Leader and Medical Manager, and co-author of the paper “Acoziborole has the potential to have a major impact on sleeping sickness treatment. This is a complicated disease, with patients living in very remote and unstable areas. We need simple approaches. We now have the promise of single-dose, oral treatment that doesn’t need to be taken with food, removes the need for hospitalization, removes the need for complex or invasive diagnosis or disease staging, and requires minimal training. In many ways, it doesn’t get any simpler than this.” The WHO Neglected Tropical Diseases Roadmap strategy sets a goal of interrupting transmission of the gambiense strain of human African trypanosomiasis (g-HAT), the scientific name of the disease, by 2030. (The gambiense strain accounts for 93% of all sleeping sickness cases). In alignment with this strategy, acoziborole could open the possibility of a simple ‘screen-and-treat’ approach, whereby any person whose rapid pinprick blood test suggests they might be infected could be treated with acoziborole, without the need for more complex parasitological confirmation or hospitalization. About the Phase II/III study Between 2016 and 2019, DNDi and its partners led an open-label, Phase II/III study to assess the safety and efficacy of acoziborole in patients with early- and late-stage g-HAT. 208 patients were recruited at 10 hospitals in the DRC and Guinea. The 18-month treatment success rate for acoziborole was 95% in late-stage g-HAT patients, corresponding to the best results from studies with existing treatments (94%). In addition, 100% of the 41 patients with early-stage g-HAT were considered as treatment successes at all timepoints. The study shows that acoziborole has a favourable safety profile, with no significant drug-related safety signals reported. These pivotal results will form the basis of Sanofi’s dossier submission to the European Medicines Agency (EMA) and represent another milestone in the quest to eliminate sleeping sickness. Upon the EMA’s positive opinion and local approval, Sanofi will donate acoziborole to the WHO through its philanthropic organization, Foundation S – The Sanofi Collective. Acoziborole is currently under clinical investigation and its safety and efficacy have not been evaluated by any regulatory authority. Dietmar Berger, MD, PhDHead of Development and Chief Medical Officer, Sanofi“Sanofi is a long-standing partner in the fight against sleeping sickness alongside DNDi and the World Health Organization. We are leveraging Sanofi’s unique expertise in developing, registering and producing new, innovative medicines to address areas of significant unmet need and in this case, support the World Health Organization’s goal to eliminate sleeping sickness in humans by 2030.” Dr Mariame Camaraprincipal investigator of the study at the HAT Treatment Centre in Dubreka, Guinea and co-author of the Lancet article“Many sleeping sickness patients live in remote villages in the mangrove swamps along the coast of Guinea – even though we have good treatment options now, we still need to send people who test positive with a sleeping sickness rapid test from their village to a confirmatory health centre. Acoziborole could potentially become the first treatment that would allow doctors to treat people on the spot, once they receive a positive rapid test in their village.” About the DNDi programme for the development of acoziborole The DNDi programme for the development of acoziborole was supported by grants from the Bill & Melinda Gates Foundation; UK aid; the Federal Ministry of Education and Research (BMBF) through KfW, Germany; the Swiss Agency for Development and Cooperation (SDC); Médecins Sans Frontières; the Dutch Ministry of Foreign Affairs (DGIS); the Norwegian Agency for Development Cooperation (Norad), Norwegian Ministry of Foreign Affairs, as part of Norway’s in-kind contribution to EDCTP2; Stavros Niarchos Foundation; the Spanish Agency for International Development Cooperation (AECID); and the BBVA Foundation (through the ‘Frontiers of Knowledge Award in Development Cooperation’). 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 DNDiA not-for-profit research and development organization, DNDi works to deliver new treatments for neglected patients, those living with Chagas disease, sleeping sickness (human African trypanosomiasis), leishmaniasis, filarial infections, mycetoma, paediatric HIV, hepatitis C, and dengue. DNDi is also coordinating the ANTICOV clinical trial to find treatments for mild-to-moderate COVID-19 cases in low-resource settings. Since its inception in 2003, DNDi has delivered twelve new treatments to date, including new drug combinations for kala-azar, two fixed-dose antimalarials, and DNDi’s first successfully developed new chemical entity, fexinidazole, approved in 2018 for the treatment of both stages of sleeping sickness. dndi.org Media Relations SanofiSandrine Guendoul | + 33 6 25 09 14 25 | sandrine.guendoul@sanofi.comEvan Berland | +1 215 432 0234 | evan.berland@sanofi.comVictor Rouault | + 33 6 70 93 71 40 | victor.rouault@sanofi.com Investor Relations SanofiEva Schaefer-Jansen | + 33 7 86 80 56 39 | eva.schaefer-jansen@sanofi.comCorentine Driancourt | + 33 6 40 56 92 21 | corentine.driancourt@sanofi.com Media Relations DNDiFrédéric Ojardias (DNDi Geneva) | +41 79 431 62 16 | fojardias@dndi.org Ilan Moss (DNDi New York) | +1 646 266 5216 | imoss@dndi.org Francine Ngalula (DNDi Kinshasa) | + 243 816 402 389 | francinengal@gmail.com Notes for the editorsAcoziborole is the first oral single-dose new chemical entity to be issued from DNDi’s lead optimization programme for sleeping sickness. It started with an initial hit identified in the chemical library of Anacor Pharmaceuticals, which was acquired by Pfizer in 2016. The initial structure was then optimized with Scynexis and Pace University and was then selected as a candidate for development and Phase I safety studies conducted successfully in France.Acoziborole is the latest innovation brought by two decades of innovation efforts by DNDi, Sanofi, and partners. In 2009, they developed a combination of existing drugs known as NECT, which was extremely effective and much safer than the toxic arsenic derivative that doctors had to use at the time. A donation programme was set up by Sanofi and Bayer to the World Health Organization (WHO), radically improving treatment options for patients.But NECT requires complex logistics, with each treatment weighing 8 kg, and must be administered in hospital settings with skilled staff. In 2018, DNDi, Sanofi, and partners developed fexinidazole, which became the first all-oral treatment available for sleeping sickness. This 10-day treatment is now available in all sleeping sickness-endemic countries. Acoziborole can be administered as a single oral dose, meaning treatment could be done in villages: healthcare workers from mobile teams will be able to immediately administer a single pill of acoziborole as soon as the patient is diagnosed, without the need of hospitalization or supervision of the treatment at home. This means it could become a major tool to facilitate the efforts to finally eliminate sleeping sickness. Sanofi Forward-Looking Statements This 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 COVID-19 will 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, 2021. 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

John Tlumacki/The Boston Globe via Getty Images Biogen has terminated its asset purchase agreement with Karyopharm Therapeutics, putting a halt to the development of KPT-350 (BIIB100), which was being tested in Phase 1 as a treatment for amyotrophic lateral sclerosis (ALS). In a U.S. Securities and Exchange Commission filing, cancer therapies developer Karyopharm wrote that the deal it signed on January 24, 2018, is no longer in force, leaving the company with no potential entitlement to any royalties or milestone payments. Biogen had paid Karyopharm $10 million upfront four years ago for the rights to KPT-350, an oral selective inhibitor of nuclear export, and related assets to bolster the company's ALS therapy development activities. Karyopharm had been eligible to receive up to $270 million more if and when more milestones were achieved. The document does not detail why the deal ended, but notably, Biogen has not been getting any positive news on the ALS front of late. In March, a Phase 1 study of BIIB078, which the company had been conducting with partner Ionis Pharmaceuticals for people with a specific type of ALS associated with the C9orf72 gene, failed to meet clinical endpoints. The program was subsequently stopped. The terminated deal with Karyopharm might not be surprising to industry observers who were somewhat wary in 2018 over the conservative amount invested into the deal. Analysts had expected Biogen to make a bigger splash, particularly as other companies at the time had been pouring billions into mergers and acquisitions. Biogen has yet to comment on the canceled deal with Karyopharm. The $3 billion ALS market remains with a huge unmet need where no cure or treatment exists. The U.S. Food and Drug Administration has approved certain drugs such as edaravone and riluzole for ALS, also called Lou Gherig's disease, but these have only demonstrated the ability to moderately slow down disease progression. All modes of treatment are related to just reducing discomfort and pain, improving nutrition and supporting mobility. Biogen is currently in Phase III studies with Ionis for tofersen, an investigational antisense drug for people living with superoxide dismutase 1 (SOD1) ALS. While the drug failed to meet the primary endpoint, new 12-month data from the Phase III VALOR study shared earlier this month demonstrated the drug's potential to slow decline in muscle strength, clinical and respiratory function. Early survival data also point to the possibility of lowering the risk for permanent ventilation or death if the drug is given early on.

Pune, India, Aug. 09, 2021 (GLOBE NEWSWIRE) -- The Global Injectable Drug Delivery Market Size is expected to value USD 800.34 million in 2021 and is projected to grow at a CAGR of 10.9 % during the forecast period. Drug delivery refers to formulations, technologies, approaches, and systems for carrying a pharmaceutical compound in the body. The injectable drug delivery devices comprise needle-free injectors, auto-injectors, conventional syringes, and pen injectors. The increase in the demand for self-injection devices is one of the key factors expected to drive market growth. Get Sample Copy of This Report @ Increasing prevalence of chronic diseases such as cancer and diabetes are also expected to contribute to market growth during the forecast period. Increasing demand for drug delivery systems and treatment of diseases, such as hepatitis C, Wilson’s disease, autoimmune diseases, hemophilia, and cystic fibrosis using injectables, is also expected to boost the market growth. Increase in healthcare spending and a rise in R&D investments are amongst major factors expected to drive the injectable drug delivery market growth. The rise in demand for generic injectables and biosimilar, rapid technological advancements in the field of injectable drug delivery will create new opportunities for the market during the forecast period. The new product introductions, and high growth in the biologics market is expected to offer significant growth opportunities for the market during the forecast period. Global Injectable Drug Delivery Market, by Type Based on type, the injectable drug delivery market is segmented into devices, and formulation type. The devices type segment is further sub segmented into conventional injection devices, self-injection devices. The formulations type is further sub segmented into conventional drug delivery formulations, novel drug delivery formulations, and long-acting injection formulations. Among these, the formulations type formulation segment is the fastest-growing segment in 2021 and is expected to remain fastest-growing during the forecast period. The segment growth can be attributed to growing patient adherence, increasing prevalence of chronic diseases, and increasing acceptance of self-injection and new technologies. Global Injectable Drug Delivery Market, by Injection Device Material Based on injection device material, the injectable drug delivery market is segmented into glass and plastics. The glass segment hold the fastest growing segment in 2021, and anticipated to remain fastest growing during the forecast period. The segment growth can be because glass poses a hindrance to atmospheric gases, such as oxygen, and carbon dioxide from entering the primary container, mitigating the risk of drug contamination. Global Injectable Drug Delivery Market, by Formulation Packaging Based on formulation packaging, the injectable drug delivery market is segmented into ampules, vials, cartridges, and bottles. The vials segment holds the largest market share in 2021 and is expected to remain the largest during the forecast period. The segment growth can be because vials offer better thermal insulation, permitting them to keep their contents at the right temperature at all times. Global Injectable Drug Delivery Market, by Therapeutic Application Based on therapeutic application, the injectable drug delivery market is segmented into auto-immune diseases, hormonal disorders, orphan diseases, oncology, and others. The oncology segment holds the largest market in 2021 and is expected to remain the largest market during the forecast period. The segment growth can be attributed to the high demand for injectable drugs from the oncology sector. Global Injectable Drug Delivery Market, by Usage Pattern Based on usage pattern, the market is segmented into curative care, immunization, and others. The curative care segment holds the largest market in 2021 and is expected to remain the largest during the forecast period. The segment growth can be attributed to the high usage of injectable drugs in curative care for various diseases such as cancer. Global Injectable Drug Delivery Market, by Site of Administration Based on site of administration, the injectable drug delivery market is segmented into skin, circulatory/musculoskeletal, organs, and central nervous system. The organs segment holds the largest market share in 2021 and is expected to be the largest during the forecast period. The segment growth can be attributed to numerous technological advancements and new product introductions to accelerate the convenience and ease of administration of parenteral therapeutics. Enquiry Before Buying This Report @ Global Injectable Drug Delivery Market, by Distribution Channel Based on distribution channel, the injectable drug delivery market is segmented into hospitals, and retail pharmacy stores. The hospitals and clinics segment holds the largest market share in 2021 and is expected to be the largest market during the forecast period. The segment growth can be due to increasing occurrence of chronic diseases, technological advancements, and patient compliance. Global Injectable Drug Delivery Market, by End Use Based on end use, the injectable drug delivery market is segmented into hospitals and clinics, home care settings, and others. The hospitals and clinics segment holds the largest market share in 2021 and is expected to be the largest market during the forecast period. The growth of market is attributed to increasing occurrence of chronic diseases, technological advancements, and patient compliance. Global Injectable Drug Delivery Market, by Region Based on region, the injectable drug delivery market is segmented into North America, Europe, Asia Pacific, the Middle East & Africa, and South America. North America holds the largest market size in 2021, and is estimated to remain the largest market during the forecast period. Factors driving the regional market growth are increasing incidence of chronic diseases, increasing demand of self-injection devices, and growth of biologics market. In addition, increased R&D investments, rise in healthcare expenditure, and government initiatives to increase awareness about chronic diseases are other factors contributing to regional injectable drug delivery market. Some Recent Developments in the Global Injectable Drug Delivery Market: COVID-19 Impact on Global Injectable Drug Delivery Market The QMI team is closely observing the effect of COVID-19 on injectable drug delivery market. The healthcare segment is overburdened with huge number of patients than the number of beds existing. Most businesses are at a temporary halt while some others are going an extra mile to create enough revenue. The injectable drug delivery market has positive impact due to increasing demand for drug delivery for COVID-19 patients and other disease as cancer, diabetics etc. Some Major Findings of the Global Injectable Drug Delivery Market Include: Browse key industry insights spread across 165 pages with 85 market data tables and 41 figures & charts from the report, “Injectable Drug Delivery Market, by Type (Devices, Formulations), Injection Device Material (Glass, Plastic), Formulation Packaging (Ampules, Vials, Cartridges, Bottles), Therapeutic Application (Auto-Immune Diseases, Hormonal Disorders, Orphan Diseases, Oncology, Other), Usage Pattern (Curative Care, Immunization, Other), Site of Administration (Skin, Circulatory/Musculoskeletal, Organs, Central Nervous System), Distribution Channel (Hospitals, Retail Pharmacy Stores), End Use (Hospitals and Clinics, Home Care Settings, Other), and Region (North America, Europe, Asia Pacific, Middle East and Africa, South America) – Market Size and Forecasting to 2030” in-depth analysis along with the table of contents (ToC). Buy Now Full Report @ Contact Us: Ajay D Quince Market Insights Pune India Phone: US +1 208 405 2835 UK +44 1444 39 0986 APAC +91 706 672 4848 Email: sales@quincemarketinsights.com Web: Browse Related Reports: ·Pharmaceutical Drug Delivery Market, By Route (Oral (Tablet), Pulmonary (Nebulizer), Injectable, Ocular (Liquid), Topical (Solid), Implantable (Active), Transmucosal), By Application (Cancer, Diabetes, Infectious Diseases, Cardiovascular Diseases, Respiratory Diseases, Central Nervous System Disorders, Autoimmune Diseases), By Facility of use (Hospital, Home Care Settings, ASC/Clinics, Diagnostic Centers), By Region (North America, Western Europe, Eastern Europe, Asia Pacific, Middle East, Rest of the World) – Market Size & Forecasting (2016-2028). ·Global Transdermal Drug Delivery Systems Market, By Type (Transdermal Patches, Transdermal Semisolids), By Application (Pain Management, Central Nervous System Disorders, Cardiovascular Diseases, Hormonal Applications, Other Applications), By End-Users (Home Care Settings, Hospitals & Clinics), By Region (North America, Eastern Europe, Western Europe, Asia Pacific, Middle East, Rest of the World) – Market Size & forecasting (2016-2025)