Narcolepsy

− Pre-Specified Interim Analysis Shows Positive Safety and Efficacy Results; Mezagitamab Safe and Well Tolerated − Takeda Intends to Initiate a Global Phase 3 Trial of Mezagitamab in Patients with Primary Immune Thrombocytopenia in Fiscal Year 2024 − Late-Stage Pipeline Continues to Gain Momentum With Five New Molecular Entities in Phase 3 Development in Fiscal Year 2024 OSAKA, Japan & CAMBRIDGE, Mass.--(BUSINESS WIRE)-- Takeda (TSE:4502/NYSE:TAK) today announced positive topline results from a Phase 2, randomized, double-blind, placebo-controlled study evaluating the safety, tolerability and efficacy of mezagitamab (TAK-079) in patients with persistent or chronic primary immune thrombocytopenia (ITP). Mezagitamab is a fully human immunoglobulin IgG1 monoclonal antibody (mAb) with high affinity for CD38 expressing cells (including plasmablasts, plasma cells, natural killer cells) resulting in their depletion. The TAK-079-1004 trial (NCT04278924) evaluated three different doses of subcutaneous mezagitamab vs placebo, given once weekly for eight weeks in patients with chronic (more than one year in duration) or persistent (3-12 months in duration) primary ITP. An interim analysis of the ongoing Phase 2 study demonstrated positive safety and efficacy results. Mezagitamab has been generally safe and well tolerated across all three cohorts. All mezagitamab doses tested demonstrated a higher platelet response rate than placebo. The increases in platelet count were dose-dependent with the greatest platelet response observed at the highest dose tested. Platelet response in mezagitamab treated patients occurred rapidly and was maintained post-therapy. Based on these positive results, and following consultation with global health authorities, Takeda plans to initiate a global Phase 3 trial of mezagitamab in ITP in fiscal year 2024. When mezagitamab advances to Phase 3 in ITP Takeda will have five new molecular entities in Phase 3 development in fiscal year 2024 including TAK-279 for the treatment of psoriasis and psoriatic arthritis, TAK-861 for the treatment of narcolepsy type 1, soticlestat for the treatment of Lennox-Gastaut syndrome and Dravet syndrome, and fazirsiran for the treatment of α1-antitrypsin associated liver disease. ITP is a rare, IgG mediated autoimmune disease caused, in part, by the development of autoantibodies to platelets (and/or megakaryocytes), which are blood cells responsible for preventing or stopping bleeding. ITP is characterized by the accelerated destruction of platelets (with or without impaired production), resulting in a decreased platelet count and an increased risk of bleeding, which can be debilitating (including fatigue and impaired quality of life), and in severe cases may be life-threatening. The precedent for approval of new drugs in this indication requires that platelet counts be maintained at 50,000/uL or more for a sustained period. Approximately 20 percent of patients with ITP do not achieve a platelet count above 50,000/uL following treatment with available first- and second-line therapies creating significant patient burden and unmet need for a disease modifying treatment that is also tolerable.1,2 “These Phase 2 results demonstrate mezagitamab’s compelling disease modifying mechanism of action, which has the potential to achieve disease remission for people with ITP. There remains considerable unmet need among ITP patients who may not respond or have inadequate response to prior treatment. In addition, some patients who do respond to available therapies struggle to manage side effects or relapse despite treatment,” said Chinwe Ukomadu, Head of the Gastrointestinal & Inflammation Therapeutic Area Unit at Takeda. “We look forward to initiating the Phase 3 trial and presenting these data at an upcoming scientific congress.” Therapy with mezagitamab is designed to deliver rapid and sustained improvement in platelet response and generally rapidly restores platelet counts to functional levels. Mezagitamab previously received Orphan Drug Designation for the treatment of ITP from the U.S. Food and Drug Administration and the program recently received Fast Track Designation. Results from the Phase 2 trial have no impact on the full year consolidated reported forecast for the fiscal year ending March 31, 2024 (Fiscal Year 2023). About Takeda Takeda is focused on creating better health for people and a brighter future for the world. We aim to discover and deliver life-transforming treatments in our core therapeutic and business areas, including gastrointestinal and inflammation, rare diseases, plasma-derived therapies, oncology, neuroscience, and vaccines. Together with our partners, we aim to improve the patient experience and advance a new frontier of treatment options through our dynamic and diverse pipeline. As a leading values-based, R&D-driven biopharmaceutical company headquartered in Japan, we are guided by our commitment to patients, our people and the planet. Our employees in approximately 80 countries and regions are driven by our purpose and are grounded in the values that have defined us for more than two centuries. For more information, visit . Important Notice For the purposes of this notice, “press release” means this document, any oral presentation, any question-and-answer session and any written or oral material discussed or distributed by Takeda Pharmaceutical Company Limited (“Takeda”) regarding this release. This press release (including any oral briefing and any question-and-answer in connection with it) is not intended to, and does not constitute, represent or form part of any offer, invitation or solicitation of any offer to purchase, otherwise acquire, subscribe for, exchange, sell or otherwise dispose of, any securities or the solicitation of any vote or approval in any jurisdiction. No shares or other securities are being offered to the public by means of this press release. No offering of securities shall be made in the United States except pursuant to registration under the U.S. Securities Act of 1933, as amended, or an exemption therefrom. 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These forward-looking statements are based on assumptions about many important factors, including the following, which could cause actual results to differ materially from those expressed or implied by the forward-looking statements: the economic circumstances surrounding Takeda’s global business, including general economic conditions in Japan and the United States; competitive pressures and developments; changes to applicable laws and regulations, including global health care reforms; challenges inherent in new product development, including uncertainty of clinical success and decisions of regulatory authorities and the timing thereof; uncertainty of commercial success for new and existing products; manufacturing difficulties or delays; fluctuations in interest and currency exchange rates; claims or concerns regarding the safety or efficacy of marketed products or product candidates; the impact of health crises, like the novel coronavirus pandemic, on Takeda and its customers and suppliers, including foreign governments in countries in which Takeda operates, or on other facets of its business; the timing and impact of post-merger integration efforts with acquired companies; the ability to divest assets that are not core to Takeda’s operations and the timing of any such divestment(s); and other factors identified in Takeda’s most recent Annual Report on Form 20-F and Takeda’s other reports filed with the U.S. Securities and Exchange Commission, available on Takeda’s website at: or at . Takeda does not undertake to update any of the forward-looking statements contained in this press release or any other forward-looking statements it may make, except as required by law or stock exchange rule. Past performance is not an indicator of future results and the results or statements of Takeda in this press release may not be indicative of, and are not an estimate, forecast, guarantee or projection of Takeda’s future results. Medical Information This press release contains information about products that may not be available in all countries, or may be available under different trademarks, for different indications, in different dosages, or in different strengths. Nothing contained herein should be considered a solicitation, promotion or advertisement for any prescription drugs including the ones under development. References Provan D, Donald A, et al. Blood Advances. 2019;26;3(22):3780-3817. Rodeghiero F. International Journal of Hematology. 2023;117:316–33.

The prevalence of ADHD among prison populations is believed to be around ten times that of the general population. Amphetimine-based drugs used to treat ADHD may cut fatality risk, suggests study published in JAMA, one of the world's most widely read medical journals Medicines used to treat children and adults with attention deficit/hyperactivity disorder (ADHD) reduce the higher-than-average risk for premature death, suggests a study in the world’s most widely distributed medical journal. Researchers in Sweden tracked 148,578 children and adults with ADHD. Within three months of being diagnosed, about 57% had started on medications. Within two years, 632 patients had died, the researchers reported. Overall, among every 10,000 individuals, mortality rates were 39.1% in the medication group compared with 48.1% in those not receiving treatment. The findings were reported in JAMA , the world’s most widely circulated  international peer-reviewed general medical journal. More than half of the deaths were due to unnatural causes, such as accidental injuries, accidental poisoning, and suicide. After accounting for other risk factors, use of medication was associated with 21% lower odds of death from any cause, and 25% lower odds of death from unnatural causes, for males in particular, the researchers found. The short-term study was not a randomised trial, so it is unclear whether the use of medication explains the results and, if it does, whether the effect is seen over time. Also unclear, according to an editorial published with the study, is whether ADHD treatment reduces the risk of premature death directly by reducing impulsive behaviours, or indirectly by alleviating core symptoms of the disease. “Future research should investigate the potential relationship between ADHD symptom severity and mortality outcomes for those receiving medication treatment,” the editorial said. ADHD pharmas include Teva, Janssen & Noven ADHD affects 5.9% of young people and 2.5% of adults worldwide, according to the 2021 World Federation of ADHD International. The prevalence of ADHD among prison populations is believed to be around ten times that of the general population. An estimated 25% of adults in prison have ADHD, compared to 2.5% of adults in the general population ( Young & Cocallis, 2021 ). ADHD medicines are amphetamine-based. Amphetamine is a central nervous system stimulant, and is also used in the treatment of narcolepsy and obesity. It was discovered as a chemical in 1887 by Lazăr Edeleanu, and used as a drug in the late 1920s. The first amphetamine pharmaceutical was Benzedrine. Modern amphetamine-based medicines include Adderall ( Teva Pharmaceutical ), Concerta ( Janssen ), Daytrana ( Noven ), Dexedrine ( Cardinal Health ) and Dyanavel XR ( Tris Pharma ). Amphetamine is a tightly controlled pharmaceutical, due to the health risks associated with recreational use. About JAMA JAMA , first published in 1883. It is a member of the JAMA Network, a consortium of peer-reviewed, general medical and specialty publications. The online version is freely available to institutions in developing countries through the World Health Organization's HINARI program.

DUBLIN, Ireland, March 05, 2024 (GLOBE NEWSWIRE) -- Avadel Pharmaceuticals plc (Nasdaq: AVDL), a biopharmaceutical company focused on transforming medicines to transform lives, announced today that members of management will participate in the following upcoming investor conferences: Leerink Global Biopharma Conference: Fireside chat on Monday, March 11 at 3:20 p.m. ET.UBS Virtual CNS Day: Fireside chat on Monday, March 18 at 12:30 p.m. ET. A live webcast of these events, as well as an archived recordings, will be available on Avadel’s Investor Relations website, investors.avadel.com, for 90 days following each conference. About Avadel Pharmaceuticals plc Avadel Pharmaceuticals plc (Nasdaq: AVDL) is a biopharmaceutical company focused on transforming medicines to transform lives. Our approach includes applying innovative solutions to the development of medications that address the challenges patients face with current treatment options. Avadel’s commercial product, LUMRYZ™, was approved by the U.S. Food & Drug Administration (FDA) as the first and only once-at-bedtime oxybate for the treatment of cataplexy or excessive daytime sleepiness (EDS) in adults with narcolepsy. For more information, please visit www.avadel.com. Investor Contact:Courtney MogerleyStern Investor Relations, Inc.Courtney.Mogerley@sternir.com (212) 698-8687 Media Contact:Lesley StanleyReal Chemistrylestanley@realchemistry.com(609) 273-3162