Narcolepsy

Takeda Pharmaceutical, Japan's largest drugmaker, reported second-quarter earnings on Thursday that beat market expectations. The company also raised its full-year earnings forecast. Takeda is currently undergoing a restructuring plan aimed at cutting costs and improving efficiency. Takeda posted an operating profit of 184.2 billion yen ($1.21 billion) in the three months through September, compared with a loss of 49.3 billion yen a year earlier, when the company was in the red. According to a survey of four analysts by LSEG, Takeda's results far exceeded the consensus forecast of 93.6 billion yen in earnings. Based on the strong first-half performance and an update to currency assumptions, Takeda raised its full-year operating profit forecast to 265 billion yen from 225 billion yen. In May, Takeda announced it would restructure to improve margins and refocus on drug discovery. The company plans to cut or reposition about 1,200 employees at centers in San Diego and Massachusetts, according to domestic media reports, and last week unveiled an early retirement plan for employees in Japan. While Takeda declined to give details on the specific positions affected, the company told Reuters that the restructuring was aimed at "maximizing the potential of our therapeutic portfolio, product lines and organizational structure for long-term growth." Takeda is trying to rebuild its drug pipeline. Last year, the company suffered failures in clinical trials for lung cancer and Crohn's disease treatments, leading to large impairment losses. However, the company has high hopes for its experimental narcolepsy treatment TAK-861 and psoriasis candidates. The drugs were acquired by Takeda from Us-Based Nimbus Therapeutics in late 2022 for up to $6 billion. While Takeda's shares are up 5.2% this year, that still underperforms the benchmark Nikkei average's 17% gain.

NEW YORK, Oct. 29, 2024 (GLOBE NEWSWIRE) -- Axsome Therapeutics, Inc. (NASDAQ: AXSM), a biopharmaceutical company developing and delivering novel therapies for the management of central nervous system (CNS) disorders, today announced seven presentations from across its broad portfolio of innovative neuroscience products and product candidates at Psych Congress 2024, being held Oct. 29 - Nov. 2, 2024, in Boston, Massachusetts. Details for the presentations are as follows: Major Depressive Disorder Title: AXS-05 (Auvelity®) in Major Depressive Disorder: Pooled Data from Two Six-Week Controlled Trials (GEMINI and ASCEND)Presentation Date and Time: Thursday, Oct. 31, 1:30 - 3 p.m. and Friday, Nov. 1, 1:30 - 3 p.m. ETLead Author: Craig Chepke, MD, SUNY Upstate Medical UniversityPoster Number: 171 Title: Effects of AXS-05 (Auvelity®) in Improving Anhedonia and Interest-Activity Symptoms of MDD and the Associated Improvements in Functional Impairment Presentation Date and Time: Thursday, Oct. 31, 1:30 - 3 p.m. and Friday, Nov. 1, 1:30 - 3 p.m. ETLead Author: Roger McIntyre, MD, Professor of Psychiatry and Pharmacology at the University of TorontoPoster Number: 172 Title: Real-World Treatment Experiences and Expectations by Symptom Severity in Patients with Major Depressive DisorderPresentation Date and Time: Thursday, Oct. 31, 1:30 - 3 p.m. and Friday, Nov. 1, 1:30 - 3 p.m. ETLead Author: Roger McIntyre, MD, Professor of Psychiatry and Pharmacology at the University of TorontoPoster Number: 198 Alzheimer’s Disease Agitation Title: Clinical Profile of AXS-05 in Treating Alzheimer’s Disease-Related Agitation: Results from The Phase 2/3 Development ProgramPresentation Date and Time: Thursday, Oct. 31, 1:30 - 3 p.m. and Friday, Nov. 1, 1:30 - 3 p.m. ETLead Author: Jeffrey Cummings, MD, ScD, Vice Chair of Research, UNLV Department of Brain HealthPoster Number: 45 OSA and Narcolepsy Title: Solriamfetol for Excessive Daytime Sleepiness in Patients with Narcolepsy and OSA Reporting Anxiety and Depression in the Real-World SURWEY StudyPresentation Date and Time: Thursday, Oct. 31, 1:30 - 3 p.m. and Friday, Nov. 1, 1:30 - 3 p.m. ETLead Author: Ulf Kallweit, MD, Assistant Professor of Neurology at the University Witten/Herdecke, GermanyPoster Number: 20 Cognition Title: Effects of Solriamfetol on Cognition in Obstructive Sleep Apnea with Excessive Daytime Sleepiness and Impaired Cognition in the SHARP Clinical TrialPresentation Date and Time: Thursday, Oct. 31, 1:30 - 3 p.m. and Friday, Nov. 1, 1:30 - 3 p.m. ETLead Author: Yaroslav Winter, MD, Mainz Comprehensive Epilepsy and Sleep Medicine Center, department of neurology, Johannes Gutenberg-University, Mainz, GermanyPoster Number: 92 Title: Effects of Solriamfetol on Cognition in Patients with Excessive Daytime Sleepiness Associated with Narcolepsy in the Real-World SURWEY StudyPresentation Date and Time: Thursday, Oct. 31, 1:30 - 3 p.m. and Friday, Nov. 1, 1:30 - 3 p.m. ETLead Author: Yaroslav Winter, MD, Mainz Comprehensive Epilepsy and Sleep Medicine Center, department of neurology, Johannes Gutenberg-University, Mainz, GermanyPoster Number: 164 About Axsome Therapeutics Axsome Therapeutics, Inc. is a biopharmaceutical company developing and delivering novel therapies for central nervous system (CNS) conditions that have limited treatment options. Through development of therapeutic options with novel mechanisms of action, we are transforming the approach to treating CNS conditions. At Axsome, we are committed to developing products that meaningfully improve the lives of patients and provide new therapeutic options for physicians. For more information, please visit the Company’s website at www.axsome.com. Forward Looking Statements Certain matters discussed in this press release are “forward-looking statements”. The Company may, in some cases, use terms such as “predicts,” “believes,” “potential,” “continue,” “estimates,” “anticipates,” “expects,” “plans,” “intends,” “may,” “could,” “might,” “will,” “should” or other words that convey uncertainty of future events or outcomes to identify these forward-looking statements. In particular, the Company’s statements regarding trends and potential future results are examples of such forward-looking statements. The forward-looking statements include risks and uncertainties, including, but not limited to, the continued commercial success of the Company’s Sunosi® and Auvelity® products and the success of the Company’s efforts to obtain any additional indication(s) with respect to solriamfetol and/or AXS-05; the Company’s ability to maintain and expand payer coverage; the success, timing and cost of the Company’s ongoing clinical trials and anticipated clinical trials for the Company’s current product candidates, including statements regarding the timing of initiation, pace of enrollment and completion of the trials (including the Company’s ability to fully fund the Company’s disclosed clinical trials, which assumes no material changes to the Company’s currently projected revenues or expenses), futility analyses and receipt of interim results, which are not necessarily indicative of the final results of the Company’s ongoing clinical trials, and/or data readouts, and the number or type of studies or nature of results necessary to support the filing of a new drug application (“NDA”) for any of the Company’s current product candidates; the Company’s ability to fund additional clinical trials to continue the advancement of the Company’s product candidates; the timing of and the Company’s ability to obtain and maintain U.S. Food and Drug Administration (“FDA”) or other regulatory authority approval of, or other action with respect to, the Company’s product candidates, including statements regarding the timing of any NDA submission; whether issues identified by FDA in the complete response letter may impact the potential approvability of the Company’s NDA for AXS-07 for the acute treatment of migraine in adults with or without aura, pursuant to the Company’s special protocol assessment for the MOMENTUM clinical trial; the Company’s ability to successfully defend its intellectual property or obtain the necessary licenses at a cost acceptable to the Company, if at all; the successful implementation of the Company’s research and development programs and collaborations; the success of the Company’s license agreements; the acceptance by the market of the Company’s products and product candidates, if approved; the Company’s anticipated capital requirements, including the amount of capital required for the continued commercialization of Sunosi and Auvelity and for the Company’s commercial launch of its other product candidates, if approved, and the potential impact on the Company’s anticipated cash runway; the Company’s ability to convert sales to recognized revenue and maintain a favorable gross to net sales; unforeseen circumstances or other disruptions to normal business operations arising from or related to domestic political climate, geo-political conflicts or a global pandemic and other factors, including general economic conditions and regulatory developments, not within the Company’s control. The factors discussed herein could cause actual results and developments to be materially different from those expressed in or implied by such statements. The forward-looking statements are made only as of the date of this press release and the Company undertakes no obligation to publicly update such forward-looking statements to reflect subsequent events or circumstance. Investors:Mark JacobsonChief Operating Officer(212) 332-3243mjacobson@axsome.com Media:Darren OplandDirector, Corporate Communications(929) 837-1065dopland@axsome.com

Data demonstrated people with narcolepsy were significantly more likely to have sleep, mood and pain disordersDUBLIN, Oct. 24, 2024 (GLOBE NEWSWIRE) -- Avadel Pharmaceuticals plc (Nasdaq: AVDL), a biopharmaceutical company focused on transforming medicines to transform lives, and nference, a health tech company dedicated to making biomedical data computable, today announced the publication of real-world data that identified the demographic characteristics and the top 20 most prevalent comorbidities of people living with narcolepsy. The paper, titled “Demographic Characteristics and Comorbidities of Patients With Narcolepsy: A Propensity-Matched Cohort Study,” was published online in Sleep Advances. These real-world findings were previously presented at the American Neurological Association (ANA) Annual Meeting in October 2022. Results describing demographic characteristics and the top 20 comorbidities were based on a review of deidentified electronic health data from more than 2,000 people with narcolepsy and 2,000 matched controls. The retrospective analysis was conducted through Mayo Clinic Platform, which leverages nference’s AI software to provide access to curated, deidentified electronic health data in a secure, privacy-protected environment. These types of data provide a more comprehensive view of patient information than claims data alone. “This is the first real-world study in narcolepsy to use aggregate electronic health record data,” said Melissa Lipford, M.D., a neurologist at the Center for Sleep Medicine at Mayo Clinic and lead author of the published paper. “We found that people with narcolepsy were more likely to have psychiatric, sleep and pain disorders. Understanding which comorbid conditions are most common should help clinicians better understand the challenges faced by patients with narcolepsy and help them optimize the management of their symptoms with the most appropriate treatment.” “We were proud to collaborate with Mayo Clinic Platform and nference to generate these real-world deidentified data from patients seen at the Mayo Clinic Health System. Narcolepsy is a chronic and complex neurological disorder and understanding the most prevalent comorbidities increased in this patient population may help to inform optimal management,” said Jennifer Gudeman, Pharm.D., Senior Vice President, Medical and Clinical Affairs of Avadel. “In contrast to claims-based studies, cardiovascular disease was not among the top 20 co-morbidities found to be increased in the narcolepsy cohort compared to the matched cohort.” About Avadel Pharmaceuticals plcAvadel 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 EDS in patients seven years and older with narcolepsy. For more information, please visit www.avadel.com. Cautionary Disclosure Regarding Forward-Looking StatementsThis press release includes “forward-looking statements” within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934. These forward-looking statements relate to our future expectations, beliefs, plans, strategies, objectives, results, conditions, financial performance, prospects or other events. Such forward-looking statements include, but are not limited to, expectations regarding the potential impact of the study on helping clinicians optimize the management of patients’ symptoms with the most appropriate treatment. In some cases, forward-looking statements can be identified by the use of words such as “will,” “may,” “could,” “believe,” “expect,” “look forward,” “on track,” “guidance,” “anticipate,” “estimate,” “project,” “next steps” and similar expressions and the negatives thereof (if applicable). The Company’s forward-looking statements are based on estimates and assumptions that are made within the bounds of our knowledge of our business and operations and that we consider reasonable. However, the Company’s business and operations are subject to significant risks, and, as a result, there can be no assurance that actual results and the results of the company’s business and operations will not differ materially from the results contemplated in such forward-looking statements. Factors that could cause actual results to differ from expectations in the Company’s forward-looking statements include the risks and uncertainties described in the “Risk Factors” section of Part I, Item 1A of the Company’s Annual Report on Form 10-K for the year ended December 31, 2023, which was filed with the Securities and Exchange Commission (SEC) on February 29, 2024, and subsequent SEC filings. Forward-looking statements speak only as of the date they are made and are not guarantees of future performance. Accordingly, you should not place undue reliance on forward-looking statements. The Company does not undertake any obligation to publicly update or revise our forward-looking statements, except as required by law. About nferencenference is unlocking the potential of healthcare data with AI, delivering real-world evidence, insights, and solutions informed by the most comprehensive multimodal data across all therapeutic areas. Our extensive de-identified, longitudinal data includes decades of clinical notes along with rich patient histories, vitals, lab tests, radiology images, digital pathology, genomics, and electrophysiology waveforms. Our federated data platform, comprised of premier healthcare organizations, including Mayo Clinic, Duke Health, Banner Health, Vanderbilt University Medical Center, and Emory Healthcare, enables advanced research and custom AI model development. With unprecedented access to data from over 40 million patient journeys, our partners can accelerate target discovery, optimize clinical trial design, and enhance lifecycle management. Follow nference on LinkedIn. Visit us at www.nference.com. AvadelInvestor Contact:Courtney MogerleyPrecision AQCourtney.Mogerley@precisionAQ.com(212) 698-8687 Media Contact:Lesley StanleyReal Chemistrylestanley@realchemistry.com (609) 273-3162 nferenceMedia Contact:Andrea SampsonPresident/CEO, Sampson Public Relations Groupasampson@sampsonprgroup.com