Endometriosis

INGREZZA® (valbenazine) Third Quarter Net Product Sales of $613 Million Representing 26% Year-Over-Year Growth INGREZZA ® (valbenazine) 2024 Net Product Sales Guidance Raised to $2.30 - $2.32 Billion Board Authorizes $300 Million Share Repurchase Plan SAN DIEGO, Oct. 30, 2024 /PRNewswire/ -- Neurocrine Biosciences, Inc. (Nasdaq: NBIX) today announced its financial results for the third quarter ended September 30, 2024 and provided an update on its 2024 financial guidance. "With continued INGREZZA growth across the tardive dyskinesia and Huntington's disease chorea indications, FDA Priority Review for crinecerfont in congenital adrenal hyperplasia, a deep neuroscience focused pipeline and a strong balance sheet, we are confident in our ability to help more patients than ever before," said Kyle W. Gano, Ph.D., Chief Executive Officer of Neurocrine Biosciences. William Rastetter, Chairman of the Board of Directors of Neurocrine Biosciences, said, "The share repurchase authorization reflects the Board's confidence in Neurocrine's significant value creation potential. Importantly, the new share repurchase authorization preserves our flexibility to drive sustained growth through investments in INGREZZA and the anticipated launch of crinecerfont, while also advancing our diverse pipeline and maintaining our strong balance sheet." Financial Highlights INGREZZA Net Product Sales Highlights INGREZZA third quarter 2024 net product sales were $613 million and grew 26% compared to the third quarter 2023 Year-over-year growth driven by strong underlying patient demand and improvement in gross-to-net dynamics Other Key Financial Highlights Differences in third quarter 2024 GAAP and Non-GAAP operating expenses compared with third quarter 2023 were driven by: Increased R&D expense in support of an expanded and advancing portfolio including investments in muscarinic compounds, gene therapy programs, and second generation VMAT2 inhibitors. Third quarter 2024 R&D expense includes $39 million for development milestones achieved under collaborations with Nxera Pharma UK Limited (Nxera, formerly known as Sosei Heptares) and Voyager Therapeutics, Inc. (Voyager). Increased SG&A expense includes incremental investment in crinecerfont-related headcount, crinecerfont-related pre-launch activities, and continued investment in INGREZZA, including the recent expansion of our psychiatry and long-term care sales teams in September 2024. Third quarter 2024 GAAP net income and earnings per share were $130 million and $1.24, respectively, compared with $83 million and $0.82, respectively, for third quarter 2023 Third quarter 2024 Non-GAAP net income and earnings per share were $189 million and $1.81, respectively, compared with $156 million and $1.54, respectively, for third quarter 2023 Differences in third quarter 2024 GAAP and Non-GAAP net income compared with third quarter 2023 driven by: Higher INGREZZA net sales and improved operating margin Third quarter 2024 includes $17 million loss from changes in fair values of equity investments compared with $40 million loss for third quarter 2023 (Non-GAAP adjustment) Third quarter 2024 includes $39 million of expense for development milestones achieved under collaborations with Nxera and Voyager At September 30, 2024, the Company had cash, cash equivalents and marketable securities totaling approximately $1.9 billion A reconciliation of GAAP to Non-GAAP financial results can be found in Table 3 and Table 4 at the end of this news release. Recent Developments Kyle W. Gano, Ph.D. appointed Chief Executive Officer effective October 11, 2024. Announced the Company's Board of Directors has authorized a $300 million share repurchase plan. The Company subsequently intends to enter into a $300 million accelerated share repurchase transaction in the coming days, subject to market conditions, which will constitute the entirety of the authorized share repurchase plan. Announced positive topline data for the Phase 2 study of NBI-1117568, a first-in-class, orally active, highly selective investigational M4 agonist, in development as a potential treatment for schizophrenia. The successful completion of the Phase 2 study triggered a $35 million milestone payment to Nxera in the third quarter of 2024. We expect to advance NBI-1117568 into Phase 3 development in the first half of 2025, which would trigger an additional $15 million milestone payment to Nxera upon initiation of the Phase 3 study. Presented KINECT ®-HD2 interim data at the 2024 MDS International Congress of Parkinson's Disease and Movement Disorders demonstrating robust and sustained improvements in chorea associated with Huntington's Disease through week 104 irrespective of antipsychotic use. Announced the ERUDITE™ Phase 2 study of luvadaxistat (NBI-1065844) in cognitive impairment associated with schizophrenia (CIAS) did not meet its primary endpoint. In addition, we provided Takeda Pharmaceutical Company Limited with written notice of termination of the license agreement to develop and commercialize luvadaxistat and NBI-1065846. The termination is anticipated to be effective in April 2025. Provided Idorsia Pharmaceuticals Ltd. with written notice of termination of the license agreement to develop and commercialize NBI-827104 in epileptic encephalopathy with continuous spike and wave during sleep. The termination is anticipated to be effective in January 2025. Raised 2024 Net Sales Guidance and Updated Expense Guidance Conference Call and Webcast Today at 8:00 AM Eastern Time Neurocrine Biosciences will hold a live conference call and webcast today at 8:00 a.m. Eastern Time (5:00 a.m. Pacific Time). Participants can access the live conference call by dialing 800-225-9448 (US) or 203-518-9708 (International) using the conference ID: NBIX. The webcast and accompanying slides can also be accessed at approximately 8:00 a.m. Eastern Time on Neurocrine Biosciences' website under Investors at . A replay of the webcast will be available on the website approximately one hour after the conclusion of the event and will be archived for approximately one month. About Neurocrine Biosciences Neurocrine Biosciences is a neuroscience-focused, biopharmaceutical company with a simple purpose: to relieve suffering for people with great needs, but few options. We are dedicated to discovering and developing life-changing treatments for patients with under-addressed neurological, neuroendocrine, and neuropsychiatric disorders. The company's diverse portfolio includes FDA-approved treatments for tardive dyskinesia, chorea associated with Huntington's disease, endometriosis* and uterine fibroids*, as well as a robust pipeline including multiple compounds in mid- to late-phase clinical development across our core therapeutic areas. For three decades, we have applied our unique insight into neuroscience and the interconnections between brain and body systems to treat complex conditions. We relentlessly pursue medicines to ease the burden of debilitating diseases and disorders, because you deserve brave science. For more information, visit neurocrine.com, and follow the company on LinkedIn, X (Formerly Twitter) and Facebook. (*in collaboration with AbbVie) NEUROCRINE BIOSCIENCES, NEUROCRINE and YOU DESERVE BRAVE SCIENCE are registered trademarks of Neurocrine Biosciences, Inc. The Neurocrine logo is a trademark of Neurocrine Biosciences, Inc. Non-GAAP Financial Measures In addition to the financial results and financial guidance that are provided in accordance with accounting principles generally accepted in the United States (GAAP), this press release also contains the following Non-GAAP financial measures: Non-GAAP R&D expense, Non-GAAP SG&A expense, and Non-GAAP net income and net income per share. When preparing the Non-GAAP financial results and guidance, the Company excludes certain GAAP items that management does not consider to be normal, including recurring cash operating expenses that might not meet the definition of unusual or non-recurring items. In particular, these Non-GAAP financial measures exclude: non-cash stock-based compensation expense, charges associated with convertible senior notes, vacated legacy campus facility costs, net of sublease income, non-cash amortization expense related to acquired intangible assets, acquisition and integration costs, changes in fair value of equity security investments, changes in foreign currency exchange rates and certain adjustments to income tax expense. These Non-GAAP financial measures are provided as a complement to results provided in accordance with GAAP as management believes these Non-GAAP financial measures help indicate underlying trends in the Company's business, are important in comparing current results with prior period results and provide additional information regarding the Company's financial position. Management also uses these Non-GAAP financial measures to establish budgets and operational goals that are communicated internally and externally and to manage the Company's business and evaluate its performance. The Company provides guidance regarding combined R&D and SG&A expenses on both a GAAP and a Non-GAAP basis. A reconciliation of these GAAP financial results to Non-GAAP financial results is included in the attached financial information. Forward-Looking Statements In addition to historical facts, this press release contains forward-looking statements that involve a number of risks and uncertainties. These statements include, but are not limited to, statements related to: the benefits to be derived from our products and product candidates; the value our products and/or our product candidates may bring to patients; the continued success of INGREZZA; our financial and operating performance, including our future revenues, expenses, or profits; our collaborative partnerships; expected future clinical and regulatory milestones; the timing of the initiation and/or completion of our clinical, regulatory, and other development activities and those of our collaboration partners; and our intention to enter into an accelerated share repurchase transaction, including the expected dollar amounts and the timing of the transaction. Among the factors that could cause actual results to differ materially from those indicated in the forward-looking statements are: our future financial and operating performance; risks and uncertainties associated with the commercialization of INGREZZA; risks that the crinecerfont New Drug Applications (NDAs) may not obtain regulatory approval, such approval may be delayed, or may not receive the benefits associated with priority review; risks related to the development of our product candidates; risks associated with our dependence on third parties for development, manufacturing, and commercialization activities for our products and product candidates, and our ability to manage these third parties; risks that the FDA or other regulatory authorities may make adverse decisions regarding our products or product candidates; risks that clinical development activities may not be initiated or completed on time or at all, or may be delayed for regulatory, manufacturing, or other reasons, may not be successful or replicate previous clinical trial results, may fail to demonstrate that our product candidates are safe and effective, or may not be predictive of real-world results or of results in subsequent clinical trials; risks that the potential benefits of the agreements with our collaboration partners may never be realized; risks that our products, and/or our product candidates may be precluded from commercialization by the proprietary or regulatory rights of third parties, or have unintended side effects, adverse reactions or incidents of misuse; risks associated with government and third-party regulatory and/or policy efforts which may, among other things, impose sales and pharmaceutical pricing controls on our products or limit coverage and/or reimbursement for our products; risks associated with competition from other therapies or products, including potential generic entrants for our products; constraints, volatility, or disruptions in the capital markets or other factors affecting our ability to enter into or complete an accelerated share repurchase transaction; and other risks described in our periodic reports filed with the SEC, including our Quarterly Report on Form 10-Q for the quarter ended September 30, 2024. Neurocrine Biosciences disclaims any obligation to update the statements contained in this press release after the date hereof other than as required by law. SOURCE Neurocrine Biosciences, Inc. WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Strategic partnership explores therapeutic effect of Lisata’s novel cyclic peptide product candidate, certepetide, in combination with bevacizumabBASKING RIDGE, N.J., Oct. 28, 2024 (GLOBE NEWSWIRE) -- Lisata Therapeutics, Inc. (Nasdaq: LSTA), a clinical-stage pharmaceutical company developing innovative therapies for the treatment of advanced solid tumors and other serious diseases, today announced it has entered into a sponsored research agreement with the University of Cincinnati to investigate Lisata’s novel cyclic peptide product candidate, certepetide, in combination with bevacizumab (a VEGF inhibitor) in a preclinical animal model for the treatment of endometriosis. Bevacizumab has previously demonstrated efficacy in treating endometriosis; however, the required dosage levels are associated with systemic side effects, limiting its use in otherwise healthy young women. Under the agreement, the University of Cincinnati will conduct the research, while Lisata will provide both funding and certepetide. “We are pleased to have entered into a strategic scientific partnership with the University of Cincinnati to investigate the potential therapeutic applications of certepetide beyond oncology,” stated Kristen K. Buck, M.D., Executive Vice President of Research and Development and Chief Medical Officer of Lisata. “Given the selective targeting of endometrial tissue by certepetide due to its interaction with the surface integrin and neuropilin-1 receptors, we believe the combination of certepetide and bevacizumab could potentially offer an effective and targeted treatment for endometriosis.” “We're excited to join forces with Lisata to explore the potential of certepetide for endometriosis,” stated Katherine Burns, PhD, Associate Professor at the University of Cincinnati College of Medicine. “By combining our expertise and resources, we aim to uncover insights that could change the treatment and management of endometriosis.” Endometriosis, a debilitating condition affecting 6.5 million women in the U.S. alone1, occurs when uterine tissue grows outside the uterus and attaches to other organs. This can result in severe pain, infertility, and other reproductive health issues, as well as nausea, depression, and anxiety. Despite its prevalence, endometriosis remains underdiagnosed and misunderstood, with limited treatment options often requiring invasive surgical procedures. Researchers are actively working to better understand its causes and develop innovative therapies, with the goal of improving the lives of those affected by this condition.______________________________________1 Ellis, K., Munro, D., & Clarke, J. (2022). Endometriosis Is Undervalued: A Call to Action. Frontiers in global women's health, 3, 902371. https://doi.org/10.3389/fgwh.2022.902371 About Certepetide Certepetide is an investigational drug designed to selectively activate the C-end rule active transport mechanism in a tissue-specific manner, resulting in systemically co-administered agents more efficiently penetrating and accumulating in the tissue. To date, certepetide has demonstrated favorable safety, tolerability, and clinical activity in completed and ongoing oncology clinical trials designed to demonstrate its ability to enhance the effectiveness of standard-of-care chemotherapy for pancreatic cancer, as well as the combination of chemotherapy and immunotherapy in a variety of solid tumors. Beyond its promising applications in oncology, certepetide's unique mechanism of action has the potential to be explored in various non-oncology settings. Its ability to selectively target specific tissues could offer new therapeutic possibilities for a range of diseases. Certepetide has been awarded Fast Track designation (U.S.) and Orphan Drug Designation for pancreatic cancer (U.S. and E.U.), as well as Orphan Drug Designation for glioma, osteosarcoma, and cholangiocarcinoma (U.S.). Additionally, certepetide has received Rare Pediatric Disease Designation for osteosarcoma (U.S.). About Lisata Therapeutics Lisata Therapeutics is a clinical-stage pharmaceutical company dedicated to the discovery, development and commercialization of innovative therapies for the treatment of advanced solid tumors and other major diseases. Lisata’s product candidate, certepetide, is an investigational drug designed to activate a novel uptake pathway that allows co-administered or tethered anti-cancer drugs to selectively target and penetrate solid tumors more effectively. Lisata has already established noteworthy commercial and R&D partnerships based on its CendR Platform® technology. The Company expects to announce numerous milestones during the next 1.5 years and believes that its projected capital will fund operations into early 2026, encompassing anticipated data milestones from its ongoing and planned clinical trials. Learn more about certepetide’s mechanism of action in our short film. For more information on the Company, please visit www.lisata.com. About University of Cincinnati Founded in 1819, the University of Cincinnati ranks among the nation’s best urban public research universities. Home to more than 53,000 students, 11,000 faculty and staff and 332,000 alumni, UC combines a Top 35 public research university with a physical setting The New York Times calls “the most ambitious campus design program in the country.” With the launch of Next Lives Here, the Cincinnati Innovation District, a $100 million JobsOhio investment, continuing record enrollment, global leadership in cooperative education, a dynamic academic health center and entry into the Big 12 Conference, UC’s momentum has never been stronger. UC’s annual budget tops $1.65 billion and its endowment totals $1.8 billion. Learn more at uc.edu. Forward-Looking Statements This communication contains “forward-looking statements” that involve substantial risks and uncertainties for purposes of the safe harbor provided by the Private Securities Litigation Reform Act of 1995. All statements, other than statements of historical facts, included in this communication regarding the Company’s clinical development programs are forward-looking statements. In addition, when or if used in this communication, the words “may,” “could,” “should,” “anticipate,” “believe,” “estimate,” “expect,” “intend,” “plan,” “predict” and similar expressions and their variants, as they relate to Lisata or its management, may identify forward-looking statements. Examples of forward-looking statements include, but are not limited to, the potential efficacy of certepetide combined with bevacizumab as a treatment for patients with endometriosis; and certepetide’s potential in oncology and non-oncology applications. Actual results could differ materially from those contained in any forward-looking statement as a result of various factors, including, without limitation: results observed from a single patient case study are not necessarily indicative of final results and one or more of the clinical outcomes may materially change following more comprehensive reviews of the data and as more patient data becomes available, including the risk that unconfirmed responses may not ultimately result in confirmed responses to treatment after follow-up evaluations; the risk that product candidates that appeared promising in early research and clinical trials do not demonstrate safety and/or efficacy in larger-scale or later clinical trials; the safety and efficacy of Lisata’s product candidates, decisions of regulatory authorities and the timing thereof, the duration and impact of regulatory delays in Lisata’s clinical programs, Lisata’s ability to finance its operations, the likelihood and timing of the receipt of future milestone and licensing fees, the future success of Lisata’s scientific studies, Lisata’s ability to successfully develop and commercialize drug candidates, the timing for starting and completing clinical trials, rapid technological change in Lisata’s markets, the ability of Lisata to protect its intellectual property rights; and legislative, regulatory, political and economic developments. The foregoing review of important factors that could cause actual events to differ from expectations should not be construed as exhaustive and should be read in conjunction with statements that are included herein and elsewhere, including the risk factors included in Lisata’s Annual Report on Form 10-K filed with the SEC on February 29, 2024, and in other documents filed by Lisata with the Securities and Exchange Commission. Except as required by applicable law, Lisata undertakes no obligation to revise or update any forward-looking statement, or to make any other forward-looking statements, whether because of new information, future events or otherwise. Lisata Therapeutics Contact:Investors:Lisata Therapeutics, Inc.John MendittoVice President, Investor Relations and Corporate CommunicationsPhone: 908-842-0084Email: jmenditto@lisata.com Media:ICR HealthcareElizabeth ColemanSenior AssociatePhone: 203-682-4783Email: elizabeth.coleman@westwicke.com University of Cincinnati Contact:Megan BurgasserPublic Information OfficerPhone: 513-545-5616Email: megan.burgasser@uc.edu

Plus, news about Interius BioTherapeutics, Hope Medicine, PMV Pharmaceuticals, Epitopea, Alyssum Therapeutics and a new dementia trial accelerator in the UK: Bayer exercises option to develop heart drug with Dewpoint Therapeutics : The companies, which first teamed up in 2019, now plan to develop a therapy for dilated cardiomyopathy, a heart muscle disease. Bayer is paying $424 million in upfront and milestone payments. — Jaimy Lee Viking Therapeutics is already thinking about an obesity launch: The company plans to meet with the FDA before the end of the year to discuss Phase 3 plans for its subcutaneous GLP-1/GIP receptor agonist, VK2735, according to remarks made by CEO Brian Lian on Wednesday’s earnings call. Viking is already in conversation with manufacturers, and Lian said he’s confident the company will be able to supply “a blockbuster-sized product at the appropriate time.” Viking is also launching a Phase 2 trial this quarter for an oral version of VK2735. Lian described the experimental oral medicine as more of an “add-on” to the portfolio, while the subcutaneous version would be the “anchor piece.” — Nicole DeFeudis Interius BioTherapeutics doses first ever patient with in vivo CAR-T: The biotech gave an in vivo CAR-T therapy to a patient enrolled in a Phase 1 study of INT2104, which it’s developing for B cell cancers. The trial, which is being run in Australia, is the first test of its kind. Umoja Biopharma has said it plans to dose a patient in its US-based study of its own in vivo CAR-T therapy by the end of this year . — Lei Lei Wu Hope Medicine touts interim data for endometriosis treatment: The company’s monoclonal antibody, dubbed HMI-115 , demonstrated statistically significant improvement in endometriosis pain at 12 weeks. The Phase 2 trial enrolled 142 female patients in the US, Poland and China. The mean dysmenorrhea pain score was reduced by 42%, and non-menstrual pelvic pain score was reduced by 50%. Hope CEO Nathan Chen said the company plans to “move quickly” toward global Phase 3 trials. — Katherine Lewin PMV Pharmaceuticals discontinues early-stage cancer trial: Citing toxicities and limited clinical benefit, the company abandoned its Phase 1b study assessing rezatapopt and Merck’s Keytruda. It had been testing the combo in patients with advanced solid tumors harboring a TP53 Y220C mutation. PMV said enrollment for a Phase 2 trial evaluating rezatapopt as a monotherapy for patients with TP53 Y220C and KRAS wild-type advanced solid tumors is still on track. — Katherine Lewin Epitopea closes $31M pre-Series A: With today’s round, the cancer biotech has raised more than $45 million. The funds are set to go toward its preclinical discovery work in solid tumors and clinical development plans for its RNA-based immunotherapies. — Katherine Lewin Alyssum Therapeutics emerges from stealth with $26M Series A: The Cambridge, MA-based biotech’s lead asset is AT-1965, a clinical-stage small molecule CMTR2 inhibitor. This round was led by co-founder Patrick Soon-Shiong and Accel, Famy and Navam Capital. — Katherine Lewin UK government pours £20M into dementia trial accelerator: The new initiative aims to increase the number of participants in late-stage dementia studies to the tens of thousands and “address pharma-identified roadblocks” to running the trials. The Dementia Trials Accelerator’s objectives include creating a biomarker toolbox and extending trial enrollment to community settings. — Ayisha Sharma