GE HealthCare unveils next-generation SIGNA MRI technology, aiming to boost efficiency, enhance patient experience, and advance sustainability




GE HealthCare unveils next-generation SIGNA MRI technology, aiming to boost efficiency, enhance patient experience, and advance sustainability

• SIGNA Bolt¹, a new 3T MRI scanner, is designed with a next-gen gradient system engineered to support precision diagnostics, clinical efficiency, and advanced research capability.
• SIGNA Sprint with Freelium,² a new sealed magnet MRI system, seeks to reimagine 1.5T MRI by combining a sustainable design with remarkable image quality, and operational autonomy to expand access to MR.
• Both new systems, 510(k) pending at the U.S. FDA, are powered by SIGNA One³, an ecosystem of AI-driven workflow solutions designed to reduce inefficiencies and support MRI exams from plan to scan and beyond.

CHICAGO–(BUSINESS WIRE)–GE HealthCare (Nasdaq: GEHC) today announced the 510(k) submissions to the U.S. Food and Drug Administration (FDA) seeking clearance for next-generation SIGNA™ MRI technology. Unveiled at the Radiological Society of North America’s 2025 Annual Meeting, these differentiated solutions are part of a wave of new GE HealthCare innovations aimed at tackling some of the most complex challenges in healthcare. Designed to enhance precision diagnosis for clinicians and help clinicians improve patient outcomes, the new technologies include GE HealthCare’s⁴ 1.5T MRI system, both equipped with an AI-powered workflow platform designed for end-to-end exam efficiency.

With imaging needs outpacing the radiologist workforce⁵, leading to longer wait times, burnout and delays in diagnosis, many departments are struggling to keep pace. At the same time, aging equipment and flat reimbursement rates are pushing providers to seek smarter, more sustainable solutions.

“The urgency for greater access, efficiency and precision in MRI has never been greater. We’ve listened to clinicians who are seeking smarter, more sustainable technologies to meet today’s challenges and tomorrow’s needs,” said Kelly Londy, President & CEO, MR, GE HealthCare. “With the introduction of our advanced SIGNA lineup, we’re delivering on a bold vision for MRI — one that intentionally puts smarter technology to work for clinicians and patients alike to make MRI more intuitive, more efficient, and more impactful in everyday care.”

SIGNA™ Bolt: Aiming to translate innovative discoveries and complex exams into everyday patient care

SIGNA Bolt⁶ aims to bring to market our most advanced high-field, clinical wide bore 3.0T MRI system and is seeking to combine ultra-high gradient performance, intelligent digital radio frequency (RF) architecture, and sustainable design to deliver precision imaging, fast workflows, and seamless clinical-to-research flexibility, all with exceptionally low energy consumption and operational costs.

SIGNA Bolt is designed to include:

• The power needed to deliver diagnostic quality in everyday scans with outstanding gradient and RF performance for both research and clinical applications
• Next-gen AI-enabled workflow of SIGNA One in combination with deep learning applications that are designed to enable accelerated examination times
• Deep-learning based advanced applications that aim to enhance diagnostic capabilities
• End-to-end clinical pathway solutions from planning to reporting for neurology and oncology
• Advanced developer toolkits to simplify research

SIGNA™ Sprint with Freelium™: Designed to be purposefully helium-free without compromising on clinical or operational efficiency

SIGNA Sprint with Freelium⁷ aims to broaden access to sustainable and equitable MRI technology. With less than 1% helium usage⁸, Freelium is designed to provide effortless sustainability without compromising clinical and operational efficiency.

Engineered for excellence, the SIGNA Sprint with Freelium 1.5T system is designed to support remarkable image quality through exceptional homogeneity, high signal-to-noise ratio (SNR) and image clarity. Integrated with deep learning solutions, including AIR™ Recon DL and Sonic DL with wide coverage of anatomies and clinical applications, the system aims to provide consistent clarity and diagnostic confidence.

Designed with flexibility in mind, its Freelium ventless magnet is designed to allow installation virtually anywhere – from inside hospitals to remote regions, expanding access to advanced MR capabilities for more patients and communities. SIGNA Sprint with Freelium features two levels of operational autonomy to empower radiology departments: Scanning Autonomy with SIGNA One Interface, an intuitive user interface aiming to simplify scanning for all levels of technologist experience, and Autoramp with intelligent sensors designed to support automated magnet recovery and supporting system uptime without the need for a field engineer.

SIGNA ™ Sprint with Freelium is designed to include:

• A design that uses less than 1% helium versus conventional designs⁹ with no additional power or cooling required
• MR magnet installation virtually anywhere – from inside hospitals to remote regions that need more access to advanced MR capabilities, due to its ventless design
• Exceptional homogeneity¹⁰ designed for impressive image quality and diagnostic capability

SIGNA One¹¹: Aiming to reduce inefficiencies with a new, AI-powered MRI workflow platform

A next-gen, AI-powered workflow platform that is designed to improve the imaging experience by combining precision with simplicity, SIGNA One offers five innovations at its core designed to streamline operations and elevate clinical confidence.

“We’re dedicated to making every interaction smooth, fast and patient centric by using predictive technology that boosts efficiency,” said Bryan Mock, PhD, General Manager, Global Product Segment, Premium MR, GE HealthCare. “SIGNA One technology is designed to simplify and improve the efficiency of experienced users, while aiming to shorten the learning curve for new users. Regardless of a users’ experience level, SIGNA One aims to liberate users from inefficiencies at every stage of the MR imaging process.”

SIGNA ™ One is designed to include¹²:

• An intuitive user experience that may help reduce training time and aims to boost productivity while aiming to shorten the learning curve for new and less experienced users
• The SIGNA One Table, designed to enhance patient comfort and simplify patient transport
• Fully automated SIGNA One Camera with In-Room Console live feed, AI-enabled landmark localization and patient positioning verification, with real-time visual guidance on a touchscreen display — aiming to improve speed and accuracy of patient setup
• Contactless respiratory and peripheral gating designed to capture physiological data effortlessly, regardless of patient orientation and without the need for external devices
• High-resolution, In-Room Consoles empowering technologists with simple patient setup control, aiming to minimize interruptions and maximizing throughput

GE HealthCare is integrating with NVIDIA technology to enhance the performance and intelligence of its MR products by utilizing NVIDIA GPUs to accelerate the development of deep learning reconstruction models. Accelerated by NVIDIA’s RTX product line and programmed with the new SIGNA One MRI Workflow solutions and advanced deep learning tools that are designed to elevate the user and patient experience, SIGNA Bolt and SIGNA Sprint with Freelium are designed to provide powerful imaging techniques for tailored patient care.

Learn more about these and other GE HealthCare MRI technologies at GE HealthCare’s booth #7334 at RSNA’s annual meeting in Chicago through Dec. 4.

About GE HealthCare Technologies Inc.

GE HealthCare is a trusted partner and leading global healthcare solutions provider, innovating medical technology, pharmaceutical diagnostics, and integrated, cloud-first AI-enabled solutions, services and data analytics. We aim to make hospitals and health systems more efficient, clinicians more effective, therapies more precise, and patients healthier and happier. Serving patients and providers for more than 125 years, GE HealthCare is advancing personalized, connected and compassionate care, while simplifying the patient’s journey across care pathways. Together, our Imaging, Advanced Visualization Solutions, Patient Care Solutions and Pharmaceutical Diagnostics businesses help improve patient care from screening and diagnosis to therapy and monitoring. We are a $19.7 billion business with approximately 53,000 colleagues working to create a world where healthcare has no limits.

GE HealthCare is proud to be among 2025 Fortune World’s Most Admired Companies™.

Follow us on LinkedIn, X, Facebook, Instagram, and Insights for the latest news, or visit our website https://www.gehealthcare.com for more information.

¹ SIGNA Bolt is 510(k) pending at U.S. FDA. Not CE marked. Not available for sale.

² SIGNA Sprint with Freelium is a configuration of SIGNA Sprint Select. SIGNA Sprint Select is 510(k) pending at U.S. FDA. Not CE marked. Not available for sale.

³ SIGNA One is 510(k) pending with the U.S. FDA. Not CE marked. Not available for sale.

⁴ Helium-free: Helium is permanently enclosed in the magnet.

⁵ Harvey L. Neiman Health Policy Institute, 2025.

⁶ SIGNA Bolt is 510(k) pending at U.S. FDA. Not CE marked, not available for sale.

⁷ SIGNA Sprint with Freelium is a sealed configuration of SIGNA Sprint Select, 510(k) pending at the U.S. FDA. Not CE marked, not available for sale.

⁸ Compared to conventional magnets.

⁹ Helium-free: Helium is permanently enclosed in the magnet

¹⁰ as conventional IPM magnet.

¹¹ SIGNA One represents features of SIGNA Bolt and SIGNA Sprint Select which are 510(k) pending at U.S. FDA. Not yet CE marked. Not available for sale.

¹² All features may not be included in all system configurations.

Contacts

GE HealthCare Media Contact:
Katie Scrivano

M +1 262-215-5281

Katherine.Scrivano@gehealthcare.com

Fifty Countries Strong: AHF ‘Keeping the Promise’ on World AIDS Day




Fifty Countries Strong: AHF ‘Keeping the Promise’ on World AIDS Day

M.O.U. cements Bangladesh as AIDS Healthcare Foundation’s newest country

Global nonprofit cares for over 2.7 million people worldwide; adds Tennessee and Michigan to U.S. roster

LOS ANGELES–(BUSINESS WIRE)–To mark World AIDS Day 2025, AIDS Healthcare Foundation (AHF) will hold major commemorative concerts and events in early December across Africa, Asia, Europe, and the Americas to emphasize that the work to end HIV/AIDS is far from over—underscored by the 1.3 million new infections that still occur globally each year.

World AIDS Day is observed annually on Dec. 1. AHF’s observances will promote HIV prevention, testing, condom access, treatment, and care, while demonstrating solidarity with people and communities affected by HIV/AIDS.

This year’s commemorations also coincide with a historic milestone for AHF: reaching operations in 50 countries worldwide, an extraordinary achievement since the organization first launched its global programs in 2002 in South Africa and Uganda. The recent signing of a memorandum of understanding (M.O.U) between AHF and the Government of the People’s Republic of Bangladesh cements that country as AHF’s newest.

“50 countries and 2.7 million lives in care: a remarkable accomplishment, one reached thanks to the dedication of thousands of AHF staff, volunteers, board members and with clients and patients who place their trust, and care, in our hands each day,” said AHF President Michael Weinstein. “However, in true AHF fashion we are not resting on our laurels. The global public health system is profoundly broken and must be repaired. STDs are at pandemic levels and being ignored. Too many patients are not retained in care. The responsibility of bringing as many people as possible into the lifeboat of care remains staggering but is a challenge AHF will continue to take on.”

In the United States, AHF also celebrates the addition of two states to its roster: Tennessee, where it recently opened a new AHF Healthcare Center in Memphis; and Michigan, where it just started operations and began treatment in Detroit. This brings AHF’s U.S. roster to include 19 states, Washington, D.C., as well as Puerto Rico. Over the past several years, the range of AHF services has also expanded to include food, shelter, disaster relief and wellness services.

Despite decades of progress in the fight against HIV, around 40 million people worldwide are living with the virus, with women and girls representing more than half of those affected. Stigma and discrimination continue to prevent many from accessing essential care, while key populations face significant barriers to treatment. These challenges are compounded by chronic underfunding of the global HIV response, highlighting the urgent need for sustained HIV/AIDS financing—including full funding of the Global Fund to Fight AIDS, Tuberculosis, and Malaria. AHF’s global World AIDS Day events will highlight these persistent gaps and reinforce the importance of keeping HIV/AIDS at the forefront of national and global public health priorities.

“This World AIDS Day reminds us that the fight against HIV is far from over. Millions of people still face barriers to testing, treatment, and ongoing care, while stigma and high drug prices put lifesaving tools out of reach,” said Terri Ford, AHF Chief of Global Advocacy & Policy. “Expanding access to prevention, including condoms and new innovations, supporting retention in care, and ensuring affordable treatment are critical to stopping new infections and saving lives. Governments and communities must work together to ensure progress is equitable for everyone living with or affected by HIV.”

Additionally, pharma greed continues to block progress for the global HIV/AIDS response. True progress demands that pharmaceutical companies put people before profits because innovation doesn’t matter if it isn’t accessible to all.

World AIDS Day serves as a vital platform for HIV/AIDS advocates to acknowledge the progress made, honor those we have lost to AIDS-related illnesses and those who carry on the fight, and call on governments worldwide to commit the necessary resources and political will to end HIV/AIDS. On this World AIDS Day, we’re reminded: It’s Not Over.

About AIDS Healthcare Foundation (AHF)

AIDS Healthcare Foundation (AHF) is a global non-profit organization providing cutting-edge medicine and advocacy to over 2.7 million people in 50 countries worldwide in Africa, the Americas, the Asia/Pacific Region and Europe. We are currently the largest non-profit provider of HIV/AIDS medical care in the world. To learn more about AHF, please visit our website: www.aidshealth.org, find us on Facebook: www.facebook.com/aidshealth and follow us on Twitter: @aidshealthcare and Instagram: @aidshealthcare

Contacts

US MEDIA CONTACT:

Ged Kenslea

Senior Director, Communications, AHF

+1.323.308.1833 work

+1.323.791.5526 mobile

gedk@aidshealth.org

Denys Nazarov
Director of Global Policy and Communications, AHF

+1 323.308.1829

denys.nazarov@aidshealth.org

CAR T-Cell Therapy for the Multiple Myeloma Market, 2025-2035: Extensive Data on Products, End Users, Regions and Companies – ResearchAndMarkets.com




CAR T-Cell Therapy for the Multiple Myeloma Market, 2025-2035: Extensive Data on Products, End Users, Regions and Companies – ResearchAndMarkets.com

DUBLIN–(BUSINESS WIRE)–The “CAR T-Cell Therapy for Multiple Myeloma Market – A Global and Regional Analysis: Focus on Product, End User, and Region – Analysis and Forecast, 2025-2035” has been added to ResearchAndMarkets.com’s offering.

The chimeric antigen receptor (CAR) T-cell therapy has emerged as a groundbreaking treatment modality, offering new hope for patients with relapsed or refractory multiple myeloma (RRMM). Building upon traditional treatments such as autologous stem cell transplantation and monoclonal antibody therapies, CAR T-cells are engineered to recognize and destroy myeloma cells by targeting specific surface antigens. The recent approval of two CAR T-cell therapies – Abecma and Carvykti – by the FDA has demonstrated promising clinical outcomes, including high response rates and improved survival in heavily pretreated patients.

Ongoing clinical trials continue to investigate novel CAR constructs, combination strategies, and earlier-line use to further enhance efficacy and overcome existing limitations such as high costs, manufacturing complexity, and side effect profiles. These developments signal a transformative shift in Multiple myeloma treatment and pave the way for more personalized and effective therapeutic approaches.

The CAR T-Cell Therapy for Multiple Myeloma market is primarily driven by the high efficacy of BCMA-targeted therapies such as Abecma and Carvykti, which have demonstrated deep, durable responses in relapsed or refractory cases, significantly outperforming traditional treatment options. Additionally, the rising global incidence of multiple myeloma, particularly among aging populations, is increasing the demand for advanced, personalized treatment approaches.

Regulatory agencies like the FDA and EMA are further propelling market growth by granting fast-track approvals and priority designations to CAR T therapies, recognizing their transformative potential. Moreover, substantial investments from pharmaceutical giants and biotech firms in manufacturing capabilities, research and development, and clinical infrastructure are accelerating the commercialization and accessibility of CAR T-cell therapies worldwide.

Despite its therapeutic promise, the CAR T-Cell Therapy for Multiple Myeloma market faces several significant challenges. One of the most pressing issues is the high cost of treatment, often exceeding USD 400,000 per patient, which poses substantial reimbursement hurdles and limits affordability – particularly in low- and middle-income countries. The complex and individualized manufacturing process further adds to the burden, creating supply chain bottlenecks and scalability issues that hinder widespread adoption.

Additionally, CAR T-cell therapies are associated with severe side effects such as cytokine release syndrome (CRS) and neurotoxicity, necessitating intensive care and restricting use to highly specialized treatment centres. In many developing regions, limited healthcare infrastructure, a shortage of trained personnel, and low awareness further constrain market expansion and equitable access.

The competitive landscape of the global CAR T-Cell Therapy for Multiple Myeloma market is rapidly advancing, fuelled by increasing demand for precision oncology treatments and breakthroughs in cell and gene therapy. The market, once limited to conventional therapies such as immunomodulatory drugs and proteasome inhibitors, is now witnessing a surge in innovation with the approval and commercialization of BCMA-targeted CAR T-cell products like Abecma and Carvykti. Leading biopharmaceutical companies, including Bristol-Myers Squibb, Johnson & Johnson (Legend Biotech), Novartis, and Gilead Sciences, are actively expanding their pipelines and scaling manufacturing capabilities to meet rising global demand.

Additionally, emerging biotech firms and academic spin-offs are entering the space with next-generation and allogeneic CAR T constructs, further intensifying competition. The growing number of strategic partnerships, licensing agreements, and M&A activities reflects a dynamic market environment focused on improving access, reducing production timelines, and enhancing therapeutic durability. As regulatory bodies worldwide offer accelerated pathways and supportive frameworks, the market is poised for robust expansion, especially as CAR T-cell therapies move into earlier lines of treatment and explore novel antigen targets beyond BCMA.

The CAR T-Cell Therapy for Multiple Myeloma market holds substantial growth potential, particularly through expansion in emerging markets such as India, China, and Latin America. Improvements in clinical infrastructure and local regulatory approvals – like NexCAR19 in India – are paving the way for greater access in cost-sensitive regions. Additionally, there is increasing momentum in diversifying the therapeutic pipeline beyond BCMA, with new targets like GPRC5D and FcRH5 offering treatment options for patients who relapse after BCMA-based therapies.

Advancements in manufacturing technologies, including automation, closed-loop systems, and point-of-care platforms, promise to enhance scalability and reduce turnaround times. Furthermore, integrating CAR T-cell therapy with combination treatments – such as checkpoint inhibitors or monoclonal antibodies – presents a promising strategy to extend response durability and reduce the risk of relapse, thereby broadening the clinical utility of these therapies.

Market Segmentation

Segmentation 1: by Product

• Abecma
• Carvykti
• Anito cel
• Elrexfio
• Others

Segmentation 2: by End Users

• Hospitals
• Cancer Treatment Centres & Specialty Clinics
• Long-Term Care facilities

Segmentation 3: by Region

• North America
• Europe
• Asia-Pacific

Companies Profiled

• Bristol-Myers Squibb Company
• Novartis AG
• Johnson & Johnson (Legend Biotech)
• Gilead Sciences
• Others

For more information about this report visit https://www.researchandmarkets.com/r/2y4leu

About ResearchAndMarkets.com

ResearchAndMarkets.com is the world’s leading source for international market research reports and market data. We provide you with the latest data on international and regional markets, key industries, the top companies, new products and the latest trends.

Contacts

ResearchAndMarkets.com

Laura Wood, Senior Press Manager

press@researchandmarkets.com

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Merck to Participate in the 8th Annual Evercore ISI HealthCONx Conference




Merck to Participate in the 8th Annual Evercore ISI HealthCONx Conference

RAHWAY, N.J.–(BUSINESS WIRE)–Merck (NYSE: MRK), known as MSD outside of the United States and Canada, announced today that Chirfi Guindo, chief marketing officer, Human Health, and Dr. Eliav Barr, senior vice president, head of global clinical development and chief medical officer, Merck Research Laboratories, are scheduled to participate in a fireside chat at the 8th Annual Evercore ISI HealthCONx Conference on Tuesday, Dec. 2, 2025, at 9:10 a.m. ET.

Investors, analysts, members of the media and the general public are invited to listen to a live audio webcast of the presentation at this weblink.

About Merck

At Merck, known as MSD outside of the United States and Canada, we are unified around our purpose: We use the power of leading-edge science to save and improve lives around the world. For more than 130 years, we have brought hope to humanity through the development of important medicines and vaccines. We aspire to be the premier research-intensive biopharmaceutical company in the world – and today, we are at the forefront of research to deliver innovative health solutions that advance the prevention and treatment of diseases in people and animals. We foster a diverse and inclusive global workforce and operate responsibly every day to enable a safe, sustainable and healthy future for all people and communities. For more information, visit www.merck.com and connect with us on X (formerly Twitter), Facebook, Instagram, YouTube and LinkedIn.

Forward-Looking statement of Merck & Co., Inc., Rahway, N.J., USA

This news release of Merck & Co., Inc., Rahway, N.J., USA (the “company”) includes “forward-looking statements” within the meaning of the safe harbor provisions of the U.S. Private Securities Litigation Reform Act of 1995. These statements are based upon the current beliefs and expectations of the company’s management and are subject to significant risks and uncertainties. If underlying assumptions prove inaccurate or risks or uncertainties materialize, actual results may differ materially from those set forth in the forward-looking statements.

Risks and uncertainties include but are not limited to, general industry conditions and competition; general economic factors, including interest rate and currency exchange rate fluctuations; the impact of pharmaceutical industry regulation and health care legislation in the United States and internationally; global trends toward health care cost containment; technological advances, new products and patents attained by competitors; challenges inherent in new product development, including obtaining regulatory approval; the company’s ability to accurately predict future market conditions; manufacturing difficulties or delays; financial instability of international economies and sovereign risk; dependence on the effectiveness of the company’s patents and other protections for innovative products; and the exposure to litigation, including patent litigation, and/or regulatory actions.

The company undertakes no obligation to publicly update any forward-looking statement, whether as a result of new information, future events or otherwise. Additional factors that could cause results to differ materially from those described in the forward-looking statements can be found in the company’s Annual Report on Form 10-K for the year ended December 31, 2024 and the company’s other filings with the Securities and Exchange Commission (SEC) available at the SEC’s Internet site (www.sec.gov).

Contacts

Media Contacts:

John Cummins

john.cummins2@merck.com

Michael Levey

michael.levey@merck.com

Investor Contacts:

Peter Dannenbaum

(732) 594-1579

Steven Graziano

(732) 594-1583

U.S. FDA Grants Priority Review to Sonrotoclax for the Treatment of Relapsed or Refractory Mantle Cell Lymphoma




U.S. FDA Grants Priority Review to Sonrotoclax for the Treatment of Relapsed or Refractory Mantle Cell Lymphoma

If approved, sonrotoclax will become the first BCL2 inhibitor for R/R MCL in the U.S., addressing a high unmet need in an aggressive cancer

Sonrotoclax previously received Breakthrough Therapy Designation based on clinically meaningful, rapid responses in R/R MCL

BeOne Medicines will present the data supporting the NDA and Priority Review for the first time at ASH 2025

SAN CARLOS, Calif.–(BUSINESS WIRE)–$ONC #BeOne—BeOne Medicines Ltd. (Nasdaq: ONC; HKEX: 06160; SSE: 688235), a global oncology company, today announced that the U.S. Food and Drug Administration (FDA) has accepted and granted Priority Review to a New Drug Application (NDA) for sonrotoclax, a next-generation BCL2 inhibitor, for the treatment of adult patients with relapsed or refractory (R/R) mantle cell lymphoma (MCL), following treatment with a Bruton’s tyrosine kinase (BTK) inhibitor.

“Sonrotoclax is advancing with remarkable speed, from Breakthrough Therapy Designation to Priority Review, all within a short window,” said Lai Wang, Ph.D., Global Head of R&D at BeOne. “That pace reflects both the strength of the data and the urgency of the need for patients with R/R MCL. With rapid, deep, and durable responses and a manageable safety profile, sonrotoclax is emerging as a potential best-in-class BCL2 inhibitor, alongside our two other transformative hematology assets – BTK inhibitor BRUKINSA, and investigational BTK degrader BGB-16673.”

The NDA is supported by data from the global, multicenter, single-arm, open-label, Phase 1/2 study, BGB-11417-201 (NCT05471843), which enrolled 125 adult patients with R/R MCL who received prior treatment with a BTK inhibitor. Sonrotoclax achieved its primary endpoint of overall response rate (ORR) as assessed by an independent review committee (IRC), demonstrating clinically meaningful responses in this heavily pretreated population. The study also showed promising results across several secondary efficacy endpoints, including complete response (CR) rate, duration of response (DOR), and progression-free survival (PFS). The treatment was well-tolerated, and the risks were manageable.

BeOne will present the full results for the first time at the 67^th American Society of Hematology (ASH) Annual Meeting and Exposition, December 6-9, in Orlando, Florida. (Oral Presentation: 663; December 7 from 5:00-5:15 PM EST).

Supporting efforts to rapidly advance global review and potential access, BeOne will participate in the FDA’s Project Orbis for sonrotoclax, an initiative that provides a framework for concurrent submission and review of oncology products among international partners. BeOne also intends to submit the Phase 1/2 data to other global regulatory bodies for the potential approval of sonrotoclax in R/R MCL, including the European Medicines Agency.

New drug applications for sonrotoclax for the treatment of R/R MCL and R/R chronic lymphocytic leukemia / small lymphocytic lymphoma (CLL/SLL) have also been accepted and are under review by the Center for Drug Evaluation (CDE) of the China National Medical Products Administration (NMPA) for potential accelerated approval.

About Mantle Cell Lymphoma

Mantle cell lymphoma (MCL) is a rare subtype of aggressive non-Hodgkin lymphoma (NHL)¹ that originates in B-cells located in the mantle zone of the lymph nodes. MCL accounts for approximately 5% of all NHL cases globally², affecting an estimated 28,000 people³. MCL is often diagnosed at advanced stages⁴ and nearly all MCL patients will eventually develop refractory or relapsed (R/R) disease.⁵ The five-year survival rate for MCL is approximately 50%, reflecting the urgent need for new therapeutic options.⁶

About Sonrotoclax (BGB-11417)

Sonrotoclax is a next-generation and potentially best-in-class investigational B-cell lymphoma 2 (BCL2) inhibitor with a unique pharmacokinetic and pharmacodynamic profile. Laboratory studies during early drug development have shown that sonrotoclax is a highly potent and specific BCL2 inhibitor with a short half-life and no drug accumulation. Sonrotoclax has shown promising clinical activity across a range of B-cell malignancies and is in development as a monotherapy and in combination with other therapeutics, including BRUKINSA. Notably, in early clinical trials, sonrotoclax plus BRUKINSA has demonstrated rapid and unprecedented rates of undetectable minimal residual disease (uMRD) in treatment-naïve patients with CLL. To date, more than 2,200 patients have been enrolled across the broad sonrotoclax global development program.

The U.S. Food and Drug Administration (FDA) granted Breakthrough Therapy Designation (BTD) for sonrotoclax for the treatment of adult patients with relapsed or refractory (R/R) mantle cell lymphoma (MCL). In addition, the FDA has granted sonrotoclax Fast Track Designation for MCL and Waldenström macroglobulinemia, as well as Orphan Drug Designation for the treatment of adult patients with MCL, WM, multiple myeloma, acute myeloid leukemia, and myelodysplastic syndrome.

About BeOne

BeOne Medicines is a global oncology company domiciled in Switzerland that is discovering and developing innovative treatments that are more accessible to cancer patients worldwide. With a portfolio spanning hematology and solid tumors, BeOne is expediting development of its diverse pipeline of novel therapeutics through its internal capabilities and collaborations. With a growing global team of nearly 12,000 colleagues spanning six continents, the Company is committed to radically improving access to medicines for far more patients who need them.

To learn more about BeOne, please visit www.beonemedicines.com and follow us on LinkedIn, X, Facebook and Instagram.

Forward-Looking Statement

This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995 and other federal securities laws, including statements regarding the potential benefits of sonrotoclax; BeOne’s expectations regarding sonrotoclax’s clinical development, regulatory milestones, submissions and approvals; BeOne’s plans to present the full data at an upcoming medical meeting; and BeOne’s plans, commitments, aspirations and goals under the caption “About BeOne.” Actual results may differ materially from those indicated in the forward-looking statements as a result of various important factors, including BeOne’s ability to demonstrate the efficacy and safety of its drug candidates; the clinical results for its drug candidates, which may not support further development or marketing approval; actions of regulatory agencies, which may affect the initiation, timing and progress of clinical trials and marketing approval; BeOne’s ability to achieve commercial success for its marketed medicines and drug candidates, if approved; BeOne’s ability to obtain and maintain protection of intellectual property for its medicines and technology; BeOne’s reliance on third parties to conduct drug development, manufacturing, commercialization, and other services; BeOne’s limited experience in obtaining regulatory approvals and commercializing pharmaceutical products; BeOne’s ability to obtain additional funding for operations and to complete the development of its drug candidates and achieve and maintain profitability; and those risks more fully discussed in the section entitled “Risk Factors” in BeOne’s most recent quarterly report on Form 10-Q, as well as discussions of potential risks, uncertainties, and other important factors in BeOne’s subsequent filings with the U.S. Securities and Exchange Commission. All information in this press release is as of the date of this press release, and BeOne undertakes no duty to update such information unless required by law.

To access BeOne media resources, please visit our Newsroom.

Contacts

Investor Contact
Liza Heapes

+1 857-302-5663

ir@beonemed.com

Media Contact
Kyle Blankenship

+ 667-351-5176

media@beonemed.com

Corstasis Therapeutics and Cardiovascular Logistics Partner to Redefine Outpatient Heart Failure Care with ENBUMYST™ (Bumetanide Nasal Spray)




Corstasis Therapeutics and Cardiovascular Logistics Partner to Redefine Outpatient Heart Failure Care with ENBUMYST™ (Bumetanide Nasal Spray)

HENDERSON, Nev.–(BUSINESS WIRE)–#CHF–Corstasis Therapeutics Inc., a commercial-stage biopharmaceutical company advancing innovative outpatient therapies for cardiovascular and renal disease, today announced that it is working with Cardiovascular Logistics (CVL) to support the clinical adoption and integration of ENBUMYST™ (bumetanide nasal spray) into modern heart failure management pathways, consistent with FDA-approved use in outpatient heart failure care.

The initiative is designed to improve access to non-oral diuretic therapy across CVL’s growing network of leading cardiovascular practices, with the shared goal of improving patient access to outpatient diuretic therapy and supporting value-based cardiovascular care.

“ENBUMYST gives clinicians a new self-administered option for decongestion outside the hospital,” said Ben Esque, CEO of Corstasis Therapeutics. “Partnering with CVL allows us to work directly with physicians who are shaping the future of cardiovascular care—those focused on earlier intervention, reduced care escalation, and better outcomes for patients.”

“CVL is committed to enabling physicians to deliver more proactive, efficient, and patient-centered cardiovascular care,” said Craig Walker, MD, Chief Medical Officer of CVL. “CVL’s mission is to expand access to therapies that support proactive, outpatient management of cardiovascular disease. We support innovations like ENBUMYST that align with FDA-approved care models designed to improve patient access and comfort.” CVL and its affiliated practices do not endorse or promote any specific product. Participation in this communication reflects a shared commitment to advancing outpatient cardiovascular care consistent with FDA-approved therapies.

The U.S. Food and Drug Administration (FDA) recently approved ENBUMYST for the treatment of edema associated with congestive heart failure, hepatic disease, and renal disease, including nephrotic syndrome in adults. The Corstasis–CVL collaboration is among the first national initiatives to operationalize the use of a nasal-delivered loop diuretic across outpatient cardiology networks.

About ENBUMYST™

ENBUMYST (bumetanide nasal spray) is a loop diuretic indicated for the treatment of edema associated with congestive heart failure, and hepatic and renal disease, including nephrotic syndrome in adults.

INDICATION AND IMPORTANT SAFETY INFORMATION FOR ENBUMYST™ (BUMETANIDE NASAL SPRAY).

INDICATION

ENBUMYST is indicated for the treatment of edema associated with congestive heart failure, and hepatic and renal disease, including nephrotic syndrome in adults.

IMPORTANT SAFETY INFORMATION

ENBUMYST is contraindicated in patients with anuria, who are in hepatic coma and have a history of hypersensitivity to bumetanide.

ENBUMYST is a diuretic that may cause fluid, electrolyte, and metabolic abnormalities. Excessive fluid loss can lead to dehydration, decreased blood volume, and increased risk of blood clots. Abnormalities may include changes in blood electrolytes, nitrogen, glucose, and uric acid. The chance of getting these abnormalities is higher in people who are elderly, use higher doses or who do not get enough electrolytes by mouth.

If increasing azotemia and oliguria occur during treatment of severe progressive renal disease, discontinue bumetanide.

Although unlikely at the recommended doses, the potential for ototoxicity must be considered a risk of intravenous therapy, at high doses, repeated frequently in the face of renal excretory function impairment.

Avoid use in patients with significant nasal mucosal or structural abnormalities, such as acute episodes of rhinitis or congestion due to any cause.

Advise lactating women treated with ENBUMYST to monitor their infants for excessive urine output, dehydration, and lethargy.

Most common adverse reactions are hypovolemia, headache, muscle cramps, dizziness, hypotension, nausea and encephalopathy (in patients with pre-existing liver disease).

These are not all of the possible side effects of ENBUMYST. To report suspected adverse reactions, contact Corstasis Therapeutics at 1-877-300-5339 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.

Please see the full Prescribing Information for ENBUMYST.

About Corstasis Therapeutics

Corstasis Therapeutics Inc. is a commercial-stage biopharmaceutical company focused on transforming the management of fluid overload in patients with heart failure, liver disease, and kidney disease. Its lead product, ENBUMYST™, was approved by the FDA on September 12, 2025. Visit www.corstasis.com for more information.

About Cardiovascular Logistics

Cardiovascular Logistics is a physician-led cardiovascular platform that partners with leading independent practices to expand access, optimize outcomes, and improve efficiency across the continuum of cardiovascular care. Cardiovascular Logistics provides affiliated practices with shared administrative, operational, and clinical infrastructure that enables physicians to focus on delivering exceptional patient care. Learn more at https://cvlhealth.com.

Contacts

Media Contacts
Ben Esque, CEO

Corstasis Therapeutics Inc.

Phone: 702-541-9222

Email: Info@corstasis.com

Pilar Lewis,

Cardiovascular Logistics,

404-401-9755

plewis@marketri.com
https://cvlhealth.com/

With Two in Five Employees Undergoing Fertility Treatment Leaving Their Jobs or Considering Quitting, Are Companies Doing Enough?




With Two in Five Employees Undergoing Fertility Treatment Leaving Their Jobs or Considering Quitting, Are Companies Doing Enough?

SAINT-PREX, Switzerland–(BUSINESS WIRE)–An international survey, spanning Australia, France, Japan, Poland and the UK, has found that many employees experiencing fertility challenges lack support in the workplace, with almost two in five (39%) leaving or considering leaving their roles while undergoing treatment.¹

‘The Impact of Fertility Challenges at Work: International Insights’ survey by Ferring Pharmaceuticals, Fertility Matters at Work and This Can Happen shows that, despite growing awareness of reproductive health, two thirds (67%) who have experienced fertility challenges say that their workplaces do not offer support for employees undergoing fertility treatment, with France the least likely to provide it (88%).¹ 60% said they were not clearly entitled to time off for fertility appointments, with time recorded as paid leave, unpaid leave or annual leave (26%), and some also reported taking sick leave due to a lack of flexibility (17%).¹

With assisted reproduction therapy, including IVF, already accounting for up to 12% of births in some countries,^2,3,4,5 employee expectations are rapidly shifting. 73% of those experiencing fertility challenges say they would be attracted to a role that provides fertility support, reinforcing the need for clearer, more consistent workplace policies.¹

A large gap also emerged regarding the impact of going through fertility treatment. While three quarters (75%) of employers feel their organisation acknowledges fertility treatment as a significant life event, only 27% of employees who have experienced fertility challenges agree.¹ As a result, more than a fifth (22%) currently felt unsupported by their organisation, rising to 38% in Poland, and more than a third (36%) have felt pressure from their employer to be at work while undergoing treatment or investigations.¹

Mental health impact was one of the most consistent themes across all countries. 94% of employees reported that fertility treatment affects their mental wellbeing, with 80% experiencing anxiety or depression during their fertility journey.¹

However, the survey did show that practice is starting to change with signs employers recognise the importance of fertility support on employee engagement and talent attraction. 58% either already include fertility and family-building challenges as part of their workforce engagement and wellbeing strategy or will be in the future.¹

“At Ferring, we believe that every individual deserves compassionate and practical support during their fertility journey. This research highlights the challenges employees face and reinforces the importance of clear policies, open conversations and workplace cultures that recognise fertility as a significant moment in people’s lives,” said Christelle Beneteau, Chief People Officer, Ferring.

“Fertility treatment is a challenging time, yet the report shows that many people are still navigating it without the support they need, from manager training and awareness, to entitled leave,” said Becky Kearns, Co-Founder of Fertility Matters at Work. “We have seen first-hand how employees who receive fertility support feel more committed to their employer. More workplace recognition and clearer fertility policies could make a huge difference for individuals and organisations, with those who feel supported less likely to leave their jobs.”

“Mental wellbeing and fertility challenges are deeply connected, yet too often overlooked in workplace strategies. This research shows that creating supporting cultures and clear support policies isn’t just compassionate – it’s essential for retaining talent and protecting mental health,” said Kathryn Courtenay-Evans, This Can Happen.

Given that infertility now affects roughly one in six people globally,⁶ the report makes it clear that workplaces must urgently transform how they support people navigating fertility treatment. This means proactively creating inclusive, open, and supportive cultures, and implementing clear fertility policies to ensure consistent guidance and access to support.

About ‘The Impact of Fertility Challenges at Work: International Insights’ survey

Jointly run by Fertility Matters at Work (FMAW), This Can Happen, and Ferring Pharmaceuticals and funded by Ferring, the survey aims to explore how fertility challenges impact people in the workplace and how employers can offer better support. The survey, of over 3,600 employees and employers, was initially conducted in the UK and has since been rolled out to Australia, Japan, Poland and France.

Report link: The Impact of Fertility Challenges at Work: International Insights

About Ferring Pharmaceuticals

Ferring Pharmaceuticals is a privately owned specialty biopharmaceutical group committed to building families and helping people live better lives. We are leaders in reproductive medicine with a strong heritage in gastroenterology and urology and are at the forefront of innovation in uro-oncology gene therapy. Ferring was founded in 1950 and employs more than 7,500 people worldwide.

About Fertility Matters at Work

Fertility Matters at Work is the leading training provider for fertility support in the workplace.

About This Can Happen

This Can Happen is your trusted partner to support mental wellbeing in your workplace.

Contacts

Matthew Worrall
Director of Corporate Communications, Ferring
+44 7442 271 811

matthew.worrall@ferring.com

Otsuka Receives FDA Accelerated Approval for VOYXACT® (sibeprenlimab-szsi) for the Reduction of Proteinuria in Adults with Primary Immunoglobulin A Nephropathy (IgAN) at Risk for Disease Progression




Otsuka Receives FDA Accelerated Approval for VOYXACT® (sibeprenlimab-szsi) for the Reduction of Proteinuria in Adults with Primary Immunoglobulin A Nephropathy (IgAN) at Risk for Disease Progression

• VOYXACT achieved a significant placebo-adjusted treatment effect of 51% (P<0.0001) reduction in proteinuria at 9 months of treatment (-50% VOYXACT vs 2% placebo) in the VISIONARY Phase 3 interim analysis.
• In the study, the most common adverse reactions (reported in ≥10% of patients treated with VOYXACT and at a higher incidence than placebo) reported in patients treated with VOYXACT and placebo, respectively, were infections (49% versus 45%) and injection site reactions (24% versus 23%).
• VOYXACT blocks A-PRoliferation-Inducing-Ligand (APRIL), resulting in reduced levels of serum galactose-deficient IgA1 (Gd-IgA1). Gd-IgA1 is implicated in the pathogenesis of IgAN.
• Proteinuria reduction is a recognized surrogate marker correlating with delaying progression to kidney failure and has been used as a surrogate endpoint in IgAN clinical trials to support accelerated regulatory approvals.
• VOYXACT is a self-administered, subcutaneous injection dosed every 4 weeks.
• Despite the current standard of care, IgAN is a progressive, immune-mediated, chronic kidney disease that typically manifests in adults aged 20-40 years and can lead to end-stage kidney disease (ESKD) over the lifetime of most patients.

PRINCETON, N.J. & TOKYO–(BUSINESS WIRE)–Otsuka Pharmaceutical Development & Commercialization, Inc. and Otsuka Pharmaceutical, Co. Ltd. (Otsuka) today announce the U.S. Food and Drug Administration (FDA) has granted accelerated approval of VOYXACT (sibeprenlimab-szsi) for the reduction of proteinuria in adults with primary immunoglobulin A nephropathy (IgAN) at risk for disease progression. VOYXACT is a self-administered, subcutaneous injection dosed every four weeks. VOYXACT was granted accelerated approval based on the VISIONARY Phase 3 interim analysis, where it achieved a significant placebo-adjusted treatment effect of 51% (P<0.0001) reduction in proteinuria at nine months (n=320) of treatment (-50% VOYXACT vs 2% placebo). VOYXACT is the first and only therapy to block A-PRoliferation-Inducing-Ligand (APRIL).

Proteinuria reduction is a recognized surrogate marker correlating with delaying progression to kidney failure and has been used as a surrogate endpoint in IgAN clinical trials to support accelerated regulatory approvals¹. This indication is approved by the FDA under the accelerated approval pathway based on reduction of proteinuria. It has not been established whether VOYXACT slows kidney function decline over the long-term in patients with IgAN. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory clinical from the ongoing Phase 3 VISIONARY study evaluating whether VOYXACT slows disease progression as measured by estimated glomerular filtration rate (eGFR) decline at 24 months. The eGFR data are expected in early 2026 and are intended to support traditional FDA approval.

“The availability of VOYXACT represents a novel targeted approach to help manage this complex disease for patients living with IgAN,” said John Kraus, M.D., Ph.D., executive vice president and chief medical officer, Otsuka. “With its targeted mechanism, strong efficacy, safety profile, and once-every-four-weeks dosing, VOYXACT offers a new option for IgAN patients. We recognize the urgent need for new treatment options for IgAN and are thankful for the patients and healthcare professionals who continue to participate in our clinical trial program.”

VOYXACT works by blocking APRIL, which plays a key role in the 4-hit process of IgAN pathogenesis and is an important initiating and sustaining factor in IgAN progression by promoting the production of pathogenic galactose-deficient IgA1 (Gd-IgA1)^2,3,4,5. Inhibition of APRIL results in reduced levels of serum galactose-deficient IgA1 (Gd-IgA1), which is implicated in the pathogenesis of IgAN.

“VOYXACT is the first approved treatment for adults with primary IgAN at risk for disease progression that blocks the activity of APRIL,” said Dr. Dana Rizk, professor of medicine in the division of nephrology at the University of Alabama at Birmingham and VOYXACT VISIONARY study investigator and co-chair of the steering committee. “I’m encouraged by its potential to help improve the outlook for IgAN patients.”

“We’re thrilled to hear the news about the accelerated approval of VOYXACT,” said Bonnie and Ed Schneider, IgA Nephropathy Foundation co-founders. “It’s important for patients who are managing IgAN to have new treatment options. On behalf of our board of directors, leadership, and the community we serve, thank you to the patients and experts involved in helping advance this option.” Otsuka is honored to support the IgAN Foundation and other organizations in raising awareness of IgA Nephropathy.

Data Supporting Approval

The Phase 3 VISIONARY study is a multicenter, randomized, double-blind, placebo-controlled trial consisting of 510 adult patients with IgAN who were receiving standard-of-care therapy (defined as maximally tolerated ACE inhibitor and/or ARB with or without SGLT2 inhibitor), designed to evaluate the efficacy and safety of sibeprenlimab 400 mg administered subcutaneously every four weeks, compared to placebo⁶.

In the trial, patients had to have a biopsy-confirmed IgAN diagnosis, urine protein-to-creatinine ratio (uPCR)≥ 0.75 g/g or urine protein ≥ 1.0g/day, eGFR≥ 30 mL/min/1.73 m², and be on a stable dose of supportive therapy (ACE inhibitor and/or ARB with or without SGLT2 inhibitor) for ≥ three months prior to screening⁶. Patients were randomized to treatment once every four weeks with VOYXACT or placebo and remained on supportive therapy throughout the study⁶.

The primary endpoint is the ratio of 24-hour uPCR at month nine compared with baseline. In the VISIONARY Phase 3 interim analysis (n=320), VOYXACT achieved a significant placebo-adjusted treatment effect of 51% (P<0.0001) reduction in proteinuria at nine months of treatment (-50% VOYXACT vs 2% placebo). Proteinuria reduction is a recognized surrogate marker correlating with delaying progression to kidney failure and has been used as a surrogate endpoint in IgAN clinical trials to support accelerated regulatory approvals¹. The study is still ongoing, and the secondary endpoint data of the annualized slope of eGFR over 24 months is expected to be available in 2026.

In the study, the most common adverse reactions (reported in ≥10% of patients treated with VOYXACT and at a higher incidence than placebo) in patients treated with VOYXACT and placebo, respectively, were infections (49% versus 45%) and injection site reactions (24% versus 23%). The most common infection was upper respiratory infection (15% versus 14%), and the most common injection site reaction was injection site erythema (13% versus 12%). Most adverse reactions were reported as mild or moderate in severity and resolved without treatment interruption or discontinuation.

About IgAN

IgAN is a progressive, immune-mediated, chronic kidney disease that typically manifests in adults aged 20-40 years and can lead to end-stage kidney disease (ESKD) over the lifetime of most patients^7,8,9. IgAN is characterized by the accumulation of Gd-IgA1 complexes in the kidneys. IgAN can lead to progressive loss of kidney function and, eventually, ESKD, imposing a significant burden on patients⁸. Despite supportive care, there is an unmet need for treatments that address the root causes of the condition⁴. Continued research in the disease remains crucial to uncovering opportunities for advancement in our understanding and treatment of patients⁴.

About VOYXACT® (sibeprenlimab-szsi)

VOYXACT (sibeprenlimab-szsi) was designed and engineered by Visterra, Inc., a wholly owned subsidiary of Otsuka. Pre-clinical and early-stage trials of sibeprenlimab were also conducted by Visterra. VOYXACT (sibeprenlimab-szsi) is a humanized monoclonal antibody that binds to and blocks APRIL, which plays a key role in the 4-hit process of IgAN pathogenesis and is an important initiating and sustaining factor in IgAN progression by promoting the production of pathogenic galactose-deficient IgA1 (Gd-IgA1)^2,5. Inhibition of APRIL results in reduced levels of serum galactose-deficient IgA1 (Gd-IgA1), which is implicated in the pathogenesis of IgAN. VOYXACT is a self-administered, subcutaneous injection dosed every four weeks.

INDICATION and IMPORTANT SAFETY INFORMATION for

VOYXACT® (sibeprenlimab-szsi)

INDICATION

VOYXACT is indicated to reduce proteinuria in adults with primary immunoglobulin A nephropathy (IgAN) at risk for disease progression.

This indication is approved under accelerated approval based on reduction of proteinuria. It has not been established whether VOYXACT slows kidney function decline over the long-term in patients with IgAN. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory clinical trial.

IMPORTANT SAFETY INFORMATION

CONTRAINDICATION

VOYXACT is contraindicated in patients with serious hypersensitivity to sibeprenlimab-szsi or any of the excipients of VOYXACT.

WARNINGS AND PRECAUTIONS

Immunosuppression and Increased Risk of Infections: VOYXACT suppresses the immune system by reducing antibody production, which may increase the risk of infections. Patients with chronic or recurring infections may have an increased risk of serious infection. In clinical trials, infections occurred in 49% of patients treated with VOYXACT compared with 45% of patients treated with placebo.

Before initiating VOYXACT, assess patients for active infections. During treatment, monitor patients for signs and symptoms of infection. If a serious infection develops, consider interrupting VOYXACT until the infection is controlled.

Immunosuppression and Immunization Risks: Because of its mechanism of action, VOYXACT may interfere with immune responses to vaccines and increase the risk of infection from live vaccines. Live vaccines are not recommended within 30 days prior to initiation of VOYXACT or during treatment with VOYXACT as safety has not been established. No data are available on the secondary transmission of infection from persons receiving live vaccines to patients receiving VOYXACT or on the efficacy of immunizations administered while receiving VOYXACT.

Common Adverse Reactions: The most common adverse reactions (reported in ≥10% of patients treated with VOYXACT and at a higher incidence than placebo) in patients treated with VOYXACT and placebo, respectively, were infections (49% versus 45%) and injection site reactions (24% versus 23%). The most common infection was upper respiratory infection (15% versus 14%), and the most common injection site reaction was injection site erythema (13% versus 12%). Most adverse reactions were reported as mild or moderate in severity and resolved without treatment interruption or discontinuation.

Pregnancy: There are no available data on VOYXACT use in pregnant women to evaluate for a drug-associated risk of major birth defects, miscarriage or other adverse maternal or fetal outcomes. Monoclonal antibodies, such as sibeprenlimab-szsi, can be actively transported across the placenta as pregnancy progresses; therefore, potential effects on a fetus are likely to be greater during the second and third trimester of pregnancy.

Lactation: There are no data on the presence of sibeprenlimab-szsi in human milk, the effects of sibeprenlimab-szsi on the breastfed infant, or the effects of sibeprenlimab-szsi on milk production.

Pediatric Use: Safety and effectiveness of VOYXACT in pediatric patients have not been established.

Geriatric Use: Clinical studies of VOYXACT did not include sufficient numbers of patients aged 65 and over to determine whether they respond differently from younger adult patients.

Pregnant women exposed to VOYXACT, or their healthcare providers, should report VOYXACT exposure by calling 1-833-869-9228 or visiting www.VOYXACT.com

To report SUSPECTED ADVERSE REACTIONS, contact Otsuka America Pharmaceutical, Inc. at 1-800-438-9927 or FDA at 1-800-FDA-1088 (www.fda.gov/medwatch).

Please see FULL PRESCRIBING INFORMATION and PATIENT INFORMATION.

About Otsuka

Otsuka Pharmaceutical Co., Ltd. is a global healthcare company with the corporate philosophy: Otsuka–people creating new products for better health worldwide. Otsuka researches, develops, manufactures, and markets innovative products, with a focus on pharmaceutical products to meet unmet medical needs and nutraceutical products for the maintenance of everyday health.

In pharmaceuticals, Otsuka is a leader in the challenging areas of mental, kidney, and cardiovascular health and has additional research programs in oncology and on several under-addressed diseases including tuberculosis, a significant global public health issue. These commitments illustrate how Otsuka is a “big venture” company at heart, applying a youthful spirit of creativity in everything it does.

Otsuka established a presence in the U.S. in 1973 and today its U.S. affiliates include Otsuka Pharmaceutical Development & Commercialization, Inc. (OPDC) and Otsuka America Pharmaceutical, Inc. (OAPI). These two companies’ 2,250 employees in the U.S. develop and commercialize medicines in the areas of mental health and nephrology, using cutting-edge technology to address unmet healthcare needs.

OPDC and OAPI are indirect subsidiaries of Otsuka Pharmaceutical Co., Ltd., which is a subsidiary of Otsuka Holdings Co., Ltd. headquartered in Tokyo, Japan. The Otsuka group of companies employed 35,340 people worldwide and had consolidated sales of approximately USD 14.7 billion in 2024.

All Otsuka stories start by taking the road less traveled. Learn more about Otsuka in the U.S. at www.otsuka-us.com and connect with us on LinkedIn and Twitter at @OtsukaUS. Otsuka Pharmaceutical Co., Ltd.’s global website is accessible at https://www.otsuka.co.jp/en/.

About Visterra

Visterra is a biologics research and early-stage clinical development biotechnology company committed to developing innovative antibody-based therapies for the treatment of patients with immune-mediated kidney diseases and other hard-to-treat diseases. Its proprietary Hierotope® platform enables the design and engineering of precision biologics-based product candidates that specifically bind to, and modulate, key disease targets that are not adequately addressed by traditional therapeutic approaches. The platform also includes Fc engineering capabilities for half-life extension, bispecific antibodies, and antibody-drug conjugates (ADCs). Visterra’s pipeline includes programs targeting kidney diseases, immunologically-driven diseases and infectious diseases. Visterra is an indirect subsidiary of Otsuka Pharmaceutical Co., Ltd. For more information, visit www.visterrainc.com.

References

1. Thompson A, Carroll K, Inker LA, et al. Proteinuria Reduction as a Surrogate End Point in Trials of IgA Nephropathy. Clin J Am Soc Nephrol. 2019;14(3):469-481.doi:10.2215/CJN.08600718
2. Mathur M, Barratt J, Suzuki Y, et al. Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of VIS649 (Sibeprenlimab), an APRIL-Neutralizing IgG2 Monoclonal Antibody, in Healthy Volunteers. Kidney Int Rep. 2022;7(5):993-1003.
3. Chang S, Li XK. The Role of Immune Modulation in Pathogenesis of IgA Nephropathy (nih.gov)
4. Cheung CK, Barratt J, Liew A, Zhang H, Tesar V, Lafayette R. The role of BAFF and April in IGA nephropathy: Pathogenic mechanisms and targeted therapies. Frontiers in nephrology. February 1, 2024.
5. Mathur M, Barratt J, Chacko B, et al. A Phase 2 Trial of Sibeprenlimab in Immunoglobulin A Nephropathy Patients. NEJM. 2023 https://www.nejm.org/doi/full/10.1056/NEJMoa2305635
6. Perkovic, et al. Evaluating Sibeprenlimab in IgA Nephropathy – Rationale and Baseline Data from the VISIONARY Trial. Kidney International Reports. https://doi.org/10.1016/j.ekir.2025.09.031
7. Pitcher, D. Braddon, et. al Long-term outcomes in IGA nephropathy. Clinical journal of the American Society of Nephrology: CJASN. https://pubmed.ncbi.nlm.nih.gov/37055195/.
8. Lai K. Iga nephropathy. Nature reviews. Disease primers. 2016
9. Cheung, Chee Kay & Boyd, JKF & Feehally, J.. (2012). Evaluation and management of IgA nephropathy. Clinical Medicine. 12. s27-s30. 10.7861/clinmedicine.12-6-s27.

Contacts

Contacts for Media

Otsuka in the U.S.
Jill Roman

Corporate Communications

Otsuka America Pharmaceutical, Inc.

jill.roman@otsuka-us.com

Otsuka in Japan
Jeffrey Gilbert

Leader, Pharmaceutical PR

Otsuka Pharmaceutical Co., Ltd.

gilbert.jeffrey.a@otsuka.jp
+81 3 6361 7379

Mirum Pharmaceuticals to Participate in Upcoming Investor Conferences




Mirum Pharmaceuticals to Participate in Upcoming Investor Conferences

FOSTER CITY, Calif.–(BUSINESS WIRE)–Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM), a biopharmaceutical company focused on the identification, acquisition, development and commercialization of novel therapies for debilitating rare and orphan diseases, today announced that it will participate in the following upcoming investor conferences:

• 8th Annual Evercore HealthCONx Conference on Tuesday, December 2^nd
  • Company presentation – Tuesday, December 2^nd starting at 2:10 p.m. ET
• Citi’s 2025 Global Healthcare Conference on Wednesday, December 3^rd

Visit the Investors and Media section of Mirum’s corporate website for webcast links and additional information.

About Mirum Pharmaceuticals, Inc.

Mirum Pharmaceuticals, Inc. is a biopharmaceutical company dedicated to transforming the treatment of rare diseases affecting children and adults. Mirum has three approved medications: LIVMARLI® (maralixibat) oral solution/LIVMARLI® (maralixibat) tablets, CHOLBAM® (cholic acid) capsules, and CTEXLI™ (chenodiol) tablets.

LIVMARLI, an IBAT inhibitor, is approved for the treatment of two rare liver diseases affecting children and adults. It is approved for the treatment of cholestatic pruritus in patients with Alagille syndrome in the U.S. (three months and older), in Europe (two months and older), and in other regions globally. It is also approved in the U.S. in cholestatic pruritus in PFIC patients 12 months of age and older; in Europe, it is approved for patients with PFIC three months of age and older. Mirum is conducting the Phase 3 EXPAND study, a label expansion opportunity for LIVMARLI in additional settings of cholestatic pruritus. CHOLBAM is FDA-approved for the treatment of bile acid synthesis disorders due to single enzyme deficiencies and adjunctive treatment of peroxisomal disorders in patients who show signs or symptoms of liver disease.

CTEXLI is FDA-approved for the treatment of cerebrotendinous xanthomatosis (CTX) in adults.

Mirum’s late-stage pipeline includes two investigational treatments for several rare diseases.

Volixibat, an IBAT inhibitor, is being evaluated in two potentially registrational studies including the Phase 2b VISTAS study for primary sclerosing cholangitis (PSC) and Phase 2b VANTAGE study for primary biliary cholangitis. Volixibat has been granted Breakthrough Therapy Designation for the treatment of cholestatic pruritus in patients with PBC. Mirum has also initiated a Phase 2 study evaluating MRM-3379, a PDE4D inhibitor for the treatment of Fragile X syndrome, a rare genetic neurocognitive disorder.

To learn more about Mirum, visit mirumpharma.com and follow Mirum on Facebook, LinkedIn, Instagram and X.

Contacts

Investor Contacts:

Andrew McKibben

ir@mirumpharma.com

Media Contact:

Meredith Kiernan

media@mirumpharma.com

Arrowhead Pharmaceuticals Reports 2025 Fiscal Year-End Results




Arrowhead Pharmaceuticals Reports 2025 Fiscal Year-End Results

– Conference Call and Webcast Today, November 25, 2025, at 4:30 p.m. ET

PASADENA, Calif.–(BUSINESS WIRE)–$arwr–Arrowhead Pharmaceuticals, Inc. (NASDAQ: ARWR) today announced financial results for its 2025 fiscal year ended September 30, 2025. The Company is hosting a conference call today, November 25, 2025, at 4:30 p.m. ET to discuss the results.

“The recent FDA approval of REDEMPLO, indicated as an adjunct to diet to reduce triglycerides in adults with familial chylomicronemia syndrome (FCS), is a transformational milestone for Arrowhead. REDEMPLO is the first and only FDA-approved siRNA medicine for people living with FCS and represents the first FDA-approval for a medicine leveraging Arrowhead’s proprietary and differentiated Targeted RNAi Molecule (TRiM™) platform,” said Christopher Anzalone, Ph.D., President and CEO at Arrowhead. “However, Arrowhead is truly just getting started and we are well positioned to execute on our aggressive goals in discovery, clinical development, business development, and commercial to enable multiple potential new launches, both independently and with partners, over the coming years.”

Webcast and Conference Call and Details

Investors may access a live audio webcast on the Events and Presentations page under the Investors section of the Arrowhead website. A replay of the webcast will be available approximately two hours after the conclusion of the call.

For analysts who wish to participate in the conference call, please register at https://register-conf.media-server.com/register/BI2700430252ab408a89dff83808477869. Once registered, you will receive the dial-in number and a personalized PIN code that will be required to access the call.

Selected Recent Events

• Announced that the U.S. Food and Drug Administration (FDA) has approved REDEMPLO (plozasiran), a small interfering RNA (siRNA) medicine, as an adjunct to diet to reduce triglycerides in adults with familial chylomicronemia syndrome (FCS).
  • FCS is a severe, rare disease, with an estimated 6,500 people in the U.S. living with genetic or clinical FCS, characterized by triglyceride levels that can be 10 to 100 times higher than normal leading to a substantially higher risk of developing acute, recurrent, and potentially fatal pancreatitis.
  • This is Arrowhead’s first FDA-approved medicine, marking a major milestone for the company as it transitions into commercial-stage.
  • REDEMPLO is the first and only FDA-approved siRNA medicine for people living with FCS and can be self-administered at home with a simple subcutaneous injection once every three months.
  • REDEMPLO is the first and only FDA-approved medicine to be studied in patients with genetically confirmed and clinically diagnosed FCS.
  • The FDA approval is based on positive results from the Phase 3 PALISADE study where REDEMPLO significantly reduced triglycerides -80% from baseline and lowered the numerical incidence of acute pancreatitis compared to placebo.
• Announced the One-REDEMPLO pricing model that creates one consistent price across current and potential future indications.
• Launched Rely On REDEMPLO, a patient support program providing support services and resources for patients at each stage of the treatment journey with REDEMPLO, including financial assistance options for eligible patients.
• Announced a global licensing and collaboration agreement with Novartis for ARO-SNCA, Arrowhead’s preclinical stage siRNA therapy against alpha-synuclein for the treatment of synucleinopathies, such as Parkinson’s Disease, and for other additional collaboration targets that will utilize Arrowhead’s proprietary Targeted RNAi Molecule (TRiM™) platform. Financial terms of the agreement include:
  • Arrowhead received a $200 million upfront payment from Novartis. Arrowhead is also eligible to receive development, regulatory, and sales milestone payments of up to $2 billion. Arrowhead is further eligible to receive tiered royalties on commercial sales up to the low double digits.
• Filed a request for regulatory clearance to initiate a Phase 1/2a clinical trial of ARO-DIMER-PA, the company’s investigational RNA interference (RNAi) therapeutic being developed as a potential treatment for atherosclerotic cardiovascular disease (ASCVD) due to mixed hyperlipidemia.
  • ARO-DIMER-PA is a dual functional RNAi therapeutic designed to silence expression of the proprotein convertase subtilisin kexin 9 (PCSK9) and apolipoprotein C3 (APOC3) genes. This represents an important step forward for the RNAi field as it is the first clinical candidate to target two genes simultaneously in one molecule.
• Filed a request for regulatory clearance to initiate a Phase 1/2a clinical trial of ARO-MAPT, the company’s investigational RNAi therapeutic being developed as a potential treatment for tauopathies including Alzheimer’s disease, a progressive neurodegenerative disease characterized by cognitive and functional decline.
  • ARO-MAPT is Arrowhead’s first investigational RNAi-based therapy to utilize a new proprietary delivery system which, in preclinical studies, has achieved blood-brain-barrier penetration and deep knockdown of target genes across the central nervous system (CNS), including deep brain regions, after subcutaneous injections.
• Earned $300 million in milestone payments from Sarepta Therapeutics.
  • $100 million milestone earned when Arrowhead reached the first of two pre-specified enrollment targets and subsequent authorization to dose escalate in a Phase 1/2 clinical study of ARO-DM1, an investigational RNA interference (RNAi) therapeutic for the treatment of type 1 myotonic dystrophy (DM1), the most common adult-onset muscular dystrophy.
  • $200 million milestone earned following a drug safety committee review and subsequent authorization to dose escalate, and achievement of the second pre-specified patient enrollment target.
• Elected to receive approximately $50 million worth of Arrowhead common stock and approximately $50 million in cash from Sarepta Therapeutics to satisfy the payment of the first $100 million milestone owed to Arrowhead. The company believed this direct stock buyback of Arrowhead shares from Sarepta at $18.79 per share was an attractive option, given Arrowhead’s significant growth potential, both near- and longer-term. These shares were subsequently placed into treasury to reduce the number of Arrowhead shares outstanding.
• Dosed the first subject in the YOSEMITE Phase 3 clinical trial of zodasiran, the company’s investigational RNAi therapeutic being developed as a potential treatment for homozygous familial hypercholesterolemia (HoFH), a rare genetic condition that leads to severely elevated LDL-cholesterol and early onset cardiovascular disease. Zodasiran is the fourth investigational RNAi-based candidate developed by Arrowhead to reach late-stage pivotal studies, after investigational drugs plozasiran, fazirsiran (licensed to Takeda) and olpasiran (licensed to Amgen).
• Announced the signing of an asset purchase agreement between Sanofi and Visirna Therapeutics, a majority-owned subsidiary of Arrowhead created to develop and commercialize four of Arrowhead’s investigational cardiometabolic candidates in Greater China. Summary terms of the agreement include:
  • Visirna will receive an upfront payment of $130 million from Sanofi. In addition, Visirna will be eligible to receive further milestone payments of up to $265 million upon approval of plozasiran across various indications in mainland China.
  • Sanofi will receive an exclusive license to develop and commercialize investigational plozasiran in Greater China from Visirna Therapeutics, offering potential treatment to people living with elevated triglycerides.

Selected Fiscal 2025 Year-End Financial Results

About REDEMPLO^® (plozasiran)

REDEMPLO (plozasiran) is approved by the U.S. Food and Drug Administration as an adjunct to diet to reduce triglycerides in adults with Familial Chylomicronemia Syndrome (FCS). REDEMPLO is an siRNA therapeutic designed to suppress the production of apoC-III, a protein produced in the liver that raises triglyceride levels by slowing their breakdown and clearance. By targeting apoC-III with sustained silencing, REDEMPLO delivers significant reductions in triglyceride levels. REDEMPLO is the first and only siRNA FDA-approved treatment studied in both genetically confirmed and clinically diagnosed patients living with FCS.

For more information about REDEMPLO, visit Our Medicines.

IMPORTANT SAFETY INFORMATION

CONTRAINDICATIONS

None.

ADVERSE REACTIONS

Most common adverse reactions in REDEMPLO treated patients (incidence ≥10% of patients treated with REDEMPLO and >5% more frequently than with placebo) are hyperglycemia, headache, nausea, and injection site reaction.

Please see full Prescribing Information for REDEMPLO^®.

About Arrowhead Pharmaceuticals

Arrowhead Pharmaceuticals develops medicines that treat intractable diseases by silencing the genes that cause them. Using a broad portfolio of RNA chemistries and efficient modes of delivery, Arrowhead therapies trigger the RNA interference mechanism to induce rapid, deep, and durable knockdown of target genes. RNA interference, or RNAi, is a mechanism present in living cells that inhibits the expression of a specific gene, thereby affecting the production of a specific protein. Arrowhead’s RNAi-based therapeutics leverage this natural pathway of gene silencing.

For more information, please visit www.arrowheadpharma.com, or follow us on X (formerly Twitter) at @ArrowheadPharma, LinkedIn, Facebook, and Instagram. To be added to the Company’s email list and receive news directly, please visit http://ir.arrowheadpharma.com/email-alerts.

Safe Harbor Statement under the Private Securities Litigation Reform Act:

This news release contains forward-looking statements within the meaning of the “safe harbor” provisions of the Private Securities Litigation Reform Act of 1995. Any statements contained in this release except for historical information may be deemed to be forward-looking statements. Without limiting the generality of the foregoing, words such as “may,” “will,” “expect,” “believe,” “anticipate,” “hope,” “intend,” “plan,” “project,” “could,” “estimate,” “continue,” “target,” “forecast” or “continue” or the negative of these words or other variations thereof or comparable terminology are intended to identify such forward-looking statements. In addition, any statements that refer to projections of our future financial performance, trends in our business, expectations for our product pipeline, products or product candidate or other characterizations of future events or circumstances are forward-looking statements. These forward-looking statements include, but are not limited to, statements about our beliefs and expectations regarding the long-term impacts of REDEMPLO (plozasiran) on patient health and the health care system; our beliefs and expectations regarding the pricing, value, or expected timing for availability of our drugs and drug candidates; and our believes and expectations around the potential uses and value of the TRiM™ platform. These statements are based upon our current expectations and speak only as of the date hereof. Actual results or outcomes may differ materially and adversely from those expressed in any forward-looking statements as a result of numerous factors and uncertainties the safety and efficacy of our products and product candidates, pricing and reimbursement decisions related to our products, demand for our products, decisions of regulatory authorities and the timing thereof, the duration and impact of regulatory delays in our clinical programs, our ability to finance our operations, the likelihood and timing of the receipt of future milestone and licensing fees, the future success of our scientific studies, the timing for starting and completing clinical trials, rapid technological change in our markets, the enforcement of our intellectual property rights, and the other risks and uncertainties described in our most recent Annual Report on Form 10-K, subsequent Quarterly Reports on Form 10-Q and other documents filed with the Securities and Exchange Commission from time to time. We assume no obligation to update or revise forward-looking statements to reflect new events or circumstances.

Source: Arrowhead Pharmaceuticals, Inc.

Contacts

Arrowhead Pharmaceuticals, Inc.

Vince Anzalone, CFA

626-304-3400

ir@arrowheadpharma.com

Investors:
LifeSci Advisors, LLC

Brian Ritchie
212-915-2578

britchie@lifesciadvisors.com

Media:
LifeSci Communications, LLC

Kendy Guarinoni, Ph.D.

724-910-9389

kguarinoni@lifescicomms.com