Mundipharma announces patient enrolment completion in Phase III ReSPECT clinical trial for REZZAYO® (rezafungin)




Mundipharma announces patient enrolment completion in Phase III ReSPECT clinical trial for REZZAYO® (rezafungin)

For Trade and Medical Media Only

• Enrolment completed in Phase III ReSPECT study in prophylaxis of fungal infections in adult patients undergoing allogeneic blood and marrow transplant
• Topline results from ReSPECT study expected by mid-2026

CAMBRIDGE, England–(BUSINESS WIRE)–Mundipharma today announced completion of patient enrolment for the ongoing, global Phase III ReSPECT trial evaluating REZZAYO^® (rezafungin).

The ReSPECT trial is a global, randomised, double-blind, controlled, pivotal Phase 3 trial of rezafungin versus the standard antimicrobial regimen to prevent invasive fungal disease due to Candida, Aspergillus and Pneumocystis in subjects undergoing allogeneic blood and marrow transplantation.

“Completing patient enrolment for the Phase III ReSPECT study marks a key milestone in addressing the unmet need for antifungal prevention in immunosuppressed patients,” said Dr Yuri Martina, Chief Development and Medical Officer at Mundipharma. “Patients have enrolled in this global study from more than 50 clinical trial centres across seven countries. We look forward to the clinical evidence that this study will produce.”

*ENDS*

About invasive candidiasis

Invasive candidiasis (IC) continues to be an area of significant unmet need, especially for critically ill patients in hospitals and patients with compromised immune systems.^1,2 Despite a number of available treatments, the mortality rate for patients with invasive candidiasis is as high as 40%.^2,3 IC is characterised as a severe, life-threatening systemic Candida infection of the bloodstream and/or deep/visceral tissues, known as candidemia and deep-seated tissue candidiasis.⁴

About rezafungin

Rezafungin is indicated for the treatment of invasive candidiasis in adults.⁵

REZZAYO^® is a registered trademark of NAPP Pharmaceutical Group Limited a member of the Mundipharma network.

About Mundipharma

Mundipharma is a global healthcare company with a presence in Africa, Asia Pacific, Canada, Europe, Latin America and the Middle East. In line with its mission, United for Patients, Mundipharma is dedicated to bringing innovative treatments to patients in the areas of pain management, infectious disease, ophthalmology, oncology, respiratory and central nervous system. For more information visit www.mundipharma.com.

Commercialisation rights to rezafungin in the United States are licensed to CorMedix Inc.

References:

1. Cortegiani A, et al. Cochrane Database Syst Rev 2016;1:CD004920
2. Kullberg BJ, Arendrup MC. N Engl J Med 2015;373:1445–56
3. Bassetti M, et al. Crit Care 2019;23(1):219
4. Cortes JA, Corrales IF. Invasive candidiasis: Epidemiology and Risk Factors. November 2018. Available at https://www.intechopen.com/chapters/64365 (last accessed October 2025)
5. REZZAYO^® Summary of Product Characteristics. Available at: https://www.medicines.org.uk/emc/product/15479/smpc#gref (last accessed October 2025)

Job code: GBL-S-RZF-2500016

Date of preparation: October 2025

Contacts

Media contact:

Media.relations@mundipharma.com

Data from GNT-018-IDES Trial Supports Feasibility of imlifidase as Pretreatment in Gene Therapy Treatment for Patients With Crigler–Najjar Syndrome Who Are Immune to AAV




Data from GNT-018-IDES Trial Supports Feasibility of imlifidase as Pretreatment in Gene Therapy Treatment for Patients With Crigler–Najjar Syndrome Who Are Immune to AAV

Results presented today at ESGCT 2025

PARIS–(BUSINESS WIRE)–Genethon, a worldwide pioneer and leader in research and development of gene therapy for rare genetic diseases, and Hansa Biopharma, a Sweden-based leader in IgG cleaving enzyme technology announced today that a patient with a rare liver disease and immunity to the AAV vector has been successfully treated with Genethon’s AAV-based GNT0003 gene therapy for Crigler-Najjar syndrome, following prior administration of imlifidase, an enzyme capable of temporarily inhibiting the immune response. This encouraging result, achieved in a clinical trial, is a significant advance in the treatment of patients with immunity to AAVs who were previously ineligible for clinical trials and existing gene therapy treatments.

Gene therapy involves injecting a gene drug into an organism using a vector, a “means of transport” usually derived from viruses, such as AAVs (adeno-associated viruses), which are commonly used for gene therapy in for example neuromuscular, liver, and eye diseases. Initial contact with the natural virus can cause the body to develop immunoglobulin G (IgG) antibodies that neutralize AAVs. It is estimated that one in three people is naturally immune to AAVs, thereby excluding a large number of patients from the possibility of benefiting from gene therapy using an AAV vector. To evaluate potential options for treating patients with natural immunity to AAVs, researchers at Genethon tested imlifidase, an enzyme developed by Hansa Biopharma, as a pre-treatment. This enzyme is capable of cleaving IgG, thereby rapidly and significantly reducing the level of anti-AAV antibodies and allowing the administration of a gene therapy drug candidate.

Dr. Jérémy Do Cao (Béclère Hospital, AP-HP, France) presented at the 2025 congress of the European Society of Gene & Cell Therapy (ESGCT), the results of using imlifidase as a pre-treatment for GNT0003 gene therapy in a patient with a severe form of Crigler–Najjar syndrome who is naturally immune to the AAV8 vector, as part of the clinical trial (GNT-018-IDES) conducted by Genethon in collaboration with Hansa Biopharma.

In this first patient treated, the study demonstrated:

– the feasibility and safety of this approach: imlifidase administered prior to GNT0003 gene therapy successfully cleaved and inactivated the patient’s antibodies and enabled treatment with GNT0003. No severe side effects related to GNT0003 or imlifidase were reported.

– initial efficacy data: GNT0003, Genethon’s gene therapy drug candidate significantly lowered the patient’s bilirubin levels, enabling her to stop hours of daily phototherapy, which had been essential to her survival until then. The phototherapy has been interrupted sixteen weeks after the injection, as planned in the protocol. Additional data will be needed to confirm this efficacy in the longer term.

This is the first time that gene therapy has been successfully administered to a patient with Crigler–Najjar syndrome who has antibodies against AAV8. If the results are confirmed in the next stages of the trial, this approach could become a promising option for patients with antibodies to AAVs, who are currently ineligible for clinical trials and existing gene therapy treatments.

“It’s incredible to have had the chance to witness the clinical translation of this project, which I saw come to life in our laboratory,” said Giuseppe Ronzitti, Head of the Immunology and Liver Disease Laboratory and Director of Scientific Forecasting at Genethon. “It’s a project that involved a significant portion of Genethon working toward a common goal. The preliminary data we’ve obtained indicate that we still have a lot to learn about the immune response to AAV vectors, but the solution is potentially there.”

About imlifidase

Imlifidase is a unique antibody-cleaving enzyme, originating from Streptococcus pyogenes, that specifically targets IgG and inhibits the IgG-mediated immune response. ⁽⁶⁾ It has a rapid onset of action, cleaving IgG antibodies and inhibiting their activity within hours of administration. Imlifidase has conditional marketing approval in EU, Norway, Iceland, Lichtenstein and the UK and is marketed under the trade name IDEFIRIX® for the desensitization treatment of highly sensitized adult kidney transplant patients with a positive crossmatch against an available deceased donor. ⁽⁶⁾ Is also approved in Switzerland and Australia.

About GNT003

The drug candidate GNT-0003, developed by Genethon to treat Crigler-Najjar syndrome, is currently being evaluated in a trial (GNT-012-CRIG) conducted in France, the Netherlands, and Italy in patients with no pre-existing immunity to AAV, with encouraging results. ⁽⁵⁾

About Crigler-Najjar syndrome

Crigler-Najjar syndrome is a rare genetic liver disease characterized by abnormally high levels of bilirubin in the blood (hyperbilirubinemia), which leads to irreversible neurological damage manifested as muscle weakness, lethargy, deafness, mental retardation, and eye movement paralysis. This accumulation of bilirubin is caused by a deficiency of the UGT1A1enzyme, responsible for transforming bilirubin into a substance that can be eliminated by the body. It can result in significant neurological damage and death if not treated quickly. At present, patients must undergo prolonged daily phototherapy (often more than 10 hours a day, sometimes up to 15 hours a day) to keep their bilirubin levels below the toxicity threshold. Crigler-Najjar syndrome is an ultra-rare disease affecting less than one in one million people per year. Liver transplantation remains the only definitive cure to date, but is associated with significant morbidity and mortality, as well as graft shortage. ⁽⁷⁾

About Hansa Biopharma

Hansa Biopharma AB is a pioneering commercial-stage biopharmaceutical company on a mission to develop and commercialize innovative, lifesaving and life-altering treatments for patients with rare immunological conditions. The company has a rich and expanding research and development program based on proprietary IgG-cleaving enzyme technology platform, to address serious unmet medical needs in autoimmune diseases, gene therapy and transplantation. The company’s portfolio includes imlifidase, a first-in-class immunoglobulin G (IgG) antibody-cleaving enzyme therapy, which has been shown to enable kidney transplantation in highly sensitized patients and HNSA-5487, a next-generation IgG cleaving molecule with redosing potential. Hansa Biopharma is based in Lund, Sweden, and has operations in Europe and the United States. The company is listed on Nasdaq Stockholm under the ticker HNSA. Find out more at https://www.hansabiopharma.com/ and on LinkedIn.

About Genethon

A pioneer in the discovery and development of gene therapies for rare diseases, Genethon is a nonprofit laboratory created by the AFM-Telethon. A first gene therapy drug, to which Genethon contributed, has been approved for marketing for spinal muscular atrophy. With more than 240 scientists and experts, Genethon’s goal is to develop innovative therapies that change the lives of patients suffering from rare genetic diseases. Thirteen gene therapy products developed by Genethon or to which Genethon has contributed are currently undergoing clinical trials for diseases of the liver, blood, immune system, muscles, and eyes. Seven other products are in preparation for clinical trials over the next five years. Find out more on visit http://www.genethon.com

References

Contacts

Press contact:
Stephanie Bardon – communication@genethon.fr / +33 (0)6 45 15 95 87

Data Analyses Show Benefit for Individuals With Infantile-Onset Niemann-Pick Disease Type C Treated With Investigational Drug Adrabetadex




Data Analyses Show Benefit for Individuals With Infantile-Onset Niemann-Pick Disease Type C Treated With Investigational Drug Adrabetadex

• Analysis shows statistically significant survival benefit in individuals with infantile-onset Niemann-Pick disease type C (NPC) treated with adrabetadex compared to matched external controls
• Findings from an analysis of key biomarkers support the disease-modifying potential of adrabetadex in individuals with NPC

THOUSAND OAKS, Calif.–(BUSINESS WIRE)–Mandos Health^® by Beren Therapeutics Public Benefit Corporation (P.B.C.) announced today that findings from analyses of its investigational therapy adrabetadex will be presented at the 54^th Child Neurology Society (CNS) Annual Meeting, taking place on October 8-11, 2025 in Charlotte, North Carolina.

Two analyses detail the clinical and biological effects of adrabetadex in Niemann-Pick disease type C (NPC), a rare and progressive neurodegenerative disorder characterized by impaired cholesterol trafficking, severe neurological deficits and premature mortality. The first abstract reports improved overall survival in adrabetadex-treated participants with neurological symptom onset before 6 years of age, compared to matched external controls. The second abstract provides biomarker data supporting the potential of adrabetadex as a disease-modifying therapy.

“These analyses include long-term treatment data allowing evaluation of the effect on survival. They represent the most comprehensive clinical assessment of adrabetadex to date and show its therapeutic potential for individuals with infantile-onset Niemann-Pick disease type C (NPC) and their families. If approved, adrabetadex could become the first treatment option that improves survival in patients with infantile-onset NPC,” said Jason Camm, Chief Executive Officer, Mandos Health by Beren Therapeutics Public Benefit Corporation (P.B.C.).

Clinically meaningful survival benefit

Dr. Elizabeth Berry-Kravis, Professor of Pediatrics, Director of RUSH Pediatric Neurosciences F.A.S.T. Center for Translational Research at Rush University Medical Center and lead study author, will present the survival analysis comparing adrabetadex-treated participants from clinical trials and an ongoing Expanded Access Program (EAP) to controls developed from four major disease databases or publications. Participants with infantile-onset NPC (neurological symptoms before 6 years of age) treated with intrathecal adrabetadex had a statistically significant increase in survival compared to matched, untreated external controls.

“These data represent an important step forward in our understanding of how treatment with adrabetadex may impact disease progression in individuals with infantile-onset Niemann-Pick disease type C,” said Berry-Kravis. “The rigorous methodology we employed in comparing treated participants to matched external controls provides valuable insights into how treatment can potentially extend the lives of these patients.”

Evidence of disease-modifying potential

Results from a biomarker analysis of cerebrospinal fluid (CSF) samples banked during a previously completed Phase 2b/3 clinical trial show that adrabetadex addresses the core pathology of NPC and is associated with reductions in biomarkers of neuronal damage and cell death. Specifically, treatment with adrabetadex increased CSF levels of 24(S)-hydroxycholesterol, which is low in individuals with NPC, reflecting the restoration of intracellular cholesterol trafficking that is impaired in NPC. Adrabetadex treatment also decreased levels of both calbindin D and fatty acid-binding protein 3 (FABP3), markers associated with neuronal injury and death.

“These biomarker findings provide compelling evidence that adrabetadex addresses the underlying pathophysiology of Niemann-Pick disease type C (NPC). These data strengthen our understanding of how adrabetadex works at the molecular level and support its potential as a disease-modifying investigational therapy for individuals with NPC,” said Dr. Forbes D. Porter, Eunice Kennedy Shriver National Institute of Child Health and Human Development, National Institutes of Health, who led the early Phase 1 and 2 clinical trials and whose team provided natural history data to support the studies.

Adrabetadex, which is not approved by the FDA, was generally well-tolerated. The main adverse events associated with adrabetadex include hearing impairment that can be managed with hearing aids when necessary, and post-dose fatigue and/or ataxia.

Both of these analyses, previously presented at the 150^th annual meeting of the American Neurological Association, will be featured in encore poster presentations during the CNS meeting:

About Niemann-Pick Disease Type C

Niemann-Pick disease type C (NPC) is a rare, autosomal-recessive, severe, neurodegenerative disorder caused by pathologic variants in the NPC1 (~95% of cases) or NPC2 genes, leading to impaired cholesterol trafficking resulting in progressive neurological decline and premature death. Earlier neurological onset is associated with more rapid progression and poorer prognosis, with mean survival of ~5.6 years for early infantile onset (age of neurological onset <2 years) and ~13.4 years for late infantile-onset (2 to <6 years). Individuals with early and late infantile-onset NPC typically present with manifestations affecting multiple organs, with the most severe and debilitating effects occurring in the brain.

About Adrabetadex (VTS-270)

Adrabetadex (VTS-270) is a proprietary mixture of 2-hydroxypropyl-β-cyclodextrin isomers under investigation as a treatment for Niemann-Pick disease type C (NPC). By re-establishing intracellular cholesterol trafficking, adrabetadex directly addresses the core pathology of NPC.

Adrabetadex has not been approved by the U.S. Food and Drug Administration (FDA) or any other health authority at this time.

About Mandos Health

Mandos LLC, a wholly-owned subsidiary of Beren Therapeutics Public Benefit Corporation (P.B.C.), is a biotechnology company committed to the development of adrabetadex for individuals living with Niemann-Pick disease type C (NPC), a condition characterized by defects in intracellular cholesterol trafficking. The company’s experienced team of scientists are focused on evaluating adrabetadex, a derivatized cyclodextrin, as a potential treatment that can target the underlying pathology of NPC. Since 2021, Mandos Health has supported the NPC community by providing access to adrabetadex* through an Expanded Access Program (EAP). The company will continue working closely with patients, families, researchers, regulators and others on a path to bring forth this potentially transformative, investigational therapy for NPC and other cholesterol-trafficking diseases. For more information, please visit mandoshealth.com/about-us.

About Rush University Medical Center

Rush University System for Health (RUSH) is an academic health system whose mission is to improve the health of the individuals and diverse communities it serves through the integration of outstanding patient care, education, research and community partnerships. RUSH comprises Rush University Medical Center, Rush University, Rush Copley Medical Center, and Rush Oak Park Hospital, as well as numerous outpatient care facilities. Rush University, with more than 2,500 students, is a health sciences university that comprises Rush Medical College, the College of Nursing, the College of Health Sciences, and the Graduate College.

*Adrabetadex is an investigational drug that has not been approved by the U.S. Food and Drug Administration and has not been found safe and effective to treat NPC or any other condition.

Contacts

Amanda Eckel

BGB Group

Aeckel@bgbgroup.com

GE HealthCare unveils Carestation 850 at ANESTHESIOLOGY 2025: its next-generation anesthesia delivery system designed to adapt to care needs




GE HealthCare unveils Carestation 850 at ANESTHESIOLOGY 2025: its next-generation anesthesia delivery system designed to adapt to care needs

• Carestation 850 is designed to help health systems navigate the evolving landscape of anesthesia care now and in the future with an ergonomic design, continuously optimized algorithms, adaptable technologies and sustainable practices.
• With the goal of enhancing clinical precision, Carestation 850 features tools and customizable applications that are designed to help adapt to the needs of each patient, from neonates to the elderly.
• GE HealthCare will showcase Carestation 850 at booth #1314 during ANESTHESIOLOGY 2025 taking place from October 10-14, in San Antonio, Texas.

CHICAGO–(BUSINESS WIRE)–GE HealthCare (Nasdaq: GEHC) today announced the unveiling of Carestation™ 850, its next-generation, FDA 510(k) pendingⁱ anesthesia delivery system that is designed to help care teams adapt to evolving clinical and operational needs. Marking the latest evolution of GE HealthCare’s Carestation portfolio, Carestation 850 has the potential to advance anesthesia care through tools, customizable applications, and a sleek, space conscious footprint. The FDA 510(k) submission for Carestation 850 follows the recent CE mark in Europe and Australian Therapeutic Goods approval in Australia and New Zealand.

Anesthesia professionals today are facing more demands, as health systems grapple with a high volume of increasingly complex surgical cases.ⁱⁱ This strain is faced across the anesthesia field, including anesthesiologists, clinical registered nurse anesthetists and anesthesia assistants. An estimated 68% of anesthesia professionals are at high risk of burnout, contributing to high turnover and staffing shortages across the industry.^iii,iv,v These trends are expected to continue, with the anesthesiologist shortage projected to reach 12,500 by 2033, underscoring the need for more efficient care delivery.^vi

“In the dynamic operating environment, anesthesia professionals are focused on safety while managing increasingly complex cases and changing patient needs,” said John Beard, MD, anesthesiologist and Chief Medical Officer of Patient Care Solutions at GE HealthCare. “As anesthesia care teams face new challenges, technology can help ease the burden and support safer and more effective care delivery. We’re excited about the potential of Carestation 850 to meet today’s demands in anesthesia delivery and evolve with health systems in the future.”

In surgical and procedural settings, optimizing patient safety is essential, with anesthesia professionals playing a central role in achieving that goal. Carestation 850 is designed to help clinicians more confidently care for each patient and optimize procedural outcomes. To support clinical decision making, the intuitive widescreen display and an enhanced user interface aim to deliver clear data visualization – empowering clinicians to focus on measurements that drive patient care. The system also features a vaporization platform that is designed with advanced alerting capabilities and the ability to refill while in use that could help anesthesia providers proactively manage cases and minimize disruptions. Additionally, Carestation 850 is designed to include tools and applications to aid health systems’ sustainability goals.

“The unveiling of Carestation 850 reinforces our commitment to advancing care that can think one step ahead with its potential to evolve with the changing health systems’ needs through continuously optimized algorithms and adaptable technologies,” said Alla K. Woodson, General Manager, Anesthesia and Global Services, GE HealthCare. “We listened to our customers, and with their help we created Carestation 850, a solution that has potential to transform the future of perioperative care and complement the expertise of clinicians in the operating room by delivering precision, sustainability and efficiency.”

Learn more about Carestation 850 and the full suite of anesthesia solutions at the GE HealthCare booth (#1314) at ANESTHESIOLOGY® 2025 or visit online at gehealthcare.com.

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.

Contacts

GE HealthCare Media Contact:
Kimberly Schmohl

M +1 929 289 1937

kimberly.schmohl@gehealthcare.com

Merck Expands Tulisokibart Clinical Development Program With Initiation of Phase 2b Trials in Three Additional Immune-Mediated Inflammatory Diseases




Merck Expands Tulisokibart Clinical Development Program With Initiation of Phase 2b Trials in Three Additional Immune-Mediated Inflammatory Diseases

Tulisokibart, an investigational anti-TL1A monoclonal antibody currently in Phase 3 trials for ulcerative colitis and Crohn’s disease, to be studied in hidradenitis suppurativa, radiographic axial spondyloarthritis and rheumatoid arthritis

RAHWAY, N.J.–(BUSINESS WIRE)–$MRK #MRK–Merck (NYSE: MRK), known as MSD outside of the United States and Canada, announced it has initiated three Phase 2b trials evaluating the safety and efficacy of tulisokibart (MK-7240), an investigational humanized monoclonal antibody targeting tumor necrosis factor (TNF)-like cytokine 1A (TL1A), in patients with three immune-mediated inflammatory diseases:

• MK-7240-12 (NCT06956235) studying patients with moderate to severe hidradenitis suppurativa (HS)
• MK-7240-013 (NCT07133633) studying patients with radiographic axial spondyloarthritis (r-axSpA; also known as ankylosing spondylitis)
• MK-7240-014 (NCT07176390) studying patients with rheumatoid arthritis (RA)

Global recruitment of these trials has begun, targeting enrollment of more than 640 patients across the three studies.

“The expansion of our tulisokibart clinical development program reflects Merck’s ongoing commitment to addressing the burden of immune-mediated inflammatory diseases,” said Dr. Aileen Pangan, vice president and head of immunology, global clinical development, Merck Research Laboratories. “We’re excited to further evaluate the potential of tulisokibart as a treatment for patients across multiple diseases in rheumatology and dermatology.”

With the initiation of these Phase 2b trials, tulisokibart is now being investigated in a total of six diseases. Merck is also currently conducting two Phase 3 studies evaluating the efficacy and safety of tulisokibart in patients with two different types of inflammatory bowel disease (IBD), ATLAS-UC (NCT06052059) in ulcerative colitis (UC) and ARES-CD (NCT06430801) in Crohn’s disease (CD), and a Phase 2 study in systemic sclerosis-associated interstitial lung disease (SSc-ILD) (NCT05270668). For an overview of Merck’s clinical development program in immunology, please click here.

About hidradenitis suppurativa (HS)

Hidradenitis suppurativa is a chronic inflammatory skin condition that affects hair follicles and is characterized by small, painful abscesses under the skin in areas of the body such as the armpits, groin, buttocks and breasts. Hidradenitis suppurativa affects a variable proportion of the population, with prevalence estimates ranging from 0.1% to 0.8%.

About radiographic axial spondyloarthritis (r-axSpA)

Radiographic axSpA (also known as ankylosing spondylitis) is characterized by chronic inflammation and pain involving the spine and the joints that connect the bottom of the spine to the pelvis. As its name suggests, damage associated with r-axSpA is visible on X-rays. Worldwide, it is thought to affect 0.1% to 1% of all people.

About rheumatoid arthritis (RA)

Rheumatoid arthritis is a chronic autoimmune condition characterized by the inflammation of joints, which can lead to pain, swelling and stiffness. Rheumatoid arthritis can also affect other parts of the body, including the skin, eyes, mouth, heart and lungs. Globally, it is estimated that 17.9 million people are affected by RA, which represents a 13.2% increase since 1990.

About tulisokibart

Tulisokibart is an investigational humanized monoclonal antibody directed to a novel target, tumor necrosis factor (TNF)-like cytokine 1A (TL1A), that is associated with both intestinal inflammation and fibrosis. Tulisokibart is thought to bind both soluble and membrane-bound human TL1A. Clinical studies suggest that tulisokibart may inhibit inflammatory pathways involved in inflammatory bowel disease (IBD), and help reduce intestinal fibrosis, which may be important in altering disease progression in IBD. Merck is developing tulisokibart for the treatment of immune-mediated inflammatory diseases including ulcerative colitis (UC), Crohn’s disease (CD), systemic sclerosis-associated interstitial lung disease (SSc-ILD), hidradenitis suppurativa (HS), radiographic axial spondyloarthritis (r-axSpA), and rheumatoid arthritis (RA).

Merck’s commitment to immunology

Advances in our understanding of human biology have led to the emergence of innovative medicines and new modalities that aim to change approaches to treatment of immune-mediated inflammatory diseases. Our scientists are applying their expertise to the discovery and development of therapies to help people with immune-mediated inflammatory diseases. Our research efforts are focused on investigating novel targets such as TL1A and CD30L and their potential role in many immune-mediated inflammatory diseases.

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. There can be no guarantees with respect to pipeline candidates that the candidates will receive the necessary regulatory approvals or that they will prove to be commercially successful. 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:

Eilyn Segura

(203) 940-6259

Elizabeth Sell

(484) 689-9978

Investor Contacts:

Peter Dannenbaum

(732) 594-1579

Steven Graziano

(732) 594-1583

Within3 Names Ian McKinnon, Ph.D., as Managing Director, Global Head of Consulting




Within3 Names Ian McKinnon, Ph.D., as Managing Director, Global Head of Consulting

Former Oracle leader joins to advance launch intelligence innovation

CLEVELAND–(BUSINESS WIRE)–#AI—Within3, the leading Launch Intelligence™ platform for life science, today announced the appointment of Ian McKinnon, Ph.D., as Managing Director, Global Head of Consulting. McKinnon joins from Oracle, where he most recently served as Global Head of Commercial Consulting, Life Sciences.

With more than two decades of experience guiding pharmaceutical and biotech companies through complex commercialization challenges, McKinnon has held senior leadership roles at Oracle, Cerner, Kantar Health, and TNS Healthcare. His expertise spans launch excellence, evidence-based strategy, advanced analytics, and the application of AI and machine learning to real-world data.

“Bringing Ian on board is another signal of our ambition to redefine how life sciences organizations approach launch,” said Lance Hill, CEO of Within3. “Top industry leaders don’t leave Oracle to join just any company. Ian’s decision underscores the disruptive work we’re doing with Launch Intelligence™ and reinforces our reputation as the partner driving pharma’s next era of innovation.”

In his new role, McKinnon will accelerate the growth of Within3’s Launch Intelligence™ platform and services, ensuring clients capture maximum value from real-time, decision-ready insights. He will lead initiatives that help pharmaceutical brand, medical, and field teams unify fragmented data, anticipate market shifts, and act with confidence at every stage of launch. By combining deep commercialization expertise with Within3’s proprietary technology, McKinnon will help expand the company’s ability to deliver intelligence and guidance that enable life science organizations to launch with confidence.

“Joining Within3 is about being part of a visionary organization,” said McKinnon. “The company is building something that simply does not exist elsewhere in the industry, and it’s an opportunity to apply my experience to a team that is setting the pace for how launches succeed in pharma.”

This leadership appointment comes as Within3 continues rapid growth, expanding its Launch Intelligence™ platform and services to support pharmaceutical companies across the launch lifecycle.

About Within3

We help pharma teams make faster, smarter decisions by unifying the most critical sources of insight — including field activity, HCP engagement, social sentiment, claims data, congresses, and more — into a single, decision-ready view of the market. Powered by Know Everything™, Within3 accelerates time-to-insight and delivers precise, actionable intelligence that drives measurable impact across brand, medical, and field teams.

Trusted by all the top 20 pharmaceutical companies, Within3 enables life sciences organizations to move at the speed of launch — without sacrificing insight or clarity.

Contacts

Media Contact:

Aly Madjerich

Within3

SVP, Global Marketing

amadjerich@within3.com

Owlstone Medical Wins up to $49.1 Million Award from ARPA-H to Develop At-home Multi-Cancer-Early Detection Tests




Owlstone Medical Wins up to $49.1 Million Award from ARPA-H to Develop At-home Multi-Cancer-Early Detection Tests

• Goal of POSEIDON is to enable accurate, low-cost, accessible cancer screening for all Americans
• Program to deliver first-in-class synthetic-sensor based MCED test for Stage I detection of 30+ solid tumors
• Owlstone to work in partnership with the Massachusetts Institute of Technology, Boston University, Georgia Tech Research Corporation, Qurin B.V., and Planned Systems International Inc.

CAMBRIDGE, England–(BUSINESS WIRE)–Owlstone Medical (“Owlstone”), the global leader in Breath Biopsy^® for applications in early disease detection and precision medicine, today announced that it has won an award of up to $49.1 million from the Advanced Research Projects Agency for Health (ARPA-H) for the Platform Optimizing SynBio for Early Intervention and Detection in Oncology (POSEIDON) program. POSEIDON aims to develop first-in-class synthetic-sensor based Multi-Cancer-Early Detection (MCED) tests for Stage I detection of 30+ solid tumors using only breath and urine samples that can be performed in the home and are available over the counter.

Almost 40% of Americans will develop cancer in their lifetimes and cancer is the second leading cause of death in the U.S. The number of new cancer diagnoses in 2025 is estimated to be more than 2 million, with over 618,000 cancer deaths, equivalent to almost 1,700 deaths every day. The patient-related economic burden of cancer in 2019 was more than $21 billion in the U.S., of which treatment costs are by far the largest component. Critically, costs associated with late-stage diagnoses are much higher than early-stage, however cancer is difficult to detect early when it is most curable.

Drastically reducing late-stage diagnoses by detecting cancer at Stage I would not only increase treatment effectiveness, but also significantly reduce cancer care costs, restoring up to $2.3 trillion to the U.S. economy. Currently available screening technologies are unable to address this need due to performance limitations, and many Americans are too remote to access clinic and hospital-based screening programs. While emerging technologies such as liquid biopsy hold great promise for later stage cancer detection and to help guide therapy selection, performance in early-stage cancer detection has been insufficient.

Owlstone’s project, in partnership with the Massachusetts Institute of Technology, Boston University, Georgia Tech Research Corporation, Qurin B.V., and Planned Systems International Inc, aims to overcome this challenge by delivering accurate, low-cost cancer screening for 30+ solid tumors to Americans aged 18 and older.

The project involves the inhalation of a mix of pan-cancer and tumor-specific synthetic sensors from a single-use inhaler, which then circulate throughout the body and accumulate on the surface of cancer cells. The reporters produced by the sensors are either DNA-based which act as a readable barcode, or a set of volatile organic compounds (VOCs), supporting the detection of 36 cancers in total. These will be collected at-home or in clinic in urine samples and from breath respectively using portable collection and analysis devices. Results will be uploadable real-time to electronic health records (EHR) for rapid review by healthcare professionals, integrating seamlessly into clinical practice and digitally enabled care.

This unique approach offers significant advantages over competing technologies. These include boosting the signal to enhance test performance such that cancer is reliably detectable from early stage, enabling simple and non-invasive sample collection at home, rapid result generation and EHR integration, and a low-cost manufacturing model such that economics are not a barrier to adoption of the technology as the new standard of care for early cancer detection.

“The field of cancer screening needs a revolution, and POSEIDON stands ready to deliver. The program allows for a better future by creating broadly accessible, at-home tests that will accurately detect 30+ cancers as early as Stage I, when tumors are still small and the chances of survival are high,” said POSEIDON Program Manager Ross Uhrich, DMD, MBA. “This revolutionary funding effort brings together experts in synthetic biology, oncology, medical devices, big cancer data, and commercialization to create test kits that will transform how and when people are screened for cancer. POSEIDON will allow every American the opportunity to test themselves long before they have symptoms and at their discretion. POSEIDON’s rigorous performance metrics and translational focus reflect our clear commitment to bring these technologies safely and directly to all Americans.”

Billy Boyle, co-founder and CEO at Owlstone Medical, said: “Access to an accurate and low-cost MCED test that does not require a doctor’s visit or laboratory testing is key to preventing late-stage diagnoses. This award validates both breath as a diagnostic approach and Owlstone’s EVOC^® probes as a reporter technology to overcome the shortcomings and challenges that have held back early cancer detection previously. We are grateful to ARPA-H for the opportunity to bring transformative MCED testing to every American within the next decade.”

Contacts

Dr Ben Rutter, Zyme Communications

ben.rutter@zymecommunications.com
+44 (0) 7920-770935

Bimiralisib, Topical PI3K/mTOR Inhibitor Shows Up To 92% Clearance Rate And Superior Tolerability In Proof-Of-Concept Actinic Keratosis Study




Bimiralisib, Topical PI3K/mTOR Inhibitor Shows Up To 92% Clearance Rate And Superior Tolerability In Proof-Of-Concept Actinic Keratosis Study

Torqur AG Announced Full Phase 2 Results at EADV Congress 2025

BASEL, Switzerland–(BUSINESS WIRE)–Torqur AG, subsidiary of Swiss Rockets incubator and a clinical-stage company advancing targeted therapies in oncodermatology and oncology, announced the results of the full Phase 2 proof-of-concept study results of topical bimiralisib gel (2%) for the treatment of actinic keratosis (AK) at the European Academy of Dermatology and Venereology (EADV) Congress 2025 in Paris, the largest dermatology conference in Europe.

The randomized, multi-center study evaluated topical bimiralisib gel as a field-directed treatment for AK on the face, scalp, and/or back of hands. Results demonstrated substantial efficacy and a favorable safety profile, supporting further clinical development:

• Efficacy: 92% of complete or near complete response with Olsen grade 1 patients, and overall, 52% of patients in the 2-week period and 71% in the 4-week period achieved an Investigator’s Global Assessment (IGA) score of 0–1 (complete or partial clearance). All patients experienced some degree of lesion clearance after the initial treatment period. Clearance was seen in Olsen grade 1 and grade 2 AK lesions. Subanalysis showed that patients with Olsen grade 2 also had significant clearance with 48% complete or near complete response.
• Safety: Treatment was well tolerated, with related adverse events being generally local skin reactions with the vast majority being mild (only four grade 2 events), all resolving without intervention. Retreatment during the optional extension phase for up to 8 weeks was feasible without any significant or new safety concerns. Further improvements of lesions were noted with additional cases of complete clearance.
• The Phase 2 study enrolled 46 patients across two leading Swiss dermatology centers: University Hospital Basel (Prof. A. Navarini) and Lausanne University Hospital (Prof. O. Gaide).

Dr. Vladimir Cmiljanovic, CEO of Torqur AG, said: “Presenting these Phase 2 results at Europe’s most prestigious dermatology congress is a tremendous milestone. The high rates of clearance and consistently favorable tolerability profile underscore bimiralisib’s potential to become a new standard for patients with actinic keratosis. We are now preparing for the pivotal Phase 3 clinical trials and discussing with potential partners to bring this therapy closer to patients worldwide.”

Prof. Dr. Alexander Navarini, Chairman of Dermatology at University Hospital Basel and Lead Principal Investigator, commented: “These results show that bimiralisib gel 2% has both efficacy and safety across different grades of actinic keratosis. This represents a promising new non-invasive option for patients, addressing a significant unmet need in dermatology.”

Prof. Dr. Olivier Gaide, Head of the Center of Excellence for Skin Cancer and Melanoma at Lausanne University Hospital and Principal Investigator, added: “With actinic keratosis being the most common precancerous skin condition, these data highlight the great potential of this targeted topical therapy to counter progression to squamous cell carcinoma.”

Actinic keratosis affects more than 10% of Europeans and is considered the most common pre-cancerous dermatological condition driven by an overactive PI3K/mTOR pathway. Bimiralisib gel is a selective pan-PI3K/mTOR inhibitor and offers a new mechanism-based approach to treat early-stage skin lesions effectively and safely.

About Torqur AG (www.torqur.com)

Torqur AG is an innovative Swiss clinical-stage biotechnology company dedicated to advancing next-generation therapies in oncology and dermatology. Its lead product, bimiralisib, is a selective pan-PI3K/mTOR inhibitor with applications across cancer and dermatological indications. Torqur AG is a fully owned subsidiary of Swiss Rockets AG (www.swissrockets.com), the leading biotechnology incubator and accelerator of Switzerland, headquartered in Basel.

Forward-Looking Statements

This press release contains forward-looking statements within the meaning of applicable securities laws, including statements regarding the clinical development, potential regulatory approval, and future commercialization of bimiralisib. Forward-looking statements are based on current expectations and involve risks and uncertainties that could cause actual results to differ materially. Torqur AG undertakes no obligation to update or revise any forward-looking statements, whether as a result of new information, future events, or otherwise.

Contacts

For media inquiries or further information, please contact:

Dr. Vladimir Cmiljanovic, Ph.D.

Chief Executive Officer Torqur AG

E-mail: vladimir.cmiljanovic@swissrockets.com

Dr. Sabina R. Korfmann-Bodenmann

KCCC Korfmann Corporate Communications Consulting AG

s.korfmann@kccc.ch

Celltrion Presents Positive Real-World Data on Switching from intravenous (IV) to subcutaneous (SC) infliximab at UEG Week 2025 Meet the Expert sessions




Celltrion Presents Positive Real-World Data on Switching from intravenous (IV) to subcutaneous (SC) infliximab at UEG Week 2025 Meet the Expert sessions

• Switching from intravenous (IV) to subcutaneous (SC) infliximab was shown to be well tolerated, with a low relapse risk for most IBD patients and high treatment persistence^{1, 2}

INCHEON, South Korea–(BUSINESS WIRE)–Celltrion, Inc. today showcased real-world evidence supporting the use of subcutaneous (SC) infliximab, confirming the efficacy and safety of switching to SC infliximab 120mg every two weeks in patients with inflammatory bowel disease (IBD).

The real-world evidence was presented at the Meet the Expert (MTE) Sessions at UEG 2025, and included presentations from Professor Nicolas Mathieu, Medical Director of MICI Institut Privé, Cliniques des Cèdres and Associate Professor of Gastroenterology, and Prof Anthony Buisson, Head of IBD Unit at University Hospital Estaing, Clermont Ferrand, France.

Professor Nicolas Mathieu presented a real-life study which demonstrated that the switch from IV infliximab to SC infliximab is well tolerated amongst patients. The presentation particularly highlighted the results from the multicentric, prospective PEREM real-life cohort study, which found high treatment persistence, with more than 95% of patients in remission remaining on therapy after one year in real-world practice.¹ In addition, there was no significant difference of SC infliximab persistence at Week 48 between patients who were on combination therapy with immunomodulator at both inclusion and 3 months and those on SC infliximab monotherapy.²

In Professor Anthony Buisson’s session, it was shared that patients switching from IV to SC infliximab is feasible and safe, even in challenging cases such as obesity, prior perianal disease, or patients with complicated phenotypes (structuring or fistulizing CD). Long term persistence of SC infliximab was also confirmed in multiple studies across the UK and France, and was generally considered effective, safe, and well accepted, with low relapse risk in most IBD patients. SC infliximab also showed promising efficacy after IV induction in patients with active perianal lesions.¹

“The Meet the Expert sessions provide an important opportunity to share real-world evidence on the role of subcutaneous infliximab,” said Nam Lee, Vice President of Global Medical Affairs at Celltrion. “The evidence confirms that switching from intravenous to subcutaneous therapy is effective and safe and supports more sustainable healthcare delivery.”

Notes to Editors:

About the subcutaneous (SC) formulation of CT-P13

CT-P13 SC is the world’s first subcutaneous formulation of infliximab. A 120mg fixed dose of CT-P13 SC has been approved for use in 60 countries including the US, UK, EU, Canada, Brazil, Australia and Taiwan, in adults regardless of body weight. The SC formulation of infliximab has the potential to enhance treatment options by providing high consistency in drug exposure and a convenient method of administration.^3,4 In July 2024, CT-P13 SC received final approval from the European Commission for an additional dosing regimen and dose escalation, which allows 3-IV induction dosing regimen and dose escalation of subcutaneous maintenance dose from CT-P13 SC 120 mg Q2W to 240 mg Q2W for patients with loss of response.⁵ Long term data from two-year extension of the LIBERTY studies (LIBERTY-CD and LIBERTY-UC) have demonstrated sustained efficacy and safety of CT-P13 SC, with clinical remission, response, and corticosteroid-free remission generally maintained through Week 102.⁶

About Celltrion

Celltrion is a leading biopharmaceutical company that specializes in researching, developing, manufacturing, marketing and sales of innovative therapeutics that improve people’s lives worldwide. Celltrion is a pioneer in the biosimilar space, having launched the world’s first monoclonal antibody biosimilar. Our global pharmaceutical portfolio addresses a range of therapeutic areas including immunology, oncology, haematology, ophthalmology and endocrinology. Beyond biosimilar products, we are committed to advancing our pipeline with novel drugs to push the boundaries of scientific innovation and deliver quality medicines.

For more information, please visit our website www.celltrion.com/en-us and stay updated with our latest news and events on our social media – LinkedIn, Instagram, X, and Facebook.

FORWARD-LOOKING STATEMENT

Certain information set forth in this press release contains statements related to our future business and financial performance and future events or developments involving Celltrion Inc. and its subsidiaries that may constitute forward-looking statements, under pertinent securities laws. This press release contains forward looking statements. These statements may be also identified by words such as “prepares”, “hopes to”, “upcoming”, “plans to”, “aims to”, “to be launched”, “is preparing”, “once gained”, “could”, “with the aim of”, “may”, “once identified”, “will”, “working towards”, “is due”, “become available”, “has potential to”, “anticipates”, the negative of these words or such other variations thereon or comparable terminology.

In addition, our representatives may make oral forward-looking statements. Such statements are based on the current expectations and certain assumptions of Celltrion Inc. and its subsidiaries’ management, of which many are beyond its control.

Forward-looking statements are provided to allow potential investors the opportunity to understand management’s beliefs and opinions in respect of the future so that they may use such beliefs and opinions as one factor in evaluating an investment. These statements are not guarantees of future performance and undue reliance should not be placed on them.

Such forward-looking statements necessarily involve known and unknown risks and uncertainties, which may cause actual performance and financial results in future periods to differ materially from any projections of future performance or results expressed or implied by such forward-looking statements.

Celltrion Inc. and its subsidiaries undertake no obligation to update forward-looking statements if circumstances or management’s estimates or opinions should change except as required by applicable securities laws.

References

¹ Buisson A et al. Clinical Gastroenterology and Hepatology 2023; Buisson A et al. Alimentary Pharmacology and Therapeutics 2023

² Mathieu N et al., Persistence and Safety of Subcutaneous Infliximab 1 Year After Switch From Intravenous Route in IBD Patients in REMission. Clinical Gastroenterology and Hepatology. 2025

³ Schreiber S et al., Gastroenterology. 2021;160(7):2340-2353

⁴ Westhovens R et al., Rheumatology. 2021;60(5):2277-2287

⁵ European Medicines Agency Summary of Product Characteristics (SmPC), Remsima®. Available at link to SmPC [Last accessed October 2025].

⁶ Colombel JF et al., Subcutaneous Infliximab (CT-P13 SC) as Maintenance Therapy for Crohn’s Disease and Ulcerative Colitis: 2-Year Results from Open Label Extensions of Two Randomized Controlled Trials (LIBERTY). Journal of Crohn’s and Colitis. 2025;19(6):jjaf060

Contacts

For further information please contact:
Donna Gandhi

dgandhi@hanovercomms.com
+44 (0) 7827 053 502

DATROWAY® Demonstrated Statistically Significant and Clinically Meaningful Improvement in Overall Survival as First-Line Therapy for Patients with Metastatic Triple Negative Breast Cancer for Whom Immunotherapy Was Not an Option in TROPION-Breast02




DATROWAY® Demonstrated Statistically Significant and Clinically Meaningful Improvement in Overall Survival as First-Line Therapy for Patients with Metastatic Triple Negative Breast Cancer for Whom Immunotherapy Was Not an Option in TROPION-Breast02

• Daiichi Sankyo and AstraZeneca’s DATROWAY is the first and only therapy to significantly improve overall survival versus chemotherapy in this patient population
• DATROWAY also demonstrated a highly statistically significant and clinically meaningful improvement in the dual primary endpoint of progression-free survival
• Plans for global regulatory submissions are underway

TOKYO & BASKING RIDGE, N.J.–(BUSINESS WIRE)–Positive topline results from the TROPION-Breast02 phase 3 trial showed DATROWAY^® (datopotamab deruxtecan) demonstrated a statistically significant and clinically meaningful improvement for the dual primary endpoints of overall survival (OS) and progression-free survival (PFS) compared to investigator’s choice of chemotherapy as first-line treatment for patients with locally recurrent inoperable or metastatic triple negative breast cancer (TNBC) for whom immunotherapy was not an option.

DATROWAY is a specifically engineered TROP2 directed DXd antibody drug conjugate (ADC) discovered by Daiichi Sankyo (TSE: 4568) and being jointly developed and commercialized by Daiichi Sankyo and AstraZeneca (LSE/STO/Nasdaq: AZN).

Approximately 70% of patients with metastatic TNBC are not candidates for immunotherapy, including all patients whose tumors do not express PD-L1 as well as patients with PD-L1 expressing tumors who cannot receive immunotherapy due to other factors.¹ Chemotherapy remains the first-line standard of care for these patients.²

“DATROWAY is the first antibody drug conjugate and the only therapy to significantly improve overall survival compared to chemotherapy in patients with metastatic triple negative breast cancer for whom immunotherapy is not an option,” said Ken Takeshita, MD, Global Head, R&D, Daiichi Sankyo. “These landmark results from TROPION-Breast02 strengthen our confidence in our ongoing clinical development program for DATROWAY in triple negative breast cancer and other tumor types. We look forward to discussing these data with global regulatory authorities and to bringing DATROWAY to patients with triple negative breast cancer as soon as possible.”

“TROPION-Breast02 is the only trial ever to show an overall survival benefit in the first-line treatment of patients with metastatic triple negative breast cancer for whom immunotherapy is not an option,” said Susan Galbraith, MBBChir, PhD, Executive Vice President, Oncology Hematology R&D, AstraZeneca. “We expect today’s results will mark an inflection point in the treatment of these patients who have the poorest prognosis of any type of breast cancer and urgently need better options.”

The safety profile of DATROWAY was consistent with previous clinical trials of DATROWAY in breast cancer. These data from TROPION-Breast02 will be presented at an upcoming medical meeting and shared with regulatory authorities.

Daiichi Sankyo and AstraZeneca are evaluating DATROWAY across stages and treatment settings of TNBC in three additional phase 3 trials. TROPION-Breast03 is evaluating DATROWAY as adjuvant therapy with or without AstraZeneca’s anti-PD-L1 therapy durvalumab versus investigator’s choice of therapy in patients with stage I-III TNBC with residual invasive disease after neoadjuvant systemic therapy. TROPION-Breast04 is evaluating neoadjuvant DATROWAY plus durvalumab versus neoadjuvant pembrolizumab plus chemotherapy in patients with stage II-III triple negative or hormone receptor (HR) low, HER2 low or negative breast cancer. TROPION-Breast05 is evaluating first-line DATROWAY with or without durvalumab versus investigator’s choice of therapy in patients with metastatic TNBC whose tumors express PD-L1.

About TROPION-Breast02

TROPION-Breast02 is a global, multicenter, randomized, open-label phase 3 trial evaluating the efficacy and safety of DATROWAY versus investigator’s choice of chemotherapy (paclitaxel, nab-paclitaxel, capecitabine, carboplatin or eribulin) in patients with previously untreated locally recurrent inoperable or metastatic TNBC for whom immunotherapy was not an option. This included patients whose tumors did not express PD-L1 as well as patients with PD-L1 expressing tumors who could not receive immunotherapy due to prior exposure in early-stage disease, comorbidities or immunotherapy not being accessible in their geography. Enrollment included patients with de novo or recurrent disease, regardless of disease-free interval, and those with poor prognostic factors such as brain metastases.

The dual primary endpoints of TROPION-Breast02 are PFS assessed by blinded independent central review and OS. Key secondary endpoints include PFS as assessed by investigator, objective response rate, duration of response, disease control rate, pharmacokinetics and safety.

TROPION-Breast02 enrolled 644 patients at sites in Africa, Asia, Europe, North America and South America. For more information visit ClinicalTrials.gov.

About Triple Negative Breast Cancer

TNBC accounts for approximately 15% of all breast cancer cases, with an estimated 345,000 diagnoses globally each year.^3,4 TNBC is diagnosed more frequently in younger and premenopausal women, and is more prevalent in Black and Hispanic women.^5,6,7 Metastatic TNBC is the most aggressive type of breast cancer and has the worst prognosis, with median overall survival of just 12 to 18 months and only about 14% of patients living five years following diagnosis.^5,8,9

While some breast cancers may test positive for estrogen receptors, progesterone receptors or overexpression of HER2, TNBC tests negative for all three.⁵ Due to its aggressive nature and absence of common breast cancer receptors, TNBC is characteristically difficult to treat.⁵ For patients with metastatic disease with PD-L1 expressing tumors, the addition of immunotherapy to chemotherapy has improved outcomes in the first-line setting.^10,11 However, for approximately 70% of patients with metastatic TNBC who are not candidates for immunotherapy, chemotherapy remains the first-line standard of care.^1,2

TROP2 is a protein broadly expressed in several solid tumors, including TNBC.¹² TROP2 is associated with increased tumor progression and poor survival in patients with breast cancer.^13,14

About DATROWAY

DATROWAY (datopotamab deruxtecan; datopotamab deruxtecan-dlnk in the U.S. only) is a TROP2 directed ADC. Designed using Daiichi Sankyo’s proprietary DXd ADC Technology, DATROWAY is one of six DXd ADCs in the oncology pipeline of Daiichi Sankyo, and one of the most advanced programs in AstraZeneca’s ADC scientific platform. DATROWAY is comprised of a humanized anti-TROP2 IgG1 monoclonal antibody, developed in collaboration with Sapporo Medical University, attached to a number of topoisomerase I inhibitor payloads (an exatecan derivative, DXd) via tetrapeptide-based cleavable linkers.

DATROWAY (6 mg/kg) is approved in more than 35 countries/regions worldwide for the treatment of adult patients with unresectable or metastatic HR positive, HER2 negative (IHC 0, IHC 1+ or IHC 2+/ISH-) breast cancer who have received prior endocrine-based therapy and chemotherapy for unresectable or metastatic disease based on the results from the TROPION-Breast01 trial.

DATROWAY (6 mg/kg) is approved in Russia and the U.S. for the treatment of adult patients with locally advanced or metastatic EGFR-mutated non-small cell lung cancer (NSCLC) who have received prior EGFR-directed therapy and platinum-based chemotherapy, based on the results from TROPION-Lung05 and TROPION-Lung01 trials. Continued approval for this indication in the U.S. may be contingent upon verification and description of clinical benefit in a confirmatory trial.

About the DATROWAY Clinical Development Program

A comprehensive global clinical development program is underway with more than 20 trials evaluating the efficacy and safety of DATROWAY across multiple cancers, including NSCLC, TNBC and urothelial cancer. The program includes eight phase 3 trials in lung cancer and five phase 3 trials in breast cancer evaluating DATROWAY as a monotherapy and in combination with other cancer treatments in various settings.

About the Daiichi Sankyo and AstraZeneca Collaboration

Daiichi Sankyo and AstraZeneca entered into a global collaboration to jointly develop and commercialize ENHERTU^® in March 2019 and DATROWAY in July 2020, except in Japan where Daiichi Sankyo maintains exclusive rights for each ADC. Daiichi Sankyo is responsible for the manufacturing and supply of ENHERTU and DATROWAY.

About the ADC Portfolio of Daiichi Sankyo

The Daiichi Sankyo ADC portfolio consists of seven ADCs in clinical development crafted from two distinct ADC technology platforms discovered in-house by Daiichi Sankyo.

The ADC platform furthest in clinical development is Daiichi Sankyo’s DXd ADC Technology where each ADC consists of a monoclonal antibody attached to a number of topoisomerase I inhibitor payloads (an exatecan derivative, DXd) via tetrapeptide-based cleavable linkers. The DXd ADC portfolio currently consists of ENHERTU, a HER2 directed ADC, and DATROWAY, a TROP2 directed ADC, which are being jointly developed and commercialized globally with AstraZeneca. Patritumab deruxtecan (HER3-DXd), a HER3 directed ADC, ifinatamab deruxtecan (I-DXd), a B7-H3 directed ADC, and raludotatug deruxtecan (R-DXd), a CDH6 directed ADC, are being jointly developed and commercialized globally with Merck & Co., Inc, Rahway, NJ, USA. DS-3939, a TA-MUC1 directed ADC, is being developed by Daiichi Sankyo.

The second Daiichi Sankyo ADC platform consists of a monoclonal antibody attached to a modified pyrrolobenzodiazepine (PBD) payload. DS-9606, a CLDN6 directed PBD ADC, is the first of several planned ADCs in clinical development utilizing this platform.

Ifinatamab deruxtecan, patritumab deruxtecan, raludotatug deruxtecan, DS-3939 and DS-9606 are investigational medicines that have not been approved for any indication in any country. Safety and efficacy have not been established.

DATROWAY U.S. Important Safety Information

Indications

DATROWAY^® is a Trop-2-directed antibody and topoisomerase inhibitor conjugate indicated for the treatment of:

• adult patients with locally advanced or metastatic epidermal growth factor receptor (EGFR)-mutated non-small cell lung cancer (NSCLC) who have received prior EGFR-directed therapy and platinum-based chemotherapy.

  This indication is approved under accelerated approval based on objective response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trial.

• adult patients with unresectable or metastatic, hormone receptor (HR)-positive, human epidermal growth factor receptor 2 (HER2)-negative (IHC 0, IHC 1+ or IHC 2+/ISH-) breast cancer who have received prior endocrine-based therapy and chemotherapy for unresectable or metastatic disease.

Contraindications

None.

Warnings and Precautions

Interstitial Lung Disease/Pneumonitis

DATROWAY can cause severe, life-threatening, or fatal interstitial lung disease (ILD) or pneumonitis.

Locally Advanced or Metastatic NSCLC

In the pooled safety population of 484 patients with NSCLC from TROPION-Lung01, TROPION-Lung05, and TROPION-PanTumor01, ILD/pneumonitis occurred in 7% of patients treated with DATROWAY, including 0.6% of patients with Grade 3 and 0.4% with Grade 4. There were 8 (1.7%) fatal cases. The median time to onset for ILD was 1.4 months (range: 0.2 months to 9 months). Eleven patients (2.3%) had DATROWAY withheld and 20 patients (4.1%) permanently discontinued DATROWAY due to ILD/pneumonitis. Systemic corticosteroids were required in 79% (26/33) of patients with ILD/pneumonitis. ILD/pneumonitis resolved in 45% of patients.

Unresectable or Metastatic Breast Cancer

In the pooled safety population of 443 patients with breast cancer from TROPION-Breast01 and TROPION-PanTumor01, ILD/pneumonitis occurred in 3.6% of patients treated with DATROWAY, including 0.7% of patients with Grade 3. There was one fatal case (0.2%). The median time to onset for ILD was 2.8 months (range: 1.1 months to 10.8 months). Four patients (0.9%) had DATROWAY withheld and 7 patients (1.6%) permanently discontinued DATROWAY due to ILD/pneumonitis. Systemic corticosteroids were required in 60% (9/15) of patients with ILD/pneumonitis. ILD/pneumonitis resolved in 40% of patients.

Patients were excluded from clinical studies for a history of ILD/pneumonitis requiring treatment with steroids or for ongoing ILD/pneumonitis.

Monitor patients for new or worsening respiratory symptoms indicative of ILD/pneumonitis (e.g., dyspnea, cough, fever) during treatment with DATROWAY. For asymptomatic (Grade 1) ILD/pneumonitis, consider corticosteroid treatment (e.g., ≥0.5 mg/kg/day prednisolone or equivalent). For symptomatic ILD/pneumonitis (Grade 2 or greater), promptly initiate systemic corticosteroid treatment (e.g., ≥1 mg/kg/day prednisolone or equivalent) and continue for at least 14 days followed by gradual taper for at least 4 weeks.

Withhold DATROWAY in patients with suspected ILD/pneumonitis and permanently discontinue DATROWAY if ≥Grade 2 ILD/pneumonitis is confirmed.

Ocular Adverse Reactions

DATROWAY can cause ocular adverse reactions including dry eye, keratitis, blepharitis, meibomian gland dysfunction, increased lacrimation, conjunctivitis, and blurred vision.

In the pooled safety population, ocular adverse reactions occurred in 36% of patients treated with DATROWAY. Twenty patients (2.2%) experienced Grade 3 ocular adverse reactions, which included keratitis, dry eye, and blurred vision, and one patient experienced a Grade 4 ocular adverse reaction of conjunctival hemorrhage. The most common (≥5%) ocular adverse reactions were dry eye (17%), keratitis (14%), and increased lacrimation (7%). The median time to onset for ocular adverse reactions was 2.3 months (range: 0.03 months to 23.2 months). Of the patients who experienced ocular adverse reactions, 39% had complete resolution, and 10% had partial improvement (defined as a decrease in severity by one or more grades from the worst grade at last follow up). Ocular adverse reactions led to dosage interruption in 3.6% of patients, dosage reductions in 2.5% of patients, and permanent discontinuation of DATROWAY in 1% of patients.

Patients with clinically significant corneal disease were excluded from clinical studies.

Advise patients to use preservative-free lubricant eye drops several times daily for prophylaxis. Advise patients to avoid use of contact lenses unless directed by an eye care professional.

Refer patients to an eye care professional for an ophthalmic exam including visual acuity testing, slit lamp examination (with fluorescein staining), intraocular pressure, and fundoscopy at treatment initiation, annually while on treatment, at end of treatment, and as clinically indicated.

Promptly refer patients to an eye care professional for any new or worsening ocular adverse reactions. Monitor patients for ocular adverse reactions during treatment with DATROWAY, and if diagnosis is confirmed, withhold, reduce the dose, or permanently discontinue DATROWAY based on severity.

Stomatitis

DATROWAY can cause stomatitis, including mouth ulcers and oral mucositis.

In the pooled safety population, stomatitis occurred in 63% of patients treated with DATROWAY, including 8% of patients with Grade 3 events and one patient with a Grade 4 reaction. The median time to first onset of stomatitis was 0.5 months (range: 0.03 months to 18.6 months). Stomatitis led to dosage interruption in 6% of patients, dosage reductions in 11% of patients, and permanent discontinuation of DATROWAY in 0.5% of patients.

In patients who received DATROWAY in TROPION-Breast01, 39% used a mouthwash containing corticosteroid for management or prophylaxis of stomatitis/oral mucositis at any time during the treatment.

Advise patients to use a steroid-containing mouthwash for prophylaxis and treatment of stomatitis. Instruct the patient to hold ice chips or ice water in the mouth throughout the infusion of DATROWAY.

Monitor patients for signs and symptoms of stomatitis. If stomatitis occurs, increase the frequency of mouthwash and administer other topical treatments as clinically indicated. Based on the severity of the adverse reaction, withhold, reduce the dose, or permanently discontinue DATROWAY.

Embryo-Fetal Toxicity

Based on its mechanism of action, DATROWAY can cause embryo-fetal harm when administered to a pregnant woman because the topoisomerase inhibitor component of DATROWAY, DXd, is genotoxic and affects actively dividing cells.

Advise patients of the potential risk to a fetus. Advise female patients of reproductive potential to use effective contraception during treatment with DATROWAY and for 7 months after the last dose. Advise male patients with female partners of reproductive potential to use effective contraception during treatment with DATROWAY and for 4 months after the last dose.

Adverse Reactions

The pooled safety population described in WARNINGS AND PRECAUTIONS reflects exposure to DATROWAY in 927 patients as a single agent at 6 mg/kg administered as an intravenous infusion once every 3 weeks (21-day cycle) until disease progression or unacceptable toxicity. This included 137 patients with NSCLC in TROPION-Lung05, 297 patients with NSCLC in TROPION-Lung01, 360 patients with HR-positive, HER2-negative breast cancer in TROPION-Breast01, and 50 patients with NSCLC and 83 patients with breast cancer in TROPION-PanTumor01 (NCT03401385). Among 927 patients who received DATROWAY, 45% were exposed for 6 months or longer and 19% were exposed for greater than one year. In this pooled safety population, the most common (≥20%) adverse reactions were stomatitis (63%), nausea (52%), fatigue (45%), alopecia (38%), constipation (28%), decreased appetite (23%), rash (23%), vomiting (22%), and musculoskeletal pain (20%). In this pooled safety population, the most common (≥2%) Grade 3 or 4 laboratory abnormalities were decreased lymphocytes (9%) and decreased hemoglobin (3.5%).

Locally Advanced or Metastatic EGFR-Mutated Non-Small Cell Lung Cancer

TROPION-Lung05, TROPION-Lung01, TROPION-PanTumor01

The safety of DATROWAY was evaluated in 125 patients with EGFR-mutated NSCLC who received DATROWAY 6 mg/kg administered as an intravenous infusion once every 3 weeks (21-day cycle) until disease progression or unacceptable toxicity in TROPION-Lung05 and TROPION-Lung01 as well as TROPION-PanTumor01 (NCT03401385). Among these patients, the median duration of treatment was 6.1 months (range 0.7 months to 41.7 months).

The median age was 63 years (range: 36 to 81), 56% of patients were <65 years, 62% of patients were female; 66% were Asian, 26% were White, 0.8% were Black, 6% were other races; and 2.4% were of Hispanic ethnicity.

Serious adverse reactions occurred in 26% of patients who received DATROWAY. Serious adverse reactions in >1% of patients who received DATROWAY were COVID-19 (4%), stomatitis (2.4%), and pneumonia (1.6%). Fatal adverse reactions occurred in 1.6% of patients who received DATROWAY, due to death not otherwise specified.

Permanent discontinuation of DATROWAY due to an adverse reaction occurred in 8% of patients. Adverse reactions which resulted in permanent discontinuation of DATROWAY in >1% of patients included ILD/pneumonitis (2.4%) and abnormal hepatic function (1.6%).

Dosage interruptions of DATROWAY due to an adverse reaction occurred in 43% of patients. Adverse reactions which required dosage interruption in >1% of patients included COVID-19 (13%), stomatitis (7%), fatigue (6%), pneumonia (4%), anemia (2.4%), amylase increased (2.4%), keratitis (2.4%), ILD/pneumonitis (1.6%), decreased appetite (1.6%), dyspnea (1.6%), rash (1.6%), and infusion-related reaction (1.6%).

Dose reductions of DATROWAY due to an adverse reaction occurred in 26% of patients. Adverse reactions which required dose reduction in >1% of patients included stomatitis (14%), keratitis (1.6%), fatigue (1.6%), decreased weight (1.6%) and COVID-19 (1.6%).

The most common (≥20%) adverse reactions, including laboratory abnormalities, were stomatitis (71%), nausea (50%), alopecia (49%), fatigue (42%), decreased hemoglobin (34%), decreased lymphocytes (32%), constipation (31%), increased calcium (31%), increased AST (28%), decreased white blood cell count (27%), increased lactate dehydrogenase (23%), musculoskeletal pain (22%), decreased appetite (20%), increased ALT (20%), and rash (20%).

Clinically relevant adverse reactions occurring in <10% of patients who received DATROWAY included dry skin, blurred vision, abdominal pain, conjunctivitis, dry mouth, ILD/pneumonitis, skin hyperpigmentation, increased lacrimation, and visual impairment.

Unresectable or Metastatic, HR-Positive, HER2-Negative Breast Cancer

TROPION-Breast01

The safety of DATROWAY was evaluated in 360 patients with unresectable or metastatic HR-positive, HER2-negative (IHC 0, IHC1+ or IHC2+/ISH-) breast cancer who received at least one dose of DATROWAY 6 mg/kg in TROPION-Breast01. DATROWAY was administered by intravenous infusion once every three weeks. The median duration of treatment was 6.7 months (range: 0.7 months to 16.1 months) for patients who received DATROWAY.

Serious adverse reactions occurred in 15% of patients who received DATROWAY. Serious adverse reactions in >0.5% of patients who received DATROWAY were urinary tract infection (1.9%), COVID-19 infection (1.7%), ILD/pneumonitis (1.1%), acute kidney injury, pulmonary embolism, vomiting, diarrhea, hemiparesis, and anemia (0.6% each). Fatal adverse reactions occurred in 0.3% of patients who received DATROWAY and were due to ILD/pneumonitis.

Permanent discontinuation of DATROWAY due to an adverse reaction occurred in 3.1% of patients. Adverse reactions which resulted in permanent discontinuation of DATROWAY in >0.5% of patients included ILD/pneumonitis (1.7%) and fatigue (0.6%).

Dosage interruptions of DATROWAY due to an adverse reaction occurred in 22% of patients. Adverse reactions which required dosage interruption in >1% of patients included COVID-19 (3.3%), infusion-related reaction (1.4%), ILD/pneumonitis (1.9%), stomatitis (1.9%), fatigue (1.7%), keratitis (1.4%), acute kidney injury (1.1%), and pneumonia (1.1%).

Dose reductions of DATROWAY due to an adverse reaction occurred in 23% of patients. Adverse reactions which required dose reduction in >1% of patients included stomatitis (13%), fatigue (3.1%), nausea (2.5%), and weight decrease (1.9%).

The most common (≥20%) adverse reactions, including laboratory abnormalities, were stomatitis (59%), nausea (56%), fatigue (44%), decreased leukocytes (41%), decreased calcium (39%), alopecia (38%), decreased lymphocytes (36%), decreased hemoglobin (35%), constipation (34%), decreased neutrophils (30%), dry eye (27%), vomiting (24%), increased ALT (24%), keratitis (24%), increased AST (23%), and increased alkaline phosphatase (23%).

Clinically relevant adverse reactions occurring in <10% of patients who received DATROWAY included infusion-related reactions (including bronchospasm), ILD/pneumonitis, headache, pruritus, dry skin, dry mouth, conjunctivitis, blepharitis, meibomian gland dysfunction, blurred vision, increased lacrimation, photophobia, visual impairment, skin hyperpigmentation, and madarosis.

Contacts

Global/US:
Jennifer Brennan

Daiichi Sankyo, Inc.

jennifer.brennan@daiichisankyo.com
+1 908 900 3183 (mobile)

Japan:

Daiichi Sankyo Co., Ltd.

DS-PR_jp@daiichisankyo.com

Investor Relations Contact:
DaiichiSankyoIR_jp@daiichisankyo.com

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