Centauri Therapeutics Granted FDA QIDP Status for CTX-187 Treatment of Gram-Negative Bacterial Infections




Centauri Therapeutics Granted FDA QIDP Status for CTX-187 Treatment of Gram-Negative Bacterial Infections

• Lead clinical candidate CTX-187 is a novel antimicrobial immunotherapy developed to treat clinically-prevalent and multi-drug resistant Gram-negative bacterial infections
• Qualified Infectious Disease Product designation awarded for the development of antibacterials and antifungals intended to treat serious or life-threatening conditions in humans

ALDERLEY PARK, England–(BUSINESS WIRE)–Centauri Therapeutics Limited (‘Centauri’ or ‘the Company’), an immunotherapy company with a unique and proprietary platform technology applicable across a wide range of therapeutic indications, today announced that the Company’s lead clinical candidate in the ABX-01 programme, CTX-187, has received Qualified Infectious Disease Product (QIDP) designation from the US Food and Drug Administration (FDA). Currently in pre-clinical development ahead of first in-human trials, CTX-187 provides a novel immunotherapeutic approach to treat Gram-negative bacterial infections and expand therapeutic options for clinically vulnerable patients.

QIDP status is awarded under the US GAIN (Generating Antibiotic Incentives Now) provisions for the development of antibacterials and antifungals intended to treat serious or life-threatening conditions in humans. Drug products receiving QIDP status gain access to several benefits to accelerate the development process, including eligibility for fast-track designation and priority review, and a five-year extension to market exclusivity.

CTX-187 is a novel antimicrobial built on Centauri’s proprietary Alphamer^® platform, featuring a dual mechanism-of-action that combines immunotherapeutic effects through complement activation and opsonophagocytosis with antibacterial activity. The molecule is currently in development, progressing to first in-human clinical trials later this year as a new treatment for clinically prevalent and multidrug-resistant Gram-negative bacteria.

Dr. Debra Barker, Chief Medical Officer, Centauri Therapeutics, said: “This designation from the FDA will be invaluable as we continue to advance toward first in-human trials for CTX-187. The recognition of the potential value of our technology further underpins our belief in its potential to transform treatment paradigms for Gram-negative bacterial infections, filling an urgently needed gap in the market for new and effective treatment options for clinically vulnerable patients.”

The Alphamer^® platform is a modularly designed immunotherapeutic that redirects the body’s natural antibody repertoire against disease. The unique design is backed by strong proof-of-concept data, demonstrating >99.9% immune-mediated bacterial clearance at sub-therapeutic doses across Gram-negative and multi-drug resistant bacterial strains in vivo¹. This novel immunotherapeutic approach enables Alphamers to be used as a viable treatment option for clinically vulnerable patient groups where current therapies are limited or ineffective, including patients that are immunosuppressed.

Daniel Hynes, VP of Development, Centauri Therapeutics, commented: “The FDA’s QIDP designation is a crucial programme designed to address the healthcare burden of serious and life-threatening infections by accelerating the development of promising therapeutics. We welcome this regulatory endorsement of CTX-187, which will enable us to streamline our route to regulatory approvals, and bring a much-needed treatment option to address serious infections in vulnerable patient groups.”

The Company has also been supported by CARB-X² (Combating Antibiotic-Resistant Bacteria Biopharmaceutical Accelerator) to undertake research and development studies since 2019, advancing the ABX-01 programme from Lead Optimization to the Preclinical phase.

1. Hairsine, B. et al. (2025) ‘Harnessing endogenous anti-glycan antibodies using a novel, bifunctional immunotherapy to treat gram-negative bacterial infections’, The Journal of Immunology, 00, pp. 1–13. doi:10.1093/jimmun/vkaf055.
2. CARB-X’s funding for this project is provided in part with federal funds from the U.S. Department of Health and Human Services (HHS); Administration for Strategic Preparedness and Response; Biomedical Advanced Research and Development Authority; under agreement number: 75A50122C00028, and by awards from Wellcome (WT224842), Germany’s Federal Ministry of Research, Technology and Space (BMFTR), and the Novo Nordisk Foundation. The content of this press release is solely the responsibility of the authors and does not necessarily represent the official views of CARB-X or any of its funders.

Contacts

Media
Katie Odgaard

katie.odgaard@zymecommunications.com

Alzheon to Present Clinical, Imaging, and Fluid Biomarker Results from Trials with Oral Valiltramiprosate/ALZ-801 at 2026 AD/PD Conference in Copenhagen, Denmark




Alzheon to Present Clinical, Imaging, and Fluid Biomarker Results from Trials with Oral Valiltramiprosate/ALZ-801 at 2026 AD/PD Conference in Copenhagen, Denmark

Valiltramiprosate Shows Promise as an Oral Potentially Disease-Modifying Agent that can Slow Alzheimer’s Disease Based on Clinical Results, MRI Data, and Fluid Biomarkers in APOE4/4 MCI Patients

Two Oral Presentations and Six Posters Highlight Consistent Effects of Oral Valiltramiprosate on Brain Volume, Microstructural Integrity, Neurodegeneration, Systems Modeling, and Long-Term Clinical Stability

Valiltramiprosate Designed to Inhibit Amyloid Aggregation via Distinct Upstream Mode of Action, Targeting Soluble Amyloid Oligomers – A Key Driver of Alzheimer’s Disease Pathology

Precision Medicine Approach Supported by Favorable Safety Results in High-Risk APOE4/4 Patients in Phase 3 & Phase 2 Trials

FRAMINGHAM, Mass.–(BUSINESS WIRE)–#ALZ801—Alzheon, Inc., a clinical-stage biopharmaceutical company developing a broad portfolio of investigational therapies and diagnostic assays for patients with Alzheimer’s disease (AD) and other neurodegenerative disorders, today announced it will present new efficacy, imaging, safety, biomarker, and quantitative systems pharmacology findings for its lead investigational therapy, valiltramiprosate/ALZ-801 during the 2026 International Conference on Alzheimer’s and Parkinson’s Disease (AD/PD) in Copenhagen, Denmark.

“Valiltramiprosate stands out as the only late-stage oral Alzheimer’s treatment with potential for a major near-term impact,” said Susan Abushakra, Chief Medical Officer of Alzheon. “Our results at AD/PD align with previous clinical, imaging, safety, and biomarker data. Targeting toxic amyloid oligomers continues to show promise for preserving cognition, daily function, and brain volume. As we prepare for regulatory discussions, our priority is approval of valiltramiprosate for Alzheimer’s patients with the APOE4 genotype and expanding to additional high-risk groups.”

Valiltramiprosate is an investigational oral therapeutic agent in Phase 3 development that works upstream of anti-amyloid antibodies by preventing the formation of neurotoxic soluble amyloid oligomers. As a valine-conjugated prodrug of tramiprosate with improved pharmacokinetics and brain penetration, valiltramiprosate targets early amyloid aggregation, a core driver of Alzheimer’s pathology and disease progression. Valiltramiprosate aims to preserve brain structure and cognitive function in individuals with early-stage Alzheimer’s disease, with particular emphasis on APOE4/4 homozygotes, which are the highest-risk genetic subgroup, characterized by aggressive neurodegeneration and limited treatment options.

New results to be presented at AD/PD include safety and ARIA analyses from the APOLLOE4 Phase 3 placebo-controlled trial, quantitative systems pharmacology (QSP) analyses of amyloid aggregation dynamics, diffusion MRI findings showing preserved microstructural integrity, and clinical-imaging correlations in the pre-specified Mild Cognitive Impairment (MCI) subgroup of the Phase 3 study. These results are further supported by responder analyses showing reversal of hippocampal atrophy, and long-term MMSE outcomes from a Phase 2 study which had disease stability over four years of treatment in APOE4 MCI carriers.

Details of Presentations at AD/PD Conference

Alzheon will deliver two oral presentations and showcase six posters covering clinical, MRI, microstructural, biomarker, and QSP analyses.

Symposium: Advances in AD Treatment

Saturday, March 21, 2026 – 2:10pm- 4:10pm

Podium Presentation: Clinical Efficacy and Imaging Results of Oral Valiltramiprosate in APOE4/4 Homozygotes with Early AD: Phase 3 Topline Results from 78-Week APOLLOE4 Trial

• Presenter: Dr. Aidan Power, Chief Development Officer, Alzheon, Inc.
• Lecture Time: 2:10pm – 2:25pm

Podium Presentation: Clinical Stabilization in MCI APOE4 Carriers Over 3 Years Correlates with Improved Hippocampal Volume in Valiltramiprosate Phase 2 LTE Study

• Presenter: Dr. John Hey, Chief Scientific Officer, Alzheon, Inc.
• Lecture Time: 2:25pm – 2:40pm

Tuesday, March 17 from 7:30am – Thursday, March 19^th 11:00am

Poster: Biomarker‑Positive APOE4 Carriers with MCI Show Disease Stability Over 4 Years of Valiltramiprosate/ALZ‑801 Treatment: MMSE Responder Analysis from the ALZ‑801 Phase 2 Long‑Term Extension Study

• Presenter: Patrick Kesslak, Senior Research Fellow, Alzheon, Inc.

Thursday, March 19^th from 1:50pm – Saturday, March 21^st 5:00pm

Poster: Positive Effects of Oral Valiltramiprosate on Grey and White Matter Microstructural Integrity by DTI in APOE4/4 Homozygotes with Early AD: APOLLOE4 78‑Week Phase 3 Results

• Presenter: Earvin Liang, VP of Clinical Operations, Alzheon, Inc.

Poster: Safety and ARIA Analyses of the Oral Anti‑Amyloid Oligomer Agent Valiltramiprosate in APOE4/4 Homozygotes with Early AD: Results of the 78‑Week Phase 3 APOLLOE4 Trial

• Presenter: Dr. David Watson, CEO, Alzheimer’s Research & Treatment Center

Poster: Quantitative Systems Pharmacology Analyses of Beta‑Amyloid Aggregation Dynamics: Additive/Synergistic Effects of Valiltramiprosate Used in Combination with Leqembi and Kisunla

• Presenter: Jean Schaefer, VP of CMC & Project Management, Alzheon, Inc.

Poster: Valiltramiprosate Reverses Neurodegeneration and Improves Clinical Outcomes in Subgroup of Early Symptomatic AD in Homozygotes: Results from the 78‑Week Phase 3 APOLLOE4 Trial

• Presenter: Dr. John Hey, Chief Scientific Officer, Alzheon, Inc.

Poster: Valiltramiprosate Effects in the Pre‑Specified MCI Group Show Significant Correlations Between Clinical and Brain Volume Benefits: Phase 3 APOLLOE4 Results in Early AD APOE4/4 Homozygotes

• Presenter: Dr. Aidan Power, Chief Development Officer, Alzheon, Inc.

About ALZ-801

Valiltramiprosate/ALZ-801 is an investigational oral agent currently in Phase 3 development as a potential first-in-class, disease-modifying treatment for Alzheimer’s disease.^3-7,9,12 Valiltramiprosate is designed to inhibit the formation of neurotoxic soluble beta amyloid oligomers that contribute to cognitive decline in individuals with AD.^4-8,10,15 Preclinical mechanism-of-action studies have demonstrated that ALZ-801 can completely block the formation of these neurotoxic oligomers at the dosage used in Phase 3 clinical trials.^3,9,12,14 Valiltramiprosate employs an enveloping molecular mechanism of action intended to prevent the aggregation of soluble amyloid oligomers in the human brain, ¹⁴ which are associated with the onset and progression of cognitive impairment in AD patients.^3,4,7,9,10 In recognition of its therapeutic promise, valiltramiprosate received Fast Track designation from the U.S. Food and Drug Administration in 2017 for the treatment of Alzheimer’s disease.

Clinical trial data indicate that valiltramiprosate exhibits strong clinical efficacy at the MCI stage, and a favorable safety profile, with no observed increase in the risk of brain vasogenic edema.^5-1-10,13,15 The initial Phase 3 program for valiltramiprosate targets Early AD patients who are homozygous for the apolipoprotein ε4 allele (APOE4/4), with plans to expand future research to include AD treatment and prevention in individuals carrying one copy of the APOE4 gene.^3–10

Valiltramiprosate APOLLOE4 Phase 3 Trial

An Efficacy and Safety Study of Valiltramiprosate in APOE4/4 Early Alzheimer’s Disease Subjects (NCT04770220): This trial was designed to evaluate the efficacy, safety, biomarker and imaging effects of 265 mg twice daily oral dose of valiltramiprosate in Early AD subjects with two copies of the apolipoprotein ε4 allele (APOE4/4 homozygotes), who constitute approximately 15% of Alzheimer’s patients. This double-blind, randomized trial compared oral valiltramiprosate to placebo treatment over 78 weeks. The APOLLOE4 trial was supported by a grant from the National Institute on Aging to Alzheon, with Susan Abushakra as the principal investigator.

Valiltramiprosate APOLLOE4 Long Term Extension Trial (Phase 3 LTE)

An ongoing long-term extension of the trial, APOLLOE4-LTE, evaluates valiltramiprosate in subjects who complete the core APOLLOE4 study for an additional 104 weeks of treatment for a total of 182 weeks or 3.5 years over the core and LTE study. This LTE study ended in January 2026 (NCT06304883).

Valiltramiprosate Phase 2 Biomarker Trial

Biomarker Effects of Valiltramiprosate in APOE4 Carriers with Early Alzheimer’s Disease (NCT04693520): This trial was designed to evaluate the effects of 265 mg twice daily oral dose of valiltramiprosate on biomarkers of AD pathology in subjects with Early AD, who have either the APOE4/4 or APOE3/4 genotype and constitute 65-70% of Alzheimer’s patients. The primary outcome was the change from baseline in plasma p-tau₁₈₁. The trial also included evaluation of clinical efficacy, safety, tolerability, and pharmacokinetic profile of valiltramiprosate over 104 weeks of treatment. A completed long-term extension of the trial evaluated the same dose of valiltramiprosate for an additional 104 weeks of treatment for a total of 208 weeks.^3,7,8

About Alzheon

Alzheon, Inc. is a clinical-stage biopharmaceutical company dedicated to advancing a diverse portfolio of product candidates and diagnostic assays for individuals affected by Alzheimer’s disease and other neurodegenerative disorders. The company is focused on innovating therapeutic solutions that directly target the underlying pathology of neurodegeneration. Its lead Alzheimer’s clinical candidate, valiltramiprosate/ALZ-801, is a first-in-class oral agent currently in Phase 3 clinical development as a potentially disease-modifying treatment for Alzheimer’s disease. Valiltramiprosate is an orally administered small molecule shown in preclinical studies to completely inhibit the formation of neurotoxic soluble amyloid oligomers. Leveraging clinical expertise and a robust technology platform, Alzheon pursues drug discovery and development using a precision medicine approach that incorporates individual genetic and biomarker profiles, aiming to advance therapies with meaningful benefits for patients.

Alzheon Scientific Publications

¹Abushakra S, et al: Hippocampal Atrophy on Magnetic Resonance Imaging as a Surrogate Marker for Clinical Benefit and Neurodegeneration in Early Symptomatic Alzheimer’s Disease: Synthesis of Evidence from Observational and Interventional Trials, CNS Drugs 2025; 40, 199-214.

²Abushakra S, et al: Clinical Efficacy, Safety and Imaging Effects of Oral Valiltramiprosate in APOEε4/ε4 Homozygotes with Early Alzheimer’s Disease: Results of the Phase III, Randomized, Double-Blind, Placebo-Controlled, 78-Week APOLLOE4 Trial, Drugs 2025; 85, 1455-1472.

³Pearson D, et al: Polymorph Analysis of ALZ-801 (Valiltramiprosate), a Valine-Conjugated Oral Prodrug of Tramiprosate in Late-Stage Clinical Development for Alzheimer’s Disease, Journal of Chemical Crystallography 2025; 55, 206-215.

⁴Hey JA, et al: Clinical Pharmacokinetics of Oral ALZ-801/Valiltramiprosate in a Two-Year Phase 2 Trial of APOE4 Carriers with Early Alzheimer’s Disease, Clinical Pharmacokinetics 2025; 64, 407-424.

⁵Aye S, et al: Point of View: Challenges in Implementation of New Immunotherapies for Alzheimer’s Disease, The Journal of Prevention of Alzheimer’s Disease 2025;12(1):100022.

⁶Abushakra S, et al: APOLLOE4 Phase 3 Study of Oral ALZ-801/Valiltramiprosate in APOE ε4/ε4 Homozygotes with Early Alzheimer’s Disease: Trial Design and Baseline Characteristics, Alzheimer’s & Dementia 2024; 10(3): e12498.

⁷Tolar M, et al: The Single Toxin Origin of Alzheimer’s Disease and Other Neurodegenerative Disorders Enables Targeted Approach to Treatment and Prevention, International Journal of Molecular Sciences 2024; 25(5), 2727.

⁸Hey JA, et al: Analysis of Cerebrospinal Fluid, Plasma β Amyloid Biomarkers, and Cognition from a 2-Year Phase 2 Trial Evaluating Oral ALZ-801/Valiltramiprosate in APOE4 Carriers with Early Alzheimer’s Disease Using Quantitative Systems Pharmacology Model, Drugs 2024; 84(7), 825-839.

⁹Hey JA, et al: Effects of Oral ALZ-801/Valiltramiprosate on Plasma Biomarkers, Brain Hippocampal Volume, and Cognition: Results of 2-Year Single Arm, Open Label, Phase 2 Trial in APOE4 Carriers with Early Alzheimer’s Disease, Drugs 2024; 84(7), 811-823.

¹⁰Tolar M, et al: Neurotoxic Soluble Amyloid Oligomers Drive Alzheimer’s Pathogenesis and Represent a Clinically Validated Target for Slowing Disease Progression, International Journal of Molecular Sciences 2021; 22(12), 6355.

¹¹Abushakra S, et al: APOE ε4/ε4 Homozygotes with Early Alzheimer’s Disease Show Accelerated Hippocampal Atrophy and Cortical Thinning that Correlates with Cognitive Decline, Alzheimer’s & Dementia 2020; 6(1): e12117.

¹²Tolar M, et al: Aducanumab, Gantenerumab, BAN2401, and ALZ-801—the First Wave of Amyloid-Targeting Drugs for Alzheimer’s Disease with Potential for Near Term Approval, Alzheimer’s Research & Therapy 2020; 12(1): 95.

¹³Tolar M, et al: The Path Forward in Alzheimer’s Disease Therapeutics: Reevaluating the Amyloid Cascade Hypothesis, Alzheimer’s & Dementia 2020; 16(11):1553-1560.

¹⁴Hey JA, et al: Discovery and Identification of an Endogenous Metabolite of Tramiprosate and Its Prodrug ALZ-801 that Inhibits Beta Amyloid Oligomer Formation in the Human Brain, CNS Drugs 2018; 32(9): 849-861.

¹⁵Hey JA, et al: Clinical Pharmacokinetics and Safety of ALZ-801, a Novel Prodrug of Tramiprosate in Development for the Treatment of Alzheimer’s Disease, Clinical Pharmacokinetics 2018; 57(3): 315-333.

¹⁶Abushakra S, et al: Clinical Effects of Tramiprosate in APOE4/4 Homozygous Patients with Mild Alzheimer’s Disease Suggest Disease Modification Potential, Journal of Prevention of Alzheimer’s Disease 2017; 4(3): 149-156.

¹⁷Kocis P, et al: Elucidating the Aβ42 Anti-Aggregation Mechanism of Action of Tramiprosate in Alzheimer’s Disease: Integrating Molecular Analytical Methods, Pharmacokinetic and Clinical Data, CNS Drugs 2017; 31(6): 495-509.

¹⁸Abushakra S, et al: Clinical Benefits of Tramiprosate in Alzheimer’s Disease Are Associated with Higher Number of APOE4 Alleles: The “APOE4 Gene-Dose Effect,” Journal of Prevention of Alzheimer’s Disease 2016; 3(4): 219-228.

Contacts

Media Contact
Yasemin Khakian, Alzheon, Inc.

508.808.2301

yasemin.khakian@alzheon.com

Investor Contact
Erwan de Naurois, Alzheon, Inc.

802.735.8789

erwan.denaurois@alzheon.com

For the First Time, Ferring Reports Revenue of Over €2.5 Billion in 2025




For the First Time, Ferring Reports Revenue of Over €2.5 Billion in 2025

• Ferring’s total revenues for 2025 exceeded €2.5 billion, an increase of 10% from 2024, mainly driven by our flagship product Menopur^®
• Continued ramp-up in the US for Adstiladrin^®, our novel gene-based therapy for bladder cancer, confirming its position as Ferring’s second major growth driver
• Commitment to sustainability demonstrated by SBTi approval of our targets to reduce greenhouse gas emissions, and by our programme to reduce maternal deaths in low- and lower middle-income countries

SAINT-PREX, Switzerland–(BUSINESS WIRE)–Ferring today published its 2025 Annual Report and Sustainability Report. The company achieved total revenues of over €2.5 billion in 2025, an increase of 7% over the previous year at actual exchange rates (AER) and 10% at constant exchange rates (CER). These were mainly driven by our flagship product Menopur^® (menotropins for injection) in reproductive medicine, and the ramp-up in the US of our breakthrough gene-based therapy for non-muscle invasive bladder cancer, Adstiladrin^® (nadofaragene firadenovec-vncg).

Operating expenses were contained to an increase of €61 million year-on-year (i.e. +5% at AER and +7% at CER), and this includes significant non-recurring items (notably impairment charges and restructuring provisions). Underlying operating expenses remained well-controlled, with increased investments targeted to support the growth of Adstiladrin and other opportunities. Thus, operating profit for the year reached €167 million, a decrease of -€24 million (‑13%) versus the prior year at AER, while remaining flat at CER – with the difference being driven by the weaker US dollar.

Following a focus on improving cash conversion after several heavy investments, free cash flow generation approached neutral despite currency headwinds from the weaker US dollar. This represents a substantial improvement compared to the previous year, and a significant step towards sustainable cash generation.

Jean-Frédéric Paulsen, Chairman of the Board of Directors and Chief Executive Officer, said: “This was a pivotal year as we continued evolving our business to become stronger, more agile and more resilient, and importantly we got back to free cash flow neutral. Moreover, in 2025, we introduced an enterprise model designed to create greater value for patients and customers while supporting sustainable growth. This reflects our long-term commitment to serving patients’ need, and fostering an environment where people can learn, grow and perform at their best.”

Ferring has always conducted business responsibly by seeking to protect the environment, create value for society, and uphold our high standards of ethics and governance. In 2025, we passed a major milestone when our targets for reducing greenhouse gas (GHG) emissions were approved by the Science Based Targets initiative (SBTi). This globally recognised standard ensures corporate goals are aligned with international climate policy. During the year, we succeeded in reducing our Scope 1 and 2 GHG emissions by 4.3% and Scope 3 emissions by 19%.

Access to affordable healthcare is embedded in Ferring’s purpose and strategic priorities. The Project Family™: Safe Birth initiative aims to reduce maternal deaths in low- and lower middle-income countries by enabling wider access to Carbetocin Ferring (carbetocin, room-temperature stable formulation). In 2025, we worked with partners to supply around 1.7 million doses of this life-saving medicine at an affordable access price, while gaining further approvals in seven low- and lower middle-income countries.

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. The company is headquartered in Saint-Prex, Switzerland, and has operating subsidiaries in more than 50 countries which market its medicines in over 100 countries.

Learn more at www.ferring.com, or connect with us on LinkedIn, Instagram and YouTube.

Contacts

For more information, please contact

Carine Julen
Senior Manager, Corporate Communications & Public Affairs
carine.julen@ferring.com

C-Further Unveils First Therapeutic Programmes Dedicated to Paediatric Oncology




C-Further Unveils First Therapeutic Programmes Dedicated to Paediatric Oncology

C-Further to advance its first two bespoke paediatric cancer therapeutics in collaboration with leading global academic and industry investigators

Selected programmes to potentially address multiple children’s cancers including Ewing sarcoma, bone sarcoma, medulloblastoma and acute myeloid leukaemia

Additional C-Further programmes to be announced in 2026

LONDON–(BUSINESS WIRE)–C-Further, an international consortium committed to creating new therapeutics for childhood cancers, today unveils the first early-stage therapeutic programmes for its pipeline, dedicated to paediatric oncology indications. Through its collaborative model, C-Further has partnered with investigators at UVA Comprehensive Cancer Center, Dana-Farber Cancer Institute and Mass General Brigham to advance CF-012 for the potential treatment of Ewing sarcoma. In parallel, the consortium has partnered with investigators at MiNK Therapeutics (Nasdaq: INKT) to advance CF-033, leveraging MiNK’s proprietary platform with translational support by investigators at the University of Southampton, for the potential treatment of multiple children’s cancers, including bone sarcoma, medulloblastoma and acute myeloid leukaemia.

Enabled by C-Further’s core partners, Cancer Research Horizons, LifeArc and Great Ormond Street Hospital Charity (GOSH Charity), the consortium will support the progression of CF-012 and CF-033 up to preclinical candidate nomination, subject to completion of scientific milestones. Together, these projects form the foundation of C-Further’s expanding pipeline which is supported by an initial $40m (£30m) budget.

Lone Friis, PhD, C-Further Programme Co-lead said: “By combining the pioneering approaches of our scientific collaborators with industry-standard drug discovery capabilities, expertise of our core partners and reach into critical paediatric-focused networks, C-Further is advancing a pipeline of potential first-in-class therapies for childhood cancers. Guided by an indication-agnostic but child-first approach, we believe the initial CF-012 and CF-033 programmes have the potential to address a profound unmet need across multiple children’s cancers.”

The selected research partners represent top academic and industry investigators from across the globe, underscoring the international reach and ambition of C-Further.

• CF-012 targets ETV6, a newly identified and critical transcriptional dependency that is essential for tumour growth and metastasis. The aim is to develop a potential first-in-class inhibitor with a precise mechanism to disrupt tumour growth and metastasis in young patients.

  John Bushweller, PhD, Professor at the University of Virginia School of Medicine and a member of UVA Comprehensive Cancer Center, investigator for CF-012, said: “We’re excited to partner with C-Further to progress CF-012. Children and young people have historically lacked effective, targeted cancer treatments and CF-012 has the potential to address an urgent unmet need in relapsed and metastatic Ewing sarcoma – a rare, aggressive bone tumour that most commonly occurs in young people. With C-Further’s collaborative, child-first drug discovery model, we believe we have the power to bring these medicines to market.”

  Other key investigators for the CF-012 therapeutic programme include Kimberly Stegmaier, MD, Chair of the Department of Pediatric Oncology at Dana-Farber Cancer Institute, and Miguel Rivera, MD, Assistant Molecular Pathologist at Massachusetts General Hospital.

• CF-033 is a potential first-in-class allogeneic iNKT cell therapy engineered with a specific T-cell receptor targeting PRAME, which bridges innate and adaptive immunity and is designed to enable both direct tumour cell killing and broader immune activation within the tumour microenvironment. As an allogeneic, off-the-shelf approach without the need for toxic lymphodepletion, CF-033 has the potential to support more timely treatment delivery and improved tolerability, which are important considerations in paediatric oncology, where new therapeutic approaches are urgently needed.

  Marco Purbhoo, PhD, Head of Translational Medicine at MiNK Therapeutics, investigator for CF-033, said: “CF-033 is a PRAME-targeted invariant NKT (iNKT) cell therapy developed to address the urgent needs of children with high-risk cancers, which are often marked by treatment resistance and a weakened immune response that cannot sustain tumour control. Unlike conventional CAR-T or TCR-T therapies, CF-033 harnesses the unique biology of iNKT cells — immune cells that can both directly kill cancer and help coordinate a broader, longer-lasting anti-tumour response. Importantly, this approach is designed to be delivered without the need for HLA matching or toxic lymphodepletion, which is particularly meaningful for this vulnerable paediatric population.”

  Ali Roghanian, PhD, Associate Professor at the University of Southampton, is the other key investigator leading the CF-033 therapeutic programme.

C‑Further welcomes expressions of interest from researchers, innovators and partners who share its mission to accelerate new tailored and well-tolerated treatments for children and young people with cancer. The deadline to be considered for the next round of submissions is 13 March 2026.

C-Further has also commenced preliminary research on a third, undisclosed project. Additional programmes are expected to be announced in 2026.

To learn more about ongoing work or to explore partnering opportunities, visit the C-Further website.

About C-Further

C-Further is an international consortium that aims to bring together drug discovery and development researchers, clinicians, partners and impact investors with a shared commitment to creating new therapeutics for childhood cancers.

Our vision is to break from the conventional approach of repurposing adult therapies causing lasting harm to developing bodies – and create a world where young patients are treated effectively with tailored, well-tolerated treatments.

Together we’re combining expertise from around the world to create an innovation ecosystem that allows us to challenge conventional approaches to developing therapies and accelerate promising ideas towards better outcomes for children living with cancer. Our operational model brings together industry-standard drug discovery and academic innovation, ensuring a clear and viable path to patients. If you share this vision and believe you can contribute, we invite you to connect with us and be part of the change: info@c-further.org. Visit www.c-further.org for more information.

About UVA Comprehensive Cancer Center and UVA Health

UVA Comprehensive Cancer Center is one of 57 National Cancer Institute (NCI) designated comprehensive cancer centers in the United States for its work in cancer research, prevention, detection and treatment. UVA Comprehensive Cancer Center provides world-class cancer treatment led by doctors who have been honored by publications such as Best Doctors in America® and America’s Top Doctors®. UVA Comprehensive Cancer Center is part of UVA Health, a leading academic health system that includes the UVA School of Medicine, UVA School of Nursing, UVA Physicians Group and the Claude Moore Health Sciences Library, as well as multiple hospitals across Virginia. In addition, UVA recently launched the Paul and Diane Manning Institute of Biotechnology to accelerate the development of new treatments and cures for cancers and other complex diseases. Visit uvahealth.com for more information, resources and social media links.

About Dana-Farber Cancer Institute

Dana-Farber Cancer Institute is one of the world’s leading centers of cancer research and treatment. Dana-Farber’s mission is to reduce the burden of cancer through scientific inquiry, clinical care, education, community engagement and advocacy. Dana-Farber is a federally designated Comprehensive Cancer Center and a teaching affiliate of Harvard Medical School.

Dana-Farber is the only hospital nationwide with a top 3 U.S. News & World Report Best Cancer Hospital ranking in both adult and pediatric care.

As a global leader in oncology, Dana-Farber is dedicated to a unique and equal balance between cancer research and care, translating the results of discovery into new treatments for patients locally and around the world, offering more than 1,200 clinical trials.

About Mass General Brigham

Mass General Brigham is an integrated academic health care system, uniting great minds to solve the hardest problems in medicine for our communities and the world. Mass General Brigham connects a full continuum of care across a system of academic medical centers, community and specialty hospitals, a health insurance plan, physician networks, community health centers, home care, and long-term care services. Mass General Brigham is a nonprofit organization committed to patient care, research, teaching, and service to the community. In addition, Mass General Brigham is one of the nation’s leading biomedical research organizations with several Harvard Medical School teaching hospitals. For more information, please visit massgeneralbrigham.org.

About MiNK Therapeutics

MiNK Therapeutics is a clinical-stage biopharmaceutical company pioneering allogeneic invariant natural killer T (iNKT) cell therapies and precision-targeted immune technologies. MiNK’s proprietary platform is designed to restore immune balance and drive cytotoxic responses across cancer, immune-mediated diseases, and pulmonary immune failure. MiNK’s lead candidate, agenT-797, is an off-the-shelf iNKT cell therapy currently in clinical development for GvHD, solid tumors, and severe pulmonary inflammation. With a scalable cryopreserved manufacturing process and differentiated biology bridging innate and adaptive immunity, MiNK is committed to developing next-generation immune reconstitution therapies. For more information, visit www.minktherapeutics.com or follow us on X @MiNK_iNKT.

About the University of Southampton

The University of Southampton drives original thinking, turns knowledge into action and impact, and creates solutions to the world’s challenges. We are among the top 100 institutions globally (QS World University Rankings 2025). Our academics are leaders in their fields, forging links with high-profile international businesses and organisations, and inspiring a 24,000-strong community of exceptional students, from over 135 countries worldwide. Through our high-quality education, the University helps students on a journey of discovery to realise their potential and join our global network of over 300,000 alumni. www.southampton.ac.uk

Contacts

C-Further Press Release Contacts:

– Megan McGrath (trade outlets): megan@ctdcomms.com
– Fiona Scott (consumer and nationals): Fiona.Scott@cancer.org.uk
– Please CC Pierre Peotta pierre.peotta@cancer.org.uk

UVA Comprehensive Cancer Center and UVA Health Press Release Contact:

– Josh Barney – jdb9a@uvahealth.org

MiNK Therapeutics Press Release Contacts:

– Investor Contact: 917-362-1370 | investor@minktherapeutics.com
– Media Contact: 781-674-4428 | communications@minktherapeutics.com

MiNK Therapeutics Corporate Spokesperson: Dr. Jennifer Buell

– Media Liaison: Stefanie Perna-Nacar: Stefanie.nacar@agenusbio.com
– Please cc Dr. Buell’s assistant, Dimitra Dariotis-Diaz dimitra.dariotis-diaz@agenusbio.com on any media inquiries sent to Stefanie

The University of Southampton Press Release Contact:

– Lucy Collie, PR Manager: l.j.collie@soton.ac.uk

ViruSure Launches First GMP-Validated Viral Safety Test Based on Oxford Nanopore Sequencing




ViruSure Launches First GMP-Validated Viral Safety Test Based on Oxford Nanopore Sequencing

First-in-class solution consolidates multiple assays into a single test, enabling broader viral detection, faster time-to-result and more confident decision-making in regulated biopharmaceutical quality control

OXFORD, England & VIENNA–(BUSINESS WIRE)–#Nanopore–ViruSure, an Asahi Kasei company and global leader in pathogen safety testing for biopharmaceuticals, today announced the launch of the world’s first GMP-validated adventitious viral agent (AVA) detection test based on Oxford Nanopore sequencing. Designed for use in regulated biopharmaceutical quality control (QC) environments, this process is designed to ensure biologics medicines are free from viral contamination.

The first-in-class solution combines Oxford Nanopore’s rapid, information-rich molecular sensing technology with ViruSure’s GMP expertise, delivering broader viral detection and a more streamlined approach to viral safety testing in commercial biologics manufacturing. This has the potential to enable rapid testing and support confidence in batch release, thus strengthening supply chain resilience.

The launch follows the industry’s first GLP-validated (Good Laboratory Practice) AVA detection test using Oxford Nanopore sequencing in August 2025. Progression to GMP (Good Manufacturing Practice) validation, the standard required for routine use in commercial biopharmaceutical manufacturing, demonstrates the regulatory readiness of Oxford Nanopore sequencing-based approaches for viral safety testing and marks an important step toward wider adoption in regulated environments.

Francis Van Parys, CEO of Oxford Nanopore Technologies, said:

“Rapid Oxford Nanopore sequencing offers a fundamentally different level of resolution for detecting viral contaminants. Through this collaboration with ViruSure, that capability is now available in a GMP-validated format, making advanced sequencing accessible for routine safety testing across biologics manufacturing.”

Faster and boarder AVA detection from a single, comprehensive test

Biopharmaceutical manufacturing underpins the supply of many critical medicines used worldwide. With the global biologics market estimated in excess of £300 billion, and a biosafety testing market alone valued at approximately £3.2 billion, even isolated viral contamination events can have cascading effects across healthcare systems, resulting in patient safety concerns, drug shortages, and substantial financial impact.

Quality control is central to ensuring safe, reliable biologics manufacturing, and new technologies are creating opportunities to make this process more reliable and efficient. While traditional AVA testing has long supported the industry with a broad panel of established assays, the ViruSure and Oxford Nanopore approach brings the benefit of consolidating multiple steps into a single, information-rich test.

Traditional AVA testing relies on numerous parallel and sequential assays to complete a full viral safety panel. Individual tests can take more than 28 days and depending on the level of characterisation can cost tens to hundreds of thousands of pounds, with full panels often taking months. As a result, viral safety testing represents a significant operational component of biologics production, and quality control can account for up to half of the total manufacturing timeline.

Despite their complexity, these approaches can still produce false negatives and false positive results. False positives can trigger unnecessary manufacturing pauses and emergency investigations, creating additional cost and potential supply disruption.

The ViruSure and Oxford Nanopore solution introduces a sequencing-based alternative capable of consolidating many of these individual assays into a single, integrated test that has been demonstrated to have the required sensitivity and specificity necessary to provide assurances around the absence of adventitious agents. By generating a comprehensive and information-rich dataset in one run, manufacturers have the potential to reduce the number of required tests from multiple assays to one, shorten overall testing timelines by weeks, and simplify laboratory workflows within GMP-regulated environments.

Oxford Nanopore’s unique ability to sequence reads of any length enables broader AVA detection and clearer differentiation between background noise, false positives, and true viral contaminants. This supports more confident, data-driven decision-making while reducing the need for unnecessary follow-up investigations, helping to maintain continuity in manufacturing operations.

Dr Andy Bailey, CEO of ViruSure, said:

“Manufacturers need viral safety tests that detect the whole range of potential contaminants, are fast and practical for GMP environments with a low rate of false positives. By validating nanopore sequencing for adventitious viral agent detection, our workflow has demonstrated that we can provide a robust and sensitive solution, with a low risk of false positives, that can strengthen safety assurances for the whole range of biological products.”

About Oxford Nanopore Technologies

Oxford Nanopore Technologies’ goal is to bring the widest benefits to society through enabling the analysis of anything, by anyone, anywhere. The company has developed a new generation of nanopore-based sensing technology for real-time, high-performance, accessible and scalable analysis of DNA and RNA. The technology is used in more than 120 countries to understand the biology of humans and diseases such as cancer, plants, animals, bacteria, viruses and whole environments. Oxford Nanopore Technologies products are intended for molecular biology applications and are not intended for diagnostic purposes.

For more, visit: https://nanoporetech.com/

About ViruSure

With over 20 years of experience, ViruSure is a leading global Contract Research Organization specializing in virus and prion biosafety testing for the life sciences industry. Located in Vienna, Austria, ViruSure offers analytical testing solutions, virus and prion validation services covering a broad portfolio of tests necessary to support drug development and ensure the safety of biopharmaceuticals, cell & gene therapies, and vaccines. ViruSure is integral to the Biosafety Testing Services Unit of Asahi Kasei Bioprocess. To learn more about ViruSure, visit www.virusure.com.

Contacts

Oxford Nanopore
media@nanoporetech.com

Operio Group Named Distributor for Schaefer Technologies in the United Kingdom and European Union




Operio Group Named Distributor for Schaefer Technologies in the United Kingdom and European Union

FORT WORTH, Texas–(BUSINESS WIRE)–Operio Group has signed a distribution agreement with Schaefer Technologies, a manufacturer of semi-automatic encapsulation equipment. Operio Group, a holding company building a global group of brands serving the solid dose manufacturing industry, will be the official distributor of Schaefer Technologies’ products in the United Kingdom and the European Union.

Schaefer Technologies develops semi-automatic capsule filling systems, including equipment that produces banded capsules for liquid and pellet formulations used by nutraceutical and pharmaceutical manufacturers.

Leadership from both companies worked together to establish the agreement, including Kevin Schaefer, CEO of Schaefer Technologies Inc., and Alastair Sanderson, Chief Business Development Officer at Operio Group.

“Schaefer Technologies is looking forward to our new relationship with Operio Group,” said Schaefer. “Their understanding of the pharmaceutical equipment market makes them a strong partner as we combine our expertise in capsule filling with their commitment to product development and customer service. We also extend our appreciation to Garry and Suzanne Coleman at Glenvale Packaging for their partnership over the past 29 years. As our representative in Europe since 1997, Glenvale helped establish Schaefer Technologies’ presence across the European market. We wish Garry and Suzanne the very best in their retirement.”

“We’re very proud of our partnership with Schaefer Technologies,” said Declan McLaughlin, CEO of Operio Group. “At Operio Group, our mission is to become the number one world-class provider of manufacturing, service, and support for capsule fillers and tablet presses serving the pharmaceutical and nutraceutical industries. This partnership advances that, and we’re committed to delivering the world-class service and support customers expect from the Schaefer Technologies brand.”

“It’s an honor to work with Schaefer Technologies,” said Sanderson. “Operio Group has long recognized the need for semi-automatic capsule filling solutions within our portfolio. Schaefer’s equipment allows us to better support manufacturers working with powders, pellets, and liquid-filled capsules.”

Through this partnership, Operio Group will support sales and distribution of Schaefer Technologies’ encapsulation systems across the United Kingdom and the European Union, strengthening Operio Group’s capsule manufacturing equipment portfolio.

About Operio Group

Operio Group is a collective of companies providing equipment, tooling, ingredients, and technical expertise to the nutraceutical, pharmaceutical, and solid dose manufacturing industries.

For more information visit: www.operiogroup.com

About Schaefer Technologies Inc.

Schaefer Technologies Inc. manufactures semi-automatic capsule filling equipment, including systems designed for banded capsules used in liquid and pellet formulations.

For more information visit: www.schaefertechnologies.com

Contacts

Whittney Hines

Operio Group
682-312-0034

Bristol Myers Squibb Announces Positive Phase 3 Results from the SUCCESSOR-2 Study of Oral Mezigdomide in Relapsed or Refractory Multiple Myeloma




Bristol Myers Squibb Announces Positive Phase 3 Results from the SUCCESSOR-2 Study of Oral Mezigdomide in Relapsed or Refractory Multiple Myeloma

Study marks the first positive Phase 3 study for mezigdomide and the second positive Phase 3 study for the Bristol Myers Squibb CELMoD program

PRINCETON, N.J.–(BUSINESS WIRE)–$BMY #CELMoD—Bristol Myers Squibb (NYSE: BMY) today announced positive interim Phase 3 results from the SUCCESSOR-2 study (NCT05552976). In the trial, oral mezigdomide in combination with carfilzomib and dexamethasone (MeziKd) demonstrated statistically significant and clinically meaningful improvement in progression-free survival (PFS) versus carfilzomib and dexamethasone alone (Kd) in patients with relapsed or refractory multiple myeloma (RRMM). Safety findings were consistent with the known profile of mezigdomide and the combination regimen. Patients will continue to be followed for survival and safety.

“We are excited by these results, which underscore Bristol Myers Squibb’s leadership in treating multiple myeloma and our unwavering commitment to patients living with this persistent and challenging disease,” said Cristian Massacesi, executive vice president, chief medical officer and head of development at Bristol Myers Squibb. “Importantly, these findings reinforce the value of our CELMoD program and our targeted protein degradation platform, and strengthen our confidence in bringing forward effective, accessible oral treatment options for patients with difficult-to-treat blood cancers and potentially beyond.”

“While treatment advances have been meaningful, far too many patients with multiple myeloma still relapse or stop responding—making the need for new options urgent,” said Paul Richardson, MD, Director of Clinical Research and Clinical Program Leader at the Jerome Lipper Multiple Myeloma Center, the Dana-Farber Cancer Institute and RJ Corman Professor of Medicine, Harvard Medical School. “These data underscore the potential of MeziKd as an oral regimen that could address a key unmet need for patients previously exposed to anti-CD38 and lenalidomide.”

“It is important for patients to have treatment options that offer enduring disease control,” said Meletios-A. (Thanos) Dimopoulos, MD, Professor and Chairman, Department of Clinical Therapeutics at Alexandra Hospital, School of Medicine, National and Kapodistrian University of Athens. “These positive interim data show that adding mezigdomide, a CELMoD specifically optimized for enhanced myeloma cell killing and immune activation compared with IMiD agents, to carfilzomib and dexamethasone may provide clinical benefit in earlier relapse.”

Data from SUCCESSOR-2 will be presented at a future medical meeting and results will be shared with health authorities.

About SUCCESSOR-2

SUCCESSOR-2 (NCT05552976) is an inferential, seamless Phase 2/3, multicenter, randomized, open-label study evaluating the efficacy and safety of mezigdomide in combination with carfilzomib and dexamethasone (MeziKd) versus carfilzomib and dexamethasone (Kd) in patients with relapsed or refractory multiple myeloma (RRMM).

The primary endpoint of the Phase 3 portion is progression-free survival (PFS). Key secondary endpoints include overall survival (OS), overall response rate (ORR), duration of response (DoR), time to progression (TTP), time to next treatment (TTNT), minimal residual disease (MRD) negativity, and health-related quality of life (HR-QoL).

About Targeted Protein Degradation and Novel CELMoD Agents

Targeted protein degradation (TPD) is a differentiated research platform at Bristol Myers Squibb built on more than two decades of scientific expertise, providing new avenues to degrade therapeutically relevant proteins that were previously considered “undruggable.” BMS is the only company that has successfully developed and commercialized protein degrader agents for the treatment of multiple myeloma. These agents, known as immunomodulatory drugs (IMiDs), helped establish the current standard of care in the treatment of this disease, which remains without a cure. BMS is building on this foundation with several investigational protein degraders in clinical trials, leveraging three different modalities including CELMoD agents, ligand-directed degraders (LDDs), and degrader antibody conjugates (DACs). This three-pronged approach enables matching the right therapeutic modality to a molecular mechanism of action to modulate targets most effectively and ultimately provide more opportunities for potential breakthroughs that may offer meaningful new options for patients across a broad range of diseases, in and beyond hematology and oncology. Learn more about the science behind TPD at Bristol Myers Squibb here.

About Bristol Myers Squibb

Bristol Myers Squibb is a global biopharmaceutical company whose mission is to discover, develop and deliver innovative medicines that help patients prevail over serious diseases. For more information about Bristol Myers Squibb, visit us at BMS.com or follow us on LinkedIn, X, YouTube, Facebook and Instagram.

Cautionary Statement Regarding Forward-Looking Statements

This press release contains “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995 regarding, among other things, the research, development and commercialization of pharmaceutical products. All statements that are not statements of historical facts are, or may be deemed to be, forward-looking statements. Such forward-looking statements are based on current expectations and projections about our future financial results, goals, plans and objectives and involve inherent risks, assumptions and uncertainties, including internal or external factors that could delay, divert or change any of them in the next several years, that are difficult to predict, may be beyond our control and could cause our future financial results, goals, plans and objectives to differ materially from those expressed in, or implied by, the statements. These risks, assumptions, uncertainties and other factors include, among others, that future study results may not be consistent with the results to date, and that mezigdomide in combination with carfilzomib and dexamethasone (MeziKd) may not receive regulatory approval for the indication described in this release in the currently anticipated timeline or at all, any marketing approvals, if granted, may have significant limitations on their use, and, if approved, whether such combination treatment for such indication will be commercially successful. No forward-looking statement can be guaranteed. Forward-looking statements in this press release should be evaluated together with the many risks and uncertainties that affect Bristol Myers Squibb’s business and market, particularly those identified in the cautionary statement and risk factors discussion in Bristol Myers Squibb’s Annual Report on Form 10-K for the year ended December 31, 2024, as updated by our subsequent Quarterly Reports on Form 10-Q, Current Reports on Form 8-K and other filings with the Securities and Exchange Commission. The forward-looking statements included in this document are made only as of the date of this document and except as otherwise required by applicable law, Bristol Myers Squibb undertakes no obligation to publicly update or revise any forward-looking statement, whether as a result of new information, future events, changed circumstances or otherwise.

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Contacts

Bristol Myers Squibb

Media Inquiries:

media@bms.com

Investors:

investor.relations@bms.com

Pfizer’s Phase 2 Study of Trispecific Antibody Positive in Moderate to Severe Atopic Dermatitis




Pfizer’s Phase 2 Study of Trispecific Antibody Positive in Moderate to Severe Atopic Dermatitis

• Study met primary endpoint, demonstrating statistically significant increase in the percentage of participants achieving EASI-75 at Week 16 across all doses tested, compared to placebo
• Tilrekimig (PF-07275315) was well-tolerated with a favorable safety profile
• Tilrekimig has the potential to be a first-in-class, once-monthly trispecific antibody targeting interleukin-4 (IL-4), interleukin-13 (IL-13), and thymic stromal lymphopoietin (TSLP) for multiple chronic Type 2 (Th2) inflammatory conditions including atopic dermatitis, asthma, and chronic obstructive pulmonary disease (COPD)
• Based on encouraging results, Pfizer plans to accelerate tilrekimig to Phase 3 development, with a pivotal study in atopic dermatitis on track to start this year

NEW YORK–(BUSINESS WIRE)–Pfizer Inc. (NYSE: PFE) today announced positive topline results from a Phase 2 study investigating tilrekimig (PF-07275315) in adults with moderate to severe atopic dermatitis. The study met its primary efficacy endpoint, demonstrating a statistically significant increase in the percentage of participants achieving EASI-75* (≥ 75% reduction in the Eczema Area and Severity Index) at Week 16, compared to placebo. In Stage 2 of the study, which evaluated monthly dosing regimens, tilrekimig showed competitive efficacy. The placebo-adjusted percentage of participants achieving EASI-75 at Week 16 was:

• 38.7% for the low tested dose;
• 51.9% for the middle tested dose; and
• 49.4% for the high tested dose.

The two highest dose levels tested with tilrekimig strongly suggest potentially meaningful improvements to approved standard of care biologics. Tilrekimig is an investigational trispecific antibody that simultaneously targets interleukin-4 (IL-4), interleukin-13 (IL-13), and thymic stromal lymphopoietin (TSLP), with the potential to be a once-monthly treatment option for multiple chronic inflammatory conditions driven by an overactive Type 2 (Th2) immune response, without affecting receptors on healthy cells.

“We are encouraged by the topline Phase 2 results for tilrekimig, which show that combining the potent inhibition of IL-4/13 and TSLP pathways has the potential to deliver improved efficacy over the standard of care for atopic dermatitis,” said Mike Vincent, Chief Inflammation & Immunology Officer at Pfizer. “We plan to advance a broad clinical development program for tilrekimig, a potential first-in-class trispecific antibody discovered at Pfizer, in atopic dermatitis and other Th2-mediated inflammatory diseases including asthma and COPD.”

Tilrekimig was well-tolerated with a favorable safety profile and no dose dependent safety signals; adverse event (AE) rates were comparable to placebo. The most common AEs were infections and infestations, skin and subcutaneous tissue disorders and general disorders, and administration site reactions. Three serious adverse events (SAEs) were observed, which were all considered to be unrelated to treatment. Of note, the observed frequency of conjunctivitis in this study was lower than rates reported with IL-4 receptor alpha inhibitors.

The Phase 2 study is an ongoing randomized, double-blind, placebo-controlled trial in adults with moderate to severe atopic dermatitis. It is being conducted in four overlapping stages:

• Stage 1 tested a high dose of tilrekimig versus placebo. In addition, Stage 1 included an arm testing a high dose of ompekimig (PF-07264660), an investigational trispecific antibody targeting IL-4, IL-13, and interleukin-33 (IL-33), versus placebo. Stage 1 has concluded and both tilrekimig and ompekimig groups met the primary endpoint.
• Stage 2 was a dose-ranging study in which participants received either tilrekimig or placebo.
• Stage 3 is an ongoing study in which participants who previously received biologic treatments receive either tilrekimig or placebo.
• Stage 4 is an ongoing dose-ranging study in which participants receive either ompekimig or placebo.

In addition to the ongoing Phase 2 study in atopic dermatitis, Pfizer is studying tilrekimig in an ongoing Phase 2 study in asthma and the company recently initiated a Phase 2b/3 study of tilrekimig in chronic obstructive pulmonary disease (COPD). Phase 3 planning for atopic dermatitis is ongoing, with a pivotal study on track to start this year.

Detailed results from the Phase 2 study of tilrekimig will be submitted to a future medical meeting and a peer-reviewed journal. Pfizer plans to share results from the ongoing portions of the study in the future, pending completion.

About Atopic Dermatitis

Atopic dermatitis is more than “just a rash.” It is a chronic Type 2 (Th2) inflammatory skin condition, affecting people of all ages and genders around the world.^i,ii Atopic dermatitis is the most common form of eczema, and sometimes the terms are used interchangeably.ⁱⁱⁱ The most frequent symptom of atopic dermatitis is itchy skin, which can lead to rashes, pain, and poor sleep.ⁱⁱⁱ The condition can be debilitating, disrupting patients’ daily lives, and negatively affecting their emotional well-being. Many patients do not experience a clinically meaningful response to currently available treatments, signaling a critical need for further innovation in this class of medicines.

About Pfizer: Breakthroughs That Change Patients’ Lives

At Pfizer, we apply science and our global resources to bring therapies to people that extend and significantly improve their lives. We strive to set the standard for quality, safety and value in the discovery, development and manufacture of health care products, including innovative medicines and vaccines. Every day, Pfizer colleagues work across developed and emerging markets to advance wellness, prevention, treatments and cures that challenge the most feared diseases of our time. Consistent with our responsibility as one of the world’s premier innovative biopharmaceutical companies, we collaborate with health care providers, governments and local communities to support and expand access to reliable, affordable health care around the world. For 175 years, we have worked to make a difference for all who rely on us. We routinely post information that may be important to investors on our website at www.Pfizer.com. In addition, to learn more, please visit us on www.Pfizer.com and follow us on X at @Pfizer and @Pfizer News, LinkedIn, YouTube and like us on Facebook at Facebook.com/Pfizer.

Disclosure Notice

The information contained in this release is as of March 9, 2026. Pfizer assumes no obligation to update forward-looking statements contained in this release as the result of new information or future events or developments.

This release contains forward-looking information about tilrekimig (PF-07275315), an investigational trispecific antibody, including its potential benefits; results from the Phase 2 study of tilrekimig in participants with moderate to severe atopic dermatitis; Pfizer’s investigational inflammation and immunology portfolio, and anticipated trial starts and clinical development plans, including plans to accelerate tilrekimig to Phase 3 development, with a pivotal study in atopic dermatitis planned to start this year, as well as their potential benefits, that involves substantial risks and uncertainties that could cause actual results to differ materially from those expressed or implied by such statements. Risks and uncertainties include, among other things, the uncertainties inherent in research and development, including the ability to meet anticipated clinical endpoints, commencement and/or completion dates for our clinical trials, regulatory submission dates, regulatory approval dates and/or launch dates, as well as the possibility of unfavorable new clinical data and further analyses of existing clinical data; risks associated with initial, preliminary or interim data; the risk that clinical trial data are subject to differing interpretations and assessments by regulatory authorities; whether regulatory authorities will be satisfied with the design of and results from the clinical studies; whether and when drug applications may be filed in any jurisdictions for tilrekimig or any other product candidates for any potential indications; whether and when any such applications may be approved by regulatory authorities, which will depend on myriad factors, including making a determination as to whether the product’s benefits outweigh its known risks and determination of the product’s efficacy and, if approved, whether tilrekimig or any such other product candidates will be commercially successful; decisions by regulatory authorities impacting labeling, manufacturing processes, safety and/or other matters that could affect the availability or commercial potential of tilrekimig or any such other product candidates; risks and uncertainties related to issued or future executive orders or other new, or changes in, laws or regulations; uncertainties regarding the impact of COVID-19 on Pfizer’s business, operations and financial results; and competitive developments.

A further description of risks and uncertainties can be found in Pfizer’s Annual Report on Form 10-K for the fiscal year ended December 31, 2025, and in its subsequent reports on Form 10-Q, including in the sections thereof captioned “Risk Factors” and “Forward-Looking Information and Factors That May Affect Future Results”, as well as in its subsequent reports on Form 8-K, all of which are filed with the U.S. Securities and Exchange Commission and available at www.sec.gov and www.pfizer.com.

*EASI-75 is the industry standard for measuring how effectively a treatment candidate reduces atopic dermatitis (eczema) severity and extent from baseline

ⁱ Laughter MR, Maymone MBC, Mashayekhi S, et al. The global burden of atopic dermatitis: lessons from the Global Burden of Disease Study 1990–2017*. British Journal of Dermatology. 2021;184(2):304-309. doi:10.1111/bjd.19580

ⁱⁱ Choragudi S, Yosipovitch G. Trends in the Prevalence of Eczema Among US Children by Age, Sex, Race, and Ethnicity From 1997 to 2018. JAMA Dermatol. 2023;159(4):454-456.

ⁱⁱⁱ Eczema (contact dermatitis): Symptoms, causes, & treatment | National Eczema Association

Contacts

Media Contact:

PfizerMediaRelations@Pfizer.com

Investors Contact:

IR@Pfizer.com

Atamyo Therapeutics Presents Promising Results in the First Patients Treated With Its ATA-200 Gene Therapy in the Clinical Trial Targeting LGMD-R5 Limb-girdle Muscular Dystrophy




Atamyo Therapeutics Presents Promising Results in the First Patients Treated With Its ATA-200 Gene Therapy in the Clinical Trial Targeting LGMD-R5 Limb-girdle Muscular Dystrophy

• Four patients with LGMD-R5 (gamma-sarcoglycanopathy, formerly LGMD-2C) have received the ATA-200 gene therapy as part of the ongoing Phase 1b/2 trial evaluating the safety, pharmacodynamics, and efficacy of ATA-200.
• Atamyo Therapeutics’ clinical trial is currently being conducted at the Powell Gene Therapy Center, University of Florida, with Dr. Barry Byrne, MD, PhD, as the principal investigator.
• Nine-month follow-up data (including muscle biopsies performed at six months) for the first two patients treated have been reported: the safety of the product has been demonstrated and the efficacy results for these first two patients treated are very encouraging.

EVRY, France–(BUSINESS WIRE)–Atamyo Therapeutics, a biotechnology company specializing in the development of next-generation gene therapies for limb-girdle muscular dystrophy (LGMD), announced at the MDA (Muscular Dystrophy Association) Conference 2026 the first safety, pharmacodynamics, and efficacy results for its ATA-200 gene therapy in LGMD-2C/R5 limb-girdle muscular dystrophy associated with γ-sarcoglycan deficiency (SGCG, gamma-sarcoglycanopathy). The results are from the first patients treated in the clinical trial conducted at the Powell Gene Therapy Center at the University of Florida by Dr. Barry Byrne and supported by The Dion Foundation for Children with Rare Diseases.

LGMD-2C/R5 is a severe form of muscular dystrophy that appears in childhood and causes loss of walking ability before adulthood, respiratory and heart failure, and premature death. This Phase 1b/2 clinical trial (NCT05973630) is a single-center study evaluating the safety, pharmacodynamics, efficacy, and immunogenicity of ATA-200 in children aged 6 to 13 years. ATA-200 is an adeno-associated virus (AAV) gene therapy carrying a normal copy of the human SGCG gene and administered as a single intravenous injection at a dose of 1.0E+14 vg/kg. This gene therapy product was developed by Isabelle Richard, a pioneer in the study of limb-girdle muscular dystrophies and the development of innovative therapies at Genethon.

At a dose of 1.0E+14 vg/kg, the following was observed in the first two patients treated with ATA-200:

• More than 90% of muscle fibers expressing the SGCG protein—demonstrating that almost all muscle fibers had received the therapeutic gene (90.2% for patient 1 and 92.1% for patient 2, biopsies at 6 months).

• A significant and sustained reduction in CPK levels (a biomarker of muscle damage) and a decrease in transaminases 9 months after treatment, demonstrating the significant efficacy of ATA-200 gene therapy.

• Also 9 months post-treatment, clinical benefits were observed on several other important parameters in ambulatory patients, particularly in timed functional tests.

No serious side effects were observed in the four patients treated, confirming the safety of the product.

“These initial results are very encouraging and demonstrate the potential of our product with biological data rarely seen in neuromuscular diseases and at such an early stage of the trial. I would like to commend the quality of the work done by the teams at Atamyo Therapeutics and, in particular, the commitment and determination of Isabelle Richard, which has made it possible to offer this hope to patients and their families. We are deeply grateful for the collaboration with the Dion Foundation and Dr. Barry Byrne of the Powell Gene Therapy Center and proud to offer children with LGMD-R5 the opportunity to receive a treatment that could change their lives.” — Angela Columbano, CEO, Atamyo Therapeutics.

Further results from this ongoing study are expected to be published in the coming months, when new longer-term follow-up data becomes available.

We are grateful to the Dion Foundation for Children with Rare Diseases and CureSCG for their financial contributions that made it possible to launch the trial.

About the ATA-200 program in LGMD-2C/R5

About the ATA-200 program in LGMD-2C/R5 LGMD-2C/R5 is a rare genetic disease caused by mutations in the gene that produces γsarcoglycan, a transmembrane protein that is involved in the connection between muscle fibers and their environment. It affects an estimated 2,000 people in Europe and in the US. In the classical form, symptoms appear in early childhood and patients suffer from progressive muscular weakness leading to loss of ambulation before adulthood. Cardiac involvement, typically presented as dilated cardiomyopathy, is reported in approximately half of patients and will eventually impact life expectancy. There is no curative treatment. The management of LGMD-2C/R5 is solely supportive. ATA-200, Atamyo’s gene therapy for LGMD-2C/R5, delivers a normal copy of the gene for production of γ-sarcoglycan. In preclinical mice models, ATA-200 demonstrated its tolerability and capability to correct symptoms and biomarkers of the pathology.

ATA-200 has been awarded Orphan Drug Designation in the US and Europe, and Rare Pediatric Disease Designation by the US FDA.

The therapy is based on the research of Atamyo Chief Scientific Officer Isabelle Richard, Ph.D., Research Director at CNRS who heads the Progressive Muscular Dystrophies Laboratory at Genethon.

About Atamyo Therapeutics

Atamyo Therapeutics is a clinical-stage biopharma focused on the development of a new generation of effective and safe gene therapies for neuromuscular diseases. A spin-off of gene therapy pioneer Genethon, Atamyo leverages unique expertise in AAV-based gene therapy and muscular dystrophies from the Progressive Muscular Dystrophies Laboratory at Genethon. Atamyo’s most advanced programs address different forms of limb-girdle muscular dystrophies (LGMD), with two clinical-stage programs targeting respectively LGMD-R9 and LGMD-R5. The name of the company is derived from two words: Celtic Atao which means “Always” or “Forever” and Myo which is the Greek root for muscle. Atamyo conveys the spirit of its commitment to improve the life of patients affected by neuromuscular diseases with life-long efficient treatments.

For more information visit www.atamyo.com

Contacts

US & Europe contacts: contact@atamyo.com  

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

Daniel Eramian – Opus Biotech Communications – danieleramian@comcast.net

Abselion Establishes US Subsidiary in Cambridge, MA




Abselion Establishes US Subsidiary in Cambridge, MA

Supports closer engagement with scientists in North America, to meet growing international adoption of its Amperia protein quantification platform

CAMBRIDGE, United Kingdom & CAMBRIDGE, Mass.–(BUSINESS WIRE)–#Abselion–Abselion, a pioneering life sciences technology company focused on simplifying biomolecule quantification, today announced that it has established a US subsidiary at The Engine in Cambridge, MA. The new location strengthens its presence in the North American life sciences ecosystem, providing a formal base to support more direct engagement with academic, biotech, and pharma research teams working across biologics characterisation and development workflows.

The US site will provide Abselion with an organisational framework to complement its established collaborations and support its next stage of development as interest in its Amperia™ protein quantification system extends beyond the UK and Europe. The Engine, built by the Massachusetts Institute of Technology (MIT), is a Tough Tech incubator and accelerator and a strategic US hub for life sciences, biotech, and other transformational technologies. It provides specialised infrastructure, deep technical collaboration, and long-term company building support. Aligning Abselion’s organisational structure with the geographic distribution of its customer base will allow the company to plan and coordinate its North American activities more effectively. This will help to support system introduction, ongoing use, and collaboration in the region, while the company continues to build on its scientific and operational foundations in the UK. Expanding the company’s global footprint will also help ensure the appropriate structures are in place to support future international growth.

Dr Ruizhi Wang, CEO and Founder, Abselion, said: “Our vision is to make high-quality quantification more accessible through closer international collaboration. Establishing a US subsidiary marks an important milestone in Abselion’s growth, strengthening our ability to operate across key life science markets globally while continuing to build on our strong scientific foundation.

The US represents a significant focus for advanced biologics research and development. A formal presence there demonstrates our commitment to supporting long-term adoption of our protein quantification systems and will allow our growing international team to work more closely with customers and partners as they explore and build workflows around our Amperia platform.”

For further information about Abselion’s US site and it’s Amperia platform, please visit: https://www.abselion.com/ or meet the team at BPI West from 9–11 March in San Diego, CA to learn more.

Contacts

Codon Communications
Dr Michelle Ricketts

Tel: +447789053885

Email: michelle.ricketts@codoncommunications.com