Levicept to Present Further Analysis from Phase II Trial of Novel Neurotrophin-3 Inhibitor, LEVI-04, at EULAR

Levicept to Present Further Analysis from Phase II Trial of Novel Neurotrophin-3 Inhibitor, LEVI-04, at EULAR




Levicept to Present Further Analysis from Phase II Trial of Novel Neurotrophin-3 Inhibitor, LEVI-04, at EULAR

  • Significant and clinically meaningful improvement in pain, function, and other outcomes in people with knee osteoarthritis
  • Rapid onset of action observed
  • Improvement in function out of proportion to pain-relieving effects
  • First-in-class novel biologic under development as non-addictive pain treatment

SANDWICH, United Kingdom, June 12, 2025 (GLOBE NEWSWIRE) — Levicept Ltd, a biotechnology company focused on the development of LEVI-04, a first-in-class treatment for osteoarthritis, is to present new data from its large-scale Phase II trial of LEVI-04 at EULAR, the European Congress of Rheumatology in Barcelona, Spain.

LEVI-04 is a proprietary p75 neurotrophin receptor fusion protein (p75NTR-Fc) that provides analgesia via inhibition of NT-3 activity, supplementing the endogenous p75NTR binding protein and modulating excess neurotrophin levels present in osteoarthritis.

The data are from Levicept’s multiarm, multicentre, randomized, double-blind, placebo-controlled, Phase II study which enrolled 518 participants with pain and disability due to osteoarthritis of the knee (ClinicalTrials.gov ID: NCT05618782). Previously presented data showed the primary efficacy endpoint of the trial was met with significant analgesia across all doses.

The further analyses for the first time show the effect sizes associated with LEVI-04 treatment and that significant and clinically meaningful improvements were observed versus placebo across a wide range of pain, function, and other outcome measures. The data also show that improvement in pain was observed after three days. Professor Philip Conaghan MBBS PhD, Director NIHR Leeds Biomedical Research Centre, Principal Investigator will present the findings in an oral presentation on June 14.

Levicept has also presented data at the conference showing that LEVI-04’s effect on physical function on both subjective and performance-based measures was disproportionately higher than expected from the reduction in pain it provides, in comparison to other treatments for knee osteoarthritis. This presentation included data from a novel model of evoked pain on movement (StEPPi) which was shown to correlate well with other assessments typically used in OA trials, and where LEVI-04 performed significantly better than placebo.

Professor Philip Conaghan MBBS PhD, Director NIHR Leeds Biomedical Research Centre, Principal Investigator said, “These new data we are presenting at EULAR further support our belief that LEVI-04 has the potential to offer a vital new treatment option to millions of patients in need. The results suggest that LEVI-04 meaningfully improves physical function and pain in people with knee osteoarthritis, with rapid onset of action, vitally important treatment characteristics, and furthermore that LEVI-04 has an impact on function greater than would be expected from its impact on pain alone.”

Eliot Forster, CEO of Levicept, said, “We continue to see significant interest in the results from our Phase II study at the high calibre international medical conferences, underlining their importance, and are excited to present these additional analyses here. We are confident that LEVI-04 could represent a genuine, and much needed, breakthrough in the treatment of osteoarthritis. We continue to advance our strategy for LEVI-04’s further development and for ensuring the best opportunity for it to reach the many patients who may benefit.”

Presentation details:

Clinical Meaningfulness of Pain Improvements and Time to Onset of Pain Relief of a Novel OA Therapy: Analyses from a Randomised Controlled Phase 2 Trial with LEVI-04, a Novel Neurotrophin-3 Inhibitor

Oral plenary presentation (OP0374) – Saturday, 14 June, 13:00 – 13:10 CEST

Authors: Philip G. Conaghan, Nathaniel Katz, Asger R. Bihlet, Laus Wullum, Kerry af Forselles, Michael Perkins, Bernadette Hughes, Claire Herholdt, Iwona Bombelka, Simon Westbrook

LEVI-04, a Novel Neurotrophin-3 Inhibitor, Improves Physical Function Out of Proportion to its Pain-Relieving Effects

Poster presentation (POS0556) Wednesday, 11 June, 15:30 – 16:30 CEST

Nathaniel Katz, Simon Westbrook, Asger R. Bihlet, Laus Wullum, Kerry af Forselles, Michael Perkins, Bernadette Hughes, Claire Herholdt, Iwona Bombelka, Philip G. Conaghan

Levicept

Eliot Forster, CEO – eliot@levicept.com

Media Enquiries

Charles Consultants

Sue Charles – Sue@charles-consultants.com +44 (0)7968 726585

Chris Gardner – Chris@CGComms.onmicrosoft.com +44 (0)7956 031077

About Levicept – www.levicept.com

Levicept Ltd is a UK-based biotechnology company developing the first in a new class of novel, safe and efficacious biological therapies, LEVI-04 [p75NTR-Fc], for the treatment of osteoarthritis and chronic pain. LEVI-04 inhibits NT-3, one of the neurotrophin family of proteins. LEVI-04 has completed a Phase II clinical trial in more than 500 patients with osteoarthritis. It is estimated that the market opportunity for drugs that treat osteoarthritis is worth in excess of $10 billion. LEVI-04 was discovered by Levicept’s founder, Simon Westbrook. Levicept’s investors include Medicxi, Advent Life Sciences, Gilde Healthcare and Pfizer Ventures.

Follow us on LinkedIn – https://www.linkedin.com/company/levicept-ltd

_______________________________
i StEPP – Staircase Evoked Pain Procedure – patients walk up and down a single stair 24 times in a standardised protocol, rating their pain intensity at the end (performance-based measure with no recall)

Enterome raises $19 million to fund clinical development of its OncoMimics™ immunotherapy to treat Follicular Lymphoma

Enterome raises $19 million to fund clinical development of its OncoMimics™ immunotherapy to treat Follicular Lymphoma




Enterome raises $19 million to fund clinical development of its OncoMimics™ immunotherapy to treat Follicular Lymphoma

  • Proceeds to fund Phase 1/2 trial of EO2463 OncoMimics™, as a monotherapy or in combination, to treat multiple forms of indolent non-Hodgkin lymphoma (iNHL)
  • $9 million from new investor The Institute for Follicular Lymphoma Innovation (IFLI)  
  • $10 million from existing specialist investors, including The Leukemia & Lymphoma Society Therapy Acceleration Program® (LLS TAP)  

Paris, France – June 12, 2025

Enterome SA
, a clinical-stage company developing first-in-class OncoMimics™ immunotherapies to treat cancer, has raised $19 million in a new private financing to advance its lead clinical program EO2463 OncoMimics™ immunotherapy to treat indolent non-Hodgkin lymphoma (iNHL). The new funds will be used to expand and finalize the ongoing Phase 1/2 SIDNEY clinical trial of EO2463 and prepare the candidate for a registrational trial.

New U.S. investor The Institute for Follicular Lymphoma Innovation (IFLI), a global non-profit foundation dedicated to advancing research and treatment for follicular lymphoma, invested $9 million in the round, of which $5 million will be allocated to Enterome upon closing and an additional $4 million in conditional tranched funding. 

Existing shareholders invested an additional $10 million including: SymBiosis, a U.S. venture capital firm; Seventure Partners, based in France; Lundbeckfonden BioCapital from Denmark; Primo Capital, an Italian venture capital and private equity firm; and The U.S. Leukemia & Lymphoma Society Therapy Acceleration Program® (LLS TAP).

“Attracting highly specialized blood cancer investor IFLI to this financing demonstrates the conviction of our new and existing investors in the potential of OncoMimics™ for blood and solid tumor cancers,” said Pierre Bélichard, Enterome’s Chief Executive Officer. “We currently are generating exciting clinical proof of concept data for EO2463 monotherapy in several iNHL patient populations included in the Phase 1/2 SIDNEY clinical trial. Most importantly, EO2463 has shown robust clinical efficacy and exceptional safety and tolerability – which is especially impressive for such a potent immunotherapy. This offers a new hope for these patients and a rare opportunity to create an entirely new market segment for an impactful therapeutic. This financing will enable us to continue the SIDNEY trial of EO2463 and prepare to launch a first pivotal Phase 3 trial of this candidate for the ‘watch-and-wait’ iNHL population.”

The company presented interim SIDNEY dataat the American Society of Hematology (ASH) conference in December 2024, showing highly encouraging responses in the Cohort 2 of “watch and wait” iNHL patients in the ongoing SIDNEY study. This population, as the name suggests, is generally not eligible to receive other treatments due to the unacceptable risk-benefit ratio (in this iNHL sub-population) of the most commonly used blood cancer therapies. 

The company also recently disclosed having held positive meetings with both FDA (Type C meeting) and EMA (Scientific Advice), outlining a clear regulatory path registration for marketing authorizations in “watch-and-wait” iNHL. 

“This investment aligns with IFLI’s mission to accelerate the development of innovative therapies and precision biomarkers for follicular lymphoma,” said Michel Azoulay, MD, Chief Medical Officer at IFLI. “EO2463 represents a novel class of synthetic, off-the-shelf Immunotherapeutics with a unique mechanism of action that selectively targets malignant B cells. We are particularly interested in supporting Enterome’s efforts to demonstrate EO2463’s clinical efficacy across multiple lines of therapy, including in relapsed and refractory settings.”

Enterome recently announced that it will present new data showing EO2463 also has a meaningful impact when tested in combination with standard of care in relapsed and refractory iNHL patients at the International Conference on Malignant Lymphoma (ICML) in Lugano on June 21. Previous findings presented at ASCO in 2024 in the relapsed and refractory patient population further suggested the potential to identify individuals most likely to benefit from EO2463 treatment, supported by biomarker analyses. 

Lore Gruenbaum, Chief Scientific Officer at LLS, said, “It is important for us to continue to support Enterome, a company working to develop novel therapeutics based on our shared commitment to create better therapies for blood cancers. LLS has invested over $1.8 billion in groundbreaking research since our inception in 1949. Our active partnership with Enterome, through our Therapy Acceleration Program, will continue to advance the clinical development of the OncoMimics™ family of novel immunotherapeutics for the benefit of blood cancer patients. We are particularly excited to help advance EO2463 which has shown promising signs of efficacy as monotherapy with excellent safety and tolerability in ‘watch-and-wait’ iNHL patients, who currently have no approved treatment options.” 

EO2463 is an innovative, off-the-shelf immunotherapy candidate that combines four synthetic OncoMimics™ peptides. These non-self, microbial-derived peptides correspond to CD8 HLA-A2 epitopes that mimic the B lymphocyte-specific lineage markers CD20, CD22, CD37, and CD268 (BAFF receptor). EO2463 also includes the helper peptide (CD4+ epitope) universal cancer peptide 2 (UCP2). The unique ability of EO2463 immunotherapy to selectively target multiple B cell markers enables the destruction of malignant B lymphocytes. By ensuring broad target coverage across malignant B cells, this novel approach aims to simultaneously improve safety and maximize efficacy, reducing the tumor cells’ capacity to develop immune-resistance mechanisms such as antigen escape.

SIDNEY is an ongoing 12-month open label Phase 1/2 study that aims to assess safety, tolerability, immunogenicity, and preliminary efficacy of EO2463 monotherapy and combination therapy in up to about 55 patients with follicular lymphoma and marginal zone lymphoma including divided into three cohorts:  newly diagnosed patients eligible to watch-and-wait (monotherapy); newly diagnosed patients in need of therapy / first line (combo with rituximab); patients with relapsed/refractory disease (combo with R2). In addition to safety, survival, response rates and other measures of efficacy are being collected.

OncoMimics™ were inspired by the microbial origin of certain autoimmune diseases. The Company uses AI and machine learning to identify microbial proteins that closely mimic the structure, effect or actions of specific cancer antigens (as well as hormones or cytokines). Memory T cells against microbial antigen are created during early development, sometimes leading to autoimmune disorders. In the case of OncoMimics™, however, this means that the immune system can mount a rapid, robust and durable immune response that is highly targeted and specific for the OncoMimics™ and the cancer antigens they closely resemble.

This is possible because, unlike cancer antigens, OncoMimics™ bypass the biological process, known as thymic deletion, that prevents the immune system from mounting an attack against the “self” proteins (e.g. antigen) on tumor and blood cancer cells. Once activated, the immune system attacks with high specificity and potency the cancer antigens targeted by the OncoMimics™, killing the cancer cells that carry them. OncoMimics™ are synthetic peptides that are easy to manufacture, store, distribute and administer as an off-the-shelf subcutaneous injection. In clinical testing to date they have been shown to be extremely well tolerated, especially compared to other potent immunotherapies.

Enterome SA (www.enterome.com) is a privately held clinical-stage biopharmaceutical company developing OncoMimics™, a new proprietary immunotherapeutic modality inspired by the microbial origin of certain autoimmune diseases, to treat cancer. The Company’s wholly-owned OncoMimics pipeline includes three distinct clinical-stage drug candidates: EO2463 to treat indolent non-Hodgkin lymphomas; EO4010 to treat third-line colorectal cancer; and EO2401 to treat glioblastoma and adrenal tumors. Each of these candidates has shown positive early clinical efficacy and exceptional safety and tolerability. 

For more information, please contact:

ENTEROME INVESTOR & MEDIA RELATIONS
Pierre Bélichard
Chief Executive Officer

 

+33 (0)1 75 77 27 85
communication@enterome.com

 

 

Cohesion Bureau
Chris Maggos / Giovanni Ca’ Zorzi

 

+41 (0)79 367 6254 / +33 (0)7 84 67 07 27
enterome@cohesionbureau.com

 

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Clariane completes its plan to strengthen its financial structure with the disposal of its Petits-fils home care services network

Clariane completes its plan to strengthen its financial structure with the disposal of its Petits-fils home care services network




Clariane completes its plan to strengthen its financial structure with the disposal of its Petits-fils home care services network

Press release
 
June 12th, 2025

 

        

Clariane completes its plan to strengthen its financial structure with the disposal of its Petits-fils home care services network

Paris, June 12th, 2025 – Clariane (CLARI.PA – ISIN FR0010386334), announces today the signing of an agreement for the disposal of its Petits-fils network to Crédit Agricole Santé & Territoires for a gross disposal value of €345 million. Subject to the fulfillment of customary conditions precedent, the closing of this transaction is expected to take place in the third quarter of 2025.

The disposal of the Petits-fils network, the terms of which were set following a competitive process, completes, six months ahead of schedule, the implementation of the plan announced by Clariane on 14 November 2023 to strengthen its financial structure. This plan included an asset disposal programme worth a gross amount of €1 billion.

The various operating assets sold under this programme were sold on good terms, with an average EBITDA multiple of c.14x, helping to strengthen the company’s balance sheet.

Founded in 2007, Petits-fils is now recognized for the high-quality home services it provides to elderly people, offering independent living, meal assistance, home care, support, and administrative assistance. When it was acquired by Clariane in November 2018, Petits-fils operated a national franchise network of 58 branches. Six years later, in 2024, Petits-fils now has a network of 292 branches serving close to 39,000 people in France. These contributed €56 million to Clariane Group revenue in 2024.

Crédit Agricole Santé & Territoires’ acquisition of Petits-fils will enable Petits-fils to continue its development path by benefiting from Crédit Agricole Santé & Territoires’ strong local presence.

Clariane and Crédit Agricole Santé & Territoires are also considering entering into a country-wide service partnership to enable patients and their carers to find the local care solutions best suited to their needs.

Sophie Boissard, Chief Executive Officer of the Clariane Group, said:

In November 2023, in a severely deteriorated financial environment for the sector, Clariane announced the implementation of an ambitious plan to strengthen its financial structure for a total of €1.5 billion. Thanks to the unwavering commitment of our teams and the full support of our shareholders, this plan has been successfully executed a year and a half later. This plan, along with the amendment and extension of our syndicated loan announced in February 2025, has significantly strengthened Clariane’s financial position and restored a more normalized access to financing.
Building on these achievements and driven by the momentum of our “At Your Side” corporate project and the “Better Support” program, we approach the coming months with determination and confidence.

Disclaimer

This document contains forward-looking statements that involve risks and uncertainties, including those included or incorporated by reference, concerning the Group’s future growth and profitability that could cause actual results to differ materially from those indicated in the forward-looking statements. These risks and uncertainties relate to factors that the Company cannot control or estimate precisely, such as future market conditions. The forward-looking statements made in this document constitute expectations for the future and should be regarded as such. Actual events or results may differ from those described in this document due to a number of risks and uncertainties described in Chapter 2 of the 2024 Universal Registration Document filed with the AMF on 1 April 2025 under registration number D.25-0209, available on the Company’s website (www.clariane.com) and that of the AMF (www.amf-france.org). All forward-looking statements included in this document are valid only as of the date of this press release. Clariane S.E. undertakes no obligation and assumes no responsibility to update the information contained herein beyond the requirements of applicable regulations.

Readers are cautioned not to place undue reliance on these forward-looking statements. Neither Clariane nor any of its directors, officers, employees, agents, affiliates or advisors accepts any responsibility for the reasonableness of any assumptions or opinions expressed or for the likelihood of any projections, prospects or performance being achieved. Any liability for such information is expressly excluded. Nothing in this document is, or should be construed as a promise or representation regarding the future. Furthermore, nothing contained in this document is intended to be or should be construed as a forecast of results. Clariane’s past performance should not be taken as a guide to future performance.

The main Alternative Performance Indicators (APIs), such as EBITDA, EBIT, net debt and financial leverage, are defined in the Universal Registration Document available on the Company’s website at www.clariane.com.

About Clariane

Clariane is the leading European community of care in times of vulnerability. It has operations in six countries: Belgium, France, Germany, Italy, the Netherlands and Spain.
Relying on their diverse expertise, each year, the Group’s 63,000 professionals provide services to nearly 900,000 patients and residents in three main areas of activity: care homes (Korian, Seniors Residencias, etc.), healthcare facilities and services (Inicea, Ita, Grupo 5, etc.), and alternative living solutions (Petits-fils, Ages & Vie, etc.).
In June 2023, Clariane became a purpose-driven company and added to its bylaws a new corporate purpose, common to all its activities: “taking care of each person’s humanity in times of vulnerability”.

Clariane has been listed on Euronext Paris, Section B since November 2006. The Group joined the SBF 120 index and the CAC® SBT 1.5° index on 23 September 2024.

Euronext ticker: CLARI.PA – ISIN: FR0010386334.

Stéphane Bisseuil                                                        Benoît Lesieur

     Head of Investor Relations                      Deputy Head of Investor Relations – ESG
        +33 6 58 60 68 69                                                        +33 6 64 80 15 90
                stephane.bisseuil@clariane.com                                benoit.lesieur@clariane.com

        Julie Mary                                                                Florian Bachelet

                                                                             Press officer                                                                      Press officer

        +33 6 59 72 50 69                                                                +33 6 79 86 78 23
                julie.mary@clariane.com                                florian.bachelet@clariane.com

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Editas Medicine Reports Proprietary Targeted Lipid Nanoparticle Delivery in Non-Human Primates Enables In Vivo HBG1/2 Promoter Editing for Sickle Cell Disease and Beta Thalassemia at the European Hematology Association 2025 Congress in June

Editas Medicine Reports Proprietary Targeted Lipid Nanoparticle Delivery in Non-Human Primates Enables In Vivo HBG1/2 Promoter Editing for Sickle Cell Disease and Beta Thalassemia at the European Hematology Association 2025 Congress in June




Editas Medicine Reports Proprietary Targeted Lipid Nanoparticle Delivery in Non-Human Primates Enables In Vivo HBG1/2 Promoter Editing for Sickle Cell Disease and Beta Thalassemia at the European Hematology Association 2025 Congress in June

Achieved 58% mean editing at five months after a single dose using high efficiency HSC delivery, demonstrating therapeutically relevant editing levels using a clinically validated strategy.

Achievement supports development of a novel, in vivo approach to treating sickle cell disease and beta thalassemia.

CAMBRIDGE, Mass., June 12, 2025 (GLOBE NEWSWIRE) — Editas Medicine, Inc. (Nasdaq: EDIT), a pioneering gene editing company, today shared new in vivo data demonstrating therapeutically relevant levels of HBG1/2 promoter editing in hematopoietic stem cells (HSCs) with a single dose of proprietary targeted lipid nanoparticle (tLNP) in non-human primates (NHPs). This clinically validated approach targeting HBG1/2 promoters to upregulate fetal hemoglobin (HbF) is in pre-clinical development as a potential transformative in vivo gene editing medicine for the treatment of sickle cell disease and beta thalassemia. The Company reported these data in a presentation available today and will detail the data in a poster session on Saturday, June 14th 6:30 – 7:30 p.m. CEST (12:30 – 1:30 p.m. EDT) at the European Hematology Association (EHA) 2025 Congress in Milan, Italy.

In this study, the Company’s proprietary tLNP formulation delivered HBG1/2 promoter editing cargo to HSCs in NHPs. Latest data from this ongoing NHP study showed that at five months a single intravenous administration of Editas’ tLNP resulted in mean on-target editing levels in the HBG1/2 promoter region of 58% in HSCs: well exceeding the predicted editing threshold of ≥25% required for therapeutic benefit. In addition to achieving therapeutically relevant editing levels, the biodistribution data in NHPs with Editas’ tLNP continue to show significant de-targeting of the liver in contrast to standard LNPs.

“These data from our in vivo HSC program confirm our ability to achieve high efficiency delivery, therapeutically relevant editing levels and favorable biodistribution in NHPs. These data validate the further development of Editas’ proprietary HSC-tLNP for editing of the HBG1/2 promoters for the treatment of sickle cell disease and beta thalassemia,” said Linda C. Burkly, Ph.D., Executive Vice President and Chief Scientific Officer, Editas Medicine.

Editas Medicine’s in vivo HSC program targets HBG1/2 promoters to mimic naturally occurring mechanisms of hereditary persistence of fetal hemoglobin (HPFH) and utilizes proprietary AsCas12a to edit with high efficiency and minimize off-target editing. Editing the HBG1/2 promoters with AsCas12a with the investigational medicine reni-cel led to robust increases in HbF and total hemoglobin (Hb) in clinical trials.

The presentation details are listed below. Abstracts can be accessed on the EHA website, and the presentation will be posted on the Editas Medicine website during the conference.

Poster Presentation Details:
Title: Targeted Lipid Nanoparticle Delivery in Non-Human Primates Enables In Vivo HBG1/2 Promoter Editing for β-hemoglobinopathies
Date/Time: Saturday, June 14, 2025, 6:30 – 7:30 p.m. CEST/ 12:30 – 1:30 p.m. EDT
Location: Allianz MiCo, Milano Convention Centre
Session: Poster Session 2

About Editas Medicine
As a pioneering gene editing company, Editas Medicine is focused on translating the power and potential of the CRISPR/Cas12a and CRISPR/Cas9 genome editing systems into a robust pipeline of in vivo medicines for people living with serious diseases around the world. Editas Medicine aims to discover, develop, manufacture, and commercialize transformative, durable, precision in vivo gene editing medicines for a broad class of diseases. Editas Medicine is the exclusive licensee of Broad Institute’s Cas12a patent estate and Broad Institute and Harvard University’s Cas9 patent estates for human medicines. For the latest information and scientific presentations, please visit www.editasmedicine.com.

CONTACT: Media and Investor Contacts:
media@editasmed.com
ir@editasmed.com

Significant efficacy benefit of IMBRUVICA® (ibrutinib) plus venetoclax versus acalabrutinib plus venetoclax in frontline treatment of patients with chronic lymphocytic leukaemia suggested by indirect treatment comparison

Significant efficacy benefit of IMBRUVICA® (ibrutinib) plus venetoclax versus acalabrutinib plus venetoclax in frontline treatment of patients with chronic lymphocytic leukaemia suggested by indirect treatment comparison




Significant efficacy benefit of IMBRUVICA® (ibrutinib) plus venetoclax versus acalabrutinib plus venetoclax in frontline treatment of patients with chronic lymphocytic leukaemia suggested by indirect treatment comparison

Cross-study findings indicate significant clinical benefit of frontline fixed-duration ibrutinib plus venetoclax with improved likelihood of undetectable minimal residual disease and progression-free survival versus acalabrutinib plus venetoclax1

Phase 2 CAPTIVATE long-term follow-up data further supports sustained efficacy and safety profile of fixed-duration ibrutinib plus venetoclax treatment in patients receiving frontline treatment for chronic lymphocytic leukaemia2

Beerse, Belgium, June 12, 2025 (GLOBE NEWSWIRE) — Janssen-Cilag International NV, a Johnson & Johnson company, today announced new data from a matching-adjusted indirect comparison (MAIC) analysis assessing the efficacy of IMBRUVICA® (ibrutinib) in combination with venetoclax (I+V) vs acalabrutinib in combination with venetoclax (A+V) as fixed-duration (FD) treatments for adults with previously untreated chronic lymphocytic leukaemia (CLL).1 The data were featured in a poster presentation at the 30th European Hematology Association (EHA) Congress (Poster presentation #PF587) and reported that the I+V regimen yielded significantly better efficacy when compared to the A+V regimen.1 Patients treated with I+V were more likely to achieve disease clearance, as measured by undetectable minimal residual disease (uMRD) three months after the end of treatment (EOT+3), from both peripheral blood (PB) and bone marrow (BM).1 In addition to this, progression-free survival (PFS) significantly favoured I+V compared to A+V.1

“In the absence of head-to-head trials, clinicians need reliable tools to effectively compare treatment options and make the best possible choices for their patients,” said Talha Munir, M.D., Consultant in Clinical Haematology at St James’s Hospital, Leeds, United Kingdom.* “The matching-adjusted indirect comparison data presented at EHA suggests that ibrutinib plus venetoclax may offer meaningful clinical advantages over acalabrutinib plus venetoclax with patients more likely to achieve higher rates of undetectable minimal residual disease, three months after treatment. This may translate into more time in remission and longer progression-free survival – outcomes that matter deeply to both patients and the healthcare professionals who treat them.”

Patients who met the AMPLIFY inclusion criteria from both the Phase 3 GLOW (NCT03462719) and Phase 2 CAPTIVATE (NCT02910583) studies were included in this analysis, and, after matching and balancing the treatment cohorts, comparative analyses between the trials suggested that I+V significantly reduced the risk of progression or death by 47 percent when compared to patients treated with A+V (hazard ratio [HR]: 0.53; 95 percent confidence interval [CI]: 0.33-0.85; p=0.0085).1,3,4,5 The results also suggested that patients treated with I+V were almost twice (95 percent CI: 1.47-2.41; p<0.0001) and 2.4 times (95 percent CI: 1.78-3.12; p<0.0001) more likely to achieve uMRD than A+V at EOT+3, in PB and BM, respectively.1 The GLOW and CAPTIVATE FD cohorts were based on individual patient-level data with a median follow-up of approximately 4.5 years, while the A+V cohort used aggregate level data from the AMPLIFY study with a median follow-up of approximately 3.4 years.1

Results from final analysis of CAPTIVATE

Long-term follow-up results from the Phase 2 CAPTIVATE study data were presented as a poster at the American Society of Clinical Oncology (ASCO) 2025 Annual Meeting (Poster presentation #219) and will also be presented as an encore oral presentation at EHA 2025 (Oral presentation #S156).6 The data reinforced the durable clinical benefit of frontline I+V.2 Phase 2 CAPTIVATE results demonstrated patients in the I+V FD cohort displayed a clinically meaningful PFS and overall survival (OS) vs the MRD-guided cohort.2 After a median follow-up of 68.9 months, the 5.5-year PFS and OS rates for all treated patients were 66 percent (95 percent CI: 58-72) and 97 percent (95 percent CI: 93-99), respectively.2 Additionally, the 5.5 year PFS rate in patients who achieved uMRD in the PB at the EOT was 71 percent (95 percent CI: 60-80).2 Furthermore, 1-year PFS and OS rates from the start of retreatment (with single-agent ibrutinib or FD I+V) were both 100 percent, whilst 2-year PFS and OS rates from the start of retreatment were 91 percent and 96 percent, respectively.2 No new safety signals were observed during the CAPTIVATE study since the previous follow-up, with COVID-19, diarrhoea and hypertension being the most frequently reported adverse events (AEs).2 In the total pooled CAPTIVATE population, 32 percent (n=64/202) of patients had progressive disease following 5.5 years of follow-up.2 Of the 53 patients with available samples, none had acquired resistance-associated mutations in BTK or PLCG2.2

“The final analysis of CAPTIVATE highlights how ibrutinib continues to raise the bar in the treatment of chronic lymphocytic leukaemia, with durable minimal residual disease response, extended treatment-free intervals, and a tolerable safety profile,” said Ester in’t Groen, EMEA Therapeutic Area Head Haematology, Johnson & Johnson Innovative Medicine. “With the longest follow-up of any oral fixed-dose regimen, ibrutinib is setting a new standard for what patients and clinicians can expect from targeted therapies. We remain committed to advancing science in complex blood cancers and improving outcomes across the cancer care landscape.”

“The updates presented at EHA add to the growing body of evidence in support of ibrutinib, the most extensively studied Bruton’s tyrosine kinase inhibitor, as the standard of care in the frontline treatment of chronic lymphocytic leukaemia,” said Jessica Vermeulen, Vice President, Lymphoma & Leukemia Disease Area Stronghold Leader, Johnson & Johnson Innovative Medicine. “Offering patients not only more time, but also the option for treatment-free remissions continues to be our goal and we are proud of the incredible contribution ibrutinib has made since its first European approval in 2014.”

About the MAIC analysis
The objective of this analysis was to compare the progression-free survival (PFS) and undetectable minimal residual disease (uMRD) data from the fixed-duration (FD) ibrutinib + venetoclax (I+V) cohorts from the Phase 3 GLOW (NCT03462719) and Phase 2 CAPTIVATE (NCT02910583) studies against the Phase 3 AMPLIFY (NCT03836261) data.1 In absence of prospective head-to-head trials investigating different Bruton’s tyrosine kinase inhibitors (BTKis) plus B-cell lymphoma 2 (Bcl-2) strategies, this study utilised matching-adjusted indirect comparison (MAIC) to obtain useful insights on comparative efficacy.1 Individual patient data from the FD I+V cohorts of the GLOW and CAPTIVATE studies were pooled and compared to aggregate published Intent-to-Treat (ITT) data of the acalabrutinib plus venetoclax (A+V) arm of the AMPLIFY study.1,3,4,5 A MAIC was performed following method published by Signorovitch et al. and guidelines from the National Institute for Health and Care Excellence (NICE).1,7 Patients who did not meet the inclusion criteria of AMPLIFY were excluded from the I+V pooled cohort to establish the I+V patient population for analysis.1 The remaining I+V patients were then reweighted so that the average baseline characteristics of the pooled I+V cohort matched those of the A+V patients in AMPLIFY.1 The reweighted outcomes from I+V were then compared to the reported outcomes for A+V using indirect treatment comparison.1 Relative effects were quantified using relative risk (RR) and hazard ratios (HR) with 95 percent confidence intervals.1 There are potential sources of bias that cannot be accounted for in MAIC, that should be considered when interpreting such data. Specifically, in this comparison, the measurement of progression was stricter in GLOW and CAPTIVATE, requiring computer or magnetic imaging regardless of suspected progression and the median follow-up was longer in I+V population.1 Both may have biased the PFS results in favour of A+V.1 Additionally, AMPLIFY reported multicolour flow cytometry use but with no details on the number of colours and comparability is assumed with the 8-colour assay used in I+V studies.1 As in any non-randomised comparison there may be additional unreported clinically important prognostic patient baseline characteristics which cannot be accounted for.1 For example, Cumulative Illness Rating Scale score data was not collected in CAPTIVATE and therefore could not be used in matching.1

About CAPTIVATE
The Phase 2 CAPTIVATE study (NCT02910583) evaluated previously untreated adult patients with chronic lymphocytic leukaemia (CLL), who were 70 years or younger, including patients with high-risk disease, in two cohorts with combined median age of 60 years: a minimal residual disease (MRD)-guided cohort (n=43) and an FD cohort (n=159; median age).2,4,8 Patients in the FD cohort received three cycles of ibrutinib lead-in followed by 12 cycles of I+V (oral ibrutinib [420 mg/d]; oral venetoclax [five-week ramp-up to 400 mg/d]) and the primary endpoint was complete response rate.4 In the MRD cohort, after completion of three cycles ibrutinib lead-in followed by 12 cycles I+V, patients with confirmed uMRD were randomly assigned to double-blind treatment with placebo, or continuous ibrutinib.4 The primary endpoint was one-year disease-free survival.4 

About the GLOW study 
The GLOW study (NCT03462719) is a randomised, open-label, Phase 3 trial that evaluated the efficacy and safety of frontline, FD I+V versus chlorambucil plus obinutuzumab in adult patients with CLL who are (a) ≥65 years old, or (b) 18-64 years old with a Cumulative Illness Rating Scale score of greater than six or creatinine clearance less than 70 mL/min, who had active disease requiring treatment per the International Workshop on CLL criteria.3 

About ibrutinib 
Ibrutinib is a once-daily oral medication that is jointly developed and commercialised by Janssen Biotech, Inc. and Pharmacyclics LLC, an AbbVie company.9 Ibrutinib blocks the Bruton’s tyrosine kinase (BTK) protein, which is needed by normal and abnormal B-cells, including specific cancer cells, to multiply and spread.10 By blocking BTK, ibrutinib may help move abnormal B-cells out of their nourishing environments and inhibits their proliferation.11 
Ibrutinib is approved in more than 100 countries and has been used to treat more than 325,000 patients worldwide.12 There are more than 50 company-sponsored clinical trials, including 18 Phase 3 studies, over 11 years evaluating the efficacy and safety of ibrutinib.10,13 In October 2021, ibrutinib was added to the World Health Organization’s Model Lists of Essential Medicines (EML), which refers to medicines that address global health priorities and which should be available and affordable for all.14 

Ibrutinib was first approved by the European Commission (EC) in 2014, and approved indications to date include:10 

  • As a single agent or in combination with rituximab or obinutuzumab or venetoclax for the treatment of adult patients with previously untreated CLL 
  • As a single agent or in combination with bendamustine and rituximab (BR) for the treatment of adult patients with CLL who have received at least one prior therapy 
  • As a single agent for the treatment of adult patients with relapsed or refractory (RR) mantle cell lymphoma (MCL) 
  • As a single agent for the treatment of adult patients with Waldenström’s macroglobulinaemia (WM) who have received at least one prior therapy, or in first line treatment for patients unsuitable for chemo-immunotherapy. In combination with rituximab for the treatment of adult patients with WM 

For a full list of adverse events and information on dosage and administration, contraindications and other precautions when using ibrutinib please refer to the Summary of Product Characteristics.

About Chronic Lymphocytic Leukaemia
CLL is typically a slow-growing blood cancer of the white blood cells.15 The overall incidence of CLL in Europe is approximately 4.92 cases per 100,000 persons per year and it is about 1.5 times more common in men than in women (based on individuals diagnosed 2000-2002).16 CLL is predominantly a disease of the elderly, with a median age of 72 years at diagnosis.17 While patient outcomes have dramatically improved in the last few decades, the disease is still characterised by consecutive episodes of disease progression and the need for therapy.18 Patients are often prescribed multiple lines of therapy as they relapse or become resistant to treatments.19 

About Johnson & Johnson
At Johnson & Johnson, we believe health is everything. Our strength in healthcare innovation empowers us to build a world where complex diseases are prevented, treated, and cured, where treatments are smarter and less invasive, and solutions are personal. Through our expertise in Innovative Medicine and MedTech, we are uniquely positioned to innovate across the full spectrum of healthcare solutions today to deliver the breakthroughs of tomorrow, and profoundly impact health for humanity.

Learn more at www.innovativemedicine.jnj.com/emea. Follow us at www.linkedin.com/company/jnj-innovative-medicine-emea. Janssen-Cilag International NV, Janssen Pharmaceutica NV, Janssen-Cilag Limited, Janssen Biotech, Inc., and Janssen Research & Development, LLC are Johnson & Johnson companies.

Cautions Concerning Forward-Looking Statements
This press release contains “forward-looking statements” as defined in the Private Securities Litigation Reform Act of 1995 regarding product development and the potential benefits and treatment impact of daratumumab. The reader is cautioned not to rely on these forward-looking statements. These statements are based on current expectations of future events. If underlying assumptions prove inaccurate or known or unknown risks or uncertainties materialise, actual results could vary materially from the expectations and projections of Johnson & Johnson. Risks and uncertainties include, but are not limited to: challenges and uncertainties inherent in product research and development, including the uncertainty of clinical success and of obtaining regulatory approvals; uncertainty of commercial success; competition, including technological advances, new products and patents attained by competitors; challenges to patents; changes in behaviour and spending patterns of purchasers of health care products and services; changes to applicable laws and regulations, including global health care reforms; and trends toward health care cost containment. A further list and descriptions of these risks, uncertainties and other factors can be found in Johnson & Johnson’s most recent Annual Report on Form 10-K, including in the sections captioned “Cautionary Note Regarding Forward-Looking Statements” and “Item 1A. Risk Factors,” and in Johnson & Johnson’s subsequent Quarterly Reports on Form 10-Q and other filings with the Securities and Exchange Commission. Copies of these filings are available online at http://www.sec.gov/, http://www.jnj.com/ or on request from Johnson & Johnson. Johnson & Johnson does not undertake to update any forward-looking statement as a result of new information or future events or developments.

*Talha Munir, M.D., Consultant in Clinical Haematology at St James’s Hospital, Leeds, United Kingdom, has provided consulting, advisory, and speaking services to Janssen-Cilag International NV; he has not been paid for any media work. 


1 Munir T, et al., Cross-Study Comparison of Ibrutinib in Combination with Venetoclax (I+V) vs Acalabrutinib in Combination with Venetoclax (A+V) in Subjects with Previously Untreated Chronic Lymphocytic Leukemia (CLL). Poster presentation at 2025 European Hematology Association (EHA) Congress; June 12–15, 2025. [Poster PF587].
2 Ghia P, et al. Final Analysis of Fixed-Duration Ibrutinib + Venetoclax for Chronic Lymphocytic Leukemia (CLL)/Small Lymphocytic Lymphoma (SLL) in the Phase 2 CAPTIVATE Study. Oral presentation at 2025 European Hematology Association (EHA) Congress; June 12–15, 2025. [Oral S156].
3 ClinicalTrials.gov. A Study of the Combination of Ibrutinib Plus Venetoclax Versus Chlorambucil Plus Obinutuzumab for the First-line Treatment of Participants With Chronic Lymphocytic Leukemia (CLL)/​Small Lymphocytic Lymphoma (SLL) (GLOW). Available at: https://clinicaltrials.gov/study/NCT03462719. Last accessed: June 2025.
4 ClinicalTrials.gov. Ibrutinib Plus Venetoclax in Subjects With Treatment-naive Chronic Lymphocytic Leukemia /Small Lymphocytic Lymphoma (CLL/SLL) (CAPTIVATE). Available at: https://clinicaltrials.gov/ct2/show/NCT02910583. Last accessed: June 2025.
5 ClinicalTrials.gov. Study of Acalabrutinib (ACP-196) in Combination With Venetoclax (ABT-199), With and Without Obinutuzumab (GA101) Versus Chemoimmunotherapy for Previously Untreated CLL (AMPLIFY). Available at: https://clinicaltrials.gov/study/NCT03836261. Last accessed: June 2025.
6 Ghia P, et al. Final Analysis of Fixed-Duration Ibrutinib + Venetoclax for Chronic Lymphocytic Leukemia/Small Lymphocytic Lymphoma in the Phase 2 CAPTIVATE Study. Poster presentation at 2025 American Society of Clinical Oncology (ASCO) Annual Meeting; May 30–June 3, 2025. [Poster #219].
7 Signorovitch JE, et al. Matching-adjusted Indirect Comparisons: A New Tool for Timely Comparative Effectiveness Research. Value Health, 2012; 15: 940-947.
8 Jacobs R, et al., Outcomes in High-risk Subgroups After Fixed-Duration Ibrutinib + Venetoclax for Chronic Lymphocytic Leukemia/Small Lymphocytic Lymphoma: Up To 5.5 years of Follow-up in the Phase 2 CAPTIVATE Study. Poster presentation at 2024 European Hematology Association (EHA) Hybrid Congress; June 13–16, 2024. [Poster P675].
9 European Medicines Agency. IMBRUVICA Summary of Product Characteristics. April 2025. Available at: https://www.ema.europa.eu/en/documents/product-information/imbruvica-epar-product-information_en.pdf. Last accessed: June 2025.
10 Turetsky A, et al. Single cell Imaging of Bruton’s tyrosine kinase using an Irreversible Inhibitor. Sci Rep. 2014; 4: 4782.
11 de Rooij MF, et al. The Clinically Active BTK Inhibitor PCI-32765 targets B-cell Receptor- and Chemokine-controlled Adhesion and Migration in Chronic Lymphocytic Leukemia. Blood. 2012; 119(11): 2590-2594.
12 J&J Data on File (RF-465355). Patients Treated on Imbruvica Worldwide. May 2025.
13 Pollyea DA, et al. A Phase I Dose Escalation Study of the Btk Inhibitor PCI-32765 in Relapsed and Refractory B Cell Non-Hodgkin Lymphoma and Use of a Novel Fluorescent Probe Pharmacodynamic Assay. Blood. 2009; 114(22): 3713.
14 World Health Organization. WHO Prioritizes Access to Diabetes and Cancer Treatments in New Essential Medicines Lists. Available at: https://www.who.int/news/item/01-10-2021-who-prioritizes-access-to-diabetes-and-cancer-treatments-in-new-essential-medicines-lists. Last accessed: June 2025.
15 American Cancer Society. What is Chronic Lymphocytic Leukemia? Available at: https://www.cancer.org/cancer/chronic-lymphocytic-leukemia/about/what-is-cll.html. Last accessed: June 2025.
16 Sant M, et al. Incidence of Hematologic Malignancies in Europe by Morphologic Subtype: Results of the HAEMACARE project. Blood. 2010; 116:3724–34.
17 Eichhorst B, et al. Chronic Lymphocytic Leukaemia: ESMO Clinical Practice Guidelines for Diagnosis, Treatment and Follow-up Ann Oncol. 2021; 32(1): 23-33.
18 Moreno C. Standard Treatment Approaches for Relapsed/refractory Chronic Lymphocytic Leukemia after Frontline Chemoimmunotherapy. Hematology Am Soc Hematol Educ Program. 2020; 2020: 33-40.
19 Bewarder M, et al. Current Treatment Options in CLL. Cancers (Basel). 2021;13(10): 2468.

CP-523458 

June 2025

CONTACT: Media contact:
Jenni Mildon
jmildon@its.jnj.com
+44 7920 418 552 

Investor contact:
Lauren Johnson
Investor-relations@its.jnj.com 

Dapirolizumab Pegol Phase 3 Data in SLE Presented at the Annual European Congress of Rheumatology (EULAR) Show Improvement in Fatigue and Reduction in Disease Activity

Dapirolizumab Pegol Phase 3 Data in SLE Presented at the Annual European Congress of Rheumatology (EULAR) Show Improvement in Fatigue and Reduction in Disease Activity




Dapirolizumab Pegol Phase 3 Data in SLE Presented at the Annual European Congress of Rheumatology (EULAR) Show Improvement in Fatigue and Reduction in Disease Activity

  • Dapirolizumab pegol (DZP) showed efficacy across multiple clinical endpoints in the PHOENYCS GO study, including fatigue and measures of disease activity
  • DZP showed consistent improvements in fatigue, a common and debilitating symptom of systemic lupus erythematosus (SLE)
  • At Week 48, more individuals receiving DZP experienced no or low disease activity compared to standard of care with differences observed as early as Week 12

BRUSSELS and CAMBRIDGE, Mass., June 12, 2025 (GLOBE NEWSWIRE) — UCB (Euronext Brussels: UCB) and Biogen Inc. (NASDAQ: BIIB) today presented additional detailed results from the Phase 3 PHOENYCS GO study evaluating dapirolizumab pegol (DZP), a novel Fc-free anti-CD40L drug candidate. In the study, DZP demonstrated significant clinical improvements in disease activity in people living with moderate-to-severe systemic lupus erythematosus (SLE), as measured by the British Isles Lupus Assessment Group (BILAG)-based Composite Lupus Assessment (BICLA) at Week 48, the primary endpoint. Improvements were also seen across additional clinical measures, including fatigue and disease activity/remission. These results were presented at EULAR 2025, the European Alliance of Associations for Rheumatology’s annual meeting, in Barcelona, Spain. The safety and efficacy of DZP in SLE have not been established, and it is not approved for use in SLE by any regulatory authority worldwide. A second Phase 3 trial of dapirolizumab pegol is ongoing with the goal of confirming the results from PHOENYCS GO.

“Despite being a common manifestation of systemic lupus erythematosus, fatigue is a difficult-to-treat symptom that can severely impact a person’s quality of life, and remains a challenge to address,” said Ioannis Parodis, MD, PhD, Associate Professor of Rheumatology, Karolinska University Hospital, Sweden. “The results we observed in this Phase 3 study indicate that participants treated with dapirolizumab pegol have the potential to achieve consistent improvements in fatigue beyond the current standard of care.”

In an analysis of the impact of DZP on patient-reported fatigue in people with SLE participating in the PHOENYCS GO study, individuals receiving DZP in addition to standard of care (SOC) treatment demonstrated improvements across two fatigue measurements:

  • Improvements in Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue scores were greater in the DZP group (change from baseline, 8.9), compared with SOC alone (5.2; nominal* p=0.0024) at Week 48.
  • Using FATIGUE-PRO, a measure recently developed to capture the patient experience of fatigue in SLE, greater improvements from baseline (nominal* p<0.05) were observed in people receiving DZP compared with SOC alone in the Physical Fatigue (change from baseline difference between groups,7.6), Mental and Cognitive (5.6), and Susceptibility to Fatigue (7.8) scales at Week 48.

“Being able to address both fatigue and remission are areas of critical unmet need in lupus care, an important treatment goal is to improve the quality of life for patients as well as to reduce the long-term risk of organ damage through disease remission,” said Eric F. Morand, MBBS, Head of the Monash Health Rheumatology Unit, Monash University, Australia. “In the PHOENYCS GO study, dapirolizumab pegol has shown meaningful impact in helping patients achieve remission and low disease activity, offering the exciting possibility for improved disease control while reducing exposure to glucocorticoids. Dapirolizumab pegol has the potential to become a significant new medication for people living with SLE, as shown by the breadth of effect seen in the study and I look forward to seeing results from the second Phase 3 study.”

In an additional analysis, improvements were seen on measures of low disease activity, as measured by Low Lupus Disease Activity State (LLDAS) and disease remission, as measured by Definition of Remission in SLE (DORIS). Both measures include assessments of disease activity, in addition to a required low dose glucocorticoid intake (<7.5 mg prednisone / day in LLDAS; <5 mg prednisone / day in DORIS).

  • At Week 48, the percentage of participants achieving low disease activity in the DZP group was twice that of the SOC only group (40.9% and 19.6%, respectively; nominal* p<0.0001). As early as Week 12, greater proportions of participants receiving DZP plus SOC achieved LLDAS versus SOC alone (nominal* p<0.05). 
  • 23.6% of participants receiving DZP plus SOC achieved low disease activity in ≥50% of visits through 48 weeks compared with 15.9% receiving SOC alone (nominal* p=0.1042).
  • A higher proportion of those receiving DZP (19.2%) versus SOC alone (8.4%) also achieved DORIS at Week 48 (nominal* p=0.0056).

* Having met the primary endpoint, improvement of moderate-to-severe disease activity as assessed by achievement of BICLA after 48 weeks and the key secondary endpoint having a p-value = 0.1776, all subsequent secondary and tertiary endpoints are descriptive and nominal p-values are included.

The safety profile of DZP was generally favorable. The safety results were consistent with previous DZP studies and with that in study participants with SLE receiving an immunomodulator. A higher proportion of individuals receiving DZP plus SOC had treatment-emergent adverse events (TEAEs) compared to SOC alone (82.6% vs. 75.0%). The proportion of participants with serious TEAEs was 9.9% in participants receiving DZP plus SOC was lower as compared to 14.8% in those receiving SOC alone. Discontinuation of treatment or study participation due to TEAEs occurred in 4.7% (10) of participants receiving DZP plus SOC and 3.7% (4) of participants receiving SOC alone.

Participants from the PHOENYCS GO study (NCT04294667) will continue to be followed in a long-term open-label study. A second Phase 3 trial of dapirolizumab pegol, PHOENYCS FLY (NCT06617325) is ongoing.

About Systemic Lupus Erythematosus (SLE)
SLE is a chronic, multifactorial autoimmune disease that is caused by the activation of autoreactive T, B and antigen-presenting cells, resulting in manifestations across multiple organ systems with periods of illness or flares alternating with periods of inactivity.1 SLE can present itself in several ways including rash, arthritis, anemia, thrombocytopenia, serositis, nephritis, seizures or psychosis.2 SLE is associated with a greater risk of death from causes such as infection and cardiovascular disease.

An estimated 90% of people living with lupus are women; most begin to see symptoms between the ages of 15-55.3,4,5 Individuals from populations of African, Hispanic, Asian and Native American descent are at a greater risk of earlier onset and more aggressive disease.6,7 Pregnancy in women with SLE is high risk, with higher maternal and fetal mortality and morbidity than the general population.8,9

About Dapirolizumab Pegol
Dapirolizumab pegol is a novel investigational humanized Fc-free polyethylene glycol (PEG)-conjugated antigen-binding (Fab’) fragment. Dapirolizumab pegol inhibits CD40L signaling which has been shown to reduce B cell activation and autoantibody production, mitigate type 1 interferon (IFN) secretion, and attenuate T cell and antigen-presenting cell (APC) activation.10 Dapirolizumab pegol is presently in Phase 3 clinical development for the treatment of systemic lupus erythematosus (SLE) under a collaboration between UCB and Biogen.11

About UCB
UCB, Brussels, Belgium (www.ucb.com) is a global biopharmaceutical company focused on the discovery and development of innovative medicines and solutions to transform the lives of people living with severe diseases of the immune system or of the central nervous system. With approximately 9,000 people in approximately 40 countries, the company generated revenue of €6.1 billion in 2024. UCB is listed on Euronext Brussels (symbol: UCB).

About Biogen
Founded in 1978, Biogen is a leading biotechnology company that pioneers innovative science to deliver new medicines to transform patient’s lives and to create value for shareholders and our communities. We apply deep understanding of human biology and leverage different modalities to advance first-in-class treatments or therapies that deliver superior outcomes. Our approach is to take bold risks, balanced with return on investment to deliver long-term growth.

The company routinely posts information that may be important to investors on its website at www.biogen.com. Follow us on social media – FacebookLinkedInXYouTube.

Forward looking statements UCB
This document contains forward-looking statements, including, without limitation, statements containing the words “potential”, “believes”, “anticipates”, “expects”, “intends”, “plans”, “seeks”, “estimates”, “may”, “will”, “continue” and similar expressions. These forward-looking statements are based on current plans, estimates and beliefs of management. All statements, other than statements of historical facts, are statements that could be deemed forward-looking statements, including estimates of revenues, operating margins, capital expenditures, cash, other financial information, expected legal, arbitration, political, regulatory or clinical results or practices and other such estimates and results. By their nature, such forward-looking statements are not guaranteeing future performance and are subject to known and unknown risks, uncertainties, and assumptions which might cause the actual results, financial condition, performance or achievements of UCB, or industry results, to be materially different from any future results, performance, or achievements expressed or implied by such forward-looking statements contained in this document. Important factors that could result in such differences include but are not limited to: global spread and impacts of wars, pandemics and terrorism, the general geopolitical environment, climate change, changes in general economic, business and competitive conditions, the inability to obtain necessary regulatory approvals or to obtain them on acceptable terms or within expected timing, costs associated with research and development, changes in the prospects for products in the pipeline or under development by UCB, effects of future judicial decisions or governmental investigations, safety, quality, data integrity or manufacturing issues, supply chain disruption and business continuity risks; potential or actual data security and data privacy breaches, or disruptions of our information technology systems, product liability claims, challenges to patent protection for products or product candidates, competition from other products including biosimilars or disruptive technologies/business models, changes in laws or regulations, exchange rate fluctuations, changes or uncertainties in tax laws or the administration of such laws, and hiring, retention and compliance of its employees. There is no guarantee that new product candidates will be discovered or identified in the pipeline, or that new indications for existing products will be developed and approved. Movement from concept to commercial product is uncertain; preclinical results do not guarantee safety and efficacy of product candidates in humans. So far, the complexity of the human body cannot be reproduced in computer models, cell culture systems or animal models. The length of the timing to complete clinical trials and to get regulatory approval for product marketing has varied in the past and UCB expects similar unpredictability going forward. Products or potential products which are the subject of partnerships, joint ventures or licensing collaborations may be subject to disputes between the partners or may prove to be not as safe, effective or commercially successful as UCB may have believed at the start of such partnership. UCB’s efforts to acquire other products or companies and to integrate the operations of such acquired companies may not be as successful as UCB may have believed at the moment of acquisition. Also, UCB or others could discover safety, side effects or manufacturing problems with its products and/or devices after they are marketed. The discovery of significant problems with a product similar to one of UCB’s products that implicate an entire class of products may have a material adverse effect on sales of the entire class of affected products. Moreover, sales may be impacted by international and domestic trends toward managed care and health care cost containment, including pricing pressure, political and public scrutiny, customer and prescriber patterns or practices, and the reimbursement policies imposed by third-party payers as well as legislation affecting biopharmaceutical pricing and reimbursement activities and outcomes. Finally, a breakdown, cyberattack or information security breach could compromise the confidentiality, integrity and availability of UCB’s data and systems.

Given these uncertainties, the public is cautioned not to place any undue reliance on such forward-looking statements. These forward-looking statements are made only as of the date of this document, and do not reflect any potential impacts from the evolving event or risk as mentioned above as well as any other adversity, unless indicated otherwise. The company continues to follow the development diligently to assess the financial significance of these events, as the case may be, to UCB.

UCB expressly disclaims any obligation to update any forward-looking statements in this document, either to confirm the actual results or to report or reflect any change in its forward-looking statements with regard thereto or any change in events, conditions or circumstances on which any such statement is based, unless such statement is required pursuant to applicable laws and regulations.

Biogen Safe Harbor
This press release contains forward-looking statements, relating to: our strategy and plans; potential of, and expectations for, our commercial business and pipeline programs; capital allocation and investment strategy; clinical development programs, clinical trials, and data readouts and presentations; regulatory discussions, submissions, filings, and approvals; the potential benefits, safety, and efficacy of our and our collaboration partners’ products and investigational therapies; the anticipated benefits and potential of investments, actions to improve risk profile and productivity of R&D pipeline, collaborations, and business development activities; and our future financial and operating results. These forward-looking statements may be accompanied by such words as “aim,” “anticipate,” “assume,” “believe,” “contemplate,” “continue,” “could,” “estimate,” “expect,” “forecast,” “goal,” “guidance,” “hope,” “intend,” “may,” “objective,” “outlook,” “plan,” “possible,” “potential,” “predict,” “project,” “prospect,” “should,” “target,” “will,” “would,” and other words and terms of similar meaning. Drug development and commercialization involve a high degree of risk, and only a small number of research and development programs result in commercialization of a product. Results in early-stage clinical trials may not be indicative of full results or results from later stage or larger scale clinical trials and do not ensure regulatory approval. You should not place undue reliance on these statements. Given their forward-looking nature, these statements involve substantial risks and uncertainties that may be based on inaccurate assumptions and could cause actual results to differ materially from those reflected in such statements. This press release includes, among others, forward-looking statements including: that Biogen is building on a new foundation with the goal of long-term sustainable growth in its commercial portfolio; the multi-billion dollar potential of its late-stage pipeline; that we believe there remains a significant long-term opportunity for our ongoing product launches including LEQEMBI; that we believe that continued execution against these key strategic elements, as well as a disciplined approach to business development, will allow us to generate long-term value for our shareholders by bringing innovative medicines to patients; and all statements and information under the heading “Full Year 2025 Financial Guidance”. These forward-looking statements are based on management’s current beliefs and assumptions and on information currently available to management. Given their nature, we cannot assure that any outcome expressed in these forward looking statements will be realized in whole or in part.

We caution that these statements are subject to risks and uncertainties, many of which are outside of our control and could cause future events or results to be materially different from those stated or implied in this document, including, among others, factors relating to: our substantial dependence on revenue from our products and other payments under licensing, collaboration, acquisition or divestiture agreements; uncertainty of long-term success in developing, licensing, or acquiring other product candidates or additional indications for existing products; expectations, plans and prospects relating to product approvals, approvals of additional indications for our existing products, sales, pricing, growth, reimbursement and launch of our marketed and pipeline products; the potential impact of increased product competition in the biopharmaceutical and healthcare industry, as well as any other markets in which we compete, including increased competition from new originator therapies, generics, prodrugs and biosimilars of existing products and products approved under abbreviated regulatory pathways; our ability to effectively implement our corporate strategy; the successful execution of our strategic and growth initiatives, including acquisitions; the drivers for growing our business; difficulties in obtaining and maintaining adequate coverage, pricing, and reimbursement for our products; the drivers for growing our business, including our dependence on collaborators and other third parties for the development, regulatory approval, and commercialization of products and other aspects of our business, which are outside of our full control; risks associated with current and potential future healthcare reforms; risks related to commercialization of biosimilars, which is subject to such risks related to our reliance on third parties, intellectual property, competitive and market challenges and regulatory compliance; failure to obtain, protect, and enforce our data, intellectual property, and other proprietary rights and the risks and uncertainties relating to intellectual property claims and challenges; the risk that positive results in a clinical trial may not be replicated in subsequent or confirmatory trials or success in early stage clinical trials may not be predictive of results in later stage or large scale clinical trials or trials in other potential indications; risks associated with clinical trials, including our ability to adequately manage clinical activities, unexpected concerns that may arise from additional data or analysis obtained during clinical trials, regulatory authorities may require additional information or further studies, or may fail to approve or may delay approval of our drug candidates; the occurrence of adverse safety events, restrictions on use with our products, or product liability claims; risks relating to technology, including our incorporation of new technologies such as artificial intelligence into some of our processes; risks related to use of information technology systems and potential impacts of any breakdowns, interruptions, invasions, corruptions, data breaches, destructions and/or other cybersecurity incidents of our systems or those of connected and/or third-party systems; problems with our manufacturing capacity, including our ability to manufacture products efficiently or adequately address global bulk supply risks; risks relating to management, personnel and other organizational changes, including our ability to attracting, retaining and motivating qualified individuals; risks related to the failure to comply with current and new legal and regulatory requirements, including judicial decisions, accounting standards, and tariff or trade restrictions; the risks of doing business internationally, including geopolitical tensions, acts of war and largescale crises; risks relating to investment in our manufacturing capacity; risks relating to the distribution and sale by third parties of counterfeit or unfit versions of our products; risks relating to the use of social media for our business, results of operations and financial condition; fluctuations in our operating results; risks related to investment in properties; risks relating to access to capital and credit markets to finance our present and future operations and business initiatives and obtain funding for such activities on favorable terms; risks related to indebtedness; the market, interest, and credit risks associated with our investment portfolio; risks relating to share repurchase programs; change in control provisions in certain of our collaboration agreements; fluctuations in our effective tax rate and obligations in various jurisdictions in which we are subject to taxation; environmental risks; and any other risks and uncertainties that are described in other reports we have filed with the U.S. Securities and Exchange Commission.

These statements speak only as of the date of this press release and are based on information and estimates available to us at this time. Should known or unknown risks or uncertainties materialize or should underlying assumptions prove inaccurate, actual results could vary materially from past results and those anticipated, estimated or projected. Investors are cautioned not to put undue reliance on forward-looking statements. A further list and description of risks, uncertainties and other matters can be found in our Annual Report on Form 10-K for the fiscal year ended December 31, 2024 and in our subsequent reports on Form 10-Q and Form 10-K, in each case including in the sections thereof captioned “Note Regarding Forward-Looking Statements” and “Item 1A. Risk Factors,” and in our subsequent reports on Form 8-K. Except as required by law, we do not undertake any obligation to publicly update any forward-looking statements whether as a result of any new information, future events, changed circumstances or otherwise.

References:

  1. Tselios K, Gladman DD, Touma Z, et al. Disease course patterns in systemic lupus erythematosus. Lupus. 2019;28(1):114-122.
  2. Fanouriakis A, Tziolos N, Bertsias G, et al. Update οn the diagnosis and management of systemic lupus erythematosus. Ann Rheum Dis. 2021;80(1):14-25. doi:10.1136/annrheumdis-2020-218272
  3. Petri M. Epidemiology of systemic lupus erythematosus. Best Pract Res Clin Rheumatol. 2002;16(5):847-58. Epub 2002/12/11. doi: 10.1053/berh.2002.0259. PubMed PMID: 12473278.
  4. Rees F, Doherty M, Grainge M, Davenport G, Lanyon P, Zhang W. The incidence and prevalence of systemic lupus erythematosus in the UK, 1999-2012. Ann Rheum Dis. 2016;75(1):136-41. Epub 2014/10/01. doi: 10.1136/annrheumdis-2014-206334. PubMed PMID: 25265938; PubMed Central PMCID: PMCPMC4717400.
  5. Pons-Estel GJ, Ugarte-Gil MF, Alarcón GS. Epidemiology of systemic lupus erythematosus. Expert Rev Clin Immunol. 2017;13(8):799-814.
  6. Carter EE, Barr SG, Clarke AE. The global burden of SLE: prevalence, health disparities and socioeconomic impact. Nat Rev Rheumatol. 2016;12(10):605-20. Epub 2016/08/26. doi: 10.1038/nrrheum.2016.137. PubMed PMID: 27558659.
  7. Kheir JM, Guthridge CJ, Johnston JR, Adams LJ, Rasmussen A, Gross TF, et al. Unique clinical characteristics, autoantibodies and medication use in Native American patients with systemic lupus erythematosus. Lupus Sci Med. 2018;5(1):e000247. Epub 2018/03/14. doi: 10.1136/lupus-2017-000247. PubMed PMID: 29531773; PubMed Central PMCID: PMCPMC5844376.
  8. Mehta B, Luo Y, Xu J, Sammaritano L, Salmon J, Lockshin M, et al. Trends in Maternal and Fetal Outcomes Among Pregnant Women With Systemic Lupus Erythematosus in the United States: A Cross-sectional Analysis. Ann Intern Med. 2019;171(3):164-71. Epub 2019/07/10. doi: 10.7326/M19-0120. PubMed PMID: 31284305.
  9. Bitencourt N, Bermas BL. Pharmacological Approach to Managing Childhood-Onset Systemic Lupus Erythematosus During Conception, Pregnancy and Breastfeeding. Paediatr Drugs.
  10. Furie RA, Bruce IN, Dörner T, et al. Phase 2 randomized, placebo-controlled trial of dapirolizumab pegol in patients with moderate to severe active systemic lupus erythematosus (SLE). Rheumatology (Oxford).2021;60(11): 5397-407.
  11. Clinicaltrials.gov (NCT04294667). A Study to Evaluate the Efficacy and Safety of Dapirolizumab Pegol in Study Participants With Moderately to Severely Active Systemic Lupus Erythematosus (PHOENYCS GO) 2023 [cited August 2024] Available at: https://clinicaltrials.gov/ct2/show/NCT04294667. Retrieved July 25, 2024.
   
MEDIA CONTACTS:
UCB
Adriaan Snauwaert
+32 497 70 23 46
Adriaan.snauwaert@ucb.com

Biogen
Jack Cox
+1 781 464 3260
public.affairs@biogen.com

INVESTOR CONTACTS:
UCB
Antje Witte
+32 2 559 9414
Antje.Witte@ucb.com

Biogen
Tim Power
+ 1 781 464 2442
IR@biogen.com

   

Dapirolizumab Pegol Phase 3 Data in SLE Presented at the Annual European Congress of Rheumatology (EULAR) Show Improvement in Fatigue and Reduction in Disease Activity

Dapirolizumab Pegol Phase 3 Data in SLE Presented at the Annual European Congress of Rheumatology (EULAR) Show Improvement in Fatigue and Reduction in Disease Activity




Dapirolizumab Pegol Phase 3 Data in SLE Presented at the Annual European Congress of Rheumatology (EULAR) Show Improvement in Fatigue and Reduction in Disease Activity

  • Dapirolizumab pegol (DZP) showed efficacy across multiple clinical endpoints in the PHOENYCS GO study, including fatigue and measures of disease activity
  • DZP showed consistent improvements in fatigue, a common and debilitating symptom of systemic lupus erythematosus (SLE)
  • At Week 48, more individuals receiving DZP experienced no or low disease activity compared to standard of care with differences observed as early as Week 12

BRUSSELS and CAMBRIDGE, Mass., June 12, 2025 (GLOBE NEWSWIRE) — UCB (Euronext Brussels: UCB) and Biogen Inc. (NASDAQ: BIIB) today presented additional detailed results from the Phase 3 PHOENYCS GO study evaluating dapirolizumab pegol (DZP), a novel Fc-free anti-CD40L drug candidate. In the study, DZP demonstrated significant clinical improvements in disease activity in people living with moderate-to-severe systemic lupus erythematosus (SLE), as measured by the British Isles Lupus Assessment Group (BILAG)-based Composite Lupus Assessment (BICLA) at Week 48, the primary endpoint. Improvements were also seen across additional clinical measures, including fatigue and disease activity/remission. These results were presented at EULAR 2025, the European Alliance of Associations for Rheumatology’s annual meeting, in Barcelona, Spain. The safety and efficacy of DZP in SLE have not been established, and it is not approved for use in SLE by any regulatory authority worldwide. A second Phase 3 trial of dapirolizumab pegol is ongoing with the goal of confirming the results from PHOENYCS GO.

“Despite being a common manifestation of systemic lupus erythematosus, fatigue is a difficult-to-treat symptom that can severely impact a person’s quality of life, and remains a challenge to address,” said Ioannis Parodis, MD, PhD, Associate Professor of Rheumatology, Karolinska University Hospital, Sweden. “The results we observed in this Phase 3 study indicate that participants treated with dapirolizumab pegol have the potential to achieve consistent improvements in fatigue beyond the current standard of care.”

In an analysis of the impact of DZP on patient-reported fatigue in people with SLE participating in the PHOENYCS GO study, individuals receiving DZP in addition to standard of care (SOC) treatment demonstrated improvements across two fatigue measurements:

  • Improvements in Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue scores were greater in the DZP group (change from baseline, 8.9), compared with SOC alone (5.2; nominal* p=0.0024) at Week 48.
  • Using FATIGUE-PRO, a measure recently developed to capture the patient experience of fatigue in SLE, greater improvements from baseline (nominal* p<0.05) were observed in people receiving DZP compared with SOC alone in the Physical Fatigue (change from baseline difference between groups,7.6), Mental and Cognitive (5.6), and Susceptibility to Fatigue (7.8) scales at Week 48.

“Being able to address both fatigue and remission are areas of critical unmet need in lupus care, an important treatment goal is to improve the quality of life for patients as well as to reduce the long-term risk of organ damage through disease remission,” said Eric F. Morand, MBBS, Head of the Monash Health Rheumatology Unit, Monash University, Australia. “In the PHOENYCS GO study, dapirolizumab pegol has shown meaningful impact in helping patients achieve remission and low disease activity, offering the exciting possibility for improved disease control while reducing exposure to glucocorticoids. Dapirolizumab pegol has the potential to become a significant new medication for people living with SLE, as shown by the breadth of effect seen in the study and I look forward to seeing results from the second Phase 3 study.”

In an additional analysis, improvements were seen on measures of low disease activity, as measured by Low Lupus Disease Activity State (LLDAS) and disease remission, as measured by Definition of Remission in SLE (DORIS). Both measures include assessments of disease activity, in addition to a required low dose glucocorticoid intake (<7.5 mg prednisone / day in LLDAS; <5 mg prednisone / day in DORIS).

  • At Week 48, the percentage of participants achieving low disease activity in the DZP group was twice that of the SOC only group (40.9% and 19.6%, respectively; nominal* p<0.0001). As early as Week 12, greater proportions of participants receiving DZP plus SOC achieved LLDAS versus SOC alone (nominal* p<0.05). 
  • 23.6% of participants receiving DZP plus SOC achieved low disease activity in ≥50% of visits through 48 weeks compared with 15.9% receiving SOC alone (nominal* p=0.1042).
  • A higher proportion of those receiving DZP (19.2%) versus SOC alone (8.4%) also achieved DORIS at Week 48 (nominal* p=0.0056).

* Having met the primary endpoint, improvement of moderate-to-severe disease activity as assessed by achievement of BICLA after 48 weeks and the key secondary endpoint having a p-value = 0.1776, all subsequent secondary and tertiary endpoints are descriptive and nominal p-values are included.

The safety profile of DZP was generally favorable. The safety results were consistent with previous DZP studies and with that in study participants with SLE receiving an immunomodulator. A higher proportion of individuals receiving DZP plus SOC had treatment-emergent adverse events (TEAEs) compared to SOC alone (82.6% vs. 75.0%). The proportion of participants with serious TEAEs was 9.9% in participants receiving DZP plus SOC was lower as compared to 14.8% in those receiving SOC alone. Discontinuation of treatment or study participation due to TEAEs occurred in 4.7% (10) of participants receiving DZP plus SOC and 3.7% (4) of participants receiving SOC alone.

Participants from the PHOENYCS GO study (NCT04294667) will continue to be followed in a long-term open-label study. A second Phase 3 trial of dapirolizumab pegol, PHOENYCS FLY (NCT06617325) is ongoing.

About Systemic Lupus Erythematosus (SLE)
SLE is a chronic, multifactorial autoimmune disease that is caused by the activation of autoreactive T, B and antigen-presenting cells, resulting in manifestations across multiple organ systems with periods of illness or flares alternating with periods of inactivity.1 SLE can present itself in several ways including rash, arthritis, anemia, thrombocytopenia, serositis, nephritis, seizures or psychosis.2 SLE is associated with a greater risk of death from causes such as infection and cardiovascular disease.

An estimated 90% of people living with lupus are women; most begin to see symptoms between the ages of 15-55.3,4,5 Individuals from populations of African, Hispanic, Asian and Native American descent are at a greater risk of earlier onset and more aggressive disease.6,7 Pregnancy in women with SLE is high risk, with higher maternal and fetal mortality and morbidity than the general population.8,9

About Dapirolizumab Pegol
Dapirolizumab pegol is a novel investigational humanized Fc-free polyethylene glycol (PEG)-conjugated antigen-binding (Fab’) fragment. Dapirolizumab pegol inhibits CD40L signaling which has been shown to reduce B cell activation and autoantibody production, mitigate type 1 interferon (IFN) secretion, and attenuate T cell and antigen-presenting cell (APC) activation.10 Dapirolizumab pegol is presently in Phase 3 clinical development for the treatment of systemic lupus erythematosus (SLE) under a collaboration between UCB and Biogen.11

About UCB
UCB, Brussels, Belgium (www.ucb.com) is a global biopharmaceutical company focused on the discovery and development of innovative medicines and solutions to transform the lives of people living with severe diseases of the immune system or of the central nervous system. With approximately 9,000 people in approximately 40 countries, the company generated revenue of €6.1 billion in 2024. UCB is listed on Euronext Brussels (symbol: UCB).

About Biogen
Founded in 1978, Biogen is a leading biotechnology company that pioneers innovative science to deliver new medicines to transform patient’s lives and to create value for shareholders and our communities. We apply deep understanding of human biology and leverage different modalities to advance first-in-class treatments or therapies that deliver superior outcomes. Our approach is to take bold risks, balanced with return on investment to deliver long-term growth.

The company routinely posts information that may be important to investors on its website at www.biogen.com. Follow us on social media – FacebookLinkedInXYouTube.

Forward looking statements UCB
This document contains forward-looking statements, including, without limitation, statements containing the words “potential”, “believes”, “anticipates”, “expects”, “intends”, “plans”, “seeks”, “estimates”, “may”, “will”, “continue” and similar expressions. These forward-looking statements are based on current plans, estimates and beliefs of management. All statements, other than statements of historical facts, are statements that could be deemed forward-looking statements, including estimates of revenues, operating margins, capital expenditures, cash, other financial information, expected legal, arbitration, political, regulatory or clinical results or practices and other such estimates and results. By their nature, such forward-looking statements are not guaranteeing future performance and are subject to known and unknown risks, uncertainties, and assumptions which might cause the actual results, financial condition, performance or achievements of UCB, or industry results, to be materially different from any future results, performance, or achievements expressed or implied by such forward-looking statements contained in this document. Important factors that could result in such differences include but are not limited to: global spread and impacts of wars, pandemics and terrorism, the general geopolitical environment, climate change, changes in general economic, business and competitive conditions, the inability to obtain necessary regulatory approvals or to obtain them on acceptable terms or within expected timing, costs associated with research and development, changes in the prospects for products in the pipeline or under development by UCB, effects of future judicial decisions or governmental investigations, safety, quality, data integrity or manufacturing issues, supply chain disruption and business continuity risks; potential or actual data security and data privacy breaches, or disruptions of our information technology systems, product liability claims, challenges to patent protection for products or product candidates, competition from other products including biosimilars or disruptive technologies/business models, changes in laws or regulations, exchange rate fluctuations, changes or uncertainties in tax laws or the administration of such laws, and hiring, retention and compliance of its employees. There is no guarantee that new product candidates will be discovered or identified in the pipeline, or that new indications for existing products will be developed and approved. Movement from concept to commercial product is uncertain; preclinical results do not guarantee safety and efficacy of product candidates in humans. So far, the complexity of the human body cannot be reproduced in computer models, cell culture systems or animal models. The length of the timing to complete clinical trials and to get regulatory approval for product marketing has varied in the past and UCB expects similar unpredictability going forward. Products or potential products which are the subject of partnerships, joint ventures or licensing collaborations may be subject to disputes between the partners or may prove to be not as safe, effective or commercially successful as UCB may have believed at the start of such partnership. UCB’s efforts to acquire other products or companies and to integrate the operations of such acquired companies may not be as successful as UCB may have believed at the moment of acquisition. Also, UCB or others could discover safety, side effects or manufacturing problems with its products and/or devices after they are marketed. The discovery of significant problems with a product similar to one of UCB’s products that implicate an entire class of products may have a material adverse effect on sales of the entire class of affected products. Moreover, sales may be impacted by international and domestic trends toward managed care and health care cost containment, including pricing pressure, political and public scrutiny, customer and prescriber patterns or practices, and the reimbursement policies imposed by third-party payers as well as legislation affecting biopharmaceutical pricing and reimbursement activities and outcomes. Finally, a breakdown, cyberattack or information security breach could compromise the confidentiality, integrity and availability of UCB’s data and systems.

Given these uncertainties, the public is cautioned not to place any undue reliance on such forward-looking statements. These forward-looking statements are made only as of the date of this document, and do not reflect any potential impacts from the evolving event or risk as mentioned above as well as any other adversity, unless indicated otherwise. The company continues to follow the development diligently to assess the financial significance of these events, as the case may be, to UCB.

UCB expressly disclaims any obligation to update any forward-looking statements in this document, either to confirm the actual results or to report or reflect any change in its forward-looking statements with regard thereto or any change in events, conditions or circumstances on which any such statement is based, unless such statement is required pursuant to applicable laws and regulations.

Biogen Safe Harbor
This press release contains forward-looking statements, relating to: our strategy and plans; potential of, and expectations for, our commercial business and pipeline programs; capital allocation and investment strategy; clinical development programs, clinical trials, and data readouts and presentations; regulatory discussions, submissions, filings, and approvals; the potential benefits, safety, and efficacy of our and our collaboration partners’ products and investigational therapies; the anticipated benefits and potential of investments, actions to improve risk profile and productivity of R&D pipeline, collaborations, and business development activities; and our future financial and operating results. These forward-looking statements may be accompanied by such words as “aim,” “anticipate,” “assume,” “believe,” “contemplate,” “continue,” “could,” “estimate,” “expect,” “forecast,” “goal,” “guidance,” “hope,” “intend,” “may,” “objective,” “outlook,” “plan,” “possible,” “potential,” “predict,” “project,” “prospect,” “should,” “target,” “will,” “would,” and other words and terms of similar meaning. Drug development and commercialization involve a high degree of risk, and only a small number of research and development programs result in commercialization of a product. Results in early-stage clinical trials may not be indicative of full results or results from later stage or larger scale clinical trials and do not ensure regulatory approval. You should not place undue reliance on these statements. Given their forward-looking nature, these statements involve substantial risks and uncertainties that may be based on inaccurate assumptions and could cause actual results to differ materially from those reflected in such statements. This press release includes, among others, forward-looking statements including: that Biogen is building on a new foundation with the goal of long-term sustainable growth in its commercial portfolio; the multi-billion dollar potential of its late-stage pipeline; that we believe there remains a significant long-term opportunity for our ongoing product launches including LEQEMBI; that we believe that continued execution against these key strategic elements, as well as a disciplined approach to business development, will allow us to generate long-term value for our shareholders by bringing innovative medicines to patients; and all statements and information under the heading “Full Year 2025 Financial Guidance”. These forward-looking statements are based on management’s current beliefs and assumptions and on information currently available to management. Given their nature, we cannot assure that any outcome expressed in these forward looking statements will be realized in whole or in part.

We caution that these statements are subject to risks and uncertainties, many of which are outside of our control and could cause future events or results to be materially different from those stated or implied in this document, including, among others, factors relating to: our substantial dependence on revenue from our products and other payments under licensing, collaboration, acquisition or divestiture agreements; uncertainty of long-term success in developing, licensing, or acquiring other product candidates or additional indications for existing products; expectations, plans and prospects relating to product approvals, approvals of additional indications for our existing products, sales, pricing, growth, reimbursement and launch of our marketed and pipeline products; the potential impact of increased product competition in the biopharmaceutical and healthcare industry, as well as any other markets in which we compete, including increased competition from new originator therapies, generics, prodrugs and biosimilars of existing products and products approved under abbreviated regulatory pathways; our ability to effectively implement our corporate strategy; the successful execution of our strategic and growth initiatives, including acquisitions; the drivers for growing our business; difficulties in obtaining and maintaining adequate coverage, pricing, and reimbursement for our products; the drivers for growing our business, including our dependence on collaborators and other third parties for the development, regulatory approval, and commercialization of products and other aspects of our business, which are outside of our full control; risks associated with current and potential future healthcare reforms; risks related to commercialization of biosimilars, which is subject to such risks related to our reliance on third parties, intellectual property, competitive and market challenges and regulatory compliance; failure to obtain, protect, and enforce our data, intellectual property, and other proprietary rights and the risks and uncertainties relating to intellectual property claims and challenges; the risk that positive results in a clinical trial may not be replicated in subsequent or confirmatory trials or success in early stage clinical trials may not be predictive of results in later stage or large scale clinical trials or trials in other potential indications; risks associated with clinical trials, including our ability to adequately manage clinical activities, unexpected concerns that may arise from additional data or analysis obtained during clinical trials, regulatory authorities may require additional information or further studies, or may fail to approve or may delay approval of our drug candidates; the occurrence of adverse safety events, restrictions on use with our products, or product liability claims; risks relating to technology, including our incorporation of new technologies such as artificial intelligence into some of our processes; risks related to use of information technology systems and potential impacts of any breakdowns, interruptions, invasions, corruptions, data breaches, destructions and/or other cybersecurity incidents of our systems or those of connected and/or third-party systems; problems with our manufacturing capacity, including our ability to manufacture products efficiently or adequately address global bulk supply risks; risks relating to management, personnel and other organizational changes, including our ability to attracting, retaining and motivating qualified individuals; risks related to the failure to comply with current and new legal and regulatory requirements, including judicial decisions, accounting standards, and tariff or trade restrictions; the risks of doing business internationally, including geopolitical tensions, acts of war and largescale crises; risks relating to investment in our manufacturing capacity; risks relating to the distribution and sale by third parties of counterfeit or unfit versions of our products; risks relating to the use of social media for our business, results of operations and financial condition; fluctuations in our operating results; risks related to investment in properties; risks relating to access to capital and credit markets to finance our present and future operations and business initiatives and obtain funding for such activities on favorable terms; risks related to indebtedness; the market, interest, and credit risks associated with our investment portfolio; risks relating to share repurchase programs; change in control provisions in certain of our collaboration agreements; fluctuations in our effective tax rate and obligations in various jurisdictions in which we are subject to taxation; environmental risks; and any other risks and uncertainties that are described in other reports we have filed with the U.S. Securities and Exchange Commission.

These statements speak only as of the date of this press release and are based on information and estimates available to us at this time. Should known or unknown risks or uncertainties materialize or should underlying assumptions prove inaccurate, actual results could vary materially from past results and those anticipated, estimated or projected. Investors are cautioned not to put undue reliance on forward-looking statements. A further list and description of risks, uncertainties and other matters can be found in our Annual Report on Form 10-K for the fiscal year ended December 31, 2024 and in our subsequent reports on Form 10-Q and Form 10-K, in each case including in the sections thereof captioned “Note Regarding Forward-Looking Statements” and “Item 1A. Risk Factors,” and in our subsequent reports on Form 8-K. Except as required by law, we do not undertake any obligation to publicly update any forward-looking statements whether as a result of any new information, future events, changed circumstances or otherwise.

References:

  1. Tselios K, Gladman DD, Touma Z, et al. Disease course patterns in systemic lupus erythematosus. Lupus. 2019;28(1):114-122.
  2. Fanouriakis A, Tziolos N, Bertsias G, et al. Update οn the diagnosis and management of systemic lupus erythematosus. Ann Rheum Dis. 2021;80(1):14-25. doi:10.1136/annrheumdis-2020-218272
  3. Petri M. Epidemiology of systemic lupus erythematosus. Best Pract Res Clin Rheumatol. 2002;16(5):847-58. Epub 2002/12/11. doi: 10.1053/berh.2002.0259. PubMed PMID: 12473278.
  4. Rees F, Doherty M, Grainge M, Davenport G, Lanyon P, Zhang W. The incidence and prevalence of systemic lupus erythematosus in the UK, 1999-2012. Ann Rheum Dis. 2016;75(1):136-41. Epub 2014/10/01. doi: 10.1136/annrheumdis-2014-206334. PubMed PMID: 25265938; PubMed Central PMCID: PMCPMC4717400.
  5. Pons-Estel GJ, Ugarte-Gil MF, Alarcón GS. Epidemiology of systemic lupus erythematosus. Expert Rev Clin Immunol. 2017;13(8):799-814.
  6. Carter EE, Barr SG, Clarke AE. The global burden of SLE: prevalence, health disparities and socioeconomic impact. Nat Rev Rheumatol. 2016;12(10):605-20. Epub 2016/08/26. doi: 10.1038/nrrheum.2016.137. PubMed PMID: 27558659.
  7. Kheir JM, Guthridge CJ, Johnston JR, Adams LJ, Rasmussen A, Gross TF, et al. Unique clinical characteristics, autoantibodies and medication use in Native American patients with systemic lupus erythematosus. Lupus Sci Med. 2018;5(1):e000247. Epub 2018/03/14. doi: 10.1136/lupus-2017-000247. PubMed PMID: 29531773; PubMed Central PMCID: PMCPMC5844376.
  8. Mehta B, Luo Y, Xu J, Sammaritano L, Salmon J, Lockshin M, et al. Trends in Maternal and Fetal Outcomes Among Pregnant Women With Systemic Lupus Erythematosus in the United States: A Cross-sectional Analysis. Ann Intern Med. 2019;171(3):164-71. Epub 2019/07/10. doi: 10.7326/M19-0120. PubMed PMID: 31284305.
  9. Bitencourt N, Bermas BL. Pharmacological Approach to Managing Childhood-Onset Systemic Lupus Erythematosus During Conception, Pregnancy and Breastfeeding. Paediatr Drugs.
  10. Furie RA, Bruce IN, Dörner T, et al. Phase 2 randomized, placebo-controlled trial of dapirolizumab pegol in patients with moderate to severe active systemic lupus erythematosus (SLE). Rheumatology (Oxford).2021;60(11): 5397-407.
  11. Clinicaltrials.gov (NCT04294667). A Study to Evaluate the Efficacy and Safety of Dapirolizumab Pegol in Study Participants With Moderately to Severely Active Systemic Lupus Erythematosus (PHOENYCS GO) 2023 [cited August 2024] Available at: https://clinicaltrials.gov/ct2/show/NCT04294667. Retrieved July 25, 2024.
   
MEDIA CONTACTS:
UCB
Adriaan Snauwaert
+32 497 70 23 46
Adriaan.snauwaert@ucb.com

Biogen
Jack Cox
+1 781 464 3260
public.affairs@biogen.com

INVESTOR CONTACTS:
UCB
Antje Witte
+32 2 559 9414
Antje.Witte@ucb.com

Biogen
Tim Power
+ 1 781 464 2442
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Nusano Announces Breakthrough HALEU Program Expected to Produce Up to 350 Metric Tons of Fuel Annually for Advanced Nuclear Reactors

Nusano Announces Breakthrough HALEU Program Expected to Produce Up to 350 Metric Tons of Fuel Annually for Advanced Nuclear Reactors




Nusano Announces Breakthrough HALEU Program Expected to Produce Up to 350 Metric Tons of Fuel Annually for Advanced Nuclear Reactors

  • Reservations available now, commercial production deliveries begin Q1 2027
  • Proprietary process with reduced environmental impacts
  • Efficient domestic supply chain needed to support projected growth in energy demand from increased electrification and energy-intensive industries
  • Critical step toward energy independence being built in Utah

WEST VALLEY CITY, Utah, June 11, 2025 (GLOBE NEWSWIRE) — Nusano, a physics company transforming radioisotope production and enabling next-generation energy solutions, announced today the launch of a High-Assay Low-Enriched Uranium (HALEU) production program to support the need for new, sustainable energy solutions and small modular nuclear reactor (SMR) infrastructure in the United States.

“Domestic HALEU production is essential to enabling sustainable energy solutions,” said Chris Lowe, CEO of Nusano. “Through our HALEU program, Nusano is working to stabilize supply chains, significantly drive down the cost of fuel, and enable the deployment of advanced nuclear energy on a scale needed to support advancements in AI, data centers, electrification, and modern logistics.”

The Nusano HALEU program is a critical step toward establishing a 100% domestic fuel supply for next-generation energy solutions and firmly positions Utah to lead the nation in advanced fuel production.

The launch also aligns with Utah Governor Cox’s broader Operation Gigawatt strategy for energy abundance founded in a secure, stable and independent energy ecosystem, as well as recent federal government directives to build a domestic uranium enrichment supply.

“Utah has long played a role in the nation’s nuclear fuel supply chain,” said Emy Lesofski, energy advisor to the governor and director of the Utah Office of Energy Development. “As we explore how to meet the energy needs of the future, we’re focused on energy resources that are reliable and clean—many of which need HALEU. The next generation of advanced nuclear technologies need fuel and companies like Nusano can fill that need safely and responsibly and we look forward to opportunities to partner with them at the Utah San Rafael Energy Lab.”

Proprietary System Enables Mass Production

Nusano brings new solutions to HALEU production, making it the first private company in the United States to offer a scalable, commercial solution for nuclear fuel with a focus on HALEU.

The Nusano HALEU program utilizes a proprietary process capable of quick, cost-effective production and scale up. Compared to modern centrifuge facilities, the Nusano solution is more energy efficient, easier to operate, and can produce on demand—overcoming the most common issues associated with gas centrifuge machines. Additionally, Nusano’s process eliminates the need for uranium hexafluoride, a bottleneck in current nuclear fuel cycles.

These capabilities position Nusano to play a critical role stabilizing the domestic HALEU supply chain while also reducing environmental impacts. A stable, domestic fuel supply will ultimately decrease U.S. dependence on foreign suppliers, increasing our national security and economic stability.

Nusano intends to produce initial commercial samples of HALEU by Q4 2026 and begin large scale production in Q1 2027. A single Nusano system will be capable of producing more than 50 metric tons of HALEU annually — with a small footprint, lower initial capital investment, and lower operating costs than other proposed solutions. Nusano plans to scale its production to approximately 350 metric tons annually by 2029.

America’s Unmet Energy Needs

The United States Department of Energy estimates that by 2035, the country will need 50 metric tons per year of HALEU to support its commercial nuclear power industry, escalating to 500 metric tons per year by 2050. Current solutions are capable of providing less than 1% of that supply, which makes this project essential if America is to regain its energy independence and stabilize its economies.

HALEU is a crucial fuel for small modular reactors (SMRs) and other advanced nuclear reactor designs. Unlike traditional reactors, which use uranium-235 (U-235) enriched to about 3-5%, HALEU is enriched to between 5-19.9% U-235, which allows for greater fuel efficiency, longer operational cycles, and more compact reactor designs. The Nuclear Regulatory Commission (NRC) oversees the establishment of new uranium facilities and the uranium enrichment process. The U.S. has been safely enriching uranium for decades and this project will continue the country’s successful and responsible production of nuclear fuel.

About Nusano
Nusano is a privately held physics company committed to: bringing supply stability and innovation to the rapidly emerging and critically undersupplied medical radioisotopes market, serving industrial and commercial markets dependent on reliable access to high quality radioisotopes for their products and services, and enabling next-generation energy solutions. Nusano’s breakthrough technologies are poised to help supply the fight against cancer and enable innovation across multiple industries. For more, please visit nusano.com.

Contacts:

Nusano Announces Breakthrough HALEU Program Expected to Produce Up to 350 Metric Tons of Fuel Annually for Advanced Nuclear Reactors

Nusano Announces Breakthrough HALEU Program Expected to Produce Up to 350 Metric Tons of Fuel Annually for Advanced Nuclear Reactors




Nusano Announces Breakthrough HALEU Program Expected to Produce Up to 350 Metric Tons of Fuel Annually for Advanced Nuclear Reactors

  • Reservations available now, commercial production deliveries begin Q1 2027
  • Proprietary process with reduced environmental impacts
  • Efficient domestic supply chain needed to support projected growth in energy demand from increased electrification and energy-intensive industries
  • Critical step toward energy independence being built in Utah

WEST VALLEY CITY, Utah, June 11, 2025 (GLOBE NEWSWIRE) — Nusano, a physics company transforming radioisotope production and enabling next-generation energy solutions, announced today the launch of a High-Assay Low-Enriched Uranium (HALEU) production program to support the need for new, sustainable energy solutions and small modular nuclear reactor (SMR) infrastructure in the United States.

“Domestic HALEU production is essential to enabling sustainable energy solutions,” said Chris Lowe, CEO of Nusano. “Through our HALEU program, Nusano is working to stabilize supply chains, significantly drive down the cost of fuel, and enable the deployment of advanced nuclear energy on a scale needed to support advancements in AI, data centers, electrification, and modern logistics.”

The Nusano HALEU program is a critical step toward establishing a 100% domestic fuel supply for next-generation energy solutions and firmly positions Utah to lead the nation in advanced fuel production.

The launch also aligns with Utah Governor Cox’s broader Operation Gigawatt strategy for energy abundance founded in a secure, stable and independent energy ecosystem, as well as recent federal government directives to build a domestic uranium enrichment supply.

“Utah has long played a role in the nation’s nuclear fuel supply chain,” said Emy Lesofski, energy advisor to the governor and director of the Utah Office of Energy Development. “As we explore how to meet the energy needs of the future, we’re focused on energy resources that are reliable and clean—many of which need HALEU. The next generation of advanced nuclear technologies need fuel and companies like Nusano can fill that need safely and responsibly and we look forward to opportunities to partner with them at the Utah San Rafael Energy Lab.”

Proprietary System Enables Mass Production

Nusano brings new solutions to HALEU production, making it the first private company in the United States to offer a scalable, commercial solution for nuclear fuel with a focus on HALEU.

The Nusano HALEU program utilizes a proprietary process capable of quick, cost-effective production and scale up. Compared to modern centrifuge facilities, the Nusano solution is more energy efficient, easier to operate, and can produce on demand—overcoming the most common issues associated with gas centrifuge machines. Additionally, Nusano’s process eliminates the need for uranium hexafluoride, a bottleneck in current nuclear fuel cycles.

These capabilities position Nusano to play a critical role stabilizing the domestic HALEU supply chain while also reducing environmental impacts. A stable, domestic fuel supply will ultimately decrease U.S. dependence on foreign suppliers, increasing our national security and economic stability.

Nusano intends to produce initial commercial samples of HALEU by Q4 2026 and begin large scale production in Q1 2027. A single Nusano system will be capable of producing more than 50 metric tons of HALEU annually — with a small footprint, lower initial capital investment, and lower operating costs than other proposed solutions. Nusano plans to scale its production to approximately 350 metric tons annually by 2029.

America’s Unmet Energy Needs

The United States Department of Energy estimates that by 2035, the country will need 50 metric tons per year of HALEU to support its commercial nuclear power industry, escalating to 500 metric tons per year by 2050. Current solutions are capable of providing less than 1% of that supply, which makes this project essential if America is to regain its energy independence and stabilize its economies.

HALEU is a crucial fuel for small modular reactors (SMRs) and other advanced nuclear reactor designs. Unlike traditional reactors, which use uranium-235 (U-235) enriched to about 3-5%, HALEU is enriched to between 5-19.9% U-235, which allows for greater fuel efficiency, longer operational cycles, and more compact reactor designs. The Nuclear Regulatory Commission (NRC) oversees the establishment of new uranium facilities and the uranium enrichment process. The U.S. has been safely enriching uranium for decades and this project will continue the country’s successful and responsible production of nuclear fuel.

About Nusano
Nusano is a privately held physics company committed to: bringing supply stability and innovation to the rapidly emerging and critically undersupplied medical radioisotopes market, serving industrial and commercial markets dependent on reliable access to high quality radioisotopes for their products and services, and enabling next-generation energy solutions. Nusano’s breakthrough technologies are poised to help supply the fight against cancer and enable innovation across multiple industries. For more, please visit nusano.com.

Contacts:

Mesoblast Maintains Momentum With FDA on Accelerated Approval Pathway for Revascor® in Ischemic Heart Failure and Label Extension for Ryoncil® in Adults With GvHD

Mesoblast Maintains Momentum With FDA on Accelerated Approval Pathway for Revascor® in Ischemic Heart Failure and Label Extension for Ryoncil® in Adults With GvHD




Mesoblast Maintains Momentum With FDA on Accelerated Approval Pathway for Revascor® in Ischemic Heart Failure and Label Extension for Ryoncil® in Adults With GvHD

NEW YORK, June 11, 2025 (GLOBE NEWSWIRE) — Mesoblast (Nasdaq:MESO; ASX:MSB), global leader in allogeneic cellular medicines for inflammatory diseases, today provided an update on continued momentum with United States Food and Drug Administration (FDA) regarding both accelerated approval pathway for Revascor® (rexlemestrocel-L) in the treatment of patients with ischemic chronic heart failure with reduced ejection fraction (HFrEF) and inflammation, and label extension for Ryoncil® (remestemcel-L-rknd) in adults with steroid refractory acute graft versus host disease (SR-aGvHD).

In the first week of June, Mesoblast held a Type B meeting with FDA under its Regenerative Medicines Advanced Therapy (RMAT) designation for REVASCOR to discuss components of a potential filing for a Biologics License Application (BLA). There was general alignment on items regarding chemistry, manufacturing & controls (CMC), potency assays for commercial product release, and proposed design and primary endpoint for the confirmatory trial post-approval. The Company will await the final minutes from FDA in order to provide detailed feedback and timelines for potential filing.

In early July, Mesoblast has an upcoming meeting with FDA to discuss a pivotal trial of Ryoncil® in adults with SR-aGvHD. This trial will be conducted with the NIH-funded Bone Marrow Transplant Clinical Trials Network (BMT-CTN), the objective being to extend the product’s label from children to adults with SR-aGvHD. Ryoncil® is the first and only mesenchymal stromal cell product approved by the FDA for any indication.

Ryoncil® became commercially available for purchase in the United States on March 28, 2025, within one quarter of receiving FDA approval to treat children with SR-aGvHD. More than 20 transplant centers will have been onboarded by the end of the quarter, exceeding the company’s expectations at product launch. Mesoblast has continued to expand coverage for Ryoncil® to over 220 million US lives insured by commercial and government payers. To date, 37 of the 51 States provide fee-for-service Medicaid coverage for Ryoncil® through Orphan Drug Lists or medical exception / prior authorization (PA) process. The remainder will come online July 1, 2025, with mandatory coverage for all 24 million lives.

“We are very pleased with the momentum of interactions with FDA on both our cardiac and GvHD programs,” said Mesoblast Chief Executive Dr. Silviu Itescu. “We are also encouraged by the strength of the of the Ryoncil® commercial launch, the rate of hospital onboarding, physician adoption, and payor coverage exceeding our expectations in the ten weeks since commercial launch. We will be providing an update on sales of Ryoncil® in our quarterly activities report at the end of next month.”  

About Mesoblast
Mesoblast (the Company) is a world leader in developing allogeneic (off-the-shelf) cellular medicines for the treatment of severe and life-threatening inflammatory conditions. The therapies from the Company’s proprietary mesenchymal lineage cell therapy technology platform respond to severe inflammation by releasing anti-inflammatory factors that counter and modulate multiple effector arms of the immune system, resulting in significant reduction of the damaging inflammatory process.

Mesoblast’s RYONCIL® (remestemcel-L) for the treatment of steroid-refractory acute graft versus host disease (SR-aGvHD) in pediatric patients 2 months and older is the first FDA approved mesenchymal stromal cell (MSC) therapy. Please see the full Prescribing Information at www.ryoncil.com.

Mesoblast is committed to developing additional cell therapies for distinct indications based on its remestemcel-L and rexlemestrocel-L allogeneic stromal cell technology platforms. RYONCIL is being developed for additional inflammatory diseases including SR-aGvHD in adults and biologic-resistant inflammatory bowel disease. Rexlemestrocel-L is being developed for heart failure and chronic low back pain. The Company has established commercial partnerships in Japan, Europe and China.

About Mesoblast intellectual property: Mesoblast has a strong and extensive global intellectual property portfolio, with over 1,000 granted patents or patent applications covering mesenchymal stromal cell compositions of matter, methods of manufacturing and indications. These granted patents and patent applications are expected to provide commercial protection extending through to at least 2041 in major markets.

About Mesoblast manufacturing: The Company’s proprietary manufacturing processes yield industrial-scale, cryopreserved, off-the-shelf, cellular medicines. These cell therapies, with defined pharmaceutical release criteria, are planned to be readily available to patients worldwide.

Mesoblast has locations in Australia, the United States and Singapore and is listed on the Australian Securities Exchange (MSB) and on the Nasdaq (MESO). For more information, please see www.mesoblast.com, LinkedIn: Mesoblast Limited and X: @Mesoblast

Forward-Looking Statements
This press release includes forward-looking statements that relate to future events or our future financial performance and involve known and unknown risks, uncertainties and other factors that may cause our actual results, levels of activity, performance or achievements to differ materially from any future results, levels of activity, performance or achievements expressed or implied by these forward-looking statements. We make such forward-looking statements pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995 and other federal securities laws. Forward-looking statements should not be read as a guarantee of future performance or results, and actual results may differ from the results anticipated in these forward-looking statements, and the differences may be material and adverse. Forward-looking statements include, but are not limited to, statements about: the initiation, timing, progress and results of Mesoblast’s preclinical and clinical studies, and Mesoblast’s research and development programs; Mesoblast’s ability to advance product candidates into, enroll and successfully complete, clinical studies, including multi-national clinical trials; Mesoblast’s ability to advance its manufacturing capabilities; the timing or likelihood of regulatory filings and approvals, manufacturing activities and product marketing activities, if any; the commercialization of Mesoblast’s RYONCIL for pediatric SR-aGVHD and any other product candidates, if approved; regulatory or public perceptions and market acceptance surrounding the use of stem-cell based therapies; the potential for Mesoblast’s product candidates, if any are approved, to be withdrawn from the market due to patient adverse events or deaths; the potential benefits of strategic collaboration agreements and Mesoblast’s ability to enter into and maintain established strategic collaborations; Mesoblast’s ability to establish and maintain intellectual property on its product candidates and Mesoblast’s ability to successfully defend these in cases of alleged infringement; the scope of protection Mesoblast is able to establish and maintain for intellectual property rights covering its product candidates and technology; estimates of Mesoblast’s expenses, future revenues, capital requirements and its needs for additional financing; Mesoblast’s financial performance; developments relating to Mesoblast’s competitors and industry; and the pricing and reimbursement of Mesoblast’s product candidates, if approved. You should read this press release together with our risk factors, in our most recently filed reports with the SEC or on our website. Uncertainties and risks that may cause Mesoblast’s actual results, performance or achievements to be materially different from those which may be expressed or implied by such statements, and accordingly, you should not place undue reliance on these forward-looking statements. We do not undertake any obligations to publicly update or revise any forward-looking statements, whether as a result of new information, future developments or otherwise.

Release authorized by the Chief Executive.

For more information, please contact:

Corporate Communications / Investors
Paul Hughes
T: +61 3 9639 6036
 
Media – Global
Allison Worldwide
Emma Neal
T: +1 603 545 4843
E: emma.neal@allisonworldwide.com
 
Media – Australia
BlueDot Media
Steve Dabkowski
T: +61 419 880 486
E: steve@bluedot.net.au