This is a long-term follow-up study for participants treated with Galapagos (GLPG) CAR T-cell therapies to evaluate the long-term safety and efficacy of GLPG CAR T-cell products for 15 years post infusion.
Per Health Authorities guidelines for gene therapy medicinal products that utilize integrating vectors (e.g. lentiviral vectors), long term safety and efficacy follow up of treated patients is required. The purpose of this study is to monitor all participants exposed to GLPG CAR T-cell therapies for 15 years following their last CAR T-cell infusion to assess the risk of delayed adverse events (AEs) and the long-term benefit/risk profile and to monitor for replication-competent lentivirus (RCL) and CAR-T cell persistence.

Start Date09 Sep 2024

Sponsor / Collaborator

Lakefront Biotherapeutics

CTIS2023-504422-19-00

/ CompletedPhase 1

A study evaluating the safety, tolerability, and efficacy of GLPG5101 in adults with treatment-refractory active systemic lupus erythematosus

Start Date30 Nov 2023

Sponsor / Collaborator

Lakefront Biotherapeutics

NCT06561425

/ TerminatedPhase 1/2

A Phase I/II, Multicenter Study Evaluating the Feasibility, Safety, and Efficacy of Point-of-care Manufactured GLPG5101 (19CP02) in Subjects With Relapsed/Refractory B-cell Non-Hodgkin Lymphoma

This study is evaluating whether an experimental treatment called GLPG5101 helps to treat non-Hodgkin lymphoma (NHL) and if it is safe to use.
This study will be carried out in 2 phases:
* The first phase is to see which doses of GLPG5101 work best with the least number of side effects.
* In the second phase, participants will receive the selected dose(s) based on the results in the first phase.

Start Date09 Mar 2022

Sponsor / Collaborator

Lakefront Biotherapeutics

100 Clinical Results associated with GLPG-5101

100 Translational Medicine associated with GLPG-5101

100 Patents (Medical) associated with GLPG-5101

Literatures (Medical) associated with GLPG-5101

01 Dec 2024·HemaSphere

Kinetics of lymphocytosis in naïve chronic lymphocytic leukemia patients treated with covalent Bruton's tyrosine kinase inhibitors: An Italian multicenter real‐life experience

Article

Author: Mosca, Antonio ; Stirparo, Luca ; Trentin, Livio ; Mattiello, Veronica ; Autore, Francesco ; Innao, Vanessa ; Martini, Francesca ; Tomasso, Annamaria ; Tedeschi, Alessandra ; Sportoletti, Paolo ; Pasquale, Raffaella ; Sanna, Alessandro ; Bellesi, Silvia ; Innocenti, Idanna ; Laurenti, Luca ; Murru, Roberta ; Catania, Gioacchino ; Fresa, Alberto ; Zamprogna, Giulia ; Laureana, Roberta ; Ghia, Paolo ; Galitzia, Andrea ; Patti, Caterina ; Visentin, Andrea ; Farina, Giuliana ; Martino, Enrica A. ; Varettoni, Marzia ; Del Principe, Maria I. ; Galli, Eugenio ; Cappello, Emilia ; Scarfò, Lydia ; Montalbano, Maria C. ; Sangiorgi, Eugenio ; Coscia, Marta ; Morelli, Francesca ; Gentile, Massimo ; Chiriu, Sabrina ; Benintende, Giulia

Chronic lymphocytic leukemia (CLL) therapy has recently undergone a revolution with the introduction of a new class of drugs: covalent Bruton's tyrosine kinase inhibitors (cBTKi), paving the way for a chemotherapy-free approach.1-4 Presently, cBTKi can be utilized in the first line of CLL management, thanks to the results of phase III clinical trials such as RESONATE-2 and ELEVATE-TN, which demonstrated the superiority of Ibrutinib over chemotherapy with chlorambucil5 and acalabrutinib over chemoimmunotherapy with chlorambucil + obinutuzumab,6 in terms of progression-free survival (PFS) in both cases. Ibrutinib exhibited better PFS, overall survival (OS), and overall response rate than the monoclonal anti-CD20 antibody ofatumumab in previously treated patients with CLL.7 Additionally, the ASCEND study, another phase III randomized clinical trial, demonstrated that acalabrutinib significantly improved PFS compared to a physician's choice of Idelalisib + rituximab or bendamustine + rituximab, in patients with relapsed/refractory CLL.8 BTK plays a pivotal role in B-cell receptor (BCR) signal transduction,9 stimulating important pathways such as NFKB10, 11 and CXCR4.12 Consequently, BTK is involved in B-cell survival, proliferation, and adhesion, while its activation promotes B-cell proliferation.13 Paradoxically, ibrutinib has shown to increase absolute lymphocyte count (ALC) in the initial phase of treatment, regardless of previous lines of therapy. Ibrutinib-induced lymphocytosis may be explained by the redistribution of lymphocytes from neoplastic nodal compartments into the peripheral blood.14 Furthermore, it was noted that Ibrutinib-induced lymphocytosis is transient in most patients, resolving within 8 months, but may rarely persist for over 12 months without impacting survival.14 This evidence led to the introduction of a new criterion in the assessment of CLL therapy response: partial response with lymphocytosis (PR-L).15 Subsequently, the kinetics of lymphocytosis in CLL treated with ibrutinib monotherapy showed that lymphocytosis occurred in the majority of patients treated in first line was higher in immunoglobulin variable heavy chain (IGHV) mutated settings and resolved in 95% of patients after a median of 18.4 months.16 Little is known about frequency and duration of lymphocytosis in patients treated with the second-generation cBTKi acalabrutinib. Therefore, the aim of this study is to outline the kinetics of lymphocytosis in CLL patients treated with acalabrutinib compared to ibrutinib. We conducted a multicenter retrospective real-life study involving 17 Italian centers. The study was carried out according to the Helsinki Declaration, Good Clinical Practice, and the applicable national regulations and approved by the local ethical committee. All patients provided written informed consent. The primary endpoint was to define the kinetics of lymphocytosis in naïve patients treated with acalabrutinib monotherapy compared to those treated with ibrutinib, over a 12-month observational period. We included patients receiving therapy with ibrutinib or acalabrutinib, in the first line at the target dose of 420 mg/day for ibrutinib and 200 mg/day for acalabrutinib. We enrolled 204 patients divided into two arms: the ibrutinib arm (n = 136) and the acalabrutinib arm (n = 68). The median age was 73 years for the ibrutinib arm and 71 for the acalabrutinib arm. For each patient, we defined the clinical and biological features of disease at baseline, including IGHV mutational status, chromosomal abnormalities by FISH, and molecular biology mutations. At baseline, we considered stage, lymph node involvement, and the presence of splenomegaly. Clinical characteristics and molecular features are reported in Table 1. Subsequently, we assessed the ALC through serial blood count tests at baseline and at different time points: 2 weeks, 1 month, 2 months, 3 months, 6 months, 9 months, and 12 months after the start of treatment. We calculated the median ALC, expressed both in terms of cells/mm3 and as a percentage compared to the baseline, at each time point. For statistical analysis, we used the Mann–Whitney test to compare median ALC values, considering only those with a p-value < 0.05 as statistically significant. We observed that in the ibrutinib group (IBR), the median ALC at baseline was 63,270/mm3, while in the acalabrutinib group (ACALA), it was 82,905/mm3. Median ALC peaked at two weeks in both arms and then immediately began to decline, reaching the baseline level at 1 month. From Month 1 to Month 12 ALC steadily declined, reaching a normal lymphocyte count (<4000/mm3, according to iwCLL guidelines) at Month 12 for ACALA. ACALA exhibited lower ALC from Month 6 to Month 12 compared to IBR. At Month 12, IBR did not reach the median normal lymphocyte count. For each data point, we calculated the percentage compared to the baseline, which provides a more representative view of lymphocyte count changes at each time point of the study (Supporting Information S1: Table S1). Subsequently, we examined the decline in lymphocytosis to understand if well-defined clinical or biological features could independently impact the kinetics of lymphocyte count in both arms. ACALA had less patients with a molecularly unfavorable prognostic profile; despite this, it seems to have no impact on the kinetics of lymphocytosis in both arms according to our subanalysis (data not shown). IGHV mutational status did not differ between the two arms, but IGHV mutational status had an impact on the lymphocytosis decline. We considered separately cases with unmutated and mutated IGHV genes for IBR and ACALA. They exhibited a similar increment at Day 14 followed by a steady decline. Unmutated IGHV curves of IBR and ACALA overlapped until Month 12. Mutated IGHV for IBR and ACALA followed a similar pattern until Month 6, after which ACALA exhibited a stronger reduction, albeit only as a percentage of baseline, due to ACALA starting from a higher baseline level (Figure 1, Supporting Information S1: Tables S2 and S3). Because there were more cases with TP53 mutations (del17p or TP53 mutated), we evaluated whether the TP53 mutational status had an impact on ALC kinetics within the subgroups. As shown in Supporting Information S1: Table S4, there was no difference in ALC kinetics according to TP53 mutational status among the IGHV mutated and unmutated cases in the IBR arm. Regarding clinical features and the burden of disease at baseline, the two arms were homogeneous with only one exception: more patients treated with acalabrutinib (53%) had Binet staging C before therapy, while only (39%) of patients treated with ibrutinib had stage C. Despite this difference being statistically significant (p = 0.04), our analysis demonstrated that the stage had no impact on the kinetics of lymphocytosis (data not shown). Even with different grades of lymphadenopathy and splenomegaly, including the presence of bulky masses, the trend of lymphocytosis during treatment with both cBTKi remained unaltered (data not shown). We have provided the first description of the kinetics of lymphocytosis in patients treated with acalabrutinib and conducted the first comparative study of lymphocyte counts during 12 months of treatment with two cBTK inhibitors. We observed that, similar to Ibrutinib, acalabrutinib leads to an increase in lymphocyte count immediately after starting therapy. This observation confirms previous works identifying lymphocytosis as a "class effect" of cBTKi.14 We observed that while there is an appreciable reduction in lymphocyte count starting from the second month of treatment in both groups, the decrease in lymphocytosis appears to be faster and more profound in patients treated with acalabrutinib from the 6th month onward. We also investigated whether certain disease's features could explain the differences in the kinetics of lymphocytosis. There was a significant difference in the distribution of patients with a poor molecular prognosis (del17p/TP53) between the two groups, with a marked prevalence in the IBR arm. This difference can be attributed to the evolution of cBTKi use in clinical practice: when ibrutinib was introduced, it was only allowed for patients with a poor prognosis. Conversely, acalabrutinib was used in clinical practice when the administration of cBTKi was already well established in all untreated patients. However, according to our data, having a del17p/TP53 mutation or NOTCH1 mutation does not interfere with the kinetics of lymphocytosis during treatment with the two different cBTKi. NOTCH1 mutations and higher CD49D expression have been associated with reduced ibrutinib-induced lymphocytosis,17, 18 but in this cohort, we did not observe any effect. The IGHV mutational status did not differ between the two groups; however, when examining lymphocyte count curves in mutated IGHV, starting from the 6th month to the end of the study period, the median percentage of baseline declined more in the ACALA arm reaching a statistical difference. Overall, IGHV mutated/unmutated patients treated with acalabrutinib had a similar increase in lymphocyte count after 14 days, followed by a sharper decline, achieving median normal lymphocyte count earlier than the IBR arm. These data suggest that the main differences of lymphocytes count between the two cBTKi is due to IGHV mutated status. The clinical burden of disease at baseline had no impact on the kinetics of lymphocytosis between the two arms. Currently, there is lack of data in the literature comparing the differences between ACALA and IBR concerning lymphocytosis, IGHV mutational status, and the extent of lymphocyte mobilization in peripheral blood. Particularly, there is scarcity of mechanistic explanations for the divergent behavior of the two cBTK inhibitors. In order to verify this hypothesis, our group is conducting research into chemokine receptors and adhesion molecules. In conclusion, we describe the kinetics of peripheral blood lymphocytosis after ibrutinib or acalabrutinib in patients with CLL treated in front line. Both treatments exhibited a similar peak at 2 weeks after initiation, followed by a more pronounced and rapid decrease in the acalabrutinib treatment group, particularly in IGHV mutated cases. MoH ricerca corrente 2024. Antonio Mosca, Annamaria Tomasso, Andrea Galitzia, Luca Stirparo, Francesca Martini, Francesca Morelli, Roberta Laureana, Giulia Benintende, Veronica Mattiello, Sabrina Chiriu, Maria I. Del Principe, Giulia Zamprogna, Massimo Gentile, Enrica A. Martino, Emilia Cappello, Maria C. Montalbano, Giuliana Farina, Vanessa Innao, Lydia Scarfò, Caterina Patti, Paolo Sportoletti, Alberto Fresa, Gioacchino Catania, Marta Coscia, Silvia Bellesi, Alessandra Tedeschi, Alessandro Sanna, Andrea Visentin, Francesco Autore, Raffaella Pasquale, Livio Trentin, Marzia Varettoni, Paolo Ghia, and Roberta Murru collected the data. Eugenio Galli performed the statistical analysis. Idanna Innocenti and Antonio Mosca wrote the manuscript and Eugenio Sangiorgi and Luca Laurenti supervised the project. Paolo Ghia received honoraria from AbbVie, AstraZeneca, BeiGene, BMS, Galapagos, Janssen, Lilly/LoxoOncology, MSD, and Roche, and research funding from AbbVie, AstraZeneca, BMS, and Janssen, and is an Editor of HemaSphere. Marzia Varettoni received honoraria from AbbVie, AstraZeneca, BeiGene, and Janssen. The remaining authors declare no conflict of interest. This research received no external funding. This study was conducted in accordance with the Declaration of Helsinki and approved by the Institutional Review Board of Fondazione Policlinico Universitario Agostino Gemelli IRCCS (Prot. ID 5765 13/10/2023). Written informed consent was obtained from the patients to publish this study. The data that support the findings of this study are available from the corresponding author upon reasonable request. Please note: The publisher is not responsible for the content or functionality of any supporting information supplied by the authors. Any queries (other than missing content) should be directed to the corresponding author for the article.

01 Nov 2023·Clinical journal of the American Society of Nephrology : CJASN

Will SGLT2 Inhibitors Be Effective and Safe in Patients with Severe CKD, Dialysis, or Kidney Transplantation

Article

Author: Stefan Berger ; Hiddo J.L. Heerspink ; Ron T. Gansevoort

The clinical benefits of sodium‐glucose cotransporter‐2 (SGLT2) inhibitors in patients with CKD have been well established in major clinical outcome trials.1,2 Secondary analyses from these landmark trials demonstrated consistent benefits of SGLT2 inhibitors across many subgroups, supporting the widespread use and incorporation in clinical practice guidelines of this new kidney and cardioprotective drug class.3,4 Notwithstanding, there are three important groups of kidney patients in whom SGLT2 inhibitors are not indicated. First are patients with severely impaired kidney function. This seems sensible because the efficacy of SGLT2 inhibitors in these patients could well be diminished. SGLT2 inhibitors block the reabsorption of glucose and sodium in the proximal tubule and enhance glycosuria and natriuresis. In patients with a low number of functioning nephrons, it may be expected that SGLT2 transporters are less effective because the number of SGLT2 transporters containing tubules is reduced. Therefore, patients with an eGFR <20 ml/min per 1.73 m2 and those receiving dialysis were excluded from recent outcome trials. Yet, there are emerging data suggesting that SGLT2 inhibitors may reduce clinically relevant outcomes in these vulnerable high-risk patients. The DAPA-CKD and EMPA-KIDNEY trials recruited 2906 patients with CKD stage 4 who had an eGFR>25 (DAPA-CKD; N=624) or >20 ml/min per 1.73 m2 (EMPA-Kidney; N=2282).1,2 In both trials, SGLT2 inhibition reduced the risk of the primary kidney outcome by 27% with no evidence that the magnitude of the effect estimate varied by baseline GFR. Moreover, in the DAPA-CKD trial, dapagliflozin reduced the relative risks of heart failure or cardiovascular death or all-cause mortality, both secondary outcomes, in participants with and without CKD stage 4.1 In the EMPA-KIDNEY trial, 245 participants had an eGFR below 20 ml/min per 1.73 m2 at randomization. Although this was a small subgroup, the 27% relative risk reduction in the incidence of the primary kidney outcome was consistent with the effect size in the overall population. These exploratory subgroup data support the hypothesis that SGLT2 inhibitors may exert beneficial effects in patients at advanced stages of CKD. However, the evidence in this specific subgroup is weak, and the benefits observed in exploratory analyses in small subgroups could be the result of chance. Second, clinical experience with SGLT2 inhibitors in dialysis patients is also limited because of perceived lack of efficacy. However, emerging data from the DAPA-CKD trial, where patients continued study medication when they started dialysis, provide new insights. Safety assessment among participants continuing dapagliflozin or placebo during dialysis did not reveal any between-group differences in adverse events. Moreover, numerically fewer deaths were reported in the dapagliflozin compared with the placebo group.1 These data should, however, be cautiously interpreted because the dapagliflozin and placebo groups were not randomized at dialysis initiation and few participants initiated dialysis (N=68 and N=99, respectively), reducing the reliability of these results. How could it be that the efficacy of SGLT2 inhibitors persists in patients with severely impaired kidney function or on dialysis? Experimental data have suggested direct effects on the kidney as well as the heart that are not mediated via SGLT2 inhibition as presented in Figure 1. For instance, in an experimental study, uremic serum from dialysis patients impaired endothelium-mediated enhancement of relaxation and contraction of cardiomyocytes. Incubation with empagliflozin reversed this process, suggesting direct effects on endothelial dysfunction in coronary microvascular beds.5 In another experimental study, it was shown that isolated endothelial cells of human coronary arteries produce on stimulation with the stressor TNFα more of vasoconstrictor reactive oxygen species and less of the vasodilator nitric oxide. These effects could be blocked by cotreating these isolated cells with the SGLT2 inhibitors empagliflozine and dapagliflozine.6 How exactly SGLT2 inhibitors exert these beneficial effects in the absence of a functioning proximal tubule requires further study. These drugs can also bind directly to sodium–hydrogen exchange 1 transporters in cardiac tissues to promote a cardioprotective effect.7 Moreover, Lee et al. showed that in the case of ischemia, SGLT2 is expressed in the myocardium and that inhibition of this cardiac SGLT2 leads to a smaller infarct size. Finally, binding of SGLT2 inhibitors to SGLT2 expressed in pancreatic α-cells may promote the release of glucagon and reduce insulin, which in turn increases ketone body levels. Increased ketone bodies exert a wide array of beneficial effects in cardiac tissues.8 This notion of SGLT2 transporter–independent cardiac benefits is further supported by a bioinformatic study that used a combined approach of in silico modeling of publicly available RNA sequence datasets coupled with RNA sequencing of cardiac tissues from an experimental model of diabetic rats with heart failure treated with empagliflozin.9 The results also suggested that the inhibition of sodium–hydrogen exchange 1 may explain the heart failure protective effects of SGLT2 inhibitors.9 Such direct cardiac and kidney effects that are independent of proximal tubular SGLT2 could play a role in patients with severe CKD and minimal diuresis, but confirmation in a clinical study is required. Thus, at present, there is no definitive evidence to support a positive benefit-to-risk ratio in patients with severe CKD.Figure 1.: Potential cardiorenal protective mechanisms for SGLT inhibition mediated via tubular SGLT2 and via mechanisms working direct on the heart and the kidney. NHE-1, sodium–hydrogen exchange 1; SGLT2, sodium‐glucose cotransporter‐2. Adapted from Udell JA, Jones WS, Petrie MC, et al. Sodium glucose cotransporter-2 inhibition for acute myocardial infarction: JACC review topic of the week. J Am Coll Cardiol. 2022;79(20): 2058–2068.The third group of patients who could benefit from SGLT2 inhibitors but were excluded from recent large-scale outcome trials are patients living with a kidney transplant. Besides the fact that many of these patients have (severely) decreased kidney function, there is another issue that deserves attention in this specific group. In general, SGLT2 inhibitors are well tolerated and safe. Main side effects are, however, an increased incidence of urinary tract and genital infections. Patients living with a kidney transplant have a higher chance of (severe) urinary tract infections, which may lead to kidney function loss. The benefit-to-risk ratio in kidney transplant recipients deserves therefore special attention. Another specific clinical dilemma in patients with kidney transplantation is post-transplant diabetes mellitus, which occurs in 10%–20% of kidney transplant recipients due to prednisolone and other immunosuppressive therapy. SGLT2 inhibitors may be salutary not only because of their kidney and cardiac benefits but also because they reduce the risk of new-onset diabetes.1 A number of studies have explored the effects of SGLT2 inhibition in kidney transplant recipients. Most of these studies were case reports or small retrospective case series, limiting the quality and reliability of the data. A small randomized controlled clinical trial in 44 kidney transplant recipients showed that 24-week treatment with empagliflozin reduced HbA1c and body weight compared with placebo. During the relatively short follow-up period, empagliflozin was well tolerated with no imbalance in adverse events or immunosuppressive drug levels, including calcineurin inhibitors.10 Kidney function over time or the incidence of clinical outcomes was not assessed. A more recent observational clinical practice study aimed to address this gap. In this study, 226 kidney transplant recipients with type 2 diabetes who received SGLT2 inhibitors >90 days were pair-matched with kidney transplant recipients with type 2 diabetes not using SGLT2 inhibitors. During a mean follow-up of 5.2 years, SGLT2 inhibition was associated with a reduction in the risk of all-cause mortality, death-censored graft survival, or doubling of serum creatinine.11 SGLT2 inhibitors were well tolerated with no difference in the incidence of bacterial or fungal urinary tract infections. Diabetic ketoacidosis did not occur during follow-up in both groups. Although these findings are reassuring, the retrospective character of this study, without randomization and without blinded end point collection, precludes a definitive assessment of benefits and risks. In conclusion, although there are some preliminary promising data, it is at present unclear whether SGLT2 inhibition will be effective in preventing clinically relevant outcomes and be sufficiently safe in patients with severe CKD that are not included in the present label. The RENAL LIFECYCLE trial (NCT05374291) was recently started to investigate the potential benefit of SGLT2 inhibitors in these patients. The trial aims to address the efficacy and safety of dapagliflozin 10 mg versus placebo in at least 1500 patients with (1) an eGFR <25 ml/min per 1.73 m2; (2) dialysis patients with residual diuresis >500 ml/24-hour; or (3) kidney transplant recipients with an eGFR ≤45 ml/min per 1.73 m2. These patients are recruited at clinical practice sites in Europe and Australia. The primary study end point is a composite end point of kidney failure, heart failure hospitalization, or all-cause mortality. The results are awaited in 4 years. In the absence of reliable data from randomized controlled clinical trials, we recommend being restrictive with the use of SGLT2 inhibitors in patients with severe CKD, on dialysis, or living with a kidney transplant. Disclosures S. Berger reports consultancy for Chiesi and Novartis; research funding from Chiesi and Novartis; honoraria from Astellas and Novartis; and advisory or leadership roles for Advisory Board for Novartis and Supervisory Board for Dutch Transplant Foundation. R.T. Gansevoort reports consultancy for AstraZeneca, Bayer, Galapagos, Mironid, and Sanofi-Genzyme; research funding from Abbvie, AstraZeneca, Bayer, Galapagos, Otsuka Pharmaceuticals, Roche, and Sanofi-Genzyme; role on the Editorial Boards of American Journal of Kidney Diseases, CJASN, Journal of Nephrology, Kidney360, and Nephron Clinical Practice; and role as Editor of Nephrology Dialysis Transplantation and member of the Council of the European Renal Association. H.J.L. Heerspink reports ongoing consultancy agreements with AstraZeneca, Bayer, Boehringer Ingelheim, Chinook, CSL Behring, Dimerix, Eli-Lilly, Gilead, Janssen, Merck, Novartis, Novo Nordisk, and Travere Pharmaceuticals; research funding from AstraZeneca, Boehringer Ingelheim, Janssen research support (grant funding directed to employer), and Novo Nordisk; lecture fees from AstraZeneca and Novo Nordisk; and speakers bureau for AstraZeneca.

01 Sep 2023·United European gastroenterology journal

Surfing the web as a patient with IBD: New horizons

Communications

Author: Rodríguez‐Lago, Iago ; Barreiro‐de Acosta, Manuel ; Catalan‐Serra, Ignacio

Inflammatory bowel disease (IBD) is a chronic lifelong condition with a disabling course in a significant proportion of patients. Additionally, it is more frequently diagnosed during childhood and in young adults, so its impact can be observed in many aspects of their life in the long-term.1 Despite this negative perspective, the therapeutic armamentarium has increased in recent years, so the probability of controlling the disease activity has increased notably. As we continue to learn about the triggers of the disease and the underlying immunological alterations, our understanding of the real impact of this condition is also growing. For instance, it is now clear how the disease may affect extraintestinal organs2 but, even more importantly, also the critical impairment of quality of life, disability, mental health and work productivity.3 This is highly relevant, since this impact on several domains of the patients' lives might be present despite an adequate control of the disease in the gut. The increasing awareness of its impact led the European Medicines Agency (EMA) to express their support to include quality of life and patient-reported outcomes (PRO) in IBD clinical trials in 2019.4 Crohn's disease (CD) and ulcerative colitis-PRO signs and symptoms diaries were developed and endorsed by EMA for this purpose,5, 6 and they are now considered as the first choice in the assessment of disease activity as the use of other scores like Crohn's Disease Activity Index and total Mayo are difficult to use in daily practice or can be biased in certain situations. These new endpoints and trial designs have already been implemented during the development of the most recently developed biologics and small molecules7, 8 and hopefully can lead to broader the concept of disease-modification strategies in the near future.9 Thus, the shift in the management of these patients has evolved from a physician-centered model to a patient-focused evaluation. This is expected to bring new perspectives with more realistic and impactful unmet needs, and they can also potentially reduce patient-care variability.10 Despite this remarkable -and positive- change, it should be stressed that both perspectives (from physicians and patients) have demonstrated significant differences in the assessment of disease activity or adverse drug reactions.11, 12 Nevertheless, in the era of electronic health records, digital PRO have been developed in parallel with telemedicine resources; thus, patient wellbeing, certain follow-up consultations and patient self-management are new tools available to improve a more comprehensive evaluation of the disease.13 We should all be involved in implementing these advances within our clinical practice, as this is a unique opportunity to work together and understand what patients need more in detail. Platforms where patients can report their perception of the disease and interact with their physicians are urgently needed. Ideally, they should also serve as a source for research that can explore new treatment alternatives and how to fulfil other patient needs. However, new tools also bring new questions.14 Digital platforms, and mainly those using cell phones, may not be suitable for all patients and across all ages. Accessibility will always be an issue, as they need to encourage the participation of those (not so) motivated patients, those with disabilities, and those without limited access to certain technologies. In this issue, Engel et al. provide new and useful information about how these resources can be applied for research.15 Here, StuffThatWorks (www.stuffthatworks.health), a platform designed to collect clinical information from chronic conditions using a machine learning algorithm, was used to gather patient experience on different medical treatments for CD. The authors showed that anti-TNF agents were ranked as the most effective by patients, followed by ustekinumab and vedolizumab, respectively. Interestingly, surgery appeared in second place. However, one of the most remarkable aspects evaluated in the study is the triggers associated with disease activity, highlighting that psychological factors and certain foods were the most important factors reported by patients. Nonetheless, we also think that validation across diseases and countries would increase the value of their interesting findings. In conclusion, the featured paper by Israeli colleagues provides novel and relevant information about how new technologies can bridge the gap between patient and physician perceptions in IBD and to include PRO in future research. Still, some questions remain open about how these data could be combined with traditional trials. Nonetheless, in our opinion, these novel digital approaches explore how we can closely collaborate with patients in order to shape and improve the future clinical care of patients affected with IBD. IRL-L is supported by a research grant from Gobierno Vasco – Eusko Jaurlaritza [Grant No 2020111061] and by a research grant from Biocruces Bizkaia Health Research Institute [Grant No BCB/I/LIB/22/008]. IR-L has received financial support for travelling and educational activities from or has served as an advisory board member for Abbvie, Adacyte, Celltrion, Chiesi, Danone, Ferring, Faes Farma, Janssen, Galapagos, MSD, Pfizer, Roche, Takeda, and Tillotts Pharma. Financial support for research: Tillotts Pharma. IC-S has received financial support for travelling and educational activities from or has served as an advisory board member for Takeda, AbbVie, Janssen, Tillots Pharma, Ferring, Lilly, and BMS. MB-A has received financial support for travelling and educational activities from or has served as an advisory board member for Pfizer, MSD, Takeda, AbbVie, Kern, Janssen, Fresenius Kabi, Galapagos, Lilly, BMS, Faes Farma, Chiesi, and Adacyte. Biocruces Bizkaia Health Research Institute, Grant/Award Number: BCB/I/LIB/22/008; Gobierno Vasco – Eusko Jaurlaritza, Grant/Award Number: 2020111061 Not applicable.

News (Medical) associated with GLPG-5101

01 Apr 2026

·www.biospace.com

Advanced Therapy Medicinal Products CDMO Market Size to Hit USD 53.11 Billion by 2035

According to Nova One Advisor, the global Advanced Therapy Medicinal Products (ATMP) CDMO market was valued at USD 9.35 billion in 2025 and is anticipated to reach approximately USD 53.11 billion by 2035, growing at a CAGR of 18.97% during the forecast period from 2026 to 2035. Key Insights on the Advanced Therapy Medicinal Products CDMO Market: · By Product: Gene therapy led the market in 2025, while cell therapy is the fastest-growing segment. · By Phase: The Phase II segment held the largest market share in 2025. However, the Phase III segment is expected to experience the fastest growth moving forward. · By Indication: Oncology was the dominant indication in 2025, but neurological and genetic disorders are projected to see the highest growth rate. · By Region: North America held the largest market share in 2025. Asia-Pacific is expected to have the fastest growth throughout the forecast period. The Complete Study is Now Available for Immediate Access | Download the Sample Pages of this Report@ Market Overview: Integrated CDMO Platforms and Advanced Analytics The advanced therapy medicinal products CDMO market includes contract development and manufacturing organizations providing specialized services such as process development, cGMP manufacturing, and testing for gene therapies, somatic cell therapies, and tissue-engineered products. ATMP CDMOs serve as vital partners for biotech and pharmaceutical companies by offering end-to-end development and GMP manufacturing services for complex, personalized living medicines . This growth is driven by increasing clinical trials for rare diseases and cancer, with high demand for outsourcing due to complex manufacturing needs. Emergence of New Transcriptomics: Major Potential The advent of advanced spatial and single-cell transcriptomics, including Xenium and MERSCOPE, presents ATMP CDMOs with a transformative opportunity to expand their service capabilities through improved cellular characterization, high-resolution quality control, and optimized manufacturing processes. These technologies help ensure better reproducibility of complex therapies. By analyzing cellular heterogeneity and tissue interactions, CDMOs can speed up process development and enhance patient-specific modeling. Combining AI with transcriptomics technologies offers a competitive advantage by automating data analysis and predictive modeling, boosting therapy effectiveness, and reducing time to market. Barriers in Regulation and Standardization: Major Obstacles The rapid adoption and commercialization of new ATMP technologies are significantly hampered by a fragmented global regulatory landscape, a lack of harmonized guidelines, and scarce standardized reference materials. These challenges increase compliance costs and hinder reproducibility. Inaccurate data interpretation, high capital investments for advanced infrastructure, and a shortage of skilled personnel make scaling up these personalized therapies difficult, especially for smaller CDMOs. Buy Now Full Report: Advanced Therapy Medicinal Products CDMO Market Report Scope Report Attribute Details Market Size in 2026 USD 11.12 Billion Market Size by 2035 USD 53.11 Billion Growth Rate From 2026 to 2035 CAGR of 18.97% Base Year 2025 Forecast Period 2026 to 2035 Segments Covered By Product, By Phase, By Indication Market Analysis (Terms Used) Value (USD Million/Billion) or (Volume/Units) Report Coverage Revenue Forecast, Company Ranking, Competitive Landscape, Growth Factors, and Trends Key Companies Profiled Celonic; Bio Elpida; CGT Catapult; Rentschler Biopharma SE; AGC Biologics; Catalent; Lonza; WuXi Advanced Therapies; BlueReg; Minaris Regenerative Medicine; Patheon You can place an order or ask any questions, please feel free to contact at sales@novaoneadvisor.com | +1 804 441 9344 Advanced Therapy Medicinal Products CDMO Market: Segmentation Analysis By Product Analysis In 2025, the gene therapy segment dominated the market, driven by a large and rapidly growing pipeline of clinical trials, increasing regulatory approvals for rare diseases, and the urgent need for complex viral vector manufacturing . Innovations in gene editing, such as CRISPR-Cas9, along with improved non-viral delivery systems, have broadened potential therapeutic applications, leading to an increase in the number of projects managed by CDMOs. Gene therapies depend on complex viral vector manufacturing, which necessitates high-level technical expertise that biotechnology companies often outsource to CDMOs. The cell therapy segment is expected to experience the fastest growth during the forecast period, primarily due to the rapid expansion of CAR-T cell therapies for cancer. There are a surging pipeline of clinical trials and a notable shift toward allogeneic off-the-shelf manufacturing. The number of clinical trials for cell therapies targeting oncology , rare diseases, and autoimmune disorders is growing quickly, necessitating advanced infrastructure for development. The high demand for complex, individualized, and sometimes regenerative therapies is pushing biotech firms to outsource production to CDMOs. Advanced Therapy Medicinal Products CDMO Market by Product, 2025-2035 (USD Billion) Year 2025 2026 2027 2028 2029 2030 2031 2032 2033 2034 2035 Gene Therapy 3.35 3.97 4.67 5.53 6.52 7.71 9.11 10.76 12.73 15.02 17.75 Cell Therapy 2.98 3.55 4.24 5.05 6.03 7.19 8.57 10.22 12.18 14.53 17.32 Tissue Engineered 0.75 0.9 1.1 1.33 1.61 1.95 2.36 2.86 3.46 4.19 5.07 Others 0.37 0.44 0.53 0.63 0.75 0.89 1.06 1.26 1.49 1.78 2.11 By Phase Analysis In 2025, the Phase II segment led the market, primarily because it handles the highest volume of ongoing trials and requires critical, complex Good Manufacturing Practice (GMP) production for intermediate efficacy trials, which demands the expertise of specialized CDMOs. The growing popularity of cell and gene therapies , coupled with high success rates from Phase I trials, is driving significant funding into this segment. CDMOs in this phase specialize in enhancing product consistency, scalability, and in preparing documentation for potential commercialization. The Phase III segment is predicted to expand at a notable CAGR. This growth is mainly driven by an increase in late-stage clinical trials for gene and cell therapies transitioning to commercialization, which demands massive process validation, large-scale viral vector production, and stringent quality assurance. As more advanced therapy medicinal products (ATMPs) move into Phase III, CDMOs will be tasked with managing pivotal, large-scale studies and preparing for product launches. Advanced Therapy Medicinal Products CDMO Market by Phase, 2025-2035 (USD Billion) Year 2025 2026 2027 2028 2029 2030 2031 2032 2033 2034 2035 Phase I 1.64 1.89 2.17 2.5 2.86 3.28 3.76 4.29 4.9 5.58 6.34 Phase II 2.82 3.32 3.9 4.57 5.37 6.29 7.38 8.66 10.15 11.9 13.94 Phase III 2.24 2.7 3.27 3.95 4.77 5.77 6.96 8.41 10.15 12.25 14.79 Phase IV 0.75 0.95 1.2 1.52 1.91 2.4 3 3.74 4.66 5.79 7.18 By Indication Analysis The oncology segment dominated the market in 2025, primarily due to the high demand for CAR-T cell therapies, gene-modified cell therapies, and oncolytic viruses. This dominance is driven by increased investment in cancer research and development, personalized medicine initiatives, and the need for specialized manufacturing capacity. To meet the growing global demand for targeted cancer treatments, CDMOs are increasingly relied upon for their specialized facilities and technical expertise, offering faster development timelines and high-precision outsourced production. The segment focused on neurological and genetic disorders is expected to grow at the fastest CAGR during the forecast period. This growth is attributed to a rapid increase in clinical trials for gene therapies, the demand for specialized vector design, and significant unmet medical needs for rare. Due to the complexity and high costs associated with developing in-house infrastructure for these advanced and often curative therapies, pharmaceutical companies are heavily partnering with specialized ATMP CDMOs with rapid apid advancements in gene editing technologies and viral vector development. Advanced Therapy Medicinal Products CDMO Market by Indication, 2025-2035 (USD Billion) Year 2025 2026 2027 2028 2029 2030 2031 2032 2033 2034 2035 Oncology 2.61 3.08 3.63 4.28 5.05 5.95 7 8.25 9.74 11.45 13.53 Cardiology 0.52 0.62 0.74 0.88 1.04 1.24 1.48 1.76 2.09 2.49 2.96 Central nervous system 0.67 0.8 0.95 1.13 1.34 1.6 1.9 2.26 2.69 3.2 3.8 Musculoskeletal 0.45 0.53 0.63 0.75 0.89 1.06 1.27 1.51 1.79 2.13 2.53 Infectious disease 0.6 0.7 0.82 0.97 1.13 1.33 1.56 1.83 2.15 2.52 2.96 Dermatology 0.3 0.35 0.42 0.5 0.6 0.71 0.84 1 1.19 1.42 1.69 Endocrine, metabolic, genetic 0.52 0.64 0.78 0.95 1.16 1.42 1.73 2.11 2.57 3.13 3.8 Immunology & inflammation 0.52 0.64 0.78 0.95 1.16 1.42 1.73 2.11 2.57 3.13 3.8 Ophthalmology 0.45 0.54 0.65 0.79 0.95 1.15 1.39 1.68 2.03 2.45 2.96 Hematology 0.37 0.44 0.53 0.63 0.75 0.89 1.06 1.26 1.49 1.78 2.11 Gastroenterology 0.22 0.27 0.34 0.41 0.51 0.62 0.76 0.93 1.13 1.39 1.69 Others 0.22 0.25 0.27 0.3 0.33 0.35 0.38 0.4 0.42 0.43 0.42 By Regional Insights North America led the market in 2025. This leadership is mainly due to high investment in research and development, strong biopharma infrastructure, and a boom in clinical trials for cell and gene therapies. A notable rise in Investigational New Drug (IND) applications for therapies in oncology and rare diseases has created high demand for specialized, scalable, and compliant manufacturing facilities. Strict but clear FDA guidelines foster a stable environment for investment and commercialization, and high outsourcing rates from biotech startups further strengthen this regional leadership. With a dense biotech network and faster FDA approvals for therapies like CAR-T, the U.S. maintains a dominant position globally. Heavy investment in automated, large-capacity viral vector manufacturing solidifies the U.S. as the top hub for translating advanced, complex cell and gene therapies from lab to market. · In March 2026, Agilent Technologies launched Agilent Advanced Therapeutics, a unified CDMO solution integrating BIOVECTRA and Nucleic Acid Solutions across North America to offer comprehensive, end-to-end manufacturing for life-changing therapies. Canada is quickly establishing itself as an affordable extension of the U.S. market, combining specialized expertise in high-growth cell and gene therapies with strict FDA cGMP standards. Canadian CDMOs provide seamless, high-quality analytical and manufacturing solutions for U.S. biotech sponsors, supporting faster, compliant innovation within North America. · In March 2025, Merck began construction on a $1 billion, 470,000-square-foot biologics center of excellence in Wilmington, Delaware, aimed at boosting U.S. manufacturing of next-generation therapies, including antibody-drug conjugates (ADCs) and Keytruda, and creating thousands of high-paying jobs. Asia Pacific is expected to experience the fastest growth during the forecast period. This rapid expansion results from increasing demand for personalized medicine, rising research and development investments, and a strategic move toward regionalizing supply chains. Governments in China, Japan, and South Korea are actively supporting the sector through incentives, subsidies, and faster regulatory pathways for advanced therapies. The high number of biotech firms in China and emerging hubs in Singapore and India focusing on orphan diseases and cancer fuels demand for external manufacturing capacity. India is becoming a key hub, offering cost-effective, FDA-compliant services to Western companies looking to diversify supply chains through the China+1 strategy. Building on its vaccine manufacturing expertise, India's market is poised for significant growth, shifting toward a complex biologics and advanced cell and gene therapies landscape. · In December 2025, India’s Bulk Drug Parks are game-changers, transforming the nation from an import-dependent pharmacy to a self-reliant powerhouse by creating world-class, cost-effective API manufacturing hubs that fuel local CDMO growth. China has become the leading low-cost, high-speed partner for global biotech, largely due to rapid regulatory reforms and significant innovation. By transitioning from a follower to an innovator with greater diversity in research targets and improved regulatory capacity at the CDE, China is establishing itself as an essential, integrated ecosystem for advanced therapy medicinal products. · In October 2025, China’s NMPA is fast-tracking the future of medicine by slashing red tape and accelerating clinical trials for advanced therapies. These sweeping regulatory reforms foster homegrown innovation, align with global standards, and promise rapid patient access to life-saving treatments. Advanced Therapy Medicinal Products CDMO Market by Region, 2025-2035 (USD Billion) Year 2025 2026 2027 2028 2029 2030 2031 2032 2033 2034 2035 North America 2.84 3.33 3.91 4.61 5.42 6.39 7.51 8.83 10.39 12.21 14.37 Europe 2.38 2.81 3.31 3.9 4.59 5.41 6.37 7.51 8.84 10.41 12.25 Asia-Pacific 1.64 2.02 2.49 3.06 3.76 4.61 5.66 6.93 8.48 10.37 12.67 Latin America 0.37 0.43 0.51 0.59 0.69 0.8 0.93 1.08 1.25 1.46 1.69 Middle East & Africa (MEA) 0.22 0.27 0.32 0.38 0.45 0.53 0.63 0.75 0.9 1.07 1.27 Advanced Therapy Medicinal Products CDMO Market Companies · Celonic GmbH · Bio Elpida · Cell and Gene Therapy Catapult (CGT Catapult) · Rentschler Biopharma SE · AGC Biologics · Catalent, Inc. · Lonza Group Ltd. · WuXi Advanced Therapies (WuXi AppTec Group) · BlueReg Group · Minaris Regenerative Medicine Pervasive Adoption of Cell and Gene Therapies in Modern Medicine Company Latest Updates Sumitomo Chemical (August 2025) Announced expansion of cell therapy manufacturing capacity in Japan, including a fourth regenerative medicine facility, supported by the 2024 supplementary budget from METI. Vector BioMed (June 2025) Partnered with Kailash Cancer Hospital and Research Center in India to bring CAR T-Cell therapy to rural areas, expanding access and local production. Lonza (Feb 2025) Expanded cell and gene therapy manufacturing capacity in the U.S. to meet growing demand for late-stage and commercial CGT programs. Catalent (Jan 2025) Partnered with Galapagos NV to support the decentralized manufacturing of CAR-T therapy GLPG5101, designed to enhance patient access to treatment. Recent Developments · In May 2025, Altaris combined Minaris Regenerative Medicine and WuXi Advanced Therapies’ U.S. and U.K. operations to create Minaris Advanced Therapies™, a global cell therapy CDMO headquartered in Philadelphia. The company manufactures two commercial cell therapies and tests over 27 products. With more than 1,400 experts, it offers advanced platforms for cell therapies and viral vectors and aims to streamline the commercialization of these therapies. · In January 2025, Immuneel Therapeutics launched Qartemi, a CAR T-cell therapy for adult B-cell Non-Hodgkin Lymphoma (B-NHL) in India. This personalized therapy addresses a crucial need as the country faces over 120,000 new blood cancer cases yearly. Co-founder Kiran Mazumdar-Shaw emphasized their goal of providing accessible, innovative cancer treatments. · In March 2026, Indena announced its expanded capabilities in Highly Potent Active Pharmaceutical Ingredients (HPAPIs) and Antibody-Drug Conjugates (ADCs) at DCAT Week 2026. They produce multiple commercial HPAPIs and integrate advanced technologies to meet the rising demand in oncology. Global CDMO Director Bernard Vianes highlighted Indena's commitment to innovation and safety, achieving low occupational exposure levels in HPAPI manufacturing. Related Report – ⬥ ︎ Active Pharmaceutical Ingredients CDMO Market - The global active pharmaceutical ingredients CDMO market size was valued at USD 139.33 billion in 2025 and is anticipated to reach around USD 292.79 billion by 2035, growing at a CAGR of 7.71% from 2026 to 2035. ⬥ ︎ Biopharmaceutical CDMO Market- The global biopharmaceutical CDMO market was valued at USD 21.15 billion in 2024 and is projected to hit around USD 49.61 billion by 2034, expanding at a CAGR of 8.9% during the forecast period of 2025 to 2034. ⬥ ︎ Biologics CDMO Market- The global biologics CDMO market size is calculated at USD 22.45 billion in 2024, grows to USD 25.92 billion in 2025, and is projected to reach around USD 94.60 billion by 2034, growing at a solid CAGR of 15.47% from 2025 to 2034. ⬥ ︎ Oligonucleotide CDMO Market- The global oligonucleotide CDMO market size is calculated at USD 3.15 billion in 2024, grows to USD 3.84 billion in 2025, and is projected to reach around USD 22.73 billion by 2034, at a compound annual growth rate (CAGR) of 21.85% from 2024 to 2034. ⬥ ︎ Cell and Gene Therapy CDMO Market- The global cell and gene therapy CDMO market size was estimated at USD 10.35 billion in 2025 and is projected to hit around USD 125.09 billion by 2035, growing at a CAGR of 28.3% during the forecast period from 2026 to 2035. ⬥ ︎ U.S. Active Pharmaceutical Ingredients CDMO Market- The U.S. active pharmaceutical ingredients CDMO market size is calculated at USD 25.85 billion in 2024, grows to USD 27.22 billion in 2025, and is projected to reach around USD 43.33 billion by 2034, growing at a CAGR of 5.3% from 2025 to 2034. ⬥ ︎ North America Topical Drugs CDMO Market- The North America topical drugs CDMO market size was exhibited at USD 15.25 billion in 2024 and is projected to hit around USD 42.87 billion by 2034, growing at a CAGR of 10.89% during the forecast period 2025 to 2034. ⬥ ︎ Topical Drugs CDMO Market- The topical drugs CDMO market size was exhibited at USD 46.15 billion in 2025 and is projected to hit around USD 137.06 billion by 2035, growing at a CAGR of 11.31% during the forecast period 2026 to 2035. ⬥ ︎ U.S. Pharmaceutical CDMO Market- The U.S. pharmaceutical CDMO market size was exhibited at USD 43.7 billion in 2025 and is projected to hit around USD 89.45 billion by 2035, growing at a CAGR of 7.43% during the forecast period 2026 to 2035. ⬥ ︎ Veterinary CRO and CDMO Market- The Veterinary CRO and CDMO market size was exhibited at USD 7.15 billion in 2024 and is projected to hit around USD 17.56 billion by 2034, growing at a CAGR of 9.4% during the forecast period 2025 to 2034. ⬥ ︎ U.S. Small Molecule Innovator API CDMO Market - The U.S. small molecule innovator API CDMO market size was exhibited at USD 9.85 billion in 2025 and is projected to hit around USD 17.96 billion by 2035, growing at a CAGR of 6.19% during the forecast period 2026 to 2035. Segments Covered in the Report This report forecasts revenue growth at country levels and provides an analysis of the latest industry trends in each of the sub-segments from 2021 to 2035. For this study, Nova one advisor, Inc. has segmented the Advanced Therapy Medicinal Products CDMO market. By Product · Gene Therapy · Cell Therapy · Tissue Engineered · Others By Phase · Phase I · Phase II · Phase III · Phase IV By Indication · Oncology · Cardiology · Central nervous system · Musculoskeletal · Infectious disease · Dermatology · Endocrine, metabolic, genetic · Immunology & inflammation · Ophthalmology · Hematology · Gastroenterology · Others By Region · North America · Europe · Asia-Pacific · Latin America · Middle East & Africa (MEA) Immediate Delivery Available | Buy This Premium Research: About-Us Nova One Advisor is a global leader in market intelligence and strategic consulting, committed to delivering deep, data-driven insights that power innovation and transformation across industries. With a sharp focus on the evolving landscape of life sciences, we specialize in navigating the complexities of cell and gene therapy, drug development, and the oncology market, enabling our clients to lead in some of the most revolutionary and high-impact areas of healthcare. Our expertise spans the entire biotech and pharmaceutical value chain, empowering startups, global enterprises, investors, and research institutions that are pioneering the next generation of therapies in regenerative medicine, oncology, and precision medicine. Web: Contact Us USA: +1 804 420 9370 Email: sales@novaoneadvisor.com For Latest Update Follow Us: LinkedIn Towards Healthcare | Towards Packaging | Towards Automotive | Towards Chem and Materials | Towards FnB | Towards Consumer Goods | Statifacts | Towards EV Solutions | Towards Dental | Market Stats Insight | Nutraceuticals Func Foods | Onco Quant | Sustainability Quant | Specialty Chemicals Analytics

VaccineGene TherapyImmunotherapyPhase 2

19 Jan 2026

·www.biospace.com

JPM26: Galapagos’ New CEO Is Here to Finally Turn The Ship Around

iStock, Hwangdaesung Henry Gosebruch, who has $3.5 billion in capital to deploy, is thinking broad as he steers the decades-old biotech out of years of turmoil.  Galapagos CEO Henry Gosebruch truly has a clean slate to work with in rebuilding the storied—but chronically unlucky—company. Brought in last year to replace Paul Stoffels , who made multiple attempts to right the ship , Gosebruch’s mandate is to find a whole new direction. The company has suffered from a series of clinical failures, FDA rejections, restructuring and leadership changes. He has cash—about €3 billion ($3.5 billion)—and the board’s backing to find whatever asset, technology or disease area he wants. “On the one hand, it’s like, well, what’s it going to be? But on the other hand, that’s the beauty of what we have, that we can be much more broad in our thinking and really just look at the best opportunity,” Gosebruch told BioSpace on the sidelines of the J.P. Morgan Healthcare Conference last week. This wide-open opportunity is what attracted Gosebruch in the first place—even if the role he was originally offered was quite different. He was brought into the fold in April 2025 to head a proposed spin-out company that would build a brand new pipeline. Barely a month later, the company reversed course on the spin-out idea and announced Stoffels’ departure. Gosebruch instead became CEO of the original Galapagos entity, which would carry on the mission of finding a new pipeline. Deals Almost 5 Years into $5B Galapagos Partnership, Gilead Execs Start Over With New Spinoff Five years ago, Gilead signed a massive deal with Galapagos. After a restructuring, the pharma is still hunting for the potential it saw at the original signing. January 22, 2025 · 5 min read · Annalee Armstrong Read More Later, Galapagos ditched the cell therapy assets that Stoffels had been carefully curating over his nearly five-year term through a series of acquisitions. Explaining the decision to back off cell therapy, Gosebruch said it was a long time coming. The investment needed would have been too great to advance the technology far enough to be competitive. Gosebruch said it would have cost hundreds of millions of dollars. Those conversations were happening well before Gosebruch arrived, but they intensified in the past year. On January 5, the board officially announced the wind-down of cell therapy at Galapagos. Galapagos originally intended to find partners for the existing programs, which include the Phase II CD19 CAR T GLPG5101 for blood cancer, but that has not materialized. “We’re continuing to be open, but at this point, nobody’s come forward, really, with a proposal that makes sense to pursue,” Gosebruch said. New Outlook Of his predecessor Stoffels, Gosebruch expressed his admiration and said the board made the decision to part ways. “Paul for many, many years and I have a lot of respect, but he was very, very focused on cell therapy,” Gosebruch explained. “That was sort of his mission, to bring this set of programs to patients. And I think, given how focused he was on that particular mission, I think he was not focused on opening up to other potential opportunities.” Gosebruch, on the other hand, has an open mind: “My approach will be that I’m not wedded to one area over another; I’m going to be looking more broadly.” Galapagos does have a legacy pipeline consisting of one molecule, a TYK2 small molecule for immunology called GLPG3667. It’s “the one program that survived from the sort of olden days,” Gosebruch said. The therapy is currently being tested in Phase II trials for systemic lupus erythematosus and dermatomyositis. But GLPG3667 may not ultimately stay in Galapagos’ hands either, Gosebruch said. “Now we are thinking about, given that we don’t have the broad development team anymore, we don’t have the big infrastructure anymore that we had back in the day, does it make sense for us to rebuild around that asset, or does it make sense for us to work with a partner who may already have that?” No ‘Synergy’ Here The new CEO was tight lipped on what he wants to bring into Galapagos. He noted that, at the J.P. Morgan conference, he had “a very packed calendar of BD discussions.” He was also meeting with investors to explain the company’s new focus. Immunology and inflammation and solid tumors are likely candidates and would match Galapagos’ historical focus. “I’d love to find a way to still see that vision through,” the CEO said. “And I think there are some very exciting opportunities in immunology.” Gosebruch also spent time at I&I king AbbVie, so assets in this area would be complimentary to his experience, while solid tumors would fit in with long-time partner Gilead. Galapagos still has strong ties with Gilead, which has a major investment in the biotech. Gosebruch said the relationship is strong and that he spent time with the pharma’s leadership even before accepting the role to understand what they want out of the partnership. He doesn’t think Gilead will simply hand off a program to Galapagos—the goal is to spend the capital on hand to find new opportunities, Gosebruch said. Gosebruch added that the company has a team exploring China for opportunities, just like many peers across the biopharma industry. “I think it’s—I’m going to use the word required—actually,” Gosebruch said. “If you want to do BD these days, you have to be in China, at least to understand what’s the competitive landscape.” Special edition Deep Dive: Chinese Biotech Innovation Can’t Be Stopped In this deep dive, BioSpace investigates China’s rise as a biotech powerhouse. October 27, 2025 · 3 min read · Annalee Armstrong Read More One important piece of leverage that Galapagos has in business development discussions is that the biotech has shrunk down over the past year via layoffs. After the cell therapy restructuring is complete, Gosebruch estimated the remaining workforce will be about 35-40 people. So a whole company could join Galapagos without risk of layoffs—which commonly happen after a merger. “One of the things we can offer to a potential transaction party is that if there’s a strong R&D team or a strong commercial team, they could, as a full team, come to Galapagos and be our team going forward,” Gosebruch said. “There won’t be any synergy.” Gosebruch didn’t write off any potential diseases or opportunities and said he plans to be thoughtful—while acknowledging that investors have been very patient amid the restructuring. “I’d rather take more time, be patient and wait for the right opportunity to come our way than rush into anything.” .responsive-container { display: flex; flex-wrap: wrap; border: 1px solid #ededed; font-family: Helvetica, Arial, Sans-Serif; padding: 20px 20px 10px 20px; } .column-left { flex: 1; max-width: 45%; padding: 20px 20px 10px 20px; } .column-right { flex: 2; padding: 20px 20px 10px 20px; } @media (max-width: 768px) { .column-left, .column-right { max-width: 100%; flex: 100%; padding: 10px 0; } } Subscribe to BioPharm Executive! 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Phase 2Cell TherapyExecutive ChangeAcquisition

08 Dec 2025

·www.globenewswire.com

Galapagos Presented New ATALANTA-1 Cell Therapy Data in MCL at ASH 2025

High complete response rates and minimal residual disease (MRD) negativity, with durable responses, in high-risk mantle cell lymphoma (MCL) with GLPG5101, a fresh, early memory-enriched CAR T-cell therapy with a 7-day vein-to-vein time Mechelen, Belgium; December 8, 2025, 07:30 CET; Galapagos NV (Euronext & NASDAQ: GLPG) announced new and updated Phase 2 data from the ongoing ATALANTA-1 study with its CD19 CAR T-cell therapy candidate, GLPG5101, during an oral presentation (#662) at the 67th American Society of Hematology (ASH) Annual Meeting. “The new and updated results from the Phase 2 ATALANTA-1 study demonstrate that GLPG5101 offers timely treatment with low rates of high-grade toxicities and durable responses for patients with relapsed or refractory MCL,” said Marie José Kersten, MD, ATALANTA-1 Principal Investigator and Professor of Hematology at Amsterdam University Medical Center. “The short 7-day vein-to-vein time enabled a low dropout rate and eliminated the need for bridging therapy, allowing more patients to receive treatment who otherwise might not have been able to access CAR T-cell therapy.” Summary of ATALANTA-1 data from the MCL cohort (pooled data across two dose levels):As of September 2, 2025 (data cut-off date), 26 heavily pretreated MCL patients had undergone leukapheresis and 25 had received an infusion of GLPG5101 (4% dropout rate). Of these, 24 patients received a fresh product, with 23 infused within seven days after apheresis. Among infused patients (N=24), the objective response rate (ORR) was 100%, with a complete response rate (CRR) of 96%. Duration of response (DOR) and progression-free survival (PFS) rates were both 83% at a median follow-up of 9 months.9 of 10 (90%) of minimal residual disease (MRD)-evaluable patients were MRD-negative at CR and 7 of 9 MRD-negative patients remained in CR at the time of the data cut-off.GLPG5101 showed an encouraging safety profile (N=24). The most common Grade ≥ 3 treatment-emergent adverse events were hematologic. No Grade ≥ 3 CRS was observed, and only one case of Grade ≥ 3 ICANS occurred.GLPG5101 demonstrated robust in vivo CAR T-cell expansion and long-term persistence with an enrichment of early memory phenotypes. Intention to wind down Galapagos’ cell therapy activities As announced on October 21, 2025, and following a comprehensive strategic and evaluation and sales process, Galapagos remains focused on the intention to wind down the cell therapy activities. This intention is subject to the conclusion of consultations with works councils in Belgium and the Netherlands, during which Galapagos will continue to operate the business and conduct ongoing clinical studies. Galapagos would still consider any viable proposal to acquire all, or part of the cell therapy business, should such a proposal emerge during the wind down process. About GLPG5101 and ATALANTA-1 (EudraCT 2021-003272-13; NCT 06561425) GLPG5101 is a second generation anti-CD19/4-1BB CAR-T product candidate, administered as a single fixed intravenous dose. The safety, efficacy and feasibility of decentralized manufactured GLPG5101 are currently being evaluated in the ATALANTA-1 Phase 1/2 study in eight hematological malignancies with high unmet need. The primary objective of the Phase 1 part of the study is to evaluate safety and to determine the recommended dose for the Phase 2 part of the study. Secondary objectives include assessment of efficacy and feasibility of decentralized manufacturing of GLPG5101. The dose levels that were evaluated in Phase 1 are 50×106 (DL1), 110×106 (DL2) and 250×106 (DL3) CAR+ viable T-cells. The primary objective of the Phase 2 part of the study is to evaluate the Objective Response Rate (ORR) while the secondary objectives include Complete Response Rate (CRR), duration of response, progression free survival, overall survival, safety, pharmacokinetic profile, and the feasibility of decentralized manufacturing. Each enrolled patient will be followed for 24 months. The ATALANTA-1 study is currently enrolling patients in the U.S. and Europe. For further information, contact Galapagos:Investor RelationsGlenn Schulman +1 412 522 6239ir@glpg.com Corporate CommunicationsMarieke Vermeersch+32 479 490 603 media@glpg.com Visit us at www.glpg.com or follow us on LinkedIn or X. Forward-looking statementsThis press release includes forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995, as amended. These statements are often, but are not always, made through the use of words or phrases such as “anticipate,” “expect,” “plan,” “estimate,” “will,” “continue,” “aim,” “intend,” “future,” “potential,” “could,” “indicate,” “forward,” “may,” as well as similar expressions. Forward-looking statements contained in this press release include, but are not limited to, statements regarding Galapagos’ plans, expectations and strategy with respect to its cell therapy business, including statements regarding its plans, expectations and strategy for GLPG5101 and its other product candidates and partnered programs, Galapagos’ intention to wind down its cell therapy business as part of its ongoing transformation, the expected timing, design and readouts of the ATALANTA-1 study, and the potential benefits of Galapagos’ product candidates. Forward-looking statements involve known and unknown risks, uncertainties and other factors which might cause Galapagos’ actual results to be materially different from those expressed or implied by such forward-looking statements. These risks, uncertainties and other factors include, without limitation, the risk that Galapagos will not be able to successfully implement the winding down of its cell therapy business within the expected timeframe or at all, or if implemented, the wind down will not achieve its anticipated economic benefits; the risk that preliminary or interim clinical results may not be replicated in ongoing or subsequent clinical trials, the risk that ongoing and future clinical studies with Galapagos’ product candidates, including GLPG5101, may not be completed in the currently envisaged timelines or at all, the inherent uncertainties associated with competitive developments, clinical trial and product development activities and regulatory approval requirements (including that data from the ongoing and planned clinical research programs may not support registration or further development of GLPG5101 due to safety, efficacy or other reasons), Galapagos' reliance on collaborations with third parties (including its collaboration partners Lonza and US WorldMeds), and that Galapagos’ estimations regarding its GLPG5101 development programs and regarding the commercial potential of GLPG5101 may be incorrect, as well as those risks and uncertainties identified in Galapagos’ Annual Report on Form 20-F for the year ended 31 December 2024 filed with the U.S. Securities and Exchange Commission (SEC) and its subsequent filings with the SEC. All statements other than statements of historical fact are statements that could be deemed forward-looking statements. The forward-looking statements contained herein are based on management’s current expectations and beliefs and speak only as of the date hereof, and Galapagos makes no commitment to update or publicly release any revisions to forward-looking statements in order to reflect new information or subsequent events, circumstances or changes in expectations. Further, Galapagos cannot assess the impact of each such factor on its business or the extent to which any factor, or combination of factors, may cause actual results to be materially different from those contained in any forward-looking statement. Attachment Galapagos Presented New ATALANTA-1 Cell Therapy Data in MCL at ASH 2025

Cell TherapyClinical ResultASHPhase 2Immunotherapy

100 Deals associated with GLPG-5101

R&D Status

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Hematologic Neoplasms	Phase 3	United States	Lakefront Biotherapeutics	09 Sep 2024
Hematologic Neoplasms	Phase 3	Belgium	Lakefront Biotherapeutics	09 Sep 2024
Hematologic Neoplasms	Phase 3	Finland	Lakefront Biotherapeutics	09 Sep 2024
Hematologic Neoplasms	Phase 3	Netherlands	Lakefront Biotherapeutics	09 Sep 2024
Hematologic Neoplasms	Phase 3	Spain	Lakefront Biotherapeutics	09 Sep 2024
Burkitt Lymphoma	Phase 2	Belgium	Lakefront Biotherapeutics	01 Nov 2025
Chronic Lymphocytic Leukemia	Phase 2	Belgium	Lakefront Biotherapeutics	01 Nov 2025
Mantle-Cell Lymphoma	Phase 2	Belgium	Lakefront Biotherapeutics	01 Nov 2025
Primary Central Nervous System Lymphoma	Phase 2	Belgium	Lakefront Biotherapeutics	01 Nov 2025
B-cell lymphoma refractory	Phase 2	United States	Lakefront Biotherapeutics	09 Mar 2022

Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT06561425 (ATALANTA-1, ASH 12025 \| ASH 22025)	Phase 1/2	Mantle-Cell Lymphoma	20	GLPG5101	xcxmwuujer \| jyjvdrjpek(ugewurjfgd) = hematologic; serious infections were reported in 3/17 (18%) pts. G1/2 CRS was reported in 11/17 (65%) pts and G1 ICANS in 5/17 (29%) pts. There were no G≥3 CRS or ICANS events. G≥3 cytopenia was reported in 5/17 (29%), 4/17 (24%), and 4/15 (27%) pts at 30, 60, and 90 days post‐infusion, respectively. qdrpgmsuvv (ikjjhwsykb )	Positive	06 Dec 2025
NCT06561425 (ATALANTA-1, ASH2025)	Phase 1/2	Diffuse large B-cell lymphoma recurrent	24	GLPG5101	wgaqswnyoy(tkhbpxyicx) = vljjtesspc uggkzqhpbw (sdncammocu ) View more	Positive	06 Dec 2025
NCT06561425 (ATALANTA-1, ASH2025)	Phase 1/2	Diffuse large B-cell lymphoma recurrent	24	GLPG5101 (3L+)	wgaqswnyoy(tkhbpxyicx) = cctlmdpgbe uggkzqhpbw (sdncammocu ) View more	Positive	06 Dec 2025
NCT06561425 (ATALANTA-1, EHA2025)	Phase 1/2	Non-Hodgkin Lymphoma CD19	64	GLPG5101	jpqsqqlmca(aiypieckjf) = There were 6 deaths: 2 due to progressive disease and 4 due to AEs (1 intra-abdominal hemorrhage, 1 pneumonia and respiratory distress considered unrelated to infusion; 1 COVID‑19 pneumonia, 1 E. coli sepsis during the follow-up period [>14 weeks post-infusion] considered possibly related to infusion) vaigyztqql (eswfxvjqdy ) View more	Positive	14 May 2025
NCT06561425 (ATALANTA-1, ASH2024) Manual	Phase 1/2	Non-Hodgkin's lymphoma refractory	45	GLPG5101	ypwkuwuriw(qoxksxetuz) = pexdwcnwts eltadxncaz (tmwmjmjqou ) View more	Positive	07 Dec 2024
NCT06561425 (ATALANTA-1, ASH2024) Manual	Phase 1/2	Non-Hodgkin's lymphoma refractory	45	GLPG5101 (MCL)	ypwkuwuriw(qoxksxetuz) = geealzuvtp eltadxncaz (tmwmjmjqou ) View more	Positive	07 Dec 2024

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