Healthcare

Patients are at the center of all our research and development efforts. We are committed to innovation in science to bring more medicines to more patients, faster. We plan to balance and expand our research and development (R&D) pipeline by acquiring programs through external innovation as well as accelerating internally developed assets that are currently in-house. This approach will enable us to build a sustainable pipeline for long-term growth.

Following an investment of € 160 million, we inaugurated the Launch and Technology Center at our campus in Darmstadt, Germany, in September 2025. The Launch and Technology Center aims to ensure that our next generations of innovative small molecule-based medicines (including high-potency compounds) are available for clinical trials, global launches and commercial supply with accelerated timeframes compared to the past. It is anticipated to be fully operational in 2026 following validation by the health authorities.

Oncology

In Oncology, we are guided by our vision to help cancer patients become cancer survivors. As a key focus area within our R&D portfolio, we are dedicated to delivering transformative treatments. Translational research is integrated throughout the entire R&D process with several projects addressing unmet needs in difficult-to-treat cancers through innovative treatment approaches and novel combinations.

Marketed therapies

We are committed to setting new standards of care for multiple tumor types and expanding access to the corresponding therapies. In 2025, we therefore continued to explore the impact of our marketed therapies by continuously analyzing data from our pivotal trials and generating real-world evidence. Additionally, we are evaluating these treatments in new clinical settings to allow more patients with cancer to experience their potential benefits. 

External research continues to reinforce Erbitux^® (cetuximab) as the backbone of treatment in metastatic colorectal cancer (CRC). At multiple congresses, data were presented from the Phase III BREAKWATER trial conducted by Pfizer Inc., USA, evaluating the clinical efficacy of the combination of mFOLFOX6, encorafenib and Erbitux^® in metastatic BRAF V600E-mutant metastatic CRC. Results from the trial showed a 51% reduction in the risk of death for patients treated with this regimen compared with standard-of-care treatment. The final analysis of the investigator-sponsored FIRE-4 trial evaluating the efficacy of Erbitux^® re-challenge in patients with RAS wild-type metastatic CRC was presented at the 2025 American Society of Clinical Oncology (ASCO^®) Annual Meeting. The trial demonstrated a significantly higher overall response rate and safety in the Erbitux^® plus FOLFIRI-containing experimental arm versus physicians’ choice of treatment. Furthermore, it demonstrated statistically similar but numerically higher overall survival, primary endpoint, and progression-free survival.

We remain committed to fostering innovation in this disease to help address unmet needs for patients with metastatic CRC as well as helping to ensure that Erbitux^®, an important backbone therapy in metastatic CRC, is made available to all patients around the world who could benefit.  

Bavencio^® (avelumab), an anti-PD-L1 antibody, is a first-line maintenance treatment for locally advanced or metastatic urothelial carcinoma (UC) in adult patients whose disease has not progressed following platinum-based chemotherapy. New analyses presented at congresses throughout 2025 continued to strengthen the robust evidence supporting its use in this setting. At multiple scientific congresses, including the ASCO^® Genitourinary Cancers Symposium, the ASCO^® Annual Meeting and the European Society for Medical Oncology (ESMO) Congress, new data were shared from the pivotal Phase III JAVELIN Bladder 100 trial alongside real-world evidence that reinforced the clinical trial findings of Bavencio^® as a first-line maintenance therapy in patients with locally advanced or metastatic UC. The data highlight the effectiveness and safety of Bavencio^® in routine clinical practice and heterogenous populations as well as the importance of personalized treatment decision-making. These data further add to the growing body of evidence supporting the use of Bavencio^® in a rapidly evolving therapy landscape.  

For Tepmetko^® (tepotinib), data from the VISION trial presented at the 2025 European Lung Cancer Congress highlighted the continued robust and durable efficacy and the manageable safety profile of this medicine in patients with treatment-naive and previously treated METex14-skipping non-small-cell lung cancer (NSCLC) after three years or more of follow-up. These findings reinforce Tepmetko^® as a meaningful treatment option in this setting. Additional analyses of VISION presented at the 2025 World Conference on Lung Cancer held by the International Association for the Study of Lung Cancer found that with three or more years of follow-up, Tepmetko^® demonstrated a continued manageable safety profile in patients with METex14-skipping NSCLC with no new safety signals and stability in health-related quality of life and patient-reported outcomes as measured by the symptom scores in the Quality of Life Questionnaire of the European Organisation for Research and Treatment of Cancer. 

At the ESMO Congress 2025, further data from the VISION trial presented at ASCO^® confirmed that Tepmetko^® continues to show robust and sustained efficacy in patients with at least three years of follow-up, irrespective of age, smoking status, the presence of brain metastases at baseline, or whether the MET (gene) alteration was detected by tissue or liquid biopsy. Treatment sequencing with Tepmetko^® was also investigated and results demonstrate that after three years or more of follow-up, Tepmetko^® delivers robust and lasting efficacy across treatment lines and particularly in the first-line setting, supporting its early use in the treatment sequence.

Novel medicines

In 2025, we made significant progress in advancing our antibody-drug conjugates (ADC) from our own research. 

We presented data from the dose optimization section of the Phase I PROCEADE-CRC 01 trial of our anti-CEACAM5 ADC precemtabart tocentecan (M9140) in advanced CRC at the 2025 ASCO^® Annual Meeting, with additional data from this trial presented at the ESMO Congress 2025. These data, which showed a higher objective response rate and overall survival along with a similar safety profile for the higher of the two doses studied, support the rationale for selecting the recommended dose for further development in CRC and other solid tumors, including cancer types being investigated in the ongoing Phase Ib/II PROCEADE-PanTumor trial. Based on the encouraging findings in patients with heavily pretreated advanced CRC, we plan to initiate a Phase III trial of precemtabart tocentecan in this setting in 2026.

Clinical development of M3554, our anti-GD2 ADC, is also underway, with recruitment ongoing in a first-in-human Phase I, multicenter open-label trial in patients with advanced solid tumors.

Within our DDR portfolio (DNS Damage Response), we refined our focus in 2025 based on the data generated to date.

For our ataxia telangiectasia and Rad3-related (ATR) inhibitor tuvusertib, we discontinued investigation of combination approaches with other DDR inhibitors, such as PARP (poly(ADP-ribose) polymerase) (niraparib and M9466) or ataxia telangiectasia mutated kinase (ATM) (lartesertib) in 2025, based on an underwhelming efficacy signal observed in an in-house Phase II trial in patients with ovarian cancer with prior exposure to PARP inhibitors. No new safety signals have been observed in combinations. The termination resulted in an impairment of an intangible asset amounting to € 12 million. Tuvusertib continues to be investigated through external collaboration, with an emphasis on monotherapy in biomarker-defined populations and in combinations with immuno-oncology in different tumor types.

For M9466 (also known as HRS-1167), the selective PARP1 (poly(ADP-ribose) polymerase 1) inhibitor licensed from Jiangsu Hengrui Pharmaceuticals Co. Ltd., China, in 2023, we have investigated opportunities with the intention of leveraging its increased potency and selectivity in combinations with tuvusertib, with cytotoxic chemotherapy, and with hormone treatments across several solid tumor types, including the traditional PARP inhibitor spaces. Based on the emerging efficacy and safety data in combination with other compounds and the rapidly evolving competitive landscape in the established PARP inhibitor space, we have made the strategic decision not to pursue further development. The termination of this trial in Phase Ib resulted in an impairment of an intangible asset amounting to € 174 million as well as the recognition of a provision for follow-up costs in the low double-digit million euro range.

Following the acquisition of SpringWorks Therapeutics, Inc., USA, we are dedicating ourselves to the targeted treatment of patients with additional rare diseases and hematological cancers. In addition to our work in desmoid tumors, we continue to support industry and academic collaborator studies evaluating nirogacestat as part of B-cell maturation antigen combination therapy regimens across treatment lines in patients with multiple myeloma.

With mirdametinib, beyond our work in NF1-PN, a Phase I/II clinical trial evaluating mirdametinib as a monotherapy in pediatric and young adult patients with low-grade gliomas is being conducted by St. Jude Children’s Research Hospital, Memphis, Tennessee, USA. The Phase II portion of the trial is ongoing, and patient enrollment is in progress.

In March 2025, we announced that we had exercised our option with Abbisko Therapeutics Co. Ltd., China, to commercialize pimicotinib in the United States and the rest of the world. We now hold worldwide commercialization rights for pimicotinib. The randomized double-blind treatment phase of the Abbisko-led global Phase III MANEUVER trial of pimicotinib for the treatment of tenosynovial giant cell tumor (TGCT) met its primary endpoint and all key secondary endpoints. Once-daily pimicotinib demonstrated a statistically significant improvement in the primary endpoint of objective response rate. The results were presented for the first time at the 2025 ASCO^® Annual Meeting, with a longer-term analysis – conducted after the last patient completed the open-label treatment phase – presented at the ESMO Congress 2025.

Based on the positive findings from MANEUVER, we submitted applications to regulatory authorities in several regions in 2025. In China, the Center for Drug Evaluation (CDE) of the National Medical Products Administration (NMPA) granted priority review to pimicotinib in May for the treatment of patients with TGCT who require systemic therapy. In June, the CDE accepted our new drug application for marketing authorization of pimicotinib as a Class 1 innovative drug for adult patients with TGCT requiring systemic treatment. Pimicotinib has been granted breakthrough therapy designation by China’s NMPA and the FDA, as well as fast track designation from the FDA and priority medicine designation from the European Medicines Agency. In December 2025, the NMPA approved pimicotinib for the treatment of adult patients with symptomatic TGCT for which surgical resection would potentially cause functional limitation or relatively severe morbidity.

Neurology & Immunology

We are continuing to expand the therapeutic focus areas of our Neurology & Immunology franchise by developing potential first-in-class treatments for conditions with high unmet medical needs. We have a pipeline focusing on discovering new therapies with potential in other neuroinflammatory and immune-mediated diseases, including systemic lupus erythematosus (SLE), cutaneous lupus erythematosus (CLE) and generalized myasthenia gravis (gMG).

Enpatoran, an investigational highly specific potential first-in-class immune modulator, is being developed as a new investigational oral therapy for lupus. It aims to overcome the limitations of currently available lupus therapies by providing selective inhibition of toll-like-receptors (TLR) 7 and 8, which are known as key lupus-relevant disease drivers.

The global Phase II WILLOW trial was uniquely designed to study enpatoran across two lupus cohorts including patients with both active SLE and CLE. In 2025, we shared encouraging results from the WILLOW trial, which indicate that enpatoran demonstrated a meaningful reduction in disease activity among patients with active lupus rash. These findings supported the potential of enpatoran as a viable treatment option for lupus patients. We have held discussions with key health authorities to determine the most effective Phase III pathway for bringing enpatoran to patients in need.

We are also exploring the potential of cladribine capsules for the treatment of gMG, a rare, serious and chronic neuromuscular autoimmune disease affecting an estimated 700,000 people worldwide that leads to progressive and significant muscle weakness, where a high unmet need remains, particularly with regard to oral treatment options. Cladribine capsules are expected to selectively target B and T lymphocytes, which are thought to be the root cause of gMG. We currently have a global Phase III clinical trial ongoing for cladribine capsules for the treatment of gMG.

In February 2025, we also presented new Mavenclad^® (cladribine) tablets data at the 2025 Americas Committee for Treatment and Research in Multiple Sclerosis (ACTRIMS) Forum. The data reinforce Mavenclad^® as being a differentiated disease-modifying therapy for adults with highly active relapsing multiple sclerosis (RMS) by showing consistent safety and high efficacy across a range of disability outcomes, combined with a suggested low treatment burden for both physicians and people living with MS.

Data presented from the CLARIFY-MS extension trial showed the continued effect of Mavenclad^® on non-traditional and patient-centric efficacy measures of disease activity, including health-related quality of life and cognitive function through four years of treatment. These data demonstrated that the mental and physical health improvements as well as cognitive benefits were seen with Mavenclad^® throughout the treatment-free period.

Additionally, two abstracts reporting four-year data from the MAGNIFY-MS extension trial suggest a positive effect of Mavenclad^® on a range of biomarkers for MS in the periphery, including immune cell dynamics, serum neurofilament light chain (sNfL) and immunoglobulins, as well as in cerebrospinal fluid NfL levels and oligoclonal bands.

Results of the two-year MAGNIFY-MS trial suggested the ability of Mavenclad^® to effectively reconstitute the immune system toward a more homeostatic and less pathogenic state without the need for continuous immunosuppression.

At the 2025 congress of the European Committee for Treatment and Research in Multiple Sclerosis (ECTRIMS), we showcased a strong scientific narrative with 37 abstracts in RMS, including four-year data indicating that nearly nine in ten RMS patients treated with Mavenclad^® remained free from progression independent of relapse activity and that Mavenclad^® effectively reduced biomarkers of chronic neuroinflammation, neuroaxonal damage and disease progression while preserving brain volume loss. These results reinforced the potential of the drug to reduce neurodegeneration and neuroinflammation beyond established clinical efficacy outcomes in RMS.

Our pipeline