Other reports


With our Healthcare research, we aspire to make a positive difference for patients. Our business sector-wide “Focused Leadership” approach to pipeline enrichment builds on our established expertise in the underlying disease biology of our core therapeutic areas of oncology, neurology and immunology as well as technological capabilities. By building on our existing strengths and maximizing synergies within our pipeline of compounds discovered in-house and with external assets, we will secure sustainable R&D productivity in order to provide innovative medicines to patients in need. In November 2022, we announced that we would aim to launch one new product or indication every 1.5 years on average, bolstered by external innovation.


In Oncology, our scientific curiosity and dedication to patients are at the heart of our efforts to improve the future of people living with cancer. In this core focus area of our R&D portfolio, we aim to deliver transformative treatments. Translational research is embedded into the whole R&D process, with several projects addressing unmet needs in difficult-to-treat cancers through innovative treatment approaches and novel combinations.

We are committed to bringing new standards of care for multiple tumor types to as many patients as possible worldwide. Therefore, in 2023 we continued to explore the impact of our marketed therapies through continued analysis of data from our pivotal studies and the generation of real-world evidence. We are assessing these treatments in new settings as well.


To date, Bavencio® (avelumab), an anti-PD-L1 antibody, has been approved in 66 countries as 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. At the 2023 American Society of Clinical Oncology (ASCO) Genitourinary Cancers Symposium, we presented long-term follow-up data from the Phase III JAVELIN Bladder 100 trial. The data demonstrated median overall survival from start of chemotherapy of 29.7 months among patients receiving Bavencio® who did not progress on first-line platinum-based chemotherapy, thus establishing a new benchmark for treatment outcomes in clinical studies.

We continue to evaluate whether optimization of first-line maintenance treatment by adding a novel therapy to avelumab could improve outcomes for patients with advanced UC whose disease did not progress with first-line platinum-based chemotherapy in the Phase II JAVELIN Bladder Medley study. Initiated in 2022, this randomized umbrella study is assessing avelumab monotherapy versus the combination of avelumab with our investigational anti-TIGIT antibody M6223, avelumab in combination with Nektar Therapeutics’ interleukin-15 (IL-15) receptor agonist, NKTR-255, and avelumab in combination with Gilead Sciences’ Trodelvy® (sacituzumab govitecan-hziy).

Bavencio® is also approved as a monotherapy for the treatment of metastatic Merkel cell carcinoma (MCC) in 63 countries. In September 2023, we received U.S. Food and Drug Administration approval of a supplemental Biologics Licensing Application for Bavencio®, converting the MCC indication from accelerated approval into full approval. This makes it the first MCC treatment to receive full approval in the U.S. market.

Additionally, Bavencio® is approved for the treatment of advanced renal cell carcinoma (RCC) in combination with axitinib in 60 countries.


In 2023, we shared multiple analyses of studies of the oral MET inhibitor Tepmetko® (tepotinib) in advanced non-small cell lung cancer (NSCLC). In a long-term follow-up analysis of the Phase II VISION study published in JAMA Oncology, Tepmetko® showed robust and durable clinical activity across therapy lines in patients with METex14-skipping NSCLC, particularly in previously untreated patients with METex14 skipping confirmed by tissue biopsy. An additional subgroup analysis presented at the 2023 World Congress on Lung Cancer (WCLC) in September demonstrated the robust and durable clinical activity of Tepmetko®, particularly as a first-line treatment, with stability in health-related quality of life and a manageable safety profile in Asian patients with advanced NSCLC with METex14 skipping. Tepmetko® is now available for the treatment of METex14-skipping NSCLC in 23 markets globally.

We also shared results of the primary analysis of the Phase II INSIGHT 2 study at the WCLC. These findings suggest the potential of tepotinib plus osimertinib as a chemotherapy-sparing oral targeted therapy option for patients with EGFR-mutant NSCLC with MET amplification who have developed resistance to prior EGFR tyrosine kinase inhibitor therapy.

Novel medicines

As we work towards our vision of creating a world where more cancer patients can become cancer survivors, we continue to pioneer novel medicines, advancing promising molecules in our pipeline that build on our expertise and leadership in core mechanisms and tumor types.

Our Phase III development program for xevinapant, the potentially first-in-class IAP (inhibitor of apoptosis protein) inhibitor, in the treatment of squamous cell carcinoma of the head and neck (SCCHN) continues to progress. Patient enrollment for the TrilynX study (NCT04459715) was completed in 2023. This international, randomized, double-blind, placebo-controlled Phase III study evaluates the efficacy and safety of xevinapant compared to placebo when administered in addition to definitive chemoradiotherapy in patients with unresected, locally advanced SCCHN. Patient recruitment continues in the international, randomized, double-blind, placebo-controlled Phase III XRay Vision (NCT05386550) study, which is evaluating the efficacy and safety of xevinapant versus placebo in combination with adjuvant, post-operative radiotherapy in patients with resected LA SCCHN who are at high risk for relapse and are ineligible for cisplatin treatment.

Additional progress in our pipeline in 2023 includes completion of Phase Ia for our anti-CEACAM5 antibody-drug conjugate (ADC), M9140, with the identification of two doses for evaluation in Phase Ib. M9140 is the first ADC based on our proprietary technology to enter clinical development.

We also continued to advance our pipeline of DNA damage response inhibition (DDRi) assets, exploring multiple hypotheses to determine which regimens may provide the most value to patients. In 2023, we initiated the Phase Ib/IIa DDRiver NSCLC 322 study of tuvusertib (M1774), our potentially best-in-class, potent and selective inhibitor of ataxia telangiectasia and Rad3-related (ATR), in combination with Regeneron Pharmaceutical’s PD-1 inhibitor cemiplimab in patients with non-squamous NSCLC that has progressed on prior anti-PD-(L)1 and platinum-based therapies. The first dose was administered in October to a person requiring treatment.

In July 2023, our collaboration partner Telix Pharmaceuticals announced the administration of the first dose in the Phase Ib STARSTRUCK trial. This open-label, single-arm, multicenter dose-escalation and dose-expansion study will evaluate the safety profile, dosing and activity of our DNA-dependent protein kinase (DNA-PK) inhibitor candidate, peposertib (M3814), in combination with Telix’s investigational targeted radiation therapy, TLX250, in patients with solid tumors expressing carbonic-anhydrase IX (CAIX).

To diversify our robust internal pipeline in our focus areas of DNA damage response inhibition and antibody-drug conjugates, in October 2023 we announced a strategic collaboration with Jiangsu Hengrui Pharmaceuticals Co. Ltd. (Hengrui). The partnership includes an exclusive global license (excluding mainland China) to develop, manufacture and commercialize Hengrui’s next-generation potent and selective PARP1 (poly (ADP-ribose) polymerase 1) trapping inhibitor HRS-1167. The agreement also includes an option for exclusive global development, manufacturing and commercialization (excluding mainland China) of Hengrui’s Claudin-18.2 antibody-drug conjugate (ADC) SHR-A1904.

In December, we announced a license agreement with Abbisko Therapeutics Co. Ltd, Shanghai, China, for pimicotinib (ABSK021), which is currently being evaluated in a Phase III study for the treatment of tenosynovial giant cell tumor (TGCT). TGCT is a benign tumor of the joints that can cause swelling, pain, stiffness, and limited mobility of the affected joints. The agreement grants us a license to commercialize pimicotinib in mainland China, Hong Kong, Macau and Taiwan, with an option for rest of world.

Highlights of congress publications in 2023

We shared additional new data for our marketed and investigational oncology medicines at major oncology congresses.

In June, 43 abstracts featuring new data for the medicines Bavencio® (avelumab), Erbitux® (cetuximab) and Tepmetko® (tepotinib) and drug candidates from our pipeline including the first-in-class investigational IAP inhibitor xevinapant were presented at the ASCO Annual Meeting.

Highlights included:

  • Clinical data for Bavencio® that reinforce its role as a standard of care in first-line maintenance for advanced urothelial carcinoma in patients without disease progression following first-line platinum-based chemotherapy. Poster discussions, including long-term safety analyses and an analysis of quality-adjusted survival from the Phase III JAVELIN Bladder 100 study, confirm the acceptable long-term benefit-risk profile as well as the net benefit estimate of Bavencio® in first-line maintenance and further support its use.
  • Long-term outcomes from the VISION study, the largest study of a MET inhibitor in patients with METex14-skipping advanced NSCLC (N=313). Detection was carried out via liquid and/or tissue biopsy. The results demonstrate the robust and sustained clinical activity of Tepmetko®, particularly in the first-line setting: with a median follow-up time of 32.6 months, the overall response rate in 164 people treated with first-line therapy was 57.3% (95% CI: 49. 4, 65.0) and the median duration of response 46.4 months (13.8, cannot be estimated).
  • Additional presentations for Tepmetko® that included analyses of the INSIGHT 2 study inNSCLC with epidermal growth factor receptor (EGFRm) mutation and MET amplification during treatment with Tepmetko® plus osimertinib.
  • Erbitux® data that add to the growing body of evidence supporting the role of cetuximab-based therapies across the continuum of care in the treatment of RAS wild-type metastatic colorectal cancer and as a backbone of treatment in SCCHN.

At the European Society for Medical Oncology (ESMO) Congress 2023, we presented 28 abstracts featuring the latest research on our oncology portfolio addressing unmet treatment needs across bladder, head and neck, lung, colorectal, and other cancers.

Highlights included:

  • New analyses and real-world evidence that reinforce the role of Bavencio® first-line maintenance in the treatment of advanced UC in patients with varying characteristics. These include long-term efficacy and safety outcomes from the Phase III JAVELIN Bladder 100 study that confirm the prolonged overall survival (OS), progression-free survival (PFS) and tolerability of first-line maintenance with Bavencio® in patients older than 65 years with advanced UC. Further evidence from France and the United States, including initial data from the French AVENANCE study on patients with advanced UC whose tumors have histological variants, support the findings of JAVELIN Bladder 100 in real-world settings.
  • Additional real-world analyses reinforcing the use of Bavencio® as a treatment for advanced/metastatic MCC. After a median follow-up of approximately 29 months, data from the MCC TRIM study showed a median OS of 52 months for patients with metastatic MCC treated with Bavencio® in a real-world setting in Germany. Most patients (approximately 86%) received first-line Bavencio®.
  • Updated findings from the Phase II VISION trial, which is the largest study of a MET inhibitor in METex14-skipping NSCLC and served as the basis for regulatory approvals, continue to show clinically meaningful long-term efficacy in patients treated with Tepmetko® regardless of line of therapy (2L, 2L+ and 3L+).
  • A new analysis of real-world survival outcomes and survival risk factors in elderly patients with locally advanced SCCHN that highlights poor survival outcomes, especially in patients aged 70 years and older with advanced disease stage and comorbidities, underscoring the need for innovative effective treatments for this population.

Neurology & Immunology

With a commitment of more than 25 years to people living with multiple sclerosis (MS), our ongoing dedication to science drives us to discover cutting-edge therapies through our research in neurological and immune-mediated disease areas.

Beyond our portfolio in MS, we have a pipeline focusing on discovering new therapies that have 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 blocking the activation of toll-like receptors (TLR7 and TLR8), is being developed as a new oral therapy for SLE and CLE. It aims to overcome limitations of currently available lupus therapies by providing selective inhibition of lupus-relevant disease drivers, which may increase efficacy while preserving immunity against infections. We anticipate data from our Phase II clinical trials for enpatoran in the first half of 2024.

We are also exploring the potential of oral cladribine beyond MS, developing it for the treatment of gMG, which affects an estimated 700,000 people and where a high unmet need remains, particularly as regards oral treatment options. Cladribine is believed to work by affecting the pathogenic pathways involved in the development of autoimmunity (auto-antibody producing B cells and T cells). In June 2023, the FDA granted Orphan Drug Designation for cladribine for the treatment of myasthenia gravis. We anticipate the initiation of a global Phase III clinical trial program in the second quarter of 2024.

In February 2023, we entered a preclinical licensing and strategic research partnership with Aqilion, a biotech company focused on developing innovative treatments for immune-mediated and neurological diseases.

New data for our existing therapy Mavenclad® (cladribine tablets), as well as for our investigational drug evobrutinib, were presented at key congresses in 2023, including the Americas Committee for Treatment and Research in Multiple Sclerosis (ACTRIMS) Forum in February, the American Academy of Neurology (AAN) Annual Meeting in April and the European Committee for Treatment and Research in Multiple Sclerosis (ECTRIMS) Congress in October.

At ACTRIMS 2023, we presented data that included analyses of the CLARIFY-MS study, showing the potential of Mavenclad® to improve outcomes in an impactful way for people living with RMS. In addition, we showed updated long-term efficacy and safety data from our Phase II program for the investigational drug evobrutinib. In December we shared that the phase III pivotal study did not meet its primary endpoint of annualized relapse rate for up to 156 weeks compared to oral teriflunomide.

At AAN 2023, we presented data from the M AGNIFY-MS study, showing sustained reductions in the memory B-cell numbers, with changes towards anti-inflammatory phenotypes in circulating B- and T-cell types for study participants taking Mavenclad® and provided updated efficacy and safety data from our Phase II program for the investigational drug evobrutinib.

At ECTRIMS 2023, we presented 31 abstracts in total, including long-term efficacy and neurofilament light chain data (from the M AGNIFY-MS study) for Mavenclad® as well as new real-world evidence data highlighting naïve use of the treatment. In addition, we shared updated five-year safety and efficacy data from the Phase II Open Label Extension for investigational evobrutinib as well as baseline demographic data of our Phase III EVOLUTION trials.


As the global market leader in fertility drugs and treatments, our Fertility franchise plays a crucial role in our Healthcare business.

Infertility is an increasing challenge globally due to demographic changes and lifestyle adjustments such as delayed childbearing. Based on the latest data from WHO, one in six people worldwide are affected by infertility.

According to updated data, more than five million babies have been born worldwide with the help of Gonal-f®, a leading therapeutic within our fertility portfolio. It contains the active ingredient follitropin alfa (r-hFSH alfa), which is a recombinant form of the natural hormone FSH and is available in a convenient and ready-to-use pre-filled injection pen. Treatment with Gonal-f® can result in increased follicles, oocytes and embryos compared with urinary gonadotropins, thereby improving the chances of pregnancy and live birth. Recent real-world evidence studies based on key European registries (D.I.R., SNDS) showed increased likelihood of live birth with Gonal-f® compared with urinary gonadotropins and biosimilar preparations of follitropin alfa.

Cardiovascular, Metabolism & Endocrinology*

In view of the significant and growing impact of chronic diseases such as diabetes, prediabetes, hypertension, and cardiovascular disease, growth hormone disorders and thyroid disorders on health and society in the 21st century, we are committed to helping patients with these conditions.

The new formulation of Euthyrox® (levothyroxine) for the treatment of hypothyroidism obtained further regulatory approvals in 2023, resulting in a total of 101 countries where this incremental innovation is registered. With its characteristics of delivering precise, fine-tuned and stable T4 doses, Euthyrox® may help optimize disease management, making it a good choice for healthcare providers and patients.

Glucophage®, containing the active ingredient metformin, is the most widely prescribed non-insulin diabetes treatment worldwide for first-line treatment of type 2 diabetes for which we achieved a successful label extension in Europe in 2022. The label update on the mechanism of action is evidence of the still growing body of knowledge and opportunities for metformin in the diabetes continuum. Those label updates are currently being rolled out in all other countries outside Europe where the Glucophage® family of products is available.

Concor®/Concor Cor®, containing bisoprolol, is a beta-blocker for treating hypertension and cardiovascular diseases such as coronary heart disease and chronic heart failure. In addition to Concor®/Concor Cor®, the Concor® franchise includes fixed-dose combinations such as Concor Plus®/Lodoz® (bisoprolol with hydrochlorothiazide) and Concor® AM (bisoprolol with amlodipine). Concor® AM has been registered in 71 countries.

Investments to speed up the availability of new medicines*

Our declared aim is to bring more medicines to more patients faster. In 2023, we supported this aim by reaching key milestones for two transformational investments focusing on complementary therapeutic modalities:

  • In June, we inaugurated our Biotech Development Center at our site in Corsier-sur-Vevey, Switzerland. This investment of over € 250 million aims to ensure that our next generations of innovative large-molecule medicines (biotech therapies and potential other new therapeutic modalities) are available for clinical trials on time and in the required quality and quantity with an accelerated process compared with the past. The Biotech Development Center is expected to be fully operational in early 2024 following validation by regulatory authorities.
  • In September, we celebrated the topping-out for our Launch and Technology Center at our site in Darmstadt, Germany. This investment of approximately € 160 million is intended to ensure that our next generations of innovative small-molecule medicines (including high-potency compounds) are available for clinical trials, global launches and commercial supply on time and in the required quality and quantity, with accelerated processes compared with the past. The Launch and Technology Center is anticipated to be fully operational by the end of 2025 following validation by regulatory authorities.

Collaborations to strengthen AI-driven drug discovery*

On September 20, we announced two strategic collaborations with Benevolent AI and Exscientia to drive accelerated drug discovery with higher probability of success. Access to end-to-end AI platform capabilities is expected to generate several novel development candidates in oncology, neurology and immunology. AI-powered R&D is an integral part of delivering on our ambition to bring more medicines to more patients, faster.

Our pipeline

Our pipeline

As of December 31, 2023










Therapeutic area















Enpatoran (TLR7/8 antagonist)


Systemic lupus erythematosus1


Phase II

Enpatoran (TLR7/8 antagonist)


Cutaneous lupus erythematosus1


Phase II

Enpatoran (TLR7/8 antagonist)


Idiopathic inflammatory myopathies (DM and PM)2


Phase II











Xevinapant (IAP inhibitor)


Locally advanced squamous cell carcinoma of the head
and neck – Unresected, cisplatin-eligible3


Phase III

Xevinapant (IAP inhibitor)


Locally advanced squamous cell carcinoma of the head
and neck – Resected, cisplatin-ineligible4


Phase III

Avelumab (anti-PD-L1 mAb) + combinations


Locally Advanced or Metastatic Urothelial Carcinoma5


Phase II

Tuvusertib/M1774 (ATR inhibitor)


Solid tumors6


Phase Ib

M4076 (ATM inhibitor)


Solid tumors7


Phase Ib

M9140 (anti-CEACAM5 Antibody drug conjugate)


Solid tumors


Phase Ia

M6223 (anti-TIGIT mAb)


Solid tumors8


Phase Ib

M9466 (HRS-1167; Selective PARPi)


Solid tumors9


Phase I






Global Health





Arpraziquantel (anthelmintic)


Pediatric schistosomiasis10



M5717 (PeEF2 inhibitor)




Phase II

On December 04, 2023, we announced a license agreement with Abbisko Therapeutics Co. Ltd, China, for pimicotinib (ABSK021), which is currently being evaluated in a Phase III study for the treatment of tenosynovial giant cell tumor (TGCT). The agreement grants us a license to commercialize pimicotinib in mainland China, Hong Kong, Macau and Taiwan, with an option for rest of world.

End of December 2023, we entered into a licensing agreement with Inspirna, Inc., United States, for ompenaclid (RGX-202), a first-in-class oral inhibitor of the creatine transport channel SLC6A8, and SLC6A8-targeting follow-on compounds. Ompenaclid is currently being evaluated in a Phase II study for the second-line treatment of RAS-mutated (RASmut) advanced or metastatic colorectal cancer (mCRC).

Unless noted otherwise, clinical programs conducted in collaboration with external partners are not shown unless we have co-ownership of data. More information on the ongoing clinical trials can be found at www.clinicaltrials.gov. Pipeline products are under clinical investigation and have not been proven to be safe and effective. There is no guarantee any product will be approved in the sought-after indication.



Clinical trial passed futility analysis.


Dermatomyositis and Polymyositis.


In combination with cisplatin and radiotherapy in unresected LA SCCHN patients eligible for the treatment with cisplatin.


In combination with radiotherapy in resected LA SCCHN patients ineligible for the treatment with cisplatin.


Combinations include Sacituzumab Govitecan, NKTR-255 and M6223.


Studies as monotherapy and in combination with cemiplimab, niraparib, avelumab or M4076 ATMi. Includes studies (phase I/II) in collaboration with/ sponsored by external partners, e.g. US National Cancer Institute (NCI).


Administered in combination with Tuvusertib/M1774 (ATRi).


Administered in combination, including combinations other than avelumab.


On October 30, 2023, we announced a collaboration with Jiangsu Hengrui Pharmaceuticals Co. Ltd., China, including an exclusive license worldwide (excluding China) to develop, manufacture and commercialize the next-generation potent and selective PARP1 trapping inhibitor HRS-1167.


On 14 December, 2023 the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) adopted a positive scientific opinion for arpraziquantel for the treatment of schistosomiasis in children aged 3 months to 6 years. The application was submitted by us, on behalf of the Pediatric Praziquantel Consortium, under the EU-M4all procedure for high-priority medicines for human use intended for countries outside the European Union.


ATM: ATM serine/threonine kinase

ATR: Ataxia telangiectasia and Rad3-related

BTK: Bruton’s tyrosine kinase

CEACAM5: Carcinoembryonic antigen-related cell adhesion molecule 5

IAP: Inhibitor of apoptosis proteins

mAb: Monoclonal antibody

PARP1: poly (ADP-ribose) polymerase 1

Phase Ia: Dose finding

Phase Ib: Dose escalation/expansion and signal seeking

PD-L1: Programmed cell death ligand 1

PeEF2: Plasmodium eukaryotic elongation factor 2

TIGIT: T cell immunoreceptor with Ig and ITIM domains

TLR7/8: Toll-like receptors 7 and 8

* The contents of this chapter or section are voluntary and therefore not audited. However, our auditor has read the text critically.

Share this page: