Innovation

Expert Q&A: The role of real-world evidence in lung cancer detection

Shuvayu Sen, Ph.D., shared how our real-world evidence research uses data to analyze patient journeys and risk prediction models for early lung cancer detection

March 23, 2026

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In the fight against lung cancer, early detection can be critical. While some countries have previously rolled out national lung cancer screening programs, in many cases, participation was low. This reality is one of the drivers behind Mission Lung Cancer, our collective effort to break down the barriers that stand in the way of early detection of lung cancer. 

At the heart of our commitment to early lung cancer detection is one of our powerful contributions: scientific insights. Our real-world evidence (RWE) research uses patient-level data to analyze patient journeys and risk prediction models. This enables us to better understand diagnostic pathways and identify opportunities that may help facilitate early detection and diagnosis of lung cancer. 

We spoke with Shuvayu Sen, Ph.D., vice president, oncology value and implementation outcomes research, about the importance of using RWE.

What is RWE and why does it matter? 

Sen: RWE is generated through the analysis of real-world data or health information routinely collected from sources such as electronic health records (EHR), registries and insurance claims. Alongside data from clinical trials, real-world data matters because it may provide contextual insights that are not possible in a controlled setting.

How is your team using RWE in lung cancer research?

Sen: Our applicable areas of research include continuing to address smoking as the leading risk factor for lung cancer while identifying additional contributing risk factors — pinpointing moments to engage at-risk individuals and building explainable risk prediction models, including for non-small cell lung cancer. Our research on the patient journey can help show where delays in care may occur, such as low screening uptake, missed follow-ups on imaging or coordination gaps between care teams.

What have you learned from RWE in lung cancer?

Sen (left) with colleague Melissa Santorelli, Ph.D., MPH, at our global headquarters.

Sen: As part of our analysis of one institution’s EHR database, we identified underutilization of low-dose computed tomography (CT) scans as an unmet need in the diagnostic pathway. Our research also showed potential for electronic medical record data to help identify patients who may be at risk of developing lung cancer. Looking ahead, we aim to explore options that could support earlier detection by leveraging this data. These insights point to potentially meaningful opportunities across the oncology ecosystem and beyond.

How else are we helping to advance research in this space?

Sen: We believe it’s critical to advance this work through research outside our company, as shared insights and investigator-led research are equally essential to accelerating innovation. That’s why we expanded our MSD Investigator Studies Program (MISP) to support independent research.

The MISP program evaluates tools and methods for lung cancer risk assessment and explores new technologies, like artificial intelligence (AI) and digital diagnostics, with the goal of improving early detection and diagnosis of lung cancer.

Together, our real-world evidence and MISP-supported research have the potential to reshape how and when lung cancer may be detected.

Learn more about Mission Lung Cancer.

Innovation

Our AI model KERMT is helping to advance drug discovery

Our scientists harness AI and machine learning in small molecule lead optimization

March 23, 2026

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In the lead optimization phase, scientists fine-tune early molecules in the hopes of finding a molecule that might one day become a medicine.

Traditionally, this stage takes months, and most drug candidates never make it to clinical testing. But advancements in artificial intelligence and machine learning (AI/ML), including our new AI foundation model KERMT, could help change that.

What is KERMT and how is it transforming small molecule research?

Developed in collaboration with Nvidia, KERMT, pronounced “Kermit” and short for Kinetic GROVER Multi-Task, is a deep-learning computer model trained on more than 11 million molecules. It learns from patterns in vast amounts of chemical data with the goal of helping scientists better predict how a molecule will behave in the body, potentially spotting issues much earlier and reducing the need for months of lab work.

KERMT isn’t just helping our researchers; as an open-source model, its code is available to the whole scientific community.

How AI models can impact drug development timelines

In a recent interview, Senior Director of Data Science Alan Cheng said AI is already “speeding up the early stages of drug development dramatically.”

“AI is sometimes cutting timelines by 30% or more, improving drug candidate quality and reducing costs,” Cheng said. “This is a very meaningful acceleration. While clinical trials remain lengthy, our models are enabling faster identification of disease targets and optimized compounds, which should increase probability of success and shorten preclinical phases.”

Open-source AI for the scientific community

AI/ML is evolving at an incredible pace: access to relevant data is growing, computing power is expanding and deep-learning algorithms are rapidly improving. Advances like KERMT can give teams a powerful new way to make better informed decisions and focus their time on the most promising drug candidates.

These changes have the potential to create unprecedented opportunities to speed and strengthen the discovery of new drugs — with the goal of bringing safer, more effective medicines to patients faster.

KERMT is available on Nvidia accelerated computing and software, including platforms like Nvidia BioNeMo and Clara Open Models.

Watch to learn more about how MSD is using AI/ML for small molecule lead optimization 

Innovation

How wearable technology powers patient-focused drug development

Our scientists are exploring the use of sensor-based technologies and digital clinical measures to improve disease understanding

February 10, 2026

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Sensor-based digital technologies like smartwatches and other wearables have surged in popularity in recent years. People are easily and conveniently tracking physical activity, sleep and other health-related data — including information that’s helpful for scientists developing new medicines.

At MSD, scientists in our digital clinical measures group are using these sensor-based tools in clinical trials to collect objective measurements which were previously difficult or impossible to obtain. Now, measurements from patients outside the clinic, including at home and work, can provide data that’s more reflective of their everyday lives — deepening our understanding of disease and enabling more efficient and patient-centric drug development.

What are digital clinical measures, and why do we use them?

Digital clinical measures are specific, objective measures of biology, health, behavior or treatment response that are generated via sensor signals from digital technologies processed with algorithms. These measures can be derived from data collected during active task-based assessments, such as timed walk or hand-turning tests performed with wearable sensors, or through passive monitoring, where data are captured continuously as part of everyday activities like walking or sleeping.

Unlike some traditional clinical study endpoints that require lengthy in-clinic exams or patients or caregivers to remember symptoms over days or weeks, sensor-based technologies can objectively and remotely track metrics of health, behavior and treatment response over time. They can also provide more precise measures compared to traditional clinical rating scales.

Marissa Dockendorf, Ph.D.

“Digital clinical measures can augment traditional study endpoints and allow us to collect richer, more frequent data that better reflect how patients live and function day to day.”

  • Marissa Dockendorf, Ph.D.
    Head of digital clinical measures

“In addition to using digital health technologies — or DHTs — to enhance the data we capture in clinical trials, we’re focused on developing more objective and precise measures from these technologies,” added Dockendorf. “These advancements can enable us to understand more quickly, or with fewer clinical trial participants, whether our drug candidates are working, which ultimately can support our ability to deliver medicines to patients faster.”

Collaborating to advance the field of digital measures

We’re working with partners including the Critical Path for Parkinson’s Consortia, the Digital Medicine Society, the University of Oxford and Koneksa Health to advance development of digital clinical measures. These collaborations focus on furthering the digital endpoint field as well as identifying promising digital measures that may improve how we assess disease progression in patients with Parkinson’s disease and, potentially, how we evaluate the efficacy of investigational therapies.

“Digital endpoints hold tremendous promise to transform how we measure and understand health in clinical research,” said Dockendorf. “To fully realize that promise, collaboration is essential as we lay the important groundwork needed to develop measures that are valid, reliable and capable of making a meaningful impact in drug development.”

Digital clinical measures in action in Parkinson's disease

Our researchers are exploring the use of digital health technologies to measure motor function in clinical trials for Parkinson’s disease. Wearable sensor arrays — devices equipped with multiple sensors worn on the body to capture comprehensive data — can provide a wide range of motor function measures, such as gait and turn speed. Collecting data from these technologies over time may provide a clearer understanding of how motor function changes over time and with treatment as compared to traditional endpoints based on categorical rating scales.

Innovation

Our Q4 and full-year 2025 financial results

February 3, 2026

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MSD’s Q4 and full-year 2025 sales performance reflects strength across oncology and animal health, as well as increasing contributions from new launches. Our company announced Q4 worldwide sales of $16.4 billion. Full-year 2025 worldwide sales were $65.0 billion.

“In 2025, we continued to advance leading-edge science to deliver transformative medicines and vaccines that are improving health outcomes for patients around the world,” said Rob Davis, chairman and CEO. “Our business benefited from demand for our innovative portfolio, including for KEYTRUDA, increasing contributions from new launches in cardiometabolic and respiratory as well as vaccines, and strong performance of Animal Health. The transformation of our portfolio, bolstered by the acquisitions of Verona Pharma and Cidara Therapeutics, is well underway, and momentum is building as we continue to execute on our strategy. Our progress positions us to continue delivering on our purpose for patients and creating durable value for shareholders.”

MSD anticipates full-year 2026 worldwide sales to be between $65.5 billion and $67.0 billion.

Find more details on Q4 and full-year 2025 results below.

4Q and full year 2025 financial highlights for MSD

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Innovation

5 ways we’re transforming artificial intelligence into impact

We’re applying AI across our company to help us work smarter and faster so we can reach patients sooner

January 9, 2026

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At MSD, we’re in the business of knowledge, insights and innovation — rooted in intelligence.

Today, artificial intelligence (AI) — or what could also be “automated”, “accelerated” or “augmented” intelligence — lies not only in software and computer systems, but in the data, development and delivery of these intelligent tools to achieve better outcomes for patients.

Here are five ways we’re using AI to drive our purpose of saving and improving lives around the world.

01.

Accelerating the discovery of new medicines

Drug discovery remains an endeavor where only about 1 in 10 drug candidates that enter clinical trials ultimately receive regulatory approval. We’re working to change that by enabling scientists to use AI and machine learning (ML) foundation models to enhance and build upon their existing approaches to drug design before experimental testing and clinical trials.

We recently developed two foundation models which uncover patterns in disease to find better drug targets, allow faster molecular design and rapidly test small molecules, including cyclic peptides, for efficacy and toxicity before going into the clinic.

By unlocking patterns within vast datasets, these AI models enable our scientists to accelerate the discovery of leading therapeutic candidates —  a process that normally takes 10 years — allowing us to potentially get therapies to patients faster without compromising scientific rigor.

bonds-science

02.

Optimizing clinical trials

Enrolling people in clinical trials and keeping them engaged once they’ve signed up remains a significant challenge across our industry, with approximately 20% of activated sites failing to enroll a single participant. We’re addressing this by using AI to help improve site selection, patient matching and retention. For example, predictive models can flag patients at higher risk of dropping out, enabling targeted interventions that improve retention and keep trials on track.

diverse crowd animation

03.

Automating workflows to improve productivity

Our enterprise-wide training program helps employees understand the latest digital technology, including generative and agentic AI, and learn how to use it responsibly. Our proprietary AI platform — which more than 80% of our workforce uses — applies large language models to enable employees to automate, simplify and digitize processes that historically took more time, freeing us up to prioritize more impactful work.

Illustration of people looking at workflow chart

04.

Modernizing manufacturing

Generative AI helps protect our supply chain when potentially disruptive events like natural disasters or port delays occur. Our systems can produce event-based risk assessments in under 30 minutes — allowing us to quickly see which products and sites are affected and act to avoid or reduce shortages and delays.

In vaccine manufacturing, we’re using computer vision — another form of AI — to inspect vials and syringes for defects. This results in less waste, lower costs and higher production speed.

teal boxes in workflow

05.

Streamlining education and engagement with health care providers

We’re using AI to streamline information for providers and patients to ensure we deliver the right details to the right people when it matters most.

We’ve embedded AI across the content life cycle — from conception through medical, legal and regulatory review — so that we can organize messages more intelligently. The result: higher quality, personalized content that gets to health care providers faster.

Supporting this is our generative AI-powered chatbot for our field representatives. It summarizes relevant insights and helps us respond in real time to provider needs.

computer screen of data

It all starts with data

Data powers AI. We have a vast repository of proprietary and secure data, but for it to be usable, it must first be structured and organized.

We’re continuously working to create a frictionless data flow so AI can reliably and accurately drive faster, more targeted and personalized outcomes.

Data is critical to our business strategy and to our pipeline. When our data is high-quality, well-manicured and organized to support powerful insights, we can make more accurate and intelligent predictions — and move faster to deliver the medicines and vaccines patients are waiting for.

Read more about how we’re using data science, AI and machine learning.

Innovation

Exploring the potential use of PET tracers in neurodegenerative disease research

MSD scientists are researching the potential of an investigational alpha-synuclein PET tracer in early identification of Parkinson’s disease

December 9, 2025

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Parkinson’s disease (PD) is a progressive neurodegenerative disease caused by the loss of certain brain cells that are involved with the control of movement. Globally, the prevalence of Parkinson’s has doubled in the past 25 years with global estimates in 2019 showing over 8.5 million individuals living with PD. Most patients with Parkinson’s are diagnosed after experiencing worsening symptoms such as tremors, slow movements and gait abnormalitie

Unfortunately, by the time symptoms become apparent, a large proportion of affected brain cells have already been irreversibly damaged, making therapeutic intervention difficult. Researchers at MSD, along with the broader scientific community, are focused on exploring new ways to identify people with PD earlier in the course of their disease and to measure changes in disease progression.

Alpha-synuclein: A biomarker for Parkinson’s disease

Research evaluating the brains of people with PD has shown that abnormal clumps of a protein called alpha-synuclein form as the disease progresses. Historically, the only way to evaluate the accumulation of alpha-synuclein is by analyzing brain tissue collected after a person dies.

Our scientists then asked: What if there was a way to detect the first signs of these abnormal protein deposits in the brain as a potential way to identify the early signs of PD before symptoms appear? Could we then also evaluate the potential of investigational therapies to slow disease progression?

Exploring noninvasive early detection for neurodegenerative disease

Advanced imaging technologies have helped evolve our understanding of neurodegenerative diseases, in some instances leading to a better understanding of the disease and potential therapeutic options.

In PD, similar imaging techniques offer the potential of early detection and the ability to monitor disease progression. A PET tracer may offer a noninvasive method of detecting alpha-synuclein.

Our scientists are working to evaluate the potential of a PET tracer as a noninvasive way to visualize and measure alpha-synuclein, with the hope that it could lead to earlier detections of Parkinson’s disease.

“A selective PET tracer for alpha-synuclein could have a significant impact on ongoing research by providing a window into what’s happening in the brain in real time,” said Dr. Eric D. Hostetler, executive director and head of translational imaging biomarkers at MSD. “We see the potential for an advanced imaging tool that could contribute to the development of novel treatments by potentially making it easier and more effective to test whether an investigational therapy that targets alpha-synuclein in the brain is working in clinical trials.”

Our research in this area was recognized by the Michael J. Fox Foundation for Parkinson’s Research Ken Griffin Alpha-synuclein Imaging Competition in 2023, and our scientists are continuing important research in multiple investigational PET tracers in the central nervous system space.

“We’re evaluating the potential of this investigational PET tracer to serve as a biomarker to measure disease progression and determine whether investigational therapies are able to slow the progression of Parkinson’s disease,” said Dr. Jason Uslaner, vice president and head of neuroscience discovery at MSD. “It’s all about identifying the right patient at the right time for the right medicine.”

Learn more about our research in neuroscience.

Innovation

Building trust and advancing inclusion: Our approach to clinical trials in Uganda

In Uganda, where women are disproportionately impacted by HIV, our community-based research prioritizes meeting patients where they are

November 24, 2025

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Rebeca Plank, M.D., Ph.D. (right) and Joan Nabawanuka, community liaison officer at MU-JHU Care Ltd., meet with Kibuuka Yahaya Galabuzi, local leader of Katiko Fishing Village in the Mityana district

In sub-Saharan Africa, young women are three times more likely than young men to be living with HIV. Now, MSD researchers are part of a coalition of community leaders, academic organizations and NGOs working in Uganda to help ensure that clinical trials for investigational HIV medicines are designed to address this disparity — and to reflect real-world challenges in local communities. By focusing on local voices and expertise, the coalition aims to build trust, improve trial design, address barriers to study participation and engage directly with the community.

To learn more about how these efforts have shaped our HIV clinical trials in Uganda, we chatted with two members of our on-the-ground research team: Ian Bradley-Perrin, Ph.D., and Rebeca Plank, M.D., MPH.

Why is inclusion important in HIV clinical trials?

Plank: People may respond differently to the same medicine based on factors like age, gender, weight or ethnicity. By prioritizing inclusivity in HIV clinical trials, our goal is to develop treatment and prevention options that work for all people affected by HIV.

SEE ALSO: Dr. Plank shares her motivations and hopes for the future of HIV research

Why is it important to include women in HIV clinical research, especially in countries like Uganda?

Bradley-Perrin: Cultural expectations, gender roles and the still-too-common stigma surrounding HIV and sexual health can make it difficult for women in Uganda to take part in HIV prevention trials. Historically, women’s underrepresentation in these prevention clinical trials limited our field’s understanding of how these medicines work specifically for them.

Plank: We’ve made an effort to expand our HIV prevention clinical trials to include pregnant and breastfeeding women because there are limited late-stage data and safety information in those populations. So, it’s important to move in that direction of greater inclusivity in our trials with the goal of driving the research and development of investigational medications to make sure there are options women can access regardless of their plans for pregnancy. Today, many health care providers may be reluctant to prescribe medicines that have not been studied in pregnant and/or lactating women, and pregnant and/or lactating women may be reluctant to take these medicines.

What’s the role of local communities in HIV prevention clinical trials?

Bradley-Perrin: We’ve learned that strong collaboration is essential — and it’s been a real two-way learning process. Partners like the University of Washington’s International Clinical Research Center bring long standing experience running clinical trials in sub-Saharan Africa, and the Gates Foundation is providing strategic partnership and financial support for the operational needs of each clinical research site, including things like experienced research staff, upgraded facilities, and help building new clinics. Equally important is the Global Community Advisory Group: HIV prevention advocates with a country and global perspective who give critical input on trial design, recruitment materials and will advise on study operations throughout the trial. They give us honest feedback about community concerns and help keep us accountable and connected to people’s real lives and priorities.

For example, in Mityana, a rural district west of Kampala, our team collaborated with community leaders to support a local engagement strategy that helped identify locations where women have historically had trouble accessing HIV clinical trials, like fishing villages and gold mining communities.

Plank: By going out to a gold mine that was two hours from the clinic, I better understood the complexities of reaching this community, especially over the duration of a multi-year clinical trial. Transportation was a huge barrier that needed to be addressed to make sure women wouldn’t be hindered from participating because of where they lived or their access to transportation.

What are some other ways MSD is supporting clinical trial participants?

Bradley-Perrin: Along with transportation, we prioritized support services such as child care and, when needed, home visits to help women participate in the trials.

Plank: Thanks to the Gates Foundation, ICRC and our other partners on the ground, the clinical trial team has trained local staff, updated facilities and even helped to construct new research clinics that could be used for new research projects. These practical solutions aim to increase trial participation in geographies that need innovations the most and help address some of the practical challenges participants face.

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  • Plank and Bradley-Perrin walk with Flavia Matovu Kiweewa, MBChB, Ph.D., principal investigator of MU-JHU Care Ltd.
  • Cyrus Badshah, M.D., Ph.D, MSD senior principal scientist and Joyce Matovu, site hospital liaison coordinator at MU-JHU Care Ltd., tour the Namulanda Mukikadde gold mine community in the Mityana district.
  • A view of Namulanda Mukikadde gold mine area in the Mityana district of Uganda, where innovative clinical trials for HIV are taking place.
  • Godfrey Ssaka, laboratory manager at MU-JHU Care Ltd., shows Susan Vaz, a clinical research manager at our company, around the lab.
  • MSD’s community-based research in Uganda prioritizes fostering trust and creating conditions to encourage inclusive trial participation.
Previous
Next
  • Plank and Bradley-Perrin walk with Flavia Matovu Kiweewa, MBChB, Ph.D., principal investigator of MU-JHU Care Ltd.
  • Cyrus Badshah, M.D., Ph.D, MSD senior principal scientist and Joyce Matovu, site hospital liaison coordinator at MU-JHU Care Ltd., tour the Namulanda Mukikadde gold mine community in the Mityana district.
  • A view of Namulanda Mukikadde gold mine area in the Mityana district of Uganda, where innovative clinical trials for HIV are taking place.
  • Godfrey Ssaka, laboratory manager at MU-JHU Care Ltd., shows Susan Vaz, a clinical research manager at our company, around the lab.
  • MSD’s community-based research in Uganda prioritizes fostering trust and creating conditions to encourage inclusive trial participation.
Innovation

Our Q3 2025 financial results

October 30, 2025

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scientists in lab

MSD’s Q3 2025 sales performance reflects strength across oncology and animal health, as well as increasing contributions from new launches. Our company announced Q3 worldwide sales of $17.3 billion.​

“In the third quarter, we continued to execute on our strategy with important pipeline advancements, significant approvals and successful new product launches,” said Rob Davis, chairman and CEO. “We’re delivering value to patients and customers through our innovative portfolio of medicines and vaccines, and we’re securing our future by making important investments in our pipeline – including through compelling, strategic business development like our completed acquisition of Verona Pharma and expanded U.S. manufacturing and R&D spending. With each milestone we achieve, my conviction that we’re well-positioned to drive the next chapter of success for our company increases.”​

​MSD anticipates full-year 2025 worldwide sales to be between $64.5 billion and $65.0 billion.​

​Take a look at the infographic below for more details on Q3 2025 results.

Q3 2025 Earnings Infographic

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Innovation

Understanding vision-threatening retinal conditions: Diabetic macular edema and wet AMD

Our scientists are researching new ways to address vision-threatening retinal conditions like diabetic macular edema and wet age-related macular degeneration (wet AMD)

October 9, 2025

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Retinal conditions such as diabetic macular edema (DME) and neovascular (wet) age-related macular degeneration (AMD) threaten the eyesight of millions worldwide. While anti-VEGF treatments have helped to improve outcomes, a significant number of patients – up to 40% – fail to respond or only partially respond.

“A diagnosis of diabetic macular edema or wet age-related macular degeneration can profoundly affect a patient’s quality of life,” said Dr. David Guyer, founder, chief executive officer and president at EyeBio, a wholly-owned subsidiary of Merck & Co., Inc., Rahway, N.J., USA, “The fear of progressive vision loss looms large, and beyond the physical challenges, many patients carry the emotional burden of worrying about their declining eyesight.”

To find alternatives for these patients, our scientists are investigating novel therapeutic targets for certain retinal conditions.

Our research exploring the Wnt signaling pathway

Our eyes have a blood-retinal barrier that protects our delicate retinal tissue. When the barrier is compromised, fluid can leak into the macula – the central part of the retina responsible for sharp, detailed vision – and other areas of the eye, causing swelling that can lead to vision loss for people living with DME and wet AMD.

Research suggests that changes in the Wnt (pronounced “wint”) pathway are associated with the breakdown of this blood-retinal barrier in DME and wet AMD. MSD and EyeBio scientists are exploring the Wnt pathway as a potential approach to help improve the integrity of the blood-retinal barrier.

“We hear from retinal physicians about the demand for novel approaches to treat serious retinal conditions like diabetic macular edema and wet AMD.”

  • Dr. Tony Adamis
    Chief scientific officer, EyeBio, a wholly-owned subsidiary of Merck & Co., Inc., Rahway, N.J., USA
Illustration of healthy eye
Illustration of eye with DME
Illustration of eye with wet AMD

NOTE: These images are diagrammatic representations of the eye for illustrative purposes only.

What is diabetic macular edema (DME)?

Diabetic macular edema (DME) is serious eye condition that poses a risk to vision in people with diabetes:

  • DME is caused by excess fluid buildup in the retina and characterized by swelling and thickening of the macula due to fluid leakage from damaged blood vessels.
  • DME may be present in patients who are not experiencing visual symptoms, at times delaying a definitive diagnosis.

What is wet age macular degeneration (wet AMD)?

Wet AMD is the most frequent cause of vision loss in older adults, caused by the growth of abnormal blood vessels under the retina.

  • Current therapies are limited to slowing or reducing AMD-associated vision loss.

Through research, our scientists hope to improve vascular stability and reduce fluid leakage. We are working to potentially redefine the treatment of certain retinal conditions to help patients worldwide.

Learn more about our research and commitment to ophthalmic health.

Innovation

Macrocyclic peptides: a new research frontier

MSD scientists are exploring macrocyclic peptides, a new way to combine the properties of a biologic in a pill

September 18, 2025

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Macrocyclic peptides-

Small molecules, generally taken as pills, make up nearly 90% of medicines used today. It’s hard to think of a world without them. The use of small molecules has been critical in expanding the reach of and access to medicines around the world.

But it’s challenging for small molecules to impact the large featureless surfaces of protein-protein interactions, which govern a wide range of biological processes in our bodies.

primary article image

To target these interactions, scientists have turned to large molecule biologic therapies, like monoclonal antibodies, which — taken by infusion or injection — have been critical in advancing the treatment of many diseases, including some cancers and autoimmune disorders.

Over a decade ago, MSD scientists began investigating a way to create a new kind of medicine that would combine the potency and precise targeting of an antibody with the stability and ease of administration of a small molecule or pill.

“Macrocyclic peptides allow us to cast a wider net on the protein interactions we want to drug, providing a vast and untapped opportunity to access a wider range of targets and potentially new ways to treat different diseases,” said Dani Schultz, director of chemistry.

An intermediate-sized modality: not too big, not too small

Macrocyclic peptides, with their intermediate size — not too big, not too small — combine the properties of both small molecules and biologics. With their larger size and unique ring shape, macrocyclic peptides can tightly bind ample surface area to disrupt protein-protein interactions compared to traditional, linear-shaped peptide therapies. 

Macrocyclic Peptide Graphic
Size differences between small molecule, peptide and antibody

“The design and invention of macrocyclic peptides is notoriously complicated,” said David Thaisrivongs, director of chemistry.

David Thaisrivongs headshot

“Similarly, scaling production up for a macrocyclic peptide small molecule, with four to five times the size and complexity of a typical small molecule, represented a bold endeavor.”

  • David Thaisrivongs
    Director of chemistry, MSD

For our researchers, this work started by screening large libraries of cyclic peptides using messenger RNA display technology. This led to the identification of cyclic peptide leads that were optimized using 3-dimensional protein structure-based design and advanced computational techniques. Further molecular iterations and refinements improved the absorption, potency and stability of the first candidate.

“A diverse, interdisciplinary team of skilled and determined people from across our chemistry organization has dedicated substantial efforts to advancing this science,” said Thaisrivongs.

Macrocyclic peptides potentially open new possibilities in drug discovery 

Our ongoing macrocyclic peptide discovery efforts represent a new era in drug discovery which may one day allow us to treat diseases that have long evaded traditional small molecule approaches and help improve access to medicines typically administered via injection or intravenously. 

“Macrocyclic peptides are a new modality, and we’re still in the early stages of understanding their potential to impact disease and patient care,” said Schultz.

“There’s no playbook here, we’re innovating and developing new techniques on how to optimize and synthesize macrocyclic peptides — it’s really thrilling for me as a scientist because the potential is huge.”

  • Dani Schultz
    Director of chemistry, MSD
Dani Schultz