Novartis in Society Integrated Report 2022

Deliver high-value medicines

Millions of people around the world live with serious diseases that cause early death and disability and place an enormous burden on healthcare systems. At Novartis, we are dedicated to discovering and developing new treatments for diseases including cancer, heart disease and neurological conditions – and delivering them at scale to reach as many patients as possible.

2022 highlights

10.0 bn

Invested in R&D (USD)
comprising approximately 19.8% of our net sales


Ongoing Phase III programs
in our development pipeline


in the US, the EU, Japan and China for new treatments as well as new indications for existing treatments


in the US, the EU, Japan and China

Van Lacour, a patient with advanced prostate cancer (Photo)
Van Lacour received a Novartis radioligand therapy, Pluvicto, for advanced prostate cancer as part of a managed access program in 2021. Today, he is living a fulfilling life in his hometown of Natchez, Louisiana, in the US, where he enjoys taking photographs and listening to his collection of old records.

Delivering new medicines is at the core of our purpose and value creation as a company: we are in the business of improving and extending people’s lives and helping governments shape healthier societies.

Improving human health is more critical than ever. Globally, people live for an average of 10 years with a disease or disability. Chronic conditions make up an increasing share of the disease burden as populations age, with cardiovascular disease and cancer together responsible for 28 million deaths annually.

We use a variety of scientific tools to meet this challenge. Medical science is advancing at an unprecedented rate and new types of treatments are coming to the fore, including RNA therapies, gene and cell therapies, and radioligand therapies that offer targeted approaches to treating serious diseases.

In 2022, Novartis continued to deliver high-value medicines to patients. We received 23 approvals in the US, EU, China and Japan, including a novel radioligand therapy for advanced prostate cancer in the US and EU. We advanced our focused pipeline of investigational medicines, with several clinical data readouts paving the way for further launches in 2023 and beyond.

However, we also had disappointments as some clinical trials of investigational compounds – including ACZ885 (canakinumab) for lung cancer and UNR844 for presbyopia – did not meet their primary goals.

Deliver high-value medicines performance indicators1





Projects entering development pipeline 2




Ongoing Phase III programs 3




US FDA breakthrough therapy designations 4




Major submissions (US, EU, JP, China) 5




Major approvals (US, EU, JP, China) 5




New molecular entity (NME) approvals 6





Includes Innovative Medicines and Sandoz biosimilars only


Includes projects having achieved first patient, first visit (FPFV) in confirmatory development (incl. projects entering confirmatory development from an acquisition or in-licensing). Figures shown for the year 2021 and 2020 consider projects applying the former methodology, i.e. projects entering confirmatory development from internal R&D activities. FPFV has occurred in post-proof-of-concept stage after NIBR.


Includes projects with FPFV in a Phase III study but not yet filed in the US, EU, Japan or China


Number of breakthrough therapy designations granted by the US Food and Drug Administration for therapies under development by Novartis


Includes small molecules or biologics; new fixed-dose combinations of existing APIs; and new target indications, defined as new disease or new line of treatment (e.g., first line vs. second line)


Includes NMEs such as small molecules, biologics; in the EU, new fixed-dose combinations of existing APIs

Bringing our medicines to patients

We focus on high-value innovative medicines with the potential to transform the treatment of diseases across our five core therapeutic areas where we have a leadership position and where there are high unmet patient needs.

To do this, we seek to maximize the value of our key in-market and launch medicines, while investing in R&D over the longer term to deliver the next generation of therapies for patients. We follow an integrated, end-to-end approach across the drug development continuum – from early research through development, launch and patient access – with close collaboration between our R&D, manufacturing and commercial teams to broaden the reach and impact of our medicines.

We also focus our activities on key markets that represent the majority of the expected growth in healthcare spending over the next several years. Foremost among these is the US, the world’s largest healthcare market, where we aim to be a top-five player by 2027.

Across our key products and markets, we pursue effective life-cycle management and launch excellence to maximize the commercial potential of our medicines and broaden access for patients in line with the Novartis Access Principles (see "Build trust with society"). This involves investing earlier in pre-launch preparation and working systematically to meet the needs of healthcare professionals and patients, including by reducing time to diagnosis and working with healthcare systems to find solutions that improve the health of entire populations.

In each of our core therapeutic areas, we have key products that together we expect to contribute more than two-thirds of our growth in the next five years.



Key products: Entresto, Leqvio

Cardiovascular disease (CVD), which includes chronic conditions affecting the heart and blood vessels such as heart failure, heart attacks and strokes, is the world’s leading cause of death and one of society’s biggest health concerns. More people die every year from heart attacks and strokes than died from COVID-19 during the recent pandemic. Many of these deaths are preventable through management of risk factors including high cholesterol. The total global cost of CVD is set to rise to more than USD 1 trillion by 2030.

Our cardiovascular portfolio comprises therapies to treat heart failure and reduce low-density lipoprotein (LDL) cholesterol (also known as “bad cholesterol”), a key contributor to the build-up of fat deposits in arteries (atherosclerosis). We are also working systematically to meet the needs of health systems and patients through innovative access approaches, such as population health agreements, that work in tandem with our medicines to reduce the impact of CVD equitably and at scale.

Our cholesterol-lowering treatment Leqvio has now been approved for use in 70 countries worldwide. Leqvio is a subcutaneous injection given by a healthcare provider every six months (after an initial dose and one at three months). In conjunction with a healthy diet and statins, Leqvio may help those who have difficulty sticking to medicines that are self-administered and have greater dosing frequency.

The launch of Leqvio in the US and other markets is ongoing, with a focus on patient on-boarding, removing access hurdles and enhancing medical education. In the US, we continue to see steady progress in expanding access to Leqvio. After securing coverage from key payers, the medicine is covered at or near label for 76% of patients around one year after launch. In addition, 67% of patients pay out-of-pocket expense of less than USD 10.

We are advancing our partnership with the National Health Service (NHS) in England focused on people who have had a cardiovascular event and have high LDL cholesterol. The aim is to help doctors to identify and treat 300 000 people over three years. In 2022, as part of our collaboration, we secured access for Leqvio in nearly all local care formularies, developed tools to more easily identify patients not being optimized, and helped the NHS improve overall cholesterol management in primary care.

We also continue to strengthen the position of Entresto, our medicine for heart failure and hypertension, which we estimate to be treating approximately 10 million patients worldwide. With the update of the 2022 guidelines issued by the American Heart Association, American College of Cardiology and Heart Failure Society of America, Entresto is now included as a treatment option for the majority of patients with heart failure and is recommended as a preferred drug in its class for the treatment of heart failure with reduced ejection fraction, a form of heart failure with high morbidity and mortality.

Addressing the factors behind heart disease in the US

Almost a quarter of deaths in the US are due to cardiovascular disease. Social factors such as living conditions, education and diet play a key role in determining a person’s heart health. These risk factors are exacerbated by racial inequity: according to the American Heart Association, heart disease accounts for nearly 40% of the disparity in life expectancy between Black and white Americans.

Our three-year Closing the Gap initiative with Thomas Jefferson University and Jefferson Health focuses on addressing the social determinants of heart health in five vulnerable neighborhoods in Philadelphia with high rates of stroke and adverse outcomes related to heart disease. The aim is to reduce the impact of undertreated and untreated heart disease risk factors by creating systems of change in these communities that enable people to live healthier lives and experience better connections to health systems.

Closing the Gap enables us to take an upstream approach. Dedicated clinical personnel and community health workers conduct screenings and connect people with resources such as food, health, education or housing assistance. Assistance is also provided to help people reduce health risks related to diabetes and hypertension, and connect them to trusted sources of care.


CVD drives health disparities

According to the American Heart Association, heart disease accounts for nearly 40% of the disparity in life expectancy between Black and white Americans.



Key product: Cosentyx

Immunology covers a broad spectrum of conditions, including chronic skin diseases such as psoriasis. Immunological diseases affect an estimated 4.5% of the world’s population, and their prevalence is increasing.

In 2022, we saw continued growth for Cosentyx, our treatment for several systemic inflammatory conditions, due to approvals for new indications in key markets and initiatives to expand patient access. Since initial approval in 2015, Cosentyx has treated more than 960 000 patients worldwide.

In the EU, Cosentyx received approval for active enthesitis-related arthritis (ERA) and active juvenile psoriatic arthritis (JPsA) in children aged six years and over, following US approval in 2021. ERA and JPsA are subtypes of juvenile idiopathic arthritis that, if left untreated, can lead to high levels of pain and disability.

In China, Cosentyx maintained a strong growth trajectory as we continued to expand hospital access for continued patient uptake. Based on internal Novartis estimates, Cosentyx is now available in approximately 1 900 hospitals in China and has been used to treat around 250 000 psoriasis and ankylosing spondylitis patients since approval in 2019.

We also reported Phase III results for Cosentyx in hidradenitis suppurativa (HS), a painful and recurrent skin disease that affects around one in 100 people. The data showed HS patients treated with Cosentyx experienced rapid and long-lasting symptom relief, with favorable safety consistent with the medicine’s well-established profile. Data from these trials has been submitted to regulatory authorities in Europe and the US and other countries, with the goal of bringing Cosentyx as a new treatment option to patients as soon as possible.



Key products: Kesimpta, Zolgensma

Around 2 million people worldwide suffer from multiple sclerosis (MS), an autoimmune disease that is the most common cause of neurological disability unrelated to trauma in people under 40 years of age.

Kesimpta, our treatment for relapsing forms of MS, which has been shown to reduce the risk of worsening disability from MS for up to four years, has now been approved in 80 countries with more than 36 000 patients treated. Kesimpta has seen strong launch uptake in the US, Japan and Europe. In Europe, it saw additional launches in Italy and Spain during 2022, and reached 10 000 active patients by the end of the year.

New data in 2022 showed that earlier treatment with Kesimpta is more effective in limiting the progression and impact of MS compared with treatment at a later stage – helping to reduce the burden of the disease for patients and healthcare systems. Kesimpta is a once-monthly injectable treatment that can be taken from home, which helps broaden access for patients who have difficulty visiting healthcare facilities.

Zolgensma, a one-time, intravenous gene replacement therapy for spinal muscular atrophy (SMA), has now launched in most major markets. SMA is a rare genetic neuromuscular disease that results in the progressive and irreversible loss of motor neurons, which causes muscle weakness and atrophy. Zolgensma has been approved for use in 45 countries. In 2022, South Korea and Poland agreed to reimburse patients for the therapy. So far, more than 2 500 patients have used Zolgensma across clinical trials, access programs and in the commercial setting.

New data in 2022 also reinforced the transformational benefit of Zolgensma, demonstrating age-appropriate development when used pre-symptomatically, and maintenance of the ability to speak, swallow and meet nutritional needs when used symptomatically.


Solid Tumors

Key products: Kisqali, Pluvicto

Cancer is the second leading cause of death worldwide, and is a growing health issue in LMICs. The WHO estimates there will be 50% more new cancer cases in 2040 than there are today.

Breast cancer is one of the most prevalent types of cancer. Kisqali, our medicine for HR+/HER2- advanced breast cancer, the most common subtype of breast cancer, has now been approved for use in more than 95 countries and has been used to treat more than 60 000 patients. Kisqali, which inhibits two enzymes involved in the control of cell cycle progression known as CDK4 and CDK6, continues to show strong growth due to the increasing recognition of its impact on overall survival and quality of life benefits in the advanced breast cancer setting. In early 2023, it was approved in China for premenopausal and perimenopausal women with advanced breast cancer.

When faced with incurable disease, the hope for many patients is to live as long and as well as possible. In 2022, Kisqali data continued to demonstrate an overall survival benefit while maintaining or improving quality of life for people living with HR+/HER2- advanced breast cancer, which has now been observed across all three Phase III clinical trials. A Phase III study of Kisqali in early-stage breast cancer – the biggest-ever clinical study of a CDK4/6 inhibitor for this disease – is ongoing.

During the year, we also secured approval in the US, EU and other countries for Pluvicto, our radioligand therapy for progressive metastatic castration-resistant prostate cancer (mCRPC), an advanced form of prostate cancer that currently has few treatment options. Prostate cancer is among the most frequently diagnosed cancers in men and causes more than 30 000 deaths in the US every year. Since launch, we have been working to scale up production of Pluvicto to meet higher-than-expected demand.

In December, we announced positive early results from a second, pivotal Phase III study of Pluvicto in mCRPC patients prior to receiving a taxane-based chemotherapy. An additional Phase III trial in an earlier line of treatment for metastatic prostate cancer is ongoing, with a data readout anticipated in 2024.



Key product: Scemblix

Around 500 000 new cases of leukemia are recorded globally every year. Approximately 15% of all cases are chronic myeloid leukemia, or CML, most common in adults over 65. Significant advances in recent years have led to improved survival rates, enabling more patients to live with the disease. Even so, many patients remain at risk of disease progression, and are resistant to, or intolerant of, most available treatments.

Our CML treatment Scemblix is now approved for use in 40 countries

In 2022, our CML treatment Scemblix received approval for use in the EU and Japan following US approval in 2021. It is now approved for use in 40 countries. New data for Scemblix also showed long-term efficacy for patients with Philadelphia chromosome–positive CML in chronic phase. Scemblix is also being studied for possible use in patients newly diagnosed with chronic CML.

Working to eliminate malaria and neglected tropical diseases

UN Sustainable Development Goal 3 is about good health and well-being. At Novartis, we have been working for decades to find new treatments for some of the world’s biggest healthcare challenges - from noncommunicable diseases like cancer and cardiovascular disease to infectious diseases such as malaria.

One of the targets to support SDG 3 is to end the epidemics of malaria and neglected tropical diseases (NTDs). Every year, NTDs trap millions of people in a cycle of poverty and illness.

In June, we endorsed the Kigali Declaration on Neglected Tropical Diseases and pledged USD 250 million over five years (2021-2025) to advance new treatments for NTDs and malaria. This commitment includes the development of next-generation antimalarials, including an optimized formulation for infants, as well as further research into Chagas disease, dengue, leishmaniasis and cryptosporidiosis, a parasitic infection that causes life-threatening diarrhea in malnourished children.

UN SDG target 3

Target 3.3 reads: By 2030, end the epidemics of AIDS, tuberculosis, malaria and neglected tropical diseases and combat hepatitis, water-borne diseases and other communicable diseases.

Please see "Measuring our impact" for more information on our impact on the SDGs

Research and development

As with our commercial strategy, we have clear priorities in R&D to maximize the value of our pipeline of investigational medicines.

From the inception of a potential therapeutic through to clinical development and launch, our teams collaborate in support of our purpose to improve and extend people’s lives. Researchers at the Novartis Institutes for BioMedical Research (NIBR) work across several therapeutic areas to uncover biological insights into the origins and pathogenesis of disease to drive the discovery and development of the next generation of medicines. Our Global Drug Development (GDD) organization leads the advanced clinical development of potential new medicines, running large clinical trials and steering the way to regulatory approval and access for patients.

This work is powered by technology platforms that help us to discover, develop and commercialize new therapies across our therapeutic areas. We focus on two established platforms – chemistry and biotherapeutics – plus three advanced platforms – RNA, radioligand therapy, and gene and cell therapy – that offer targeted approaches to treating disease. We are investing to build our capabilities in these advanced platforms and expect them to play an increasingly important role in developing new medicines in the future.

Within this ‘two plus three’ approach, we focus our development resources on our priority assets to maximize high potential early programs and ensure flawless execution of late-stage pipeline programs, in addition to supporting lifecycle management by growing the evidence base for key in-market assets.

‘Two plus three’– our key technology platforms

‘Two plus three’– our key technology platforms (Graphic)

We continue to make significant investments to support our R&D strategy. In 2022, we invested USD 10.0 billion in R&D, compared with USD 9.5 billion in the prior year. Approximately 21 000 Novartis employees work in R&D – around one fifth of our total workforce.

Delivering high-value medicines requires a focus on productivity and prioritization to increase our returns on R&D investment – for example by moving quickly to expand projects with high potential and stop or out-license non-core programs. At the same time, we are using AI to optimize drug discovery and development, and reduce cycle times (see "Scale data science and technology").

While most of our research is conducted internally, we increase our chances of discovering new medicines by collaborating with outside researchers and biotech companies. Our network consists of more than 300 outside academics and 100 industry alliances working on joint research and drug discovery.

Significant pipeline advancements in 2022 include the approval of novel radioligand therapy Pluvicto, and further approvals and indication expansions for Scemblix, Kymriah and Cosentyx. We also presented several late-stage data readouts that pave the way for further launches in 2023 and beyond.

For example, our complement factor B inhibitor iptacopan (LNP023) met its primary endpoints in two Phase III studies in patients with paroxysmal nocturnal hemoglobinuria (PNH), a rare and deadly blood disorder. Global regulatory submissions for iptacopan for the treatment of PNH are anticipated to be filed in 2023. If approved, iptacopan has the potential to become the first oral monotherapy for PNH.

Iptacopan was discovered at NIBR through research targeting the immune system’s alternative complement pathway, of which complement factor B is a key part. When it's working properly, this pathway helps clear the body of pathogens and damaged cells. When it’s not, it can cause the destruction of critical cells in the body, leading to a variety of debilitating diseases..

Iptacopan is also being studied for other indications such as kidney diseases and other rare blood disorders. Its potential as an oral treatment for several complement-mediated diseases was recognized early, prompting the design of a streamlined, parallel development plan in multiple patient populations.

Creating new possibilities in cancer care

Cancer is a spectrum of diseases with diverse characteristics that share a common feature – abnormal cells that grow out of control. Novartis has developed a world-class portfolio of compounds and biotherapeutics that inhibit key pathways driving cell survival and growth in a number of cancers. While tremendous progress has been made, an urgent need for new and better treatments remains. We are pursuing technologies that hold the potential for more diverse, targeted therapeutics to treat additional forms of cancer.

Novartis scientists are working to make CAR-T therapy, a treatment for certain blood cancers that sits at the intersection of cell-, gene-, and immuno-based therapies, better and more effective. The technology, known as T-Charge, shortens the time it takes to genetically engineer T cells during the CAR-T manufacturing process, so they can be given back to patients faster. T-Charge also helps these cells persist in patients longer, with the goal of giving them a better chance to control cancer. We are studying T-Charge in clinical trials to gauge its potential efficacy.

Radioligand therapy uses radiation to destroy cancer cells in a more targeted way than conventional radiotherapy. By using special molecules that carry a therapeutic radioisotope and circulate throughout the bloodstream, this technology can target cancer cells anywhere in the body while limiting damage to surrounding cells. Radioligand therapy is used for certain neuroendocrine tumors and advanced stages of prostate cancer. Novartis scientists are working to apply it to other cancer types.

Finally, targeted protein degradation takes advantage of the proteasome, a cell’s internal machinery for eliminating unwanted proteins. With this technology, scientists will potentially be able remove proteins from the cell, rather than just blocking their activity – opening the way to develop targeted therapies for cancer-causing proteins that have long eluded drug hunters.


More cancer cases

The WHO estimates there will be 50% more new cancer cases in 2040 than there are today.

Innovation for global health

In 2022, we also advanced our efforts to develop novel medicines for diseases that predominantly affect underserved patients in LMICs. For example, we announced we will progress ganaplacide/lumefantrine (KAF156) into Phase III development for the treatment of patients with acute uncomplicated malaria.

Ganaplacide is a novel agent with a new mechanism of action and is active against malaria parasites that are partially resistant to existing antimalarials. Combined with a once-daily formulation of lumefantrine, this combination has the potential not only to clear malaria infection, but also to block the transmission of the malaria parasite.

The Phase III trial is planned to start in 2023 and will include clinical sites in sub-Saharan Africa. Ganaplacide/lumefantrine is being developed with support from the Medicines for Malaria Venture and their partners.

Progress against ESG targets Deliver high-value medicines



Invest USD 250m to advance R&D for NTDs and malaria (over 5 years from 2021-2025)

On track: progress in 2022 on R&D milestones for malaria and NTDs

Implement a global access strategy for all new medicines launched

All new launches in 2022 had a global access strategy.

Our approach to clinical trials

Clinical trials play a critical role in delivering high-value medicines: they help determine whether our investigational medicines are effective and safe before they are approved by regulatory authorities for general use in patients.

For every clinical trial our primary responsibility is to protect the safety, well-being and legal rights of all participants and ensure adherence to the highest ethical standards for clinical research. For more information, see our position on responsible clinical trials.

As part of the Novartis Commitment to Patients and Caregivers, we are committed to seeking patient input into the design of our clinical trials, helping us to develop medicines that address patient needs. We are also committed to registering new medicines in every country where patients have taken part in clinical trials and to publishing the results. See "Stakeholder engagement" for more information on patient engagement.

Clinical trials (phases I-V)

Clinical trials (phases I-V) (Graphic)

Diversity in clinical trials

Diverse representation in clinical trials helps us understand how patient groups who are most likely to be treated for a disease will respond to a medicine – ultimately improving the quality of care for every patient, while also helping to address health inequities in society. In the US, for example, only 8% of participants across all clinical trials in the country in 2020 identified as Black or African American, despite this group having one of the lowest life expectancies and higher mortality from diseases such as CVD.

We include diversity and inclusion principles in all Phase III studies with US participation. For example, we consider epidemiology variances for race, ethnicity and gender for target disease indications during feasibility planning and trial recruitment. We plan to extend this approach to all studies worldwide.

To increase recruitment of under-represented groups in our trials, we are working with patient groups and through community partnerships – including with churches in Atlanta and with our partners in Beacon of Hope (see "Build trust with society" for details of this program). In one example, we leveraged these relationships to help enroll patients in a Phase III trial of Entresto for heart failure in the US. As a result, 50% of patients enrolled from these sites and 22% of patients overall in the trial were from under-represented groups.

To increase recruitment of under-represented groups in our trials, we are working with patient groups and through community partnerships

In 2022, the FDA issued new guidance on diversity in clinical trials. In response, we are now updating our guidelines to incorporate the guidance into our priority programs from 2023.

Managed and post-trial access programs

Our managed access programs (MAPs) provide eligible patients – those with serious or life-threatening illnesses – with access to medicines not yet approved or available in their home countries. In 2022, we reviewed 7 730 MAP requests from physicians from 80 countries and across 55 compounds. We approved 94% of these. By the end of 2022, more than 9 700 patients were receiving treatment under Novartis MAPs.

We are the first pharmaceutical company to publish peer-reviewed research on our MAPs, spanning more than 30 000 patient requests from 110 countries over three years

We are the first pharmaceutical company to publish peer-reviewed research on our MAPs, spanning more than 30 000 patient requests from 110 countries over three years. Results showed an average approval rate of approximately 95%. Most programs (87%) took place in high-income countries, those with compassionate use regulations made available online (94%) and those with a high level of clinical trial activity (96%). This research is intended to inform discussions among policymakers, regulators and the industry on how to broaden access to patients through compassionate use.

Our post-trial access (PTA) policy, which applies to all confirmatory clinical trials and trials for serious and/or life-threatening conditions, ensures patients who have derived clinical benefit from an investigational treatment can continue to receive it, free of charge, until it is commercially available and accessible locally. Our PTA policy applies regardless of the severity of the disease, the availability of alternative therapies, or the location of the clinical trial. PTA plans were incorporated in all in-scope trials approved in 2022. See the Novartis Position on Post-Trial Access for more information.

Key assets in our R&D pipeline

The table below provides an update on select R&D programs across our core therapeutic areas as well as select programs linked to our global health priorities. Note that some assets are in development across multiple therapeutic areas. For information on our full R&D pipeline, please see the Novartis corporate website.

Product / compound name



Potential indication(s)

Current phase



RNA therapy

Lowers ‘bad cholesterol’ in conjunction with a healthy diet and statins. Approved in 70 countries worldwide. We are developing Leqvio for other potential indications.

Secondary prevention of cardiovascular events in patients with elevated levels of low-density lipoprotein cholesterol



Investigational medicine targeting part of the immune system involved in triggering inflammation and fighting infection. Phase III studies are underway into potential use as a treatment for kidney diseases. Also being studied for a rare blood disorder (please see “Hematology” below)

IgA nephropathy

C3 glomerulopathy


RNA therapy

Potential, first-of-its-kind investigational treatment for cardiovascular events in patients with elevated levels of lipoprotein(a), an inherited risk factor that cannot be effectively addressed by diet or other lifestyle changes

Secondary prevention of cardiovascular events in patients with elevated levels of lipoprotein(a)




Treatment for various autoimmune and inflammatory diseases. Approved by the EU in 2022 for use against active juvenile psoriatic arthritis and active enthesitis-related arthritis in children aged six years and over. Also in 2022, separate Phase III trial data showed positive results in treating HS.

Giant cell arteritis

Lupus nephritis



Potential multi-indication investigational treatment for a variety of autoimmune and chronic inflammatory diseases. Also being studied in multiple sclerosis (please see “Neuroscience” below)

Chronic spontaneous urticaria

Sjögren’s syndrome



Investigational therapy with unique, dual action being studied for treatment of autoimmune conditions. In 2022, we reported positive data from a Phase Il trial for Sjögren’s syndrome, a disease of the immune system affecting over two million people worldwide.

Autoimmune hepatitis

Lupus nephritis

Sjögren’s syndrome



Gene therapy

Investigational gene therapy for patients between 2 and 18 years of age with Type 2 spinal muscular atrophy (SMA). While disease progression is slower in patients with later-onset SMA, there are significant unmet needs. Potential to be first one-time treatment for this population

Spinal muscular atrophy (intrathecal formulation)



Potential multi-indication investigational treatment for variety of autoimmune and chronic inflammatory diseases (see also “Immunology” above).

Multiple sclerosis




Ongoing Phase III clinical trials for early-stage breast cancer as an adjuvant therapy

Hormone receptor-positive (HR+)/human epidermal growth factor receptor 2-negative (HER2-) early breast cancer (adjuvant)

lutetium (177Lu) vipivotide tetraxetan

Radioligand therapy

Approved in 2022 for treatment of a progressive, deadly form of prostate cancer known as mCRPC. Development is ongoing in several indications for certain other types of prostate cancer.

Metastatic castration-resistant prostate cancer, pre-taxane

Metastatic hormone-sensitive prostate cancer



Investigational oral therapy for KRAS-mutated tumors, which make up around 25% of all cancers. We reported positive data in 2022 from a Phase Ib study in advanced non-small cell lung cancer, showing that the compound is an anchor for combination strategies to improve outcomes for patients with specific KRAS-driven cancers

Non-small cell lung cancer



Potential first-in-class therapy in development for pancreatic cancer and other solid tumor types. Designed to disrupt a pathway known to play an important role in the most common type of pancreatic cancer, which afflicts nearly half a million people worldwide

Pancreatic cancer




Investigational oral medicine to prevent the destruction of red blood cells. Also being investigated for other blood disorders and complement-mediated kidney diseases. (please see “Cardiovascular” above)

Paroxysmal nocturnal hemoglobinuria



Gene therapy

Acquired in 2022 as part of acquisition of Gyroscope Therapeutics. Experimental one-time gene therapy for treatment of geographic atrophy, an advanced form of age-related macular degeneration affecting more than five million people worldwide

Geographic atrophy




Anti-malarial with novel mechanism of action to address threat of artemisinin resistance and potentially block disease transmission. In 2022, we announced our decision to move to Phase III trials.

Uncomplicated malaria



Entering Phase II trial for visceral leishmaniasis, a neglected tropical disease spread by sand flies that is typically fatal without treatment.

Visceral leishmaniasis

Phase I Phase II Phase III Phase IV

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