Innovation for global health

Our R&D efforts include discovering and developing novel medicines for our company’s flagship global health priorities, as well as other diseases that predominantly affect underserved patients in lower-income countries, while exploring new ways to treat COVID-19, the most urgent public health issue in the world today.

COVID-19

We are collaborating with Molecular Partners to develop ensovibep, a potential new treatment option for COVID-19 that targets the virus using proprietary DARPin technology to neutralize SARS-CoV-2.

The topline results of a Phase II study reported in early 2022 showed that a single intravenous dose of ensovibep reduced viral load through Day Eight, shortened symptom duration, reduced emergency room visits and/or hospitalizations related to COVID-19, and reduced deaths, compared to placebo. In separate studies, it maintained potent in-vitro pan-variant activity against all variants of concern identified so far, including Omicron. Novartis in-licensed ensovibep from Molecular Partners. We will accelerate manufacturing scale-up and plan to seek expedited regulatory authorizations globally.

Separately, we are working to discover a novel therapy targeting the main protease – an enzyme essential to viral replication across coronaviruses, including SARS-CoV-2. The goal is to develop a therapy that could treat many or all other forms of coronaviruses, potentially addressing future pandemics.

Malaria

Malaria continues to burden societies across the globe, particularly in Africa, and evidence suggests that cases may rise as a result of climate change. Novartis has taken a multipronged approach to fighting the disease, working with partners such as Medicines for Malaria Venture to research, develop and manufacture a portfolio of essential antimalarial drugs.

In 1999, we introduced our first malaria drug, a highly effective fixed dose of artemisinin-based combination therapy. In 2021, we crossed the 1 billion mark in antimalarial treatments delivered to patients worldwide (please see “Build trust with society” for more information).

For more than 20 years, the Novartis Institute for Tropical Diseases (NITD), part of NIBR, has been at the forefront in the search for novel medicines for malaria and other neglected diseases. Two Novartis compounds for uncomplicated and severe malaria, KAF156 (ganaplacide) and KAE609 (cipargamin), respectively, are in clinical trials in Africa and Asia. These compounds could potentially address resistance to current therapies as well as provide simplified therapeutic regimens. In 2021, a Phase IIb study of KAF156 and its partner medicine, lumefantrine, in adults and children with malaria reported positive results, supporting continued development of the combination treatment.

Neglected tropical diseases

NITD, with funding from the Wellcome Trust, is in the early stages of a discovery program aimed at finding first-in-class curative anti-parasitic therapies for Chagas disease, a serious condition affecting more than 6 million people mainly in Latin America. We are also conducting a Phase IV trial of our heart failure medicine Entresto in patients with chronic Chagas cardiomyopathy. Chagas disease is a tropical parasitic infection but can manifest in the long term as chronic heart failure or other complications, causing disability and even death.

With an estimated 50 000 to 90 000 new cases per year, visceral leishmaniasis is the most serious form of leishmaniasis, causing fever, weight loss, spleen and liver enlargement, and death if left untreated. In 2020, we announced a collaboration with the Drugs for Neglected Diseases initiative (DNDi) to jointly develop LXE408, a first-in-class inhibitor of the kinetoplastid proteasome, for the treatment of visceral leishmaniasis. Within the scope of the agreement, Novartis is responsible for Phase I studies, technical research and development activities, and regulatory interactions. Upon approval, we have committed to distributing the drug on an affordable basis worldwide to maximize access in endemic countries. DNDi will lead Phase II and III clinical development activities, with the first Phase II study scheduled to start in 2022 in India. Additional trials are planned in East Africa, which has the highest burden of visceral leishmaniasis.

Sickle cell disease

Sickle cell disease (SCD) is a hereditary blood condition that afflicts millions around the globe, particularly people of African descent, with sub-Saharan Africa bearing roughly 80% of the disease burden. Our commitment to addressing SCD includes a therapeutic pipeline as well as a holistic approach to diagnose, treat and manage the disease in sub-Saharan Africa.

While the genetic cause of SCD has been known for decades, only recently has medical science gained the tools to potentially mitigate the biological effects of the errant gene that causes the disease. We also are pursuing an ex vivo hematopoietic stem cell program for SCD, using CRISPR gene editing technology licensed from Intellia Therapeutics, and in 2021 we initiated patient dosing. Also in 2021, Novartis announced a pioneering collaboration with the Bill & Melinda Gates Foundation to support the discovery of a single-administration in vivo gene therapy to cure SCD, designed from the start to be practical for use in lower-resourced settings.