Novartis employs a diverse array of advanced technologies in its research and development efforts to address significant medical needs. The company utilizes established platforms such as chemistry and biotherapeutics, which focus on the discovery and optimization of low molecular weight synthetic molecules and biologics derived from natural life molecules, respectively. Additionally, Novartis is at the forefront of innovative approaches, including xRNA therapies that leverage ribonucleic acids to modify biological pathways, radioligand therapy that delivers targeted radiation to cancer cells, and gene and cell therapies aimed at treating genetic disorders by altering genetic makeup or restoring cellular functions.

Furthermore, Novartis emphasizes the importance of collaboration with independent researchers through Investigator Initiated Trials (IITs) and Investigator Initiated Research (IIRs). This commitment to ethical research allows the company to complement its own studies with valuable insights from third-party investigations, enhancing the understanding of the benefit-risk profiles of its therapies. By fostering a research ecosystem that includes patient engagement and insights, Novartis aims to improve patient outcomes and advance the practice of medicine, ultimately striving to create sustainable value for both stakeholders and society.

Switzerland

Novartis is reimagining medicine to improve and extend people’s lives. We deliver high-value medicines that alleviate society’s greatest disease burdens through technology leadership in R&D and novel access approaches. In our quest to find new medicines, we consistently rank among the world’s top companies investing in research and development. About 106,000 people of more than 140 nationalities work together to bring Novartis products to nearly 800 million people around the world.

Age-related diseases

Cancer

Cardiovascular Diseases

Central Nervous System (CNS)

Digestive System Diseases

Eye Diseases

Hematologic Diseases

Immune System Diseases

Infectious Diseases

Metabolic Diseases

Pathological Conditions

Regenerative medicine

Urogenital Diseases

Various

Novartis 

Glomerular diseases

IgA nephropathy

Kidney disease

Atrasentan is a potent and selective, small molecule inhibitor of the endothelin A receptor for the potential treatment of IgA Nephropathy (IgAN) and other proteinuric glomerular diseases.

Vanrafia® (atrasentan)

Blood cancer

Chronic myelomonocytic leukemia

Leukemia

SCEMBLIX

Fabhalta (iptacopan) is an oral, Factor B inhibitor of the alternative complement pathway1.
Discovered at Novartis, Fabhalta is currently in development for a range of rare diseases including IgAN, C3G, atypical hemolytic uremic syndrome (aHUS), immune complex membranoproliferative glomerulonephritis (IC-MPGN) and lupus nephritis (LN).
Fabhalta was approved by the FDA in December 2023 for the treatment of adults with the rare blood disorder paroxysmal nocturnal hemoglobinuria (PNH) and received a positive opinion from the CHMP of the EMA in March 2024

Fabhalta® (iptacopan) 

Breast cancer

Kisqali® (ribociclib) is a selective cyclin-dependent kinase inhibitor, a class of drugs that help slow the progression of cancer by inhibiting two proteins called cyclin-dependent kinase 4 and 6 (CDK4/6). These proteins, when over-activated, can enable cancer cells to grow and divide too quickly. Targeting CDK4/6 with enhanced precision may play a role in ensuring that cancer cells do not continue to replicate uncontrollably.

Kisqali® (ribociclib) + endocrine therapy (ET)

C3 Glomerulopathy

Fabhalta® (iptacopan)  

Chronic spontaneous urticaria

Remibrutinib is an investigational, highly selective, covalent, oral BTK inhibitor that blocks the BTK cascade and prevents the release of histamine that causes itchy hives (wheals) and swelling9-11. When remibrutinib is used alongside standard-dose antihistamines, it results in a “two-pronged approach” where two parts of the inflammatory pathway are targeted, with remibrutinib inhibiting histamine release and antihistamines inhibiting histamine receptors, reducing CSU symptoms12,13. In the pivotal Phase III studies, REMIX-1 and REMIX-2, remibrutinib met all primary endpoints in patients with CSU who remained symptomatic despite second-generation H1-antihistamine use1. Treatment with remibrutinib showed significant symptom improvement early, which was sustained up to Week 521. Remibrutinib has been shown to be well-tolerated, with a favorable safety profile up to 52 weeks, including balanced liver function tests versus placebo1. Most commonly (≥5%) observed AEs in the Phase III REMIX studies were respiratory tract infections (including COVID-19 and nasopharyngitis) and headache, all comparable with placebo1,14. If approved in CSU, remibrutinib would offer an effective oral option within the Novartis immunology portfolio, which currently includes Xolair® (omalizumab), the first and only injectable biologic indicated for CSU15. In the US, Novartis Pharmaceuticals Corporation and Genentech, a member of the Roche Group, work together to develop and co-promote Xolair. In addition to CSU, remibrutinib is being investigated in several other immune-mediated conditions and has the potential to be a pipeline in a product

Remibrutinib [BTK inhibitor]

B-cell malignancies

Follicular lymphoma

Marginal zone lymphoma

Tafasitamab-cxix is an Fc-modified monoclonal antibody that binds to CD19 antigen expressed on the surface of pre-B and mature B lymphocytes and on several B-cell malignancies, including diffuse large B-cell lymphoma (DLBCL).
Upon binding to CD19, tafasitamab-cxix mediates B-cell lysis through apoptosis and immune effector mechanisms, including antibody-dependent cellular cytotoxicity (ADCC) and antibody-dependent cellular phagocytosis (ADCP).
In studies conducted in vitro in DLBCL tumor cells, tafasitamab-cxix in combination with lenalidomide resulted in increased ADCC activity compared to tafasitamab-cxix or lenalidomide alone.
<a href="https://www.monjuvi.com/pi/monjuvi-pi.pdf">Source</a>

Tafasitamab (CD19) 

BION-1301 is a humanized IgG4 monoclonal antibody that blocks APRIL binding to its receptors with no adverse effects reported in NHP tox studies of IV BION-1301 for up to 6 months and SC for up to 1 month.
APRIL is a TNF-family cytokine involved in B-cell signaling. It drives IgA production and survival of IgA-secreting plasma cells. Higher APRIL levels in IgAN patients is correlated with higher Gd-IgA1 and proteinuria and lower eGFR3.&nbsp;
<a href="https://investors.chinooktx.com/static-files/9a4d8ed0-97b0-46a2-9c9e-14f288e17c9c">Source</a>

BION-1301 (Zigakibart)

Gastroenteropancreatic neuroendocrine tumors

Lutathera® (INN: lutetium (177Lu) oxodotreotide / USAN: lutetium Lu 177 dotatate) is approved in the US for the treatment of adult patients with SSTR-positive GEP-NETs, including those in the foregut, midgut and hindgut, an indication which includes the NETTER-2 population. Lutathera is also approved in Europe for unresectable or metastatic, progressive, well-differentiated (G1 and G2), SSTR-positive GEP-NETs in adults7,8, and in Japan for SSTR-positive NETs.

Lutathera®

Atherosclerotic cardiovascular disease

Leqvio is the first and only small interfering RNA (siRNA) therapy to lower LDL-C. It is a subcutaneous injection given by a healthcare provider (HCP) with an initial dose, another at three months, and then every six months. As a twice-yearly, HCP-administered treatment, Leqvio may help to circumvent the challenges of treatment adherence, a common issue in cholesterol management.

Leqvio (inclisiran)

Paroxysmal nocturnal hemoglobinuria

Intrathecal onasemnogene abeparvovec (OAV101 IT) is an investigational, one-time gene therapy for patients with spinal muscular atrophy (SMA)

Intrathecal onasemnogene abeparvovec (OAV101 IT)

Atrasentan

Malaria

Viral infection

Coartem® (artemether- lumefantrine)

Primary hyperoxaluria

CHK-336 is a novel, potent and selective small molecule lactate dehydrogenase A (LDHA) inhibitor with the potential for once-daily oral dosing. CHK-336 has an engineered liver-targeted tissue distribution profile, which targets its activity to the primary site of oxalate production and minimizes extra-hepatic LDH inhibition. Preclinical studies have demonstrated that CHK-336 produced significant and dose-dependent reductions in urinary oxalate in a primary hyperoxaluria type 1 (PH1) mouse model into the range observed in wild-type mice.

Novartis

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