Rigel Pharmaceuticals is a biotechnology company focused on developing innovative therapies aimed at improving the lives of patients suffering from hematologic disorders and cancer. The company has a robust pipeline that includes several key products, such as TAVALISSE® (fostamatinib), which targets spleen tyrosine kinase (SYK) for the treatment of adult chronic immune thrombocytopenia. Additionally, Rigel is advancing REZLIDHIA® (olutasidenib), designed to target mutant isocitrate dehydrogenase-1 (mIDH1) in relapsed or refractory acute myeloid leukemia (AML), and GAVRETO® (pralsetinib), which targets rearranged during transfection (RET) in non-small cell lung cancer and advanced thyroid cancer. These therapies are at various stages of clinical development, with some already receiving regulatory approval.

In addition to its commercialized products, Rigel is actively engaged in clinical trials and investigator-sponsored research to further explore the efficacy of its therapies. The company is particularly interested in understanding the mechanisms of resistance to treatments and optimizing sequencing strategies for its drugs. Rigel's commitment to advancing scientific knowledge is reflected in its willingness to support independent research that aligns with its therapeutic areas of interest, ensuring that studies are rigorously assessed for scientific merit and safety. This approach not only enhances the understanding of their therapies but also contributes to the broader field of oncology and hematology.

United States

Rigel Pharmaceuticals, Inc., is a biotechnology company dedicated to discovering, developing and providing novel small molecule drugs that significantly improve the lives of patients with immune and hematologic disorders, cancer and rare diseases.

Cancer

Central Nervous System (CNS)

Hematologic Diseases

Immune System Diseases

Lung Diseases

Musculoskeletal Diseases

Rigel Pharmaceuticals

Acute myeloid leukemia

Glioma

Olutasidenib is an oral, potent and small molecule investigational agent designed to selectively bind to and inhibit mutated IDH1 enzymes. This targeted treatment has the potential to provide therapeutic benefit by reducing 2-HG levels and restoring normal cellular differentiation.

REZLIDHIA (Olutasidenib) (FT-2102)

Autoimmune disease

Fostamatinib is a tyrosine kinase inhibitor with demonstrated activity against spleen tyrosine kinase (SYK). The major metabolite of fostamatinib, R406, inhibits signal transduction of Fc-activating receptors and B-c

TAVALISSE® (fostamatinib disodium hexahydrate)

Olutasidenib is a small-molecule inhibitor of mutated isocitrate dehydrogenase-1 (IDH1). In patients with AML, susceptible IDH1 mutations are defined as those leading to increased levels of 2-hydroxyglutarate (2-HG) in the leukemia cells and where efficacy is predicted by 1) clinically meaningful remissions with the recommended dose of olutasidenib and/or 2) inhibition of mutant IDH1 enzymatic activity at concentrations of olutasidenib sustainable at the recommended dosage according to validated methods. The most common of such mutations in patients with AML are R132H and R132C substitutions. In vitro, olutasidenib inhibited mutated IDH1 R132H, R132L, R132S, R132G, and R132C proteins; wild-type IDH1 or mutated IDH2 proteins were not inhibited. Olutasidenib inhibition of mutant IDH1 led to decreased 2-HG levels in vitro and in in vivo xenograft models.

REZLIDHIA (Olutasidenib) (FT-2102) 

Non-small cell lung carcinoma

GAVRETO&nbsp;is indicated for the treatment of adult patients with metastatic rearranged during transfection (RET) fusion-positive non-small cell lung cancer (NSCLC) as detected by an FDA-approved test and adult and pediatric patients 12 years of age and older with advanced or metastatic RET fusion-positive thyroid cancer who require systemic therapy and who are radioactive iodine-refractory (if radioactive iodine is appropriate).*
 
*Thyroid indication is approved under accelerated approval based on overall response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trial(s).

GAVRETO (pralsetinib)

COVID-19

Fostamatinib is a tyrosine kinase inhibitor with demonstrated activity against spleen tyrosine kinase (SYK). The major metabolite of fostamatinib, R406, inhibits signal transduction of Fc-activating receptors and B-c
SYK is involved in the intracellular signaling pathways of many different immune cells. Therefore, SYK inhibition may improve outcomes in patients with COVID-19 via inhibition of key Fc gamma receptor (FcyR) and c-type lectin receptor (CLR) mediated drivers of pathology such as pro-inflammatory cytokine release by monocytes and macrophages, production of neutrophil extracellular traps (NETs) by neutrophils, and platelet aggregation. Furthermore, SYK inhibition in neutrophils and platelets may lead to decreased thromboinflammation, alleviating organ dysfunction in critically ill patients with COVID-19.

Rheumatoid arthritis

R552, a systemic molecule being developed for the treatment of autoimmune and inflammatory disorders, and brain penetrating RIPK1 inhibitors for central nervous system (CNS) diseases. In preclinical studies, R552 demonstrated prevention of joint and skin inflammation in a RIPK1-mediated murine model of inflammation and tissue damage.
 
RIPK1 is a critical signaling protein implicated in a broad range of key inflammatory cellular processes including necroptosis, a type of regulated cell death, and cytokine production. In necroptosis, cells rupture leading to the dispersion of cell contents, which can trigger an immune response and enhance inflammation. RIPK1 inhibition has therapeutic potential in treating autoimmune, inflammatory, and neurodegenerative disorders.

R552

REZLIDHIA (Olutasidenib) (FT-2102)+ temozolomide

Lower-risk myeloid dysplastic syndrome

R289 is a prodrug formulation of R835. In preclinical studies, R835 demonstrated activity in multiple animal models of inflammatory disease2,3 and showed that dual inhibition of IRAK1 and IRAK4 with R835 provided more complete suppression of inflammatory cytokines when compared to an IRAK4-selective inhibitor4.
 
 
R289 blocks inflammatory cytokine production in response to toll-like receptor (TLR) and interleukin-1 receptor family (IL-1R) signaling. TLRs and IL-1Rs play a critical role in the innate immune response and dysregulation of these pathways can lead to a variety of inflammatory conditions such as psoriasis, rheumatoid arthritis, and inflammatory bowel disease. Chronic stimulation of both receptor systems is also thought to be behind the bone marrow pro-inflammatory environment responsible for persistent cytopenias in lower-risk myelodysplastic syndrome (MDS) patients1.

R289

REZLIDHIA (Olutasidenib) [triplet therapy]

Blood disorder

Fostamatinib is a tyrosine kinase inhibitor with demonstrated activity against spleen tyrosine kinase (SYK). The major metabolite of fostamatinib, R406, inhibits signal transduction of Fc-activating receptors and B-c
SYK is involved in the intracellular signaling pathways of many different immune cells. Therefore, SYK inhibition may improve outcomes in patients with COVID-19 via inhibition of key Fc gamma receptor (FcyR) and c-type lectin receptor (CLR) mediated drivers of pathology such as pro-inflammatory cytokine release by monocytes and macrophages, production of neutrophil extracellular traps (NETs) by neutrophils, and platelet aggregation. Furthermore, SYK inhibition in neutrophils and platelets may lead to decreased thromboinflammation, alleviating organ dysfunction in critically ill patients with COVID-19.

Rigel Pharmaceuticals

Company Details