Rigel Pharmaceuticals

Final data from the Phase 1/2 ARROW study demonstrates GAVRETO's durable efficacy in RET fusion-positive NSCLC, with a 70.3% overall response rate and median overall survival of 44.3 months.

DelveInsight's 2025 assessment reveals a robust sickle cell disease pipeline with 55+ companies developing 60+ therapeutic candidates across various clinical development stages.

• DelveInsight's latest report reveals a robust pipeline with 110+ companies developing 120+ therapies for acute myeloid leukemia (AML), showing significant investment in this aggressive blood cancer. • Several promising candidates are advancing through clinical trials, including GlycoMimetics' uproleselan in Phase III, BioSight's aspacytarabine (BST-236) in Phase II, and novel approaches like Senti Biosciences' logic-gated CAR-NK cell therapy. • Recent developments include Moleculin Biotech's Phase III MIRACLE trial for annamycin, Qurient's adrixetinib IND approval, and Rigel Pharmaceuticals' trial of REZLIDHIA in combination therapy for IDH1-mutated AML.

Rigel Pharmaceuticals achieved significant revenue growth in 2024, with TAVALISSE sales increasing 12% to $104.8 million and REZLIDHIA sales growing 118% to $23.0 million.

Rigel Pharmaceuticals has announced the enrollment of the first patient in a Phase I clinical trial of fostamatinib for sickle cell disease (SCD).

The FDA has granted Orphan Drug designation to R289 for treating myelodysplastic syndromes (MDS), offering incentives for its development.

The FDA has granted Fast Track designation to R289, Rigel Pharmaceuticals' IRAK1/4 inhibitor, for previously treated transfusion-dependent lower-risk myelodysplastic syndrome (MDS).

An ad-hoc analysis of the AcceleRET-Lung trial revealed a statistically significant increased risk of severe infections in patients treated with pralsetinib compared to standard of care.

Rigel Pharmaceuticals has enrolled the first patient in a Phase Ib/II clinical trial evaluating REZLIDHIA (olutasidenib) in combination with decitabine and venetoclax for acute myeloid leukemia (AML).

• Sanofi and Denali Therapeutics' investigational drug SAR443820 (DNL788), a RIPK1 inhibitor, did not meet the primary endpoint in a Phase 2 clinical trial for amyotrophic lateral sclerosis (ALS). • The trial assessed the change in the ALS Functional Rating Scale-Revised (ALSFRS-R) as the primary outcome, with Sanofi planning to present detailed efficacy and safety results at a future scientific meeting. • Despite the setback in ALS, the Phase 2 study of SAR443820 in multiple sclerosis (MS) is ongoing, and a 52-week open-label extension study in ALS will further evaluate the drug's safety and efficacy. • The failure raises questions about RIPK1 inhibition as a therapeutic strategy for ALS, impacting other companies like Eli Lilly, which has a partnership with Rigel Pharmaceuticals for RIPK1-blocking molecules.