Rezlidhia (olutasidenib) / Rigel Pharmaceuticals, Novo Nordisk, Kissei, Dr. Reddy’s - LARVOL DELTA

Isocitrate Dehydrogenase Inhibitors in Acute Myeloid Leukemia. (PubMed, Chem Biodivers) - "Inhibitors of mutated IDH1 and IDH2, vorasidenib, ivosidenib, olutasidenib, and enasidenib, respectively, were recently approved by the FDA for relapsed/refractory AML. In this review, we mainly focus on IDH inhibitors in leukemia therapy, including the discovery, structure optimization, activity of IDH inhibitors, and applications, which provided the reference for the discovery of new anticancer agents."

Journal • Review • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Oncology • IDH1 • IDH2

A Phase II open-label study of Olutasidenib Post-Transplant Maintenance Therapy for Patients with IDH1-Mutated Myeloid Malignancies (TCT-ASTCT-CIBMTR 2026) - P2 | "Efficacy of Olutasidenib post transplant as maintenance 3. Correlatives of effect on immunobiology with Olutasidenib post stem cell transplant"

Clinical • P2 data • Post-transplantation • Acute Graft versus Host Disease • Acute Myelogenous Leukemia • Bone Marrow Transplantation • Chronic Graft versus Host Disease • Chronic Myelomonocytic Leukemia • Graft versus Host Disease • Hematological Malignancies • Immunology • Leukemia • Oncology • Transplantation • IDH1

MULTICENTER PILOT CLINICAL TRIAL OF OLUTASIDENIB AS MAINTENANCE THERAPY AFTER ALLOGENEIC HEMATOPOIETIC CELL TRANSPLANTATION IN PATIENTS WITH AML/MDS CARRYING IDH1 MUTATIONS (EBMT 2026) - P1 | "GVHD prophylaxis was with post-transplant cyclophosphamide in majority of patients (n=5; 83%) At the data cutoff of December 2025, 4 patients are currently on study with median follow-up of 140 days (range: 112-293). In conclusion, post-HCT maintenance therapy with Olutasidenib was safe and feasible with favorable survival outcomes in AML/MDS patients carrying IDH1 mutations. No unusual toxicity or safety signal was noted in patients treated so far, the trial continues to accrue patients in multicenter setting"

Clinical • Acute Myelogenous Leukemia • Graft versus Host Disease • Hematological Malignancies • Immunology • Infectious Disease • Myelodysplastic Syndrome • Pneumonia • Pulmonary Disease • Respiratory Diseases • Transplantation • IDH1

Olutasidenib Single Plus Combo Therapy in IDH1mut AML After Induction and Consolidation (clinicaltrials.gov) - P1 | N=15 | Recruiting | Sponsor: Virginia Commonwealth University | Not yet recruiting ➔ Recruiting | Trial completion date: Oct 2029 ➔ Oct 2030 | Trial primary completion date: Oct 2027 ➔ Oct 2028

Enrollment open • Trial completion date • Trial primary completion date • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Oncology • IDH1

Olutasidenib in recurrent/relapsed locally advanced or metastatic IDH1-mutated chondrosarcoma: phase 1b/2 trial. (PubMed, Nat Commun) - P1/2 | "Olutasidenib was well tolerated and conferred disease control in cCS. Study limitations include open-label design and low patient sample due to rarity of cCS."

Journal • P1/2 data • Oncology • Sarcoma • Solid Tumor • IDH1

Olutasidenib in post-venetoclax patients with mIDH1 AML. (ASCO 2023) - P1/2 | "VEN was used with azacytidine (AZA) in 8 patients, after AZA (1), and with decitabine (5), cytarabine +/- idarubicin (5), and dinaciclib (2). Olutasidenib induced durable remissions in patients with mIDH1 R/R AML, including those failing prior treatment with a venetoclax-based regimen. Clinical trial information: NCT02719574."

Clinical • Acute Myelogenous Leukemia • Hematological Disorders • IDH1

Olutasidenib for mutated IDH1 acute myeloid leukemia: Final five-year results from the phase 2 pivotal cohort. (ASCO 2024) - P1/2 | "This analysis provides an additional 2 years of data beyond the results that led to FDA approval of olutasidenib. This first report of the five-year data further demonstrates the rapid and durable responses observed with olutasidenib in heavily pretreated patients with mIDH1 AML, including those R/R to prior venetoclax."

P2 data • Acute Myelogenous Leukemia • Bone Marrow Transplantation • Constipation • Fatigue • Febrile Neutropenia • Gastroenterology • Gastrointestinal Disorder • Hematological Disorders • Hematological Malignancies • Leukemia • Neutropenia • Oncology • Transplantation • IDH1

Olutasidenib alone or combined with azacitidine in patients with mutant IDH1 myelodysplastic syndrome. (PubMed, Blood Adv) - P1/2 | "Olutasidenib with or without azacitidine demonstrated encouraging clinical activity and tolerability in patients with higher-risk mIDH1 MDS. NCT02719574."

Journal • Acute Myelogenous Leukemia • Fatigue • Hematological Disorders • Hematological Malignancies • Leukemia • Myelodysplastic Syndrome • Oncology • IDH1

Olutasidenib (FT-2102) induces durable complete remissions in patients with relapsed or refractory IDH1-mutated AML. (PubMed, Blood Adv) - P1/2 | "Response rates were similar in patients who had and who had not received prior venetoclax. The observed efficacy represents a therapeutic advance in this molecularly defined, poor-prognosis patient population with mIDH1 R/R AML. This trial is registered at www.clinicaltrials.gov as NCT02719574."

Journal • Acute Myelogenous Leukemia • Febrile Neutropenia • Hematological Disorders • Hematological Malignancies • Leukemia • Neutropenia • Oncology • Thrombocytopenia • IDH1

A Phase 2 Study of Olutasidenib in Relapsed/ Refractory Acute Myeloid Leukemia: Outcomes by Number of Prior Treatment Regimens (SOHO 2025) - P1/2 | "Prior treatment with hypomethylating agents was reported in 43% and 33%, respectively; prior venetoclax use in 11% and 4%, respectively. Patients who received olutasidenib earlier in the treatment course (after 1-2 prior regimens) had better clinical outcomes than patients treated later (after ≥3 prior regimens), supporting the potential benefit of earlier use of olutasidenib in the R/R setting. Study Funding: Forma Therapeutics, Inc. Abstract Development: Rigel Pharmaceuticals, Inc."

P2 data • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Oncology • IDH1

Effectiveness of olutasidenib versus ivosidenib in patients with mutated isocitrate dehydrogenase 1 acute myeloid leukemia who are relapsed or refractory to venetoclax: the 2102-HEM-101 trial versus a US electronic health record-based external control arm. (PubMed, Leuk Lymphoma) - "Following weighting, treatment with OLU versus IVO was associated with significantly higher rates of complete response (RD: 0.25; 95%CI: 0.01, 0.49), transfusion independence (RD: 0.27; 95%CI: 0.01, 0.53), and OS (HR: 0.33; 95%CI: 0.11, 0.94). Results suggest favorable effectiveness of OLU versus IVO in this population."

Journal • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Oncology • IDH1

OLUTASIDENIB FOR MUTATED IDH1 ACUTE MYELOID LEUKEMIA: FINAL FIVE-YEAR RESULTS FROM THE PHASE 2 PIVOTAL COHORT (EHA 2024) - P1/2 | "This analysis provides an additional 2 years of data beyond the results that led to FDA approval of olutasidenib. This first report of the five-year data further demonstrates the rapid and durable responses observed with olutasidenib in heavily pretreated patients with mIDH1 AML, including those R/R to prior venetoclax."

P2 data • Acute Myelogenous Leukemia • Bone Marrow Transplantation • Constipation • Fatigue • Febrile Neutropenia • Gastroenterology • Gastrointestinal Disorder • Hematological Disorders • Hematological Malignancies • Leukemia • Neutropenia • Oncology • Transplantation • IDH1

A phase 2 study of olutasidenib in relapsed/refractory acute myeloid leukemia: Outcomes by number of prior treatment regimens. (ASCO 2025) - P1/2 | "Forty-three percent and 33% of patients had prior treatment with a hypomethylating agent, and 11% and 4% received prior venetoclax therapy (1-2 and ≥3 prior regimens groups, respectively). Higher response rates (including CR and CRh) and greater survival were observed in patients receiving OLU following 1-2 versus ≥3 prior treatment regimens, providing rationale for initiating OLU earlier in the R/R treatment paradigm. Efficacy of OLU stratified by number of prior regimens.NR, not reached."

P2 data • Acute Myelogenous Leukemia • Bone Marrow Transplantation • Fatigue • Hematological Malignancies • Leukemia • Oncology • IDH1

Olutasidenib as Maintenance Therapy After Treatment Response in Mutated IDH1 (mIDH1) Acute Myeloid Leukemia (AML) (SOHO 2025) - P1/2 | "Two patients with prior venetoclax therapy entered with a CRi, improved to CR with partial hematologic recovery (CRh) at 1 month and 8.3 months, and then were in CR at 8.3 and 20.3 months. Olutasidenib monotherapy demonstrated clinically meaningful activity as a switch maintenance strategy in AML patients with CR/CRi and persistent MRD ≥0.01% after prior therapy. These results support the potential benefit of switching to olutasidenib in response to therapy, with the goal of prolonging remission. Supported by Forma Therapeutics, Inc.; Rigel Pharmaceuticals, Inc."

Clinical • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Oncology • IDH1

Efficacy and Safety of Olutasidenib Monotherapy in Primary Refractory Acute Myeloid Leukemia: A Post Hoc Analysis of a Phase 2 Study (SOHO 2025) - P1/2 | "Sixty-one percent received a median of one prior treatment regimen (28/46; range, 1.0-4.0), including hypomethylating agents (25/46; 54%), cytarabine+anthracycline (20/46; 43%), venetoclax (3/46; 7%), or allogeneic stem cell transplant (2/46; 4%). Olutasidenib monotherapy showed meaningful response rates and durable response duration in AML patients refractory to induction, with a 30% CR/CRh rate and 17.6-month duration. Olutasidenib may offer an effective therapeutic option with acceptable ntolerability for patients with primary refractory AML."

Clinical • Monotherapy • P2 data • Retrospective data • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Oncology • IDH1

Olutasidenib in post-venetoclax patients with mutant isocitrate dehydrogenase 1 (mIDH1) acute myeloid leukemia (AML). (PubMed, Leuk Lymphoma) - "Safety was consistent with the overall profile of olutasidenib. Olutasidenib offers a valuable treatment option for patients with mIDH1 AML previously treated with venetoclax."

Journal • Acute Myelogenous Leukemia • Hematological Disorders • Hematological Malignancies • Leukemia • Oncology • IDH1

Olutasidenib alone or with azacitidine in IDH1-mutated acute myeloid leukaemia and myelodysplastic syndrome: phase 1 results of a phase 1/2 trial. (PubMed, Lancet Haematol) - P1/2 | "Olutasidenib, with or without azacitidine, was well tolerated and showed meaningful clinical activity in patients with IDH1-mutated acute myeloid leukaemia. The results of this phase 1 study provide rationale for the continued evaluation of olutasidenib in multiple patient populations with myeloid malignancies."

Journal • P1/2 data • Acute Myelogenous Leukemia • Febrile Neutropenia • Hematological Disorders • Hematological Malignancies • Leukemia • Myelodysplastic Syndrome • Neutropenia • Oncology • Thrombocytopenia • IDH1

Olutasidenib Alone or in Combination with Azacitidine Induces Durable Complete Remissions in Patients with mIDH1 Myelodysplastic Syndromes/Neoplasms (MDS) (ASH 2023) - P1/2 | "This treatment had a tolerable and manageable safety profile. These encouraging results, which warrant further investigation with a larger number of patients, show that olutasidenib has clinically meaningful activity in patients with mIDH1 MDS."

Clinical • Combination therapy • Acute Myelogenous Leukemia • Constipation • Fatigue • Gastroenterology • Gastrointestinal Disorder • Hematological Disorders • Hematological Malignancies • Infectious Disease • Leukemia • Myelodysplastic Syndrome • Neutropenia • Oncology • Pneumonia • Respiratory Diseases • Thrombocytopenia • IDH1

Safety and efficacy of olutasidenib, an IDH1 mutant inhibitor, for the treatment of recurrent/relapsed or locally advanced or metastatic IDH1 mutated chondrosarcoma (ESMO 2024) - P1/2 | "OLU was well tolerated and conferred durable disease control in cCS. OLU may represent a treatment option for patients with IDH1m+ cCS who otherwise have no other effective treatment."

Clinical • Metastases • Oncology • Sarcoma • Solid Tumor • IDH1 • IDH2

Rigel expects to report fourth quarter of 2025 gross product sales of $84.5 million. Net product sales are expected to be $65.4 million, compared to $46.5 million for the same period of 2024, including (Rigel Press Release) - "TAVALISSE (fostamatinib disodium hexahydrate) net product sales of $45.6 million...GAVRETO (pralsetinib) net product sales of $10.2 million....REZLIDHIA (olutasidenib) net product sales of $9.6 million compared to $7.4 million for the same period of 2024."

Sales projection • Acute Myelogenous Leukemia • Immune Thrombocytopenic Purpura • Non Small Cell Lung Cancer

Onco360, the nation’s leading independent specialty pharmacy, has been chosen by Rigel Pharmaceuticals as limited distribution specialty pharmacy partner for TAVALISSE (fostamatinib disodium hexahydrate), GAVRETO (pralsetinib), and REZLIDHIA (olutasidenib) (GlobeNewswire)

Licensing / partnership • Acute Myelogenous Leukemia • Immune Thrombocytopenic Purpura • Non Small Cell Lung Cancer • Thyroid Gland Medullary Carcinoma

Olutasidenib With Azacitidine Followed by Olutasidenib Maintenance for the Treatment of IDH1-mutated Acute Myeloid Leukemia in Patients With Prior Treatment With Venetoclax Plus a Hypomethylating Agent (clinicaltrials.gov) - P2 | N=28 | Recruiting | Sponsor: University of California, Davis | Not yet recruiting ➔ Recruiting

Enrollment open • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Oncology • IDH1

Efficacy of novel agents in the treatment of acute myeloid leukemia and myelodysplastic syndrome: A systematic review and meta-analysis (ASH 2025) - "Newer agents included for AML were Guadectiabine, Magrolimab, Alvocidib, Enasidenib, Flotetuzumab, Vadastuximab, Mitoxantrone, Pevonedistat, Entospletinib, Eprenetapopt, Belinostat, Onvansertib, Panobinostat, Cediranib Maleate, Nilotinib, Emavusertib, and anti-CD45 antibody (DOTA-BC8). The newer agents investigated for MDS included Rigosertib, Imetelstat, Pembrolizumab, Enasidenib, Sabatolimab, Ivosidenib, Elitercept, Pevonedistat, Emavusertib, Atezolizumab, and Olutasidenib...All patients were treated concomitantly with either azacitidine (77%) or decitabine (23%)... This meta-analysis and systematic review demonstrate promising efficacy for novel agents in AML and MDS patients. There is a need for prospective trials with larger patient populations to investigate these agents further."

Retrospective data • Review • Acute Myelogenous Leukemia • Hematological Disorders • Hematological Malignancies • Leukemia • Myelodysplastic Syndrome • Neutropenia • TP53

Outcomes of Patients With IDH1-Mutated Myeloid Neoplasms Treated With Olutasidenib. (PubMed, Clin Lymphoma Myeloma Leuk) - "Olutasidenib-based therapy demonstrated 100% response rates in previously untreated IDH1-mutated AML and MDS and modest efficacy in heavily pretreated R/R AML and MDS. Durable remissions occurred in select responders and with stem cell transplant. Further evaluation of olutasidenib as frontline therapy in IDH1-mutated AML or MDS and as a bridge to transplant is warranted."

Journal • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Myelodysplastic Syndrome • Oncology • Transplantation • IDH1

Olutasidenib With Azacitidine Followed by Olutasidenib Maintenance for the Treatment of IDH1-mutated Acute Myeloid Leukemia in Patients With Prior Treatment With Venetoclax Plus a Hypomethylating Agent (clinicaltrials.gov) - P2 | N=28 | Not yet recruiting | Sponsor: University of California, Davis

New P2 trial • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Oncology