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

Olutasidenib DDI Study in Patients With IDH1 Mutation Positive Malignancies (clinicaltrials.gov) - P4 | N=16 | Recruiting | Sponsor: Rigel Pharmaceuticals

New P4 trial • Acute Myelogenous Leukemia • Biliary Cancer • Brain Cancer • Cholangiocarcinoma • Gastrointestinal Cancer • Glioma • Hematological Malignancies • Leukemia • Oncology • Solid Tumor

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 in Relapsed IDH1 Mutated AML Patients Who Have Previously Received Venetoclax (clinicaltrials.gov) - P2 | N=25 | Not yet recruiting | Sponsor: Timothy Pardee

New P2 trial • Acute Myelogenous Leukemia • 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

Differentiation Syndrome in Acute Myeloid Leukemia: Molecular Mechanisms, Clinical Spectrum, and Emerging Therapeutic Paradigms. (PubMed, Int J Mol Sci) - "While differentiation therapy revolutionized acute promyelocytic leukemia (APL) with all-trans retinoic acid (ATRA) and arsenic trioxide (ATO), its extension into non-APL AML has been limited until recent targeted agents...IDH1/2 inhibitors (ivosidenib, enasidenib, olutasidenib) yield overall response rates (ORRs) of 30-94% in AML with DS in 10-19%. Menin inhibitors (revumenib, ziftomenib, enzomenib, bleximenib) achieve ORRs of 33-88% in KMT2A-rearranged or NPM1-mutated AML, with DS in 10-25% and QT prolongation as key toxicities. FLT3 inhibitors (gilteritinib, quizartinib) improve survival in FLT3-mutated AML with DS in 1-5%...Improved recognition of DS and rational combination approaches will be essential to maximize the therapeutic benefit. Future research should address mechanisms of resistance and biomarkers to achieve cures beyond APL."

Journal • Review • Acute Myelogenous Leukemia • Acute Promyelocytic Leukemia • Hematological Malignancies • Leukemia • Oncology • FLT3 • IDH1 • IDH2 • KMT2A • NPM1

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

The Care and Cure of the Leukemias in 2026. (PubMed, Am J Hematol) - "Currently, most leukemias are effectively treated with immunotherapies (highly effective monoclonal antibodies targeting CD19 [blinatumomab], or CD22 [inotuzumab ozogamicin]), BCR::ABL1 tyrosine kinase inhibitors (TKIs; e.g., dasatinib, ponatinib), Bruton TKIs (e.g., ibrutinib, acalabrutinib), BCL-2 inhibitors (venetoclax), IDH1/2 inhibitors (ivosidenib, olutasidenib, and enasidenib), FLT3 inhibitors (e.g., midostaurin, quizartinib, and gilteritinib), menin inhibitors (revumenib, ziftomenib), and chimeric antigen receptor T-cell therapies. Herein, we provide a high-level overview of prominent clinical developments across all leukemias. In contemporary times, harnessing the benefits of novel targeted therapies and the evolving treatment landscape bolster the optimistic view that most, if not all, leukemias are curable."

IO biomarker • Journal • Review • Acute Lymphocytic Leukemia • Acute Myelogenous Leukemia • Bone Marrow Transplantation • Chronic Lymphocytic Leukemia • Chronic Myeloid Leukemia • Hematological Malignancies • Leukemia • Oncology • Transplantation • ABL1 • CD22 • FLT3 • IDH1 • IDH2 • KMT2A • TP53

Matching-Adjusted Indirect Comparison of Olutasidenib and Ivosidenib in Isocitrate Dehydrogenase 1-Mutated Relapsed/Refractory Acute Myeloid Leukemia. (PubMed, Adv Ther) - "While not confirmatory, these findings may be clinically relevant in the context of this difficult-to-treat R/R IDH1m AML population."

Journal • Acute Myelogenous Leukemia • Bone Marrow Transplantation • Hematological Disorders • Hematological Malignancies • Leukemia • Oncology • Transplantation • IDH1

Phase 1/1b Trial Of Olutasidenib And Ziftomenib For NPM1 And IDH1 Co-Mutated Acute Myeloid Leukemia (clinicaltrials.gov) - P1 | N=20 | Not yet recruiting | Sponsor: M.D. Anderson Cancer Center

New P1 trial • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Oncology • IDH1 • NPM1

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

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 (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