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

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

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

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 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

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

Postmarketing Safety Signals of Revumenib (Menin-KMT2A Inhibitor) vs Acute Myeloid Leukemia-Targeted Therapies: A Class-Restricted Disproportionality Analysis of the FDA Adverse Event Monitoring System (2016–2026) (SOHO 2026) - " AE reports (January 2016–April 2026) were extracted for revumenib (n = 716) and eight targeted AML therapies (n = 70179): ziftomenib, enasidenib, ivosidenib, olutasidenib, gilteritinib, midostaurin, quizartinib, and venetoclax. Analysis of 716 revumenib reports (median age, 50 years; 50.2% male; 57.5% US-sourced; 55.6% serious) identified AML as the recorded indication in 59.2% of cases. Eleven AEs qualified as robust signals. Established toxicities included differentiation syndrome (n = 32; ROR, 9.12; 95% CI, 6.31–13.20), QTc prolongation (n = 29; ROR, 8.88; 95% CI, 6.03–13.08), and decreased platelet count (n = 113; ROR, 3.91; 95% CI, 3.19–4.79)."

Adverse events • Clinical • P4 data • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Oncology • FLT3 • KMT2A

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

Treatment Patterns and Outcomes With Olutasidenib After Venetoclax in IDH1-Mutated AML Using Real-World Data From Chart Review (SOHO 2026) - "Olutasidenib demonstrated robust effectiveness in adults with R/R mIDH1 AML previously treated with venetoclax, a subgroup with historically poor outcomes, with a CR/CRh rate of 60.8% and median response duration of 30.3 months. Reduced transfusion dependence further supports olutasidenib as a viable postvenetoclax option. These findings suggest earlier sequencing of olutasidenib may optimize patient outcomes."

Clinical • Real-world • Real-world evidence • Review • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Myelodysplastic Syndrome • Oncology • DNMT3A • FLT3 • IDH1 • NPM1

Real-World Treatment Patterns and Outcomes With Olutasidenib After Venetoclax in IDH1-Mutated Acute Myeloid Leukemia Using Electronic Health Record Data (SOHO 2026) - "These real-world data demonstrate that olutasidenib was commonly used directly after venetoclax and produced clinically meaningful responses in post-venetoclax R/R mIDH1 AML, supporting olutasidenib as a viable treatment option in this setting. AML: acute myeloid leukemia, CR: complete response, CRc: composite complete response, CRh: complete response with partial hematologic recovery, CRi: complete response with incomplete blood count recovery, DoR: duration of response, EHR: electronic health record, FLT3: fms related receptor tyrosine kinase 3, HMA: hypomethylating agent, HSCT: hematopoietic stem cell transplantation, IDH1: isocitrate dehydrogenase 1, IQR: interquartile range, MLFS: morphologic leukemia-free state, NPM1: nucleophosmin 1, ORR: objective response rate, OS: overall survival, R/R: relapsed/refractory, RUNX1: RUNX family transcription factor 1."

Clinical • HEOR • Real-world • Real-world evidence • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Oncology • FLT3 • IDH1 • NPM1 • RUNX1

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

Disentangling disease from drug: neurological adverse events with IDH inhibitors in glioma (EANO 2026) - "Extracted outcomes focused on the incidence, severity, and characterization of neurological adverse events, with particular attention to reporting methodology and attribution. Across phase I-III trials of IDH inhibitors—including vorasidenib, ivosidenib, olutasidenib, and safusidenib—neurological adverse events were commonly reported, with headache being the most frequent (approximately 13-46%). The interpretation of neurological adverse events in IDH-mutant gliomas is limited by significant methodological heterogeneity, lack of standardized symptom reporting, and confounding from tumor-related manifestations and prior therapies. Neurological adverse events in IDH inhibitor-treated glioma patients are common but undercharacterized. Available evidence suggests that some symptoms-particularly headache-may reflect the underlying disease rather than drug toxicity, at least in certain contexts."

Adverse events • Brain Cancer • Glioma • Oncology • Solid Tumor

Predictors of Long-Term Response to Olutasidenib in Mutant IDH1 Acute Myeloid Leukemia (SOHO 2026) - P1/2 | "Among patients with a CR/CRh duration >12 months, baseline median age was 72 years, 69% were female, 73% had an R132C mutation, 12% received prior venetoclax, 88% were platelet TI, and 73% were RBC TI. Olutasidenib enabled long-term CR/CRh in half of patients with R/R mIDH1 AML in CR/CRh without transplant. Relapsed (vs refractory) AML and female sex were significant predictors of long-term response with olutasidenib. Previously presented at ASCO 2026."

Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Oncology • DNMT3A • FLT3 • IDH1 • NPM1

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

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

Olutasidenib Alone or in Combination With Azacitidine as a Bridge to Transplant in Relapsed/Refractory mIDH1 AML. (PubMed, Eur J Haematol) - No abstract available

Journal • Acute Myelogenous Leukemia • Transplantation

Phase 1/1b Trial Of Olutasidenib And Ziftomenib For NPM1 And IDH1 Co-Mutated Acute Myeloid Leukemia (clinicaltrials.gov) - P1 | N=32 | Active, not recruiting | Sponsor: M.D. Anderson Cancer Center | N=20 ➔ 32 | Initiation date: Aug 2026 ➔ May 2026 | Not yet recruiting ➔ Active, not recruiting

Enrollment change • Enrollment closed • Trial initiation date • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Oncology • IDH1 • NPM1

For the second quarter ended June 30, 2026, total revenues were $78.7 million, consisting of $67.0 million in net product sales and $11.7 million in contract revenues from collaborations (PRNewswire) - "Net product sales increased 14% compared to $58.9 million in the same period of 2025....GAVRETO net product sales were $10.7 million, a decrease of 10% compared to $11.8 million in the same period of 2025. REZLIDHIA net product sales were $8.9 million, an increase of 27% compared to $7.0 million in the same period of 2025."

Sales • Acute Myelogenous Leukemia • Non Small Cell Lung Cancer • Thyroid Gland Carcinoma

Prognostic Implications and Therapeutic Landscape of IDH1-Mutated AML: An Updated Review of Evidence and Indian Perspective. (PubMed, Asian Pac J Cancer Prev) - "This review summarizes the prognostic implications of IDH1 mutations in AML and critically examines emerging IDH1 targeted therapies, highlighting their impact on disease biology and evolving treatment algorithms."

Journal • Review • Acute Myelogenous Leukemia • Hematological Disorders • Hematological Malignancies • Leukemia • Oncology • DNMT3A • IDH1 • NPM1

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 | Trial completion date: Jan 2032 ➔ Dec 2029 | Trial primary completion date: Jan 2031 ➔ Dec 2027

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

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

Enrollment open • Acute Myelogenous Leukemia • IDH1