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

Real-world outcomes of myelodysplastic syndrome and chronic myelomonocytic leukemia treated with IDH1 and IDH2 inhibitors in the frontline setting (ASH 2025) - "Selective IDH1/2 inhibitors including ivosidenib (IDH1), olutasidenib (IDH1), and enasidenib (IDH2) are currently approved for IDH1/2-mutated (mIDH1/2) relapsed/refractory (r/r) acute myeloid leukemia; ivosidenib is also approved for mIDH1 r/r MDS based on clinical benefit observed in 18 patients on trial AG120-C-001...Patients previously treated with venetoclax (VEN) or a hypomethylating agent (HMA) were excluded... This study highlights safety and efficacy of IDH inhibitors, particularly among older adults with treatment-naïve MDS or CMML. Nearly half of our cohort were adults >80 years, a group often underrepresented in pivotal clinical trials. The overall response rate (CR + CRl + CRh) was 88.9% among 9 patients with BME; 50% of the entire cohort achieved PB-CR and median OS was 26 months."

Clinical • Real-world • Real-world evidence • Acute Myelogenous Leukemia • Chronic Myelomonocytic Leukemia • Hematological Disorders • Hematological Malignancies • Leukemia • Myelodysplastic Syndrome • Neutropenia • IDH1 • IDH2

Real-world AML survival paradox: Early escalation vs. sustained remission (ASH 2025) - "Patients were stratified into two cohorts: Cohort A (early escalation, n=1,460), comprising patients who received either second-line chemotherapy (including fludarabine, cladribine, gilteritinib, enasidinib, revumenib, olutasidenib, etoposide, or clofarabine) or hematopoietic stem cell transplant (HSCT) as salvage therapy within 6 months of completing initial therapy; and Cohort B (no escalation, n=10,540), consisting of patients who received no additional therapy during this same period. Our findings highlight the complex interplay between disease biology and treatment interventions: achieving MRD-negativity remains critical, but when MRD-positivity occurs, subsequent treatment decisions based on disease genetics prove critical. Prospective studies incorporating molecular MRD monitoring are needed to confirm these findings and further optimize post-remission therapy approaches."

Clinical • Real-world • Real-world evidence • Acute Myelogenous Leukemia • Bone Marrow Transplantation • Hematological Malignancies • Leukemia

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

Olutasidenib monotherapy in patients with mIDH1 Acute Myeloid Leukemia who received prior intensive chemotherapy (ASH 2025) - P1/2 | "This post hoc analysis evaluated the efficacy andsafety of olutasidenib monotherapy in a subset of patients (n=105) from the phase 2 pivotal cohort whohad received prior intensive chemotherapy (IC; e.g., high-dose cytarabine with daunorubicin,mitoxantrone with etoposide and cytarabine, or transplant conditioning regimens). The pivotal cohort of the global, multicenter, registrational, open-label phase 2 trial assessedolutasidenib 150 mg BID in adults with R/R AML harboring mIDH1. Among this cohort of patients with R/R mIDH1 AML who had received prior IC, treatmentwith olutasidenib monotherapy produced clinically meaningful response rates that closely align withthose observed in the overall population, with durable responses and acceptable tolerability. Thesefindings support the utility of olutasidenib in patients previously treated with intensive chemotherapy."

Clinical • Monotherapy • Acute Myelogenous Leukemia • Constipation • Febrile Neutropenia • Gastroenterology • Gastrointestinal Disorder • Hematological Malignancies • Leukemia • Neutropenia • IDH1

Analysis of hematologic improvement (HI) by time to response in Relapsed/Refractory Acute Myeloid Leukemia (AML) patients treated with olutasidenib (ASH 2025) - P1/2 | "Later responders tended to have lower baseline platelet counts and higher bone marrowblast percentages, suggesting lower hematopoietic reserve and greater disease burden. Some patientswith best overall response of SD experienced modest improvements in hematologic parameters,including platelets, hemoglobin, and transfusion independence. These findings suggest that continuingolutasidenib treatment beyond 2 cycles may offer hematologic benefits, even in the absence of an earlyclinical response."

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

Assessment of real-world treatment patterns and outcomes of olutasidenib in patients with mutated isocitrate dehydrogenase 1 Acute Myeloid Leukemia previously treated with venetoclax using electronic health record data (ASH 2025) - P1/2 | "In this real-world cohort, despite the small sample size,50% of patients responded to post-venetoclax olutasidenib consistent with the clinical efficacy observedin the pivotal Phase 2 trial. These findings support the use of olutasidenib as a viable therapeutic optionin post-venetoclax treatment settings although more data is required to confirm these findings."

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

The transition from myelodysplastic neoplasm to secondary acute myeloid leukemia is revealed by molecular analysis, while functional drug screening demonstrates novel sensitivity patterns with clinical implication (ASH 2025) - "There are only a limited number of agentsthat are FDA approved for treatment of MDS including BCL2 and IDH inhibitors, hypomethylating agents(HMAs), ESAs, luspatercept and imetelstat...For MDS patients, weidentified the most effective agents with the proportion of sensitive samples noted in parentheses:mitoxantrone (100%), olutasidenib (78%), venetoclax (67%), dinaciclib (67%), trametinib (67%), olaparib(56%), GSK3368715 (56%), pacritinib (44%), lenalidomide (44%), fludarabine (55%), tasquinimod (44%),veliparib (44%). For sAML patients, we identified lenalidomide, olutasidenib, GSK3368715 have high DSSsin all tested samples, while fludarabine, fedratinib, tasquinimod, trametinib, veliparib, mitoxantrone andolaparib have high DSSs in 75% of the AML samples...Cancer Research 2024), and our samples included SF3B1 and U2AF1 mutations.Summary This study of the transition of MDS to sAML highlights molecular changes and reveals new drugsensitivity with agents that could be..."

Biomarker • Clinical • IO biomarker • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Myelodysplastic Syndrome • CD34 • FLT3 • JAK2 • NRAS • PTPN11 • SF3B1 • U2AF1

Trial in progress: Pilot trial of olutasidenib maintenance post allogeneic hematopoietic cell transplantation in patients carrying IDH1 mutation with AML, MDS, or CMML (ASH 2025) - P1 | "In summary, this trial aims to establish the safety and feasibility of olutasidenib maintenance post-alloHCT in patients with mIDH1+ myeloid malignancies. Given the high risk of relapse in this populationand the promising activity of olutasidenib, this study may provide critical groundwork for largerprospective maintenance strategies in the post-transplant setting."

Clinical • Acute Myelogenous Leukemia • Chronic Graft versus Host Disease • Chronic Myelomonocytic Leukemia • Graft versus Host Disease • Hematological Malignancies • Immunology • Infectious Disease • Leukemia • Myelodysplastic Syndrome • Transplantation • IDH1

IDH mutant MDS: Proposal for disease subset recognition based on molecular and clinical features, and the availability of targeted therapies (ASH 2025) - "For example, del5q MDS accounts for <5% of MDS, but the impact of lenalidomide treatmentemphasizes the need to identify those patients (pts)...Reponses to azacitidine were higher among IDH1 & 2 MT pts compared to WT (ORR (CR+PR+HI) 49.1%,48.1%, and 37.5%, respectively, and CR was 14.3%, 14% and 10.4%, respectively) (p=0.024 compared toWT and p=1.0 between IDH1 & 2).Among IDH1 MT pts, 16 pts received ivosidenib at any timepoint including AML...Three ptsreceived olutasidenib after ivosidenib, no response was observed.Among IDH2 MT pts, 42 received enasidenib at any timepoint including AML...Treatment with IDH inhibitors during the disease coursesuggests survival benefit among those pts. Our findings suggest the recognition of IDH MT MDS as aunique disease subset."

Clinical • Acute Myelogenous Leukemia • Hematological Malignancies • Immunology • Myelodysplastic Syndrome • ASXL1 • IDH1 • IDH2 • RUNX1 • SF3B1 • SRSF2 • TET2 • TP53

Clinical characteristics and response in olutasidenib-treated Relapsed/Refractory mIDH1 Acute Myeloid Leukemia (AML) patients with stable disease following two treatment cycles (ASH 2025) - P1/2 | "Themost common IDH1 mutation type was R132C (44%), followed by R132 L/G/S and R132H (28% each).Prior therapy included hypomethylating agents (n=16; 44%), hematopoietic stem cell transplant (n=6; 17%), and venetoclax therapy (n=2; 6%). Patients with SD after 2 cycles of olutasidenib may experience meaningful clinical benefitwith continued treatment, as one-third of these patients subsequently achieved a late response resultingin a lower risk of death compared to patients with no later response. These findings suggest that early SDdoes not predict treatment failure and support continuing olutasidenib for at least 6 cycles or untildisease progression."

Clinical • Acute Myelogenous Leukemia • Bone Marrow Transplantation • Cough • Febrile Neutropenia • Hematological Disorders • Hematological Malignancies • Infectious Disease • Leukemia • Neutropenia • Pneumonia • Respiratory Diseases • IDH1

Real-world practice patterns of IDH inhibitors and outcomes of on-label IDH inhibitor use for Acute Myeloid Leukemia in the United States (ASH 2025) - "In the U.S., ivosidenib (ivo) is approved with or without azacitidine (aza) in the frontline (FL) and asmonotherapy in relapsed/refractory (R/R) IDH1-mutated (mut) AML. Olutasidenib (oluta) is approved asmonotherapy for R/R IDH1-mut AML...Overall, 209 of387 patients (54%) received IDHi regimens on-label.In FL IDH1-mut AML (n=45), 11 received ivo monotherapy, 25 aza/ivo, and 9 used off-label combinations:3 hypomethylating agent (HMA) + venetoclax (ven) + ivo, 5 decitabine/ivo, 1 intensive chemotherapy(IC)/ivo...In R/R IDH2-mut AML(n=189), 119 patients received ena monotherapy and 70 received off-label combinations (25 HMA/ena, 30HMA/ven/ena, 7 IC/ena, 6 ena/gilteritinib, and 2 other).In on-label treatment of FL IDH1-mut AML, one of four patients with response assessment of FL ivomonotherapy had a CR/CRh...Practice patterns for IDH inhibitors in AML vary considerably, with high amounts of off-label use.Response rates for ivosidenib and enasidenib in the real-world setting..."

Clinical • Real-world • Real-world evidence • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • IDH1 • IDH2

Phase 1b Study of IDH Inhibition with Enasidenib and MEK Inhibition with Cobimetinib in Patients with Relapsed or Refractory Acute Myeloid Leukemia Who Have Co-Occurring IDH2 and RAS Signaling Gene Mutations (ASH 2024) - P1 | "Background : Targeting of methylation by mutant isocitrate dehydrogenase (IDH) has changed the therapeutic landscape of relapsed or refractory (R/R) AML, culminating in the approval of IDH inhibitors, enasidenib, ivosidenib, and olutasidenib. Concurrent RAS-signaling mutations represent a growing problem in the management of R/R IDH mutant AML, since they are associated with resistance to IDH inhibitors and other approved therapies such as venetoclax-based regimens...As enasidenib is a moderate CYP3A inducer, the protocol was amended to also allow concomitant use of the moderate CYP3A4 inhibiting antifungal, isavuconazonium sulfate (cresemba)...To date, four patients have enrolled with three patients completing at least 1 cycle of treatment. Enrollment is open at City of Hope and the Fred Hutchinson Cancer Center (NCT05441514)."

Clinical • P1 data • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Oncology • BRAF • IDH2 • KRAS • NF1 • NRAS • PTPN11 • RIT1 • TET2

Molecularly-guided phase 2 umbrella trial for children and young adults with newly-diagnosed high-grade glioma (HGG) including diffuse intrinsic pontine glioma (DIPG): CONNECT TarGeT (Targeted pediatric HGG therapy) trial in progress (WFNOS 2025) - P, P2 | "The following TarGeT treatment arms (most involving upfront radiotherapy), are open or soon to open, selected based on prevalence of targets in HGG/DIPG, relevant pre-clinical and clinical data, established pediatric safety data, and prioritizing combinations: (A) ribociclib and everolimus (target: cell cycle or PI3K//mTOR pathway alterations) [NCT05843253], (A-2) ribociclib and temozolomide (H3G34 mutation) (B) tovorafenib (MAPK pathway alterations), (D) olutasidenib and temozolomide (IDH1 mutation) [NCT06161974], (F) nivolumab and relatlimab (high tumor mutational burden, mismatch repair deficiency), (L) lorlatinib (+/- chemotherapy or radiation) (ROS1, ALK fusion). Development of additional treatment arms is underway, with possibility of incorporating new arms as supporting data allows."

Clinical • P2 data • Tumor mutational burden • Brain Cancer • Diffuse Intrinsic Pontine Glioma • Pediatrics • Solid Tumor • ALK • IDH1 • ROS1 • TMB

OLUVENAZA: Olutasidenib, Venetoclax, and Azacitidine in IDH1 Mutated Newly Diagnosed Acute Myeloid Leukemia Patients Eligible for Intensive Induction Chemotherapy (clinicaltrials.gov) - P2 | N=16 | Recruiting | Sponsor: Justin Watts, MD | Trial completion date: Nov 2028 ➔ Feb 2029 | Initiation date: Nov 2025 ➔ Feb 2026 | Trial primary completion date: Nov 2028 ➔ Feb 2029

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

Molecularly-guided phase 2 umbrella trial for children and young adults with newly-diagnosed high-grade glioma (HGG) including diffuse intrinsic pontine glioma (DIPG): CONNECT TarGeT (Targeted pediatric HGG therapy) trial in progress (SNO 2025) - P, P2 | "The following TarGeT treatment arms (most involving upfront radiotherapy), are open or soon to open, selected based on prevalence of targets in HGG/DIPG, relevant pre-clinical and clinical data, established pediatric safety data, and prioritizing combinations: (A) ribociclib and everolimus (target: cell cycle or PI3K//mTOR pathway alterations) [NCT05843253], (A-2) ribociclib and temozolomide (H3G34 mutation) (B) tovorafenib (MAPK pathway alterations), (D) olutasidenib and temozolomide (IDH1 mutation) [NCT06161974], (F) nivolumab and relatlimab (high tumor mutational burden, mismatch repair deficiency), (L) lorlatinib (+/- chemotherapy or radiation) (ROS1, ALK fusion). Development of additional treatment arms is underway, with possibility of incorporating new arms as supporting data allows."

Clinical • P2 data • Tumor mutational burden • Brain Cancer • Diffuse Intrinsic Pontine Glioma • Glioma • High Grade Glioma • Pediatrics • Solid Tumor • ALK • IDH1 • ROS1 • TMB

Racial Disparities in Acute Myeloid Leukemia and Myelodysplastic Syndrome US FDA Drug Approval Trials (ASH 2023) - "(Table 1) Olutasidenib, glasdegib plus low dose cytarabine, gemtuzumab-ozogamicin, liposome-encapsulated combination of daunorubicin and cytarabine, ivosidenib, and midostaurin were approved for AML. An oral combination of decitabine and cedazuridine was approved for MDS... Despite being affected adversely to a significant degree by AML, black patients are underrepresented in drug trials. More efforts should be made to include them in trials so that better treatment plans can be formulated for this underrepresented group of patients."

Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Myelodysplastic Syndrome • Oncology

Rigel Announces Publication of Final 5-year Data on REZLIDHIA (olutasidenib) in Patients with R/R mIDH1 AML in the Journal of Hematology & Oncology (Rigel Press Release) - "The safety profile remained consistent with what was previously reported, with no new safety signals identified...Additional key points from the paper include...Of 147 efficacy evaluable patients, complete remission (CR) or CR with partial hematologic recovery (CRh) was achieved in 35%. The median duration of CR/CRh was 25.3 months. Overall response rate (ORR) was 48%, with median duration of 15.5 months. Median overall survival (OS) was 11.5 months....Patients with one to two prior regimens had a higher CR/CRh rate (41%), ORR (54%), and longer median OS (13 months) compared to those with ≥3 prior regimens (CR/CRh: 24%; ORR: 39%; median OS: 8.9 months)."

P2 data • Acute Myelogenous Leukemia

Olutasidenib for mutated IDH1 acute myeloid leukemia: final five-year results from the phase 2 pivotal cohort. (PubMed, J Hematol Oncol) - P1/2 | "These 5-year data support the durable efficacy and manageable safety profile of olutasidenib in R/R mIDH1 AML, including heavily pretreated patients. Findings highlight the potential role of olutasidenib in earlier lines of treatment, and support sustaining therapy for at least 6 months to allow for a clinical response. Further research is warranted to optimize treatment sequencing and patient selection."

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

Olutasidenib for mutated IDH1 acute myeloid leukemia: final five-year results from the phase 2 pivotal cohort (J Hematol Oncol) - "Among the 147 efficacy-evaluable patients, 51 achieved complete remission (CR) or CR with partial hematologic recovery (CRh), resulting in a CR/CRh rate of 35% (P < 0.001; 95% CI, 27–43), with 32% of responders achieving CR. The median time to CR/CRh was 1.9 months (range, 0.9–5.6 months). Among responders, 33% achieved CR/CRh within 2–4 months and 12% required ≥ 4 months. The overall response rate (ORR) was 48% (n = 71; 95% CI, 40–56.7)."

P2 data • Acute Myelogenous Leukemia

Outcomes of Patients with IDH1/2 Mutated Accelerated/Blast-Phase Myeloproliferative Neoplasms in the Era of IDH Inhibitors (ASH 2024) - "The IDH1 inhibitors ivosidenib and olutasidenib and the IDH2 inhibitor enasidenib are approved for use in acute myeloid leukemia (AML) and reports have demonstrated the efficacy of IDH inhibition in IDH1/2-mutated MPN AP/BP (Patel et al, BJH 2020; Chifotides et al, Blood Adv 2020, Gangat et al, BJH 2023)...For hypomethylating agent (HMA) + venetoclax (VEN)-based therapy (HMA+VEN) (n=9) ORR was 67% (2 CR, 4 CRi), for HMA-based therapy (HMA monotherapy or HMA+JAKi) (n=8) ORR was 50% (3 CRi, 1 PR), and for IDH inhibitor (IDHi)-based (HMA+IDHi or IDHi), (n=6) the ORR was 33% (1 CRi, 1 PR)...There was no significant difference in mOS based on frontline treatment, although mOS was numerically longest in the HMA-based and IDH inhibitor based groups. IDH inhibitors also retained efficacy in the R/R setting with an ALR-C/ALR-P rate of 50% and mOS of 1.1 years."

Clinical • Acute Myelogenous Leukemia • Essential Thrombocythemia • Hematological Disorders • Hematological Malignancies • Leukemia • Myelofibrosis • Myeloproliferative Neoplasm • Oncology • Polycythemia Vera • Thrombocytosis • ASXL1 • CALR • DNMT3A • IDH1 • IDH2 • JAK2 • SRSF2 • TET2 • TP53

Phase 1b/2 Study of Decitabine and Venetoclax in Combination with the Targeted Mutant IDH1 Inhibitor Olutasidenib for Patients with Relapsed/Refractory AML, High Risk MDS, or Newly Diagnosed AML Not Eligible for Chemotherapy with an IDH1 Mutation (ASH 2024) - "Preliminary outcomes of "IDH1 triplet regimens" including azacitidine, venetoclax and ivosidenib and oral decitabine/cedazuridine, venetoclax and ivosidenib, have confirmed IDH1-triplet regimens are well tolerated and highly effective for both newly diagnosed (ND) and relapsed/refractory (R/R) patients with IDH1 mutated AML and high-risk MDS. Key secondary objectives are to determine composite remission rate (CRc; CR, CRh, and CRi) and overall response rate (ORR; CR, CRh, CRi, MLFS and PR) as well as the duration of response, event-free survival, and overall survival along with evaluation of occurrence of minimal residual disease (MRD) negative status by multiparameter flow cytometry and molecular evaluation. In addition, characterization of the PK profiles of VEN and OLUTA in plasma samples will be performed in the Phase 1b portion."

Clinical • Combination therapy • P1/2 data • Acute Myelogenous Leukemia • Hematological Malignancies • Myelodysplastic Syndrome • Oncology • IDH1

Outcomes of Patients with IDH1-Mutated Myeloid Neoplasms Treated with Olutasidenib (ASH 2024) - "Most of the patients were treated on clinical trials with olutasidenib +/- azacitidine, but 8 of the RR AML received standard-of-care olutasidenib...Olutasidenib was administered as monotherapy in 8 patients (6 RR AML and 2 RR MDS) and in combination with various agents, including hypomethylating agents in 13 (all 4 frontline AML/MDS, 8 RR AML, 1 RR MDS), chemotherapy in 2 (both RR AML), and lenalidomide in 1 RR AML patient with concomitant del(5q).In the AML cohort, both (100%) frontline patients responded (1 complete remission [CR], 1 CR with incomplete count recovery [CRi])...When analyzing particular subgroups in the RR AML cohort, no significant differences in overall survival were observed by secondary AML status (3.32 vs 3.43 months, p=0.877), prior ivosidenib administration (2.69 vs 4.17 months, p=0.146), or previous venetoclax exposure (2.69 vs 4.17 months, p=0.492)...Durable long-term remissions were achieved in those who achieved CR or underwent allogeneic..."

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

Olutasidenib for the Treatment of mIDH1 Acute Myeloid Leukemia in Patients Relapsed or Refractory to Hematopoietic Stem Cell Transplant, Prior mIDH1 Inhibitor, or Venetoclax (ASH 2023) - "The phase 1/2 study included 10 cohorts for a total of 335 patients, and there were patient subsets that were R/R to previous hematopoietic stem cell transplant (HSCT), ivosidenib (IVO) or venetoclax (VEN)...This descriptive analysis suggests that olutasidenib alone or in combination with azacitidine may induce complete remissions in patients with mIDH1 AML or MDS that is R/R to VEN, IVO or even HSCT. Further investigation of these difficult-to-treat subpopulations is needed. Although only a small number of patients receiving maintenance therapy were included in this analysis, the data show that maintenance of a CR and even improvement of response from CRi to CR is possible with olutasidenib."

Clinical • Acute Myelogenous Leukemia • Bone Marrow Transplantation • Hematological Malignancies • Leukemia • Myelodysplastic Syndrome • Oncology • Transplantation • IDH1

The Argument for Targeted Therapy in IDH1 Mutated Myelodysplastic Syndromes (MDS): Poor Outcomes Post-Hypomethylating Agent Failure in Higher Risk MDS and Reduced Leukemia Free Survival in Lower Risk MDS (ASH 2024) - "The upfront HMA regimens included azacitidine (61.5%), decitabine (14.4%), oral decitabine (1.5%), HMA with venetoclax (11.1%), and other HMA combinations (11.5%)...Four IDH1-MT pts received IDH1 inhibitors post-HMA failure (ivosidenib, n=3; olutasidenib, n=1), showing a significantly improved median OS of 28.6 months compared to 5.1 months for those who did not receive IDH1 inhibitors (n=28) (p<0.05)...In IDH1-MT LR-MDS, a higher rate of AML transformation and worse LFS are observed. Our data provide the historical outcomes for IDH1-MT HR-MDS post-HMA and provide rational to target IDH1 mutation among LR-MDS given high rate of leukemia transformation."

Acute Myelogenous Leukemia • Hematological Disorders • Hematological Malignancies • Leukemia • Leukopenia • Myelodysplastic Syndrome • Neutropenia • Oncology • ASXL1 • DNMT3A • EZH2 • IDH1 • RUNX1 • SRSF2

Treatment Patterns and Outcomes of Olutasidenib in Patients with Relapsed/Refractory (R/R) Mutated IDH1 Acute Myeloid Leukemia (AML) in the Real World (ASH 2024) - "Most (78%) patients had prior treatment with venetoclax, mostly in combination with a hypomethylating agent or a targeted therapy. While almost 40% of patients received olutasidenib as a fourth or later regimen in this study, results from the pivotal study showed a higher response rate for patients having received 1-2 prior therapies vs 3+, suggesting a case for treating earlier. Previous treatment with ivosidenib did not appear to adversely impact treatment duration with olutasidenib suggesting that olutasidenib has comparable clinical utility for patients with and without prior ivosidenib exposure."

Clinical • Real-world • Real-world evidence • Acute Myelogenous Leukemia • Hematological Malignancies • Leukemia • Oncology • IDH1