Rezlidhia (olutasidenib)

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.

P2, N=28, Recruiting, University of California, Davis | Not yet recruiting --> Recruiting

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.

P2, N=28, Not yet recruiting, University of California, Davis

P1, N=15, Not yet recruiting, Virginia Commonwealth University | Initiation date: Oct 2025 --> Feb 2026

P2, N=132, Not yet recruiting, National Cancer Institute (NCI) | Initiation date: Feb 2026 --> May 2026

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.

P2, N=68, Recruiting, M.D. Anderson Cancer Center | Not yet recruiting --> Recruiting

Venetoclax, an oral B-cell lymphoma-2 (BCL-2) inhibitor, has shown promise in clinical trials but requires further validation. Isocitrate dehydrogenase 1 (IDH1) inhibitors, including ivosidenib and olutasidenib, have demonstrated efficacy and tolerability, while ongoing investigations explore other novel agents like IDH2 inhibitors and FMS-like tyrosine kinase 3 (FLT3) inhibitors. By summarizing the latest advancements, this review emphasizes the importance of developing safe, effective, and personalized therapies to improve outcomes and quality of life for older patients with HR-MDS, with a focus on age-specific clinical trials.

P1, N=15, Not yet recruiting, Virginia Commonwealth University

Olutasidenib with or without azacitidine demonstrated encouraging clinical activity and tolerability in patients with higher-risk mIDH1 MDS. NCT02719574.

The drug ibrutinib serves as a standard for comparison. The approach was shown accurate throughout a range of 3.0-60.0 ng ml-1 and correlation values of (r 2) ≥ 0.999.6 replicates including olutasidenib at 4 distinct QC levels were analyzed to determine intra-assay precision and accuracy; the Coefficient of variations (CV) were reported to be 3.41% to 0.58% to 0.31% to 0.36, and the accuracy ranged from 97.40, 99.69, 99.4, and 99.16%, respectively, for LOQQC, LQC, MQC, and HQC. In a pharmacokinetic investigation using rat plasma, this strategy has proven effective.