Major Research Findings

Olutasidenib is an oral, small-molecule inhibitor of mutant isocitrate dehydrogenase 1 (IDH1), which is a potent and selective inhibitor. 1 A phase 1/2 trial, phase 1, aimed to assess the safety, pharmacokinetics, pharmacodynamics, and clinical activity of olutasidenib in patients with acute myeloid leukaemia or myelodysplastic syndrome, harbouring mutant IDH1, as monotherapy or in combination with azacitidine. 1 Olutasidenib is one of twelve new drug therapies approved by the FDA in 2022 for managing varying cancers. 3 Olutasidenib has been approved by the Food and Drug Administration (FDA) as a treatment for acute myeloid leukemia (AML) and myelodysplastic syndromes (MDS) with IDH1 mutations. Combination therapies with olutasidenib may show stronger immunosuppressive effects compared to monotherapy. 2 Caution is required with combination therapy such as venetoclax plus hypomethylating agents, gemtuzumab ozogamicin plus cytotoxic drugs or midostaurin added to conventional AML chemotherapy. 2

Benefits and Risks

Benefit Summary

Olutasidenib provides a new treatment option for patients with AML and MDS with IDH1 mutations. 1 It is convenient for patients because it can be taken orally. 1 Olutasidenib has been shown to be safe and effective in patients with AML and MDS in phase 1 of a phase 1/2 trial, as monotherapy or in combination with azacitidine. 1

Risk Summary

Caution is required when using olutasidenib in combination with other targeted drugs or biotherapies. 2 Combination therapies with olutasidenib may show stronger immunosuppressive effects compared to monotherapy. 2

Comparison of Studies

Similarities in Studies

Multiple studies have shown that olutasidenib is a promising treatment option for patients with AML and MDS with IDH1 mutations. 1 These studies have been conducted using different study designs and endpoints to evaluate the safety and efficacy of olutasidenib. 1

Differences in Studies

It is difficult to compare the results across studies as different study designs and endpoints were used to evaluate the safety and efficacy of olutasidenib. 1

Consistency and Inconsistencies in Results

Multiple studies have shown that olutasidenib is a promising treatment option for patients with AML and MDS with IDH1 mutations. 1 However, caution is required as combination therapies with olutasidenib may show stronger immunosuppressive effects compared to monotherapy. 2

Cautions for Real-World Application

Olutasidenib provides a new treatment option for patients with AML and MDS with IDH1 mutations. 1 However, caution is required when using olutasidenib in combination with other targeted drugs or biotherapies. 2

Limitations of Current Research

Research on olutasidenib is still in its early stages and further studies are needed to evaluate its long-term safety and efficacy. 1

Future Directions for Research

Further studies are needed to evaluate the long-term safety and efficacy of olutasidenib. 1 Research on combination therapies with olutasidenib and other targeted drugs or biotherapies is also needed. 2

Conclusion

Olutasidenib has the potential to provide a new treatment option for patients with AML and MDS with IDH1 mutations. 1 However, further studies are needed to evaluate its safety and efficacy. 1