Table 2.

Clinical trials evaluating emavusertib in myeloid malignancies.

Clinical Trials.gov Identifier	Diseases Evaluated	Median Age, range	Median # of Prior Treatments, range	Any Grade Treatment Related Adverse Events	≥Grade 3 Treatment Related Adverse Events	Response	RP2D
NCT04278768	Phase I (Emavusertib Monotherapy) AML MDS N=49	NR	2 (1-5)	NR	No Grade 4 or 5 treatment-related adverse events were reported Reversible, manageable Grade 3 rhabdomyolysis occurred in 1/26 (4%) patients at 300 mg BID, 2/17 (12%) at 400 mg BID, and 1/3 (33%) at 500 mg BID	In the 5 evaluable AML patients with spliceosome mutations, 40% reached CR/CRh (1 CR, 1 CRh) In the 7 spliceosome-mutated high risk-MDS patients, 57% reached marrow CR Among the 29 patients without SF3B1/U2AF1/FLT3 mutations, 1 reached CR and 2 PR	300mg BID

AML, acute myeloid leukemia; MDS, myelodysplastic syndrome; NR, not reported; ORR, overall response rate; CR, complete response; CRh, complete response with partial hematologic recovery; PR, partial response; RP2D, recommended phase II dose.

#, Number.