Curis, Inc. announced that the U.S. Food and Drug Administration (FDA) has placed a partial clinical hold on the Company's TakeAim Leukemia Phase 1/2a study (NCT04278768). The TakeAim Leukemia study is a Phase 1/2a open-label, single arm dose escalation and expansion study of orally-administered emavusertib (CA-4948) as monotherapy and in combination with azacitidine or venetoclax in patients with relapsed or refractory (R/R) acute myeloid leukemia (AML) or high-risk myelodysplastic syndrome (MDS).
While the partial hold is in place, no new patients will be enrolled in the study, and current study participants benefitting from treatment may continue to be treated with emavusertib at doses of 300mg BID or lower.
With the partial hold, the FDA is requesting additional data from the study, including data related to the death of a R/R AML patient who experienced, among several conditions, rhabdomyolysis, which has previously been identified as a dose-limiting toxicity of emavusertib. Additionally, the FDA is requesting safety, efficacy, and other data, including data related to rhabdomyolysis and the Company's determination of the Recommended Phase 2 Dose for emavusertib in this study.
"We are committed to ensuring the safety of patients in our studies and to working collaboratively with the FDA to develop therapies that meaningfully improve and extend patients' lives," said James Dentzer, Chief Executive Officer of Curis. "Given the clinical profile of emavusertib observed to date, we are hopeful that the study can be resumed soon, after appropriate review. We continue to be confident in the potential of emavusertib to address the high unmet need of patients with AML or MDS."
Curis expects to provide updated guidance on the timing of discussing the potential for a rapid registrational path for emavusertib with the FDA after the partial clinical hold is resolved and the related impact on the trial can be determined.
While this partial hold does not affect the TakeAim Lymphoma study (NCT03328078), a Phase 1/2 open-label dose escalating clinical trial investigating emavusertib in patients with B-cell malignancies, the Company has decided to pause enrollment of new patients in that study as well, pending resolution of the partial clinical hold in the TakeAim Leukemia study.
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