What question did this study set out to answer?

To determine the recommended Phase 2 dose, safety profile, and clinical efficacy of molibresib monotherapy in patients with relapsed or refractory hematological malignancies.

November 9, 2022Clinical Cancer ResearchOpen Access

A Phase I/II Open-Label Study of Molibresib for the Treatment of Relapsed/Refractory Hematologic Malignancies

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Authors

MDMark A. DawsonThe University of Melbourne GBGautam BorthakurThe University of Texas MD Anderson Cancer Center BHBrian J.P. HuntlyWellcome/MRC Cambridge Stem Cell Institute

Discussion

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Implication

Randomized trial reveals modest antitumor activity but dose-limiting toxicities of molibresib in hematologic malignancies, highlighting potential for combination therapy.

Key Points

To determine the recommended Phase 2 dose, safety profile, and clinical efficacy of molibresib monotherapy in patients with relapsed or refractory hematological malignancies.
Open-label, two-part Phase I/II clinical trial (NCT01943851) enrolling 111 patients across dose-escalation (Part 1, n=87) and dose-expansion (Part 2, n=24) cohorts.
Part 1 evaluated molibresib in acute myeloid leukemia, non-Hodgkin lymphoma, and multiple myeloma to establish the recommended Phase 2 dose.
Part 2 evaluated safety and objective response rate at the recommended Phase 2 dose in relapsed/refractory myelodysplastic syndrome and cutaneous T-cell lymphoma.
Recommended Phase 2 doses were established at 75 mg daily for myelodysplastic syndrome and 60 mg daily for cutaneous T-cell lymphoma.
The objective response rate was 10% (95% CI, 4.8–18.7) in Part 1, 25% (95% CI, 7.3–52.4) in Part 2, and 13% (95% CI, 6.9–20.6) across the entire study population, comprising 6 complete and 7 partial responses.
Grade 3 or higher adverse events were prominent, most notably thrombocytopenia (37%), anemia (15%), and febrile neutropenia (15%).

Cite This Study

Dawson et al. (2022) studied this question.

synapsesocial.com/papers/6a719b57b5c1fe5ca9dfde96 https://doi.org/10.1158/1078-0432.ccr-22-1284

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Implication

Randomized trial reveals modest antitumor activity but dose-limiting toxicities of molibresib in hematologic malignancies, highlighting potential for combination therapy.

A Phase I/II Open-Label Study of Molibresib for the Treatment of Relapsed/Refractory Hematologic Malignancies

Discussion

Member takes

Implication

Key Points

Cite This Study

A Phase I/II Open-Label Study of Molibresib for the Treatment of Relapsed/Refractory Hematologic Malignancies

Discussion

Member takes

Implication

Key Points

Cite This Study

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