Joseph M. Scandura, MD, PhD

Joseph M. Scandura, MD, PhD, Weill Cornell Medicine, provides an overview on the research of pelabresib (CPI-0610) which will be discussed at the 2022 American Society of Hematology (ASH) Annual Meeting.

Pelabresib is an oral, small-molecule, investigational BET inhibitor. The agent works by downregulating NF-κB signaling along with other genes involved in myelofibrosis disease pathways.

According to Scandura, 3 presentations at ASH this year will provide updated information on pelabresib, including it in combination with ruxolitinib (Jakafi) and its potential impact on the long-term outcomes for patients with myelofibrosis.

There's 3 oral presentations related to pelabresib at ASH this year. John Mascarenhas, MD, is presenting an update of the long-term follow-up of patients treated with the combination of ruxolitinib with pelabresib, ruxolitinib being a first-in-class and standard of care JAK inhibitor. What he's showing are the remarkable early responses that have been observed and are being maintained through the most recent follow-up. Then, Claire Harrison, MD, FRCP, FRCPat, is presenting data on another arm of the phase 2 study where in pelabresib was added to ruxolitinib in people who are not achieving an adequate response.

What I'm presenting is an update on the translational work related to pelabresib trying to understand what some of these markers are, and these changes were observed in the bone marrow. From that standpoint, I think that’s where the excitement was generated with pelabresib. It's 1 of the first agents that has been able to reverse fibrosis in the marrow in a sizable percentage of people. Of course, this disease is myelofibrosis, so the idea of reducing fibrosis is appealing.

The reality of it is we don't know, besides that kind of intuitive appeal, what it means when patients respond and have reversion of fibrosis in terms of long-term outcomes or in terms of what that goes along with our patients feeling better. [Those are] some of the other measures of response correlating with the marrow response. That's what I'm presenting, a more recent update of some of those correlations.

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Joseph M. Scandura, MD, PhD, provides an overview on the research of pelabresib which will be discussed at the 2022 American Society of Hematology Annual Meeting.

Joseph M. Scandura, MD, PhD | Authors

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Updated Data of Pelabresib in Myelofibrosis