Increased levels of TGF-β1 were detected in serum, plasma and BM and positively correlated
with both grade of BMF and extent of leukemic cell infiltration in the marrow. TGF-β likely
plays a dual role in promoting myelofibrosis and myeloproliferation, both of which are the
bone marrow morphologic hallmark of MF. AVID200 is a drug that targets TGF-β1 and TGF-β3. The
study team hypothesizes that inhibiting the TGF-β signaling pathway in MF will decrease the
fibrogenic stimuli leading to myelofibrosis and concomitantly interrupt myeloproliferation
and restore normal hematopoiesis.
This is a first in human, open-label, multicenter, Phase I/Ib trial of AVID200. Patients must
have intermediate-2 or higher primary myelofibrosis (PMF), post-essential thrombocythemia or
polycythemia-vera related MF (Post ET/PV MF). This study will enroll up to 24 patients.
AVID200 is delivered by IV infusion on day 1 of each 3 week cycle.
Phase:
Phase 1
Details
Lead Sponsor:
Icahn School of Medicine at Mount Sinai John Mascarenhas
Collaborators:
Formation Biologics Myeloproliferative Neoplasm Research Consortium (MPN-RC) National Cancer Institute (NCI)