MPN-RC 118 AVID200 in Myelofibrosis

Summary

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.

Timeline

Start

2019-02-15

Primary completion

2022-05-16

Completion

2022-05-16

Publications

• Varricchio L, Mosoyan G, Elghaity-Beckley S, Mia MB, Handa S, Salib C, Mascarenhas J, Hoffman R. Thrombocytopenia in myelofibrosis is characterized by inflammatory megakaryocytes with reduced G6B expression. Blood. 2025 Sep 25;146(13):1612-1624. doi: 10.1182/blood.2024027363.

Drugs

Indications