Forbius, a clinical-stage company that develops novel biologics for the treatment of fibrosis and cancer, announced that the first patient has been dosed in a Phase 1b trial assessing AVID200, a novel TGF-beta 1 & 3 inhibitor, in patients with myelofibrosis (MF). This multicenter trial is sponsored by the Icahn School of Medicine at Mount Sinai and the Myeloproliferative Neoplasm Research Consortium (MPN-RC), with the support of a peer-reviewed NIH grant.
AVID200’s novel dual mode of action in MF centers around its anti-fibrotic effects and ability to restore hematopoiesis, as demonstrated in MF patient cells and in vivo models of the disease (Varricchio et al., 2018). Notably, treatment of cells from MF patients with AVID200 promoted proliferation of normal hematopoietic progenitors while decreasing the proportion of MF malignant progenitor cells.
“This clinical trial will evaluate the ability of AVID200 to achieve the disease-modifying outcomes of reversing bone marrow fibrosis and restoring normal hematopoiesis. Preclinical data demonstrate that selective neutralization of TGF-beta 1 & 3 by AVID200 results in both of these critical outcomes. We believe that AVID200 has the potential to become the first disease-modifying treatment for MF,” commented Dr. Ronald Hoffman, founder of the MPN-RC and Director of the Myeloproliferative Disorders Research Program at the Icahn School of Medicine at Mount Sinai.
The single-arm, dose-escalation Phase 1b trial (AVID200-02; NCT03895112) plans to enroll up to 24 patients with primary MF, post-essential thrombocythemia MF, or post-polycythemia vera MF with ≥ grade 2 bone marrow fibrosis. Outcome measures include safety, response, clinical and hematological improvement, as well as assessment of the degree of bone marrow fibrosis.