Forbius: First Patient Dosed in a Phase 1b Myelofibrosis Trial of AVID200, a Novel TGF-beta 1 & 3 Inhibitor

  • Investigator-initiated trial to evaluate AVID200’s safety,
    anti-fibrotic activity, and ability to restore hematopoiesis in
    patients with myelofibrosis
  • AVID200 is a rationally designed, highly potent inhibitor of TGF-beta
    1 & 3, the principal drivers of fibrosis in myelofibrosis and other
    fibrotic diseases
  • AVID200 spares TGF-beta 2, the isoform that promotes hematopoiesis and
    normal cardiac function

AUSTIN, Texas & MONTREAL–(BUSINESS WIRE)–lt;a href="https://twitter.com/hashtag/AVID200?src=hash" target="_blank"gt;#AVID200lt;/agt;–Forbius, a clinical-stage company that develops novel biologics for the
treatment of fibrosis and cancer, announced today 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.

About AVID200 and the AVID200-02 Trial

AVID200 is an isoform-selective and highly potent inhibitor of TGF-beta
1 & 3, the two principal pro-fibrotic TGF-beta isoforms. These TGF-beta
isoforms are central regulators in the pathogenesis and progression of
fibrotic diseases, including MF (Chagraoui
et al., 2002
). AVID200 was rationally designed to be minimally
active against TGF-beta 2, which is a promoter of hematopoiesis and
normal cardiac function. This optimal selectivity positions AVID200 to
be an effective and well-tolerated therapeutic for MF and other fibrotic

AVID200-02 (NCT03895112)
is an investigator-initiated, open-label, multicenter, Phase 1b trial to
evaluate the safety and anti-fibrotic activity of AVID200, as well as
its ability to restore normal hematopoiesis in patients with MF.

About the Myeloproliferative Neoplasm Research Consortium (MPN-RC)

The MPN-RC was founded in 2006 and is the only independent, multicenter,
international consortium of scientists and clinicians dedicated to
developing novel therapeutic strategies for MF and other
myeloproliferative neoplasms (MPN). The MPN-RC is funded by the NIH to
conduct clinical trials based on the most promising preclinical MPN
research. The goal of the consortium is to adapt quickly in response to
scientific advances and a changing clinical landscape, in order to
develop effective therapeutics for MPN patients.

About Myelofibrosis (MF)

MF is a rare, life-threatening blood cancer characterized by progressive
bone marrow fibrosis, which causes ineffective hematopoiesis.
Approximately 30,000 people in the US alone are affected by this
disease. Currently, there are no approved therapies targeting the
underlying bone marrow fibrosis available to MF patients.

About Forbius: Targeting TGF-beta and EGFR Pathways in Fibrosis and

Forbius is a clinical-stage protein engineering company that designs and
develops novel biologics for the treatment of fibrosis and cancer. Our
current focus is the development of agents targeting the transforming
growth factor-beta (TGF-beta) and epidermal growth factor receptor
(EGFR) pathways.

For more information, please visit www.forbius.com.


Ilia A. Tikhomirov
[email protected]

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