On February 14, 2019 Forbius, a clinical-stage company that develops biologics for the treatment of cancer and fibrosis, reported a collaboration agreement with the Icahn School of Medicine at Mount Sinai and the Myeloproliferative Neoplasm Research Consortium (MPN-RC) (Press release, Forbius, FEB 14, 2019, View Source [SID1234533334]). This collaboration will launch an investigator-initiated trial (IIT) evaluating AVID200, a highly potent and isoform-selective TGF-beta inhibitor, as a potential treatment for myelofibrosis (MF). The Phase 1/2 clinical trial will be sponsored by the MPN-RC with NIH grant support and is expected to start during Q1 2019.
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"We have demonstrated that blocking TGF-beta signaling reverses bone marrow fibrosis and restores hematopoiesis in preclinical models. We believe that selective TGF-beta inhibition by AVID200 could address the underlying cause of bone marrow failure and become the first disease-modifying therapy in MF. Our consortium is eager to commence evaluation of AVID200 in the upcoming clinical study," 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.
Forbius is evaluating the immuno-oncology and anti-fibrotic effects of AVID200 in Phase 1 trials, including solid tumors and systemic sclerosis. Additionally, Forbius is expanding the AVID200 clinical development program by supporting IITs through the provision of drug, scientific input, and collaboration on the conduct of translational studies. Additional details pertaining to the prospective IIT in MF will be disclosed in due course.
About the Myeloproliferative Neoplasm Research Consortium (MPN-RC)
The MPN-RC was founded in 2006 and is the only independent, multi-center, international consortium of scientists and clinicians that is 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 AVID200
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 in MF and other fibrotic diseases.
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.