Rare Disease Report

Pre-Clinical Data Regarding BGB601 Indicates Efficacy for Fibrosis, IPF, and NASH

APRIL 13, 2018
Krista Rossi
This morning, BerGenBio ASA announced positive pre-clinical data regarding its product, BGB601, for the treatment of fibrosis, idiopathic pulmonary fibrosis (IPF), and non-alcoholic steatohepatitis (NASH).

BGBC601 (ADCT-601) is an antibody drug conjugate (ADC) drug that is composed of a humanized IgG1 antibody against human AXL.

According to pre-clinical data results, selective AXL inhibition counteracts the progression of aggressive fibrosis in the lung and liver.

The data, published in the American Journal of Respiratory and Critical Care Medicine (AJRCCM), found that selective inhibition of AXL using bemcentinib (BGB324) impacted IPF fibroblast functions and the development of fibrosis in pre-clinical models of IPF. In the study, Milena Espindola and colleagues from Cedars-Sinai in Los Angeles, CA, conclude that Gas6/TAM receptor activity contributes to the activation of pulmonary fibroblasts in IPF suggesting that targeting this RTK pathway might be an effective anti-fibrotic strategy in this disease.2

Also, in the study, it was found that the potential of using AXL levels as a biomarker to identify patients with a poor prognosis who may respond to treatment with an AXL inhibitor and to enrich populations in future clinical trials. 

Data pertaining to AXL targeting in NASH will be presented by Anna Tutusaus et al. at the European Association for the study of the Liver (EAL) on Friday, April 13th. Data regarding the anti-tumor activity of BGB601 in human cancer cell lines and animal models from Lorenzo Zammarchi et al. will be presented at the at the American Association of Cancer Research (AACR) Annual Meeting April 16, 2018.

Richard Godfrey, CEO if BerGenBio, stated his optimism regarding the data. "This patient data and pre-clinical findings in IPF and cirrhotic NASH are very compelling and suggest that selective AXL inhibition may have potential as a new approach to treating life-threatening fibrotic diseases.”

“While our focus remains clearly on completing our phase II clinical program with bemcentinib and to establish proof of concept for its role as a cornerstone of cancer therapy, we are intrigued by the possibility of therapeutic benefit from our AXL inhibitors in fibrotic diseases. We will continue to support this research and look forward to integrating it into our pipeline development strategies. Furthermore, we are encouraged by pre-clinical data supporting the advancement of BGB601, our out-licensed AXL ADC drug candidate, towards the clinic."

Pre-clinical data of BerGenBio's out-licensed AXL ADC candidate BGB601 has been accepted for presentation at the AACR Annual Meeting 2018.

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  1. Promising Pre-clinical Data Supporting BerGenBio's Pipeline to be Published and Presented at Upcoming Leading Conferences. Prnewswire.com. https://www.prnewswire.com/news-releases/promising-pre-clinical-data-supporting-bergenbios-pipeline-to-be-published-and-presented-at-upcoming-leading-conferences-300629447.html. Accessed 13, Apr. 2018.
  2.   Targeting of TAM Receptors Amerliorates Fibrotic Mechanisms in Idiopathic Pulmonary Fibrosis. Atsjournals.org. https://www.atsjournals.org/doi/abs/10.1164/rccm.201707-1519OC. Accessed 13, Apr. 2018.

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