Dosing of the first patient in the phase I/II trial evaluating PFL-002/VERT-002, a targeted therapy in oncology

PFL-002/VERT-002 is a monoclonal antibody with a novel and differentiated mechanism of action, acting as a degrader of c-MET, with the potential to be a best-in-class treatment for cancer driven by MET alterations 

Castres, France, October 24, 2024 – Pierre Fabre Laboratories announced today that the first patient has been dosed with PFL-002/VERT-002, a monoclonal antibody acting as a degrader of c-MET, in a phase I/II first-in-human dose-escalation, dose-optimization and dose-expansion trial, for patients with Non-Small Cell Lung Cancer (NSCLC) harbouring MET alterations.

The PFL-002/VERT-002 phase I/II trial is an open label, multi-centre study that aims to assess the safety, tolerability, pharmacokinetics, pharmacodynamics and preliminary clinical efficacy of PFL-002/VERT-002, as a monotherapy for patients with MET-dependent tumors, including those emerging with acquired resistance to other treatments.

Non-small cell lung cancer (NSCLC) is the most prevalent form of lung cancer, accounting for approximately 85% of newly diagnosed lung cancer cases, and MET, also known as hepatocyte growth factor receptor (HGFR), is an oncogene driver in subsets of patients suffering from NSCLC.^1-4  MET exon 14 skipping mutation and MET amplification are found as primary oncogenic drivers and MET amplification as a resistance mechanism to selected targeted therapies.  

“PFL-002/VERT-002 targets a clinically validated oncogenic driver with a unique and differentiated mechanism of action, triggering the degradation of the c-MET oncogene. Thus, it provides the opportunity to test a novel therapeutic approach for patients with MET driven tumors. We are looking forward to collaborating with the investigators participating in the first-in-human trial to assess the safety and efficacy of this new agent.” said Francesco Hofmann, Head of Research and Development for Medical Care at Pierre Fabre Laboratories.

MET TKIs (Tyrosine Kinase Inhibitors) are approved therapeutic options for the treatment of NSCLC with MET exon 14 skipping mutation. However, there is no approved MET targeted therapy for patients presenting with MET amplification.  

PFL-002/VERT-002 is a monoclonal antibody developed by Vertical Bio, offering a unique and differentiating mechanism of action, acting as a degrader of c-MET, a known disease driver in patients with solid tumors, including non-small cell lung cancer (NSCLC) presenting mutations or amplification of MET. The antibody has been optimized preclinically by Vertical Bio, which has been acquired by Pierre Fabre Laboratories.

PFL-002/VERT-002 is a potential best-in-class therapeutic option for patients with MET alterations, including resistance settings. 

Pierre Fabre Laboratories has expanded its efforts in precision oncology by adding several assets to its R&D pipeline. In partnership with Scorpion Therapeutics, PFL-241/STX-241 and PFL-721/STX-721, two mutant-selective EGFR inhibitors, will be developed for the treatment of EGFR-driven non-small cell lung cancer (NSCLC) patients. Through the acquisition of Vertical Bio, PFL-002/VERT-002 will undergo clinical testing in solid tumours driven by MET genetic alterations. More recently, the pan-RAF inhibitor exarafenib was acquired from Kinnate Biopharma with the aim to expand targeted therapy options for RAS/RAF-driven solid tumours. These new additions to its clinical development portfolio complement Pierre Fabre Laboratories’ existing precision oncology portfolio targeting BRAF, MEK, HER2, with encorafenib, binimetinib and neratinib, respectively.