FibroGen (FGEN) Receives FDA Clearance to Proceed with FG-3019 Study in DMD

July 27, 2015 4:19 PM EDT
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FibroGen (NASDAQ: FGEN) announced that the U.S. Food and Drug Administration (FDA) has completed its review of the Company's investigational new drug (IND) application for the study of FG-3019 in patients with Duchenne muscular dystrophy (DMD), and clinical study may proceed. FG-3019 is a fully human monoclonal antibody that inhibits the activity of connective tissue growth factor (CTGF), a common mediator of fibrotic disease. FG-3019 is currently in Phase 2 clinical studies for idiopathic pulmonary fibrosis and pancreatic cancer.

The IND enables FibroGen to initiate a multi-site Phase 2 trial exploring FG-3019 for the treatment of DMD in non-ambulatory patients, scheduled to begin later this year. This submission is the culmination of nearly 10 years of research by FibroGen and its collaborators to understand the role of CTGF in DMD. Data from these investigations, including the testing of FG-3019 in preclinical models of DMD, have appeared in several peer reviewed publications.(1) FibroGen has met with panels of international muscular dystrophy experts, including the TREAT-NMD Advisory Committee for Therapeutics, to review these findings, discuss the rationale for FG-3019 as a potential treatment for DMD, and refine the clinical trial design.(2) There has been consistent support in these panels for the advancement of FG-3019 development in DMD.

In DMD, muscle cells are damaged due to the absence of the dystrophin protein complex necessary for normal muscle fiber function. Absence of dystrophin leads to muscle damage, inflammation, fibrosis, and progressive dysfunction and weakness in the muscles of DMD patients. The extent of diminished muscle function directly correlates with the extent of intra-muscular fibrosis. The rationale for FG-3019 in DMD is based on data that show CTGF reduces the ability of damaged muscle cells to repair themselves and promotes muscle fibrosis. In preclinical studies, FG-3019 reduced muscle fibrosis and significantly improved muscle function. Separately, in clinical trials of another fibrotic condition, idiopathic pulmonary fibrosis, FG-3019 was shown to reverse fibrosis in a significant proportion of patients.

"We believe FG-3019 may have the ability to not only stabilize but also reverse the progression of fibrosis in disease," stated Frank H. Valone, MD, Chief Medical Officer of FibroGen. "If FG-3019 shows a positive effect on fibrosis in clinical studies of DMD patients, and inhibits or reverses the build-up of fibrosis in their skeletal muscle, FG-3019 could become a potential therapy for the muscle weakness and wasting associated with the disease. Whereas other investigational treatments focus on overcoming certain genetic mutations present in a subpopulation of DMD patients, FG-3019 addresses the disease process of muscle displacement by fibrosis seen in a broader population of DMD patients. We plan to initiate our first DMD trial at US sites and begin enrolling non-ambulatory subjects this year."

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