First DMD Patients Enroll in Phase 2 Study of FibroGen’s FG-3019

FibroGen, a biotechnology company with expertise in connective tissue growth factor (CTGF) and hypoxia-inducible factor (HIF) biology, recently announced enrolling the first two patients in its new, open-label, multicenter Phase 2 clinical trial to investigate the FG-3019 compound in Duchenne muscular dystrophy (DMD) patients.  The trial is currently recruiting participants and further information can be found here.

FG-3019 is a fully human monoclonal antibody raised against CTGF, a key player in fibrosis. A previous Phase 2 trial with FG-3019 in 89 patients with idiopathic pulmonary fibrosis (IPF), and an undergoing randomized placebo-controlled Phase 2 study in IPF, support further investigation of the compound’s potential to treat fibrotic disease.

The novel Phase 2 clinical trial, to be held at sites in Missouri and Ohio, will continue to investigate the therapeutic potential of FG-3019 in up to 22 non-ambulatory DMD patients (who are at least 12 years of age), specifically:

1. In preserving or improving pulmonary function in non-ambulatory DMD patients (by targeting pathways and preventing the loss of diaphragm and intercostal muscle strength).
2. In regaining DMD patients’ upper body muscle strength, allowing them to perform daily activities.
3. In slowing, preserving, or even reversing patients’ cardiomyopathy (i.e., the deterioration of the ability of the myocardium, the heart muscle, to contract, ultimately leading to heart failure).

Thomas Voit, MD, director designate, Biomedical Research Centre, Institute of Child Health, Great Ormond Street Hospital Trust, London, U.K., said in a press release, “While the fibrotic component of DMD pathology is well recognized, treatment options for attacking this aspect of the disease have been limited. This study of FG-3019, a novel anti-fibrotic monoclonal antibody, has the potential to establish fibrosis mitigation as a foundational component of comprehensive DMD therapy.”

“FG-3019, if shown to be safe and effective, has the potential to be a disease modifying therapy for DMD. By countering the activity of elevated CTGF and its harmful effects on muscle tissue, FG-3019 may be able to help DMD patients regardless of genetic subtype. We look forward to working closely with clinical trial investigators, study coordinators, and the DMD community as we advance the clinical development of FG-3019 in this devastating disease,” added Thomas Neff, chief executive officer of FibroGen.

The trial’s estimated completion date is September 2018.

DMD is a genetic disorder caused by mutations in the dystrophin gene. The alteration in the gene leads to myofiber degeneration and muscle wasting. Affecting mainly boys, symptoms usually arise between 3 to 5 years of age. By ages 9 to 12,  these patients likely have lost ambulation and are at risk of dying in their second or third decade due to respiratory or cardiac failure.