PPMD 2019 – Fibrogen muscles in on DMD chase

Early data have given Fibrogen’s pamrevlumab a path forward in Duchenne muscular dystrophy (DMD). The company is chiefly studying the project, a monoclonal antibody against connective tissue growth factor, in idiopathic pulmonary fibrosis and pancreatic cancer, but it could also combat the fibrosis that leads to declines in lung and cardiac function in DMD. Preliminary data from a single-arm, 21-patient phase II trial, reported on Friday at the Parent Project Muscular Dystrophy (PPMD) meeting, have at least done nothing to scupper this theory. Fibrogen did not give data on the primary endpoint, change in percent predicted annual forced vital capacity, but did say there was a less steep decline from baseline compared with that seen in natural history studies of DMD. On left ventricular ejection fraction, a secondary endpoint, the group reported a 0.29% increase from baseline with pamrevlumab in the intent-to-treat population, versus a decline of 0.82% per year in natural history studies. The lack of a placebo arm makes the data difficult to interpret, but a decent safety profile means this avenue is worth exploring. Still, Fibrogen will still need to carry out a randomised placebo-controlled trial in order to move pamrevlumab forward in DMD.  

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