FibroGen’s antibody fails PhIII in Duchenne muscular dystrophy

FibroGen’s top antibody candidate has failed a Phase III study in Duchenne muscular dystrophy, marking a disappointing start to a string of readouts slated for the next few months.

Dubbed pamrevlumab, the drug is listed as a lead candidate for FibroGen alongside roxadustat — an anemia drug that’s run into multiple clinical and regulatory setbacks of its own. Shares $FGEN fell 9% to below $17 in pre-market trading.

The LELANTOS-1 study is the first of two Phase III studies in Duchenne to read out, and among a total of five late-stage trials that FibroGen has devised for pamrevlumab, which is designed to block a signaling and regulatory molecule called connective tissue growth factor.

LELANTOS-1 enrolled non-ambulatory patients with Duchenne who are receiving background corticosteroids. It flunked the primary endpoint, measured by the Performance of the Upper Limb 2.0 (PUL 2.0) score at week 52 compared to baseline.

The second study, LELANTOS-2, focuses on ambulatory patients; data are expected in the third quarter. FibroGen did not immediately reveal plans for the program, saying only it will present the results at a medical meeting.

Enrique Conterno

“While disappointed with these results, we look forward to sharing the data at a future medical conference to contribute towards the understanding of this devastating disease,” CEO Enrique Conterno said in a statement.

FibroGen is also testing pamrevlumab in idiopathic pulmonary fibrosis and locally advanced unresectable pancreatic cancer — and the drug received fast track and orphan designations for all three indications from the FDA.

At the most recent quarterly call, Conterno told analysts that “given these differences in disease pathology, we believe there is limited or no efficacy read-through from one of these conditions to another.”

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