La Jolla gets new injection of life

La Jolla Pharmaceutical might have finally caught a break. Positive interim data from a phase II trial of its subcutaneous synthetic hepcidin, LJPC-401, have sent the group’s stock up 92%, albeit from a very low base. The LJ401-HH01 study was testing LJPC-401 in hereditary haemochromatosis, which is caused by deficiencies in hepcidin, a hormone that regulates iron absorption. So perhaps it was no surprise that replacing hepcidin appeared to work: 26 subjects in the 60-patient trial were evaluable at 16 weeks, and there was a 42% mean reduction in transferrin saturation in the LJPC-401 group versus a 6% drop with placebo. Patients receiving LJPC-401 also had fewer phlebotomy procedures, a blood removal process that is the current standard for hereditary haemochromatosis. A bigger challenge for La Jolla will be showing a benefit in beta-thalassaemia, another disorder linked with iron overload, where the project is in a phase II trial set to read out in mid-2020. Here La Jolla could go up against Protagonist Therapeutics, which is set to report phase II data soon with its hepcidin mimetic, PTG-300. After La Jolla's 2009 lupus blowup and the recent disappointing Giapreza launch, the group still has a long road to recovery.