NOVATO, California—BioMarin Pharmaceutical Inc, which recently paid $15 million upfront to La Jolla Pharmaceutical for a piece of the experimental lupus nephritis drug Riquent^® (abetimus sodium), has axed further development of the drug. The company announced Feb. 12 that the first interim efficacy analysis (IEA) of the third major clinical test of the drug came up empty, as did 2 prior studies.

In the Phase 3 ASPEN (Abetimus Sodium in Patients with a History of Lupus Nephritis) trial, according to a company statement, “the Independent Data Monitoring Board (DMB) determined that the continuation of the trial is futile.”

BioMarin and partner La Jolla Pharmaceutical stopped the study and will unblind the data and evaluate all of the clinical results including secondary endpoints. These include disease activity indices and proteinuria.

"While the results of this first interim efficacy analysis are obviously disappointing, we decided to take a calculated risk on this program and with the carefully staged agreement, we have spent a total of $15 million, a relatively modest amount for a late Phase 3 asset addressing a large market opportunity," said Jean-Jacques Bienaime, Chief Executive Officer of BioMarin. "We remain dedicated to advancing our development pipeline by making investments in future growth and continue to look for attractive late-stage in-licensing or acquisition opportunities."

The Riquent Phase 3 program was the subject of a special protocol assessment and had fast track designation from the Food and Drug Administration and Orphan Drug designation in the United States and Europe.