NOVATO and SAN DIEGO, California—BioMarin Pharmaceuticals has bet $289 million that the third time is the charm for La Jolla Pharmaceutical's experimental lupus drug Riquent^® (abetimus sodium), which failed to reach primary endpoints in 2 previous clinical trials.

Riquent is being evaluated in the international Phase 3 ASPEN trial designed to demonstrate that the drug delays the time to renal flare and reduces proteinuria in patients with lupus renal disease. The Riquent Phase 3 program is the subject of a special protocol assessment and has fast track designation from the Food and Drug Administration and Orphan Drug designation in the United States and Europe. The first and second interim efficacy analyses are expected in the first quarter of 2009 and mid-2009, with final efficacy analysis in late 2009. Assuming good results from ASPEN, analysts expect a New Drug Application to be filed in early 2010. If approved, Riquent would be the first new drug specifically for lupus in more than 45 years.

BioMarin will pay La Jolla Pharmaceutical $15 million up-front, up to $147.5 million in regulatory and clinical milestones, and up to $125 million for sales goals.

If the Phase 3 trial is successful, the companies will share equally in all losses and profits. BioMarin CEO Jean-Jacques Bienaime said, "We are very pleased to become La Jolla Pharmaceutical's partner for Riquent. The development history of Riquent has been long and challenging, but we feel the current study addresses the shortcomings of prior studies and provides the best possible opportunity to demonstrate that Riquent reduces the frequency of renal flares in lupus nephritis patients. The ASPEN Phase 3 study is the largest clinical study ever conducted in lupus nephritis and there is increasing evidence that Riquent targets one of the most important underlying causes of kidney disease in lupus patients, antibodies to double stranded DNA.”

La Jolla expects money from the deal to significantly cover the remaining costs of the Phase 3 ASPEN study.