BioMarin Pharmaceuticals is betting that the third late-stage trial will be the charm for Riquent, La Jolla Pharmaceutical's experimental therapy for lupus. BioMarin has agreed to pay up to $289 million for Riquent, which has twice failed to hit key primary endpoints. La Jolla will get $15 million in an up-front fee and equity, up to $147.5 million in regulatory and clinical milestones and up to $125 million for sales goals.
BioMarin Chief Medical Officer Emil Kakkis told analysts this morning that despite the two earlier clinical failures, the developer feels the ongoing Phase III trial has a "good chance" of success. And BioMarin's Jean-Jacques Bienaime said that Riquent stands a good chance of becoming the company's biggest product. Data from the trial is expected in the second half of this year, and if it is approved, Riquent would become the first new lupus therapy OK'd in 45 years.
The licensing deal provides joint commercialization rights in the U.S. with BioMarin gaining the rights to Europe. Investors liked the deal, despite the troubled clinical history. BioMarin shares were boosted by the news and La Jolla's stock doubled to $2 a share.
"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," said Bienaime. "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."
- check out the BioMarin release