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news.BioMarin Pharmaceutical, a biopharmaceutical company, has announced that in the first interim efficacy analysis for the Riquent Phase III Aspen trial, the Independent Data Monitoring Board determined that the continuation of the trial is futile.
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BioMarin and partner La Jolla Pharmaceutical have decided to stop the study, unblind the data and evaluate all of the clinical results including secondary endpoints such as systemic lupus erythematosus disease activity indices and proteinuria.
Jean-Jacques Bienaime, CEO of BioMarin, said: 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 III asset addressing a large market opportunity.
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.
BioMarin develops and commercializes innovative biopharmaceuticals for serious diseases and medical conditions. The company’s product portfolio comprises three approved products and multiple clinical and preclinical product candidates.
Approved products include Naglazyme; Aldurazyme; and Kuvan. Other product candidates include 6R-BH4 for cardiovascular indications, and PEG-PAL (PEGylated recombinant phenylalanine ammonia lyase).