BioMarin, Serono release Phenoptin data in three clinical studies

By Elaine Rigoli

Tampa, Fla., Sept. 12 - BioMarin Pharmaceutical, Inc. and Serono released preliminary data from clinical studies of Phenoptin (sapropterin dihydrochloride), an investigational oral small-molecule therapeutic for the treatment of phenylketonuria (PKU):

In the first study, ASHG Program No. 57, results from the phase 3, double-blind, placebo-controlled clinical study of Phenoptin (commonly referred to as 6R-BH4, or BH4) in patients with elevated blood phenylalanine (Phe) levels demonstrated a statistically significant reduction at six weeks in blood Phe levels in patients receiving 10 mg/kg/day of phenoptin, compared with those receiving a placebo.

In the second study, ASHG Program No. 2332/C, results from the phase 2 screening study conducted to identify appropriate patients for inclusion in the phase 3, double-blind, placebo-controlled study of PKU, demonstrate that Phenoptin was well-tolerated and rapidly reduced blood Phe levels by varying degrees in PKU patients across the complete spectrum of PKU phenotypes.

In a final study, ASHG Program No. 1415/A, results from a BioMarin-sponsored meta-analysis conducted to determine whether blood Phe levels can be used as a predictive biomarker of clinical outcomes for the development of new PKU treatments confirm findings from previous studies. Those studies reported a significant correlation between concurrent and long-term blood Phe levels and intelligence quotient in individuals with PKU.

BioMarin and Serono are biopharmaceutical companies based in Novato, Calif., and Geneva, Switzerland, respectively.

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