Merck Serono Announces Positive Results From Phase III Diet Study of Phenoptin for Phenylketonuria
January 16 2007 - 9:30AM
PR Newswire (US)
Phenoptin Significantly Increases Phenylalanine Tolerance in
BH4-Responsive Patients; All Pre-Specified Endpoints Met GENEVA,
Switzerland, January 16 /PRNewswire-FirstCall/ -- Merck Serono
(virt-x: SEO and NYSE: SRA) today announced positive results from
the Phase III diet study of Phenoptin(TM) (sapropterin
dihydrochloride), in combination with diet, in 4-12 year old
patients. Phenoptin is an investigational oral small molecule,
being developed in partnership with BioMarin, for the treatment of
patients with phenylketonuria (PKU), who have elevated
phenylalanine (Phe) levels. The results show that all pre-specified
efficacy and safety endpoints of the double-blind,
placebo-controlled study were met. Phenoptin treatment caused a
significant increase in phenylalanine tolerance as well as a
reduction in blood phenylalanine levels. In addition, the data
showed that Phenoptin was well tolerated in younger PKU patients
who were under dietary control. Dr. Harvey Levy, Professor of
Pediatrics at Harvard Medical School and Senior Associate in
Medicine and Genetics at Children's Hospital Boston, stated, "This
is the first time a controlled study has demonstrated the potential
of tetrahydrobiopterin, or 6R-BH4, to liberalize diet in PKU
patients. If approved, Phenoptin offers patients who respond to BH4
a real opportunity to relax their diets and the possibility to
perhaps reduce or even eliminate the need for nutritional
supplementation from medical food." Key findings from the study: -
In the primary endpoint, Phenoptin enabled a mean increase of 20.9
mg/kg/day in Phe supplementation for those patients on Phenoptin (p