NOVATO, Calif., June 9 /PRNewswire-FirstCall/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) today announced results from the Phase 1 clinical study of PEG-PAL (PEGylated recombinant phenylalanine ammonia lyase) for the treatment of phenylketonuria (PKU). The company expects to initiate the Phase 2 clinical study in late June or early July, pending institutional review board (IRB) approval from the clinical trial sites. The U.S. Food and Drug Administration (FDA) has reviewed the Phase II clinical trial protocol.
Key findings from the Phase 1 study:
- Substantial blood Phe level reductions in the range of 36% to 97% (mean of 62%) were observed in all patients in the fifth dosing cohort (0.1 mg/kg) with a mean baseline blood Phe level of 1113 umol/L. No notable blood Phe level reductions were observed in the first four dosing cohorts (0.001 to 0.03 mg/kg).
- No serious immune reactions, including hives or anaphylactoid reactions, were observed.
- Seven out of 25 patients developed late mild to moderate injection-site reactions, of which two also developed skin rashes without other symptoms and two patients developed reactions to Depo-Provera (medroxyprogesterone acetate).
Click here for graph: Correlation between blood Phe level and plasma concentration of PEG-PAL in Cohort 5.
'We are encouraged to see significant reductions in blood Phe levels in all patients in the fifth dosing cohort of the Phase 1 trial. Importantly, there were no serious reactions, and both the number and severity of the mild to moderate reactions were in-line with expectations of an enzyme of this nature,' said Hank Fuchs, M.D., Chief Medical Officer of BioMarin. 'The Phase 2 study will answer the critical questions of optimal dose, dosing regimen and tolerability.'
Phase 1 Study Design
The Phase 1 clinical trial was an open-label, multi-center study conducted in 25 PKU patients in a series of five dose-escalating cohorts ranging from 0.001 to 0.1 mg/kg, with each cohort receiving a single dose, and a 6-week follow-up period.
The primary objective of the study was to assess the safety and tolerability of single, subcutaneous injections of PEG-PAL in subjects with PKU.
