BioMarin Announces Decision To Start Phase 3 Program For PEG-PAL In 2Q 2013

Preliminary Phase 2 Results Indicate Convenient and Accelerated Dosing Regimen Identified

Conference Call and Webcast to be Held Today at 5:00 p.m. ET

NOVATO, Calif., Sept. 26, 2012 (GLOBE NEWSWIRE) -- BioMarin Pharmaceutical Inc. (Nasdaq:BMRN) announced today preliminary results from the Phase 2 program of PEG-PAL (PEGylated recombinant Phenylalanine Ammonia Lyase) for the treatment of phenylketonuria (PKU) demonstrating long-term retention, tolerability and providing evidence of efficacy. Based on these results, the company expects to start a pivotal Phase 3 study in the second quarter of 2013, following an anticipated end of Phase 2 meeting with the FDA in the first quarter of 2013.

A total of 56 patients older than 16 years have been enrolled into the four main studies that compose the Phase 2 program. During the studies, patients were not required to adhere to restricted dietary intake of phenylalanine (Phe). Mean baseline blood Phe levels were 1,360 umol/L, consistent with a diagnosis of severe PKU. Of the 25 patients who have been treated with PEG-PAL for at least one year, blood Phe measures were on average 68% lower than pre-treatment baseline levels. While the NIH guideline for control of blood Phe in adults recommends patients maintain a blood Phe of less than 900 umol/L, all 25 patients had blood Phe measurements below 600 umol/L. In contrast, only 20% of patients treated with Kuvan in the Kuvan clinical program were considered responders, and the average reduction in blood Phe for these patients was 29%. Average reduction of blood Phe levels is anticipated to be the primary endpoint of the Phase 3 program. Twenty patients in the Phase 2 program had experience with self-administration, some for over 200 days. They were able to effectively dose PEG-PAL at home, maintain efficacy and had a similar safety profile to other patients in the program.

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