"At the conclusion of the Phase 2 study, we have achieved our goal of identifying a dosing regimen that allows us to advance to the pivotal Phase 3 trial," said Hank Fuchs, M.D., Chief Medical Officer of BioMarin. "Based on the Phase 2 experience, we expect that while most patients will experience hypersensitity-type side effects during introduction to the drug, the side effects will be transient and will allow most patients to achieve safe, long-term Phe control. We look forward to meeting with the FDA in the first quarter of 2013 and reviewing the Phase 2 data and our preliminary Phase 3 study design."
Jean-Jacques Bienaimé, Chief Executive Officer of BioMarin added, "We believe PEG-PAL has a strong value proposition in treating adult severe PKU patients, especially in light of the expected ease of self, at-home administration. The initial target market for PEG-PAL is adult PKU patients in our database who are Kuvan non-responders, about 1,500 patients in the U.S. Eventually, we should be able to treat other adult patients and current pediatric patients on Kuvan as they get older."
The preliminary Phase 3 study design includes (1) an open-label study to evaluate safety and blood Phe levels in naive patients and (2) a randomized controlled study in the Phase 2 extension study patients to evaluate blood Phe levels and psychiatric and executive function endpoints. All patients in the Phase 3 trial will be offered the opportunity to remain on drug as part of an extension trial. Based on discussions with the FDA, reduction in Phe levels is expected to be the primary endpoint in the Phase 3 trial. The company expects to initiate the Phase 3 program in the second quarter of 2013 following an end of phase 2 meeting with the FDA in the first quarter of 2013.
Conference Call DetailsBioMarin will host a conference call and webcast today, Wednesday, September 26, 2012 at 5:00 p.m. ET. This event can be accessed on the investor section of the BioMarin website at www.BMRN.com.
|Date: September 26, 2012|
|Time: 5:00 p.m. ET|
|U.S. / Canada Dial-in Number: 877.303.6313|
|International Dial-in Number: 631.813.4734|
|Conference ID: 30936364|
|Replay Dial-in Number: 855.859.2056|
|Replay International Dial-in Number: 404.537.3406|
|Conference ID: 30936364|
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